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The season 1 finale of Netflix's hit TV series saw the Atlas crew complete the first half of their mission but with significant complications.

WARNING: The following contains SPOILERS for Away,season 1.

The Netflix Original TV showAwayends with a twist forRussian cosmonautMishaPopov and the situation is likely to get worse as the Atlas crew continues their mission on Mars in season 2.Awaystars Hillary Swank as Emma Green, the NASA astronaut leading humankind's first manned mission to Mars. Emma must lead her international team through multiple challenges during the 3-year trip; one such challenge is the revelation that Misha, the ship's most veteran crew member and an engineer, is rapidly loosing his ability to see.

While Netflix hasn't officially orderedAwayseason 2 yet, viewers can expect a renewal based on the show's enduring popularity.Awayis a drama series that premiered on the streaming platform on September 4, 2020. Since its debut, the show has gained a strong following online, and quickly rose to the top of Netflix's Top 10 charts.

Related:Netflix: Every Movie & TV Show Releasing In September 2020

TheAwayseason 1 finale gave the Atlas crew a much-needed win after 10 episodes of drama, tension, and general adversity. The first half of their journey was fraught with challenges even before the mission began in earnest, Emma was faced with the impossibly-difficult decision of choosing between completing her life's ambition and being present for her family during her husband's medical crisis. Realizing that staying behind would ultimately do more harm than good, she chose to proceed with the mission as planned a decision she grappled with throughout season 1. Like Emma, Misha too faced familial difficulties, but unlike his captain, his career pursuits resulted in him becoming estranged from his daughter. To add salt to the wound, season 1 gradually revealed another unfortunate ramification of his extended bouts in space: Misha has developed space blindness.

Awaymay be a fictional show, but it's based on real science. Nasahas admittedthat deep space travel may cause blindness or visual impairment in astronauts. Astudypublished in 2012 found evidence of eyeball deformities in astronauts who had been in space, and posited that that the microgravity astronauts are exposed to on long-duration missions can cause permanent changes to the optic nerve region in other words, the pressure from space flight gradually flattens the back of the eye, which in turn makes one increasingly far-sighted. This is what happens to Misha in Awayseason 1, who at one point tells fellow astronautVivian Wu that he can no longer see her face, describing it as "like an out-of-focus painting." Given that he is an engineer and previously was responsible for fixing complex machinery, his inability to seeobjectsat a close range is a significant hurdle.

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Away Season 2: What Misha's Space Blindness Means For The Mars Mission - Screen Rant

This true story of resilience comes as the pandemic creates a need for greater resilience in everyone.

KNOXVILLE, Tenn Growing up in Knoxville, Chad Foster always knew that he had Retinitis Pigmentosa. And he knew the inherited genetic disorder would eventually take away his sight.

But then it actually happened when he was a student at the University of Tennessee.

"A lot of kids are asked a question when they're growing up, what do you want to be when you grow up? And none of them, and I mean none of them, said they want to grow up to be a blind guy," he said.

For a year or so he felt sorry for himself. Then he made a decision.

"I'm not responsible for my blindness, but I have to be accountable for my life and its outcomes," he said. "I have to figure out how to improve upon or how to control what's what I call inside my sphere of influence, how can I change what I can control? How can I paint a picture of my future that includes this unchangeable fact of blindness? How can I take that and then paint the vision for myself? That's something that I can get behind and get inspired to actually chase my dream."

He was 38 years old when his friend, Paul, suggested that Chad try downhill skiing.

"And I said, 'I don't know. That doesn't sound very safe, Paul.' He said, 'no, they actually have guides that can guide you down the mountain. And, you know, safely in quotes, right? That sounds interesting, I'll consider it,'" he said.

To get down the mountain, a guide follows behind him telling Chad which way to turn so he doesn't smash into trees or other skiers.

"And the texture of the voice tells you something, too. So if there's a real severe voice inflection, you know that you're on the razor's edge. So you should probably carve and really dig in to it," he said.

"I feel free. You know, for so many years I've been tethered to either a dog or a cane or something like that and out there on the mountain, you're completely free."

Conquering the slopes is not surprising.

At the University of Tennessee, Chad Foster made straight As and the Deans list and was later honored as a distinguished alumni.

He built a successful career in technology, software innovations, and motivational speaking.

He was the first blind executive to graduate from the Harvard Business School's Leadership Program.

"A lot of people can benefit from the tools and techniques that I've developed in overcoming my personal adversity, my personal adversity of going blind, and then the changes that we've seen in the business world, and the environments that I've worked in," he said. "So I've really tied that together in Blind Ambition to help people learn how to move their mindset from victim to visionary."

The book comes out in February. He's scheduled a ski trip for January. It seems like a natural way to start 2021 for a guy who knows how to pick himself up again.

"You're going to fall and if you're trying to do something great whether it's you know achieving what you want in the academic or business world or you know skiing a black diamond, you're probably going to fall in pursuit of that and you have to get comfortable with the idea that eventually I'm going to fall and it might hurt every now and then but I'll be okay," he said.

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Knoxville native writes Blind Ambition: How To Go From Victim to Visionary - WBIR.com

On the surface, The Impending Blindness of Billie Scott is simply a coming-of-age story, a sweet and delightful portrait of the wild and wonderful people you can encounter in your early twenties, provided youre amenable. As the title implies, the titular Billie Scott is about to go blind. That would likely panic just about anyone, but Billie has the additional complication of needing to complete ten paintings for a prestigious art gallery before a two-week deadline (imposed by her impending loss of vision) is up. So she forges ahead, out of her comfortable but claustrophobic life in a student flat and out into the world, finding people to paint as she goes.

B

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Zoe Thorogood, not so far in age from Billie, has created a book thats rich with detail without feeling fussy or overly refined. Theres a panel early on where the reader can see the bottom of Billies coffee mug, which features an intact UPC sticker, the sort of detail that grounds and humanizes a large and sometimes complicated story. The books backgrounds are filled with a cacophony of posters and people and mess, lending Billies adventure a tight and confined feeling, even after shes left the borders of her tiny room. The book is largely in grayscale, with swaths of one or two accent colors that change with the scenes and help set the tone for every interaction. The art is lovely but doesnt shy away from grime or the truly absurd, and the story is stronger for it.

Every character is drawn with a level of detail that tells you a lot about them from the moment they appear, but are still given space to surprise readers with their needs and motivations. Billies sketchbook fills quickly with drawings and observations about each one, guiding the reader to view them through her perspective as a young person who might not be aware of just how vulnerable she really is. Thorogood continues this delicate tightrope walk by giving Billie a supporting cast thats neither completely threatening nor saintly, but simply human. Her roommates are friendly despite her long-term avoidance of them, and her encounters throughout the book run the gamut from warm to awful: Shes embraced by a chaotic group of drunk women at a bachelorette party, but shes also robbed and assaulted. Overall, there are more people willing to come to her aid than want to take advantage of her, which feels honest to the world in which Billie lives.

Image: Zoe Thorogood

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Its clear that her navet and unwillingness to trust people are both hindrances to her safety and progress, and Thorogood navigates this complicated idea very well. Billie is granted space to really screw up and make mistakes, but also given second and third chancesopportunities to not only reevaluate and redefine her goals, but also reach them. Its a touching exploration of what it means to make art and how to find your people, and why both things are important. To quote Billies friend Rachel, its about why you should care.

Theres a period towards the end where the metaphors and imagery Thorogood has used thus far give way to something more explicit, and it veers towards preachiness. Billie herself even makes a joke about how cringe-inducing some of it is, which helps to defuse the awkwardnessbut its definitely not as graceful as the vast majority of the book. It feels like Thorogood doesnt trust the readers, or herself, enough to come to the books conclusions on their own, which is a shame; her storytelling up to that point is far more confident and deft than she seems to believe it was. Given this is her first published book, theres no doubt shell continue to hone her craft and use her skills for future projects that will surpass even this great book.

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The Impending Blindness of Billie Scott tells a poignant story of youth, art, and purpose - The A.V. Club

TipRanks

In the investing game, its not only about what you buy; its about when you buy it. One of the most common pieces of advice thrown around the Street, buy low is touted as a tried-and-true tactic.Sure, the strategy seems simple. Stock prices naturally fluctuate on the basis of several factors like earnings results and the macro environment, amongst others, with investors trying to time the market and determine when stocks have hit a bottom. In practice, however, executing on this strategy is no easy task.On top of this, given the volatility that has ruled the markets over the last few weeks, how are investors supposed to gauge when a name is flirting with a bottom? Thats where the Wall Street pros come in.These expert stock pickers have identified three compelling tickers whose current share prices land close to their 52-week lows. Noting that each is set to take back off on an upward trajectory, the analysts see an attractive entry point. Using TipRanks database, we found out that the analyst consensus has rated all three a Strong Buy, with major upside potential also on tap.Progenity (PROG)Offering clear and actionable genetic results, Progenity specializes in providing testing services. The company started trading on Nasdaq in June and saw its shares tumbling 44% since then. With shares changing hands for $8.11, several members of the Street recommend pulling the trigger before it heats up.Piper Sandler analyst Steven Mah points out that even against the backdrop of COVID-19, PROG managed to deliver with its Q2 2020 performance. We are encouraged by the recovery in late Q2 2020 with 75,000 accessioned tests (~79,000 in Q1 2020), driven by noninvasive prenatal testing (NIPT) and carrier screening, the analyst noted. Expounding on this, Mah stated, Progenity did not provide guidance, but June test volumes of ~28,000 were strong (Q1 2020 monthly average was ~26,000) which we believe showcases the durability of its reproductive tests and the success that Progenity has in co-marketing and attaching carrier screening to the more essential NIPT. Of note, despite the pandemic disruptions, Progenity was able to maintain its leading pre-COVID test turnaround times.Additionally, health insurer Aetna is temporarily extending coverage of average-risk NIPT until year-end as a result of the pandemic, with the American College of Obstetricians and Gynecologists (ACOG) also expected to endorse average-risk in the future given its clinical utility, in Mahs opinion.Reflecting another positive, the fourth generation NIPT (single-molecule counting assay) test was able to measure fetal fraction, a key milestone according to Mah, and will continue to be developed into 2021. As the technology could potentially be applied to DNA, RNA, epigenetic markers and proteins for additional clinical applications such as oncology, the analyst is looking forward to the completion of the preeclampsia verification in Q4 2020 and a possible 2H21 launch. We believe preeclampsia (~2.3 billion serviceable market) is a major differentiator for Progenity, allowing them to cross-sell across the full-continuum of reproductive testing, the analyst added.If that wasnt enough, PROG signed its first GI Precision Medicine partnership agreement with a top-20 Pharma company in August. The Oral Biotherapeutic Delivery System (OBDS), an ingestible drug and device combination designed to precisely deliver biologics systemically through a needle-free liquid jet injection into the submucosal tissues of the small intestine, is set to be utilized as part of the collaboration. Mah commented, We believe Progenity can sign additional Pharma deals and look forward to the newsflow coming out on this front.To sum it all up, Mah said, We believe Progenity shares are undervalued given the robust recovery in the core testing business and multiple upcoming growth catalysts.To this end, Mah rates PROG an Overweight (i.e. Buy) along with a $17 price target. Should his thesis play out, a twelve-month gain of 105% could potentially be in the cards. (To watch Mahs track record, click here)Are other analysts in agreement? They are. Only Buy ratings, 4, in fact, have been issued in the last three months. Therefore, the message is clear: PROG is a Strong Buy. Given the $13.33 average price target, shares could climb 60% higher in the next year. (See PROG stock analysis on TipRanks)Tactile Systems Technology (TCMD)Developing at-home therapy devices, Tactile Systems Technology wants to provide new treatments for lymphedema, which occurs when the lymphatic system is impaired, disrupting normal transport of fluid within the body, and chronic venous insufficiency. Down 52% year-to-date, its $32.67 share price lands close to its $29.47 52-week low. Thus, with business trends improving, the Street is pounding the table.Writing for Canaccord, analyst Cecilia Furlong acknowledges that the pandemic has hampered the company, with COVID-19 weighing on both volumes and sales. In the second half of March, volumes were down 50% compared to the first half of the month, and TCMDs patient volumes in April and May remained challenged. That being said, trends started to improve at the end of May.Going forward, given the vast majority of TCMDs clinician customers practice in outpatient or office-based settings, we remain positive on TCMDs ability to demonstrate better insulation against COVID impacts and likely experience a greater bounce-back relative to overall med-tech volume trends, with TCMD further benefitting from its expanding using of technology to remotely engage with clinicians and support patients, Furlong explained.The analyst added, Furthermore, recent trends among some providers to prescribe Flexitouch (an advanced intermittent pneumatic compression device to self-manage lymphedema and nonhealing venous leg ulcers) earlier along the therapy process, as a means to reduce in-person contact, could provide upside near term, as well as potentially transition to a longer-term tailwind.On top of this, Furlong is also optimistic about new CEO Dan Reuvers and the reprioritization of the companys investment and market development efforts. TCMD will shift focus away from its acquired Airwear product line, with it redirecting investments toward its Flexitouch and Entre (a pneumatic compression device used to assist in the home management of chronic swelling and venous ulcers associated with lymphedema and chronic venous insufficiency) products.Given significant under-penetration in the lymphedema/phlebolymphedema market targeted by Flexitouch alongside the large patient population with limited treatment options today targeted by the firms Head & Neck platform, we view the combination of education and clinical data as key to further developing and penetrating these markets... Going forward, we expect management to continue to compile a broad base of clinical data to support reimbursement and drive broad adoption, Furlong commented.All of this prompted Furlong to keep a Buy rating and $62 price target on the stock. This target conveys her confidence in TCMDs ability to soar 90% in the next year. (To watch Furlongs track record, click here)In general, other analysts are on the same page. With 3 Buy ratings and 1 Hold, the word on the Street is that TCMD is a Strong Buy. The $62.33 average price target brings the upside potential to 91%. (See TCMD stock analysis on TipRanks)uniQure N.V. (QURE)Last but not least we have uniQure, which delivers curative gene therapies that could potentially transform the lives of patients. Even though shares have fallen 44% year-to-date to $40, not much higher than its 52-week low of $36.20, multiple analysts still have high hopes.Representing SVB Leerink, 5-star analyst Joseph Schwartz acknowledges that shares struggled after news broke of its collaboration and licensing agreement with CSL Behring for AMT-061, QUREs gene therapy for Hemophilia B, he argues the shareholder base turnover is likely now complete as investors and QURE shift focus to next-in-line AMT-130, its AAV5 gene therapy for Huntingtons Disease (HD).Schwartz further added, With the M&A premium now out of the stock, we see the QUREs current level as an attractive buying opportunity for those investors interested in the companys up and coming CNS gene therapies, internal manufacturing, and robust intellectual property and knowhow.Looking more closely at the agreement with CSL Behring, QURE will be tasked with the completion of the pivotal Phase 3 HOPE-B trial as well as the manufacturing process validation and manufacturing supply of AMT-061.According to management, 26-week Factor IX (FIX) data from all 54 patients enrolled in the trial remains on track, and topline data from the pivotal trial is still slated to read out by YE20. It should be mentioned that in a Phase 2b dose-confirmation study, QURE reported 41% FIX activity out to one year. Additionally, Schwartz points out that with HOPE-B progressing as planned, QURE has continued its manufacturing process validation work ahead of the anticipated BLA/MAA submissions in the U.S. and EU in 2021.On top of this, as part of the deal, QURE is eligible to receive more than $2 billion including a $450 million upfront cash payment, $1.6 billion in regulatory and commercial milestones and double-digit royalties ranging up to the low-twenties percentage of net product sales.With a strengthened cash position, QURE is well funded to rapidly advance CNS assets including AMT-130 (AAV5 gene therapy for Huntingtons Disease (HD)) and AMT-150 (AAV gene therapy for Spinocerebellar Ataxia Type 3/SCA3)...We continue to believe that as QUREs CNS pipeline assets mature, the company could once again be an attractive partner to larger biopharma companies that have recently acquired many publicly traded gene therapy platforms with substantial manufacturing capabilities, Schwartz noted.Everything that QURE has going for it convinced Schwartz to reiterate an Outperform (i.e. Buy) rating. Along with the call, he attached a $67 price target, suggesting 68% upside potential from current levels. (To watch Schwartzs track record, click here)What does the rest of the Street have to say? 9 Buys and 3 Holds have been issued in the last three months, so the consensus rating is a Strong Buy. In addition, the $69.89 average price target indicates 75% upside potential. (See QURE stock analysis on TipRanks)To find good ideas for beaten-down stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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October Declared as Contact Lens Safety Month by Prevent Blindness to Provide Public with Information on How to Keep Eyes Healthy - Yahoo Finance

More News24 Sep 2020 | 4:20 PM

Lucknow, Sep 24 (UNI) Havells India Limited, a leading Fast-Moving Electrical Goods (FMEG) company, on Thursday announced its entry into the refrigerator segment under its consumer durable brand - Lloyd.

Kolkata, Sep 24 (UNI) To meet the diverse requirement of its Tier II and III customers, Ujjivan Small Finance Bank with a long term vision to build a better life for its people and communities announced the launch of Small Commercial Vehicle Finance.

(Rptg, correcting slug)Mumbai, Sep 24 (UNI) IndusInd Bank on Thursday slid by 7.24 pc to Rs 489.45 a major loser in 30 scrips of BSE.

Mumbai, Sep 24 (UNI) IndusInd Bank on Thursday slid by 7.24 pc to Rs 489.45 a major loser in 30 scrips of BSE.

Mumbai, Sep 24 (UNI) The BSE Sensex on Thursday slumped by 1114.82 pts to settle at 36,553.60 as selling was seen across the board amid weak Asian Market.

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Why Corneal Blindness Remains a Massive Problem in India - United News of India

The agreed statement of facts offers a template for risk teams seeking to avoid a similar fate.

The complexity of Westpac's IT systems, especially when an upgrade was needed, sits at the heart of its core failures to report millions of "international fund transfer instructions" (IFTIs). These mainly related to just two of its "corresponding banks" offshore banks that Westpac receives money from and sends money to.

The relationships are critical to allow funds to move across borders. But they contain pitfalls, especially ascertaining the identity of the source of funds and customers to which money is being sent.

AUSTRAC CEO Nicole Rose at a press conference on Thursday to discuss the Westpac settlement.Alex Ellinghausen

Westpac's "Australasian Cash Management" (ACM) arrangements allowed its corresponding banks to use Westpac's direct access to the Australian clearing system to process payments. The offshore banks liked this: it reduced costs for them, by allowing them to send "structured", or aggregated, files, which was more efficient than sending payment messages, via a system known as SWIFT, for each and every payment.

When these corresponding banks send money into Australia, they must provide Westpac with an IFTI, which Westpac has to send on to AUSTRAC. In 2010, Westpac was upgrading its systems to ensure it could provide AUSTRAC with the reports in the right format. This became the source of the drama that would unfold a decade later.

The bank failed to match the tech upgrades with different ACM arrangements it had set up for different corresponding banks. Its new systems were not properly configured.

One of these was its financial crime IT system, known as Detica, built by BAE Systems. It was the primary tool for screening customers, conducting risk assessments and reporting suspicious transactions to AUSTRAC.

But to get to AUSTRAC, files received from corresponding banks went through another system, known as WIBS. They were then converted into an IFTI format, passing through Detica via another piece of software known as an integrator, and then being processed again into an IFTI that could be uploaded to AUSTRAC.

Westpac realised it had issues with the process as far back as 2010. AUSTRAC queried its IFTI reporting process in late 2011. Westpac was in regular contact with AUSTRAC in late 2010 and early 2011, as it tried to work out a reporting solution for its "structured files".

The statement of facts suggests the bank for which Westpac failed to report the vast majority of IFTIs fell outside the scope of a technical release relating to the Detica system and this was not picked up.

Then, between August 2011 and August 2012, 15 members of Westpac's IT team left to join ANZ, taking with them the bank's corporate knowledge about the complexity of the reporting system. This prevented it setting up a proper audit process that should have ensured incoming IFTIs for two banks were actually going through to AUSTRAC. The regulator described this as "an absence of appropriate end-to-end reconciliation, assurance and oversight processes for IFTI reporting".

"Westpac did not identify that over 72 per cent of all incoming IFTIs received by Westpac for the period 5 November 2013 to 3 September 2018 had not been reported," the statement says.

"As a result of the failure to file the IFTIs on time, AUSTRAC, the ATO and other law enforcement agencies have been deprived of timely information relating to over $11 billion in international payments."

Frustratingly, there were several chances for these reporting errors to have been caught by Westpac.

For example, in July 2013, AUSTRAC recommended it perform a review of its payment instructions to see if they were not being reported to the regulator. The bank prepared a "group assurance report" but it did not catch the non-reporting.

Then, in 2016, a remediation project also failed to identify the reports were not being sent in to AUSTRAC. When the non-reporting from one of the correspondent banks was identified in 2017 by a team leader, it was not escalated to senior management for action.

It was not until 2018 that the proper level of senior management was made aware of the issues. The bank swung into action but by then, the millions of legal breaches had accumulated in dramatic fashion.

The agreed statement of facts also details the holes in Westpac's due diligence that it was obliged to conduct on its corresponding banks. While it asked them questions via a questionnaire, AUSTRAC said these weren't regular enough for higher-risk banks, while the process failed to respond to new risks emerging from the sale of new products to the corresponding banks until November 2019.

There were also issues with the reporting of outgoing IFTIs, when money was being sent from Westpac abroad. One of the products that tripped up the bank was LitePay, which was set up in August 2016 to send up to $3000 to various countries overseas. Again, a systems upgrade was the root cause of the problem.

In May 2017, a technical issue affected database replication, meaning instructions for some IFTIs were not passed between Westpac systems. Then, in November 2018, another technical issue forced internal IT support teams to manually intervene to set the payment status of each transaction, but this then prevented the automated process for completing the reports which means AUSTRAC did not receive them.

Again, the statement of facts says the bank "did not have appropriate end-to-end reconciliation, assurance and oversight processes in place to identify the IFTI reporting failures relating to the LitePay outgoing IFTIs". The issue was not identified until July last year.

And then there is the failure to heed AUSTRAC's warnings, over a six-year period, about conducting proper due diligence on specific customers showing tendencies of criminal activity.

AUSTRAC published information on child exploitation risks associated with sending frequent, low-value amounts into the Philippines and some other jurisdictions in 2013. The Attorney-General's Department did the same in 2016. Westpac was briefed by AUSTRAC in December 2016 and January 2017, about adopting the new methodology to screen for suspicious payments.

But when it launched LitePay, its detection scenarios for child exploitation risk "did not adequately reflect the guidance, and did not apply to other payment channels". It was not properly implemented until October 2019.

The bank has paid a very heavy price for its failures to action AUSTRAC's requests and its governance failures to audit and proactively identify the issues. AUSTRAC said on Thursday while its contraventions were not the result of any deliberate intention to breach the legislation, "there were opportunities to prevent and detect the non-reporting and, when it was identified, failures to escalate it".

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The tech failures that cost Westpac $1.3b - The Australian Financial Review

WRT the first LTTE today (9/12/20). The concept of white supremacy was not invented; it is a description of the way that some people fantasize about their rightful place in the world. However, this total lack of personal insight often results in similar thinking errors. I wonder if the writer believes that social injustice is also an invention?

The idea that social supremacy is earned by merit, must be similar to the way Pope Urban VIII believed when he cast Galileo into prison. I am confident that his belief that the Earth was the center of the universe helped reinforce his conceit that since he was the head of the church, then he must be the CORE of the center. Such dangerous thinking errors have a long and troubled history in human society. Willful blindness to facts will not help solve our social problems. Some insight might help.

Disclaimer: As submitted to the Arizona Daily Star.

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Letter: Willful Blindness to the Problem of White Supremacy - Arizona Daily Star

BOSTON MAN CHARGED WITH ASSAULT THAT LEFT THE VICTIM BLINDED IN ONE EYE - Andover Leader

September 23, 2020 4:30 pm EDT

It was on September 10th that the police officers responded to a call reporting an unarmed robbery of a 72-year-old man who said he was attacked on Freemont Street in Bostons Mattapan section, police said in a statement Sunday.

During the incident, a male suspect violently punched and kicked a 72-year-old victim, resulting in the permanent loss of vision in the victims left eye, police said.

Adding on to the blindness,The suspect, later identified by police as Willis McGhee, 50, of Boston, was also accused of ripping off the mans pants while trying to steal his wallet from his back pocket, police said.The victim was take to the hospital to be treated for his injuries.

On his arrest,McGhee voluntarily returned to a police station, where he was charged with unarmed robbery, assault and battery of a person over the age of 60, assault and battery by means of a dangerous weapon and mayhem, police said.

McGhee was expected to be arraigned Monday in Dorchester District Court, police said.

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BOSTON MAN CHARGED WITH ASSAULT THAT LEFT THE VICTIM BLINDED IN ONE EYE - Andover Leader

Inherited retinal diseases marketis expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to an annual growth rate of 4.40% in the above-mentioned forecast period.

Inherited Retinal Diseasesmarket research report includes historic data, present and future market trends, environment, technological innovation, upcoming technologies and the technical progress in the allied industry. This market report contains extensive study about miscellaneous market segments and regions, emerging trends, major market drivers, challenges and opportunities in the market. The report has been generated with the comprehensive market insights and analysis that offers superior perspective of the market place. Market segmentation has also been carried out extensively in the Inherited Retinal Diseases Market business report based on a variety of parameters that include applications, verticals, deployment model, end user, and geography.

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The major players covered in the inherited retinal diseases market report are American Society of Gene & Cell Therapy, Spark Therapeutics, Inc., Invitae Corporation., The Choroideremia Research Foundation Inc, American Council of the Blind, Foundation Fighting Blindness, Guide Dogs for the Blind, Inc., Fighting Blindness, FIGHTING BLINDNESS CANADA, Lighthouse Guild, The RDH12 Fund For Sight, Sofia Sees Hope, Retina International, Usher Syndrome Coalition, VisionServe Alliance among other domestic and global players. Market share data is available for global, North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Europe is assumed to be the most lucrative inherited retinal disease market due to a relatively bigger prevalence of acquired retinal complications copulated with a higher treatment-seeking rate.

Global Inherited Retinal Diseases Market Scope and Market Size

Inherited retinal diseases market is segmented on the basis of type, treatment type and distribution channel. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Based on type, inherited retinal diseases market is segmented into leber congenital amaurosis (LCA), retinitis pigmentosa, choroideremia, stargardts disease, and achromatopsia.

Inherited retinal diseases market has also been segmented based on treatment type such as gene therapy treatment, and symptomatic treatment.On the basis of distribution channel, the inherited retinal diseases market is segmented into hospital pharmacies, and retail pharmacies.

Inquiry For Customize Report With Discount at :https://www.databridgemarketresearch.com/inquire-before-buying/?dbmr=global-inherited-retinal-diseases-market

Global Inherited Retinal Diseases Market Drivers:

he growing awareness amongst the physicians and patients regarding the benefits associated with the usage of inherited retinal diseases has been directly impacting the growth of inherited retinal diseases market.

The swelling prevalence of the acquired retinal disorders is foreseen to push the growth of inherited retinal diseases treatment market. Expanding knowledge and treatment-seeking flow is anticipated to feed the germination of the inherited retinal diseases therapy business. Emphasizing knowledge of the inherited retinal disease surgery options and analysis of the condition is moreover awaited to magnify the growth of the inherited retinal disease surgery market.

The production businesses in the inherited retinal diseases market is moreover concentrating on the gene mutation-specific way for developing innovative outputs which is anticipated to accelerate the growth of inherited retinal diseases market. Numerous companies are contracting in collaborations, businesses to perform clinical experiments to promote acquired retinal disorder medication alternatives more efficiently. These certain mentioned factors are expected to drive the market growth in the forecasted period of 2020 to 2027.

TOC of Inherited retinal diseases Market Report Contains:

To know more about the study,https://www.databridgemarketresearch.com/reports/global-inherited-retinal-diseases-market

About Data Bridge Market Research

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Nanoscience has made an impact on a range of industries. With continuous developments, it will only get more exciting for investors.

Through nanotechnology, nanoscience has undeniably impacteda range of industries, from energy to medicine. In the face of continuous nanotechnology research and development, experts are promising an exciting future for the industry.

The terms nanoscience and nanotechnology have been around for a long time, and its common for them to be used interchangeably. However, its important to note that they are not the same.

According toErasmus Mundus, the European Unions higher education program, nanoscience refers to the study, manipulation and engineering of particles and structures on a nanometer scale. For its part, nanotechnology is described as the design and application of nanoscience.

In simple terms, nanoscience is the study of nanomaterials and properties, while nanotechnology is using these materials and properties to create a new product.

Here the Investing News Network provides a comprehensive look at nanoscience investing and nanomaterials, with an overview of the subjects and where they are headed in the future.

The University of Sydneys Nano Institute describes nanoscience as the study of the structure and function of materials on the nanometer scale.

Nanometers are classified as particles that are roughly the size of about 10 atoms in a row. Under those conditions, light and matter behave in a different way as compared to normal sizes.

These behaviours often defy the classical laws of physics and chemistry and can only be understood using the laws of quantum mechanics, the universitys research page states.

The Institute of Nanoscience of Aragon identifies carbon nanotubes (CNTs) as one example of a component that is designed at the nanoscale level. These structures are stronger than steel at the macroscale level. CNT powders are currently used in diverse commercial products, from rechargeable batteries to automotive parts to water filters.

Scientists, researchers and industry experts are enthusiastic about nanoscience and nanoparticles.

As noted in a study published by Jeffrey C. Grossman, a University of California student, quantum properties come into play at the nanoscale level. In simple terms, at the nanoscale level, a materials optical properties, such as color, can be controlled.

Further, the paper states that the surface-to-volume ratio increases at the nano size, opening up new possibilities for applications in catalysis, filtering, and new composite materials, to name only a few.

In other words, the opening up of surface area, which adds new possibilities, can have drastic effects on industries such as manufacturing. New applications in catalysis can allow manufacturing to be sped up, while new composite materials can add more dimension to an end product.

Nanoscale developments could also lead to increased resources and could play a role in the energy sector by increasing efficiency.

As the Royal Society putsit, the aim of nanoscience and nanotechnologies is to produce new or enhanced nanoscale materials.

Nanomaterials are formed when materials have their properties changed at the nanoscale level. Nanomaterials involve elements that contain at least one nanoscale structure, but there are several subcategories of nanomaterials based on their shape and size.

According to the Royal Society, nanowires, nanotubes and nanoparticles like quantum dots, along with nanocrystalline materials, are said to be nanomaterials.

While these are broader classifications of nanomaterials, each of them has several submaterials. Graphene is one popular submaterial and is an example of a nanoplate.

The Integrated Nano-Science & Commodity Exchange, a self-regulated commodity exchange, includes a wide range of nanomaterials and related commodities and lists more than 1,000 nanomaterials.

The exchange states that its entire product range is in excess of 4,500 products, including CNTs, graphene, graphite, ceramics, drug-delivery nanoparticles, metals, nanowires, micron powders, conductive inks, nano-fertilizers and nano-polymers.

As can be seen, nanoscience and nanotechnology are used in a variety of applications across diverse fields, from energy to manufacturing. The University of Sydneys Nano Institute highlights how nanoscienceimpacts manufacturing, energy and the environment through the continuous development of new nano and quantum materials.

With the advancement of materials science and technology, solutions are being worked on for the health and medicine fields, with nanobots gaining popularity in the medical field.

Similarly, nanomaterials like graphene are having a major impact in the technology field graphene is used for various purposes, including in cooling and in batteries.

According to IndustryARC, the global nanotechnology market is projected to reach US$121.8 billion by 2025, growing at a compound annual growth rate of 14.3 percent between 2020 and 2025.

In the US, the National Nanotechnology Initiative, a US government research and development initiative that involves 20 federal and independent agencies, has received cumulative funding of US$27 billion since 2001 to advance research and development of nanoscale projects.

With growth predicted across multiple areas and industries, and with researchers and institutes working on developing the nanoscience field, investors have a slew of nanotechnology stocks to consider.

One popular investment avenue is via graphene, with companies in the space including Applied Graphene Materials (LSE:AGM,OTC Pink:APGMF) and Haydale Graphene Industries (LSE:HAYD). Meanwhile, nanotech stock options include firms such as NanoViricides (NYSE:NNVC), Nano Dimension (NASDAQ:NNDM) and Sona Nanotech (CSE:SONA).

This is an updated version of an article first published by the Investing News Network in 2019.

Dont forget to follow us @INN_Technology or real time updates!

Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.

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What is Nanoscience? | Outlook and How to Invest | INN - Investing News Network

Bioengineering professor Andrew Tsourkas leads the TITAN Lab. (Photo from Andrew Tsourkas)

The National Institutes of Health awarded a Penn Engineering lab and Penn-based startup $667,000 to improve COVID-19 detection technology.

Led by Bioengineering professor Andrew Tsourkas, the Targeted Imaging Therapeutics and Nanomedicine (Titan) Lab partnered with AlphaThera, a startup located at the Pennovation Center, to improve the ELISA technology, which is widely used to detect COVID-19 infections. ELISA, also known as enzyme-linked immunosorbent assay, is used to detect antibodies, proteins the body makes to fight an infection like COVID-19.

By improving ELISA technology, the Titan lab and AlphaThera will enable faster and more sensitive antibody tests for COVID-19 patients. According to the Centers for Disease Control and Prevention, antibody tests may allow patients to determine if they have previously been infected with COVID-19, but do not detect a current COVID-19 infection.

James Hui, who founded AlphaThera alongside Tsourkas, said the company has been working on reducing the time it takes to complete an ELISA assay in detecting antibodies for COVID-19.

These assays can take anywhere between three hours to six hours, but we can do these ELISA assays about probably half the time if not more," said Hui, who received a Ph.D. in Bioengineering from Penn in 2015 and Doctor of Medicine from the Perelman School of Medicine in 2017.

AlphaThera is responsible for developing the ELISA assays, while Tsourkas lab conducts pre-clinical testing for further development of the technology before testing on human samples. After AlphaThera's assays have been validated with the test samples, the team will begin clinical trials in Ping Wang lab at Penn Medicine on about 80 human samples from both COVID-19 and non-COVID-19 patients, Tsourkas said.

The main benefit for the lab is that it shows a practical application for some basic technology we developed in the lab, Tsourkas said. It's always nice to see something turned into a product and be useful for laboratories beyond our own.

Tsourkas said his lab's research also focuses on improving ELISA's sensitivity to detect antibodies, which will help healthcare workers identify a greater number of patients who were previously infected with the virus.

We have some cool antibody labeling technologies that we think will give us a significant boost in sensitivity and how rapid we can read out whether the patient has those antibodies in their serum, he said.

Their goal is to have the technology's validation studies be completed and for kits to be made available to the public within a year, Tsourkas said.

Vaccines imitate an infection while almost never causing illness, prompting the body to produce antibodies and white blood cells that will remember how to fight the disease in the future. Tsourkas said, therefore, fast antibody tests to determine whether a vaccine has prompted the body to produce antibodies will become more important as vaccine development progresses.

As vaccines are being developed now, we need to check to see whether they're working and to see how much of the population was really exposed [to the virus]," Tsourkas said.

Get our newsletter, Dear Penn, delivered to your inbox every weekday morning.

Tsourkas' research can also be applied to other antibody-detecting assays already on the market to improve their speed and sensitivity, he said.

While AlphaThera has been working to make the technology commercially viable and competitive in the market, Hui said their priority is to ensure the assays' speed and sensitivity in detecting antibodies.

We're not trying to be the first to roll off the market and try to get as much sales as possible, he said. Ultimately, we are a technology company [that is] more interested in developing a better product.

More:
Penn lab and startup awarded $667000 NIH grant to improve COVID-19 antibody testing - The Daily Pennsylvanian

SAN DIEGO--(BUSINESS WIRE)--Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announces that recent new contracting with partners and investments in manufacturing capacity have contributed to its Captisol business operating at the highest levels in the history of the technology and position Captisol for major growth. Significant new clinical and regulatory developments with Evomela and Kyprolis, among other drugs, are reinforcing the role the proprietary technology serves in enabling important medicines. During 2020, Ligand has facilitated the successful installation of equipment to allow production at significantly higher levels to support anticipated demand. In addition to manufacturing at partner Hoviones facilities in Ireland and Portugal, Ligand has now added final step processing capacity for Captisol in both the United States and England. Ligand also introduces guidance for 2021 Captisol material sales of approximately $200 million.

The global medical need for Captisol-enabled drugs has never been higher, said John Higgins, Chief Executive Officer of Ligand. Our recently expanded operating team has successfully positioned our Captisol technology for the substantial growth we now expect in 2021 and beyond. There is significant ongoing investment by our partners for over 30 Captisol-enabled medicines in clinical development. We have entered into more contracts this year than any other year and are proud to be working closely with Gilead under our recently extended 10-year supply contract. We continue to be pleased with the momentum relating to Captisol, as it is a critical component in multiple life-saving medicines.

Recent Captisol technology business highlights include the following:

Ligands forecast for 2021 Captisol material sales of approximately $200 million is based on information it has on anticipated demand from its major partners given growth in existing and new markets, clinical requirements for Captisol-enabled development programs and binding orders from certain commercial or pre-commercial partners. The 2021 Captisol outlook compares with the Companys guidance for 2020 Captisol material sales of approximately $90 million.

About Captisol

Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella, University Distinguished Professor at the University of Kansas Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled several FDA-approved products, including Gileads VEKLURY, Amgens KYPROLIS, Baxter Internationals NEXTERONE, Acrotech Biopharma L.L.C.s and CASI Pharmaceuticals EVOMELA, Melinta Therapeutics BAXDELA and Sage Therapeutics ZULRESSO. There are many Captisol-enabled products currently in various stages of development. Ligand maintains a broad global patent portfolio for Captisol with more than 400 issued patents worldwide relating to the technology (including 37 in the U.S.) and with the latest expiration date in 2033. Other patent applications covering methods of making Captisol, if issued, extend to 2040.

About Ligand Pharmaceuticals

Ligand is a revenue-generating biopharmaceutical company focused on developing or acquiring technologies that help pharmaceutical companies discover and develop medicines. Our business model creates value for stockholders by providing a diversified portfolio of biotech and pharmaceutical product revenue streams that are supported by an efficient and low corporate cost structure. Our goal is to offer investors an opportunity to participate in the promise of the biotech industry in a profitable, diversified and lower-risk business than a typical biotech company. Our business model is based on doing what we do best: drug discovery, early-stage drug development, product reformulation and partnering. We partner with other pharmaceutical companies to leverage what they do best (late-stage development, regulatory management and commercialization) to ultimately generate our revenue. Ligands OmniAb technology platform is a patent-protected transgenic animal platform used in the discovery of fully human mono- and bispecific therapeutic antibodies. The Captisol platform technology is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. The Vernalis Design Platform (VDP) integrates protein structure determination and engineering, fragment screening and molecular modeling, with medicinal chemistry, to help enable success in novel drug discovery programs against highly challenging targets. Ab Initio technology and services for the design and preparation of customized antigens enable the successful discovery of therapeutic antibodies against difficult-to-access cellular targets. Icagen has established deep biological expertise focused on ion channels and transporters and has a strong track record in ion channel drug discovery from screening to lead optimization. Ligand has established multiple alliances, licenses and other business relationships with the worlds leading pharmaceutical companies including Amgen, Merck, Pfizer, Sanofi, Janssen, Takeda, Servier, Gilead Sciences and Baxter International. For more information, please visit http://www.ligand.com.

Follow Ligand on Twitter @Ligand_LGND.

Forward-Looking Statements

This news release contains forward-looking statements by Ligand that involve risks and uncertainties and reflect Ligand's judgment as of the date of this release. Words such as plans, believes, expects, anticipates, and will, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding: Ligands expectation that Captisol demand will increase significantly in 2021 and beyond (particularly for sales to Gilead and to partners in Gileads consortium) and Ligands ability to supply Captisol to Gilead and other partners, including Ligands ability to increase supply capacity; the timing of initiation, enrollment and expected results with respect to the planned clinical trial of CE-Iohexol; and guidance regarding Ligands 2020 and 2021 Captisol material sales. Actual events or results may differ from Ligand's expectations due to risks and uncertainties inherent in Ligands business, including, without limitation: Ligand may not receive expected revenue from Captisol sales; the COVID-19 pandemic has disrupted Ligands and its partners business, including delaying manufacturing, preclinical studies and clinical trials and product sales, and impairing global economic activity, all of which could materially and adversely impact Ligands results of operations and financial condition; Ligand may not achieve its Captisol material sales guidance for 2020 and/or 2021; remdesivir may be later shown to not be effective or safe for the treatment of COVID-19 and/or the FDA (and/or equivalent agencies in other countries) may revise or revoke its emergency use authorization for remdesivir for the treatment of COVID-19 in patients hospitalized with moderate or severe disease if the FDA (and/or another such agency) determines that authorization no longer meets the statutory criteria for issuance; alternative COVID-19 therapies or vaccines may be approved or the risk of coronavirus infection could significantly diminish, any of which could materially and adversely affect the commercial opportunity for remdesivir; Gilead may terminate the supply agreement without cause upon 30 days prior written notice; Ligand may be unable to scale-up the supply of Captisol or at acceptable prices; Ligand is currently dependent on Hovione as a single source sole supplier for certain Captisol manufacturing functions and failures by such supplier may result in delays or inability to meet the Captisol demands of its partners; Amgen, Acrotech Biopharma or other Ligand partners may not execute on their sales and marketing plans for marketed products for which Ligand has an economic interest; Ligand or its Captisol partners may not be able to protect their intellectual property and patents covering certain products and technologies may be challenged or invalidated; Ligand's Captisol partners may terminate agreements or development or commercialization of products; Ligand may not generate expected revenues under its existing license agreements and may experience significant costs as the result of potential delays under its supply agreements; Ligand and its Captisol partners may experience delays in the commencement, enrollment, completion or analysis of clinical testing for product candidates, or significant issues regarding the adequacy of clinical trial designs or the execution of clinical trials, which could result in increased costs and delays, or limit the ability to obtain regulatory approval; unexpected adverse side effects or inadequate therapeutic efficacy of Ligand's or its Captisol partners product(s) could delay or prevent regulatory approval or commercialization; and ongoing or future litigation could expose Ligand to significant liabilities and have a material adverse effect on the company. The failure to meet expectations with respect to any of the foregoing matters may reduce Ligand's stock price. Additional information concerning these and other risk factors affecting Ligand can be found in prior press releases available at http://www.ligand.com as well as in Ligand's public periodic filings with the Securities and Exchange Commission available at http://www.sec.gov. Ligand disclaims any intent or obligation to update these forward-looking statements beyond the date of this release. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

a Monahan, et al. Biology of Blood and Marrow Transplantation, September 2020

See the article here:
Ligand Announces its Captisol Business is Positioned for Major Growth and Forecasts 2021 Captisol Material Sales of $200 Million - Business Wire

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

Dear friend,

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

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

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

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

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

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

In kindness,

Amit

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

Original post:
Our genes know kindness is the best medicine - Brainerd Dispatch

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

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

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

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

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

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

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

About Inagene Diagnostics

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

About dnaPower Inc

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

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

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

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

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

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

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

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

The prevalence of HRR alterations found is shown below.

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

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

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

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

Read more from the original source:
ESMO Virtual Congress 2020: Real-World Patterns of Genomic Testing in Patients with Metastatic Castration-R... - UroToday

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

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

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

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

Abstract Title

PresentationType

PresentingAuthor

Date/ Time

ELX/TEZ/IVA

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

ECFS Oral Presentation

Professor Griese

September 24, 2020

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

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

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

NACFC Poster Presentation #447

Professor Fajac

Oct 7 Oct 23, 2020

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

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

NACFC Poster Presentation #478

Professor Majoor

Oct 7 Oct 23, 2020

IVA

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

NACFC Poster Presentation

#466

Professor Kors van der Ent

Oct 7 Oct 23, 2020

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

NACFC Poster Presentation

#415

Dr. Rosenfeld

Oct 7 Oct 23, 2020

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

NACFC Poster Presentation

#141

Dr. Volkova

Oct 7 Oct 23, 2020

LUM/IVA

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

ECFS Published abstract:

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

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

ECFS Published abstract:

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

NACFC Poster Presentation

#190

Dr. Bower

Oct 7 Oct 23, 2020

About Cystic Fibrosis

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

About Vertex

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

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

Special Note Regarding Forward-looking Statements

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

(VRTX-GEN)

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

MIAMI, Sept. 22, 2020 /PRNewswire/ -- The Victor Center for the Prevention of Jewish Genetic Diseases, which offers preconception screening and genetic counseling for prospective parents, has recently expanded services to offer genetic screening and virtual clinical consults to couples and individuals in New York, New Jersey and Maryland.

The center, which also serves Florida, Massachusetts and Pennsylvania, helps future parents identify whether they are at risk of passing on genetic diseases, including those common among people of Jewish ancestry.

"Not everyone knows their full ancestral heritage, so we encourage anyone planning to start a family and their partner to undergo screening," said Debbie Wasserman, Outreach Coordinator/Genetic Counselor for the Victor Center.

Jewish genetic diseases are a group of recessive, inherited disorders that occur with significant frequency in the Ashkenazi Jewish community (those of eastern or central European descent). Individuals of Ashkenazi descent have higher carrier rates for diseases such as Tay-Sachs, Canavan, familial dysautonomia, and Gaucher. Many of the diseases are severe, and some are fatal in childhood.

One in two of those of Ashkenazi descent is a carrier for at least one Jewish genetic condition, and Sephardic and Mizrachi Jews are also at increased risk for certain genetic disorders. More than half of participants in the Victor Center screening program are carriers for one or more of the 225 plus conditions on the expanded screening panel, which also includes disorders found in other ethnicities.

The Victor Center offers a convenient screening process during this time of social distancing. Upon request, a genetic counseling session is scheduled and a screening kit is mailed to the home. Recipients provide saliva samples and return the kit to a lab for processing. Once results are available, a video consult is coordinated to convey understanding and address questions.

There is a fee for Victor Center screening services. However, most medical insurance plans cover the service. For more information, please call 786-897-9587 or visit http://www.victorcenter.org

About the Victor Center The Victor Center was founded in 2002 by Lois B. Victor in partnership with Einstein Healthcare Network in Philadelphia. Ms. Victor lost two children to a Jewish genetic disease before a test for the disorder became available. The experience galvanized her commitment to ensuring that no family endures the heartache of a preventable illness by making certain that Jews of childbearing age are screened and get the information they need to have healthy children. Nicklaus Children's Hospital was appointed the National Office for the Victor Center in 2017. The Nicklaus Children's Hospital Victor Center maintains the nation-wide collaborative work of the center in promoting education related to preconception screening while increasing knowledge, awareness, and access to genetic services.

About Nicklaus Children's HospitalFounded in 1950 by Variety Clubs International, Nicklaus Children's Hospital is South Florida's only licensed specialty hospital exclusively for children, with nearly 800 attending physicians and more than 475 pediatric subspecialists. The 309-bed hospital, known as Miami Children's Hospital from 1983 through 2014, is renowned for excellence in all aspects of pediatric medicine with several specialty programs routinely ranked among the best in the nation by U.S. News & World Report since 2008. The hospital is also home to the largest pediatric teaching program in the southeastern United States and has been designated an American Nurses Credentialing Center (ANCC) Magnet facility, the nursing profession's most prestigious institutional honor. For more information, please visit http://www.nicklauschildrens.org.

For more information: Nicklaus Children's Hospital Rachel Bixby, 305-663-8476

SOURCE Nicklaus Childrens Health System

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Victor Center for the Prevention of Jewish Genetic Diseases Now Serving New York, New Jersey and Maryland - PRNewswire

NEW YORK, Sept. 23, 2020 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today the awarding of 15 new MDA grants totaling more than$4 million toward research focused on a variety of neuromuscular diseases (NMDs), including Duchenne muscular dystrophy (DMD), Charcot-Marie-Tooth disease (CMT), Becker's muscular dystrophy (BMD), spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). This round of grant funding reinforces MDA's unwavering commitmentin the face of declining income due to the COVID-19 pandemicto the progress of neuromuscular disease research and builds on the more than$1 billionMDA has already invested in research to uncover new treatments and cures for NMDs since its inception. Some grants will go into effect this year, while others will be awarded in 2021.

"We continue to fund the most innovative research that will lead us to cures for a range of neuromuscular diseases," saysSharon Hesterlee, PhD, executive vice president and chief research officer for Muscular Dystrophy Association. "We have already seen our investment pay off with the first effective neuromuscular disease therapies, and these grantees are pushing the envelope even further in diseases once thought incurable."

Dr. Hesterlee added, "Although COVID led the cancellation of MDA's spring review session, we are pleased to announce the funding of these projects, which were reviewed in 2019."

The newly funded projects will aim to advance research discoveries and new therapy development in multiple areas. The awarded grants will fund studies to further advance our understanding of genetic causes of and risk factors for NMDs, investigate new approaches to developing gene therapies and other innovative potential treatments, including stopping disease progression and improving genetic testing technologies.

For a complete list of individual awards for this grant cycle, visit MDA's website and explore theGrants at a Glancesection. Highlights from thegrant awards for this grant cycleinclude:

ALS grants will be announced separately later this month, as will grants being given jointly by MDA and other organizations.

About the Muscular Dystrophy AssociationFor 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this throughinnovations in scienceandinnovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures.Research we have supportedis directly linked to life-changing therapies across multiple neuromuscular diseases.MDA's MOVRis the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports thelargest network of multidisciplinary clinicsproviding best in class care at more than 150 of the nation's top medical institutions. OurResource Centerserves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. Each year thousands of children and young adults learn vital life skills and gain independence atsummer campand through recreational programs, at no cost to families.During the COVID-19 pandemic, MDA continues to produce virtual events and programming to support our community when in-person events and activities are not possible. MDA's COVID-19 guidelines and virtual events are posted atmda.org/COVID19. For more information, visitmda.org.

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Muscular Dystrophy Association Awards 15 Grants Totaling More Than $4 Million for Neuromuscular Disease Research - PRNewswire

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Sept. 22, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated(Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD).

PRIME is a regulatory mechanism that provides early and proactive support to developers of promising medicines, to optimize development plans and speed up evaluations so these medicines can reach patients faster. The goal of PRIME is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address an unmet medical need. PRIME designation was granted based on clinical data from CRISPR and Vertexs ongoing Phase 1/2 trial of CTX001 in patients with severe SCD.

About CTX001CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from transfusion-dependent beta thalassemia (TDT) or severe SCD, in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations from the U.S. Food and Drug Administration (FDA), and Orphan Drug Designation from the European Commission, for both TDT and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. CTX001 is the most advanced gene-editing approach in development for TDT and SCD.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the benefits of PRIME designation; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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

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

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding CTX001s PRIME designation or its development, the potential benefits of CTX001, our plans and expectations for our clinical trials and clinical trial sites, and the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and potential outcomes. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Pharmaceuticals Announce Priority Medicines (PRIME) Designation Granted by the European Medicines Agency (EMA) to...

New research suggests that Parkinson's disease is not one but two diseases, starting either in the brain or in the intestines. Which explains why patients with Parkinson's describe widely differing symptoms,. The findings points towards personalized medicine as the way forward for people with Parkinson's disease.

This is the conclusion of a study which has just been published in the leading neurology journalBrain.

The researchers behind the study are Professor Per Borghammer and Medical Doctor Jacob Horsager from the Department of Clinical Medicine at Aarhus University and Aarhus University Hospital, Denmark.

"With the help of advanced scanning techniques, we've shown that Parkinson's disease can be divided into two variants, which start in different places in the body. For some patients, the disease starts in the intestines and spreads from there to the brain through neural connections. For others, the disease starts in the brain and spreads to the intestines and other organs such as the heart," explains Per Borghammer.

He also points out that the discovery could be very significant for the treatment of Parkinson's disease in the future, as this ought to be based on the individual patient's disease pattern.

Parkinson's disease is characterised by slow deterioration of the brain due to accumulated alpha-synuclein, a protein that damages nerve cells. This leads to the slow, stiff movements which many people associate with the disease.

In the study, the researchers have used advanced PET and MRI imaging techniques to examine people with Parkinson's disease. People who have not yet been diagnosed but have a high risk of developing the disease are also included in the study. People diagnosed with REM sleep behaviour syndrome have an increased risk of developing Parkinson's disease.

The study showed that some patients had damage to the brain's dopamine system before damage in the intestines and heart occurred. In other patients, scans revealed damage to the nervous systems of the intestines and heart before the damage in the brain's dopamine system was visible.

This knowledge is important and it challenges the understanding of Parkinson's disease that has been prevalent until now, says Per Borghammer.

"Until now, many people have viewed the disease as relatively homogeneous and defined it based on the classical movement disorders. But at the same time, we've been puzzled about why there was such a big difference between patient symptoms. With this new knowledge, the different symptoms make more sense and this is also the perspective in which future research should be viewed," he says.

The researchers refer to the two types of Parkinson's disease as body-first and brain-first. In the case of body-first, it may be particularly interesting to study the composition of bacteria in the intestines known as the microbiota.

"It has long since been demonstrated that Parkinson's patients have a different microbiome in the intestines than healthy people, without us truly understanding the significance of this. Now that we're able to identify the two types of Parkinson's disease, we can examine the risk factors and possible genetic factors that may be different for the two types. The next step is to examine whether, for example, body-first Parkinson's disease can be treated by treating the intestines with faeces transplantation or in other ways that affect the microbiome," says Per Borghammer.

"The discovery of brain-first Parkinson's is a bigger challenge. This variant of the disease is probably relatively symptom-free until the movement disorder symptoms appear and the patient is diagnosed with Parkinson's. By then the patient has already lost more than half of the dopamine system, and it will therefore be more difficult to find patients early enough to be able to slow the disease," says Per Borghammer.

The study from Aarhus University is longitudinal, i.e. the participants are called in again after three and six years so that all of the examinations and scans can be repeated. According to Per Borghammer, this makes the study the most comprehensive ever, and it provides researchers with valuable knowledge and clarification about Parkinson's disease - or diseases.

"Previous studies have indicated that there could be more than one type of Parkinson's, but this has not been demonstrated clearly until this study, which was specifically designed to clarify this question. We now have knowledge that offers hope for better and more targeted treatment of people who are affected by Parkinson's disease in the future," says Per Borghammer.

According to the Danish Parkinson's Disease Association, there are 8,000 people with Parkinson's disease in Denmark and up to eight million diagnosed patients worldwide.

This figure is expected to increase to 15 million in 2050 due to the ageing population, as the risk of getting Parkinson's disease increases dramatically the older the population becomes.

Reference:

This article has been republished from materials provided by Aarhus University. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Second Variant of Parkinson's Disease That Begins in the Gut Is Identified - Technology Networks