PHILADELPHIA, Oct. 28, 2020 (GLOBE NEWSWIRE) -- Scout Bio, a biotechnology company focused on revolutionizing pet medicine by delivering a pipeline of one-time therapeutics for major chronic pet health conditions, today announced it has initiated two pilot clinical studies to demonstrate effectiveness of an AAV expressing a GLP-1 analog (SB-009) in treating diabetic felines.

Scout Bio envisions two potential therapeutic applications for SB-009. First, to replace daily insulin injections with a single injection of SB-009 to treat feline diabetes and second to significantly increase the percentage of cats entering remission when SB-009 is given with insulin.

The two initiated pilot clinical studies will investigate each of these two potential treatment paradigms.

Mark Heffernan, Ph.D., Chief Executive Officer of Scout Bio remarked, This one-time injectable therapy for feline diabetes has the potential to provide a convenient single treatment that is an alternative to the burden of twice daily insulin injections. We believe SB-009 has the potential to be a blockbuster product for animal health and that our pipeline of gene therapy products for pets will disrupt and grow major markets.

SB-009 was developed under a collaboration between scientists at Scout Bio and the University of Pennsylvanias Gene Therapy Program, where the protein was engineered to improve potency, circulating accumulation kinetics and manufacturability. The design of SB-009 makes the clinical dose both affordable and commercially attractive.

Matthew Wilson, VP Product Discovery and External Innovation said, This is a further example of our strong collaborative relationship with UPenns Gene Therapy Program and Scouts internal capabilities of executing preclinical research to rapidly identify highly potent AAV gene therapies. In less than 12 months after initiating a discovery program, we are now in a position to transition into patients.

Scout Bio has conducted robust preclinical studies with various GLP-1 constructs in rodents and healthy cats. Key findings include:

Dr. Anne Traas, Scout Bios Chief Development Officer reflected, Owners can be devastated to learn their pet has this life-threatening disease and unfortunately, many are unable to give twice daily insulin injections and have to make the difficult choice to euthanize their beloved pet. A one-time safe and efficacious therapy, given by a veterinarian, that eliminates the need for insulin and worry of hypoglycemia, would greatly improve the current treatment paradigm and result in an improved quality of life for diabetic cats and their owners.

Feline diabetes, a severe disease lacking recent innovation, remains a major challenge for veterinarians and owners to safely and effectively manage. Diabetes in most cats is similar to type 2 diabetes in people. Insulin resistance, caused by factors such as obesity, leads to Beta-cell disfunction (the cells that produce insulin). Cats become insulin dependent when blood sugar levels rise, commonly 3-10 times normal, leading to the development of clinical signs which can seriously and negatively impact both the owner and the cat. The most common signs are increased drinking, increased urination and weight loss despite ravenous appetite.

About SB-009 SB-009 is a recombinant AAV gene therapy viral vector utilizing a novel capsid expressing an engineered feline GLP-1 agonist for the treatment of feline diabetes. GLP-1 has been shown to be a safe and highly efficacious molecule in the treatment of humans with type 2 diabetes and SB-009 is the first gene-therapy delivered GLP-1 to be studied in clinical studies in cats with a view to treat the disease.

The expressed feline GLP-1 analog protein functions by stimulating the beta-cells in the pancreas to produce more insulin and may also have an effect in decreasing insulin resistance. GLP-1 receptor agonists do not decrease glucose levels in animals with normal blood glucose, so there is expected to be a very low, or no risk of hypoglycemia.

About Feline DiabetesMost diabetic cats appear to have disease similar to human type 2 diabetes, which is primarily defined as a combined problem with insulin production by the beta-cells in the pancreas, as well as a decrease in the sensitivity to the normal action of insulin (insulin resistance). In cats, one of the most common factors contributing to insulin resistance is obesity which reduces insulin sensitivity.

Lack of insulin production and decreased sensitivity to insulin causes the glucose (sugar) in the blood to become very high leading to the clinical signs. Very high levels of blood glucose also hurt the beta-cells in the pancreas, leading to further reductions in insulin production.

Substantial progress has been made in the treatment of human type 2 diabetes, even in the early stages of the disease. However, insulin therapy remains the only FDA-approved treatment for diabetes in cats.

About Current Treatment | Feline DiabetesCurrent therapy aims to replace the insulin that the cats body no longer makes by injecting insulin twice daily. Giving insulin in the right amounts may bring the blood sugar levels down. If the blood glucose can be brought under control for the majority of a 24-hour period each day, then the clinical signs will be reduced to manageable levels. Too much insulin can cause the blood sugar to drop to dangerous levels (hypoglycemia), so there has to be a careful balance made between maintaining ideal blood glucose levels andadministering too much insulinwhich may result in life threatening low blood sugar levels.

Often owners find the prospect of administering injections to their cats daunting and the strict regimen of twice daily injections and feedings can be difficult to fit into a busy lifestyle. Unfortunately, not treating the cats is simply not a viable option and usually results in a rapid decline in physical health. Even with insulin treatment, some cats diabetes is not well controlled, resulting in the continuation of clinical signs and/or euthanasia.

About Diabetic RemissionGood control of blood glucose may also allow the beta-cells to rest. That rest may increase their capacity to regain some of their insulin-secreting ability. Insulin administration helps to decrease and control the excess blood glucose levels and complement whatever insulin producing ability the cat has left. In some cases, cats regain enough function to allow the insulin injections to stop. This is called diabetic remission. A cat is determined to be in remission when blood sugar is normal and there is complete correction of clinical signs once insulin has been discontinued.

About Scout BioScout Bio is a biotechnology company focused on revolutionizing pet medicine by delivering a pipeline of one-time therapeutics for major chronic pet health conditions. Scout Bios therapeutics are designed to induce long-term expression of therapeutic proteins in pet patients using AAV vector technology. Scout Bio has an exclusive research and development collaboration with the University of Pennsylvanias Gene Therapy Program. Scout Bios innovative partnerships build on a 20-year history with AAV leaders and is complemented by Scout Bios global leaders in gene therapy research and development. Scout Bio is a private company headquartered in Philadelphia, Pennsylvania. For more information, see http://www.scoutbio.coFor further information, please contact:

Investors:Sarah McCabeStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.com

Media:Fran Gaconnier214.417.4142Fran.gaconnier@scoutbio.co

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Scout Bio Advances Novel Gene Therapy for the Treatment of Feline Diabetes - BioSpace

DUBLIN, Oct. 28, 2020 /PRNewswire/ -- The "Competitive Landscape Analysis of Recent Cell and Gene Therapy Innovations" report has been added to ResearchAndMarkets.com's offering.

This research identifies some of the key developments across CAR-T cell therapies and provides insights across technological, IP, and investment landscapes. The study also provides an analysis of the competitive landscape while highlighting the key growth opportunities within the CAR-T cell therapy platform.

Key Topics Covered:

1.0 Executive Summary1.1 Research Focus: Emerging Technologies Enabling chimeric antigen receptor (CAR) T-cell Therapies1.2 Analysis Framework: The Author's Core Value1.3 Research Methodology: Five Steps Toward Success1.4 Key Findings of Technology Breakthrough Driving Sepsis Diagnosis

2.0 Technology Snapshot2.1 Rising Pace of Cell and Gene Therapy Approvals2.2 Regulatory and Ethical Perspectives on Gene Therapy2.3 Rising Demand for Precision Medicine Strategies2.4 Manufacturing Continues to be the Key Bottle Neck2.5 II Generation Chimeric Antigen Receptors Likely to Dominate the Cell Therapy Landscape in the Future2.6 CD-19 is the Most Common Target Antigen for Allogeneic CAR-T Therapies

3.0 Emerging Patent Landscape3.1 Steady Increase in Patent Grants for CAR-T Cell Therapies3.2 University of Pennsylvania and Novartis Lead the Patent Landscape for CAR-T Cell Therapies3.3 China and the US Lead the Patent Landscape for CAR-T Cell Therapies3.4 Snapshot of Key Patent Grants: Novartis3.5 Snapshot of Key Patent Grants: Cellectis and BlueBird Bio

4.0 Analysis of the Investment Landscape4.1 Key M&A Trends Across the Global Life Sciences Sector4.2 Gene Therapy - Venture Capital Funding Assessment4.3 Gene Therapy - Big Pharma In-licensing Deals Assessment4.4 Strategic Insights: Cell Therapies and Gene Therapies, Viral Vector CMOS

5. Analysis of the Competitive Landscape5.1 Allogene Therapeutics5.2 Precision BioSciences Inc.5.3 CRISPR Therapeutics AG5.4 Cellectis S.A.5.5 Celyad5.6 Bristol-Myers Squibb (BMS)5.7 Gilead5.8 Novartis5.9 BlueBird Bio5.10 Summary of the Scoring Methodology5.11 Competitive Analysis of CAR-T Participants

6.0 CAR-T Cell Therapies: Growth Opportunity Universe6.1 Growth Opportunity: CAR-T for Solid tumors, 2020

7.0 Industry Influencers

For more information about this report visit https://www.researchandmarkets.com/r/dgf6f3

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Analysis of Recent Cell and Gene Therapy Innovations: 2020 Competitive Landscape Report - CD-19 is the Most Common Target Antigen for Allogeneic CAR-T...

News Release

Tuesday, October 27, 2020

Researchers from the National Institutes of Health (NIH) have discovered a new inflammatory disorder called vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic syndrome (VEXAS), which is caused by mutations in the UBA1 gene. VEXAS causes symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in myeloid cells. The scientists reported their findings in the New England Journal of Medicine.

Nearly 125 million people in the U.S. live with some form of a chronic inflammatory disease. Many of these diseases have overlapping symptoms, which often make it difficult for researchers to diagnose the specific inflammatory disease in a given patient.

Researchers at the National Human Genome Research Institute (NHGRI), part of the NIH, and collaborators from other NIH Institutes took a unique approach to address this challenge. They studied the genome sequences from more than 2,500 individuals with undiagnosed inflammatory diseases, paying particular attention to a set of over 800 genes related to the process of ubiquitylation, which helps regulate both various protein functions inside a cell and the immune system overall. By doing so, they found a gene that is intricately linked to VEXAS, a disease which can be life-threatening. So far, 40% of VEXAS patients who the team studied have died, revealing the devastating consequences of the severe condition.

Usually, researchers discover a previously unknown disease by studying several patients with similar symptoms, then searching for a gene or multiple genes that may play a role in causing the disease. However, this was not a viable option for the NIH research team.

We had many patients with undiagnosed inflammatory conditions who were coming to the NIH Clinical Center, and we were just unable to diagnose them, said David B. Beck, M.D., Ph.D., clinical fellow at NHGRI and lead author of the paper. Thats when we had the idea of doing it the opposite way. Instead of starting with symptoms, start with a list of genes. Then, study the genomes of undiagnosed individuals and see where it takes us.

Out of the genome sequences of 2,560 patients with undiagnosed inflammatory conditions, over 1,000 patients had undiagnosed recurrent fevers and body-wide inflammation. The rest, part of the NIH Undiagnosed Diseases Network, had unusual and unclassified disorders.

Our objective was to see if any of the 2,560 patients shared variations in the same gene, said Daniel Kastner, M.D., Ph.D., scientific director of the Intramural Research Program at NHGRI and a senior author of the paper. Instead of looking at clinical similarities, we were instead taking advantage of shared genomic similarities that could help us discover a completely new disease.

Out of the 800 genes, one stood out. Three middle-aged males had rare and potentially damaging genomic variants in the UBA1 gene, but each of the three males appeared to have two copies of the UBA1 gene with one copy harboring the mutation, which was not unexpected because humans usually have two copies of every gene. However, the UBA1 gene resides in the X chromosome, and males have only one X chromosome (and one Y chromosome).

We were amazed to see this and wondered what it could mean. And thats when it clickedthis was only possible if there was mosaicism in these men, said Dr. Beck.

Mosaicism occurs when some people have groups of cells with mutations that are different from the rest of the body. The team predicted that there were specific cells in the patients bodies that carried the UBA1 gene in its normal form while other cells carried the gene in its mutated form.

Using DNA-sequencing methodologies, the researchers found that the mosaicism was indeed present in the patients myeloid cells, which are responsible for systemic inflammation and act as the first line of defense against infections.

The researchers then analyzed the genome sequences of additional individuals from various NIH cohorts and databases, which led to the discovery of an additional 22 adult males with the UBA1 gene mutations. Most of the individuals had symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in the myeloid cells.

Out of the combined 25 individuals, researchers were able to find a link between the various clinical rheumatologic and blood-related diagnoses made for the patients. Because these conditions exist in people with UBA1 mutations, the team grouped the various conditions into a new disease: VEXAS.

By using this genome-first approach, we have managed to find a thread that ties together patients carrying all of these seemingly unrelated, disparate diagnoses, Dr. Kastner said.

The researchers hope that this new genome-first strategy will help healthcare professionals improve disease assessments and provide appropriate treatments for thousands of patients who have various inflammation-related conditions. The study may also pave the way for a new and more appropriate classification of inflammatory diseases.

Additional research support for this study was provided by the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute of Dental and Craniofacial Research, the National Heart, Lung, and Blood Institute, National Institute of Allergy and Infectious Diseases, the National Cancer Institute and the NIH Clinical Center.

NHGRI is one of the 27 institutes and centers at the National Institutes of Health. The NHGRI Extramural Research Program supports grants for research, and training and career development at sites nationwide. Additional information about NHGRI can be found athttps://www.genome.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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Scientists use clues in the human genome to discover new inflammatory syndrome - National Institutes of Health

Image: SARS-CoV-2 viral particles (blue) in a clinical isolate. (Photo courtesy of CDC)

Researchers at Yale University (New Haven, CT, USA) and the Broad Institute of MIT and Harvard (Cambridge, MA, USA) screened hundred of millions of cells exposed to the SARS-CoV-2 and MERS viruses, and have identified dozens of genes that enable the viruses to replicate in cells, as well as those that seem to slam the door on the virus. The pro-viral and anti-viral roles of these genes will help guide scientists in development of new therapies to combat COVID-19, according to the researchers.

Scientists have identified how SARS-CoV-2 attaches to and invades cells, but less is known about why some cells are more susceptible to infection. Understanding the genetics behind host cells susceptibility to infection may help explain why some people exposed to the virus experience few or no symptoms while others become extremely ill or die. Researchers performed a genome-wide screen of a line of green monkey cells, which are more sensitive to SARS-CoV-2 infection than commonly used human cell lines. The screens for the first time allowed researchers to simultaneously track interactions of virus and cells. The screens confirmed earlier findings that the ACE2 gene, which encodes a receptor on the cell surface, promotes infection by SARS-CoV-2.

However, the screens also identified two previously unknown pro-viral factors, as well as a third that seems to assist in preventing infection. They found that members of the SWI/SNF protein complex, which turns genes on and off, and HMGB1, which has a myriad of functions including regulation of inflammation, were linked to increased cell death after infection. The researchers then introduced small molecule drugs that inhibit function of two of the identified gene products, and found they could increase survival of cells after infection in a dish. By contrast, the histone H3 complex, which helps regulate expression of genes within the cell nucleus, seemed to provide a protective effect, inhibiting the ability of SARS-CoV-2 to infect and kill cells.

It is very important to understand wide variation of responses to COVID-19, for instance why advanced age makes it much more likely that people will die, said Yales Craig Wilen, assistant professor in laboratory medicine and immunobiology. We have identified both proviral and antiviral genes that may help us predict who is likely to get severely ill and what kind of drugs would be helpful or detrimental in treating patients.

Related Links:Yale University Broad Institute of MIT and Harvard

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Discovery of Helpful and Harmful COVID-19-Related Genes to Aid Development of New Therapies - HospiMedica

NEW YORK, Oct. 22, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that it will host a virtual R&D Day on Friday, October 30, 2020 at 11:30 AM Eastern time, to discuss the Companys AXO-Lenti-PD gene therapy for Parkinsons disease.

Axovants Parkinsons disease R&D Day will be moderated by Chief R&D Officer, Gavin Corcoran, M.D., and will feature presentations on the current treatment landscape and unmet medical need for people living with Parkinsons disease from the following key opinion leaders:

In addition, the Company will present data from the second cohort of the Phase 2 SUNRISE-PD trial for AXO-Lenti-PD including:

Drs. Adler, Palfi, and Eberling will be joined by Dr. Corcoran to answer questions following the formal presentations.

AXO-Lenti-PD is the only investigational gene therapy for Parkinsons disease that delivers three genes via a lentiviral vector to encode a set of critical enzymes required for endogenous dopamine synthesis, with the goal of improving motor function and restoring steady, tonic levels of dopamine in the brain. The gene therapy aims to provide patient benefit for years following a single administration.

To register for the R&D webcast, please click here.

A live audio webcast of the R&D Day can be accessed through the Events & Presentations section of the company's website at investors.axovant.com. An archived replay of the webcast will be available on the company's website following the event.

Biographies of R&D Day Panelists:

Dr. Adler has received numerous grants to investigate experimental treatments for Parkinson's disease, essential tremor, dystonia, restless legs syndrome, and chronic traumatic encephalopathy (CTE). He serves as an advisory member to many different international medical societies such as the International Parkinson and Movement Disorder Society, MDS Industry Education and Services Committee, and the American Academy of Neurology Section of Movement Disorders. Dr. Adler has a commitment to education having trained residents, 14 fellows and graduate students, and has given many invited lectures. Dr. Adlers main research interests are investigating tissue diagnostic tests for Parkinsons disease, biomarkers for an early diagnosis of Parkinsons disease and PD with dementia, and identification of new treatments for PD and PD with dementia. He also has been investigating essential tremor, restless legs syndrome, and dystonia. He has published over 400 research papers and reviews, and edited a book entitled Parkinson's Disease and Movement Disorders: Diagnosis and Treatment Guidelines for the Practicing Physician. In 2006, Dr. Adler was awarded the Mayo Clinic Distinguished Investigator of the Year Award.

Dr. Palfi has published extensively on trophic factor- and enzyme-based gene therapy in Parkinsons disease and Huntingtons disease. He is a principal investigator on numerous preclinical and clinical studies and has been involved in studies of many novel agents including implanted brain devices, optogenetic, homeoprotein, trophic factors GDNF, CNTF and dopamine lentiviral vectors.

Dr. Eberling earned undergraduate and graduate degrees in biological psychology from the University of California at Berkeley, later moving to the Lawrence Berkeley National Laboratory where she developed expertise in neuroimaging techniques and gene therapy approaches for Parkinsons disease.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523investors@axovant.com

Media

Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.commedia@axovant.com

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Axovant Gene Therapies to Host Virtual Parkinson's Disease R&D Day on October 30, 2020 - GlobeNewswire

HARRIS COUNTY, Ala. (WRBL) A Harris County family is celebrating overcoming a major obstacle in treating their 8-year-old-son who has a rare form of Muscular Dystrophy, thanks to a village of supporters and a phone call from the Chief Medical Officer of a major insurance company.

8-year-old Jacob Saalman is like most kids. He loves being outdoors, riding four-wheelers, and playing baseball. This Halloween, he carved pumpkins with his family. Jacob loves his dad, a former Fort Benning Ranger, and his mom. Jacob adores his four-year-old brother, Hudson.

Jacob is very protective of Hudson. Hes a mother hen. Oftentimes I have to tell Jacob to stop parenting, shared Ryan and Brooke Saalman, Jacobs parents.

However, Jacob is not like most kids when it comes to his health. Jacobs handled more medical procedures, biopsies, and tests than many adults. Its heartbreaking. Jacobs mom and dad say hes resilient with a quiet strength.

I would say he is very strong and adaptable. All the things he has to do, all the medical stuff he has been through, pokes and prods and port placement and muscle biopsies. He has been through a lot, said Brooke.

Jacob is very sick. So is his brother Hudson. Both boys have Duchenne Muscular Dystrophy, a rare genetic disease, preventing their bodies from making a protein called Dystrophin that protects our muscles from breaking down with use.

Jacob plays ball now. By 12, he will most likely need a wheelchair. After that, the prognosis remains bleak for the brothers and the parents who love them.

The life expectancy is upper 20s and into the 30s. But you are not walking around or living a normal life. You are most likely on a ventilator and having a parent care of you 24/7, shared Ryan.

Jacobs medical team is convinced an FDA approved home infusion called Exondys 51 will significantly improve Jacobs quality of life by helping him produce partial Dystrophin proteins.

Its not a cure. It slows the rate of the disease, and also its been proven to help kids walk longer, and it is also shown to improve pulmonary function, and thats the issue later on down that causes a lot of problems and even death from this disease, said Brooke.

The Saalmans goal is to stave off the disease for as long as possible. They believe Exondys 51 can help keep Jacob as healthy as possible until a gene therapy is ready.

We still have hope. What gives us the most hope is there are gene therapy trials in phase three. So we are hoping within the next two or three years we will be able to have a gene therapy that will make this a much milder disease, said Brooke.

One of Jacobs physicians, Han C. Phan, MD, with Rare Disease Research LLC in Atlanta, Georgia, shared this statement with News 3:

Jacob has Duchenne Muscular Dystrophy, a slowly progressive condition for which without treatment could limit his life expectancy. Every child with DMD should receive the treatment he deserves and without such could be detrimental to his overall health.

The family says Horizon Blue Cross Blue Shield of New Jersey had twice denied Jacob Exondys 51, despite other major insurance companies approving similar patients treatment. The drug costs 300,000 a year. The company listed several reasons for denying coverage for the medicine in a letter sent to the Saalman family.

Ryan Saalman shared the familes heartbreak and frustration on his Facebook page earlier in the week. The status update was shared several thousand times and sent to News 3.

Monday, the Sallman family spoke with News 3 about their struggle since April to get the medication approved with their insurance company.

Its just frustrating to know there is a child that has this need, and we pay good money for our insurance. You think that if a doctor says this is medically necessary that they will get the medication, and thats not the case. These kids deserve that. Everything they go through. They deserve some help, said Brooke.

News 3 reached out to Horizon Blue Cross Blue Shield of New Jersey on Monday. Tuesday, News 3 worked with The Saalman family and Horizon BCBS of New Jersey to get the necessary medical privacy forms filled out to get information on the insurance companys case. However, before the process was complete, The Saalman family says they got a phone call from the insurance companys Chief Medical Officer. The family was thrilled to learn Exondys 51 has been approved for Jacob.

Today, I think we truly saw the power of community. In this fight against Duchenne Muscular Dystrophy, it takes a village, and I think our village got really big really fast. Our new village gave an 8-year-old boy a loud enough voice that Horizon BCBS has overturned last weeks denial. Jacob will be able to get the medication he needs. The Saalman Family wants to thank the thousands of people that shared our story; we truly believe it is because of all of you that this has happened,

The Saalman family is overjoyed. Time is not on their side as this disease doesnt slow down. With each delay comes another day Duchenne Muscular Dystrophy robs Jacobs muscles of their ability to function. The family hopes the newly approved infusion will be ready for Jacob within a few weeks. The treatment should help slow the diseases progression down and give the family the time they need to wait for a promising gene therapy they pray is on the horizon.

The day before The Saalman family learned the medication had been approved, Jacob hit the winning RBI for the final game of his baseball season in a tie game with two outs. Jacob got the game ball in the final game of what may be his last season of baseball.

And sometimes God shows up in the last game, in the last inning, with two outs and two strikes on the board, when the hope of a win is almost gone and He crushes our expectations. Man, did I ever need that reminder tonight, and maybe you do too, shared Jacobs father on Facebook.

For more information on Jacob and Hudsons battle with Duchenne MD and ways you can help, you can visit their blog: Saalman Strong. The family has been encouraged to establish a GoFundMe page: Jacob and Hudsons fight against Duchenne MD

News 3 will keep you updated on the Saalman family and the journey with their sons Jacob and Hudson.

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Parents win insurance appeal for 8-year-olds Muscular Dystrophy treatment - WWTI - InformNNY.com

Newswise PITTSBURGH, Oct. 26, 2020 James J. Collins, Ph.D., an innovator in synthetic biology whose ideas have contributed to novel diagnostics and treatments targeting infections and complex diseases, has been awarded the 2020 Dickson Prize in Medicine, the University of Pittsburgh School of Medicines highest honor.

The prize is given annually to an American biomedical researcher who has made significant, progressive contributions to medicine. The award consists of a specially commissioned medal, a $50,000 honorarium and an invitation to present the keynote lecture during the Universitys annual campus-wide showcase of scientific research. Due to the COVID-19 pandemic, both the annual showcase and Collins lecture have been postponed until 2021 at a date to be determined.

Dr. Collins is defining whats possible in the disciplines of synthetic and systems biology. His highly creative work applying engineering design principles to molecular biology has generated numerous new diagnostics and therapeutics with wide application to medicine, said Anantha Shekhar, M.D., Ph.D., Pitts senior vice chancellor for the health sciences and John and Gertrude Petersen Dean of Medicine. It is our honor to recognize him with the School of Medicines most prestigious award.

Im grateful to work with outstanding lab members and collaborators whose dedication and insight have been critical to what weve achieved, said Collins, who is the Termeer Professor of Medical Engineering and Science in the Department of Biological Engineering at Massachusetts Institute of Technology and is affiliated faculty with the Broad Institute of MIT and Harvard University, and the Wyss Institute at Harvard. I am thrilled and honored to receive the Dickson Prize in Medicine.

A seminal 2000 publication describing the successful creation of a stable, synthetic gene circuit in Escherichia coli bacteria has been cited more than 4,000 times and marked the arrival of an important new discipline in biomedicine. Collins later demonstrated that synthetic gene networks could be linked with a cells genetic circuitry as a regulatory mechanism to create programmable cells for biomedical applications.

More recently, Collins has created engineered microbes and whole-cell biosensors to serve as in vivo diagnostics and therapeutics. One innovative platform that he and colleagues developed embeds freeze-dried, cell-free synthetic gene networks onto paper and other materials with a wide range of potential clinical and research applications.

The resulting materials contain properties of a living cell, are stable at room temperature and can be activated by simply adding water. Collinss work on freeze-dried, cell-free synthetic biology has established a platform for a new class of rapid, programmable in vitro diagnostics for emerging pathogens, including drug-resistant bacteria and viruses. Collins and his team currently are developing a rapid self-activating COVID-19 face mask as a wearable diagnostic.

Collins earned an A.B. in physics at the College of the Holy Cross in Worcester, Mass., before completing a Ph.D. in medical engineering at the University of Oxford with the distinction of Rhodes Scholar. He has received a MacArthur Foundation Genius award, NIH Directors Pioneer Award and Sanofi-Institut Pasteur Award. Collins is an elected member of the National Academy of Sciences, National Academy of Engineering, National Academy of Medicine and the American Academy of Arts and Sciences. He is a charter fellow of the National Academy of Inventors.

To read this release online or share it, visit http://www.upmc.com/media/news/102620-Dickson-Prize-2020.

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2020 Dickson Prize in Medicine Awarded to Pioneer Researcher in Synthetic Biology - Newswise

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Foundation Medicine, Inc. today announced that the U.S. Food and Drug Administration (FDA) approved FoundationOneLiquid CDx for three new companion diagnostic indications to help match patients who may benefit from treatment with specific FDA-approved targeted therapies. The new indications are for Piqray (alpelisib) in advanced or metastatic breast cancer; Rubraca (rucaparib) in advanced ovarian cancer; and Alecensa (alectinib) in a certain type of metastatic non-small cell lung cancer (mNSCLC). The FDA also approved a label expansion for FoundationOne Liquid CDx to report additional select copy number alterations and genomic rearrangements.

FoundationOne Liquid CDx analyzes the largest genomic region of any FDA-approved comprehensive liquid biopsy test and was approved in August to provide tumor mutation profiling in accordance with professional guidelines for patients with any solid tumor. Concurrently, it was approved as a companion diagnostic for a poly (ADP-ribose) polymerase (PARP) inhibitor approved by the FDA for the treatment of metastatic castration-resistant prostate cancer patients with qualifying BRCA1/2 alterations, and for three first-line EGFR tyrosine kinase inhibitors (TKIs) for the treatment of non-small cell lung cancer patients.

FoundationOne Liquid CDx offers oncologists an important and minimally invasive tool to consider when making treatment decisions for their patients, regardless of the type of cancer they have, said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. These three additional companion diagnostic claims expand the tests clinical utility into breast and ovarian cancer, demonstrating our commitment to bringing precision medicine to more patients, and we plan to continue working with our biopharma partners to increase that reach.

Todays approval expands FoundationOne Liquid CDxs companion diagnostic indications to include the following targeted therapies:

PIK3CA is the most commonly mutated gene in HR+/HER2- breast cancer; approximately 40% of patients living with HR+/HER2- breast cancer have this mutation.1

An estimated one in four women with epithelial ovarian cancer have a mutation of the BRCA1 or BRCA2 gene.2

Using a blood sample, FoundationOne Liquid CDx analyzes over 300 cancer-related genes for genomic alterations. FoundationOne Liquid CDx results are delivered in an integrated report that identifies alterations matched to FDA-approved therapies. It also enables accelerated companion diagnostic development for biopharma companies developing precision therapeutics.

As a laboratory professional service which has not been reviewed or approved by the FDA, the FoundationOne Liquid CDx report delivers information about the genomic signatures microsatellite instability (MSI) and blood tumor mutational burden (bTMB), as well as single gene alterations, including NTRK fusions, to help inform the use of other therapies including immunotherapies. Also, as a laboratory professional service, the report provides relevant clinical trial information and includes interpretive content developed in accordance with professional guidelines in oncology for patients with any solid tumor.

About FoundationOne Liquid CDx

FoundationOne Liquid CDx is a qualitative next generation sequencing based in vitro diagnostic test for prescription use only that uses targeted high throughput hybridization-based capture technology to analyze 324 genes utilizing circulating cell-free DNA (cfDNA) isolated from plasma derived from anti-coagulated peripheral whole blood of advanced cancer patients. The test is FDA-approved to report short variants in over 300 genes and is a companion diagnostic to identify patients who may benefit from treatment with specific therapies (listed in Table 1 of the Intended Use) in accordance with the approved therapeutic product labeling. Additional genomic findings may be reported and are not prescriptive or conclusive for labeled use of any specific therapeutic product. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Patients who are negative for companion diagnostic mutations should be reflexed to tumor tissue testing and mutation status confirmed using an FDA-approved tumor tissue test, if feasible. For the complete label, including companion diagnostic indications and complete risk information, please visit http://www.F1LCDxLabel.com.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine and FoundationOne are registered trademarks of Foundation Medicine, Inc.

PIQRAY is a registered trademark of Novartis AG.

RUBRACA is a registered trademark of Clovis Oncology, Inc.

ALECENSA is a registered trademark of Chugai Pharmaceutical Co., Ltd., Tokyo, Japan.

Source: Foundation Medicine

1 The Cancer Genome Atlas Network. Comprehensive molecular portraits of human breast tumours. Nature. 2012;490(7418):61-70.2 Pennington et al, Clin Cancer Res. 2014; 20(3):764-7753 Dearden et al. Ann Oncol. 2013 Sep; 24(9): 23712376.

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FDA Approves New FoundationOneLiquid CDx Companion Diagnostic Indications for Three Targeted Therapies That Treat Advanced Ovarian, Breast and...