StemCell Therapy

By Deborah Borfitz

October 15, 2020| The Bio-IT World Conference & Expo closed out with a plenary keynote presentation on preventive genomics by Robert Green, M.D., professor of medicine at Harvard Medical School and a physician-scientist who directs the G2P Research Program at Brigham and Womens Hospital and the Broad Institute. Data-sharing difficulties were a recurring theme at this years conference but, as the COVID-19 Host Genetics Initiative has demonstrated, it is possible to combine genomic data to rapidly explore markers of disease, he says. But far more daily deaths are caused by cancer and cardiovascular diseasenot the pandemic virusand 59 of the causal genes are already known and actionable.

Genomic information is rarely incorporated into clinical care partly because labs, not care providers, are doing most of the testing and doctors are unclear if the benefits outweigh the costs and risks, says Green. The clinical value of DNA sequencing is also unproven, although its the central feature of personalized medicine programs that have been popping up around the country.

Green presented lessons learned from the MedSeq, exploring the impacts of incorporating genomic sequencing into everyday medicine for people with and without a suspected genetic cardiac disease, and BabySeq, testing methods for integrating sequencing into the care of newborns. Both are randomized trials funded by the National Institutes of Health.

MedSeq involved primary care physicians taking comprehensive family histories on participants with or without the addition of one-page genomic reports and following their outcomes. Reports from preventive genomic testing focused on defined, disease-specific variants with the highest clinical actionability, says Green, as distinct from indication-based testing looking at a wider universe of variants known or suspected of being pathogenic.

Notably, Green says, neither doctors nor patients experienced test-related anxietyeven when a monogenetic risk variant was discovered. In 100 individuals, 20% were found to carry a dominant mutation for a monogenetic condition. In fact, among the top four genetic mutations, sequencing often discovered ongoing disease that the healthcare system had missed.

Participating doctors, after only six hours of training, did not make any errors in communicating the results, adds Green. Healthcare spending six months post-disclosure was higher but not extraordinarily more. Two years later, 22% had been reclassified (e.g., variant of uncertain significance now likely benign or likely pathogenic variant now pathogenic).

In the smaller BabySeq Project, 11% of participants were identified as having monogenetic disease risk, Green says. As with MedSeq, a substantial number with genetic mutations already had phenotypic evidence of disease previously missed by their healthcare providers.

BabySeq additionally revealed no difference in bonding or vulnerability, says Green. Catastrophic distress is not an obstacle [to sequencing], as has often been suggested. The falling cost of genomic sequencing and interpretation should further improve the benefit-to-cost ratio.

Exactly how often does sequencing reveal something important? Herere the stats from Green: 91% of the time for recessive mutations, 80% for atypical responses to medications, 15% for dominant mutation, and 50% for elevated polygenic risk specific to at least one condition such as diabetes or cancer.

Polarizing Topic

The Mass General Brigham Biobank, which looked for the 59 genes linked to disease, has identified such mutations in over 350 of the roughly 36,000 people it has sequenced. In 75% if those cases, the mutations were linked to either cardiovascular disease or cancer and the individuals had no idea they were carrying mutations, says Green.

A significant number did not even want to know of their risk, he adds. A similarly high number met National Comprehensive Cancer Center criteria for genetic testing but had never before been tested.

The Preventive Genomics Clinic at Brigham and Womens Hospital, staffed by genetics experts and counselors, offers individuals a menu of testing options (whole genome sequencing as well as smaller panels) and also gives patients the option of being seen via telemedicine. The heart-touching stories shared on its website include a man nudged by discovered mutations to finally get a colonoscopy, revealing two cancerous lesions that were subsequently extracted, and another with worsening heart disease who learned the underlying cause was Fabry diseasea rare but treatable condition.

Genomics is a notoriously polarizing subject, Green says. The challenge in convincing the skeptics is that genomics crosses multiple therapeutic domains and testing needs to be repeated over individuals lifetime.

The exceptionalism of genomics is sometimes misplaced, he later adds, referring to the disproportionate amount of fear about misuse of genetic information relative to psychological or infectious disease data. Its perfectly possible for large groups to share genomic data that is not identifiable. Its not full-proof, but its [technically] feasible.

Federal genetic privacy laws prevent genetics-based discrimination by employers and health insurers, Green says. In July, Florida became the first state in the nation to enact a DNA privacy law that also prohibits life, disability and long-term care insurance companies from using genetic tests for coverage purposes.

Editors Note: Even if you missed the start of the event, Bio-IT World Conference & Expo virtualis still live. Register nowfor on-demand presentations.

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Proving The Value Of Preventive Genomics - Bio-IT World

PHILADELPHIA, Oct. 13, 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 publication of data in a murine model of GM1 gangliosidosis (GM1) demonstrating that a single intracerebroventricular injection of an optimized adeno-associated virus (AAV) into the cerebral spinal fluid (CSF) resulted in significant expression of Beta-galactosidase (-gal) in the brain and peripheral tissues, and demonstrated dose-related reductions in neuronal lysosomal storage lesions, neurological impairment and improvement in survival. These data were published online ahead of print in the November issue of the peer-reviewed scientific journal Human Gene Therapy (HGT).

This study suggests that delivery of an AAV vector optimized to express b-gal directly into the CSF restored b-gal activity in the brain and, if further developed and tested in human clinical trials, may be effective in modifying and preventing the devastating effects of the genetic disease GM1, said James Wilson, M.D., Ph.D., director of the Gene Therapy Program at the University of Pennsylvania (Penn) and chief scientific advisor of Passage Bio. The AAV vector used in the study is the same as Passage Bios PBGM01 gene therapy, which is designed to deliver a functional human GLB1 gene into the brain and optimized to express -gal. These preclinical study data support the further development of PBGM01 as a potential therapy for patients suffering from GM1.

GM1 is a rare and often life-threatening monogenic lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid -gal. Reduced -gal activity results in the accumulation of toxic levels of GM1 in neurons throughout the brain, causing rapidly progressing neurodegeneration. GM1 manifests as a continuum of disease and is most severe in the infantile form, which is characterized by onset in the first six months of life with hypotonia (reduced muscle tone), progressive CNS dysfunction, and rapid developmental regression. Life expectancy for infants with GM1 is two to four years, and infantile GM1 represents approximately 60 percent of the incidence of 0.5 to 1 in 100,000 live births. Currently, there are no approved disease-modifying therapies available.

Results of the PBGM01 preclinical study were reported in the paper titled, A single injection of an optimized AAV vector into cerebrospinal fluid corrects neurological disease in a murine model of GM1 gangliosidosis, by Christian Hinderer, M.D., Ph.D., and colleagues, including gene transfer pioneer Dr. Wilson, from the Gene therapy Program, Department of Medicine, University of Pennsylvania Perlman School of Medicine. The study in part was previously presented at the 22nd annual Meeting of the American Society for Cell and Gene Therapy (ASCGT) in 2019.

This research evaluated the impact of single intracerebroventricular administration of the human -gal containing AAV vector on -galactosidase enzyme activity in the murine brain and peripheral tissues, lysosomal storage lesions, neurological function (including neurological exams and gait analysis) and survival in mice lacking the -galactosidase gene. The mice received the single administration at age one month and were evaluated over 300 days. -gal activity was increased significantly in the cerebral spinal fluid and serum of the vector-treated mice compared to vehicle control-treated mice. Significant improvements in gait assessments as measured by stride length and hind paw print length and significant preservation of neurological function as measured by neurological exam scores were observed throughout the study period in the human -gal vector-treated mice. There were significant decreases in lysosomal storage lesions of vector-treated animals and by day 300 all animals that received the two highest doses were still alive, whereas none of the vehicle control-treated animals had survived.

Were excited about being able to soon advance PBGM01 into the clinic, and the potential promise it holds for patients with GM1, the majority of whom are infants and for whom there are no approved disease modifying treatments, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. Our plan is to administer PBGM01 through intra-cisterna magna delivery into the brain, which we believe may offer several benefits in terms of safety, efficiency and distribution compared to other approaches.

Passage Bio expects to initiate dosing of PBGM01 in a Phase 1/2 trial late in the fourth quarter of 2020 or early in the first quarter of 2021 and remains on track to report initial 30-day safety and biomarker data late in the first half of 2021.

This research was supported by a research, collaboration and license agreement with Passage Bio. HGT is the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies. Click here to read the full-text article on the HGT website.

About PBGM01PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual -gal enzyme activity and subsequent neurodegeneration. PBGM01 utilizes a next-generation AAVhu68 capsid administered through intra-cisterna magna (ICM) to deliver a functional GLB1 gene encoding -gal to the brain and peripheral tissues. By reducing the accumulation of GM1 gangliosides, PBGM01 has the potential to halt or prevent neuronal toxicity, thereby restoring developmental potential. In preclinical models, PBGM01 has demonstrated broad brain distribution and wide uptake of the -gal enzyme in both the central nervous system (CNS) and critical peripheral organs, suggesting potential treatment for both the CNS and peripheral manifestations of GM1. The Company has received Orphan Drug and Rare Pediatric Disease designation for PBGM01 for patients with GM1 and expects to initiate dosing of its Phase 1/2 trial late in the fourth quarter of 2020 or early in the first quarter of 2021 and remains on track to report initial 30-day safety and biomarker data late in the first half of 2021.

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.

University of Pennsylvania (Penn)Financial DisclosureDr. Wilson is a Penn faculty member and also a scientific collaborator, consultant and co-founder of Passage Bio. As such, he holds an equity stake in the company, receives sponsored research funding from Passage Bio, and as an inventor of certain Penn intellectual property that is licensed to Passage Bio, he may receive additional financial benefits under the license in the future. He is an inventor of intellectual property covering the technology described in paper published in HGT that is licensed from Penn to Passage Bio, and he may receive financial benefits under this license in the future. Penn also holds equity and licensing interests in Passage Bio.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including our planned IND submissions, initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators and partners ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. 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 statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and 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; the risk that positive results in a preclinical study or 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; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates 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; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with theSecurities and Exchange Commission(SEC), and other reports as filed with theSEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:Sarah McCabe and Zofia MitaStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.comzofia.mita@sternir.com

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

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Passage Bio Announces Publication of Preclinical Data That Show Single Injection of Optimized AAV Vector into Cerebral Spinal Fluid - BioSpace

BALA CYNWYD, Pa., Oct. 13, 2020 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Nasdaq:LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases,today announced the formation of its Scientific Advisory Board (SAB). Larimars SAB is comprised of distinguished research scientists, professors and industry experts recognized as key opinion leaders in the fields of rare disease, pediatrics and mitochondrial disease.

Larimar is privileged to have this group of prestigious, multidisciplinary advisors who are committed to advancing the research and development of CTI-1601 for Friedreichs ataxia, said Nancy M. Ruiz, MD, FACP, FIDSA, Chief Medical Officer of Larimar Therapeutics. Their scientific perspectives will be invaluable to determine our strategic scientific pathway and support the development of other potential treatments for complex rare diseases to help fill unmet medical needs in this space.

Formalizing the SAB adds to our recent accomplishments, which include resuming our Phase 1 clinical trial of CTI-1601 for Friedreichs ataxia and receiving a positive opinion on orphan drug designation for CTI-1601 from the European Medicines Agencys Committee for Orphan Medicinal Products, said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar Therapeutics. This progress helps position Larimar for success as we continue to execute our strategy of developing treatments for complex rare diseases.

The members of Larimars SAB are as follows:

About Larimar TherapeuticsLarimar Therapeutics, Inc. (Nasdaq:LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The companys lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program in the U.S. as a potential treatment for Friedreichs ataxia, a rare and progressive genetic disease. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit: https://larimartx.com.

Investor Contact:Joyce AllaireLifeSci Advisors, LLC(212) 915-2569jallaire@lifesciadvisors.com

Media Contact:Gina Cestari6 Degrees(917) 797-7904gcestari@6degreespr.com

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Larimar Therapeutics Announces Formation of Scientific Advisory Board - GlobeNewswire

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.

More on pregnancy loss:

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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

Aro Biotherapeutics today announced the appointments of Christopher K. Mirabelli, Ph.D., to its Board of Directors and Masad J. Damha, Ph.D., to its Scientific Advisory Board. Drs. Mirabelli and Damha will provide strategic and scientific counsel on the development of novel Centyrin-RNA therapeutic candidates.

"We are excited to welcome Chris, a well-recognized leader in drug development, to our Board of Directors. As the founder of multiple successful biotechnology companies, and an industry veteran with demonstrated experience in the development of new therapies, his insight and guidance will be invaluable as we advance the development of our pipeline of Centyrin-based therapeutic candidates," said Susan Dillon, Ph.D., Co-Founder and Chief Executive Officer of Aro.

Dr. Mirabelli has spent over 35 years in the pharmaceutical and biotechnology industries as a research scientist, senior executive, and venture capital investor. Currently, he is a managing director of the venture capital firm HealthCare Ventures. Dr. Mirabelli is currently Chairman of the Board of Directors of Leap Therapeutics, where he was the Chief Executive Officer from its founding in 2015 until 2020. Previously, Dr. Mirabelli was Chief Executive Officer and Chairman of the Board of Directors of LeukoSite from its founding in 1993 until its acquisition by Millennium Pharmaceuticals in 1999. Dr. Mirabelli was also a Co-Founder of Ionis Pharmaceuticals. He began his industry career in R&D at SmithKline & French Laboratories, and earned his Ph.D. in Molecular Pharmacology from Baylor College of Medicine and his B.S. in Biology from State University of New York (SUNY) at Fredonia.

"I am looking forward to serving on Aros Board of Directors and working with Sue and her team to further advance their proprietary drug-targeting technology," commented Dr. Mirabelli. "The use of antibodies to target drugs to sites of disease in patients has many limitations. Aros novel approach to overcoming these barriers is unique, and Im excited to contribute to the continued development of their pipeline of next-generation biotherapeutic candidates."

Story continues

"We are pleased to have Masad join Aros Scientific Advisory Board. His work in nucleic acid drug discovery and development has been at the forefront of oligonucleotide research, and his scientific insights will be instrumental as we continue to leverage the promise of Centyrins to achieve new therapeutic mechanisms of action," said Karyn ONeil, Ph.D., Co-Founder and Chief Scientific Officer of Aro.

Dr. Damha is a Distinguished James McGill Professor of Chemistry at McGill University. His research group focuses on synthesis and structural analyses of chemically modified oligonucleotides directed toward biomedical and diagnostic applications. His 200+ publications and dozens of issued patents have been widely cited and used in both fundamental sciences and oligonucleotide-based therapeutic applications. Dr. Damha is the recipient of several awards from the Canadian Society for Chemistry, the Queen Elizabeth II Diamond Jubilee Medal (Governor General of Canada), and McGills Fessenden Professorship in Science Innovation. He is currently President of the International Society of Nucleosides, Nucleotides, Nucleic Acids, and he has served as President of the Oligonucleotide Therapeutics Society. Dr. Damha was awarded a Ph.D. in Organic Chemistry and B.Sc. in Chemistry from McGill University.

Dr. Damha commented, "I am very pleased to join Aro Biotherapeutics Scientific Advisory Board and contribute to the development of innovative therapies that enable efficient and specific delivery of nucleic acid conjugates that address important disease targets."

About Aro Biotherapeutics

Aro is a biotechnology company focused on the research and development of a new generation of protein biologics called Centyrins. The company is developing a wholly-owned pipeline of Centyrins and is working with leaders in the industry on leveraging Centyrins for tissue-specific targeting of therapeutics, including nucleic acid drugs for a diverse set of diseases. For more information, visit http://www.arobiotx.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201013005040/en/

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Mike BeyerSam Brown Inc.mikebeyer@sambrown.com 312-961-2502

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Aro Biotherapeutics Appoints Biotechnology Executive Dr. Christopher Mirabelli to its Board of Directors and Nucleic Acid Therapeutics Expert Dr....

Pharmaceutical & Biotechnology Environmental Monitoring Market Analysis, Growth by Top Companies, Trends by Types and Application, Forecast to 2026Published: 11 hours ago Author: Ashwin NaphadeCategory: #news

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The 63 rating InvestorsObserver gives to Kiniksa Pharmaceuticals Ltd (KNSA) stock puts it near the top of the Biotechnology industry. In addition to scoring higher than 75 percent of stocks in the Biotechnology industry, KNSAs 63 overall rating means the stock scores better than 63 percent of all stocks.

Searching for the best stocks to invest in can be difficult. There are thousands of options and it can be confusing on what actually constitutes a great value. Investors Observer allows you to choose from eight unique metrics to view the top industries and the best performing stocks in that industry. A score of 63 would rank higher than 63 percent of all stocks.

These rankings allows you to easily compare stocks and view what the strengths and weaknesses are of a given company. This lets you find the stocks with the best short and long term growth prospects in a matter of seconds. The combined score incorporates technical and fundamental analysis in order to give a comprehensive overview of a stocks performance. Investors who then want to focus on analysts rankings or valuations are able to see the separate scores for each section.

Kiniksa Pharmaceuticals Ltd (KNSA) stock is trading at $21.13 as of 3:25 PM on Monday, Oct 12, an increase of $0.17, or 0.84% from the previous closing price of $20.95. Volume today is 531,806 compared to average volume of 460,930. The stock has traded between $20.21 and $21.24 so far today.

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Is Kiniksa Pharmaceuticals Ltd (KNSA) the Top Pick in the Biotechnology Industry? - InvestorsObserver

Turning Point Therapeutics Inc (TPTX) is near the top in its industry group according to InvestorsObserver. TPTX gets an overall rating of 64. That means it scores higher than 64 percent of stocks. Turning Point Therapeutics Inc gets a 78 rank in the Biotechnology industry. Biotechnology is number 27 out of 148 industries.

Searching for the best stocks to invest in can be difficult. There are thousands of options and it can be confusing on what actually constitutes a great value. Investors Observer allows you to choose from eight unique metrics to view the top industries and the best performing stocks in that industry. A score of 64 would rank higher than 64 percent of all stocks.

This ranking system incorporates numerous factors used by analysts to compare stocks in greater detail. This allows you to find the best stocks available in any industry with relative ease. These percentile-ranked scores using both fundamental and technical analysis give investors an easy way to view the attractiveness of specific stocks. Stocks with the highest scores have the best evaluations by analysts working on Wall Street.

Turning Point Therapeutics Inc (TPTX) stock is trading at $121.08 as of 11:51 AM on Monday, Oct 12, a gain of $9.27, or 8.29% from the previous closing price of $111.81. The stock has traded between $113.22 and $121.91 so far today. Volume today is 388,776 compared to average volume of 403,050.

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Is Turning Point Therapeutics Inc (TPTX) the Top Pick in the Biotechnology Industry? - InvestorsObserver

A rating of 24 puts CEL-SCI Corporation (CVM) near the bottom of the Biotechnology industry according to InvestorsObserver. CEL-SCI Corporation's score of 24 means it scores higher than 24% of stocks in the industry. CEL-SCI Corporation also received an overall rating of 36, putting it above 36% of all stocks. Biotechnology is ranked 27 out of the 148 industries.

Analyzing stocks can be hard. There are tons of numbers and ratios, and it can be hard to remember what they all mean and what counts as good for a given value. InvestorsObserver ranks stocks on eight different metrics. We percentile rank most of our scores to make it easy for investors to understand. A score of 36 means the stock is more attractive than 36 percent of stocks.

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CEL-SCI Corporation (CVM) stock is trading at $14.94 as of 3:23 PM on Monday, Oct 12, a rise of $0.07, or 0.47% from the previous closing price of $14.87. Volume today is less active than usual. So far 366,839 shares have traded compared to average volume of 545,816 shares. The stock has traded between $14.20 and $15.23 so far today.

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Is CEL-SCI Corporation (CVM) The Right Choice in Biotechnology? - InvestorsObserver

The research report on the global Pharmaceutical and Biotechnology Machines Market outlines Pharmaceutical and Biotechnology Machines market size, value chain structure, regional assessment, industrial environment, and forecast between 2020 to 2026. The rapidly averting industry scenario and current as well as future evaluation of the world Pharmaceutical and Biotechnology Machines market also reported in this study. The report on the global Pharmaceutical and Biotechnology Machines market has been characterized into topological regions, product types, application and prime vendors. Moreover, it delivers different investment opportunities and possible threats based on the intelligent analysis.

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Air LiquideLinde HealthcarePraxairAir ProductsTaiyo Nippon SansoMatheson GasAtlas Copco ABMesser GroupSOL GroupNorcoSicgil India LimitedShenzhen GaofaShenwei MedicalBeijing OrientNanning Lantian

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OxygenNitrous OxideMedical AirOthers(Nitrogen, Carbon Dioxide and Helium)

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Hospitals (Labs & Clinics)Home HealthcareUniversities/Research InstitutionsPharmaceutical & Biotechnology Industries

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Study On Pharmaceutical and Biotechnology Machines Market (impact of COVID-19) 2020-2026: Air Liquide, Linde Healthcare, Praxair, Air Products -...

SAN FRANCISCO, Oct. 13, 2020 (GLOBE NEWSWIRE) -- Nitrome Biosciences (Nitrome), a privately-held biopharmaceutical company developing a platform around its newly discovered class of enzymes to target Parkinsons disease and many other age-related and inflammation disorders, today announced that it has appointed Perry Karsen, a seasoned leader with 35 years of life sciences experience, as executive chairman of its board of directors.

Perry is a highly respected industry executive with significant expertise in leading and growing successful biopharmaceutical companies, said Irene Griswold-Prenner, Ph.D., founder, chief executive officer and chief scientific officer of Nitrome. Perrys strategic counsel and business acumen will be invaluable to Nitrome as we build out a wholly-owned first-in-class enzyme therapeutics platform based on our novel scientific discoveries.

Perry Karsen was the chief executive officer of Celgene Cellular Therapeutics, a division of Celgene Corporation, from 2013 until his retirement in 2015. Previously, Perry held the position of executive vice president and chief operations officer at Celgene, serving in various management positions, including as senior vice president and head of worldwide business development and president of the Asia/Pacific region. Perry was the president and chief executive officer of Pearl Therapeutics, a biotechnology company subsequently acquired by Astra-Zeneca. In addition, Perry held executive positions at Human Genome Sciences, Bristol-Myers Squibb, Genentech and Abbott Laboratories earlier in his career. He also was a general partner at Pequot Ventures focusing on investments in biotechnology and medical devices. Perry has a Bachelor of Science in biological sciences from the University of Illinois, Urbana, a Master of Arts in teaching biology from Duke University, and an MBA from the Kellogg School of Management at Northwestern University. Perry serves on the board of directors and as chair of several private and public biotechnology companies, and on the board of The Gladstone Foundation. He is also a former member of the board of directors and the executive committee of the Biotechnology Innovation Organization (BIO).

Perry Karsen added, I am very excited to be a part of the Nitrome team and look forward to helping the company realize the vast potential for patients of its enzyme therapeutics platform. Irenes breakthrough discovery could be the key to unraveling degeneration evidenced in many age and inflammation related conditions. The potential for Nitrome to advance science and medicine while impacting patients is profound.

About Nitrome BiosciencesNitrome Biosciences is a platform company developing drugs against a newly identified class of enzymes which play a harmful role in a variety of age and inflammation dependent diseases. The therapies that Nitrome is developing will target these enzymes and potentially help slow or halt the progression of diseases including Parkinsons, Nitromes lead indication. The company also aims to expand its proprietary platform to include other diseases, such as other neurodegenerative diseases, macular degeneration, heart disease and cancer. Nitrome has been widely recognized and has won multiple awards including the prestigious Target Advancement grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF). For more information, please visit the companys website at http://www.nitromebiosciences.com.

Investor Relations Contact:Alexandra SantosWheelhouse Life Science Advisorsasantos@wheelhouselsa.com

Media Contact:Aljanae Reynolds Wheelhouse Life Science Advisorsareynolds@wheelhouselsa.com

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Nitrome Biosciences Appoints Industry Veteran Perry Karsen as Executive Chairman of the Board of Directors - GlobeNewswire