StemCell Therapy

COPENHAGEN, Denmark, Oct. 13, 2020 /PRNewswire/ -- Novo Seeds, the early stage investment and company creation team of Novo Holdings, today announced an investment in Rappta Therapeutics ("Rappta"), an emerging biotech company focused on developing first-in-class anti-cancer drugs activating protein phosphatase 2A (PP2A). The investment is a part of a EUR 9M series A financing round.

PP2A is a critical enzyme regulating protein de-phosphorylation and a key tumor suppressor which to date has been very difficult to target pharmaceutically. Rappta has developed proprietary tools and a unique understanding of PP2A which allows it to therapeutically reactivate PP2A. As a result of PP2A's central role in the regulation of protein de-phosphorylation, Rappta's PP2A-reactivating technologies offer the potential to develop multiple lead compounds and build a platform for a new class of anti-cancer drugs.

Rappta has assembled a strong scientific, management and commercial team based in Finland and the US. Rappta's scientific team, led by CSO and co-founder, Professor Goutham Narla, Division Chief of Genetic Medicine at the University of Michigan, represents world-leading expertise in PP2A. The scientific team has published seminal papers onthe structural, functional and biological mechanisms of PP2A inactivation in human cancer. The team will be supported by the Scientific Advisory Board lead by Dr. William Hahn, a Professor of Medicine at the Harvard Medical School and the Chief Scientific Officer at the Dana-Farber Cancer Institute.

As a resut of the financing, Jeroen Bakker, Principal at Novo Seeds will join the Board.Other investors in the Series A round include Novartis Venture Fund ("NVF"), Advent Life Sciences ("Advent") and one family office.

Jeroen Bakker, Principal, Novo Seeds, said: "We are impressed by the team's pioneering work in PP2A-reactivating technologies. Novo Seeds' strategy is to back teams from all over the globe with world class science and attract other bluechip investors to help transform these entreprises into successful business in the Nordics. We are very pleased to see renowned investors such as NVF and Advent investing in the region. We look forward to working with them as we support Rappta's world-leading team translate their scientific and medical expertise in phosphatase biology into a clinical oncology biotech."

Mikko Mannerkoski, CEO and co-founder of Rappta Therapeutics, commented: "We are very pleased to attract such a strong syndicate of international investors which validates our approach to developing novel therapies to target the previously undruggable target protein PP2A. This funding will enable us to accelerate the development of our platform and advance the lead compounds towards clinical development."

The investment in Rappta follows recent Novo Seeds participation in Galecto's Series D and Chromologics seed financing rounds.

About Rappta Therapeutics

Rappta Therapeutics, based in Finland and the US, is developing first-in-class anti-cancer drugs activating protein phosphatase 2A (PP2A). It has developed proprietary tools and a unique understanding of PP2A which allows it to therapeutically reactivate PP2A, a critical enzyme regulating protein de-phosphorylation and tumor growth, with the potential to create a new class of anti-cancer drugs. Rappta has a strong scientific, management and commercial team. Its scientific team, led by CSO and co-founder, Professor Goutham Narla, Head of Cancer Research at the University of Michigan, represent world-leading expertise in PP2A. It is backed by blue-chip investors Advent Life Sciences, Novartis Venture Fund, Novo Seeds and one family office. For more information, go to http://www.rappta-therapeutics.com.

About Novo Holdings A/S

Novo Holdings A/S is a private limited liability company wholly owned by the Novo Nordisk Foundation. It is the holding and investment company of the Novo Group, comprising Novo Nordisk A/S and Novozymes A/S, and is responsible for managing the Novo Nordisk Foundation's assets.

Novo Holdings is recognized as a leading international life science investor, with a focus on creating long-term value. As a life science investor, Novo Holdings provides seed and venture capital to development-stage companies and takes significant ownership positions in growth and well-established companies. Novo Holdings also manages a broad portfolio of diversified financial assets. Further information: http://www.novoholdings.dk

SOURCE Novo Holdings

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Novo Seeds co-leads Rappta Therapeutics Series A Financing for the Development of Phosphatase 2A drugs - PRNewswire

People with blood type O may be less vulnerable to COVID-19 and have a reduced likelihood of getting severely ill, according to two studies published Wednesday. Experts say more research is needed.The research provides further evidence that blood type (also known as blood group) may play a role in a person's susceptibility to infection and their chance of having a severe bout of the disease. The reasons for this link aren't clear and more research is needed to say what implications, if any, it has for patients.Studies add to growing evidenceA Danish study found that among 7,422 people who tested positive for COVID-19, only 38.4% were blood type O even though, among a group of 2.2 million people who were not tested, that blood type made up 41.7% of the population.By contrast, 44.4% of group A tested positive, while in the wider Danish population that blood type makes up 42.4%.In the other study, researchers in Canada found that among 95 patients critically ill with COVID-19, a higher proportion with blood type A or AB 84% required mechanical ventilation compared with patients with blood group O or B, which was 61%.The Canadian study also found those with blood type A or AB had a longer stay in the intensive care unit, a median of 13.5 days, compared with those with blood group O or B, who had a median of nine days."As a clinician ... it is at the back of my mind when I look at patients and stratify them. But in terms of a definitive marker we need repeated findings across many jurisdictions that show the same thing," said Dr. Mypinder Sekhon, an intensive care physician at Vancouver General Hospital and an author of the Canadian study."I don't think this supersedes other risk factors of severity like age and co-morbities and so forth," added Sekhon, who is also a clinical assistant professor in the Division of Critical Care Medicine and Department of Medicine at the University of British Columbia."If one is blood group A, you don't need to start panicking. And if you're blood group O, you're not free to go to the pubs and bars."No need to worryMost humans fall into one of four blood groups: A, B, AB or O. In the United States, the most common blood groups are O and A.It makes very little difference to most people's daily lives unless you have to have a blood transfusion. Nor should people worry unduly about the link between blood type and COVID-19, said Dr. Torben Barington, the senior author of the Danish paper and a clinical professor at Odense University Hospital and the University of Southern Denmark."We do not know whether this is some kind of protection of group O, or whether it's some kind of vulnerability in the other blood groups," he said."I think this has scientific interest, and when we find out what the mechanism is, perhaps we're able to use that proactively in some way in regard to treatment."In the Danish study, researchers analyzed data on Danish individuals who were tested between February 27 and July 30, and the distribution of blood types among those people was compared with data from people who had not been tested. They found that blood group wasn't a risk factor for hospitalization or death from COVID-19.Both studies were published in the journal Blood Advances.While there are several theories, researchers don't yet know what mechanism could explain the link between different blood groups and COVID-19.Sekhon said it could be explained by people with blood type O having less of a key clotting factor making them less prone to coagulation problems in the blood. Clotting has been a major driver of the severity of COVID-19.Other possible explanations involve blood group antigens and how they affect the production of infection fighting antibodies. Or it could be linked to genes associated with blood types and their effect on receptors in the immune system."It's a repeated, interesting scientific observation that really warrants further mechanistic work," he said.'Important research question'The findings of the two new studies provide "more converging evidence that blood type may play a role in a person's susceptibility to COVID infection and their chance of having a severe bout of COVID-19," said Dr. Amesh Adalja, senior scholar at the Johns Hopkins University Center for Health Security in Baltimore, who was not involved in either of the studies.A separate study, published in The New England Journal of Medicine in June, found genetic data in some COVID-19 patients and healthy people suggesting that those with Type A blood had a higher risk of becoming infected, and those with type O blood were at a lower risk.That previous genetic study, paired with the two new studies in Blood Advances, are "suggestive that this is a real phenomenon that we're seeing," said Adalja, whose work is focused on emerging infectious disease."While we're not quite to the point where this is ironclad, it's clearly suggestive, and we have not seen anything inconsistent with this. The same pattern has been emerging with O blood type tending to be the one that's standing out," Adalja said.Adalja said that blood types and their susceptibility to various infections have been studied in the medical literature before. For instance, research suggests that people with blood type O appear to be more susceptible to norovirus infection.As for the novel coronavirus that causes COVID-19, "We need to figure out the mechanism and understand it at the molecular level to be able to say for sure how this is occurring that this is really the O blood type and not something that kind of tracks with O blood type," Adalja said."We're starting to see enough now that I think it's an important research question to answer," he said. "There's more science to be done here, but it seems to me that there's more evidence accumulating for this hypothesis."

People with blood type O may be less vulnerable to COVID-19 and have a reduced likelihood of getting severely ill, according to two studies published Wednesday. Experts say more research is needed.

The research provides further evidence that blood type (also known as blood group) may play a role in a person's susceptibility to infection and their chance of having a severe bout of the disease. The reasons for this link aren't clear and more research is needed to say what implications, if any, it has for patients.

A Danish study found that among 7,422 people who tested positive for COVID-19, only 38.4% were blood type O even though, among a group of 2.2 million people who were not tested, that blood type made up 41.7% of the population.

By contrast, 44.4% of group A tested positive, while in the wider Danish population that blood type makes up 42.4%.

In the other study, researchers in Canada found that among 95 patients critically ill with COVID-19, a higher proportion with blood type A or AB 84% required mechanical ventilation compared with patients with blood group O or B, which was 61%.

The Canadian study also found those with blood type A or AB had a longer stay in the intensive care unit, a median of 13.5 days, compared with those with blood group O or B, who had a median of nine days.

"As a clinician ... it is at the back of my mind when I look at patients and stratify them. But in terms of a definitive marker we need repeated findings across many jurisdictions that show the same thing," said Dr. Mypinder Sekhon, an intensive care physician at Vancouver General Hospital and an author of the Canadian study.

"I don't think this supersedes other risk factors of severity like age and co-morbities and so forth," added Sekhon, who is also a clinical assistant professor in the Division of Critical Care Medicine and Department of Medicine at the University of British Columbia.

"If one is blood group A, you don't need to start panicking. And if you're blood group O, you're not free to go to the pubs and bars."

Most humans fall into one of four blood groups: A, B, AB or O. In the United States, the most common blood groups are O and A.

It makes very little difference to most people's daily lives unless you have to have a blood transfusion. Nor should people worry unduly about the link between blood type and COVID-19, said Dr. Torben Barington, the senior author of the Danish paper and a clinical professor at Odense University Hospital and the University of Southern Denmark.

"We do not know whether this is some kind of protection of group O, or whether it's some kind of vulnerability in the other blood groups," he said.

"I think this has scientific interest, and when we find out what the mechanism is, perhaps we're able to use that proactively in some way in regard to treatment."

In the Danish study, researchers analyzed data on Danish individuals who were tested between February 27 and July 30, and the distribution of blood types among those people was compared with data from people who had not been tested. They found that blood group wasn't a risk factor for hospitalization or death from COVID-19.

Both studies were published in the journal Blood Advances.

While there are several theories, researchers don't yet know what mechanism could explain the link between different blood groups and COVID-19.

Sekhon said it could be explained by people with blood type O having less of a key clotting factor making them less prone to coagulation problems in the blood. Clotting has been a major driver of the severity of COVID-19.

Other possible explanations involve blood group antigens and how they affect the production of infection fighting antibodies. Or it could be linked to genes associated with blood types and their effect on receptors in the immune system.

"It's a repeated, interesting scientific observation that really warrants further mechanistic work," he said.

The findings of the two new studies provide "more converging evidence that blood type may play a role in a person's susceptibility to COVID infection and their chance of having a severe bout of COVID-19," said Dr. Amesh Adalja, senior scholar at the Johns Hopkins University Center for Health Security in Baltimore, who was not involved in either of the studies.

A separate study, published in The New England Journal of Medicine in June, found genetic data in some COVID-19 patients and healthy people suggesting that those with Type A blood had a higher risk of becoming infected, and those with type O blood were at a lower risk.

That previous genetic study, paired with the two new studies in Blood Advances, are "suggestive that this is a real phenomenon that we're seeing," said Adalja, whose work is focused on emerging infectious disease.

"While we're not quite to the point where this is ironclad, it's clearly suggestive, and we have not seen anything inconsistent with this. The same pattern has been emerging with O blood type tending to be the one that's standing out," Adalja said.

Adalja said that blood types and their susceptibility to various infections have been studied in the medical literature before. For instance, research suggests that people with blood type O appear to be more susceptible to norovirus infection.

As for the novel coronavirus that causes COVID-19, "We need to figure out the mechanism and understand it at the molecular level to be able to say for sure how this is occurring that this is really the O blood type and not something that kind of tracks with O blood type," Adalja said.

"We're starting to see enough now that I think it's an important research question to answer," he said. "There's more science to be done here, but it seems to me that there's more evidence accumulating for this hypothesis."

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People with blood type O may have lower risk of COVID-19 infection and severe illness, studies suggest - WCVB Boston

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Phenylketonuria (PKU) is a congenital disease in which patients lack an enzyme that breaks down the amino acid Phenylalanine (Phe). Untreated, PKU leads to intellectual disability, seizures and movement disorders.

Today, babies are typically diagnosed with PKU 3-5 days after birth, from a heel prick blood test. And the primary treatment sounds simple avoid foods that contain Phe. But thats easier said than done.

One of the problems with PKU, even though newborn screening and control of diet of young kids is good, the diet is bland and not too interesting. When kids become teens we know they want to be able to go out and have a meal with friends and they struggle to keep symptoms in check, said Dr. Rob Nicholls, professor of pediatrics and director of the Birth Defects Laboratories at the University of Pittsburgh School of Medicine.

Since Phe is an amino acid, its present in any food that contains a lot of protein steak, fish, tofu, beans, etc. so people with PKU end up purchasing Phe-free food to get the protein they need to grow and stay healthy.

But, besides being bland, this special food can also be quite expensive. Families often need help paying for it, since its not covered by health insurance.

There are a few gene therapies in the pipeline, as well as a drug that works for some, as well as enzyme substitution therapy that involves frequent painful injections, Nicholls said, but most people still end up with a prescription for a special diet.

A major roadblock for developing better treatments is the lack of a good animal model for PKU research.

Genetically engineered laboratory rodents the workhorse of preclinical research dont display PKU symptoms, and researchers have yet to establish another animal that does the job.

A PKU pig and its unaffected littermate at 2 months of age.

So, Nicholls and colleagues developed a pig model of PKU, which they describe in a paper published today in JCI Insight.

According to study coauthor Dr. Jerry Vockley, chief of medical genetics at UPMC Childrens Hospital of Pittsburgh, who sees many PKU patients and their families each year in the clinic, this new pig model of PKU will allow for the advancement of better treatment options beyond a difficult-to-sustain diet.

Development of this model of PKU for the first time allows us the opportunity to develop improved therapies and test them in a meaningful way, opening the door to a better life for patients, Vockley said.

To create a pig model of PKU, the researchers crossed a Yucatan mini pig which grows to about the size of a small human with a domestic pig and used CRISPR gene editing to cut out part of the gene for phenylalanine hydroxylase (PAH), the enzyme that breaks down Phe.

The animals were born at the University of Missouri, in collaboration with Dr. Randall Prather, Director of the National Swine Resource and Research Center.

During infancy, the PKU model pig, who was born with two copies of the edited PAH gene, was much smaller than a littermate with only one copy of the edited gene. There were also clear brain differences.

Blood Phe levels were much higher in the PKU pig than either PKU patients off their diet or mice engineered to lack the PAH gene. A completely Phe-free diet brought the animals levels down to normal, only to spike again with a diet of 50% Phe-free chow.

To verify that the gene editing protocol didnt accidentally clip off any adjacent genes, lead author Dr. Erik Koppes, a postdoctoral fellow in Nichollss lab, sequenced the genome of the PKU pig and verified that the edited segment was within the bounds of the gene known to cause PKU.

Koppes also looked for off-target mutations across the genome a common concern with CRISPR but didnt find any.

It took quite a bit of genetic detective work, Nicholls said.

Next, Nicholls plans to begin breeding PKU pigs for future studies. He hopes to one day move the animals to a nearby farm, where the operation can scale up at a lower cost, with the ultimate goal of making the pig the preferred model of preclinical PKU research.

This research was funded by the National PKU Alliance (NPKUA) and an IGNITE grant from the National Institute of Neurological Disorders and Stroke.

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For PKU Research, Pigs Could Be the New Stand-In for People - UPMC

CAMBRIDGE, Mass. & SAN FRANCISCO--(BUSINESS WIRE)--Gemini Therapeutics, a clinical stage precision medicine company developing innovative treatments for genetically defined age-related macular degeneration (AMD), and FS Development Corp. (Nasdaq: FSDC), a special purpose acquisition company sponsored by Foresite Capital, today announced they have entered into a definitive merger agreement. Upon closing of the transaction, the company will be renamed Gemini Therapeutics, Inc. (Combined Company) and will be led by Jason Meyenburg, Chief Executive Officer of Gemini. The Combined Companys common stock is expected to be listed on Nasdaq.

In addition to the approximately $121 million held in FS Development Corp.s trust account (assuming no redemptions are effected), a group of premier healthcare investors has committed to participate in the transaction through a common stock PIPE of approximately $95 million at $10.00 per share. Investors in the PIPE include lead investor Foresite Capital, an affiliate of FS Development Corp.s sponsor, as well as Fidelity Management & Research Company LLC, Wellington Management, Boxer Capital of Tavistock Group, Alyeska Investment Group, L.P., Suvretta Capital Management, CVF, DAFNA Capital, and Acorn Bioventures, in addition to existing Gemini Therapeutics shareholders including Orbimed Healthcare Fund Management, Atlas Venture, Lightstone Ventures and Wu Capital.

This mornings announcement is important for the advancement of AMD research, as it ensures we have the necessary capital to advance our clinical programs and continue applying our insights in genetics and biology to pioneer first-in-class medicines to restore regulation of the complement system in the eye and throughout the body, bringing forward targeted precision therapies based on genetically defined populations, said Mr. Meyenburg. I would like to thank all those involved in making this transaction a success, particularly our new and existing blue chip investors, and the entire Gemini team.

Gemini embodies the type of company we had in mind when forming FSDC: a platform focused on the next generation of medicines utilizing genetics, said Jim Tananbaum, M.D., Chief Executive Officer of Foresite Capital and President and Chief Executive Officer of FS Development Corp. Gemini is developing treatments for patients losing their vision because of genetically driven macular degeneration. We are excited about the tremendous potential of this transaction, which we believe creates value for investors along with the potential to bring innovative new treatment options to patients.

Proceeds from the transaction are expected to provide Gemini with the capital needed to further develop its clinical programs and preclinical portfolio, including the following programs:

Post-closing of the transaction, Mr. Meyenburg and Dr. Tananbaum will be joined by board members from Gemini to form the seven-person board of directors.

Summary of Transaction

Current Gemini shareholders are converting 100% of their existing equity interests into common stock of the Combined Company. In addition to the approximately $121 million held in FSDCs trust account (assuming no redemptions are effected), an additional group of premier healthcare investors has committed to participate in the transaction through a common stock PIPE of approximately $95 million at $10 per share.

The Combined Company is expected to receive gross proceeds of approximately $216 million at the closing of the transaction (assuming no redemptions are effected), which is expected by January 2021. The close of this transaction is subject to approval of FSDCs shareholders and the satisfaction or waiver of certain other customary closing conditions.

Jefferies LLC and SVB Leerink acted as co-lead private placement agents for FS Development Corp. Jefferies LLC also acted as lead financial and capital markets advisor to FS Development Corp. Goldman Sachs & Co. LLC acted as lead financial advisor to Gemini in the transaction. Stifel acted as additional capital markets advisor to Gemini. Goodwin Procter LLP acted as legal counsel to Gemini. White & Case LLP acted as legal counsel to FS Development Corp.

The description of the business combination contained herein is only a high-level summary. Additional information about the transaction will be provided in a Current Report on Form 8-K that will contain an investor presentation to be filed by FS Development Corp. with the Securities and Exchange Commission (SEC) and will be available at http://www.sec.gov. In addition, FS Development Corp. intends to file a registration statement on Form S-4 with the SEC, which will include a proxy statement/prospectus, and will file other documents regarding the proposed transaction with the SEC.

In connection with the proposed business combination, FS Development Corp. intends to file a Registration Statement on Form S-4, including a preliminary proxy statement/prospectus and a definitive proxy statement/prospectus with the SEC. FS Development Corp.s stockholders and other interested persons are advised to read, when available, the preliminary proxy statement/prospectus and the amendments thereto and the definitive proxy statement/prospectus and documents incorporated by reference therein filed in connection with the proposed business combination, as these materials will contain important information about Gemini, FS Development Corp., and the proposed merger. When available, the definitive proxy statement/prospectus and other relevant materials for the proposed merger will be mailed to stockholders of FS Development Corp. as of a record date to be established for voting on the proposed business combination. Stockholders will also be able to obtain copies of the preliminary proxy statement/prospectus, the definitive proxy statement/prospectus, and other documents filed with the SEC that will be incorporated by reference therein, without charge, once available, at the SECs website at http://www.sec.gov, or by directing a request to press@foresitecapital.com.

Conference Call Information

Gemini and FS Development Corp. will host a conference call today, Thursday, October 15, 2020, at 10:30 a.m. Eastern Time, to discuss the proposed transaction. To access the conference call, please dial (888) 317-6003 (local) or (412) 317-6061 (international) at least 10 minutes prior to the start time and reference conference ID: 4983831.

About Gemini Therapeutics

Gemini Therapeutics is a clinical stage precision medicine company developing innovative treatments for age-related macular degeneration (AMD) by developing drugging strategies that are matched to specific genetic mutations found in patients with high clinical unmet need. Geminis lead clinical stage candidate, GEM103, is a recombinant form of the naturally occurring complement factor H protein currently in a Phase 2a trial in dry AMD patients with a complement factor H mutation. The company has generated a rich pipeline including recombinant proteins, gene therapies, and monoclonal antibodies. Geminis CLARITY natural history study is designed to provide unprecedented insight into the role of genetic risk in common retinal diseases and began in December 2018. Gemini was launched with funding from leading life science investors and powered by academic partnerships globally.

For more information, visit http://www.geminitherapeutics.com.

About FS Development Corp. (FSDC)

FS Development Corp., sponsored by Foresite Capital, is a blank check company formed for the purpose of effecting a business combination with one or more businesses in the biotechnology sector. The company is led by Jim Tananbaum, M.D., the CEO of Foresite Capital, an investment firm funding visionary healthcare entrepreneurs with approximately $3 billion in assets under management. The firm is headquartered in San Francisco.

Important Information About the Merger and Where to Find It

A full description of the terms of the business combination will be provided in a registration statement on Form S-4 to be filed with the SEC by FS Development Corp. that will include a prospectus with respect to the Combined Companys securities to be issued in connection with the business combination and a proxy statement with respect to the shareholder meeting of FS Development Corp. to vote on the business combination. FS Development Corp. urges its investors, shareholders and other interested persons to read, when available, the preliminary proxy statement/ prospectus as well as other documents filed with the SEC because these documents will contain important information about FS Development Corp., Gemini and the business combination. After the registration statement is declared effective, the definitive proxy statement/prospectus to be included in the registration statement will be mailed to shareholders of FS Development Corp. as of a record date to be established for voting on the proposed business combination. Once available, shareholders will also be able to obtain a copy of the S-4, including the proxy statement/prospectus, and other documents filed with the SEC without charge, by directing a request to: FS Development Corp., Attn: Secretary, 600 Montgomery Street, Suite 4500, San Francisco, California 94111. The preliminary and definitive proxy statement/prospectus to be included in the registration statement, once available, can also be obtained, without charge, at the SECs website (www.sec.gov).

Participants in the Solicitation

FS Development Corp. and Gemini Therapeutics and their respective directors and executive officers may be considered participants in the solicitation of proxies with respect to the proposed business combination described in this press release under the rules of the SEC. Information about the directors and executive officers of FS Development Corp. is set forth in FS Development Corp.s final prospectus filed with the SEC pursuant to Rule 424(b) of the Securities Act of 1933, as amended (the Securities Act) on August 13, 2020, and is available free of charge at the SECs website at http://www.sec.gov or by directing a request to: FS Development Corp., Attn: Secretary, 600 Montgomery Street, Suite 4500, San Francisco, California 94111. Information regarding the persons who may, under the rules of the SEC, be deemed participants in the solicitation of the FS Development Corp. shareholders in connection with the proposed business combination will be set forth in the registration statement containing the proxy statement/prospectus for the proposed business combination when it is filed with the SEC. These documents can be obtained free of charge from the sources indicated above.

Forward-Looking Statements

This press release contains forward-looking statements that are based on beliefs and assumptions and on information currently available. In some cases, you can identify forward-looking statements by the following words: may, will, could, would, should, expect, intend, plan, anticipate, believe, estimate, predict, project, potential, continue, ongoing or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we caution you that these statements are based on a combination of facts and factors currently known by us and our projections of the future, about which we cannot be certain. Forward-looking statements in this press release include, but are not limited to, statements regarding the proposed business combination, including the timing and structure of the business combination, the proceeds of the business combination, the initial market capitalization of the Combined Company and the benefits of the business combination, as well as statements about the potential attributes and benefits of Geminis product candidates and the format and timing of Geminis product development activities and clinical trials. We cannot assure you that the forward-looking statements in this press release will prove to be accurate. These forward-looking statements are subject to a number of significant risks and uncertainties that could cause actual results to differ materially from expected results, including, among others, the ability to complete the business combination due to the failure to obtain approval from FS Development Corp.s shareholders or satisfy other closing conditions in the Merger Agreement, the occurrence of any event that could give rise to the termination of the Merger Agreement, the ability to recognize the anticipated benefits of the business combination, the outcome of any legal proceedings that may be instituted against FS Development Corp. or Gemini following announcement of the proposed business combination and related transactions, the impact of COVID-19 on Geminis business and/or the ability of the parties to complete the business combination, the ability to obtain or maintain the listing of FS Development Corp.s common stock on Nasdaq following the proposed business combination, costs related to the proposed business combination, changes in applicable laws or regulations, the possibility that FS Development Corp. or Gemini may be adversely affected by other economic, business, and/or competitive factors, and other risks and uncertainties, including those to be included under the header Risk Factors in the registration statement on Form S-4 to be filed by FS Development Corp. with the SEC and those included under the header Risk Factors in the final prospectus of FS Development Corp. related to its initial public offering. Most of these factors are outside of FS Development Corp.s and Geminis control and are difficult to predict. Furthermore, if the forward-looking statements prove to be inaccurate, the inaccuracy may be material. In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified time frame, or at all. The forward-looking statements in this press release represent our views as of the date of this press release. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we have no current intention of doing so except to the extent required by applicable law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release.

Non-Solicitation

This press release is not a proxy statement or solicitation of a proxy, consent or authorization with respect to any securities or in respect of the proposed business combination and shall not constitute an offer to sell or a solicitation of an offer to buy any securities nor shall there be any sale of securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. No offer of securities shall be made except by means of a prospectus meeting the requirements of the Securities Act.

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Gemini Therapeutics and FS Development Corp. Announce Merger Agreement Creating Publicly Listed Precision Medicine Company Focused on Age-Related...

A new study by University of California researchers shows the promise of high-throughput DNA-sequencing technologies to improve prenatal diagnosis and pregnancy outcomes for women who have experienced an abnormal prenatal ultrasound.

In the UC San Francisco-led study, scientists used a technique called exome sequencing to identify genetic diseases as the underlying cause in 37 of 127 cases of nonimmune hydrops fetalis (NIHF), a life-threatening condition in which the fetus is overloaded with fluid. The study was published online Oct. 7 in The New England Journal of Medicine (NEJM).

Corresponding author Teresa Sparks, MD, MAS, a UCSF assistant professor in the Department of Obstetrics, Gynecology & Reproductive Sciences, led the study with senior study author Mary Norton, MD, a professor in the same department. The cause of most cases of NIHF is not identified with standard testing, but when we apply exome sequencing, we find a genetic diagnosis in nearly 30 percent of cases of previously unknown cause, Sparks said.

NIHF affects about one in every 1,700 to 3,000 pregnancies in the United States and is associated with high risks of stillbirth, preterm birth, neonatal death and other complications. Although NIHF often leads to death, identifying the precise genetic cause is critical, as associated outcomes vary widely in severity.

NIHF can be a manifestation of many genetic diseases, but evidence of abnormal fluid accumulation in the fetus detected through an ultrasound exam whether it occurs under the skin, in the abdomen, or around the heart or lungs does not pinpoint an underlying cause.

Participants in the study were referred from throughout the United States after NIHF was identified with prenatal ultrasound but no underlying genetic disease was found using long established methods for detecting genetic abnormalities. These traditional genetic tests karyotype and chromosomal microarray analysis detect large abnormalities in chromosomes, not disorders caused by a defect in a single gene as are identified with exome sequencing.

Exome sequencing is the complete spelling out of the genetic code for DNA segments within the genome that serves as the blueprints for proteins. This has become possible to perform quickly and accurately in recent years, thanks to the continual refinement of technology that can sequence DNA strands that are thousands of nucleotide building blocks long, often in a massively parallel manner that helps ensure accurate results. Exome sequencing can identify even the smallest mutations, such as a change in a single building-block nucleotide base pair.

Importantly, many of the disorders identified in the study have not previously been reported in association with NIHF, so the findings broaden knowledge of genetic diseases that can present with the condition. Among the most common of 37 genetic disorders identified in the NEJM study were 11 cases affecting a key intracellular signaling pathway called RAS-MAPK, four cases of inborn errors of metabolism, four cases of musculoskeletal disorders, and three cases each of lymphatic, neurodevelopmental, cardiovascular and blood disorders. Many of these diagnoses would also have been missed by commercial gene panels, Sparks said.

Most mutations identified in the study newly arose in the fetus, but several were inherited, with the potential to affect future pregnancies with the same biologic mother or father.

There is a very wide range in genetic diagnoses underlying NIHF, and identifying the diagnosis is essential for families and healthcare providers, Sparks said. With advanced genetic testing, there is much more we can discover for families to help them understand the situation, for obstetricians and neonatologists to better take care of the pregnancy and anticipate the needs of the newborn, and ultimately to guide the development of novel prenatal management strategies such as in-utero therapies to improve health outcomes over the long term.

For some of the genetic disorders identified in the study, prenatal interventions that can improve or save lives already have been identified. For example, genetic causes of anemia in the fetus may be closely monitored, and the fetus may receive a blood transfusion if needed.

Similarly, for some of the inborn errors of metabolism identified in the study, enzyme therapies already are available after birth. Early diagnosis and treatment of these metabolic disorders leads to better outcomes. A co-author of the NEJM study, Tippi MacKenzie, MD, a professor with the UCSF Department of Surgery, is investigating in utero treatments for specific genetic disorders underlying NIHF in a new clinical trial. Sparks, Norton, and co-authors are also pursuing further investigations to identify additional genomic abnormalities underlying NIHF for the cases that remain unsolved.

Co-Authors: All co-authors of the NEJM study are affiliated with the University of California FetalMaternal Consortium or the UCSF Center for Maternal-Fetal Precision Medicine. Additional UCSF co-authors of the study include Billie Lianoglou, Sarah Downum, Sachi Patel, Amanda Faubel, Anne Slavotinek, Patrick Devine, Ugur Hodoglugil, Jessica Van Ziffle, and Stephan Sanders.

Funding: The study was funded by the UCSF Center for Maternal-Fetal Precision Medicine, the Fetal Health Foundation, the Brianna Marie Foundation, Ultragenyx, and the National Institutes of Health.

The University of California, San Francisco (UCSF) is exclusively focused on the health sciences and is dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care.UCSF Health, which serves as UCSFs primary academic medical center, includes top-ranked specialty hospitals and other clinical programs, and has affiliations throughout the Bay Area.

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DNA Test Identifies Genetic Causes of Severe Fetal and Newborn Illness - UCSF News Services

Olaparib (Lynparza) significantly improved overall survival (OS) versus enzalutamide (Xtandi) or abiraterone acetate (Zytiga) in patients with metastatic castration-resistant prostate cancer (mCRPC) who harbor BRCA1, BRCA2, and/or ATM aberrations, according to results from the final OS analysis of the pivotal phase 3 PROfound trial (NCT02987543).1

In the trial, patients with mCRPC who progressed on previous treatment with a new hormonal agent and harbored BRCA1, BRCA2, or ATM aberrations (n = 245; cohort A) or other alterations in the homologous recombination repair (HRR) pathway (n = 142; cohort B) were randomized 2:1 to receive either olaparib or enzalutamide or abiraterone acetate.

Final OS data from cohorts A and B were presented during the 2020 ESMO Virtual Scientific Program. Results showed that the median OS in cohort A was significantly longer with olaparib than with physicians choice (HR 0.69; 95% CI 0.50-0.97; P = .0175).1In cohort B, the median OS was 14.1 months with olaparib versus 11.5 months with the control (HR, 0.96; 95% CI, 0.63-1.49).

What is exciting about this particular trial is that it showed the feasibility of personalizing care and using precision medicine strategies to preselect patients to maximize the chance of benefit for those who are candidates for these treatments, said Maha H.A. Hussain, MD, FACP, FASCO.We can also help patients avoid unnecessary exposure to ineffective treatments.

Previously published data showed that olaparib resulted in a 66% reduction in the risk of disease progression or death compared with abiraterone or enzalutamide (HR, 0.34; 95% CI, 0.25-0.47;P<.0001).2 Based on these results, the FDA approved olaparib in May 2020 for the treatment of adult patients with deleterious or suspected deleterious germline or somatic HRR genemutated mCRPC who have progressed following prior treatment with enzalutamide or abiraterone.

In an interview with OncLive, Hussain, the Genevieve E. Teuton Professor of Medicine in the Department of Medicine of the Division of Hematology Oncology and the deputy director at the Robert H. Lurie Comprehensive Cancer Center of the Northwestern University Feinberg School of Medicine, further discussed the updated findings from the PROfound trial, its clinical significance in the treatment of patients with mCRPC paradigm, and the promise of precision medicine.

Hussain: PROfound is a randomized phase 3 clinical trial. It is one of the first precision medicine clinical trials to complete; patients were preselected based on specific genomic alterations and then randomized accordingly. Patients with mutations in the HRR genes or DNA damage repair genes were assigned to 2 different cohorts. The primary cohort was [comprised of] patients who had BRCA1/2 or ATM mutations, while cohort 2 included [those who harbored] other genes that are involved in the HRR pathway. Patients were randomized 2:1 to olaparib or standard of care per physicians choice of either abiraterone and prednisone or enzalutamide. The primary end point [of the trial] was radiographic progression-free survival (rPFS), which is a meaningful clinical end point, while OS was one of the several key secondary end points [examined].

Data from the Stand Up to Cancer highlighted the fact that over 20% of patients with mCRPC have significant mutations in the DNA repair pathway or the HRR genes. That [research] underscored the fact that this is a clinically relevant pathway to go after. At the time that [the PROfound trial was being designed] we saw evidence of benefit [with this approach] in other tumors [such as] breast and ovarian cancers, and then subsequently, in pancreatic cancer.

The specific pathway relevance is that both normal cells and cancer cells need to repair themselves when there is damage; the HRR pathway is involved in that repair process. However, are alterations or mutations [are present], the cells are not able to repair themselves and they fall back into a different pathway, which is the PARP pathway. Basically, PARP agents tend to inhibit that enzyme so that the [cancer] cells cannot repair themselves.

[Earlier data from the trial were previously published] this past summer. Johann de Bono, MB, ChB, PhD, of The Institute of Cancer Research was the first author on the publication in the New England Journal of Medicine, which highlighted [data regarding] the primary end point of rPFS. In this particular presentation delivered at the 2020 ESMO Virtual Congress, [investigators] reported OS [data from] cohorts A and B.

We saw that the benefit [with olaparib is] not only in terms of rPFS; the benefit translated into a median OS benefit of over 4 months between the arms, despite crossover from the control arm to the olaparib arm at time of progression. Additionally, the risk of death was reduced by 31%, which is very clinically significant. In [the cohort of patients who harbored the] other 12 genes, other than BRCA1/2 and ATM, we saw a trend in OS improvement but it was not statistically significant. The trend was about a little bit over 2 months of a difference. When adjusting for crossover, the trend improved although it was still not statistically significant. However, several patients in cohort B experienced clinical benefits from treatment. The primary benefit [with olaparib] still seems to be driven by BRCA primarily.

No; the overall safety was very much consistent with what was known about olaparib. The most common adverse effects observed included anemia, nausea, and fatigue. Most of these were low-grade events, aside from the anemia. Many of these patients were heavily pretreated; while they might have previously received abiraterone or enzalutamide, they would have also received chemotherapy and other potential anticancer treatment and be fairly advanced in the course of their disease. The findings, overall, are really not surprising and the safety profile very much consistent with what has been observed with the agent in other tumors.

We have reached a major benchmark in the management of this disease. Ever since the original observations regarding androgen deprivation [therapy] in prostate cancer and subsequent treatments, and certainly since the time I entered the field in the early 1990s, prostate cancer management has been more of a one-size-fits-all approach. In fact, when we give chemotherapy and hormone treatment we don't preselect [patients].

We still have [a lot of work to do]. Patients with metastatic castration-resistant disease continue to die from prostate cancer; they also suffer from pain and other factors involved with this disease. This [research] highlights the feasibility of performing precision medicine trials. It also shows us that meaningful clinical benefits could be achieved in these patients. I would hope that our partners across the spectrum will invest further in conducting more clinical trials.

The observation that we've seen with olaparib also opens up the door for potential combination clinical trials, both in castration-resistant disease and potentially in earlier stages of disease, where we might get a better return on investment from a clinical perspective.

Genomic profile evaluation for patients is critical moving forward, not only for the purpose of treatment for the patient. Conducting or counseling the patient regarding germline testing and tumor genomics evaluation in preparation for future treatment is also very critical. Obviously, genetic testing is associated with genetic counseling, [which may allow patients] and potential blood relatives [to get ahead of the game].

Tissue-based genomic evaluation will open the door for the patient to explore different treatments, and [certain] genomic alterations might qualify them for different clinical trials opportunities. [This work] underscores the hope for patients that their cancer can be managed better with genomically targeted treatments, specifically, in this case, the PARP inhibitor. [Now we can build on] these observations in terms of different treatment strategies and combinations.

Excerpt from:
PROfound Trial With Olaparib Shows Feasibility of Personalizing Care in mCRPC - OncLive

Fifteen weeks into her second pregnancy, Katrina Villegas and her husband learned their baby girl had trisomy 13, a chromosomal disorder that causes severe disabilities and is usually fatal.

"Its really just a whirlwind situation for a few weeks," said Villegas, recalling how her baby's heart defects and other abnormalities began showing up on her ultrasounds. "We just tried to do as much research as we could about trisomy 13 and what that would mean for our daughter. At the end of the day we decided that we didnt want her suffering, so we ended up inducing the pregnancy early."

Villegas, who contributes to the TODAY Parenting Team, recalls feeling blindsided when she had to sign paperwork for a "medical abortion" prior to her induction.

"I lost it because it wasnt an abortion in my head," said Villegas. "This was a child that we wanted. We were making one of the hardest decisions you can make as a parent, which was to spare her pain."

In the face of fetal abnormalities that will cause death or severe suffering, it's not uncommon for a expectant mother to choose a termination for medical reasons (TFMR). Though it's not an official medical term, online loss support groups and parents who have been through it coined the term to describe their unique experience. The American College of Obstetricians and Gynecologists has released an official statement explaining that in some cases, abortion is a medical necessity.

Still, parents who choose TFMR often feel weighed down by feelings of guilt or secrecy, unsure how to grieve the loss and caught between identifying with having had a miscarriage or an abortion. Some don't tell friends and family for fear of judgment. Some feel unwelcome in pregnancy loss support groups.

On August 8, 2017, Villegas gave birth to her daughter, April Rey Villegas, who lived for 11 minutes before passing away in her mom's arms.

"She held my hand pretty much the entire time," the Germantown, Maryland, mom recalled. "Everybody in the family that wanted to come meet April got to do so and have a few special moments with her."

Villegas, who blogs about her loss at Terminations Remembered, says making the decision to end a pregnancy for medical reasons was isolating.

"When you think about coping with grief, one of the things people need to do is be able to process and talk, especially to the people they love and care about," said Villegas. "But often in this situation, parents dont tell the whole story and they dont talk about it openly even to people in their close circle because of the fear of judgment."

Wilmaris Soto-Ramos was 16 weeks into her first pregnancy when she received devastating news at an ultrasound appointment.

"Doctors came into the room and told me the extremities of our baby were really abnormal," Soto-Ramos told TODAY Parents. "Her feet were clubbed, her arms werent moving and she also had fluid in her brain."

Soto-Ramos and her partner were referred to a genetic counselor, who performed an amniocentesis, a procedure used to test for genetic abnormalities.

"In that amniocentesis they kind of found the same results," Soto-Ramos, who lives in Pawtucket, Rhode Island, shared. "The doctor looked at me and said, 'Most parents would terminate a pregnancy like this and you should, too,' which was really heartbreaking."

As she considered how to proceed with her own pregnancy, Soto-Ramos went on a quest for answers.

For the next five weeks Soto-Ramos visited specialists for more tests, until finally learning her daughter had a severe case of arthrogryposis, a rare genetic condition that affects the joints and muscles.

"Because it also affected her brain, she really wasnt viable," explained Soto-Ramos. "If she was to live, she would be wheelchair bound. She wouldnt be able to speak, talk, hear or eat. They also told me if I continued the pregnancy she would probably pass away regardless because of the fluid in her brain."

Soto-Ramos made the decision to end her pregnancy, allowing doctors to induce labor at 22 weeks gestation. On May 9, 2019, her daughter, Angelis Yawa Larbi, was delivered.

"She passed away in the birth canal as she was coming out," Soto-Ramos recalled. "Its something I really struggled with because I'm somebody who is pro-choice and, for me, this was a very-much-wanted pregnancy."

Dr. Christine Greves, an OB-GYN in Orlando, Florida, says it's the job of doctors to support, not judge, women who are faced with this devastating scenario.

"If a patient of mine was to come and talk to me about it, I would tell her there are different options and encourage her to find out more information," said Greves, who does not perform TFMR and typically refers patients to a maternal-fetal medicine specialist when abnormalities are detected in a pregnancy. "More knowledge can be helpful from the standpoint of providing peace with whatever decision they choose."

She said a doctor may suggest termination for medical reasons to a pregnant woman for two reasons: when a fetus is considered not viable due to a medical condition detected in utero or when a mother's life is at risk if she continues a pregnancy.

"When people get this sad news, their doctor tells them the possible outcome of carrying the pregnancy to term," explained Greves. "They explain what could happen either fully delivering the baby and then having the baby pass away, or as far as the maternal indication goes, making sure the patient is aware they could die if they go through with the pregnancy."

At the time of her daughter April's birth, Villegas' older daughter, Caroline, was two. Caroline asked to meet her baby sister after she was born, and has continued asking difficult questions about grief and loss in the years since her death. It's these conversations, combined with a desire to tell her 1-year-old son, William, about the sister that died before his birth, that led Villegas to write five children's books dealing with TFMR.

With titles like "Our Baby is Going to Die," and "The Baby Before You Died," the books explain both surgical termination and induction, and tackle difficult topics like expressing grief and remembering the baby who was lost.

Soto-Ramos says she's experienced isolation, even feeling excluded from some infant loss support groups because she chose to terminate her pregnancy. A licensed clinical social worker, Soto-Ramos is in the process of becoming a bereavement doula and hopes to help women cope with their grief and get the answers they need about their options.

"I found, especially for me being a woman of color, it was really difficult to get answers within the medical system and I had to go look for them, which was traumatizing on its own," said Soto-Ramos. "But Im glad I did because had I listened to the first doctor who told me to terminate my pregnancy without knowing what was going on, I would have always wondered, 'What if?'"

"I love my daughter so much and so deeply and know my decision was out of love, not out of fear about something a doctor told me," Soto-Ramos added. "It was because I loved her and knew what was going on."

Soto-Ramos kept trying to have a child, and she was pregnant when she talked to TODAY Parents for this story. On October 4, she gave birth to a healthy baby girl.

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Termination for medical reasons: Moms share TFMR stories - TODAY

Children with chronic kidney disease spent about 30% longer in the hospital (an average of 2.8 days compared to 1.8 days for those without a chronic kidney disease) with nearly 60% more in hospital expenses ($8,755 per hospitalization compared to $5,016.)

Children with chronic kidney disease were also 50% more likely to die during hospitalization.

Data on in-hospital mortality for children with chronic illnesses is lacking, but we know that hospitalizations with a chronic kidney disease diagnosis have a higher mortality than those with other chronic condition diagnoses with the exception of heart failure, Modi says.

The fact that these children are potentially at higher risk of death while hospitalized should prompt providers to closely evaluate management strategies.

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That may mean bringing nephrologists in earlier if they are not already involved in patients care, making sure to avoid medications that could make kidney function worse as well as other steps that will improve care for these patients, Modi notes.

The high health care expenses for hospitalized pediatric patients with end-stage kidney disease, including dialysis, transplantation, and associated complications may be comparable to hospitalized heart failure patients, authors say.

Kidney disease may be associated with more medical complexities, authors say. The causes of chronic kidney disease in children include genetic disorders, congenital anomalies that may be part of a multi-organ system syndrome and systemic inflammatory disorders. A recent study from the UK reported that adult kidney disease patients also have a greater degree of medical complexity than patients seen by any other specialty.

Chronic kidney disease can be a devastating illness with many long-term consequences, Modi says. Some features of chronic kidney disease that start during childhood will have a significant impact on patients lives through adulthood.

We need further studies to better understand the health care needs and delivery of care to hospitalized children with chronic kidney disease in order to optimize health outcomes."

Excerpt from:
Children with Chronic Kidney Disease Have Outsized Health Burden - Michigan Medicine

For a honey bee, few things are more important than recognizing your nestmates. Being able to tell a nestmate from an invader could mean the difference between a honey-stocked hive and a long, lean winter.

New research from Washington University in St. Louis shows that honey bees rely on chemical cues related to their shared gut microbial communities, instead of genetic relatedness, to identify members of their colony.

Most people only pay attention to the genetics of the actual bee, said Yehuda Ben-Shahar, professor of biology in Arts & Sciences and corresponding author of the study published Oct. 14 in Science Advances. What we show is that it is genetic, but its the genetics of the bacteria.

Honey bees recognize and respond to chemical signals from other bees that they detect from skin compounds known as cuticular hydrocarbons, or CHCs. This study determined that a bees particular CHC profile is dependent on its microbiome the bacteria that make up its gut microbial community and is not something innate or genetic to the bee alone.

Different colonies do in fact have colony-specific microbiomes, which has never been shown before, said Cassondra L. Vernier, postdoctoral associate at the University of Illinois, who earned her biology PhD working with Ben-Shahar at Washington University.

Bees are constantly sharing food with one another and exchanging this microbiome just within their colony, said Vernier, first author of the new study.

Co-authors include Gautam Dantas, professor of pathology and immunology and of molecular microbiology at Washington University School of Medicine in St. Louis, and Joel Levine at the University of Toronto Mississauga. The work was conducted in part with bees housed at Tyson Research Center, the environmental field station for Washington University.

The importance of this paper is that its one of the first papers that actually shows that the microbiome is involved in the basic social biology of honey bees and not just affecting their health, Vernier said. The microbiome is involved in how the colony as a whole functions, and how they are able to maintain nest defenses, rather than just immune defense within an individual.

The gut microbial community or microbiome supplies humans and other animals with vitamins, helps digest food, regulates inflammation and keeps disease-causing microbes in check. Increasingly a topic of research interest, scientists have discovered many ways that the microbiome blurs the borders between a host and its bacteria.

The microbiome has been found to influence communication in several different organisms including, notably, large animals like hyenas.

For honey bees, this study shows that the microbiome plays a critical role in defining the tightly regulated chemical signals for group membership.

Until recently, most scientists thought that honey bees identified nestmates by picking up on a homogenized scent that they recognize from members of their own colony a kind of hive B.O., Ben-Shahar joked.

Bee colonies are usually composed of highly related individuals. But the chemical signals that allow bees to recognize each other are not determined by genetics alone. Researchers know this because baby bees can be placed into other colonies without being rejected up until a certain age and level of development.

It has to be something that they acquire during their lifetime that defines their nestmate recognition cues, Vernier said.

In previous work, Vernier and Ben-Shahar showed that bees develop different scent profiles as they age, and that gatekeeper bees respond differently to foragers returning to the hive compared with younger bees that have never ventured outside.

That research established a relationship between nestmate recognition and the clearly defined, age-dependent division of labor typical to honey bee hives.

Only when a bee is old enough to interact with others outside of the hive does it become recognizable to others. That was a clue for the researchers.

If you grow a honey bee in isolation, it will never develop a complete microbiome, Vernier said. It actually has to acquire most of it from interactions with other bees.

For this study, researchers determined that forager bees from different honey bee colonies have different gut microbial communities and CHC profiles by sequencing gut samples and analyzing cuticular extracts. The scientists also conducted cross-hive fostering experiments, raising groups of newly hatched bees in either their own colonies or unrelated colonies.

In the fostering experiments, the researchers found that both source- and host-colony related factors contribute to variations in the overall gut microbial community of individual bees. Of the 14 microbial taxa that significantly differed between treatments, six were similar between bees that shared the same hive environment while they grew up regardless of actual genetic relatedness.

The researchers also found that they could manipulate the microbiome of sister bees by feeding different microbes to newly hatched bees. In addition to developing different gut microbial communities, the bees also grew to have different CHC profiles.

They were unrecognizable to their siblings, Vernier said. Manipulating the microbiome was enough to cause sister bees to develop different scent profiles.

This new work is significant in part because it shows an integral role for the microbiome in the essential, everyday social interactions of honey bees, the Earths most important pollinators, researchers said.

For bees, some of the most complex aspects of their social behavior basically depends on bacteria more than anything else! Ben-Shahar said.

It doesnt matter how related they are, he said. Their ability to say you belong to this group basically depends on getting the right bacteria at the right time. Otherwise, they are blind to it.

And bee ID is key.

The biggest enemy to honey bees is other bees.

During fall, when plants stop producing nectar, there is a period of time when robbing is very prevalent in colonies, Vernier said. Robbing bees will find other colonies, and if theyre able to get in and take some honey, they will go back to their own nests and signal, Hey, go over there. Theres a nest thats not good at guarding, and we can steal their honey.

Robber bees will take that honey and leave the other colony to starve, she said. Its a very strong pressure.

Robbing deprives both the host bees and their associated bacteria with important resources which may have been the original drive to form this special bacteria-animal partnership, the researchers said.

Ben-Shahar and Verniers previous research with honey bees showed that graduating to forager status was a requirement for social membership. Read more.

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'Honey bee, it's me' | The Source - Washington University in St. Louis Newsroom

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Tools developed to probe the virome could aid in variety of research

Researchers at Washington University School of Medicine in St. Louis have received an $8.5 million grant to study the role of gut viruses in inflammatory bowel disease. Tools developed in the course of the project could accelerate research into other roles of the virome in health and disease.

The communities of bacteria that live in our digestive tracts help digest food and produce vitamins, protect against pathogens, and promote the healthy functioning of our immune system. But alongside gut bacteria thrives a vast community of viruses, and we know little about their impact on health and disease.

Efforts to study the gut viral community known as the virome have been hindered by a lack of tools to analyze viral diversity. Researchers at Washington University School of Medicine in St. Louis have received an $8.5 million grant from the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health (NIH) to develop the tools needed to study the role of the virome in inflammatory bowel disease. Once developed, such tools could be applied widely, opening up new avenues of research into the role of the virome in normal physiology and development, as well as diseases such as diabetes, AIDS and cancer.

The virome has been linked to a number of conditions inflammatory bowel disease, malnutrition, graft-versus-host disease and there is also some evidence that the virome supports human health in some ways, said principal investigator David Wang, PhD, a professor of molecular microbiology, and of pathology and immunology. But the problem that plagues virome studies is that people find an association, and then they cant pursue it. Once you find an association, the next step is to see what happens when you introduce the virus to an animal. Does it cause the disease? Make it worse? But there are no tools to carry this out.

Tools to analyze the viral community are relatively scarce partly because viruses are more diverse than bacteria. All bacteria carry certain basic housekeeping genes necessary for life, notably the 16S ribosomal RNA gene. Scientists use this universal gene to screen mixed communities of unidentified bacteria by pulling out all the 16S ribosomal RNA genes and using the sequences to classify the bacteria into families. There is no equivalent universal gene among viruses.

Wang and colleagues previously have discovered differences between the viromes of people with inflammatory bowel disease and healthy people. Inflammatory bowel disease is caused by chronic inflammation in the digestive tract and characterized by persistent diarrhea and abdominal pain. The researchers found that people with the condition carry more Caudovirales, a group of viruses that infects bacteria, and anelloviruses, a family of viruses that infects human cells, in their intestines. But they do not yet know what, if anything, the presence of these viruses means.

The new grant will allow the researchers to follow a group of people with inflammatory bowel disease over time, along with healthy members of their households for comparison. Inflammatory bowel disease tends to be cyclical, flaring up and then resolving again and again. By taking repeated stool samples and analyzing the genetic material of the viruses in such samples, the researchers will be able to see how the makeup of the gut viral community changes over the course of the disease, and gauge whether any particular groups of viruses become more abundant during flare-ups or resolutions. They also will assess what effect treatment has on the virome.

Such analysis will require the development of computational tools to identify the viruses by their genetic sequences, classify them into family groups, identify potential genes within viral sequences, and propose functions of the genes.

With the tools we have now, more than half the sequences cant be classified because they are not similar enough to known sequences, Wang said. We frequently cant even tell whether weve found a virus that infects bacteria or human cells.

Wang and colleagues also will develop ways to cultivate viruses so they can study them. As nonliving things, viruses require a living cell to multiply, which makes cultivation in the lab tricky. To grow viruses that infect human cells, researchers must first culture human cells and then infect them with viruses. But the majority of the viruses in the intestinal tract are likely to infect bacteria, not human cells. Such viruses known as bacteriophages, Latin for bacteria eaters are even more complicated: Researchers must first identify the correct bacterial species from among the thousands in our intestines, culture that species, and then attempt to grow the virus within the bacterial culture.

In previous work, we established the first culture system for a gut virus, Wang said. Were relying on our experience there to try to culture more of these novel viruses. Some of these might actually grow in a quite straightforward way, its just that no one has tried yet. And once we have the viruses, then we can use them to start doing experiments in animal models of inflammatory bowel disease.

The impact of the gut bacterial community on human health is a hot topic of study, with a possible role in health conditions ranging from autoimmunity to heart disease to psychiatric illnesses. The virome may prove to be equally consequential if only we can find a way to investigate it.

This isnt a typical grant, because part of its goal is to build resources that will then be available to the scientific community, Wang said. Creating tools is unsexy and usually unfundable. But we have to build these tools before we can answer the exciting questions.

Along with Wang, the research team includes Michael S. Diamond, MD, PhD, the Herbert S. Gasser Professor of Medicine, and Scott Handley, PhD, an associate professor of pathology and immunology, both at Washington University; Thaddeus Stappenbeck, MD, PhD, of the Cleveland Clinic; and collaborators at Cambridge University in the United Kingdom, San Diego State University in California, and Flinders University in Australia.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Role of gut viruses in inflammatory bowel disease is focus of $8.5 million grant - Washington University School of Medicine in St. Louis

Sponsored Content by the Janssen Pharmaceutical Companies of Johnson & Johnson

When it comes to new therapies, patients with lung cancer face a significant global unmet medical need. Not only does lung cancer have the highest mortality rate of all cancers worldwide, its associated with significant heterogeneity with many driver mechanisms that make it complex and difficult to treat.

The driver mechanisms we have come to understand have provided important insights into new approaches for treating lung cancer, said Matt Lorenzi, PhD., Vice President, Disease Area Leader, Solid Tumor Targeted Therapy, Janssen. Through deeper knowledge about the biology of lung cancer, we are making progress in the advancement of precision medicines that target specific pathways, including patients who have developed a resistance to other therapies.

Ongoing research efforts are continuing to show promise. Targeted therapy approaches that have derived from an understanding of driver mechanisms have helped contribute to a decline in mortality from non-small cell lung cancer (NSCLC), the most common type of lung cancer, as recently reported in The New England Journal of Medicine. In addition, immunotherapy-based approaches to reactivate or redirect a patients immune system for tumor targeting are standard of care or showing future promise, respectively.

For key oncogenic driver pathways, researchers are learning much more about the role that various genetic mutations and alterations play, and development efforts are focused on precisely targeting these defects. One of the most common genetic alterations in NSCLC is epidermal growth factor receptor (EGFR), a mutation that helps cells grow and divide. While targeting EGFR has proven to be an effective treatment intervention, tumors eventually become resistant to treatment. Therefore, the role of bispecific therapies, meaning treatments that target more than one pathway, is becoming increasingly important in the treatment of lung and other types of cancer, along with new combination-based regimens.

As researchers learn more about non-small cell lung cancer specifically as it relates to how certain genetic factors can influence the severity of disease and how a patient responds to treatment the importance of genetic testing is becoming clear, as is the importance of novel modalities and combination therapies, said Lorenzi. The complexity of lung cancer highlights the importance of developing regimens that incorporate multiple mechanisms of action to target the key pathways underlying the disease. We aim for treatment to act against each persons unique cancer, as effectively and safely as possible.

While targeted therapies are among the most discussed current research avenues, other areas are proving promising as well. One uses an approach called synthetic lethality against additional genetic alterations in lung cancer, and another uses immune cell re-direction to leverage the immune system to specifically target lung cancer cells with tumor-associated antigens.

These therapeutic developments offer a promising outlook for the future of lung cancer treatment and are further complemented by exciting efforts underway through the Johnson & Johnson Lung Cancer Initiative, which are focused on unique approaches to intercept the disease at its earliest stage or to prevent it altogether.

Despite many encouraging treatment advances in lung cancer, its five-year survival rate remains among the lowest of any cancer. Improving this statistic will require the ongoing development of novel treatments, the identification of strategic collaborators, and the advancement of scientific leadership in disease prevention and interception. As an organization that has been at the forefront of the discovery and development of new cancer therapies for more than a decade, Janssen, together with the Johnson & Johnson Lung Cancer Initiative, is committed to advancing novel approaches to support efforts throughout the medical community to transform the trajectory of lung cancer.

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Addressing Unmet Medical Needs of Patients with Lung Cancer - Cancer Therapy Advisor

PHILADELPHIA, Oct. 06, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that management will participate in the virtual 2020 Cell & Gene Meeting on the Mesa, October 12-16, 2020.

Passage Bios Chief Operating Officer, Jill M. Quigley, J.D., will deliver an overview of the company as part of the 2020 company presentations segment of the conference. Passage Bios company presentation will be available to view on-demand throughout the entirety of the conference.

Additionally, Alex Fotopoulos, Chief Technical Officer of Passage Bio, will participate in the panel discussion titled, Accelerating Biotherapeutics Development and Production for Increased Market Impact of Life Saving Therapeutics, moderated by Susan DCosta, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific. The session will cover key topics such as integrated vs. modular manufacturing approaches, ensuring security of supply, proactive approaches to regulatory compliance, and ultimately, the panels vision for the future. The panel session will be available on-demand to attendees as of Monday, October 12, 2020.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a five-day virtual conference featuring more than 120 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies. The meeting also includes over 100 panelists and featured speakers taking part in 20 in-depth sessions covering all aspects of cell and gene therapy commercialization.

Please visit http://www.meetingonthemesa.com for full information including registration. Complimentary attendance at this event is available for credentialed investors and members of the media only. Investors should contact Laura Stringham at lstringham@alliancerm.org and interested media should contact Kaitlyn Dupont at kdupont@alliancerm.org.

About Passage BioPassage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe disease.

For further information, please contact:

Investors:Sarah McCabe and Zofia MitaStern Investor Relations, Inc.sarah.mccabe@sternir.com zofia.mita@sternir.com

Media:Gwen FisherPassage Bio215.407.1548gfisher@passagebio.com

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Passage Bio to Participate in 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire

ALISO VIEJO, Calif., Oct. 1, 2020 /PRNewswire/ --Ambry Genetics(Ambry), a leading clinical genetic testing lab, is launching its CARE for COVID Program with Western Springs School District 101 and The Green Alliance International. The Ambry Genetics Comprehensive, Assessment, Risk, and Education (CARE) for COVID Program is designed to help identify and test individuals in need of coronavirus testing. The program provides Western Springs School District 101 with the system, tools, and support needed to screen and test their faculty and other employees as they return to their offices and classrooms this fall. The Western Springs School District, located in a suburb of Chicago, Illinois, serves students from kindergarten to eighth grade. The Green Alliance International will be using the CARE for COVID Program's screening and exposure questionnaire as part of their Gateway Entry Systems program. The Gateway Entry Systems program provides school systems, sports venues, and businesses across the U.S. with the tools needed to safely reopen, including disinfecting technology, wristband body temperature screening, and the CARE for COVID program's symptom and exposure digital questionnaire for remote monitoring.

The CARE for COVID program includes viral testing by RT-PCR for individuals who are exhibiting symptoms or have known exposure, with results returnedwithin 24-48hours of receipt of the sample. Ambry's RT-PCR test uses saliva collection and creates a simpler and more convenient experience than the nasopharyngeal swabs commonly used by other labs.

The CARE for COVID Program also includes:

The robust, one-stop nature of the CARE for COVID Program ensures that individuals are not missed through multiple engagement points. The CARE for COVID Program incorporates the latest guidance from federal agencies, including the CDC. Ambry worked directly with noted experts in academia, industry, and government to develop the program.

"Over the last six months or so, our team has worked tirelessly to transition our lab and the CARE Program into a comprehensive, end-to-end solution to support workforce and community testing efforts," Ambry Genetics CEO Aaron Elliott said. "We look forward to helping more businesses and schools to safely reopen."

Ambry is expanding the CARE for COVID Program to other school districts and businesses this fall. The Program can be tailored depending on the organization's needs, and employers can choose the frequency for asking individuals to complete the screening questionnaire, whether daily or less often. Ambry is contracted with health plans representing over 90% of insured individuals, making it convenient for the employers to bill insurance for the testing performed.

"With the development of our CARE for COVID program, we learned that organizations want an end-to-end solution," Ambry Genetics Chief Commercial Officer Tom Schoenherr said. "If a critical component is missing in the solution, it will not work. The CARE for COVID program includes education, evaluation, assessment, counseling, testing, post-test counseling, workplace exposure tracing, and reporting."

The Ambry CARE program is a population-health, precision-medicine tool that can address employee health and wellness across a range of medical issues. Already used to help people learn whether they are at risk for hereditary cancers including, breastand colon cancers, the program can also be adapted to help identify and manage diabetes, cardiovascular, and other diseases. To learn more about the CARE program and hereditary cancer risk, please visitambrygen.com/care.

ABOUT AMBRY GENETICS

Ambry Genetics, as part of Konica Minolta Precision Medicine, excels at translating scientific research into clinically actionable test results based upon a deep understanding of the human genome and the biology behind genetic disease. Our unparalleled track record of discoveries over 20 years, and growing database that continues to expand in collaboration with academic, corporate and pharmaceutical partners, means we are first to market with innovative products and comprehensive analysis that enable clinicians to confidently inform patient health decisions. We care about what happens to real people, their families, and the people they love, and remain dedicated to providing them and their clinicians with deeper knowledge and fresh insights, so together they can make informed, potentially life-altering healthcare decisions. For more information, please visitambrygen.com.

For more information on risk factors for hereditary cancer, please visit cancer.gov's fact sheet on hereditary cancer and genetic testing.

Press Contact:Liz Squirepress@ambrygen.com(202) 617-4662

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Konica Minolta Precision Medicine (KMPM) Company Ambry Genetics Announces Comprehensive COVID-19 Screening, Management, and Testing Program To Help...

Philadelphia, October 5, 2020Through genetic sequencing and targeted treatment, researchers from Childrens Hospital of Philadelphia (CHOP) have resolved a severe lymphatic disorder in a young woman with kaposiform lymphangiomatosis (KLA), a complex and rare disorder that causes lymphatic vessels around the heart and lung to leak fluid, causing breathing difficulties, infections, and often death. The treatment, which the research team has used successfully in other patients with lymphatic disorders, led to a complete resolution of the patients symptoms and fully remodeled her lymphatic system within a matter of months.

The findings were recently published in EMBO Molecular Medicine.

The resolution of lung disease with lymphatic remodeling is remarkable and potentially should change how we evaluate and treat lung disease in this patient population, said first author Jessica B. Foster, MD, an attending physician in CHOPs Division of Oncology. These results offer hope to other patients with lymphatic-induced lung disease and warrant further investigation.

Brenna, the patient described in the paper, first developed symptoms related to KLA at age 6 and was diagnosed with the condition when she was 10. For nearly a decade, she was treated with a variety of therapies, including rapamycin, an immunosuppressant; prednisone, a steroid; and vincristine, a chemotherapy drug. Despite aggressive treatment, her debilitating symptoms persisted, and so her clinical team at CHOP decided to explore other therapies.

Many patients with KLA have a mutation in the NRAS gene, and the team had previously used a mitogen-activated protein kinase (MEK) inhibitor called trametinib in another patient with a severe lymphatic disorder and NRASmutation. The drug resolved his symptoms and completely remodeled his lymphatic system. Although that patient did not have KLA, the researchers felt the success of trametinib in treating his severe lymphatic symptoms, combined with Brennas rapidly deteriorating condition, warranted the request for expanded access to treat Brenna with the drug.

As Brenna began taking trametinib, a drug historically used to treat metastatic melanoma, the researchers sent samples of her lymph fluid for genetic sequencing. The researchers learned that Brenna did not harbor an NRAS mutation and instead had a mutation in a different gene: CBL, a gene that operates along the Ras pathway, the same genetic pathway implicated in other lymphatic disorders, including the one for which they had used trametinib with great success. Mutations along the Ras pathway result in an overproduction of MEK, which leads to the uncontrolled proliferation of lymphatic vessels. A MEK inhibitor like trametinib brings the production of MEK under control, putting the brakes on a system in overdrive.

Brenna was the first patient with KLA to take trametinib for a lymphatic issue, and within four weeks of starting a low dose, her symptoms improved. Her shortness of breath, coughing, and difficulty breathing while lying flat disappeared. She was able to exercise again, and during the second cycle of her therapy, she began training for a 5K race.

The relatively low dose required for dramatic improvement in this case highlights that a small amount of trametinib may be sufficient to treat lymphatic disorders, which will likely limit the side effect profile compared to the higher doses used for oncologic cases, said senior author Yoav Dori, MD, PhD, Director of the Jill and Mark Fishman Center for Lymphatic Disorders at CHOP. We are now preparing ongoing prospective studies to evaluate Ras pathway inhibition in clinical trials of large cohorts of patients.

Follow up tests have shown significant improvement in Brennas restrictive lung disease, and the fluid in her lungs has disappeared. Her overabundant lymph vessels have remodeled themselves and are now behaving normally. Now 20 years old, she continues to take a daily dose of trametinib, which has kept her symptoms at bay.

Genomic evaluation of vascular anomalies such as KLA have only just begun in recent years, said Denise Adams, MD, Director of the Comprehensive Vascular Anomalies Program (CVAP), a CHOP Frontier Program. This case study demonstrates the power of collaborative, cutting-edge research that reaches across disciplines, from genetics to oncology to cardiology, to bring breakthrough treatments to patients.

Foster et al. Kaposiform lymphangiomatosis effectively treated with MEK inhibition, EMBO Molecular Medicine, online September 7, 2020, DOI: 10.15252/emmm.202012324

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About Childrens Hospital of Philadelphia: Childrens Hospital of Philadelphia was founded in 1855 as the nations first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, Childrens Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 564-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu

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CHOP Researchers Use Precision Medicine to Reverse Severe Lymphatic Disorder in a Patient with KLA - Newswise

CAMBRIDGE, Mass., Oct. 01, 2020 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that management will participate in the following upcoming investor conference:

Chardan Virtual 4th Annual Genetic Medicines ConferenceFireside ChatDate: Monday, October 5, 2020Time: 10:30 a.m. ET

The event will be webcast live and may be accessed on the Editas Medicine website in the Investors and Media section. Archived recordings will be available for approximately 30 days following the event.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a (also known as Cpf1) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Investor ContactMark Mullikin(617) 401-9083mark.mullikin@editasmed.com

Media ContactCristi Barnett(617) 401-0113cristi.barnett@editasmed.com

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Editas Medicine to Participate in Upcoming Investor Conference - GlobeNewswire

Oct. 7, 2020 11:00 UTC

BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc., (Nasdaq: STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced plans to move forward with dosing of STK-001 in children and adolescents in its ongoing Phase 1/2a MONARCH study for Dravet syndrome. Following recent interactions with the U.S. Food and Drug Administration (FDA) related to the partial clinical hold on higher dose levels in the MONARCH study, the FDA will allow the Company to add an additional higher dose level to the single ascending dose (SAD) portion of the study (previously Part A). A total of three dose levels will now be evaluated in this portion of the study: 10 mg, 20 mg and 30 mg. Dosing above 30 mg in this study remains on FDA partial clinical hold.

In addition, subject to FDA review, the Company is preparing to add a multiple ascending dose (MAD) portion to the MONARCH study, replacing Part B. This decision is based on new preclinical repeat-dose toxicology data, which were reviewed by the FDA as part of ongoing discussions with the Company. There were no adverse effects observed in the non-human primate (NHP) repeat dose study. The Company plans to submit a protocol amendment to the FDA, which will reflect these changes to the SAD and MAD portions of the study.

There is an urgent need for more effective medicines for people who are living with Dravet syndrome, so we are pleased to be moving ahead quickly with our plans to continue dosing children and adolescents in this important Phase 1/2a study of STK-001, said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. We appreciate the FDAs timely review of our additional data and look forward to evaluating a total of three individual dose levels in the single ascending dose portion of the study. In addition, we are encouraged by preclinical data that demonstrated the ability to safely achieve greater exposure levels with multiple doses of STK-001. Based on these data, we plan to also evaluate multiple ascending doses of up to 30 mg in this ongoing study. Our team is working diligently to submit a revised protocol to the FDA in the coming days.

In March 2020, the Company announced the FDA had placed a partial clinical hold on higher doses of STK-001 in the MONARCH study, pending additional preclinical testing to determine the safety profile of doses higher than the current no observed adverse effect level (NOAEL). When intrathecal doses above the NOAEL were administered to NHPs, adverse hind limb paresis was observed. This finding is known to occur following intrathecal delivery of antisense oligonucleotides (ASOs) to NHPs and is not known to translate to the human experience. When extremely high dose levels were administered, acute convulsions were observed immediately following STK-001 administration. The dose levels were well above the range of corresponding human doses that would ever be administered in the clinic, and were delivered in a formulation that was at a higher concentration than would be administered in the clinic. There is no apparent correlation of these acute adverse events with the mechanism of action of STK-001.

Enrollment and dosing in MONARCH is ongoing. Preliminary safety and pharmacokinetic data are still anticipated in 2021.

About STK-001

STK-001 is an investigational new medicine for the treatment of Dravet syndrome. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-001. STK-001 has been granted orphan drug designation by the FDA as a potential new treatment for Dravet syndrome.

About Phase 1/2a Clinical Study (MONARCH)

The MONARCH study is a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a pathogenic genetic mutation in the SCN1A gene. The primary objectives for the study will be to assess the safety and tolerability of STK-001, as well as to characterize human pharmacokinetics. A secondary objective will be to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency over a 12-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as quality of life, as secondary endpoints. Stoke plans to enroll approximately 48 patients across 20 sites in the United States.

About Dravet Syndrome

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures, beginning within the first year of life. Dravet syndrome is difficult to treat and has a poor long-term prognosis. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. The effects of the disease go beyond seizures and often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, chronic infections, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom, and it is not concentrated in a particular geographic area or ethnic group.

About Stoke Therapeutics

Stoke Therapeutics (Nasdaq: STOK), is a clinical-stage biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company on Twitter at @StokeTx.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectation about the dosing, timing and execution of our Phase 1/2a MONARCH study of STK-001, including our ability to include a multiple ascending dose portion in the study and the partial clinical hold on Part B of the study; the expansion of our pipeline and the use of the TANGO platform to treat other genetic diseases; our ability to use study data to advance the development of STK-001; the ability of STK-001 to treat the underlying causes of Dravet syndrome; and the ability of TANGO to design medicines to increase protein production and the expected benefits thereof. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop, obtain regulatory approval for and commercialize STK-001 and future product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; failure to protect and enforce our intellectual property, and other proprietary rights; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; environmental risks; risks relating to the use of social media for our business; and the other risks and uncertainties that are described in the Risk Factors section of our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements.

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Stoke Therapeutics Announces Plans to Move Forward With Dosing of STK-001 in Children and Adolescents in its Ongoing Phase 1/2a MONARCH Study for...

Editor's note: Life can be stressful, and many of us have a hard time administering self-care. The current world situation ripe with conflicts, shortages and a pandemic makes things even harder. Dr. Amit Sood, formerly of Mayo Clinic and now head of the Global Center for Resiliency and Wellbeing, is the author of books including "The Mayo Clinic Handbook for Happiness" and "SMART with Dr. Sood, and creator of the mobile app Zizo: Your Resilience Pal. Now, he is writing a weekly column answering readers' questions on these topics. See the tagline to learn how to send him your questions, and he will answer in future columns.

Dear friend,

Of the hundreds of ice-creams I have eaten so far, the only one I remember is the ice-cream I never ate.

That was in 1993. My wife Richa and I were sitting outside a shop in the sweltering New Delhi. We had just bought an ice cream to cool down. From the corner of my eye, I saw a little boy who could use a few calories. I walked up to him and offered my cone that he gladly took. I have savored the gleam I saw in his eyes at least a dozen times since then.

In a very interesting study, researchers looked at the genetic fingerprint of the two types of happiness hedonistic (self-centric) and eudaimonic (other-centric). People who were hedonistically happy had a higher inflammatory and lower anti-viral gene expression. It was just the opposite for the eudaimonic ones. With many illnesses caused by inflammation, you can see why this is so important for our health.

My take on this research is that our genes and the immune system know what is right for us and society. In the current times when a healthy immune system is extremely important for us, promoting kindness is imperative.

Kindness, research shows, pays three times over. Your kind words and actions enhance your health and wellbeing, help others, and the memory of kind actions by itself enhances your wellbeing. A very simple way to enhance your self-worth and happiness today is to count your previous kind actions.

I suggest take out a pen and paper and write the three most selfless things you have done in your life. If you feel up to it, share your experience with someone. Just counting previous acts of kindness can enhance your self-worth and bring happiness to others (you guessed it right witnessing or hearing about others kind actions also increases happiness).

In kindness,

Amit

Dr. Amit Sood answers your questions about stress, resilience, happiness, relationships, and related topics in his column. Email dearfriend@postbulletin.com.

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Our genes know kindness is the best medicine - Brainerd Dispatch

dnaPower Inc, a biotechnology company that offers DNA testing for health and wellness, announces their partnership with Inagene Diagnostics. Inagene Diagnostics is a DNA testing company that uses genetic information to predict response to medications commonly used to treat pain and mental health conditions. These two Canadian DNA testing companies have joined forces to crack the code on personalized healthcare, empowering people to take control of their health and live their best lives.

dnaPower is committed to helping people make better data-driven decisions about their day to day health using genetics. We are delighted to be partnering with Inagene, a strong female-led, Canadian DNA company to include their pharmacogenetic testing.In addition to providing tailored health solutions for your diet, exercise and lifestyle, we can now add the ability to identify the medications best suited for your body to keep you safe and get the best health outcome. Dr. Lois Nahirney, CEO, dnaPower Inc.

Through their partnership, dnaPower and Inagene offer a suite of tailored health solutions for anyone looking to maximize insights to optimize their health. Comprehensive DNA testing is available for preventative health and medical treatment optimization. Personalized insights include tailored diet, exercise, lifestyle recommendations, and medications to optimize ones treatment plan based on their genetic information. Together, these powerful genetic insights act as a biological roadmap to help individuals navigate their best health.

Advancements in technology have opened up a world of opportunities to personalized healthcare in ways never seen before. There is no one size fits all solution when it comes to health. Unique genes can respond differently to foods, types of exercise, and medications. Spending years through trial and error to find the best diet, exercise, and medication is a way of the past. Individuals can now enjoy the ability to take control of their health without the guesswork through genetic testing.

We are excited and proud to partner withdnaPower another leading-edge Canadian company! Our teams care deeply about helping clients and are passionate about bringing innovation and personalized medicine to consumers. Helping individuals and their healthcare providers determine which drug and dose will work best, with less trial and error, is just part of the equation; the opportunity to learn what diet and exercise regimen works best for you, further enhances the client experience. Nancy White, CEO, Inagene Diagnostics.

To celebrate their partnership, dnaPower and Inagene are offering a combined special on their DNA tests. The Ultimate Insights Package includes the suite of dnaPower and Inagene DNA tests at the incredible price of $648 (retail value $1495). See this special offer.

Together, dnaPower and Inagene are passionate about empowering individuals to control their health with genetic testing, sharing a unified principle that knowledge is power.

About Inagene Diagnostics

With over two decades of experience in genetic research and diagnostics, combined with over 30 years of commercial health experience, Inagene has witnessed and been a part of the growing technology that now makes personalized healthcare possible. Inagene believes patients are not simply seeking more information, but practical and individualized information they can use to help make the best decisions about their health. Inagene Personalized InsightsTM makes it easy for patients and health care practitioners to navigate to the safest and most effective treatment options for medications used for pain and mental health conditions, and steer clear of those that arent.

About dnaPower Inc

dnaPower was one of the first in the world to offer DNA health testing, launching in 2008. They saw enormous potential for new DNA technology to help support peoples health and wellness, particularly in diet and fitness. dnaPower was a pioneer in applying leading-edge gene research to develop targeted gene panels. Since then, they have been providing personalized testing and professional support to help people like you make better, data-driven decisions about your health. With years of experience, dnaPower provides clear results and specific recommendations to help you take proactive and positive action for your unique body.

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Cracking the code to live your best life: Canadian DNA testing companies, dnaPower and Inagene, partner to unlock true personalized health through...

(UroToday.com) Since 2015, multiple studies together have suggested that approximately a quarter of metastatic castration-resistant prostate cancer (mCRPC) tumors have mutations in DNA damage repair (DDR) genes. These alterations, especially those in genes for homologous recombination repair (HRR), are associated to varying degrees with response to PARP inhibitor therapy. Two PARP inhibitors are now approved for the care of patients with mCRPC and mutations in certain HRR genes: olaparib (based on PROfound trial) and rucaparib (based on TRITON2 trial). Given the significance of alterations in HRR genes in mCRPC, genomic testing for HRR alterations has been included in mCRPC treatment guidelines with the purpose of guiding genetic counseling and therapy selection.

In this poster, Dr. Neal Shore and colleagues describe real-world patterns and predictors of tissue testing for alterations in HRR genes. They focused on the community cancer care setting. Data were collected from the Flatiron Health Electronic Health Record derived database that was collected between 2013 and March 2019, prior to the approval of PARP inhibitor therapy in mCRPC, and focused on testing for alterations in BRCA1, BRCA2, ATM, CDK12, FANCA, and PALB2). The Flatiron database contains information on patient demographics, prostate cancer characteristics, and treatment, as well as genetic testing results.

From the Flatiron database, 5,213 patients with mCRPC were identified, 91% of which were managed at community oncology care centers. Of these patients, 674 (12.9%) underwent testing for the specified HRR gene alterations. Patient characteristics are shown below.

The breakdown of the 674 patients by testing method and number of genes tested is shown below.

BRCA1 and BRCA2 were the most commonly tested genes. A substantial portion of patients underwent only blood/saliva testing (40.5%, capable of detecting germline or circulating tumor DNA) or tumor-tissue testing only (42.1%, capable of detecting germline or tumor tissue DNA alterations). Only 1.8% of patients undergoing blood/saliva testing were tested for all 6 HRR genes, and 69% of tumor-tissue testing covered all 6 genes. Amongst the 286 patients who had negative blood/saliva testing, only 12.6% underwent tumor-tissue profiling, and 5 of these patients (13.9%) had an HRR alteration found.

The most common testing platform utilized was Foundation Medicine, followed by Guardant. Testing rates and the inclusion of more genes increased with time.

The prevalence of HRR alterations found is shown below.

Treatment at an academic medical center or having received multiple prior lines of therapy was associated with a higher likelihood of having HRR mutational profiling. Older age and higher PSA value at diagnosis of mCRPC were significantly associated with a lower likelihood of HRR mutation profiling.

The poster concludes that rates of HRR mutational testing did not increase dramatically with changes to the NCCN guidelines recommending this testing in 2017. Increased awareness of recommendations for testing, especially with the approval of two therapeutic agents contingent upon the presence of HRR alterations, is critical for the care of patients with mCRPC.

Presented by: Neal Shore, MD FACS, Urologist, and Director of the Carolina Urologic Research Center, Myrtle Beach, South Carolina

Written by: Alok Tewari, MD, Ph.D., Medical Oncologist at the Dana-Farber Cancer Institute, at the 2020 European Society for Medical Oncology Virtual Congress (#ESMO20), September 19th-September 21st, 2020

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ESMO Virtual Congress 2020: Real-World Patterns of Genomic Testing in Patients with Metastatic Castration-R... - UroToday

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that data from the companys portfolio of cystic fibrosis (CF) medicines will be presented at the 43rd European Cystic Fibrosis Digital Conference (ECFS) held September 24-25, 2020 and the 2020 North American Cystic Fibrosis Virtual Conference (NACFC) taking place October 7-23, 2020. An oral presentation at the ECFS Digital Conference will highlight, for the first time, interim results from the TRIKAFTA open-label extension study, which showed safety and efficacy consistent with the results of the Phase 3 pivotal studies in patients with CF ages 12 and older with F508del/Minimal Function (F/MF) or F508del/F508del (F/F) genotypes. Four additional scientific abstracts for ORKAMBI and TRIKAFTA were published in the Journal of Cystic Fibrosis as part of the ECFS conference. In addition, six scientific presentations will occur at NACFC regarding KALYDECO, ORKAMBI and TRIKAFTA, including new data from KALYDECO in infants ages 4 to less than 6 months old.

As we continue to reach additional people with CF with our medicines, gaining a better understanding of their long-term and real-world impact becomes even more important, said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. We are pleased to report the first longer-term data for TRIKAFTA which show the significant benefits seen early are maintained through one year of treatment.

Data highlighting interim results from the ongoing TRIKAFTA open-label extension (OLE) study to evaluate long-term safety and efficacy in people with CF ages 12 and older with F508del/Minimal Function (F/MF) or F508del/F508del (F/F) genotypes who completed pivotal studies will be presented at the ECFS Digital Conference. In the interim analysis, TRIKAFTA was generally well-tolerated, with no new safety concerns. The data show that the marked improvements observed in the prior pivotal studies across multiple efficacy endpoints, including, percent predicted forced expiratory volume in 1 second (ppFEV1), sweat chloride (SwCl), Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score, and body mass index (BMI), were sustained with continued treatment with TRIKAFTA.

A full listing of Vertex scientific presentations at ECFS and NACFC are below:

Abstract Title

PresentationType

PresentingAuthor

Date/ Time

ELX/TEZ/IVA

A phase 3, open-label extension study of elexacaftor/tezacaftor/ivacaftor: interim analysis of safety and efficacy in people with cystic fibrosis and F508del/minimal function or F508del/F508del genotypes

ECFS Oral Presentation

Professor Griese

September 24, 2020

11:21-11:45 a.m. CET

Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis heterozygous for F508del and a minimal function mutation: results from a phase 3 clinical study

ECFS published abstract: Journal of Cystic Fibrosis 19S2 (2020) S55S168, P221

NACFC Poster Presentation #447

Professor Fajac

Oct 7 Oct 23, 2020

Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis homozygous for F508del: results from a phase 3 clinical study

ECFS published abstract: Journal of Cystic Fibrosis 19S2 (2020) S1S36, WS19.6

NACFC Poster Presentation #478

Professor Majoor

Oct 7 Oct 23, 2020

IVA

An observational study of ivacaftor in people with cystic fibrosis and selected non-G551D gating mutations: outcomes from the third interim analysis of the VOCAL study

NACFC Poster Presentation

#466

Professor Kors van der Ent

Oct 7 Oct 23, 2020

Ivacaftor in 4 to < 6-month-old infants with a gating mutation: results of a 2-part, single-arm, phase 3 study

NACFC Poster Presentation

#415

Dr. Rosenfeld

Oct 7 Oct 23, 2020

Real-world outcomes in children aged 2-5 with CF treated with ivacaftor

NACFC Poster Presentation

#141

Dr. Volkova

Oct 7 Oct 23, 2020

LUM/IVA

Long-term safety of lumacaftor/ivacaftor in persons with cystic fibrosis aged 2-5 years homozygous for the F508del-CFTR mutation (F/F)

ECFS Published abstract:

Journal of Cystic Fibrosis 19S2 (2020) S1S36, WS19.2

Disease progression in F508del homozygous (F/F) persons with cystic fibrosis treated with lumacaftor/ivacaftor (LUM/IVA): interim results of a long-term safety study using data from the US Cystic Fibrosis Foundation Patient Registry (CFFPR)

ECFS Published abstract:

Journal of Cystic Fibrosis 19S2 (2020) S1S36, WS13.1

NACFC Poster Presentation

#190

Dr. Bower

Oct 7 Oct 23, 2020

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO (ivacaftor), TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), and ORKAMBI (lumacaftor/ivacaftor)

What is KALYDECO?KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 months and older who have at least one mutation in their CF gene that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 6 months of age.

What is TRIKAFTA?TRIKAFTA is a prescription medicine used for the treatment of CF in patients aged 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age.

What is ORKAMBI?ORKAMBI is a prescription medicine used for the treatment of CF in patients age 2 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. ORKAMBI should only be used in these patients. It is not known if ORKAMBI is safe and effective in patients under 2 years of age.

Patients should not take KALYDECO or TRIKAFTA if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; or St. Johns wort.

Patients should not take ORKAMBI if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; the seizure medicines phenobarbital, carbamazepine, or phenytoin; the sedatives and anti-anxiety medicines triazolam or midazolam; the immunosuppressant medicines cyclosporine, everolimus, sirolimus, or tacrolimus; or St. Johns wort.

Before taking KALYDECO, TRIKAFTA, or ORKAMBI, patients should tell their doctor about all of their medical conditions, including if they: have or have had liver problems; have kidney problems; have had an organ transplant; are pregnant or plan to become pregnant because it is not known if KALYDECO, TRIKAFTA, or ORKAMBI will harm an unborn baby; or are breastfeeding or planning to breastfeed because it is not known if KALYDECO, TRIKAFTA, or ORKAMBI passes into breast milk. Before taking ORKAMBI, patients should tell their doctor if they are using birth control as hormonal contraceptives, including oral, injectable, transdermal, or implantable forms should not be used as a method of birth control when taking ORKAMBI.

KALYDECO, TRIKAFTA, or ORKAMBI may affect the way other medicines work, and other medicines may affect how KALYDECO, TRIKAFTA, or ORKAMBI work. Therefore, the dose of KALYDECO, TRIKAFTA, or ORKAMBI may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.

KALYDECO or TRIKAFTA can cause dizziness in some people who take it. Patients should not drive a car, use machinery, or do anything that needs them to be alert until they know how KALYDECO or TRIKAFTA affects them.

When taking ORKAMBI, patients should tell their doctor if they stop taking ORKAMBI for more than 1 week as their doctor may need to change the dose of ORKAMBI or other medicines the patient is taking.

Patients should avoid food or drink containing grapefruit or Seville oranges while taking KALYDECO. Patients should avoid food or drink containing grapefruit while taking TRIKAFTA.

KALYDECO, TRIKAFTA, and ORKAMBI can cause serious side effects, such as:

High liver enzymes in the blood have been reported in patients receiving KALYDECO, TRIKAFTA, or ORKAMBI. The patient's doctor will do blood tests to check their liver before starting treatment with KALYDECO, TRIKAFTA, or ORKAMBI; every 3 months during the first year of treatment; and every year while on treatment. For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.

Breathing problems such as shortness of breath or chest tightness in patients when starting ORKAMBI, especially in patients who have poor lung function. If a patient has poor lung function, their doctor may monitor them more closely when starting ORKAMBI.

An increase in blood pressure in some people receiving ORKAMBI. The patients doctor should monitor their blood pressure during treatment with ORKAMBI.

Abnormality of the eye lens (cataract) in some children and adolescents treated with KALYDECO, TRIKAFTA, or ORKAMBI. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with KALYDECO, TRIKAFTA, or ORKAMBI to look for cataracts.

The most common side effects of KALYDECO include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.

The most common side effects of TRIKAFTA include headache, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.

The most common side effects of ORKAMBI include breathing problems, such as shortness of breath and chest tightness; nausea; diarrhea; fatigue; increase in a certain blood enzyme called creatinine phosphokinase; rash; gas; common cold, including sore throat, stuffy or runny nose; flu or flu-like symptoms; and irregular, missed, or abnormal periods (menses) and increase in the amount of menstrual bleeding. Additional side effects seen in children include: cough with sputum, stuffy nose, headache, stomach pain, and increase in sputum.

These are not all the possible side effects of KALYDECO, TRIKAFTA, or ORKAMBI. Please click product link to see the full Prescribing Information for KALYDECO, TRIKAFTA, or ORKAMBI.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency, and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Carmen Bozic in this press release, statements regarding the potential benefits, safety and efficacy of TRIKAFTA, KALYDECO and ORKAMBI, and our plans to present data at the ECFS and the NACFC, including data from our TRIKAFA open-label extension study, scientific abstracts for ORKAMBI and TRIKAFTA, and scientific presentations regarding KALYDECO, ORKAMBI and TRIKAFTA. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration, approval or further development of its compounds due to safety, efficacy or other reasons, risks related to approval and commercialization of our medicines, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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Vertex to Present New Data at European and North American Virtual Cystic Fibrosis Conferences Highlighting Long-Term Use of CFTR Modulators - Business...