Monthly Archives: June 2020

MENLO PARK, Calif.--(BUSINESS WIRE)--Personalis, Inc. (Nasdaq: PSNL) today announced a collaboration with Sarepta Therapeutics (Nasdaq: SRPT), a leader in precision genetic medicine for rare disease. As part of this research collaboration, Sarepta will be working with the Personalis team to characterize immune response to precision genetic therapeutics, utilizing Personalis advanced proprietary analytics.

We are excited to announce this collaboration with Personalis. By bringing together Sareptas expertise in precision genetic medicine and Personalis advanced neoepitope prediction, our goal is to better characterize certain types of immune response to benefit patients with rare disease, said Dr. Tanya Teslovich, Senior Director, Genomics at Sarepta.

We are excited to work with Sarepta to apply our proprietary analytics in the rapidly emerging area of genetic medicine. This collaboration demonstrates the extensibility of our technology platform beyond cancer to additional therapy development areas, said Dr. Richard Chen, CSO at Personalis.

About Personalis, Inc.

Personalis, Inc. is a growing cancer genomics company transforming the development of next-generation therapies by providing more comprehensive molecular data about each patients cancer and immune response. The Personalis ImmunoID NeXT Platform is designed to adapt to the complex and evolving understanding of cancer, providing its biopharmaceutical customers with information on all of the approximately 20,000 human genes, together with the immune system, from a single tissue sample. Personalis also provides genomic information to the VA Million Veterans Program as part of their goal to sequence over a million veteran genomes. The Personalis Clinical Laboratory is GxP aligned as well as CLIA88-certified and CAP-accredited. For more information, please visit http://www.personalis.com and follow Personalis on Twitter (@PersonalisInc).

Forward-Looking Statements

This press release contains or may imply "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. For example, forward-looking statements include statements regarding potential positive outcomes resulting from the collaboration of Personalis and Sarepta, such as the ability of the collaboration to yield novel discoveries. These forward-looking statements are subject to risks and uncertainties, including those discussed in Personalis filings with the Securities and Exchange Commission (SEC), including in the Risk Factors and Managements Discussion and Analysis of Financial Condition and Results of Operations sections of the Companys most recently filed periodic report on Form 10-K and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Personalis disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

Here is the original post:
Personalis Announces Scientific Collaboration with Sarepta Therapeutics on Immune Response to Precision Genetic Therapeutics - Business Wire

BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced the appointment of Julie Anne Smith to its Board of Directors. Ms. Smith has also been appointed to the Compensation Committee of the Board of Directors. Ms. Smith will replace Samuel Hall, Ph.D., whose term on the Board of Directors expired.

Julie brings more than two decades of experience in the life sciences industry, with a strong track record of successfully developing and commercializing medicines for rare and inherited diseases. Her expertise in drug development for neurodegenerative diseases will be particularly valued as we advance STK-001 for Dravet syndrome into the clinic later this year, said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. We thank Sam for his many important contributions to Stoke from our inception and as we matured through our successful IPO to become a public company prepared to enter the clinic with STK-001, the first potential medicine developed using our TANGO platform. We welcome Julie to the Board and look forward to her insights and contributions.

This is an exciting time for Stoke as it transitions to a clinical stage company and looks to the future, said Ms. Smith. I am pleased to work with the Board members and the executive leadership team as they advance their work in Dravet and expand the pipeline to help people who are living with severe genetic diseases and realize the potential of the TANGO platform.

Ms. Smith currently serves as President and CEO of ESCAPE Bio, Inc., a biotechnology company developing precisely targeted therapeutics for genetic forms of neurodegenerative disease. She previously served as President and CEO of Nuredis, Inc., a biotechnology company developing small-molecule therapies for nucleotide repeat disorders such as Huntingtons disease. In 2014, Ms. Smith was appointed President and CEO at Raptor Pharmaceuticals, a public biotechnology company with two commercial medicines for orphan diseases, where she served until its acquisition in 2016 (by Horizon Pharmaceuticals, Inc.). Prior to joining Raptor, Ms. Smith served as the Chief Commercial Officer at Enobia Pharmaceuticals (acquired by Alexion Pharmaceuticals, Inc.). Earlier in her career, she held positions of increasing responsibility at Jazz Pharmaceuticals plc, Genzyme, Novazyme and Bristol-Myers Squibb Company.

Ms. Smith previously served on the board of directors of Audentes Therapeutics, Inc., a genetic medicines company, and as a director on the Health and Emerging Companies Section Governing Boards of the Biotechnology Industry Organization (BIO). She currently serves on the board of directors of Exelixis, Inc., a public genomics-based drug discovery company. Ms. Smith holds a B.S. in biological and nutritional sciences from Cornell University.

About Stoke Therapeutics

Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company on Twitter at @StokeTx.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: Stokes expectation about timing and execution of anticipated milestones with respect to STK-001, including advancement of STK-001 to the clinical stage, and expansion of the Companys pipeline. Statements including words such as plan, continue, expect, or ongoing and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Forward-looking statements are subject to risks and uncertainties that may cause Stokes actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to Stokes ability to develop, obtain regulatory approval for and commercialize STK-001 and its future product candidates, the timing and results of preclinical studies and clinical trials, Stokes ability to protect intellectual property; and other risks set forth in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These forward-looking statements are based on our current believes and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements.

See the original post:
Stoke Therapeutics Appoints Julie Anne Smith to its Board of Directors - Business Wire

HOUSTON, June 4, 2020 /PRNewswire/ -- The Golden Helix Foundation and the Pharmacogenomics Access & Reimbursement Coalition (PARC) will co-present the inaugural Pharmacogenomics Access & Reimbursement Symposium (PARS) at the National Academy of Sciences Building on October 8, 2020 in Washington D.C. to define opportunities to expand patient access to personalized medicine. Public- and private- sector members across healthcare will assemble to develop a path forward through discussions of best practices, improved economic evaluation and strategic alignment.

"Since genetic variation impacts medication responses, it is important to leverage technologies that can translate genetic information into care tailored for each patient.Market access is a critical step in achieving widespread adoption of personalized medicine." Sara Rogers, PARC Co-Chairman and Director at the American Society of Pharmacovigilance.

The symposium program includes speakers from industry, government agencies, payer organizations, health systems and health policy organizations. Thought leaders will explore the newest developments in health technology assessment, health economics and value-based payment strategies for pharmacogenomics. In addition to convening decision-makers from around the world, the symposium will develop solutions that identify actionable ways that stakeholders can work together to democratize personalized medicine.

"Defining the value of personalized medicine interventions is of utmost importance to expedite the incorporation of these innovative healthcare solutions in routine clinical practice that would directly impact patient care and quality of life." Christina Mitropoulou, The Golden Helix Foundation Managing Director, Executive Board member and Principal Investigator of the Ubiquitous Pharmacogenomics Consortium.

Organizations across health care have partnered to support the symposium, including Intermountain Precision Genomics, Medical Device Innovation Consortium (MDIC) and Pharmacogenomics Research Network (PGRN). Stakeholders are encouraged to join the discussion by registering to participate athttp://www.parcoalition.org/symposium. A live webcast will be provided to accommodate attendees who prefer to participate remotely.

About the Golden Helix Foundation The Golden Helix Foundation is an international non-profit research organization (registered London-based UK charity) aiming to advance research and education in the area of genome and personalized medicine. The Golden Helix Foundation aims to promote the development of research and the transfer and communication of knowledge from researchers and scientists in the wider scientific community through collaborative projects and conferences in the field of pharmacogenomicsand personalized medicine.

About the Pharmacogenomics Access & Reimbursement Coalition (PARC)PARC seeks to address barriers to patient access and payer coverage of Pharmacogenomics (PGx) testing by sharing resources and leveraging shared expertise in PGx. For more information, please contact [emailprotected] and follow @PGxARC

American Society of PharmacovigilanceP.O. Box 20433Houston, TX 77225www.stopADR.org

Contact: Geneva MorelDirector of Communications Email: [emailprotected] phone: 469-939-8475

SOURCE American Society of Pharmacovigilance

Here is the original post:
The Golden Helix Foundation and PARC Co-Present International Meeting to Expand Patient Access to Personalized Medicine - PRNewswire

Mark Williams The Columbus Dispatch

TuesdayJun2,2020at5:11PM

A Massachusetts-based biopharmaceutical company plans to create 100 jobs as part of an expansion of its Columbus operations.

Sarepta Therapeutics will open an 85,000-square-foot building at 3435 Stelzer Rd. as part of its Gene Therapy Center of Excellence.

The company says it will invest more than $30 million, and that hiring for research, technician and general operations positions will begin immediately.

Employees currently working out of the companys offices in Dublin will move to the new building over time.

Sarepta, based in Cambridge, focuses on gene therapy programs to treat rare diseases.

It has two approved drugs for Duchenne muscular dystrophy and more than 40 treatments in development.

Duchenne slowly steals muscle, making children weaker and weaker as they grow older. Many died by their mid-20s. The disease afflicts mostly boys.

We are confident that gene therapy will revolutionize genetic medicine, and we chose Ohio for our Gene Therapy Center of Excellence because we believe Columbus will become a hub for genetic medicine innovation, the companys president and CEO, Doug Ingram, said in a statement.

mawilliams@dispatch.com

@BizMarkWilliams

Excerpt from:
Sarepta to expand Columbus operations, add 100 jobs - The Columbus Dispatch

CARLSBAD, Calif., June 2, 2020 /PRNewswire/ --Prescient Medical, a subsidiary of Prescient Medicine Holdings, Inc., announced today a research collaborationwith the Harvard Chan Microbiome in Public Health Center (HCMPH Center), a group at Harvard T.H. Chan School of Public Health dedicated to expanding research on the microbiome to improve public health. The aim of the collaboration is tostudy microbial biomarkers to identify the presence of precancerous adenomas and carcinomas in the colon. The initial collaboration will investigate prevalent gut microbial biomarkers for colorectal cancer (CRC) by analyzing known, recent CRC cases across populations with which the HCMPH Center works and applying cutting-edge statistical and bioinformatic techniques for microbiome meta-analysis.

"The ongoing research collaboration will further enhance diagnostic screening for colon cancer," said Keri Donaldson, M.D, chief executive officer at Prescient Medicine. "Offering a non-invasive alternative to colonoscopies that screen for colorectal adenomas and carcinomas could represent a paradigm shift in CRC screening driven by the microbiome. Therefore, research to better understand the microbiome's role in CRC is needed at this time."

Curtis Huttenhower, Ph.D., professor of computational biology at Harvard Chan School and co-director of the HCMPH Center, said, "The mission of the HCMPH Center is to improve population health via microbiome science, and there are few chronic disease conditions as well-positioned to benefit from microbiome screening as colorectal cancer. It is one of the most common causes of cancer deaths, but also one of the most preventable cancers if detected early. It's exciting to embark on this collaboration to advance the latest science and, I hope, eventually deploy our findings to the clinic."

The past decade has seen a dramatic expansion of research on the human microbiome, including investigation into the role of microbes and microbiota in the gastrointestinal track in the origin and development of CRC. The advancements in this field parallel the preceding decade's growth in personalized genetic medicine, with the microbiome offering opportunities for both therapeutic and diagnostic biomarker discovery.

According to the American Cancer Society, colorectal cancer is the third leading cause of cancer-related deaths in both men and in women. The U.S. spends approximately $14 billion each year for the diagnosis and treatment of CRC with costs largely due to delayed detection. There is a lack of non-invasive screening tests that can accurately detect precancerous polyps as effectively as a colonoscopy, the current standard of care. Screening recommendations currently suggest acolonoscopy for average-risk patients starting at age 45 every 10 years and earlier for high-risk patients, but approximately one in three patients are not in compliance with these recommendations.Research indicates that early detection of precancerous adenomas and carcinomas could lead to significantly better patient outcomes.

About Prescient Metabiomics Prescient Metabiomics LLC, a privately held company and subsidiary of Prescient Medicine Holdings, Inc., is an early stage molecular diagnostics company developing in-vitro diagnostics that leverage breakthroughs in next-generation DNA sequencing, computational systems biology, and human microbiome sciences. To learn more, visit http://www.metabiomics.com.

About Prescient Medicine HoldingsPrescient Medicine Holdings, Inc. is a privately held company focused on developing diagnostic tools that advance the precision healthcare movement.Prescient Medicine's mission is to accelerate the development, commercialization and deployment of advanced clinical diagnostics to address the most pressing public health issues in the U.S. Prescient Medicine designs powerful tests and analytic solutions to offer deep predictive insights so doctors and patients have the data they need to make more informed clinical decisions and achieve the best possible patient outcomes. Prescient Medicine technologies include LifeKitPrevent designed to detect colon cancer and precancerous adenomas and LifeKitPredict, an in vitro diagnostic test commercialized in partnership with its subsidiary AutoGenomics, used for the identification of patients who may be at risk for opioid dependency. To learn more, visit http://www.prescientmedicine.com.

View original content:http://www.prnewswire.com/news-releases/prescient-metabiomics-and-the-harvard-chan-microbiome-in-public-health-center-collaborate-to-advance-research-in-colon-cancer-screening-301068742.html

SOURCE Prescient Medicine; Prescient Metabiomics

More here:
Prescient Metabiomics and the Harvard Chan Microbiome in Public Health Center Collaborate to Advance Research in Colon Cancer Screening - BioSpace

Visit the News Hub

Chemical compound restores tetracycline's effectiveness by blocking bacterial resistance

Shown above are two different 3D views of TetX7 (green), a tetracycline-destroying enzyme that causes resistance to all tetracycline antibiotics (the small multicolored molecule in the center). Researchers at Washington University in St. Louis and the National Institutes of Health (NIH) have found that genes that confer the power to destroy tetracyclines are widespread in bacteria that live in the soil and on people.

The latest generation of tetracyclines a class of powerful, first-line antibiotics was designed to thwart the two most common ways bacteria resist such drugs. But a new study from researchers at Washington University in St. Louis and the National Institutes of Health (NIH) has found that genes representing yet another method of resistance are widespread in bacteria that live in the soil and on people. Some of these genes confer the power to destroy all tetracyclines, including the latest generation of these antibiotics.

However, the researchers have created a chemical compound that shields tetracyclines from destruction. When the chemical compound was given in combination with tetracyclines as part of the new study, the antibiotics lethal effects were restored.

The findings, available online in Communications Biology, indicate an emerging threat to one of the most widely used classes of antibiotics but also a promising way to protect against that threat.

We first found tetracycline-destroying genes five years ago in harmless environmental bacteria, and we said at the time that there was a risk the genes could get into bacteria that cause disease, leading to infections that would be very difficult to treat, said co-senior author Gautam Dantas, PhD, a professor of pathology and immunology and of molecular microbiology at Washington University School of Medicine in St. Louis. Once we started looking for these genes in clinical samples, we found them immediately. The fact that we were able to find them so rapidly tells me that these genes are more widespread than we thought. Its no longer a theoretical risk that this will be a problem in the clinic. Its already a problem.

In 2015, Dantas, also a professor of biomedical engineering, and Timothy Wencewicz, PhD, an associate professor of chemistry in Arts & Sciences at Washington University, discovered 10 different genes that each gave bacteria the ability to dice up the toxic part of the tetracycline molecule, thereby inactivating the drug. These genes code for proteins the researchers dubbed tetracycline destructases.

But they didnt know how widespread such genes were. To find out, Dantas and first author Andrew Gasparrini, PhD then a graduate student in Dantas lab screened 53 soil, 176 human stool, two animal feces, and 13 latrine samples for genes similar to the 10 theyd already found. The survey yielded 69 additional possible tetracycline-destructase genes.

Then they cloned some of the genes into E. coli bacteria that had no resistance to tetracyclines and tested whether the genetically modified bacteria survived exposure to the drugs. E. coli that had received supposed destructase genes from soil bacteria inactivated some of the tetracyclines. E. coli that had received genes from bacteria associated with people destroyed all 11 tetracyclines.

The scary thing is that one of the tetracycline destructases we found in human-associated bacteria Tet(X7) may have evolved from an ancestral destructase in soil bacteria, but it has a broader range and enhanced efficiency, said Wencewicz, who is a co-senior author on the new study. Usually theres a trade-off between how broad an enzyme is and how efficient it is. But Tet(X7) manages to be broad and efficient, and thats a potentially deadly combination.

In the first screen, the researchers had found tetracycline-destructase genes only in bacteria not known to cause disease in people. To find out whether disease-causing species also carried such genes, the scientists scanned the genetic sequences of clinical samples Dantas had collected over the years. They found Tet(X7) in a bacterium that had caused a lung infection and sent a man to intensive care in Pakistan in 2016.

Tetracyclines have been around since the 1940s. They are one of the most widely used classes of antibiotics, used for diseases ranging from pneumonia, to skin or urinary tract infections, to stomach ulcers, as well as in agriculture and aquaculture. In recent decades, mounting antibiotic resistance has driven pharmaceutical companies to spend hundreds of millions of dollars developing a new generation of tetracyclines that is impervious to the two most common resistance strategies: expelling drugs from the bacterial cell before they can do harm, and fortifying vulnerable parts of the bacterial cell.

The emergence of a third method of antibiotic resistance in disease-causing bacteria could be disastrous for public health. To better understand how Tet(X7) works, co-senior author Niraj Tolia, PhD, a senior investigator at the National Institute of Allergy and Infectious Diseases at the NIH, and co-author Hirdesh Kumar, PhD, a postdoctoral researcher in Tolias lab, solved the structure of the protein.

I established that Tet(X7) is very similar to known structures but way more active, and we dont really know why because the part that interacts with the tetracycline rings is the same, Kumar said. Im now taking a molecular dynamics approach so we can see the protein in action. If we can understand why it is so efficient, we can design even better inhibitors.

Wencewicz and colleagues previously designed a chemical compound that preserves the potency of tetracyclines by preventing destructases from chewing up the antibiotics. In the most recent study, co-author Jana L. Markley, PhD, a postdoctoral researcher in Wencewiczs lab, evaluated that inhibitor against the bacterium from the patient in Pakistan and its powerful Tet(X7) destructase. Adding the compound made the bacteria two to four times more sensitive to all three of the latest generation of tetracyclines.

Our team has a motto extending the wise words of Benjamin Franklin: In this world nothing can be said to be certain, except death, taxes and antibiotic resistance, Wencewicz said. Antibiotic resistance is going to happen. We need to get ahead of it and design inhibitors now to protect our antibiotics, because if we wait until it becomes a crisis, its too late.

Gasparrini AJ, Markley JL, Kumar H, Wang B, Fang L, Irum S, Symister C, Wallace M, Burnham CAD, Andleeb S, Tolia NH, Wencewicz TA, Dantas G. Tetracycline-inactivating enzymes from environmental, human commensal, and pathogenic bacteria cause broad-spectrum tetracycline resistance. Communications Biology. May 15, 2020. DOI: 10.1038/s42003-020-0966-5

This work is supported by the National Institute of Allergy and Infectious Diseases of the National Institutes of Health (NIH), grant number R01 AI123394; the National Institute of General Medical Sciences, training grant number T32 GM007067; the National Institute of Diabetes and Digestive and Kidney Diseases, training grant number T32 DK077653; and Washington University, W.M. Keck Postdoctoral Program in Molecular Medicine and the Chancellors Graduate Fellowship Program.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

Continued here:
Antibiotic-destroying genes widespread in bacteria in soil and on people - Washington University School of Medicine in St. Louis