Category Archives: Gene Medicine

CAMBRIDGE, Mass., June 30, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the retirement of Sandy Mahatme, Sareptas executive vice president, chief financial officer and chief business officer, from the company effective July 10, 2020. The company has commenced a search process to identify the future chief financial officer. During the interim period, the finance and accounting functions will report directly to Sareptas Chief Executive Officer, Doug Ingram, and other departments reporting to Mr. Mahatme will be overseen by members of Sareptas executive committee.

The Sarepta from which Sandy retires is a very different one from the organization he joined as our chief financial officer some eight years ago. And the Sarepta of today a financially solid biotechnology organization with perhaps the industrys deepest and most valuable pipeline of genetic medicine candidates with the potential to extend and improve lives would not have been possible without Sandys business acumen and dedication, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. On behalf of our board of directors and the entire organization, I want to wish Sandy all the best in his next journey and thank him for his invaluable and numerous contributions to our success and for having built a strong team of finance leaders who will continue to perform as he departs.

Said Mr. Mahatme, It has been a privilege to serve as Sareptas CFO and CBO for almost eight years and to have participated in its remarkable transformation and extraordinary growth. Working with this leadership team and our talented colleagues, we have built a strong foundation for Sareptas ongoing success in achieving its goal of changing the lives of patients with rare diseases around the world. Having built a strong team of finance, IT, facilities, manufacturing and business development professionals, I feel confident that this is a good time to transition to other opportunities, knowing that Sarepta is well-positioned to continue to lead the industry.

Sandy will continue to serve on the Board of Directors for Flexion Therapeutics, Inc., Aeglea BioTherapeutics, Inc., and Idorsia Pharmaceuticals Ltd.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking StatementThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the search process to identify the future chief financial officer, the reporting structure during the interim period and the performance of the finance team; Sareptas potential to extend and improve lives; Sareptas goal of changing the lives of patients with rare diseases around the world; and Sarepta being well-positioned to continue to lead the industry.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics Announces Retirement of Sandy Mahatme, Chief Financial Officer and Chief Business Officer - GlobeNewswire

Newswise Massachusetts Eye and Ear, a member hospital of Mass General Brigham, is entering into an exclusive licensing agreement with Biogen to develop a potential treatment for inherited retinal degeneration due to mutations in the PRPF31 gene, which are among the most common causes for autosomal dominant retinitis pigmentosa.

Inherited retinal degenerations (IRDs), such as retinitis pigmentosa, are a group of blinding eye diseases caused by mutations in over 270 different genes. Mutations in the PRPF31 gene are the second most common cause of dominant IRD and lead to defects in the function of the retinal pigment epithelial (RPE) cells and photoreceptors of the retina. Previous lab-based research performed by members of the Ocular Genomics Institute at Harvard Ophthalmology, led by Eric A. Pierce, MD, PhD, demonstrated that adeno-associated virus (AAV)-mediated gene augmentation therapy for PRPF31 can restore normal function to PRPF31 mutant RPE cells.

Biogen (Nasdaq: BIIB), a biopharmaceutical company that discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies, will build upon this prior work, and conduct the studies needed for clinical development of PRPF31 gene therapy. This includes the pre-clinical studies needed to support progression to clinical trials of PRPF31 gene therapy. As part of the agreement, Biogen will receive an exclusive license to develop the product worldwide and will be responsible for all U.S. Food and Drug-Administration (FDA) required investigational new drug (IND) enabling studies, clinical development and commercialization.

The treatment of IRDs with highly effective AAV-based gene therapies is core to Biogens ophthalmology strategy, said Chris Henderson, Head of Research, Biogen. This agreement underscores our commitment to that strategy and builds off of our acquisition of Nightstar Therapeutics in 2019 and our active clinical trials of gene therapies for different genetic forms of IRD. We are excited to work with Massachusetts Eye and Ear and look forward to applying our preclinical and clinical experience to their leading PRPF31 program.

We are thrilled to work with Biogen, who will bring to this effort its deep experience with the clinical development process, as we work toward our goal of developing a gene therapy for people with PRPF31-related eye disease, added Dr. Pierce, who is the William F. Chatlos Professor of Ophthalmology at Harvard Medical School. My ultimate hope for patients with inherited retinal disorders due to mutations in PRPF31 is that a gene therapy will preserve and potentially restore some of their vision.

About the Ocular Genomics Institute

The Ocular Genomics Institute at Harvard Ophthalmology aims to translate genomic medicine into precision ophthalmic care for patients with inherited eye disorders. It is home to one of the leading centers for early-phase clinical trials of therapies for inherited retinal degenerations, with seven gene-based and one stem cell trial currently in progress. The group works in conjunction with other departments throughout Harvard Medical School and Mass. Eye and Ear, including the Bioinformatics Center and Grousbeck Gene Therapy Center.

Dr. Pierces lab, established in 2011, is dedicated to research in an effort to improve the understanding of the molecular bases of IRDs so that rational therapies can be developed for these diseases.

In 2018, Mass. Eye and Ear surgeons performed the first post-FDA approval gene therapy for patients with a form of inherited retinal blindness caused by mutations in the gene RPE65 by injecting an AAV-based drug treatment into a patients eye, which restored vision in a 13-year-old boy. This therapy, called Luxturna, is now being used to treat patients with RPE65-associated retinal degeneration around the world.

One of the exciting aspects of our collaboration with Biogen is that mutations in the PRPF31 gene affect approximately 10 to 20 times more people than mutations in the RPE65 gene, said Dr. Pierce. Success with PRPF31 gene therapy could provide visual benefit to more patients, which is our ultimate goal.

Mass. Eye and Ear was one of the first centers to offer life-changing gene therapies to patients with inherited retinal disease, and we are thrilled with this new opportunity to develop a translational retinal therapy that could help even more patients, said Joan W. Miller, MD, Chief of Ophthalmology at Mass. Eye and Ear, Massachusetts General Hospital, and Brigham and Womens Hospital, and Chair of Ophthalmology and the David Glendenning Cogan Professor of Ophthalmology at Harvard Medical School.

According to Chris Coburn, Chief Innovation Officer, Mass General Brigham, the collaboration with Biogen illustrates the importance of academia and industry teaming to solve problems for patients worldwide. We are eager to see this progress reach patients who are challenged by blinding, degenerative eye disease, said Coburn. We look forward to working with Biogen to advance this break-through innovation.

Patients with an inherited retinal disease require genetic testing prior to being considered for any gene therapy treatment.

About Massachusetts Eye and Ear

Massachusetts Eye and Ear, founded in 1824, is an international center for treatment and research and a teaching hospital of Harvard Medical School. A member of Mass General Brigham, Mass. Eye and Ear specializes in ophthalmology (eye care) and otolaryngologyhead and neck surgery (ear, nose and throat care). Mass. Eye and Ear clinicians provide care ranging from the routine to the very complex. Also home to the world's largest community of hearing and vision researchers, Mass. Eye and Ear scientists are driven by a mission to discover the basic biology underlying conditions affecting the eyes, ears, nose, throat, head and neck and to develop new treatments and cures. In the 20192020 Best Hospitals Survey,U.S. News & World Reportranked Mass. Eye and Ear #4 in the nation for eye care and #2 for ear, nose and throat care.For more information about life-changing care and research at Mass. Eye and Ear, visit our blog,Focus, and follow us onInstagram,TwitterandFacebook.

About Harvard Medical School Department of Ophthalmology

The Harvard Medical SchoolDepartment of Ophthalmologyis one of the leading and largest academic departments of ophthalmology in the nation. Composed of nine affiliates (Massachusetts Eye and Ear, which is home to Schepens Eye Research Institute; Massachusetts General Hospital; Brigham and Womens Hospital; Boston Childrens Hospital; Beth Israel Deaconess Medical Center; Joslin Diabetes Center/Beetham Eye Institute; Veterans Affairs Boston Healthcare System; Veterans Affairs Maine Healthcare System; and Cambridge Health Alliance) and several international partners, the department draws upon the resources of a global team to pursue a singular goaleradicate blinding diseases so that all children born today will see throughout their lifetimes. Formally established in 1871, the department is committed to its three-fold mission of providing premier clinical care, conducting transformational research, and providing world-class training for tomorrows leaders in ophthalmology.

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Massachusetts Eye and Ear Enters Licensing Agreement with Biogen to Develop Treatment for Inherited Retinal Disorder - Newswise

Christine Stanley, Ph.D., Chief Director of Clinical Genomics at Variantyx

The answer to questions about human disease can be found in our genes. The difficulty in the past has been the testing process, a sort of trial and error approach of drilling down into the multitude of variants that can be found within the genes, variants that when analyzed in tandem with detailed clinical histories can actually tell the story and lead to a faster diagnosis.

Human beings carry around 20,000 genes and, of those, approximately 5,000 are somewhat understood, and those genes can be associated with several diseases and each disease can be associated with dozens of clinical symptoms or more. It was believed that five percent or less of the human population carry variants involved in genetic diseases. But a recent study in the Annals of Internal Medicine, now suggests the number of people with variants linked to genetic diseases is closer to 20 percent. Many other factors may determine whether an individual actually develops a disorder, but these numbers suggest the acceptance of a new approach that provides the most useful diagnostic data from a single test thats easier on the patients and families and provides the shortest time to a diagnosis and the best chance at implementing treatments.

Here is an important reason. Parents with children suspected of having a genetic disease routinely face a diagnostic odyssey that typically lasts five to seven years and entails seeing an average of seven different physicians. Its an odyssey that comes with an average cost of diagnosis reaching $21,099, more than seven times the cost of a single whole-genome sequencing test.

Historically, genetic testing has been really disjointed. Tests that were developed 10 to 15 years ago are still being run today by laboratories. These tests target extremely specific areas for an exceedingly small number of changes that cause a certain disease. It is like looking under a lamp post. And an individual, who is suspected of having the disease, will be tested for one particular variant or a small number of variants. It is an approach that is lacking in quick, definitive, and accurate results. Unless the tested area accounted for the majority of the disease-causing variants, it then forces the ordering of more tests to try to find other causes of the disease, either within that same gene or within other genes. This is happening sequentially, so the patient keeps receiving negative results, and then additional tests are ordered and the merry-go-round can continue for years. It cost families financially and emotionally. Delaying the time to diagnosis can also close the effective treatment window in cases where early treatment is important for a good prognosis.

Ordering a single whole-genome sequencing (WGS) test right off the bat replaces almost all of those long, cumbersome, and costly processes. It all but eliminates having to endure multiple genetic tests because a patient needs only one sample and one turnaround time for the greatest chance to arrive at the correct diagnosis. More importantly, if the test results were negative and then a new gene associated with the patients disease is reported the next day, and that patient has a variant in that gene, a clinician can make that connection by reanalyzing the data rather than by bringing the patient back in for a new sample. In that way, genomic testing has really revolutionized the entire genetic testing industry by providing a comprehensive analysis with the shortest time to diagnosis.

Whole-genome sequencing does not require the mechanical step of isolating genes first. It enables the identification of different types of variants that labs do not typically see when one isolates genes. It also enables the use of sophisticated algorithms applied via software to allow for the ranking of variants in a way that pulls variants that are known to cause the disease to the top of the list for examination. Variants can also be ranked by looking at the severity of the effect of the variant on genes that most closely match the patients clinical symptoms. Those results are parsed based on the known inheritance patterns of these genes. Patients can be looked at through both of those lenses at the same timethe severity of the changes that are identified, and the changes that match with the clinical symptoms of the patient.

Whole-genome testing will soon become the first line of defense, rather than a last resort for families or individuals seeking clarity on genetic diseases because of its ability to incorporate sophisticated bioinformatics and data interpretation. It is a faster route for the proper diagnosis and treatment for both early-onset diseases like epilepsy and intellectual disabilities, as well as late-onset disorders like ataxia and ALS. It can be used to diagnose almost any genetic disorder spanning such areas as neurology, endocrinology, nephrology, hearing and vision loss, blood disorders like thalassemia, muscular dystrophy, etc. While insurance reimbursement can be challenging today, the insurance payers will come around, as they have always done in the past, because this test saves time, money, and supports better outcomes for patients.

About Christine Stanley, Ph.D.

Christine Stanley, Ph.D., is the Chief Director of Clinical Genomics for Variantyx, a provider of highly specialized genetic testing to clinicians and their patients. Christine is responsible for overseeing clinical genomic interpretations and regulatory compliance for the clinical laboratory.

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Eliminating the Long, Cumbersome and Costly Diagnosis of Genetic Diseases - HIT Consultant

DUBLIN, July 1, 2020 /PRNewswire/ -- The "Rare Disease Diagnostics: Technologies and Global Markets" report has been added to ResearchAndMarkets.com's offering.

The global rare disease diagnostics market should reach $26.7 billion by 2024 from $17 billion in 2019, rising at a CAGR of 9.5% over the forecast period.

The scope of the report includes rare disease diagnostic technologies, applications, industries, initiatives, patents and companies. The market for rare disease diagnostic products and services is given for 2018 and 2019, and then forecast through 2024.

This report reviews the main diagnostic technologies and explains why genetic variation is important in clinical testing and disease. It then discusses significant large-scale research initiatives that impact rare disease diagnostic applications. Of particular interest is a discussion of global population-scale sequencing projects and their likely impact in linking genetic variation to rare disease diagnostics. The main market driving forces for rare disease diagnostic products and services are listed and discussed.

The report categorizes and quantifies the rare disease diagnostics market by the disease category, technology platform, test purpose, analysis target and geography segments.

More than 95 companies in the rare disease diagnostic industry are profiled in this report.

The research also provides a summary of more than 50 of the main industry acquisitions and strategic alliances that took place from April 2018 through April 2020, including key alliance trends.

The report includes:

Market Insights

Rare diseases comprise a growing public health priority, as they affect upward of 300 million people globally and they are difficult to diagnose and treat.

There is a pressing need for better ways to detect and diagnose rare diseases, as well as to provide companion diagnostics for therapy guidance, clinical trials enrollment and therapy monitoring applications.

Better diagnostic tests for rare diseases can make significant differences in the lives of those affected by these conditions. Many rare diseases go undiagnosed for long periods of time because patients, families and physicians may have limited awareness of certain diseases, and the symptoms may not be informative to healthcare workers who may not have encountered such diseases before.

Extended time to diagnosis of a rare disease, along with so-called diagnostic odysseys, can lead to negative outcomes, including misdiagnosis or disease progression. Rapid, accurate diagnostics can significantly shorten these diagnostic odysseys.

In addition to early detection and diagnostic potential, rare disease therapeutics will be important in orphan drug development and use. Orphan drugs address rare disease patient populations, and they are expected to have a high growth rate through 2024. By 2024, orphan drugs may make up as much as one-fifth of global prescription sales. Rare disease diagnostics can be used to help physicians make proper decisions regarding which therapies to use and ways to monitor the efficacy of those therapies during treatment courses. Rare disease diagnostics can also be used to help select patients for orphan drug clinical trials.

More than 70% of rare diseases are inherited conditions, and they thus have genetic components, so this industry relies heavily on genetic analysis methods, including polymerase chain reaction (PCR), next-generation sequencing (NGS) and Sanger sequencing.

Key Topics Covered

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Overview

Chapter 4 Technology Background

Chapter 5 Rare Disease Diagnostics Initiatives

Chapter 6 Rare Disease Diagnostic Industries

Chapter 7 Rare Disease Diagnostics Strategic Alliances and Acquisitions

Chapter 8 Rare Disease Diagnostics Markets

Chapter 9 Rare Disease Diagnostics Patents and Intellectual Property

Chapter 10 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/rp6ok6

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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Rare Disease Diagnostics Industry Anticipated to Reach $26.7 Billion by 2024 - Market Shares by Disease Class, Indication, Analysis Platform, Analysis...

A special thank you to Kathleen Dickson and Dr. John Bergeron for pointing out that yes, indeed, there are also many women who have made and continue to make significant contributions to health. We have added their additions below, but this list is by no means complete.

From open heart surgery to child-resistant containers, prestigious awards and bombs (not that kind), Canada has a long history of Canadians whose ideas and inventions have played huge roles in defining this nations healthcare.

DNA and cancer

Nada Jabado at McGill affiliated Childrens Hospital is a pioneer in pediatric cancer and her discovery of the role of what is known as the epigenome that marks the DNA in our genes in cancer. She is a leader in innovation in Health research and recognized for her leadership in the application of discoveries to address brain tumours in children.

Insulin

Perhaps the most famous health innovation to come out of Canada, if such a thing can be measured. The arrival of insulin has saved countless lives since its creation in 1922 when Frederick Banting and Charles Best isolated and extracted insulin from the pancreas of dogs. Their Nobel Prize arrived swiftly thereafter in 1923.

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28 cool health things that started with a Canadian - Regina Leader-Post

Azure pools rich in magnesium and calcium carbonate but low in phosphorus provide an ideal habitat for ancient bacterial reefs at Cuatro Cinegas, in theChihuahuan Desert of Mexico.

By Rodrigo Prez Ortega Jun. 30, 2020 , 3:40 PM

Valeria Souza Saldvar never planned to devote her life to a remote and ancient oasis more than 1000 kilometers north of her laboratory in Mexico City. But a call in early 1999 changed that.

Its one of the best cold calls Ive ever made, says James Elser, a limnologist at the University of Montana. He had picked up the phone to invite Souza Saldvar to join a NASA-funded astrobiology project in Cuatro Cinegasa butterfly-shaped basin with colorful pools, or pozas, in the middle of Mexicos Chihuahuan Desert.

Neither Souza Saldvar, a microbial ecologist at the National Autonomous University of Mexico, University City, nor her ecologist husband and research partner Luis Eguiarte Fruns, also at UNAM, had ever visited Cuatro Cinegas. That first trip convinced them to completely change their research plans. Looking at those mountains and the water, I fell in love, Souza Saldvar says.

The landscapemore than 300 turquoise-blue pozas scattered across 800 square kilometers, among marshes and majestic mountainswasnt the only draw. The waters, whose chemistry resembled that of Earths ancient seas, teemed with microbes; unusual bacterial mats and formations called stromatolites carpeted the shallows. When Souza Saldvar first cultured the organisms from the pozas, The amount of microbes was enormous, as was the diversity of colors and colony sizes, she recalls. For her, this remote microbial hot spot was an irresistible mystery.

Since then, work by Souza Saldvar, Eguiarte Fruns, and a widening circle of collaborators in Mexico and the United States has shown that Cuatro Cinegaswhich means four marshes in Spanishis one of the most biodiverse places on the planet. Theres nowhere that has so much ancient diversity of microorganisms, says Michael Travisano, an evolutionary ecologist at University of Minnesota, Twin Cities, who has collaborated with the Mexican researchers since 2001. Among the most recent additions to that menagerie are hundreds of species of archaea, the ancient microbes that may have given rise to eukaryotesorganisms with complex, nucleated cells.

At the Pozas Azules ranch in Cuatro Cinegas, about 100spring-fed pools dapple the desert. Each has a unique microbial and mineral composition.

The diversity includes strains with unusual adaptations, such as the ability to build their lipid membranes with sulfur instead of the usual phosphorus, which is scarce in the waters of thepozas. It includes potential sources of new compounds for medicine and agriculture. And it poses a question that has occupied Souza Saldvar and Eguiarte Fruns for the past 20 years: How did this Noahs Ark of ancient microbes arise? Its a dream for every biologist to know the origin of diversification, Souza Saldvar says.

But her dream might be short-lived. Since the 1970s, farmers have intensively drained water from thepozasand rivers to irrigate nearby fields of alfalfa, grown for cattle fodder, gradually drying the improbable oasis. Souza Saldvar has galvanized a conservation effort that has slowed the drainage; in the coming weeks, a canal that removes 100 million cubic meters of Cuatro Cinegass water annually is scheduled to close. In the meantime, the researchers have been trying to describe as much as they can, as fast as they can, before their belovedpozasdry up and the precious microscopic life that has survived undisturbed for millions of years dies off.

Cuatro Cinegasservedas a stopping point for hunter-gatherers for thousands of years. To date, 50 archaeological sites with cave paintingssome dating to 2275 B.C.E.have been found in mountain cavesaround the basin. Much later, the region made a mark on history when Venustiano Carranza, born in a village at the basins margin, became a leader of the Mexican Revolution and president of Mexico from 1917 to 1920. Nowadays, the village is called Cuatro Cinegas de Carranza after him.

But in the 1960s, Cuatro Cinegas started to become famous for its biodiversity, as biologists began to describe new species of snails, fish, turtles, and plants found in the pools and marshesand often nowhere else.

Wendell Minck Minckley, a renowned ichthyologist at Arizona State University (ASU), Tempe, was first lured to Cuatro Cinegas after learning thatthe worlds only aquatic box turtle(Terrapene coahuila) lived there. Over the years, Minckley made frequent trips to thepozas, describing their snails and fish (Herichthys minckleyi, a cichlid, bears his name) while making connections with the local people.

In the Cuatro Cinegas Basin, ringed with mountains and desert, an aquifer feeds hundreds of pools and marshes. But canals tapping water for agriculture threaten the wetlands and the biodiversity they host.

(MAP) N. DESAI/SCIENCE; (DATA) E. MAMER AND T. NEWTON/NEW MEXICO BUREAU OF GEOLOGY AND MINERAL RESOURCES; VALERIA SOUZA SALDVAR; NATIONAL COMMISSION OF NATURAL PROTECTED AREAS MEXICO

Minckley also noticed peculiar, rocky structures in the pools. They were stromatolites, biological structures normally found as fossils dating back as much as 3.5 billion years. Colonies of photosynthesizing bacteria, which boosted early Earths oxygen, created the layered formations by depositing carbonates and trapping sediment in ancient, shallow seas. But these stromatolites were alive. Also found in other extreme environments such as Australias warm, salty Shark Bay, living stromatolites are sort of a window into early Earth, Elser says. Thepozasalso nurture bacterial mats, a soft form of stromatolites normally found deep in the ocean.

As early as the 1970s, Minckley realized the pools and their diversity were under threat: Local farmers were carving canals to tap their water. Thanks in part to his lobbying, the Mexican government in 1994 designated an 85,000-hectare protected area. But the drainage continued. Minckley knew that Cuatro Cinegas was going to die, Souza Saldvar says. He thought NASA might be its salvation.

In 1998, NASA established its Astrobiology Institute, a network of researchers studying life in extreme environments that might resemble conditions on other planets. Minckley saw an ideal astrobiology study site in the waters of thepozas, with their seemingly inhospitable chemistry and living stromatolites. But he was no expert on extreme environments, so he enlisted Elser, who specializes in how water chemistry affects ecosystems and also works at ASU. After they submitted a 1998 proposal to fund the project, however, NASA said they should add experts on microbiology and evolutionand those experts had to be Mexican to help secure permits to obtain samples. Based on colleagues suggestions, Elser called Souza Saldvar and Eguiarte Fruns, newly minted professors at UNAM. They joined, and NASAapproved the 3-year project.

Stromatolites, reeflike colonies of carbonate-secreting cyanobacteria, abounded in Precambrian seasand thrive at Cuatro Cinegas.

With two children in tow, the couple met Minckley and Elser at Cuatro Cinegas. Next to the turquoise-blue waters of La Becerrapoza, Minckley told them he believed the ecosystem was a glimpse of deep time. Do you see these miniature snails in my hand? Souza Saldvar recalls him saying. I just scooped them from the springhead, but their direct ancestors were eating sulfur bacteria in hydrothermal vents 220 million years ago in the bottom of the ancient Pacific.

Based on the water chemistrylow in phosphorus, iron, and nitrogenand the presence of living stromatolites, Minckley believed Cuatro Cinegas re-created the marine conditions found worldwide millions of years ago. He challenged the two researchers to explore its mysteriesand to protect itspozas. Only you, as Mexicans, can save them from the extinction caused by humans, Souza Saldvar recalls him saying.

Minckleydied2 years later, in 2001.

To inventory the full diversityof microbes at Cuatro Cinegas and trace their relationships, Souza Saldvar needed to study their DNA. To do so, scientists normally take microbial samples from a site and grow them in a lab. But many bacteria and archaea are difficult to culture, and only a few groups at the time had successfully analyzed DNA isolated directly from the environment. High magnesium levels in the water and slime from the microbes made isolating DNA from thepozasespecially difficult.

But Souza Saldvar and her students Ana Escalante and Laura Espinosa Asuar made a start. In 2006, they reported in theProceedings of the National Academy of Sciencesthat they had found 38 distinct groups of microbesfour times as many as in a typical salt marshcorresponding to 10 major lineages of bacteria and one of archaea. Half the bacterial groupswere most closely related to marine microbes. Almost 10% of the groups resembled ones that live on hydrothermal ventsfissures deep in the ocean where microbes thrive despite extreme heat and mineral concentrations.

As Minckley had suspected, Cuatro Cinegas had somehow preserved ancient marine life forms deep in the desert, more than 500 kilometers from the Gulf of Mexico, at a site where the last seas retreated some 20 million years ago.

Valeria Souza Saldvar and Luis Eguiarte Fruns (top) have spent 20 years studying biodiversity at Cuatro Cinegas, where they have found thousands of new species in living structures like a bacterial mat (bottom).

The deep time aspect [of Cuatro Cinegas] is very surprising, Travisano says. It is a true lost world, preserved by the hostile water chemistry, he and the Mexican team argued in a 2018 paper ineLife. Millions of years ago, they proposed, ancient marine ancestors found their way to the place,adapted to the extreme environment, and didnt change much.

Thepozasthemselves are not particularly ancient. The springs that nurture them are fed by deep aquifers in Sierra San Marcos y Pinos, filled with water accumulated during the last ice ages, Eguiarte Fruns says. Now, the water seeps to the surface because of an active fault beneath the basin. It rises through ancient marine sediments, picking up its unusual chemistry along the way. Somehow, the ancient microbespersisted and diversifiedin a succession of springs that must have appeared and vanished throughout geologic time. As in an ancient clock, Souza Saldvar says, all the original mechanisms are still working together to sustain unusual life.

To Frederick Cohan, a microbial ecologist at Wesleyan University who is not part of the Cuatro Cinegas project, the fact that many of the microbes are related to marine species and not species found inland is compelling. I think its saying those organisms are anciently there.

When the researcherslooked at the stromatolites, theyfound even more diversity. Samples from one site, Pozas Azules II, yielded more than 58,000 distinct microbial sequences, predominantly from bacterianot a direct count of species, but an indicator of biodiversity. In the Ro Mezquites, a stream that flows through the northern part of the basin and recharges several pools, they identified 30,000 sequences, mostly from cyanobacteria. More than 1000 sequences from Pozas Azules II appeared to be from archaea, the researchers reported inEnvironmental Microbiologyin 2009. The stromatolites also teemed with bacteria-infecting virusesstrains that wereunique to each pooland resembled marine viruses.

Studying the microbes hasnt been easy. There are thousands and thousands of new bacteria that we cant grow in culture, Souza Saldvar says. They could, however, identify some startling adaptations to the extreme conditions. In one bacterium found only in El Churince, a system of lagoons andpozason the western part of the basin, researchers sequenced the smallest genome ever found in its genus,Bacillus. The work, led by Gabriela Olmedo lvarez, a genetic engineer at Center for Research and Advanced Studies of the National Polytechnic Institute, Irapuato, also showed that the microbeB. coahuilensiscould synthesize membrane sulfolipids. This meant that, like some plants and cyanobacteria, it could use sulfur from the environmentinstead of phosphorusto form its cell membranes.

Shallow, mineral-rich pools and lagoons, with conditions like those in ancient oceans, are hot spots of microbial diversity. Floating mats at Cuatro Cinegas teem with the primordial microbes known as archaea, leading researchers to call them archaean domes.

(GRAPHIC) N. DESAI/SCIENCE; (DATA) GARCIA-MALDONADO ET AL., EXTREMOPHILES, DOI 10.1007/S00792-018-1047-2; CENTENO ET AL., MICOBIOLOY ECOLOGY, DOI: 10.1111/J.1574-6941.2012.01447

It likely stole these genes from a cyanobacterium, Olmedo lvarez says, enabling it to cope with scarce phosphorus, a condition thought to have prevailed in Earths earliest oceans. The microbes small genome may also have helped it thrive, as it required less phosphorus to build its DNA. Olmedo lvarez thinks the organism may offer a glimpse of the stratagems used by early microbes to adapt to their new environment.

Were just starting to understand the depth of diversity, says Olmedo lvarez, who found thatB. coahuilensisis itself starting tosplit into strainswith variations in phosphorus metabolism.

The low phosphorus conditions found in Cuatro Cinegas not only promoted local adaptations, but alsoaccelerated microbial diversification, Souza Saldvar and Elser argued in a perspective published in 2008 inNature Reviews Microbiology. Bacteria normally share bits of DNA with their neighbors in a process called horizontal gene transfer, which blurs the divisions between strains. But in Cuatro Cinegas, the microbeshungry for phosphorusessentially consume free DNA rather than incorporating it into their genomes. They will eat the DNA to get the phosphorus, Elser says.

Besides offering insights into evolution, Cuatro Cinegass microbial diversity may hold practical payoffs. Cuatro Cinegas is one of the richest places on the planet for genetic resources, Souza Saldvar says. For example, most modern antibiotics are derived from actinobacteria, which are abundant in thepozas. Susana De la Torre Zavala, a biotechnologist at the Autonomous University of Nuevo Len (UANL), University City, is searching for potential antibiotics in a library of 350 actinobacteria from the basin. Her team has also found that an extract from a microalga living in the poolsshows anticancer activity.

Agriculture, too, could benefit, Olmedo lvarez says. By 2050, the reservoirs of phosphorus that help sustain global harvests could become scarce, and the microbesability to concentrate the element from different sourcescould hold solutions. Were understanding Cuatro Cinegas, but were also understanding basic principles of ecological interactions that have an application in medicine and agriculture, she says.

As the scientific storyof Cuatro Cinegas unfolded, its fate has hung in the balance, with Souza Saldvar fighting a long series of battles over its water with local farmers and landowners, dairy companies, and politicians. Her weapons have been her rising scientific profile and a tireless outreach to the public, especially young people.

Souza Saldvar has drawn fireduring a 2013 microbiology congress, police had to protect her from protesting localsbut she has won a series of victories. In 2007, the daughter of the CEO of LALA, a giant dairy consortium with roots in the state of Coahuila, told her father she wouldnt speak to him because he was killing Cuatro Cinegas, Souza Saldvar says. The executive promptly scheduled a meeting with the scientist. You need to change your cows diet, Souza Saldvar says she told him, refusing to accept a courtesy yogurt he offered. Ill accept your yogurt when you do so. He promised not only to stop buying the regions alfalfa, but also to invest in environmental education projects for local children.

Two years later, she won an unusual ally, the powerful Mexican billionaire Carlos Slim. His foundation collaborated with the World Wildlife Fund (WWF) to buy the land surrounding El Churince in the western basin, and to provide researchers with a 5-year, 18 million Mexican peso ($1.4 million) grant to study Souza Saldvars favoritepoza. This allowed them to set up the infrastructure to perform long-term experiments. But it did not save the water.

Endemic fishes and turtles first drew scientists to Cuatro Cinegas, where they stumbled on its less visible microbial riches.

In 2010, Mexicos National Water Commission (CONAGUA) set out to replace the open, leaky canals, which lose 75% of the drained water, with less wasteful enclosed conduits. But the project was abandoned midwaymost likely because of corruptionand the old canals were never closed. As Cuatro Cinegas continued to dry up, the researchers raced to study El Churince, finding 5167 distinct species of bacteria and archaea in the last remaining pool. A close inspection of the genomes ofBacillusbacteria from one single square kilometer increased the known diversity of the group by more than 20%. By comparing DNA sequences, the team traced theBacillusdiversity to two ancient ancestors, one dating back 680 million years, the other 160 million years. Those dates coincide with the breakup of the supercontinents Rodinia and Pangaea, respectively, and the team thinks theoceans that formed during those convulsions carried the ancestral microbesto what is now the Cuatro Cinegas Basin, where they have persisted ever since.

Cohan says thats plausible.Bacillusfrom elsewhere fail to thrive in Cuatro Cinegas, most likely because they are outcompeted by the local microbes and cant adapt to the extreme conditions. And theBacillusspecies from Cuatro Cinegas are not found anywhere else in the world. Its just bizarre, Cohan says, but it makes thepozasso much more valuable and worth saving. Its kind of a paleontological microbial park.

In 2016, El Churince dried up just after the funding from the WWFCarlos Slim Foundation ended. The researchers felt devastated. Souza Saldvar says it was painful to see turtle shells lying on the now-barren soil. Its really sad, Olmedo lvarez says. Its gone.

On the eastern sideof the basin, things are looking brighter. In 2000, the conservation nongovernmental organization Pronatura Noreste acquired the Pozas Azules ranch: 2721 hectares hosting about 100pozas. Pronatura eventually gained rights to the water as well, enabling it to close canals draining thepozasin the ranch. Farmers are now encouraged to adopt water-sparing drip irrigation, and some are growing nopalan edible cactus popular in Mexican cuisinewhich requires much less water than alfalfa.

The researchers have focused their recent studies on Pozas Azules. In 2019, after an unusual spring rain, the team noticed alien-looking structures in the shallow waters of a site near Pozas Azules II: white microbial mats buoyed by gas. The gas appeared to be largely methane, and a genetic analysis showed the mats were teeming with archaea230 distinct species,they report in a preprint. That makes the spot the most diverse place of archaea that we know of, De la Torre Zavala says.

Now, the team hopes to analyze samples from the structures, which it calls archaean domes, in search of the elusive Asgard archaea, organisms previously found only in the deep ocean and thought tohold clues to the evolution of simple microbesinto complex eukaryotes. Although some in her team are skeptical, Souza Saldvar is convinced they will find them. Valerias usually right, De la Torre Zavala says.

Shaped and seeded with life by ancient seas, the Cuatro Cinegas Basin lies at the foot of the distant Sierra San Marcos. The white dunes bordering the basin are made of gypsum, a legacy of a Jurassic ocean.

Such prospects have added to Souza Saldvars determination to preserve Cuatro Cinegas, and she is enlisting young people for support. In every field trip since 2004, her team has spent time with students from the local high school, showing them how to use a microscope and take simple environmental measurements, and teaching them about sustainable agriculture. In 2011, with funding from the LALA Foundation and the WWFCarlos Slim Foundation, the scientists set up a college-level molecular biology lab at the school, which is now ranked among the best rural high schools in Mexico.

Hctor Arocha Garza is one of its graduates. Inspired by the secrets of Cuatro Cinegas, he pursued a Ph.D. in biotechnology at UANL with De la Torre Zavala, then returned to his hometown. My heart was in Cuatro Cinegas, he says. Now, hes leading the scientific branch of a privately fundedmegaproject called Cuatro Cinegas 2040that aims to build a science museum and make Cuatro Cinegas a scientific tourism destination, while supporting education and medical care for the villages young people.

The effort comes at a critical moment. More than 90% of the marshes are gone, and somepozasand lagoons are dry. But this year, CONAGUA committed toregulating water usageand closing illegal wells, and Pronatura Noreste will close the Saca Salada Canal, which drains the Ro Mezquites, as soon as the COVID-19 pandemic permits.

Those developments, and stories like Arocha Garzas, give Souza Saldvar hope for the future of Cuatro Cinegas. It has been a very complicated, long, and difficult process, she says. But now, she wrote in a recent book, There is a revolution occurring in this oasis: Science is the tool and kids are the drivers.

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Pools in the Mexican desert are a window into Earth's early life - Science Magazine

Global Personalized Cancer Drugs Market: Snapshot

Genetic sequencing has proven that no two cancer cases are absolutely identical, heavily depending on genetic profiles of the patients, which defines their immunity power. But frequently, several promising pipeline drugs fail to reach the market for not being commonly useful for the masses. In this scenario, a small but increasing number of personalized cancer drugs are allowed by the FDA for the treatment of particular mutations. Nearly one third of cancer drugs are prescribed off-label, as it provides help to the patients immediately. These targeted agents are directed at specific molecular feature of the cancer cells and hence produce greater effectiveness with significantly less toxicity.

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The global market for personalized cancer drugs market is gaining traction from increased government support for precision-medicine. For example, in June 2016, the U.S. National Cancer Institutes revealed its plans to enroll thousand patients in a trial called NCI-MATCH, which is aimed at matching patients to twenty possible compounds on the basis of their genetic abnormalities. Along the similar lines, The American Society of Clinical Oncology has also announced a registry termed as TAPUR, collecting data on the fate of patients who receive personalized cancer drugs off-label.

Another factor driving the global personalized cancer drugs market is the falling cost of genetic sequencing, which is enabling the quick approval of drugs for off-label clinical trials on patients in need across the world.

Personalized Cancer Drugs Market: Overview

Personalized drugs, or customized drugs, are tailored to suit the needs of individual patients. Earlier, various patients suffering from the same type of disease were given the similar treatment plan. However, it became evident to physicians that a particular treatment worked differently for different patients, mainly owing to a varied genetic makeup. The concept of personalized medicine is based on the analysis of etiology of disease in individual patients and offers treatment that is more efficient, predictable, and precise.

Cancer is a common chronic disease and a major cause of fatality around the globe. The development of personalized cancer drugs has gained pace as they have relatively fewer side effects compared to standard drugs. Personalized cancer drugs target a specific protein or gene responsible for the growth and survival of a cancer type.

Personalized Cancer Drugs Market: Trends and Opportunities

The personalized cancer drugs market is primarily fueled by the rising prevalence of various cancer types such as lung cancer, breast cancer, prostate cancer, melanoma and leukemia, and colorectal cancer. According to the Surveillance, Epidemiology, and End Results Program sponsored by the National Cancer Institute (NCI), an estimated 13,397,159 people in the United States were affected with various cancer types in 2011. Moreover, in 2014, around 1,666,540 new cancer cases were diagnosed in the country, with nearly 585,720 deaths resulting from cancer. The personalized cancer drugs market is also driven by several advantages associated with this new treatment therapy and ongoing developments in the field of genetic science.

On the flip side, high cost associated with the genetic testing of patients and tumor samples may serve as a growth restraint on the market for personalized cancer drugs. In addition to this, the lack of insurance plans to cover these tests in developing nations of Asia Pacific and Rest of the World hampers the market to some extent. This can be attributed to low per capita income and poor reimbursement scenario.

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Personalized Cancer Drugs Market: Geographical Assessment

From a geographical perspective, the personalized cancer drugs market has been broadly segmented into Europe, Asia Pacific, North America, and Rest of the World (RoW). The market for personalized cancer drugs is led by North America. The chief factors responsible for the regions lead position are aggressive research and development activities, technical advancements, higher affordability for expensive treatments and therapies, and greater healthcare awareness. Europe is also a key market for personalized cancer drugs owing to significant funding from several governments and the growing penetration by U.S.-based companies.

Asia Pacific holds immense promise for players in the personalized cancer drugs market, powered mainly by Japan. The regional market is likely to be fueled by the presence of a large pool of cancer patients and improving healthcare infrastructure. The growth of the APAC personalized cancer market can also be attributed to the rapidly evolving medical tourism industry. In the RoW segment, Mexico, Brazil, Russia, and South Africa represent potential markets.

Personalized Cancer Drugs Market: Competitive Landscape

Some of the key players competing in the personalized cancer drugs market are F. Hoffmann-La Roche Ltd., Pfizer Ltd., Cell Therapeutics, Inc., H3 Biomedicine, Inc., bioTheranostics, GlaxoSmithKline, and Abbott Laboratories. Zelboraf (vemurafenib) by F. Hoffmann-La Roche Ltd. and Xalkori (crizotinib) by Pfizer Ltd. are some notable targeted drugs for the treatment of cancer.

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Personalized Cancer Drugs Market Expected to Witness a Sustainable Growth over 2025 - 3rd Watch News

Andre Watson

SAN FRANCISCO (PRWEB) June 18, 2020

Vaccines currently in development face the dual challenge of overcoming the temporary immunity presented by coronavirus infection, and the SARS-CoV-2 viruss highly effective cloaking mechanism that effectively renders it invisible to the immune system.

Ligandals technology has been designed around the unique genetic signature of the virus, which has informed the development of a peptide nanoscaffold. This peptide prevents the virus from binding with human cells, halting infection. The peptide also simultaneously disables the viral cloaking mechanism, making the virus vulnerable to an immune response.

Andre Watson, CEO and Founder of Ligandal, says, I started Ligandal to create practical genetic medicine technology that solves the worlds most pressing health problems. We had been developing a way of training T-cells to attack cancer tumours, but when the COVID-19 pandemic began, I realised our technology would be effective at neutralising this single, virulent pathogen without requiring a gene therapy component. The beauty of our solution is that it can be used as treatment and vaccine. In infected people, the peptide will prevent viral entry and replication, while bolstering the immune response and formation of neutralising antibodies that can eliminate the virus. Other approaches may neutralise the virus, but many alternatives such as antibody therapies and viral-neutralising compounds will leave the body vulnerable to repeat infection. In those who havent been infected, the peptide will display critical immunoepitopes for antibody and T cell responses against the key parts of the virus necessary for forming a neutralising response. The peptide can also be used in conjunction with other treatments and vaccinesand may bolster the efficacy of spike protein vaccines in particularalthough that shouldnt be necessary if we achieve in vivo results suggested by the in silico modelling.

Adam Hamdy, a medical consultant and author, who recently joined the companys advisory board, says, The microbiological characteristics of SARS-CoV-2 make it extremely unlikely that current vaccine approaches, which rely on training the immune system but present key challenges with this virus, will offer anything more than partial protection at best. It was clear to me that any effective response to this virus had to target it directly while also bolstering immune response.

Importantly, Ligandals peptide has advantages over other technologies in development because it is room temperature stable, meaning it represents a genuinely global solution, both in terms of logistics and the storage of the medicine. It also has a low cost per dose once at mass production scale, which means global producton is entirely feasible. It can also move rapidly through preclinical and clinical studies given the accelerated global regulatory environment and simple at scale manufacturing process.

Andre Watson says, As hostile actors take note of the havoc caused by the SARS-CoV-2 virus, it is clear we need better ways to ramp up our biodefense capabilities. A rapid four-year conventional vaccine cycle to trial a single solution isnt going to be sufficient to meet future threats. Ligandal modelled its peptide within five hours of receiving a genetic sequence of the virus, and was ready to test its response in two months. We anticipate being ready to commence clinical studies later this year or very early next year. Our only limitation in getting this to the general public is how quickly we can move through the clinical studies.

Adam Hamdy says, A long term solution to the COVID-19 problem lies in genetic medicine. Ligandals peptide is an exciting leap forward in our ability to address and neutralise the virus directly. Unlike conventional vaccines, which only get one shot at proving efficacy, Ligandals peptide can be improved by rewriting its genetic code in response to novel viral variants, giving us the opportunity to rapidly iterate and keep at the problem until we get the answer absolutely right.

About Ligandal

Based in San Francisco, Ligandal is a genetic medicine company that uses nanotechnology to develop targeted and personalised therapies.

For more information visit http://www.ligandal.com

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Genetic Medicine will be the Solution to COVID-19, and Ligandal is Leading the way - PR Web

Application of ImmTOR plus Sareptas investigational gene therapies will be evaluated for Duchenne Muscular Dystrophy and Limb-Girdle Muscular Dystrophies

CAMBRIDGE, Mass. and WATERTOWN, Mass., June 18, 2020 (GLOBE NEWSWIRE) --Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, and Selecta Biosciences, Inc. (NASDAQ: SELB) today announced that they have entered into a Research License and Option agreement granting Sarepta an option to license the rights to develop and commercialize Selectas immune tolerance platform, ImmTOR, for use in Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMDs). In advance of exercising its option, Sarepta will conduct research and evaluate the utility of ImmTOR to minimize or prevent the formation of neutralizing antibodies (NAb) to adeno-associated virus (AAV) in connection with the administration of Sareptas DMD and LGMD gene therapy candidates.

Sareptas late-stage gene therapy candidates are delivered using AAV in particular, AAVrh74. AAVrh74 was selected because of its safety profile, superior muscle tropism, empirical demonstration of high expression, and low screen-out rate for pre-existing antibodies. Currently, however, all systemic AAV-delivered constructs are one-time therapies that cannot be re-dosed due to the robust post-administration development of NAbs specific to the AAV vector. Selecta is a leader in immune tolerance and has generated strong preclinical evidence to support the potential for re-dosing patients receiving gene therapy.Selecta has reported that in preclinical studies, when used in combination with AAV gene therapy vectors,Selectas ImmTOR immune tolerance platform inhibits the development of NAbs to the vector, permitting re-dosing of the gene therapy. i

As we build our enduring gene therapy engine, we intend not only to rapidly advance treatments for rare, life-ending diseases, but at the same time, to advance the state of genetic medicine science by continually improving the utility of gene therapy. If successful, the ability to re-dose will be an enormous leap forward in the science of gene therapy and provide invaluable benefits to patients beyond those we anticipate with one-time dosing. We are encouraged by the data generated on the ImmTOR platform and excited to join with Selecta to explore the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed, said Doug Ingram, President and Chief Executive Officer, Sarepta Therapeutics.

We are pleased to build on our already strong foundation of strategic partnerships and expand the clinical application of the ImmTOR platform into neuromuscular diseases. The ability to re-dose gene therapy addresses one of the major challenges of one-time therapies today, said Carsten Brunn Ph.D., President and Chief Executive Officer of Selecta Biosciences. We are excited to collaborate with a leader in genetic medicine like Sarepta, and are confident that their expertise in rare diseases combined with our immune tolerance platform has the potential to enhance the long-term therapeutic benefit to patients with these debilitating conditions.

DMD is a rare, degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. LGMDs are a group of over 30 distinct diseases that cause weakness and wasting of the muscles around the hips and shoulders, eventually progressing to the arms and legs. LGMD can be caused by a single gene defect that affects specific proteins within the muscle cell, including those responsible for keeping the muscle membrane intact.

Under the terms of the research license and option agreement, Sarepta will make an initial payment to Selecta, and Selecta is eligible to receive certain pre-clinical milestone fees. If Sarepta exercises its options to enter any commercial license agreements, Selecta will be eligible for additional development, regulatory, and commercial milestone payments, as well as tiered royalties on net product sales. Additional financial details are not being disclosed.

About Selecta Biosciences, Inc.

Selecta Biosciences, Inc. is a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its pioneering immune tolerance platform (ImmTOR). Selecta is committed to utilizing ImmTOR to potentially improve the efficacy of biologics, enable re-dosing of life-saving gene therapy, and create novel immunotherapies for autoimmune diseases. Selectas late-stage product candidate, SEL-212, is designed to be a monthly treatment for chronic refractory gout, a debilitating rare disease with a significant unmet medical need. SEL-212 consists of a combination of our ImmTOR platform co-administered with pegadricase, an enzyme designed to treat patients with symptomatic gout, refractory to standard uric acid lowering treatment. Selectas proprietary gene therapy product candidates are in development for certain rare inborn errors of metabolism and incorporate our ImmTOR platform with the goal of addressing barriers to repeat administration. In addition to our own pipeline of core discovery and clinical candidates, Selecta has established collaborative relationships with leading biopharmaceutical companies, including Asklepios BioPharmaceutical (AskBio) for gene therapy, and Swedish Orphan Biovitrum AB (Sobi) for SEL-212. Selecta is based in Watertown, Massachusetts. For more information, please visit http://www.selectabio.com.

Selecta Forward-Looking Statements:

SelectaBiosciences, Inc. (the company), including without limitation, the companys actions regarding the monitoring and assessment of COVID-19 on the companys operations, clinical trials and manufacturing, Sareptas plans to evaluate its gene therapies in combination with the companys ImmTOR technology, the possibility of Sarepta exercising an option to enter into a commercial license agreement, the unique proprietary technology platform of the company and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the ability of the companys ImmTOR platform to unlock the full potential of biologic therapies, the potential treatment applications for product candidates utilizing the ImmTOR platform in areas such as enzyme therapy and gene therapy, the novelty of treatment paradigms that Sarepta is able to develop in combination with the companys ImmTOR technology, the potential of any therapies developed by Sarepta in combination with the companys ImmTOR technology to fulfill unmet medical needs, the companys plan to apply its ImmTOR technology platform to a range of biologics for rare and serious diseases, the ability of Sareptas existing therapies to target the heart and skeletal muscle, expected payments to be made to the company under the Research License and Option Agreement, the potential of the ImmTOR technology platform generally and the companys ability to grow its strategic partnerships, the sufficiency of the companys cash, cash equivalents and short-term investments, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including their uncertain outcomes, the effect of the COVID-19 outbreak on any of the companys planned or ongoing clinical trials, manufacturing activities, supply chain and operations, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the unproven approach of the companys ImmTOR technology, Sareptas ability to research and develop therapeutic candidates using the companys ImmTOR technology, undesirable side effects of the companys product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials as well as the impact of the COVID-19 outbreak on those third parties and their ability to continue their operations, the companys inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, managements ability to perform as expected, potential delays in regulatory approvals, Sareptas ability to make up-front and milestone payments, the companys business development strategy, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the companys recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock including stock market fluctuations that occur as a result of the COVID-19 outbreak, and other important factors discussed in the Risk Factors section of the companys most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

AboutSarepta Therapeutics

At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Sarepta Forward-Looking Statement

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the parties undertakings under the agreement and potential payments and fees; the potential benefits of Sareptas gene therapy product candidates; the potential of ImmTOR to enable re-dosing of AAV gene therapy; Sareptas intention to rapidly advance treatments for rare, life-ending diseases, and to advance the state of the genetic medicine science by continually improving the utility of gene therapy; the potential of re-dosing to provide invaluable benefits to patients beyond those Sarepta anticipates with one-time dosing;the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed; and the potential of the collaboration between Sarepta and Selecta to enhance the long-term therapeutic benefit to patients with these debilitating conditions.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Selecta may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; success in preclinical trials does not ensure that later clinical trials will be successful; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Selecta Contacts:For Media:Joshua R. MansbachSolebury Trout+1-646-378-2964jmansbach@soleburytrout.com

For Investors:Lee M. SternSolebury Trout+1-646-378-2922lstern@soleburytrout.com

Sarepta Contacts: Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

_____________________________iNature Communications,October 2018.

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Sarepta Therapeutics and Selecta Biosciences Enter into Research License and Option Agreement for Selecta's ImmTOR Immune Tolerance Platform in...

A research team led by Francesca Luca, Ph.D., associate professor of Wayne State Universitys Center for Molecular Medicine and Genetics, has published a study that annotated environmental components that can increase or decrease disease risk through changes in gene expression in 43 genes that could exacerbate or buffer the genetic risk for cardiovascular disease. Their results highlight the importance of evaluating genetic risk in the context of gene-environment interactions to improve precision medicine.

Interpreting Coronary Artery Disease Risk Through GeneEnvironment Interactions in Gene Regulation was published in Genetics, the journal of the Genetics Society of America.

The study, said Dr. Luca, also of the WSU Department of Obstetrics and Gynecology, illustrates that combining genome-wide molecular data with large-scale population-based studies is a powerful approach to investigate how genes and the environment interact to influence risk of cardiovascular disease.

By identifying regions of DNA important for endothelial cell response to different common environmental exposures, the researchers discovered that caffeine can influence the risk of cardiovascular disease. The study demonstrates the potentially beneficial and/or detrimental effects of certain environmental exposures on the cardiovascular disease risk differ depending on individual DNA sequence.

The study focused on cardiovascular disease, Dr. Luca said, because it is the leading cause of death, both in the United States and worldwide. Also, the disease is highly multifactorial, with large contributions from both environmental and genetic risk factors. By treating endothelial cells under a controlled environment, we can discover how these genetic and environmental risk factors influence each other at the molecular level, she said. Our lab has developed expertise in cardiovascular research, with additional projects using endothelial cells to develop new assays to test the regulatory activity of genetic variants. The approach outlined in this paper can be applied to many different diseases; for example, our lab has also focused on how bacteria in the human gut affect gene expression in the colon, and also on the effect of psychosocial stress on asthma.

While the work identified regions of the genome important for how endothelial cells respond to the environment and can influence the risk of cardiovascular disease, the researchers do not yet know exactly which genetic variants are directly responsible. A former graduate student, Cynthia Kalita, developed an assay to test thousands of genetic variants for gene regulatory activity. The researchers can test the variants discovered in their study using that assay to validate and explore the mechanisms by which they exert their effects, Dr. Luca said. They also are developing computational/statistical methods that can yield better personalized risk scores.

We have extended our approach to study cardiomyocytes, which are the muscle cells of the heart. Healthy heart tissue is difficult to obtain, so we have collaborated with researchers at the University of Chicago to derive cardiomyocytes from stem cells, Dr. Luca said. This will allow us to shift our focus from the vasculature to the heart itself, where we can study diseases like cardiomyopathies and arrhythmias.

As the cost of DNA sequencing continues to decrease, Dr. Luca expects that genetic testing will play a greater role in preventive health care. To fully realize the potential of precision medicine, we need to consider both genetic and environmental risk factors of disease, and how they interact. While there are already direct-to-consumer tests that prescribe an individualized diet based on DNA, these products currently offer no demonstrated clinical value. However, with very large numbers of individuals for whom we have both DNA sequencing and information on diet and lifestyle, we may one day be able to offer better recommendations.

Others involved in the study included Anthony Findley, an M.D./Ph.D. student; Allison Richards, Ph.D., a research scientist; Cristiano Petrini, of the Center for Molecular Medicine and Genetics; Adnan Alazizi, lab manager; Elizabeth Doman, of the Center for Molecular Medicine and Genetics; Alexander Shanku, Ph.D., research scientist; Gordon Davis, of the Center for Molecular Medicine and Genetics; Nancy Hauff, Department of Obstetrics and Gynecology; Yoram Sorokin, M.D., professor of Obstetrics and Gynecology; Xiaoquan Wen, of the Department of Biostatistics at the University of Michigan; and Roger Pique-Regi, Ph.D., associate professor of the Center for Molecular Medicine and Genetics, and of the Department of Obstetrics and Gynecology.

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Researchers identify environmental components that affect gene expression in cardiovascular disease - The South End