Major depressive disorder (MDD) is a common mental disorder that affects more than 264 million people worldwide and is a leading cause of disability, including death by suicide.1 MDD is a complicated disorder that involves the interaction of social, psychological, and biological factors.1 MDD can prevent patients from living healthy, productive lives and can complicate treatment of other comorbid conditions.1

Although MDD commonly is encountered in primary care settings, its treatment has become integrated into all fields of medicine due to its high prevalence. Cognitive behavioral therapy, interpersonal psychotherapy, and antidepressants, such as selective serotonin reuptake inhibitors and tricyclic antidepressants, are the mainstays of MDD treatment.1

Prescribing an antidepressant may be simple, but that does not make it easy. Efficacy and tolerability of antidepressants vary among patients, which can make it challenging to relieve patients symptoms.2 Although no genes have been associated with depression,3 several genetic variants may help clinicians predict how patients with MDD will metabolize antidepressants.4 Performing genetic testing of patients with MDD and matching patients with an antidepressant class based on identification of genetic variants that convey sensitivity to particular antidepressants could improve response to drug therapy in patients with MDD.5

The process of selecting an antidepressant should take into account cost, tolerability, adverse effect profiles, and patient preferences.2 When evaluating treatment options for patients with MDD, the current standard of care is to initiate an antidepressant at a starting dose and reassess effectiveness within 2 to 4 weeks, with adjustments to monitoring frequency dependent on the patients suicide and self-harm risk, comorbid conditions, age, and concomitant medication use.2

Several metrics are used to determine whether a selected antidepressant is working:

Clinicians can mitigate adverse effects by decreasing the dosage or switching to a different class of antidepressant. However, several weeks are needed after each change in drug or dose alteration to truly assess response. Finding and settling on a drug that produces a response with minimal adverse effects can take months. During the trial period, patients may become frustrated with the process and stop therapy and/or may be at increased risk for suicide or self-harm.

The study of drug metabolism in patients with MDD is a growing area of interest.3-5 A management approach incorporating pharmacogenetic testing in combination with clinical judgment may be superior to the standard trial and error method for finding an effective antidepressant regimen and could improve patient outcomes.5

Genome-wide association studies are used to identify single nucleotide polymorphisms (SNPs) in genes related to a particular disease or drug metabolism.6 Several laboratory testing companies offer pharmacogenetic panels to evaluate metabolism of drugs used to treat MDD.3 The FDA also has approved direct-to-consumer genetic testing panels (eg, 23andMe), which are widely available to the public without a health care providers prescription.7 A concern with these latter tests is that the results of these tests are reported directly from the company to the patient; thus, the patient decides whether or not to present the information to his or her health care provider. Many pharmacogenetic testing panels also include genes that have shown correlations with the pathogenesis of MDD, despite the lack of clinical research replicating the role of these genes in the disorder.3

This article originally appeared on Clinical Advisor

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Pharmacogenetic Testing: Does it Improve Therapy in Patients With MDD? - Neurology Advisor

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Eli Lilly said it planned to discuss with regulators the possible emergency use of baricitinib for hospitalized patients. Other news is about early research on an antibody that might neutralize COVID and how the virus controls the brain, as well.

AP:Anti-Inflammatory Drug May Shorten COVID-19 Recovery TimeA drug company says that adding an anti-inflammatory medicine to a drug already widely used for hospitalized COVID-19 patients shortens their time to recovery by an additional day. Eli Lilly announced the results Monday from a 1,000-person study sponsored by the U.S. National Institute of Allergy and Infectious Diseases. The study tested baricitinib, a pill that Indianapolis-based Lilly already sells as Olumiant to treat rheumatoid arthritis. (Marchione, 9/14)

The Hill:Drugmaker Says Anti-Inflamatory Medicine May Shorten COVID-19 Recovery TimeThe use of Baricitinib, arheumatoidarthritis drug from Eli Lilly, led to a one-day reduction in recovery time for patients when combined with Remdesivir compared to patients who only took Remdesivir, according to a trial. The finding was statistically significant, Eli Lilly said in a statement. The company did not release the full results of the study but stated the National Institute of Allergy and Infectious Diseases (NIAID) is expected to publish full results in peer-review studies and that additional analyses are ongoing to understand clinical outcome data, including safety and morbidity data. (9/14)

In other scientific developments

Fox News:University Of Pittsburgh Scientists Discover Antibody That 'neutralizes' Virus That Causes CoronavirusScientists at the University of Pittsburgh School of Medicine have isolated the smallest biological molecule that completely and specifically neutralizes SARS-CoV-2, the virus that causes the novel coronavirus. The antibody component is 10 times smaller than a full-sized antibody, and has been used to create the drug Ab8, shared in the report published by the researchers in the journal Cell on Monday. The drug is seen as a potential preventative against SARS-CoV-2. (Deabler, 9/14)

Fox News:Coronavirus Can 'Hijack' Brain Cells To Replicate Itself, Yale Researchers DiscoverThe coronavirus can affect the brain and hijack brain cells to replicate itself, Yale University researchers have discovered. A new study from Yale University, on BioRXiv, which is awaiting peer review, found that the brain is another organ susceptible to an attack by the novel coronavirus. (McGorry, 9/14)

Stat:23andMe Research Finds Possible Link Between Blood Type And Covid-19A forthcoming study from genetic testing giant 23andMe shows that a persons genetic code could be connected to how likely they are to catch Covid-19 and how severely they could experience the disease if they catch it. Its an important confirmation of earlier work on the subject. People whose blood group is O seemed to test positive for Covid-19 less often than expected when compared to people with any other blood group, according to 23andMes data; people who tested positive and had a specific variant of another gene also seemed to be more likely to have serious respiratory symptoms. (Sheridan, 9/14)

Read more:
Drug Company Touts Anti-Inflammatory Drug's Role In Shortening COVID Recovery - Kaiser Health News

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CHICAGO, Sept. 16, 2020 /PRNewswire/ -- BCS Financial (BCS) announces today it has partnered with 2nd.MD to provide top-tier medical navigation, expert consultation, and personalized second opinions to four million policyholders. Under the agreement, members covered under BCS Stop Loss and Excess of Loss policies can work with highly skilled, experienced nurses to help them navigate diagnoses, virtually connect with elite doctors who specialize in their specific condition, and receive assistance finding a local doctor or specialist in their area at no cost to them.

As the number one reinsurer of Blue Cross Blue Shield plans, BCS offers a wide range of products and services to help insurance carriers and their members navigate healthcare costs, particularly those exceeding $500,000 annually. The partnership is one of several features included in BCS Financial's Risk Navigator solutions, a highly predictive and actionable approach to managing claims risk.

"Second opinions can play a vital part in ensuring healthcare costs are not wasted on unnecessary or incorrect care," said Peter Costello, presidentand chief executive officer, BCS Financial. "BCS is committed to helping our policyholders and their members manage their risks while improving healthcare outcomes."

Members see direct benefit from the second-opinion consultation. "Members facing a complex diagnosis, surgeryor healthcare decision often find it difficult to navigate the healthcare system and manage costs," said Chris Kurtenbach, vice president, Services & Operations, BCS Financial. "With this partnership, we can help members get the appropriate care they need by connecting them with board-certified, elite specialists from top medical institutions, and reducing their costs along the way."

In many cases, the second opinion can change the treatment trajectory 85% of 2nd.MD's consultations result in improved treatment plans, 35% lead to alternative diagnoses, and nearly 30% of surgery consults result in surgery cancellation.

This is an especially important service as the market for gene therapy and cellular immunotherapy continues to ramp up. An estimated 50-75 therapies will be approved for the U.S. market by 2030, with costs for cellular immunotherapies averaging around $400,000, and gene therapy costs estimated in the $2-$4 million range. For those with complex cancers, rare or orphan conditions, and aggressive recommendations, access to top specialists can make all the difference.

"Better care and outcomes for members is always our priority," said Costello. "This partnership is just one of the ways we are delivering value to our Blue Cross and Blue Shield partners and their members by helping them continue to manage healthcare costs and quality."

This service is currently available to members covered by BCS Stop Loss and Excess of Loss policies.

About BCS Financial Corporation (BCS)

BCS Financial Corporation has more than 70 years of experience delivering a wide-range of insurance and financial solutions for Blue Cross and Blue Shield organizations nationwide. Through its subsidiaries, BCS Insurance Company and 4 Ever Life Insurance Company, BCS is licensed in all 50 states and is rated A- (Excellent) by A.M. Best. BCS Financial is headquartered in Oakbrook Terrace, Illinois. Learn more at bcsf.com.

About 2nd.MD

2nd.MD offers medical certainty by connecting you with board-certified, leading specialists from across the country for an expert second opinion via video or phone within three to five days, from the comfort of your own home. From the minute you reach out, 2nd.MD's Care Team of nurses will put you at the center of your medical care by coordinating all the details. This includes understanding your health goals, gathering all of your medical records, and connecting you with a doctor who specializes in your specific medical condition, so that you can focus on what matters most getting the best care possible. No travel. No hassle. No cost. If you haven't used 2nd.MD before, it can be life-changing. In fact, 98% of people who use 2nd.MD recommend it to their friends and family.

For more information, please contact Nathan Post at 630-472-7860.

SOURCE BCS Financial Corporation

Link:
BCS Announces Partnership With 2nd.MD to Improve Member Healthcare Cost, Quality, and Outcomes - PRNewswire

Recommendation and review posted by Bethany Smith

IRVINE, Calif. & BOSTON--(BUSINESS WIRE)--Novus Therapeutics, Inc. (Novus) (NASDAQ: NVUS) today announced it has completed the acquisition of Anelixis Therapeutics, Inc. (Anelixis), a privately held clinical stage biotechnology company developing a next generation anti-CD40 Ligand (CD40L) antibody as a potential treatment for organ and cellular transplantation, autoimmune diseases, and neurodegenerative diseases. Concurrent with the acquisition of Anelixis, Novus entered into a definitive agreement for the sale of non-voting convertible preferred stock (the Preferred Stock) in a private placement to a group of institutional accredited investors led by BVF Partners L.P., with participation from Cormorant Asset Management, Ecor1 Capital, Logos Capital, Fidelity Management and Research Company, Adage Capital Partners L.P., Woodline Partners LP, Ridgeback Capital, Janus Henderson Investors, and Samsara BioCapital, as well as additional investors. The private placement is expected to result in gross proceeds to Novus of approximately $108 million before deducting placement agent and other offering expenses. The proceeds from the private placement will be used to fund the Companys operations, including to advance Phase 2 clinical trials of AT-1501, a humanized IgG1 anti-CD40L antibody with high affinity for CD40L, in renal transplantation, islet cell transplantation, autoimmune nephritis, and amyotrophic lateral sclerosis (ALS).

We are excited about AT-1501 and the potential to develop and commercialize the next generation anti-CD40L antibody, a well-validated target with broad therapeutic possibilities, said Keith A. Katkin, Chairman of the Board of Directors of Novus. After exploring a range of strategic options to maximize shareholder value, we believe this acquisition represents the greatest value creation opportunity for Novus stockholders, and we are confident that we have the management and scientific leadership team to fully realize this opportunity for patients in need of new treatment options.

Leadership & Organization

In addition to the strategic acquisition and private placement, Novus announced its Board of Directors has previously appointed David-Alexandre DA C. Gros, M.D. to serve as Chief Executive Officer and Director. Dr. Gros joins Novus from Imbria Pharmaceuticals Inc., where he served as Co-Founder, Chief Executive Officer and Director. Prior to Imbria, Dr. Gros was President and Chief Operating Officer of Neurocrine Biosciences, Inc., Chief Business and Principal Financial Officer of Alnylam Pharmaceuticals, Inc., and Chief Strategy Officer of Sanofi, S.A. Before Sanofi, Dr. Gros held leadership positions in healthcare investment banking at Centerview Partners, LLC, and Merrill Lynch, Pierce, Fenner & Smith, Inc., and in healthcare consulting at McKinsey & Company. Dr. Gros earned a Doctor of Medicine from Johns Hopkins University School of Medicine, a Master of Business Administration from Harvard Business School, and a Bachelor of Arts from Dartmouth College.

I am both thrilled and humbled to join the Novus management team and Board during this new phase of the companys evolution, as we prepare to initiate multiple Phase 2 trials for AT-1501 said Dr. Gros. Through this acquisition and financing, we now have the scientific, organizational and financial resources to build upon a deep historical understanding of the CD40/CD40L pathway, as well as Anelixis preclinical and Phase 1 data, to address the needs of people undergoing organ or cellular transplantation, or living with autoimmune and neurodegenerative diseases.

Joining Dr. Gros on the Novus management team and Board of Directors is Steven Perrin, Ph.D., Founder and Chief Executive Officer of Anelixis, who will take on the role of President and Chief Scientific Officer. Dr. Perrin brings 20 years of drug development experience to Novus, having held R&D positions at the Hoechst-Ariad Genomics Center, Aventis Pharmaceuticals, Inc., and Biogen Idec, Inc. Over the past decade, Dr. Perrin has worked with the ALS Therapy Development Institute to develop the worlds largest ALS drug development program, bridging preclinical and clinical programs. Dr. Perrin received a Ph.D. in biochemistry from Boston University Medical Center, where he also started his career as Associate Professor of Medicine, and a Bachelor of Science from Boston College.

The activation of CD40/CD40L signaling is critical to mediating antibody and cellular inflammatory response. We are developing antibodies to inhibit the activation of this pathway with the hope of offering new treatment modalities for people living with conditions such as autoimmune nephritis and ALS, or those requiring a potentially life-saving transplant, said Dr. Perrin. I have dedicated my career to developing better medicines for these patients and their families, and I look forward to working with the team to advance these clinical programs.

Concurrent with the acquisition, former Anelixis Chairman of the Board Walter Ogier has been appointed to the Novus Board of Directors. Mr. Ogier has more than 30 years of experience developing therapeutic medical products ranging from pharmaceuticals to medical devices, stem and immune cell therapies, and gene therapies. He has served in multiple CEO roles including Genetix Pharmaceuticals, Inc. (now bluebird bio, Inc.) and Acetylon Pharmaceuticals, Inc., which Celgene Corporation acquired in 2016. In addition to Novus, he serves as a director of Biothera Pharmaceuticals, Inc., Thetis Pharmaceuticals, LLC, and Nemucore Medical Innovations, Inc., and as Board advisor to Kodikaz Therapeutic Solutions, Inc., and ME Therapeutics, Inc.

Novus Board members will also include Keith A. Katkin, Chairman of the Board; Gary A. Lyons; and John S. McBride. The company will continue to maintain its executive offices in Irvine, Calif. and will have research and development facilities in Boston, Mass.

About the Transactions

The acquisition of Anelixis was structured as a stock-for-stock transaction whereby all of Anelixis outstanding equity interests were exchanged in a merger for a combination of shares of Novus common stock and shares of Preferred Stock. Concurrently with the acquisition of Anelixis, Novus entered into definitive agreements for a PIPE investment with existing and new investors to raise approximately $108 million in which the investors will be issued shares of Preferred Stock at a price of approximately $500 per share (or, $0.50 per share on an as-converted-to-common basis). The PIPE offering is expected to close on September 14, 2020. Subject to stockholder approval, each share of Preferred Stock will, at the option of the holder, be convertible into 1,000 shares of common stock, subject to certain beneficial ownership limitations set by each holder. The acquisition was approved by the Board of Directors of Novus and the equity holders of Anelixis.

Ladenburg Thalmann & Co. Inc. is serving as exclusive financial advisor and Gibson, Dunn & Crutcher LLP is serving as legal counsel to Novus. Goodwin Procter LLP is serving as legal counsel to Anelixis. SVB Leerink is serving as financial advisor and lead placement agent for the private placement, and Noble Life Science Partners, a division of Noble Capital Markets, Inc., is acting as co-placement agent.

Additional details are available in an updated corporate presentation that can be found online at http://www.novustherapeutics.com.

Webcast Details

Novus will host an audio webcast on Tuesday, September 15, 2020, at 8:30 a.m. EDT to discuss the acquisition. The live audio webcast will be accessible through a direct link and the investor section of http://www.novustherapeutics.com. To access via phone, please dial (833) 614-1390 (toll-free) or (914) 987-7111 (international) and provide the conference ID 4046285. Please visit the investor section of the Novus website at http://www.novustherapeutics.com for the archived webcast and for more information on the acquisition.

About AT-1501

AT-1501 is a humanized IgG1 anti-CD40L antibody with high affinity for CD40L, a well-validated target with broad therapeutic potential. The CD40/CD40L pathway plays a central role in generating pro-inflammatory responses in autoimmune disease, allograft transplant rejection, and neuroinflammation. In a Phase 1 safety study of healthy volunteers and patients with ALS, AT-1501 was well tolerated at all doses tested.

About Novus Therapeutics

Novus Therapeutics, Inc. is a clinical stage biotechnology company using its expertise in targeting the CD40L pathway to develop potential treatments for people requiring an organ or cell-based transplant, and for people with autoimmune and neurodegenerative disease. Novus is headquartered in Irvine, Calif. For more information, please visit the companys website at http://www.novustherapeutics.com.

Follow Novus Therapeutics on social media: @Novus_Thera and LinkedIn.

Notice of Issuance of Inducement Grants

Pursuant to their employment agreements, Drs. Gros and Perrin have been awarded options to purchase a total of 18,279 and 7,857 shares of Preferred Stock, respectively, subject to time-based vesting (the "Inducement Grants"). The Inducement Grants have an exercise price of $500 per share of Preferred Stock, which is equal to the price at which the Preferred Stock is being offered and sold in the PIPE financing and represents (on an as-converted basis) a premium of approximately 30% over the last reported closing price of the Novus common stock prior to grant. The Inducement Grants have been approved by the Novus Board of Directors and the Compensation Committee of the Board of Directors. The Inducement Grants will be issued outside of the Company's stockholder-approved equity incentive plans as an inducement grant in accordance with Nasdaq Listing Rule 5635(c)(4).

Forward-Looking Statements

This press release contains forward-looking statements that involves substantial risks and uncertainties. Any statements about the companys future expectations, plans and prospects, including statements about its strategy, future operations, development of its product candidates, and other statements containing the words believes, anticipates, plans, expects, estimates, intends, predicts, projects, targets, looks forward, could, may, and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, although not all forward-looking statements include such identifying words. Forward-looking statements include, but are not limited to statements regarding: risks related to market conditions; expectations regarding the timing for the commencement of future clinical trials; expectations regarding the success of clinical trials; the rate and degree of market acceptance and clinical utility of the companys products; the companys estimates regarding expenses and cash runway; and the impact of the ongoing coronavirus pandemic. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the companys actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Q, annual 10-K, and other filings with the SEC, which can be found at http://www.sec.gov. Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise.

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Novus Therapeutics Announces Acquisition of Anelixis Therapeutics - Business Wire

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Helixmith said Wednesday that it has submitted a phase 3-3 extension study protocol of its gene therapy Engensis (VM202) for diabetic peripheral neuropathy (DPN), to the U.S. Food and Drug Administration.

The company set a one-year follow-up period to confirm the pain reduction and safety of VM202 in treating DPN. DPN is one of the most common complications of diabetic diseases. About 30 million Americans have diabetes, and 28.5 percent of them develop DPN. Among the DPN patients, 40 to 50 percent experience painful symptoms.

The study's primary endpoint is the average pain reduction effect measured and recorded in the pain diary over the last week of the sixth month from the first injection.

The study will be carried out with patients who had not taken Gabapentinoids, such as Pregabalin and Gabapentine, in 15 research laboratories across the U.S., including Northwestern University in Chicago.

"Existing painkillers for DPN patients are not a fundamental treatment for the disease as they only relieved pain while often accompanying serious side effects and high addiction," Helixmith CEO Kim Sun-young said. "We will try our best for the success of phase 3-3 clinical trials as well as the ongoing phase 3-2 study."

FDA recognized the clinical results of Engennsis and designated it as an advanced regenerative medicine advanced therapy (RMAT) in 2018, the company said. RMAT is a new system designed to speed up the development and approval of innovative regenerative therapies. It gives special privileges of the U.S. fast track and priority or accelerated screening.

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Helixmith eyeing P3-3 clinical trials of gene therapy Engensis - Korea Biomedical Review

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BOSTON, Sept. 15, 2020 /PRNewswire/ --GreenLight Bioscienceshas received a $3.3 million grant from the Bill & Melinda Gates Foundation to develop new mRNA-based gene therapies for Sickle Cell Disease and other global health challenges.

The funding will support GreenLight's research and testing of affordable therapies using the company's novel messenger RNA (mRNA) approach to gene editing. mRNA technology is already being used to develop vaccine candidates for infectious diseases, including the COVID-19 pandemic.

While initial research will focus on a cure for Sickle Cell Disease, GreenLight plans to develop a versatile gene editing platform to address a variety of diseases affecting underserved patient populations, such as treating HIV in developing countries.

Sickle Cell Disease is a group of inherited blood disorders in which red blood cells develop abnormally, causing pain and anemia. More than 4 million people currently suffer from the disease, with another 40+ million having the sickle cell trait, which can be passed on to future generations. The disease primarily targets people of African, Hispanic, or Middle Eastern descent. Current treatment regimens including blood transfusions and bone marrow transplants are costly, invasive, and impractical for treating large segments of affected patient populations.

"Funders are recognizing the potential of our innovative approach to gene editing that, in combination with our proprietary RNA manufacturing capability, has the potential to deliver accessible gene therapies and improve human health globally," said Marta Ortega-Valle, senior vice president of Human Health and Corporate Development at GreenLight Biosciences. "Finding a safe and effective therapy is critical, but equally important is the ability to produce it affordably for broader access. We are grateful for the Gates Foundation's support to advance novel gene editing approaches for populations in which those therapies are currently out of reach."

Gene editing therapies hold significant promise in the treatment of Sickle Cell Disease since it is a disorder caused by gene mutation. Using RNA as its core, GreenLight Biosciences is working to develop an in vivo gene therapy that could ultimately offer a cure to the disease.

Once the therapy candidate is validated and moves into clinical use, GreenLight Biosciences' biomanufacturing platform will accelerate production of affordable treatments at scale. "Manufacturing sufficient quantities of high-quality RNA at an accessible cost is critical for achieving the full potential of new therapies that aim to reach a global patient population. That capability does not yet exist in the market, but GreenLight's end-to-end, self-contained manufacturing platform aims to make that possible for all mRNA-based therapies and vaccines," Ortega-Valle added.

About GreenLight Biosciences, Inc.GreenLight is a bio-performance company with a unique, cell-free production platform that delivers high-performing RNA solutions to human, plant and animal challenges. GreenLight develops RNA products for plant and life science applications, and collaborates with industry leaders to advance vaccine development, pandemic preparation, crop management, and plant protection. The cutting-edge, natural platform delivers higher-quality RNA at a lower cost and higher speed than was ever before possible. The GreenLight team values diversity, inclusion, and equality and promises to use collaboration to remain scientifically imaginative and passionately focused on making a difference in the world. For more information, visithttps://www.greenlightbiosciences.com/.

SOURCE GreenLight Biosciences

Originally posted here:
GreenLight Biosciences Receives $3.3 Million Grant to Develop Sickle Cell Disease Cure Using mRNA Gene Therapy - PRNewswire

Recommendation and review posted by Bethany Smith

Moderna has agreed to separate deals with Vertex and Chiesi for research and development spanning cystic fibrosis (CF) therapies and pulmonary arterial hypertension (PAH).

In its agreement with Vertex, Moderna will receive $75m to collaborate on the research and development into gene therapies for CF. As part of the deal, Moderna will discover and develop lipid nanoparticles (LNPs) and mRNAs for the delivery of gene-editing therapies for the treatment of CF.

Under the terms of the deal, Moderna will receive $75m upfront to conduct research activities to discover and develop LNPs for gene-editing CF therapies. The US-based biotech company will also be eligible to receive up to $380m in further development, regulatory and commercial milestone payments, as well as tiered royalties on any products that come out of the collaboration.

While Moderna will be responsible for the initial discovery and manufacturing of LNPs and mRNA constructs for the gene-editing treatments, Vertex will be responsible for providing other components of the therapies to be formulated into LNPs, as well as the preclinical and clinical development and commercialisation efforts of any potential candidates.

This is the second agreement reached between Moderna and Vertex, with the previous collaboration aimed at the discovery and development of mRNA therapeutics for CF having been recently extended.

Our first collaboration with Vertex to deliver mRNA coding for cystic fibrosis protein in lung cells is advancing well and this second collaboration aims at using Modernas technologies to explore the use of gene editing in lung cells, said Stphane Bancel, chief executive officer of Moderna.

Prior to announcing the Vertex deal, Moderna revealed that it had come to a separate agreement with Italian-domiciled pharma company Chiesi, for the development of mRNA treatments for PAH.

As part of that deal, Moderna will receive $25m upfront as well as a potential $400m in milestone payments to conduct research and development activities in this therapy area.

PAH is a rare and progressive disorder, which occurs when arteries in the lungs constrict, which forces the heart to work harder, often causing heart failure. The condition affects around two to five million adults across the globe, and remains an area of high unmet medical need.

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Moderna inks R&D agreements with Vertex and Chiesi - PMLiVE

Recommendation and review posted by Bethany Smith

Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that it has submitted the Marketing Authorisation Application (MAA) for its lead product LUMEVOQ to the European Medicines Agency (EMA), seeking approval for the treatment of patients with vision loss due to Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene.

This first regulatory submission for GenSight is a major milestone in our progression from a pure research organization to one with commercial capabilities. It validates a technology platform that has the potential to address the high unmet medical needs of patients suffering from a range of rare diseases. I would like to thank all GenSight employees and partners whose motivation, focus and effort made this submission possible, said Bernard Gilly, Co-founder and Chief Executive Officer of GenSight Biologics.

LHON is a rare, mitochondrial genetic disease, mainly affecting young males. The ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There continues to be a high unmet medical need for the 800-1200 new LHON patients in Europe and the U.S. each year, particularly those who are struck blind in their prime working years.

Lenadogene nolparvovec (tradename: LUMEVOQ) is a recombinant adeno-associated viral vector, serotype 2 (rAAV2/2), containing a cDNA encoding the human wild-type mitochondrial NADH dehydrogenase 4 protein (ND4), which has been specifically developed for the treatment of LHON associated with mutation in the ND4 gene. It received orphan drug designation status for the treatment of LHON from the EMA in 2011 and from the U.S. Food and Drug Administration (FDA) in 2013.

GenSight submitted the MAA based on the benefit-risk balance established by results from a Phase-I/IIa study (CLIN-01), two pivotal Phase-III efficacy studies (CLIN-03A: RESCUE, and CLIN-03B: REVERSE) and the long-term follow up study of RESCUE and REVERSE (CLIN 06 readout at Year 3 post injection). To demonstrate the efficacy of LUMEVOQ in the context of a contralateral effect, the Company used a statistics-based indirect comparison methodology to assess the visual outcomes in LUMEVOQ-treated patients (from LUMEVOQ efficacy studies) against those in untreated patients from Natural History studies and GenSights REALITY Natural History Registry.

GenSight expects to submit the Biologics License Application (BLA) for LUMEVOQ to the FDA in H2 2021. First-in-human data from GenSights second clinical stage program, GS030, are expected to be available in H2 2021.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQ (GS010; lenadogene nolparvovec), is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to the eye by intravitreal injection to offer patients a sustainable functional visual recovery.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200914005857/en/

GenSight BiologicsChief Financial Officer

Thomas Gidointgidoin@gensight-biologics.com+33 (0)1 76 21 72 20

LifeSci AdvisorsInvestor Relations

Guillaume van Renterghem

gvanrenterghem@lifesciadvisors.com+33 (0)6 69 99 37 83

RooneyPartnersMedia Relations

Marion Janic

mjanic@rooneyco.com+1-212-223-4017

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James Palmerj.palmer@orpheonfinance.com+33 (0)7 60 92 77 74

Excerpt from:
GenSight Biologics Submits EU Marketing Authorisation Application for LUMEVOQ Gene Therapy to Treat Vision Loss Due to Leber Hereditary Optic...

Recommendation and review posted by Bethany Smith

David Dismuke, Ph.D., joins as Chief Technical Officer, Christopher Shilling as Vice President of Regulatory Affairs and Quality Assurance, and Christina Perry as Vice President of Finance and Operations.All three leaders will be in place in September and join Forge's executive leadership team.

"The addition of David, Chris, and Christina to the Forge leadership team brings immeasurable value to the Forge team and expands our team of gene therapy experts," said Timothy J. Miller, Ph.D., Forge's Co-Founder, President & CEO. "We believe that our accomplished and passionate team, with demonstrated experience in manufacturing and developing gene therapy products, is the foundation of fulfilling our mission to accelerate transformative medicines to reach those who need them most."

David Dismuke joins Forge with more than 15 years of experience in large-scale manufacturing. He is an authority in the bioprocessing and design of gene therapy vectors and has led CMC operations in the large-scale manufacturing of pre-clinical and clinical-grade AAV vectors for more than 10 years. Prior to joining Forge, David was the Vice President of Manufacturing at StrideBio where he directed the development of manufacturing and analytical processes for AAV vectors that utilize novel capsids. In addition, he led the design of therapeutic and reporter transgenes and innovative molecular enhancements to improve AAV production and therapeutic function.He was also previously the Head of Vector Production at Voyager Therapeutics where he led teams in the manufacturing and analytical testing of AAV using the baculovirus/Sf9 production system.Prior to Voyager, David was the Director of the UNC Vector Core, where he oversaw GMP operations as well as the production of research-grade vectors.He earned his PhD from Vanderbilt University, focusing on the molecular biology and lifecycle of HIV-1, and then performed his postdoctoral research at UNC Chapel Hill.

Christopher Shilling joins Forge as Vice President of Regulatory Affairs and Quality. He has over 15 years of experience in development of novel gene therapies for rare and severe disorders. Christopher is an experienced leader in gene therapy regulatory affairs, pharmacology, toxicology, and project management focused on developing strategies for early phase clinical trials in support of a variety of transformative therapeutics for pediatric and rare diseases. Prior to joining Forge, Christopher started the Drug and Device Development program at Nationwide Children's Hospital which was instrumental in gaining acceptance from regulators for over twenty first-in-human gene therapy clinical trials of novel biologic products, a dozen orphan drug designations, and two fast track designations. He received his Bachelor of Science degree in biology and a Master of Science degree in Pathology both from the Ohio State University.

Christina Perry joins Forge Biologics as the Vice President of Finance and Operations. She spent the last several years as the CFO of Drive Capital where she built out all accounting and finance operations, back-office needs, and investor relations and reporting. Christina managed complex entity legal structures and has had extensive exposure to equity and debt financings.Over the last 15+ years Christina has worked with public and private companies and startups across varying industries, developing operational processes to scale with high growth. She began her career at Deloitte, is a licensed CPA in the state of Ohio, earned her Bachelor of Business Administration at the University of Notre Dame and a Master of Accountancy from Miami University.

These additions to the Forge Biologics leadership team continue Forge's momentum within the biotechnology industry in Columbus Ohio, bringing positive impact to both Ohio and the global rare disease community.

About Forge BiologicsThe mission of Forge Biologics is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft2 facility in Columbus, Ohio, referred to as "The Hearth", to serve as their headquarters. The Hearth will be home to a custom-designed cGMP facility, dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

For more information, please visit https://www.forgebiologics.com.

Media Inquiries:Dan SalvoForge Biologics Director of Communications and Community Development[emailprotected]

Business Inquiries:Erandi De Silva, Ph.D.Forge BiologicsCo-Founder and Vice President of Product Development[emailprotected]

SOURCE Forge Biologics

https://forgebiologics.com

The rest is here:
Forge Biologics Strengthens Gene Therapy Leadership with Key Hires in AAV Manufacturing, Regulatory Affairs and Finance to Support Strategic Growth -...

Recommendation and review posted by Bethany Smith

When a researchers talk about gene editing, theyre usually thinking about several steps. First you need to zero in on the defective gene; then, depending on the need, youd want to knock out, replace or insert genetic material.

CRISPR/Cas9 technologies have transformed the field by making a breakthrough for the first problem. Inducing double-stranded DNA breaks, or achieving single-letter changes as base editing allows, have promising applications in multiple diseases that are starting to get tested in humans.

But Versant Ventures, one of the pioneering investors in the space, still sees a gap. And theyre teaming up with a group of prominent Stanford researchers plus a seasoned biotech exec to fill it.

Achieving high-efficiency targeted gene integration has been a critical objective of gene editing for more than 15 years, but only now is this technologically possible, Jerel Davis, Versants Vancouver-based managing director, said.

Graphite Bio has $45 million to start things off, but the money matters little when compared to the rich brain bank its drawing from.

Matthew Porteus, an academic founder of CRISPR Therapeutics, is lending the tech platform to create this next-gen play alongside gene therapy expert Maria Grazia Roncarolo.

Drawing from research work led by Danny Dever while a postdoc at Porteus lab, Graphites big promise is to increase integration efficiency from less than 1% to greater than 50% across diverse genetic lesions in a wide range of cell types.

Specifically, Dever and Porteus showed that they could correct the sickle globin gene in patient-derived hematopoietic stem cells ex vivo by combining Cas9 ribonucleoproteins with a donor molecule that serves as a template, delivered in recombinant adeno-associated viral vectors of serotype 6 (rAAV6).

That could make for a much more scalable replacement for transplants, they wrote in a 2017 paper describing mouse studies.

Notably, we devise an enrichment paradigm to purify a population of HSPCs with >90% targeted integration, they wrote. We also show efficient correction of the SCD-causing E6V mutation in patient-derived HSPCs that after differentiation into erythrocytes, express adult -globin (HbA) mRNA, confirming intact transcriptional regulation of edited HBB alleles.

Chief executive Josh Lehrer is leading the charge to start Phase I for this program in early 2021, bringing all the sickle cell knowledge and experience from a six-year run as Global Blood Therapeutics, most recently as CMO. More preclinical therapies are in the works for unnamed serious diseases. Samsara BioCapital is joining Versant for the launch round and sending Abe Bassan to the board, which also features Davis and Carlo Rizzuto from Versant.

Read more here:
Versant teams up with Stanford gene editing experts on a $45M next-gen play marrying CRISPR and AAV to fix sickle cell - Endpoints News

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Viral Vector & Plasmid DNA Manufacturing Market Research Report by Type, by Application, by End User - Global Forecast to 2025 - Cumulative Impact of COVID-19" report has been added to ResearchAndMarkets.com's offering.

The Global Viral Vector & Plasmid DNA Manufacturing Market is expected to grow from USD 551.56 Million in 2019 to USD 2,101.82 Million by the end of 2025 at a Compound Annual Growth Rate (CAGR) of 24.97%.

This research report categorizes the Viral Vector & Plasmid DNA Manufacturing to forecast the revenues and analyze the trends in each of the following sub-markets:

The FPNV Positioning Matrix evaluates and categorizes the vendors in the Viral Vector & Plasmid DNA Manufacturing Market on the basis of Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. The Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth.

Cumulative Impact of COVID-19:

COVID-19 is an incomparable global public health emergency that has affected almost every industry so far and the long-term effects projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlaying COVID-19 issues and potential paths forward. The report is delivering insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecast, considering the COVID-19 impact on the market.

The report provides insights on the following pointers:

1. Market Penetration: Provides comprehensive information offered by the key players

2. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets

3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments

4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players

5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments

The report answers questions such as:

1. What is the market size and forecast of the Global Viral Vector & Plasmid DNA Manufacturing Market?

2. What are the inhibiting factors and impact of COVID-19 shaping the Global Viral Vector & Plasmid DNA Manufacturing Market during the forecast period?

3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Viral Vector & Plasmid DNA Manufacturing Market?

4. What is the competitive strategic window for opportunities in the Global Viral Vector & Plasmid DNA Manufacturing Market?

5. What are the technology trends and regulatory frameworks in the Global Viral Vector & Plasmid DNA Manufacturing Market?

6. What are the modes and strategic moves considered suitable for entering the Global Viral Vector & Plasmid DNA Manufacturing Market?

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/w76mxp

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Global Viral Vector & Plasmid DNA Manufacturing Market Analysis and Forecasts to 2025 - Cumulative Impact of COVID-19 - ResearchAndMarkets.com -...

Recommendation and review posted by Bethany Smith

More than 1 million Americans are diagnosed with a chronic brain disorder each year, yet effective treatments for most complex brain disorders are inadequate or even nonexistent.

A major new research effort at the McGovern Institute for Brain Research at MIT aims to change how we treat brain disorders by developing innovative molecular tools that precisely target dysfunctional genetic, molecular, and circuit pathways.

The K. Lisa Yang and Hock E. Tan Center for Molecular Therapeutics in Neuroscience was established at MIT through a $28 million gift from philanthropist Lisa Yang and MIT alumnus Hock Tan 75. Yang is a former investment banker who has devoted much of her time to advocacy for individuals with disabilities and autism spectrum disorders. Tan is president and CEO of Broadcom, a global technology infrastructure company.This latest gift brings Yang and Tans total philanthropy to MIT to more than $72 million.

In the best MIT spirit, Lisa and Hock have always focused their generosity on insights that lead to real impact," says MIT President L. Rafael Reif. Scientifically, we stand at a moment when the tools and insights to make progress against major brain disorders are finally within reach. By accelerating the development of promising treatments, the new center opens the door to a hopeful new future for all those who suffer from these disorders and those who love them. I am deeply grateful to Lisa and Hock for making MIT the home of this pivotal research.

Engineering with precision

Research at the K. Lisa Yang and Hock E. Tan Center for Molecular Therapeutics in Neuroscience will initially focus on three major lines of investigation: genetic engineering using CRISPR tools, delivery of genetic and molecular cargo across the blood-brain barrier, and the translation of basic research into the clinical setting. The center will serve as a hub for researchers with backgrounds ranging from biological engineering and genetics to computer science and medicine.

Developing the next generation of molecular therapeutics demands collaboration among researchers with diverse backgrounds, says Robert Desimone, McGovern Institute director and the Doris and Don Berkey Professor of Neuroscience at MIT. I am confident that the multidisciplinary expertise convened by this center will revolutionize how we improve our health and fight disease in the coming decade. Although our initial focus will be on the brain and its relationship to the body, many of the new therapies could have other health applications.

There are an estimated 19,000 to 22,000 genes in the human genome and a third of those genes are active in the brain the highest proportion of genes expressed in any part of the body. Variations in genetic code have been linked to many complex brain disorders, including depression and Parkinsons disease. Emerging genetic technologies, such as the CRISPR gene editing platform pioneered by McGovern Investigator Feng Zhang, hold great potential in both targeting and fixing these errant genes. But the safe and effective delivery of this genetic cargo to the brain remains a challenge.

Researchers within the new Yang-Tan Center will improve and fine-tune CRISPR gene therapies and develop innovative ways of delivering gene therapy cargo into the brain and other organs. In addition, the center will leverage newly developed single-cell analysis technologies that are revealing cellular targets for modulating brain functions with unprecedented precision, opening the door for noninvasive neuromodulation as well as the development of medicines. The center will also focus on developing novel engineering approaches to delivering small molecules and proteins from the bloodstream into the brain. Desimone will direct the center and some of the initial research initiatives will be led by associate professor of materials science and engineering Polina Anikeeva; Ed Boyden, the Y. Eva Tan Professor in Neurotechnology at MIT; Guoping Feng, the James W. (1963) and Patricia T. Poitras Professor of Brain and Cognitive Sciences at MIT; and Feng Zhang, James and Patricia Poitras Professor of Neuroscience at MIT.

Building a research hub

My goal in creating this center is to cement the Cambridge and Boston region as the global epicenter of next-generation therapeutics research. The novel ideas I have seen undertaken at MITs McGovern Institute and Broad Institute of MIT and Harvard leave no doubt in my mind that major therapeutic breakthroughs for mental illness, neurodegenerative disease, autism, and epilepsy are just around the corner, says Yang.

Center funding will also be earmarked to create the Y. Eva Tan Fellows program, named for Tan and Yangs daughter Eva, which will support fellowships for young neuroscientists and engineers eager to design revolutionary treatments for human diseases.

We want to build a strong pipeline for tomorrows scientists and neuroengineers, explains Hock Tan. We depend on the next generation of bright young minds to help improve the lives of people suffering from chronic illnesses, and I can think of no better place to provide the very best education and training than MIT.

The molecular therapeutics center is the second research center established by Yang and Tan at MIT. In 2017, they launched the Hock E. Tan and K. Lisa Yang Center for Autism Research, and, two years later, they created a sister center at Harvard Medical School, with the unique strengths of each institution converging toward a shared goal: understanding the basic biology of autism and how genetic and environmental influences converge to give rise to the condition, then translating those insights into novel treatment approaches.

All tools developed at the molecular therapeutics center will be shared globally with academic and clinical researchers with the goal of bringing one or more novel molecular tools to human clinical trials by 2025.

We are hopeful that our centers, located in the heart of the Cambridge-Boston biotech ecosystem, will spur further innovation and fuel critical new insights to our understanding of health and disease, says Yang.

Originally posted here:
New molecular therapeutics center established at MIT's McGovern Institute - MIT News

Recommendation and review posted by Bethany Smith

IndustryGrowthInsights (IGI), one of the worlds prominent market research firms has announced a novel report on Global Gene Therapy Market. The report contains vital insights on the market which will support the clients to make the right business decisions. This research will help both existing and new aspirants for Gene Therapy market to figure out and study the market needs, market size, and competition. The report incorporates data regarding the supply and demand situation, the competitive scenario, and the challenges for market growth, market opportunities, and the threats faced by key players during the forecast period of 2020-2026.

Request Free Sample Report @ https://industrygrowthinsights.com/request-sample/?reportId=168032

Impact of COVID-19 on Gene Therapy Market

The report also includes the impact of ongoing global crisis i.e. COVID-19 on the Gene Therapy market and what the future holds for it. It provides an analysis of the effects of the pandemic on the global economy. The outbreak has directly disturbed the demand and supply chain. The report also analyzes the financial impact on firms and financial markets. IndustryGrowthInsights (IGI) has gathered insights from several delegates of the industry and got involved in the primary and secondary research to provide the clients with data and strategies to combat the market challenges during and after COVID-19 pandemic.

Benefits of buying the report:

Industry experts and research analysts have worked extensively to prepare the research report which will help you to give that extra edge in the competitive market. The market research report can be customized according to you to your needs. This means that IndustryGrowthInsights (IGI) can cover a particular product, application, or can provide a detailed analysis in the report. You can also purchase a separate report for a specific region.

You can buy the complete report @ https://industrygrowthinsights.com/checkout/?reportId=168032

Some of the major companies that are covered in this report:

Bluebird BioSangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVical Inc.AdvantageneGene Therap

*Note: Additional companies can be included on request

The market scenario is likely to be fairly competitive. To analyze any market with simplicity the market is fragmented into the following segments:

By Application:

CancerMonogenicInfectious diseaseCardiovascular diseaseOther

By Type:

Ex vivoIn VivoGene Therap

By Geographical Regions

Asia Pacific: China, Japan, India, and Rest of Asia PacificEurope: Germany, the UK, France, and Rest of EuropeNorth America: The US, Mexico, and CanadaLatin America: Brazil and Rest of Latin AmericaMiddle East & Africa: GCC Countries and Rest of Middle East & Africa

Segmenting the market into smaller components helps in analyzing the dynamics of the market with more clarity. Another key component that is included in the report is the regional analysis to assess the global presence of the Gene Therapy market. You can also opt for a yearly subscription of all the updates on the Gene Therapy market.

Request a sample before buying this report @ https://industrygrowthinsights.com/request-sample/?reportId=168032

Below is the TOC of the report:

Executive Summary

Assumptions and Acronyms Used

Research Methodology

Gene Therapy Market Overview

Gene Therapy Supply Chain Analysis

Gene Therapy Pricing Analysis

Global Gene Therapy Market Analysis and Forecast by Type

Global Gene Therapy Market Analysis and Forecast by Application

Global Gene Therapy Market Analysis and Forecast by Sales Channel

Global Gene Therapy Market Analysis and Forecast by Region

North America Gene Therapy Market Analysis and Forecast

Latin America Gene Therapy Market Analysis and Forecast

Europe Gene Therapy Market Analysis and Forecast

Asia Pacific Gene Therapy Market Analysis and Forecast

Middle East & Africa Gene Therapy Market Analysis and Forecast

Competition Landscape

If you have any questions on this report, feel free to reach us! @ https://industrygrowthinsights.com/enquiry-before-buying/?reportId=168032

About IndustryGrowthInsights (IGI):

IndustryGrowthInsights (IGI) has a vast experience in making customized market research reports in various industry verticals. Our motto is to provide complete client satisfaction. We cover in-depth market analysis, which consists of stipulating lucrative business strategies, especially for the new entrants and the emerging players of the market. We make sure that each report goes through intensive primary, secondary research, interviews, and consumer surveys before final dispatch.

We invest in our analysts to ensure that we have a full roster of experience and expertise in any field we cover. Our team members are selected for stellar academic records, specializations in technical fields, and exceptional analytical and communication skills. We also provide ongoing training and knowledge sharing to keep our analysts tapped into industry best practices and loaded with information.

Contact Info: Name: Alex MathewsAddress: 500 East E Street, Ontario,CA 91764, United States.Phone No: USA: +1 909 545 6473Email: [emailprotected]Website: https://industrygrowthinsights.com

See the original post:
Impact Of Covid-19 On Gene Therapy Market 2020 Analysis By Geographical Regions, Type And Application Till 2027 With Top Key Players: Bluebird Bio,...

Recommendation and review posted by Bethany Smith

NEW YORK--(BUSINESS WIRE)--As part of a two-day virtual Investor Day, Pfizer Inc. (NYSE: PFE) provided an extensive overview of pipeline advances and shared updates on the Companys efforts to battle the COVID-19 pandemic on multiple fronts, including new data on the BNT162b2 vaccine candidate being developed in collaboration with BioNTech SE. The pipeline updates contribute to the Companys expectation of at least a 6% revenue CAGR over the next five years, as well as delivery of longer-term topline growth beyond that period.

Pfizers goal of delivering up to 25 breakthroughs to patients by the year 2025 has 38 such opportunities to draw from as of today, including the companys 20-valent pneumococcal conjugate vaccine candidate (20vPnC). On a non-risk adjusted basis, these opportunities collectively represent more than $15 billion (excluding 20vPnC) in potential incremental revenue for Pfizer from 2020 to 2025, as well as aggregate peak annual sales potential of $35 billion to $40 billion (including 20vPnC). If successful, the Companys COVID-19 programs would be incremental to these estimates.

Pfizers purpose Breakthroughs that change patients lives has never been more relevant, and our R&D pipeline has never been more dynamic, said Dr. Albert Bourla, Pfizer Chairman and CEO. I am proud of the truly transformational science that our research and clinical teams are bringing to the fight against disease, as well as the unprecedented speed with which we are advancing our clinical programs in the battle against COVID-19. In the coming months and years, I look forward to the new Pfizer continuing to demonstrate the agility and innovative spirit of a biotech combined with the scale of Big Pharma. With the depth and breadth of our current portfolio, the tremendous potential of our pipeline and scientific engine, and the power of our culture of innovation, we are poised to continue delivering meaningful value to patients by addressing some of the worlds most difficult health challenges.

UPDATES ON COVID-19 DEVELOPMENT PROGRAMS

Pfizer announced several key advances in its efforts to protect humankind from the COVID-19 pandemic and prepare the pharmaceutical industry to better respond to future global health crises.

BNT162 mRNA-based Vaccine Program

Pfizer and BioNTech shared several updates from their BNT162 mRNA-based vaccine program against SARS-CoV-2, the virus that causes COVID-19 disease, including:

Protease Inhibitor Program

The company announced the initiation of its Phase 1b clinical trial to evaluate the safety of a novel investigational therapeutic for COVID-19, PF-07304814. Of note,

THERAPEUTIC AREAS OF FOCUS

Pfizer shared significant research advances across its various therapeutic areas including candidates with blockbuster potential expected to launch by 2025.

Vaccines

In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval. Updates on these late-stage clinical development programs include:

Rare Disease

Pfizers Rare Disease late-stage pipeline currently includes three gene therapy programs that, if successful, are expected to gain regulatory approval by the end of 2023, with an additional pipeline of 10 preclinical initiatives that are at various stages of maturity. Key updates include:

Oncology

Pfizers Oncology pipeline has the potential to deliver up to 14 approvals expected by the end of 2025 and the potential for 24 new molecular entities in the clinic by the end of 2021. Key updates included, for the first time, early-stage opportunities obtained from the 2019 acquisition of Array BioPharma:

Inflammation and Immunology

The Inflammation & Immunology pipeline is focused on patients with autoimmune and chronic inflammatory diseases across rheumatology, gastroenterology and dermatology, with five distinct immuno-kinases, in oral and topical formulations, studied for potential treatment of 10 diseases, and three additional novel biologics in Phase 2 studies. Key updates included:

Internal Medicine

The Internal Medicine pipeline addresses the increasing global burden of cardiometabolic disease, with nine investigational medicines in active clinical studies and additional therapies in the pre-clinical pipeline. Key updates included:

To access a replay of the webcast, including audio, video and presentation slides, visit our web site at http://www.pfizer.com/investors.

About Pfizer: Breakthroughs That Change Patients Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice: The information contained in this release is as of September 15, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release or the webcast as the result of new information or future events or developments.

This release and the webcast contain forward-looking information about Pfizers anticipated operating and financial performance, business plans and prospects, Pfizers pipeline portfolio (including anticipated regulatory submissions, data read-outs, study starts, approvals, revenue contributions and market opportunities), and our efforts to respond to COVID-19, including our investigational vaccine candidate against SARS-CoV-2 and our investigational protease inhibitor, including their potential benefits, among other things, that are subject to substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any drug applications, biologics license applications and/or emergency use authorization applications may be filed in any jurisdictions for any potential indication for Pfizers product candidates; whether and when any such applications that may be filed for any of Pfizers product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether any such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of Pfizers product candidates, including development of products or therapies by other companies; manufacturing capabilities or capacity; uncertainties regarding the ability to obtain recommendations from vaccine technical committees and other public health authorities and uncertainties regarding the commercial impact of any such recommendations; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

More:
Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas - Business Wire

Recommendation and review posted by Bethany Smith

Here are the top biotech companies you will find in Oxford, a city with an old scientific tradition and an enormous output of biotech applications.

Oxford is well known for its university, one of the oldest in Europe and considered to be one of the best in the world. Recently, the University of Oxford has been the center of attention thanks to an experimental Covid-19 vaccine that the university is developing in partnership with big pharmaceutical companies. If successful, the vaccine, already in phase III testing, could be one of the first to get approval for this new disease.

The University of Oxford has hosted thousands of bright minds over the years. Thanks to its emphasis on technology transfer, the university has also helped a large number of them turn their ideas into successful spinout companies. The environment created around the university has also attracted many talents and businesses to the city, making it the ideal melting pot for new and promising ideas.

Biotech is one of the fortes of the innovation seen in the city of Oxford. So we consulted with local experts to put together a list of the most remarkable companies in the city, be it for their size, innovation, or influence in the sector.

Founded in 2008, Immunocore is one of just a few private biotech companies in Europe that are estimated to be worth over 1B. The company is tackling multiple forms of cancer as well as infectious and autoimmune diseases using T-cell receptor (TCR) technology. TCRs are proteins on the surface of immune T cells that are responsible for identifying a threat that must be destroyed, such as cancerous or infected cells. Immunocore aims to patients with engineered TCRs to circumvent the mechanisms by which these threats evade the immune system, restoring its ability to fight disease.

The company is collaborating on several projects with Genentech, AstraZeneca, Eli Lilly, and GSK. Its most advanced program is a treatment for uveal melanoma that is currently in phase III trials. Other programs target solid tumors, hepatitis, HIV, and type 1 diabetes.

Adaptimmune Therapeutics was founded at the same time as Immunocore with the goal of exploiting TCR technology in the form of T-cell therapy. The company engineers the TCRs naturally present on the patients own immune T cells to improve their ability to identify cancerous cells.

Adaptimmune is now getting ready to start late-stage clinical trials in multiple cancer types. Thanks to a deal with Astellas Pharma, the company is also gearing up to start clinical testing of a version of its T-cell therapy that doesnt require engineering each dose individually for each patient, using donor cells instead. Adaptimmune also has several partnerships with companies including GSK, Noile-Immune Biotech, and Alpine Immune Sciences.

Oxford Biodynamics was spun out of Oxford University in 2007 with the goal of developing liquid biopsy tests that can perform a diagnosis from just a drop of blood. The company specializes in epigenetics, that is changes to the structure of our DNA that determines which genes are switched on or off.

Oxford Biodynamics works in a wide range of indications, including cancer, diabetes, Alzheimers, multiple sclerosis, and rheumatoid arthritis among many others. The tests are not only designed to diagnose a disease; they can also be made to determine which patients are going to benefit the most from a specific drug, and how likely the disease is to progress faster or relapse.

With these tests, the company is supporting the development of personalized medicine approaches and helping drug developers increase their chances of succeeding in clinical trials. Partners include big pharma such as Pfizer and EMD, as well as universities and research institutes.

Chronos Therapeutics started out in 2009 as a spinout of the University of Oxford with the goal of developing drugs for age-related conditions. The companys lead program targets amyotrophic lateral sclerosis (ALS), the most common motor neuron disease, for which there are no treatments able to stop its progression.

Over time, the company has expanded its portfolio into other indications, particularly those that affect the brain, by acquiring assets from third parties. They include programs targeting fatigue caused by multiple sclerosis, addictive behaviors such as binge eating and alcohol use disorder, and post-traumatic stress disorder.

Evox Therapeutics is developing a drug delivery technology based on exosomes nanoparticles that our bodies naturally use to transport molecules. The company engineers exosomes to carry different types of drugs, such as proteins, RNA, or small drugs. The key advantage is that these natural carriers are able to reach targets that conventional drug delivery systems cant, such as the brain.

Founded in 2016, the company has signed big deals with Takeda and Eli Lilly. Its programs are all in preclinical testing and mostly target rare diseases. One of them targets the rare liver disorder argininosuccinic aciduria and is scheduled to enter clinical trials in 2021.

Exscientia is a pioneer in the application of artificial intelligence to drug discovery. The company uses AI to identify potential drug candidates and optimize their structure to maximize their chances of success in clinical trials. The goal is to speed up and reduce the costs of the drug discovery process.

Earlier this year, the companys drug candidate for obsessive-compulsive disorder became one of the first AI-designed drugs to enter clinical trials. While a drug typically takes five years from identification until clinical trials, this one did so in just a year.

Exscientia is partnered with Bayer, BMS, Sanofi, and GSk among others. In the wake of the Covid-19 pandemic, the company set out to go through a database of 15,000 approved and investigational drugs that had already passed safety testing to find candidates that can then be fast-tracked to clinical testing in Covid-19 patients.

Oxford Biomedica was set up in 1995 as a developer and provider of lentiviral vectors for gene and cell therapy. These vectors allow the permanent introduction of a desired DNA sequence into a target cell, be it in the test tube or directly in the patients body.

The technology of Oxford Biomedica is regularly used by companies such as Novartis, Sanofi, Boehringer Ingelheim, Imperial Innovations, and Orchard Therapeutics. Notably, the vectors developed by the company are used in Novartis Kymriah, the first CAR T-cell therapy approved in Europe and the US as a cancer treatment.

Oxford Biomedica also has a preclinical pipeline of proprietary programs in a wide range of applications, including cancer, eye disease, ALS, and liver disease. Last year, the company struck a deal with Microsoft to reduce the complexity and costs of gene and cell therapy manufacturing using artificial intelligence.

Founded in 2016, Arctoris aims to bring the benefits of automation to cancer research. Through the companys services, a researcher could just order an experiment online and spend their valuable time designing experiments and analyzing results rather than performing the repetitive tasks needed to complete them.

Arctoris aims to contribute to reducing the costs of drug discovery, which are increasing every year as treatments become personalized and results more difficult to replicate. In the context of the Covid-19 pandemic, Arctoris has established assays that allow scientists the possibility of running Covid-19 experiments remotely.

Founded in 2015, Orbit Discovery is a drug discovery company focusing on the identification of peptide drugs. The company has developed a technology that significantly improves on conventional methods of drug screening such as phage display or mRNA display.

The technology consists of fusing peptides to the DNA sequence encoding them and presenting them to live cells. This method allows the screening of peptide targets that were previously missed by other technologies, and to study their effect on live cells to better predict their function.

The company has already identified several candidates in the areas of cardiology, immunology, and cancer, and is working with partners such as Zealand Pharma in their preclinical development.

OxStem is a drug discovery company with an unusual approach to stem cell treatments. Instead of using stem cells as a therapy, the firm focuses on developing small molecule drugs that can reprogram the patients own stem cells to treat a wide range of diseases related to aging, including cancer and diabetes as well as neurological, cardiovascular, and ocular conditions.

Founded in 2013, the strategy of OxStem is to spin out companies that specialize in each disease area to focus on the development of the drugs found by the parent company, with five subsidiaries set up so far.

Originally posted here:
The Top 10 Biotech Companies Brewing at... - Labiotech.eu

Recommendation and review posted by Bethany Smith

LONDON--(BUSINESS WIRE)--Technavio has been monitoring the biologic therapeutics market and it is poised to grow by $ 190.94 bn during 2020-2024, progressing at a CAGR of almost 12% during the forecast period. The report offers an up-to-date analysis regarding the current market scenario, latest trends and drivers, and the overall market environment.

Although the COVID-19 pandemic continues to transform the growth of various industries, the immediate impact of the outbreak is varied. While a few industries will register a drop in demand, numerous others will continue to remain unscathed and show promising growth opportunities. Technavios in-depth research has all your needs covered as our research reports include all foreseeable market scenarios, including pre- & post-COVID-19 analysis. We offer $1000 worth of FREE customization

The market is concentrated, and the degree of concentration will accelerate during the forecast period. AbbVie Inc., AstraZeneca Plc, Bristol-Myers Squibb Co., F. Hoffmann-La Roche Ltd., GlaxoSmithKline Plc, Johnson & Johnson, Merck & Co. Inc., Novartis AG, Pfizer Inc., and Sanofi are some of the major market participants. To make the most of the opportunities, market vendors should focus more on the growth prospects in the fast-growing segments, while maintaining their positions in the slow-growing segments.

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Strong R&D pipeline has been instrumental in driving the growth of the market. However, the high cost of biologics might hamper the market growth.

Technavio's custom research reports offer detailed insights on the impact of COVID-19 at an industry level, a regional level, and subsequent supply chain operations. This customized report will also help clients keep up with new product launches in direct & indirect COVID-19 related markets, upcoming vaccines and pipeline analysis, and significant developments in vendor operations and government regulations. Download a Free Sample Report on COVID-19 Impacts

Biologic Therapeutics Market 2020-2024: Segmentation

Biologic Therapeutics Market is segmented as below:

Biologic Therapeutics Market 2020-2024: Scope

Technavio presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources. The biologic therapeutics market report covers the following areas:

This study identifies the introduction of biosimilars as one of the prime reasons driving the biologic therapeutics market growth during the next few years.

Technavio suggests three forecast scenarios (optimistic, probable, and pessimistic) considering the impact of COVID-19. Technavios in-depth research has direct and indirect COVID-19 impacted market research reports.

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Biologic Therapeutics Market 2020-2024: Key Highlights

Table of Contents:

Executive Summary

Market Landscape

Market Sizing

Five Forces Analysis

Market Segmentation by Product

Market segmentation by application

Customer landscape

Geographic Landscape

Vendor Landscape

Vendor Analysis

Appendix

About Us

Technavio is a leading global technology research and advisory company. Their research and analysis focus on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavios report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

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COVID-19 Recovery Analysis: Biologic Therapeutics Market | Strong R&D Pipeline to Boost the Market Growth | Technavio - Business Wire

Recommendation and review posted by Bethany Smith

This month, Tmunity Therapeutics is dosing the first patients who have enrolled in an early clinical trial testing an engineered CAR-T immune cell therapy for metastatic prostate cancer. The trial's startis notable, and not just because it's been challenging to recruit patients for cancer trials during the COVID-19 pandemic. The experimental CAR-T is among the first cell therapies to target a solid tumoran enemy that was once considered too difficult to tackle with that type of treatment.

As the first FDA-approved CAR-T cell therapies start to take hold in the blood cancer sphere, a second wave of cell therapies for cancer is following close behind, promising to offer even more choices for patients. In addition to personalized CAR-T therapies for solid tumors, the pipeline includes off-the-shelf CAR-Ts for blood cancers and cell therapies that harness the bodys innate ability to kill cancer.

The CAR-T treatments on the marketNovartis Kymriah, and Yescarta and Tecartus from Gileads Kite Pharmaall target CD19 on the surface of B cells that drive some blood cancers. The treatments involve removing T cells from individual patients and engineering them to be able to recognize and kill their cancers.

ESMO Post Show: Highlights From the Virtual Conference

Cancer experts and pharma execs will break down the headline-making data from ESMO, sharing their insights and analysis around the conferences most closely watched studies. This discussion will examine how groundbreaking research unveiled over the weekend will change clinical practice and prime drugs for key new indications, and panelists will fill you in on the need-to-know takeaways from oncologys hottest fields.

Much of the work on next-gen cell therapies for cancer revolvesaround finding new targets for the engineered immune cells to home in on, as well as improving the therapies to make them more efficient and to reduce the risk of dangerous immune side effects. Theres a lot of activity in this field and different strategies that I think will prove to be effective in the next few years, said Dan Kaufman, M.D., Ph.D., a hematologist and professor of medicine at the University of California, San Diego in an interview.

RELATED: Andreessen Horowitz leads $75M round for Tmunity as it eyes phase 2 studies for 2021

Tmunitys prostate cancer CAR-T is engineered to target prostate-specific membrane antigen, a protein thats often overexpressed by prostate cancer cells, as well as transforming growth factor (TGF) beta, which can promote tumor growth.

Normally, TGF beta binds to T cells in a way that prevents them from mounting an immune attack, Tmunity CEO Usman Oz Azam, M.D., said in an interview. The company's engineering process changes the TGF beta receptor so it cant interfere with the T cells' work.

Its a combination of targeting the antigen but also giving the CAR-T cells a chance to persist and endure, which is where the TGF beta component comes in, he said. The prostate cancer CAR-T is now in a phase 1 trial at six clinical sites in the U.S.

Like the three FDA-approved CAR-Ts, Tmunitys prostate cancer therapy is made from patients own immune cells, a time-consuming and expensive process. Several companies are trying to avoid those costswith off-the-shelf, or allogeneic CAR-Ts. These cell therapies are made from donor cells and engineered so that they dont cause the rejection reaction known as graft-versus-host disease (GVHD).

Allogene, co-founded by Kite pioneer Arie Belldegrun, is one of the pioneers in off-the-shelf CAR-T, and in May it unveiled early data from a trial of its CD19-targeted candidate, ALLO-501, in patients with lymphoma. The company reported a 63% response rate.

RELATED: ASCO: Allogene reports 63% response rate in early trial of off-the-shelf CAR-T in lymphoma

Off-the-shelf CAR-T cells are genetically engineered to avoid GVHD. Precision Biosciences, another player working on a CD19-directedoff-the-shelf CAR-T, developed a technology that removes the TCR gene, which causes GVHD, and inserts the CAR (chimeric antigen receptor) all in one stepan approach that could offer some advantages, Precision CEOMatt Kane said in an interview.

With the first generation of CAR-T cells, the CAR randomly goes into the genome of cells, and as a result they can express at different levels of potency. We insert the CAR into one location, which takes away that variability, Kane said. The company expects to share clinical trial data late this year or in early 2021.

The off-the-shelf CAR-T field has hit some obstacles, though.Cellectis is developing an anti-CS1 CAR-T for multiple myeloma thats also designed for off-the-shelf use, but the FDA put a phase 1 trial on hold after one patient on the higher dose being tested developed cytokine release syndrome (CRS)and died of cardiac arrest.

The company is reviewing the case, and once Cellectis provides feedback to the FDA, the agency will have 30 days to decide whether to restart the trial, Cellectis CEO Andr Choulika, Ph.D., said in an interview.

Another potential option for off-the-shelf cell therapies for cancer is to engineer them not with T cells, but with different residents of the immune system known as natural killer or NK cells. These cells can be taken from donors and given to patients without causing GVHD, and they have an innate ability to recognize and destroy cancers. But there typically arent enough NK cellsto infiltrate tumors effectively, and the cells tend to be short-lived.

Nkarta is developing NK cell therapies that are designed to address both those shortcomings. Nkartas cells are engineered so they have an enhanced ability to recognize eight ligands that are upregulated in several blood cancers and solid tumors. The cells also express interleukin-15 (IL-15), a cytokine growth factor.

"Were able to maintain the growth of the cells for a long period of time, said James Trager, Ph.D., chief scientific officer of Nkarta, in an interview. The company recently raised $114 million in a series B round and expects to move its lead candidate, NKX101, into clinical trials by year-end.

RELATED: Nkarta nets $114M to push natural killer cell therapies into the clinic

Several NK cell developers are following close on Nkartas heals. Fate Therapeutics is working on both off-the-shelf CAR-T and NK therapies and recently signed a $50 million co-development deal with Johnson & Johnson.

Two startups are hoping to harness the power of anti-cancer drugs to enhance the potency of NK cells. Artiva debuted in June with $78 million in series A financing and a plan to test its NK cells in combination with anti-cancer monoclonal antibodies.

Acepodia was founded in 2016 by Patrick Yang, Ph.D., a former exec at CAR-T maker Juno Therapeutics, and is now pursuing NK cell therapies conjugated with therapeutic antibodies. Its lead program combines an NK cell with a monoclonal antibody targeting HER2, an antigen found in breast, gastric and other solid tumor types.

Meanwhile, Gilead is in on the NK act as well, building on its strong position in CAR-T therapies. In April, it penned a partnership with Australia-based oNKo-innate, which is using genomic screening to find pathways that boost the ability of NK cells to combat tumors.

Were looking at the intracellular pathways that are either inhibiting or preventing the optimal expansion and development of an NK therapy, Peter Emtage, Ph.D., Gilead's senior vice president of cell therapy research, said in an interview. We need to identify the optimal structure that will make that NK cell the best it can be.

Gilead envisions the field of cell therapy moving beyond the single-modality approach and towarda more holistic view of treating cancer, Emtage said. We have to start thinking outside of the classical Yescarta CD19 therapy and start looking for methods to harness the endogenous immune system, so that we can make a concerted impact on the tumor.

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From natural killer cells to off-the-shelf CAR-Ts, biopharma targets cancer with next-gen cell therapies - FierceBiotech

Recommendation and review posted by Bethany Smith

An international collaboration involving Monash University and Duke-NUS researchers have made an unexpected world-first stem cell discovery that may lead to new treatments for placenta complications during pregnancy.

While it is widely known that adult skin cells can be reprogrammed into cells similar to human embryonic stem cells that can then be used to develop tissue from human organs - known as induced pluripotent stem cells (iPSCs) - the same process could not create placenta tissue.

iPSCs opened up the potential for personalised cell therapies and new opportunities for regenerative medicine, safe drug testing and toxicity assessments, however little was known about exactly how they were made.

An international team led by ARC Future Fellow Professor Jose Polo from Monash University's Biomedicine Discovery Institute and the Australian Research Medicine Institute, together with Assistant Professor Owen Rackham from Duke-NUS in Singapore, examined the molecular changes the adult skin cells went through to become iPSCs. It was during the study of this process that they discovered a new way to create induced trophoblast stem cells (iTSCs) that can be used to make placenta cells.

This exciting discovery, also involving the expertise of three first authors, Dr. Xiaodong Liu, Dr. John Ouyang and Dr. Fernando Rossello, will enable further research into new treatments for placenta complications and the measurement of drug toxicity to placenta cells, which has implications during pregnancy.

"This is really important because iPSCs cannot give rise to placenta, thus all the advances in disease modelling and cell therapy that iPSCs have brought about did not translate to the placenta," Professor Polo said.

"When I started my PhD five years ago our goal was to understand the nuts and bolts of how iPSCs are made, however along the way we also discovered how to make iTSCs," said Dr Liu.

"This discovery will provide the capacity to model human placenta in vitro and enable a pathway to future cell therapies," commented Dr Ouyang.

"This study demonstrates how by successfully combining both cutting edge experimental and computational tools, basic science leads to unexpected discoveries that can be transformative," Professor Rackham said.

Professors Polo and Rackham said many other groups from Australian and international universities contributed to the study over the years, making it a truly international endeavour.

Reference:Liu, X., Ouyang, J.F., Rossello, F.J. et al. Reprogramming roadmap reveals route to human induced trophoblast stem cells. Nature (2020). https://doi.org/10.1038/s41586-020-2734-6

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

Original post:
Researchers Discover a Way To Create Induced Tropoblast Stem Cells - Technology Networks

Recommendation and review posted by Bethany Smith

Brady Feeney hadnt even taken any classes at Indiana University when he fell ill with Covid-19. Three weeks after he moved to Bloomington, the incoming freshman was in the emergency room, struggling to breathe. Before his illness, Feeney had been a perfectly healthy teenager, with no preexisting conditions. In high school, he was a three-time all-state football player and won two state titles in Missouri. But after two weeks of hell fighting the virus, his mother said, his bloodwork indicated possible heart problems.

When SARS-CoV-2 first struck the United States, the medical community had two working assumptions: First, this was primarily a respiratory disease, and second, it seemed to hit older people much harder than younger people, with eight out of 10 confirmed Covid-19 deaths in the U.S. happening in adults 65 or older. But now, new research is challenging both of these assumptions.

Growing evidence suggests that SARS-CoV-2 doesnt only infect the lungs. It also affects the brain, kidneys, and heart. At first, doctors and researchers wondered if these issues beyond the lungs came just from the stress of having Covid-19 and being on a ventilator or life support. But increasingly, research indicates that the virus may be attacking other organs in the body directlyand this may be more common than previously thought, even among those who arent sick enough to be hospitalized. Some have suggested that Covid-19 is actually a blood vessel disease; the lungs are merely the way the virus enters the body, but from there it gets into the bloodstream and takes up residence in major organs, leaving patients with complex, long-lasting symptoms. Moreover, experts now believe, healthy young people can get mild cases of the coronaviruseven not knowing they were sickthat could leave them with lasting cardiovascular damage. Even those who seem to have recovered from the deadly respiratory illness are not free of its complications.

Heart failure could be the next chapter of the coronavirus illness, Dr. Gregg C. Fonarow, interim chief of UCLAs Division of Cardiology, recently argued in a co-authored editorial in the journal JAMA Cardiology. Even if in younger adults Covid-19 may not be fatal, there still may be important health consequences, he told me.

Myocarditis, or inflammation of the heart, is usually a rare condition that can occur with viral infections, including the flu. But from the start of the pandemic, doctors were seeing heart inflammation among patients hospitalized with serious cases of Covid-19, Fonarow said: Early research showed that 20 to 30 percent of those hospitalized had heart issues. Left untreated, myocarditis can damage the heart and lead to heart attacks and arrhythmias, among other complications.

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What Is Covid-19 Doing to Our Hearts? - The New Republic

Recommendation and review posted by Bethany Smith

TOKYO and BOTHELL, Wash., Sept. 18, 2020 /PRNewswire/ --Astellas Pharma Inc.(TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and Seattle Genetics, Inc. (Nasdaq:SGEN) today announced that a phase 3 trial of PADCEV (enfortumab vedotin-ejfv) met its primary endpoint of overall survival compared to chemotherapy. The results were reviewed by an independent Data Monitoring Committee following a planned interim analysis. The global EV-301 clinical trial compared PADCEV to chemotherapy in adult patients with locally advanced or metastatic urothelial cancer who were previously treated with platinum-based chemotherapy and a PD-1/L1 inhibitor.

In the trial, PADCEV significantly improved overall survival (OS), with a 30 percent reduction in risk of death (Hazard Ratio [HR]=0.70; [95% Confidence Interval (CI): 0.56, 0.89]; p=0.001). PADCEV also significantly improved progression-free survival (PFS), a secondary endpoint, with a 39 percent reduction in risk of disease progression or death (HR=0.61 [95% CI: 0.50, 0.75]; p<0.00001).

For patients in the PADCEV arm of the trial, adverse events were consistent with those listed in the U.S. Prescribing Information, with rash, hyperglycemia, decreased neutrophil count, fatigue, anemia and decreased appetite as the most frequent Grade 3 or greater adverse event(s) occurring in more than 5 percent of patients.Data from EV-301 will be submitted for presentation at an upcoming scientific congress. Patients in the chemotherapy arm of the trial will be offered the opportunity to receive PADCEV.

The results will be submitted to the U.S. Food and Drug Administration (FDA) as the confirmatory trial following the drug's accelerated approval in 2019. EV-301 is also intended to support global registrations.

"EV-301 is the first randomized trial to show overall survival results compared to chemotherapy in patients with locally advanced or metastatic urothelial cancer who previously have received platinum-based treatment and a PD-1 or PD-L1 inhibitor, and we are encouraged by the potential this may have in helping patients who have otherwise limited alternatives," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head, Astellas. "We look forward to discussing these results with global health authorities."

"These survival results from the confirmatory trial for PADCEV are welcome news for patients whose cancer has progressed after platinum-based chemotherapy and immunotherapy," said Roger Dansey, M.D., Chief Medical Officer at Seattle Genetics. "We continue to explore PADCEV's activity across the spectrum of urothelial cancer including its potential for use in earlier lines of therapy."

Globally, approximately 580,000 people will be diagnosed with bladder cancer in 2020.1Urothelial cancer accounts for 90 percent of all bladder cancers and can also be found in the renal pelvis (where urine collects inside the kidney), ureter (tube that connects the kidneys to the bladder) and urethra.2Approximately 80 percent of people do not respond to PD-1 or PD-L1 inhibitors after a platinum-containing therapy has failed as an initial treatment for advanced disease.3

About the EV-301 TrialThe EV-301 trial (NCT03474107) is a global, multicenter, open-label, randomized phase 3 trial designed to evaluate PADCEV versus physician's choice of chemotherapy (docetaxel, paclitaxel or vinflunine) in approximately 600 patients with locally advanced or metastatic urothelial cancer who were previously treated with a PD-1 or PD-L1 inhibitor and platinum-based therapies. The primary endpoint is overall survival of participants treated with PADCEV compared to those treated with chemotherapy. Secondary endpoints include progression-free survival, duration of response, and overall response rate, as well as assessment of safety/tolerability and quality-of-life parameters.

For more information about the EV-301 clinical trial, please visit http://www.clinicaltrials.gov.

About PADCEV (enfortumab vedotin-ejfv)PADCEV was approved by the U.S. Food and Drug Administration (FDA) in December 2019 and is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy before (neoadjuvant) or after (adjuvant) surgery or in a locally advanced or metastatic setting. PADCEV was approved under the FDA's Accelerated Approval Program based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.4

PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.4,5 Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).4 PADCEV is co-developed by Astellas and Seattle Genetics.

PADCEV Important Safety Information

Warnings and Precautions

Adverse ReactionsSerious adverse reactions occurred in 46% of patients treated with PADCEV. The most common serious adverse reactions (3%) were urinary tract infection (6%), cellulitis (5%), febrile neutropenia (4%), diarrhea (4%), sepsis (3%), acute kidney injury (3%), dyspnea (3%), and rash (3%). Fatal adverse reactions occurred in 3.2% of patients, including acute respiratory failure, aspiration pneumonia, cardiac disorder, and sepsis (each 0.8%).

Adverse reactions leading to discontinuation occurred in 16% of patients; the most common adverse reaction leading to discontinuation was peripheral neuropathy (6%). Adverse reactions leading to dose interruption occurred in 64% of patients; the most common adverse reactions leading to dose interruption were peripheral neuropathy (18%), rash (9%) and fatigue (6%). Adverse reactions leading to dose reduction occurred in 34% of patients; the most common adverse reactions leading to dose reduction were peripheral neuropathy (12%), rash (6%) and fatigue (4%).

The most common adverse reactions (20%) were fatigue (56%), peripheral neuropathy (56%), decreased appetite (52%), rash (52%), alopecia (50%), nausea (45%), dysgeusia (42%), diarrhea (42%), dry eye (40%), pruritus (26%) and dry skin (26%). The most common Grade 3 adverse reactions (5%) were rash (13%), diarrhea (6%) and fatigue (6%).

Lab AbnormalitiesIn one clinical trial, Grade 3-4 laboratory abnormalities reported in 5% were: lymphocytes decreased (10%), hemoglobin decreased (10%), phosphate decreased (10%), lipase increased (9%), sodium decreased (8%), glucose increased (8%), urate increased (7%), neutrophils decreased (5%).

Drug Interactions

Specific Populations

For more information, please see the full Prescribing Information for PADCEV here.

About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+ healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. For more information, please visit our website at https://www.astellas.com/en/.

About Seattle Genetics Seattle Genetics, Inc. is a global biotechnology company that discovers, develops and commercializes transformative medicines targeting cancer to make a meaningful difference in people's lives. The company is headquartered in the Seattle, Washington area, with locations in California, Switzerland and the European Union. For more information on our robust pipeline, visit http://www.seattlegenetics.comand follow @SeattleGeneticson Twitter.

About the Astellas and Seattle Genetics CollaborationAstellas and Seattle Genetics are co-developing PADCEV (enfortumab vedotin-ejfv) under a 50:50 worldwide development and commercialization collaboration that was entered into in 2007 and expanded in 2009.

Astellas Cautionary NotesIn this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties.

Information about pharmaceutical products (including products currently in development), which is included in this press release is not intended to constitute an advertisement or medical advice.

Seattle Genetics Forward Looking Statements Certain statements made in this press release are forward looking, such as those, among others, relating to the submission of data from the EV-301 trial for presentation at an upcoming scientific congress; intended regulatory actions, including plans to submit the results of the EV-301 trial to the FDA as the confirmatory trial following the drug's accelerated approval in the U.S. and plans to discuss the results with global health authorities and seek global registrations; conduct of a comprehensive clinical development program for PADCEV, which includes exploring PADCEV's activity in other types of urothelial cancer and its potential for use in earlier lines of therapy;the therapeutic potential of PADCEV,including its efficacy, safety and therapeutic uses, and anticipated development activities, including ongoing and future clinical trials. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include that the data from the EV-301 trial may not be selected for presentation at scientific congresses; the possibility of delays in the submission of results to the FDA; that the results from the EV-301 trial may not be enough to convert PADCEV's accelerated approval in the U.S. to regular approval or to support any other global registrations; that, even if PADCEV receives regular approval in the U.S. or any other global registrations, the product labeling may not be as broad or desirable as anticipated; the possibility that ongoing and subsequent clinical trials may fail to establish sufficient activity; the risk of adverse events or safety signals; and the possibility that adverse regulatory actions may occur. More information about the risks and uncertainties faced by Seattle Genetics is contained under the caption "Risk Factors" included in the company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 filed with the Securities and Exchange Commission. Seattle Genetics disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

1 International Agency for Research on Cancer. Cancer Tomorrow: Bladder. http://gco.iarc.fr/tomorrow. Accessed 07-31-2020.2 American Society of Clinical Oncology. Bladder cancer: introduction (10-2017).3 Shah, Manasee V., et al "Targeted Literature Review of the Burden of Illness in UC" (PCN108), Nov 2018.4PADCEV [package insert] Northbrook, IL: Astellas, Inc.5Challita-Eid P, Satpayev D, Yang P, et al. Enfortumab Vedotin Antibody-Drug Conjugate Targeting Nectin-4 Is a Highly Potent Therapeutic Agent in Multiple Preclinical Cancer Models. Cancer Res 2016;76(10):3003-13.

SOURCE Astellas Pharma Inc.

https://www.astellas.com/en/

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Astellas and Seattle Genetics Announce PADCEV (enfortumab vedotin-ejfv) Significantly Improved Overall Survival in Phase 3 Trial in Previously Treated...

Recommendation and review posted by Bethany Smith

The European Medicines Agency is on the verge of releasing revised guidance for advanced therapy medicinal products containing genetically modified cells, which includes chimeric antigen receptor (CAR)-T cell therapies.

The Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells was originally issued in 2012 but underwent revision and consultation from July 2018-July 2019. The revised version is expected to be adopted in October and published in November, according to Ana Hidalgo-Simon, MD, PhD, head of advanced therapies at EMA. She previewed the major changes at RAPS Convergence 2020.

There were an enormous number of comments on the document, Hidalgo-Simon said.The agency is also working on a Q&A document on principles of good manufacturing practices (GMP) for Advanced Therapy Medicinal Products (ATMP) starting material. There will likely be consultation on the document in 2021, she said. (RELATED: Regulation of advanced therapy medicinal products in the EU, Regulatory Focus, 16 July 2020.)

Major changesEMA chose to update the guidance to reflect the increase in clinical experience with these therapies, particularly chimeric antigen receptor-T (CAR-T) cells; to cover new categories of products, such as induced pluripotent stem (iPS) cells; and to allow for consideration of new tools for genetic modification of cells, such as genome editing technologies, she said.

The main quality updates are related to starting materials, the manufacturing process, and characterization and release. For example, the starting materials guidance will now include genome editing tools, while the manufacturing process includes a new section on comparability. The characterization and release portion of the guidance includes specific advice for CAR-T cells.

Additionally, the guidance calls for dose-finding studies to explore safety, toxicity, and anti-tumor activity at different dose levels, to define the threshold dose required for anti-tumor effect, and to define the recommended dose or range for Phase 2 studies. She said sponsors need to show a solid rationale for the criteria being used to find the dose.

The guidance also calls for Phase 3 confirmatory trials to follow a randomized controlled design, comparing the CAR-T cell therapy to a reference regimen, unless otherwise scientifically justified. Single-arm studies will continue to be allowed, but they will be the exception, Dr. Hidalgo-Simon said.

Be very careful with the design of the trials, she advised. The assumptions need to be really, very well backed.

When it comes to safety, the guidance calls for a 15-year follow period. While sponsors wont have all the answers at the time of submission, Hidalgo-Simon said they should have a plan that includes monitoring during the post-authorization period.

Hidalgo-Simon also advised sponsors to think beyond the approval process and consider what evidence will be needed to convince other stakeholders -- from patients to payers -- about the safety and efficacy of the therapy.

Avoiding development pitfallsRichard Dennett, PhD, the senior director of chemistry, manufacturing and controls regulatory affairs at PPD, also participated in the RAPS Convergence 2020 session on advanced therapies. He reviewed development points where companies can run into trouble with advanced therapies, particularly CAR-T cell products.Dennett recommended that product sponsors keep the end in mind when developing advanced therapies by focusing on the target product profile at the beginning of development. That profile includes the indication for which approval will be sought and the incidence of that indication; other considerations include mode of action, demographics, how much of the product needs to be produced, and market access and reimbursement considerations.

He also outlined several areas where developers should focus to create a watertight regulatory package, including sufficient product characterization, potency assay, impurities, formulation, stability, lack of sufficient development batches, and validation strategy.

Dennett urged developers to dive into the growing number of regulatory guidance documents for advanced therapies. In addition to the European guidance documents, developers should consultthe US Food and Drug Administrations Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), which was released in January 2020. (RELATED: Advanced therapies: Trip hazards on the development pathway, Regulatory Focus, 02 August 2020)

Live and breathe the guidances that are out there, Dennett advised. They allow us to understand what expectations we need to meet.

The key to success in advancing CAR-T cell therapies is the mitigation of risk, Dennett said: The biggest risk is the one that you havent thought of.RAPS 2020 Convergence

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Convergence: EMA close to finalizing guidance for advanced therapies - Regulatory Focus

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A surface view of the organ of hearing (cochlea) from a mouse, using confocal microscopy. The sensory cells are named hair cells because of their apical projections (stereocilia) which move from stimulation by sound.

University of Maryland School of Medicine

Researchers at the University of Maryland School of Medicine (UMSOM) have conducted a study that has determined the role that a critical protein plays in the development of hair cells. These hair cells are vital for hearing. Some of these cells amplify sounds that come into the ear, and others transform sound waves into electrical signals that travel to the brain. Ronna Hertzano, MD, PhD, associate professor in the Department of Otorhinolaryngology Head and Neck Surgery at UMSOM and Maggie Matern, PhD, a postdoctoral fellow at Stanford University, demonstrated that the protein, called GFI1, may be critical for determining whether an embryonic hair cell matures into a functional adult hair cell or becomes a different cell that functions more like a nerve cell or neuron.

The study was published in the journalDevelopment, and was conducted by physician-scientists and researchers at the UMSOM Department of Otorhinolaryngology Head and Neck Surgery and the UMSOM Institute for Genome Sciences (IGS), in collaboration with researchers at the Sackler School of Medicine at Tel Aviv University in Israel.

Hearing relies on the proper functioning of specialized cells within the inner ear called hair cells. When the hair cells do not develop properly or are damaged by environmental stresses like loud noise, it results in a loss of hearing function.

In the United States, the prevalence of hearing loss doubles with every 10-year increase in age, affecting about half of all adults in their 70s and about 80 percent of those who are over age 85. Researchers have been focusing on describing the developmental steps that lead to a functional hair cell, in order to potentially generate new hair cells when old ones are damaged.

To conduct her latest study, Hertzano and her team utilized cutting-edge methods to study gene expression in the hair cells of genetically modified newborn mice that did not produce GFI1. They demonstrated that, in the absence of this vital protein, embryonic hair cells failed to progress in their development to become fully functional adult cells. In fact, the genes expressed by these cells indicated that they were likely to develop into neuron-like cells.

"Our findings explain why GFI1 is critical to enable embryonic cells to progress into functioning adult hair cells," said Hertzano. "These data also explain the importance of GFI1 in experimental protocols to regenerate hair cells from stem cells. These regenerative methods have the potential of being used for patients who have experienced hearing loss due to age or environmental factors like exposure to loud noise."

Hertzano first became interested in GFI1 while completing her MD, PhD at Tel Aviv University. As part of her dissertation, she discovered that the hearing loss resulting from mutations in another protein called POU4F3 appeared to largely result from a loss of GFI1 in the hair cells. Since then, she has been conducting studies to discover the role of GFI1 and other proteins in hearing. Other research groups in the field are now testing these proteins to determine whether they can be used as a "cocktail" to regenerate lost hair cells and restore hearing.

"Hearing research has been going through a Renaissance period, not only from advances in genomics and methodology, but also thanks to its uniquely collaborative nature among researchers," said Hertzano.

The new study was funded by the National Institute on Deafness and Other Communication Disorders (NIDCD) which is part of the National Institutes of Health (NIH). It was also funded by the Binational Scientific Foundation (BSF).

"This is an exciting new finding that underscores the importance of basic research to lay the foundation for future clinical innovations," said E. Albert Reece, MD, PhD, MBA, executive vice president for medical affairs, UM Baltimore, and the John Z. and Akiko K. Bowers Distinguished Professor and dean, University of Maryland School of Medicine. "Identifying the complex pathways that lead to normal hearing could prove to be the key for reversing hearing loss in millions of Americans."

- This press release was originally published on theUniversity of Maryland School of Medicine website

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Researchers ID Role of Protein in Development of New Hearing Hair Cells - Lab Manager Magazine

Recommendation and review posted by Bethany Smith

When it comes to scientific advancements, there is no question that the past 100 years have been characterized by a number of innovations that have propelled us much farther into the future many of them in the medical field. One of those techniques that are considered groundbreaking by some but with skepticism by others, seems to be more like science-fiction rather than reality. Cryonics is the process of deep-freezing the bodies of people who have just died, in the hope that scientific advances may allow them to be revived in the future this has been regarded with skepticism within the mainstream scientific community but for others, it presents hope.

In 2015, Matheryn Naovaratpong became the youngest person to be cryogenically frozen at just two years old. Matheryn aka Einz was the second child of Sahatorn and Nareerat Naovaratpong, whose first child, Matrix, had wanted a younger sibling. The baby brought joy to the family when she was born. However, she developed a rare form of brain cancer just after her second birthday -- a form of cancer for which the survival rate was minimal if not zero. Sahatorn made the decision to cryogenically freeze his child, in the hopes that she could be revived in the future when there would be a cure for her cancer.

The Naovaratpong family's quest to give their child a chance to live and the aftermath of that decision is the subject of Netflix's latest documentary, 'Hope Frozen: A Quest to Live Twice'. Sahatorn passes on his dreams of reviving Einz to his son, Matrix, who himself holds up that dream with fervor. Matrix goes to visit an American scientist in the latter half of the documentary to learn how close they are to reviving those who have been cryogenically frozen. What he learns there is heartbreaking.

The scientist who worked on successfully freezing and reviving a rabbit tells him that current techniques mostly will not ensure that revival will be a successful process. The scientist stresses that the cell structure needs to be intact to ensure that the person being revived remains the same. However, with current techniques, he believes there will only be a 0.1% chance of success.

How exactly does cryonics work? Once the patient is declared clinically dead, cryonic technicians drain their blood and replace it with a solution designed to preserve organs, then follow it up with a cryoprotectant solution that freezes cells without causing the crystal formation that would damage them when returned to normal temperature. Bodies are then placed in tanks of liquid nitrogen for long-term storage -- the nitrogen must be regularly topped up.

The scientific community, however, is much more skeptical about the process, as we have seen in the documentary. What makes a person who they are, are their thoughts, memories, and their knowledge and the cryonics process could destroy the structure of the brain by dehydrating it. The method to recreate synaptic connections or the nervous system's workings in a virtual scenario is still an impossible task. A 2015 article from MIT Technology Review states that such a technology does not exist, even in principle, and says the cryonics industry is offering "an abjectly false hope that is beyond the promise of technology."

'Hope Frozen: A Quest to Live Twice' is now streaming on Netflix.

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'Hope Frozen': The cryonics industry offers strength to grieving families but does the technology actually wor - MEAWW

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Live Conference Call and Webcast at 4:30 ET

ENGLEWOOD, CO / ACCESSWIRE / September 16, 2020 / Aytu BioScience, Inc. (NASDAQ:AYTU) (the "Company"), a specialty pharmaceutical company focused on commercializing novel products that address significant patient needs, announced today that the Company will present its operational results for the fiscal fourth quarter ended June 30, 2020 on September 24, 2020, at 4:30 p.m. ET. The Company will review accomplishments from the quarter and provide an overview of its business and growth strategy.

Conference Call Information

1-877-407-9124 (toll-free)1-201-689-8584 (international)

The webcast will be accessible live and archived at the following link, https://www.webcaster4.com/Webcast/Page/2142/37506 and on Aytu BioScience's website, within the Investors section under Events & Presentations, at aytubio.com, for 90 days.

A replay of the call will be available for fourteen days. Access the replay by calling 1-877-481-4010 (toll-free) or 919-882-2331 (international) and using the replay access code 37506.

About Aytu BioScience, Inc.

Aytu BioScience is a commercial-stage specialty pharmaceutical company focused on commercializing novel products that address significant patient needs. The company currently markets a portfolio of prescription products addressing large primary care and pediatric markets. The primary care portfolio includes (i) Natesto, the only FDA-approved nasal formulation of testosterone for men with hypogonadism (low testosterone, or "Low T"), (ii) ZolpiMist, the only FDA-approved oral spray prescription sleep aid, and (iii) Tuzistra XR, the only FDA-approved 12-hour codeine-based antitussive syrup. The pediatric portfolio includes (i) AcipHex Sprinkle, a granule formulation of rabeprazole sodium, a commonly prescribed proton pump inhibitor; (ii) Cefaclor, a second-generation cephalosporin antibiotic suspension; (iii) Karbinal ER, an extended-release carbinoxamine (antihistamine) suspension indicated to treat numerous allergic conditions; and (iv) Poly-Vi-Flor and Tri-Vi-Flor, two complementary prescription fluoride-based supplement product lines containing combinations of fluoride and vitamins in various for infants and children with fluoride deficiency. Aytu also distributes a COVID-19 IgG/IgM rapid antibody test and rapid antigen tests. These assays are used in the rapid, qualitative diagnostic assessment of the 2019 Novel Coronavirus. Additionally, Aytu recently licensed worldwide rights to develop the Healight technology platform from Cedars-Sinai Medical Center. Healight is a pre-clinical medical device being studied as a prospective treatment for COVID-19 and other respiratory infections.

Aytu also operates a consumer health subsidiary, Innovus Pharmaceuticals, Inc. ("Innovus"), a specialty pharmaceutical company commercializing, licensing and developing safe and effective consumer healthcare products designed to improve men's and women's health and vitality. Innovus commercializes over thirty-five consumer health products competing in large healthcare categories including diabetes, men's health, sexual wellness and respiratory health. The Innovus product portfolio is commercialized through direct-to-consumer marketing channels utilizing the company's proprietary Beyond Human marketing and sales platform.

Aytu's strategy is to continue building its portfolio of revenue-generating Rx and consumer health products, leveraging its focused commercial team and expertise to build leading brands within large therapeutic markets. For more information visit aytubio.com and visit innovuspharma.com to learn about the company's consumer healthcare products.

Forward-Looking Statement

This press release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, or the Exchange Act. All statements other than statements of historical facts contained in this presentation, are forward-looking statements. Forward-looking statements are generally written in the future tense and/or are preceded by words such as ''may,'' ''will,'' ''should,'' ''forecast,'' ''could,'' ''expect,'' ''suggest,'' ''believe,'' ''estimate,'' ''continue,'' ''anticipate,'' ''intend,'' ''plan,'' or similar words, or the negatives of such terms or other variations on such terms or comparable terminology. These statements are just predictions and are subject to risks and uncertainties that could cause the actual events or results to differ materially. These risks and uncertainties include, among others: the regulatory and commercial risks associated with introducing the COVID-19 rapid tests, the accuracy of the COVID-19 rapid tests as compared to other COVID-19 tests, market acceptance of the tests, the ability to obtain FDA approval or authorization for the tests, our ability to obtain sufficient tests to meet consumer demand, if any, the manufacturers' ability to scale up manufacturing to meet customer demand, if any, reputation risks if the tests are not as effective as anticipated, and that the current regulatory environment continues to permit the sale of the tests. We also refer you to the risks described in ''Risk Factors'' in Part I, Item 1A of the company's Annual Report on Form 10-K and in the other reports and documents we file with the Securities and Exchange Commission from time to time.

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Aytu BioScience to Report Fourth Quarter FY 2020 Results and Business Update on Thursday, September 24, 2020 - Yahoo Finance

Recommendation and review posted by Bethany Smith

With an ever-growing CRISPR genome-editing toolbox, scientists are creating crops that can resist diseases and pests, withstand global warming, and offer better nutrition. The emergence of this technology offers a crucial opportunity for renewed public engagement around crop engineering. In order to actualize the potential of CRISPR-edited food, we must work together to create and share strategies for productive dialogue. This article identifies one area of necessary improvement in communication and public engagement.

Describing how CRISPR-edited crops are arguably more natural than GMOs, or how these crops could potentially use fewer chemicals than their GMO predecessors reinforces pervasive societal suspicions of GMOs. If we think that engineered crops will play a key role in addressing environmental and public health issues, then promoting CRISPR-edited crops at the expense of GMOs is short-sighted. Instead, we must use CRISPR as a new avenue for renewing productive discourse with the public. CRISPR offers a way to bring everyone back to the table, reintroducing voices into vital conversations that will impact us all.

The question, Is this safe? captures this tension between distancing CRISPR from GMOs in order to separate a new technology from its polarized relative, while not discarding GMOs and avoiding difficult conversations. Science communicators can use the question Is this safe? as a case study to further identify problematic practices and offer strategies for communication alternatives. Before answering this question, we must better understand the consumers decision-making process.

The processes behind engineering a CRISPR-edited crop and a GMO share many commonalities and, in some instances, lead to nearly identical outcomes .

In the wake of an incoming wave of CRISPR-edited crops, communicators have an opportunity to renew conversations surrounding what is natural, and in doing so, address concerns about naturalness and safety. For science communicators, do we suggest that CRISPR-edited crops are more natural? Do we explain how brands with a natural label dont always align with what consumers think they are buying? Or do we do we zoom out and try to separate natural from safe, so we dont tacitly buy into notions that GMOs are all unsafe?

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Promoting CRISPR crops at the expense of GMOs is short-sighted when we need both - Genetic Literacy Project

Recommendation and review posted by Bethany Smith