Category Archives: Genetic medicine

Aug. 21, 2020 13:07 UTC

For the first time, up to 10,000 people in Europe ages 12 years and older with one F508del mutation and one minimal function mutation will be eligible for a medicine that treats the underlying cause of cystic fibrosis

People 12 years of age and older who have two F508del mutations will also be eligible for the new triple combination regimen

LONDON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission (EC) has granted marketing authorization for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor to treat people with cystic fibrosis (CF) ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

For the first time, up to 10,000 people in Europe ages 12 years and older with CF who have one F508del mutation and one minimal function mutation will be eligible for a CFTR modulator that treats the underlying cause of the disease. Approval of the triple combination regimen also expands the number of treatment options available to people ages 12 years and older with CF who have two copies of the F508del mutation, the most common CF-causing mutation worldwide.

Today is a significant day for those with CF, their families and Vertex, and one that brings us one step closer towards our ultimate goal of discovering and developing treatments for all patients with CF, said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. I would like to thank our dedicated scientists, as well as study investigators and people with CF who participated in our clinical trials to enable this innovative medicine to be approved in Europe today. Without their commitment, this milestone would not have been possible.

As a result of long-term reimbursement agreements in England, Denmark and the Republic of Ireland, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the triple combination regimen in the upcoming weeks. Vertex is committed to working closely with national health authorities and governments in all other countries in Europe to secure access for eligible patients as quickly as possible.

Marketing authorization was based on the results of two global Phase 3 studies, which showed statistically significant and clinically meaningful improvements in lung function (primary endpoint) and all key secondary endpoints, in people with CF ages 12 years and older with one F508del mutation and one minimal function mutation or two F508del mutations in the CFTR gene. The triple combination regimen was generally well tolerated in both studies.

The triple combination regimen has been shown to have a major impact on several outcome measures in people with CF, said Professor Harry Heijerman, Professor and Head of the Department of Pulmonology at University Medical Center Utrecht, Netherlands. The clinical data showed significant improvements in lung function and other important measures, such as sweat chloride levels and quality of life as measured by the CFQ-R respiratory domain score, in patients treated with the triple combination therapy. I now look forward to seeing the impact of the medicine in clinical practice.

About Cystic Fibrosis

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

About KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a Combination Regimen With ivacaftor

KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg was developed for the treatment of cystic fibrosis (CF) in patients ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KAFTRIO is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The EU submission for KAFTRIO was supported by positive results of two global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a four-week Phase 3 study in 107 people with two F508del mutations (F/F).

About Vertex

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

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America,Europe,AustraliaandLatin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Reshma Kewalramani and Professor Harry Heijerman in this press release, statements regarding the eligible patient population in Europe, our expectations regarding the timing of access to the triple combination regimen across countries in Europe, and our plans to secure access to our medicine for additional patients in Europe. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, risks related to commercializing medicines in Europe, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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European Commission Approves KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibrosis in People Ages 12 Years...

Sarepta Therapeutics Inc. (SRPT) is priced at $140.97 after the most recent trading session. At the very opening of the session, the stock price was $144.00 and reached a high price of $144.345, prior to closing the session it reached the value of $148.66. The stock touched a low price of $138.71.

Recently in News on August 11, 2020, Sarepta Therapeutics and University of Florida Announce Collaboration to Accelerate the Discovery and Development of Therapies for Rare Genetic Diseases. Sarepta Therapeutics Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and the University of Florida today announced a strategic collaboration to enable cutting-edge research for novel genetic medicines. Through the agreement, Sarepta will fund multiple research programs at the University, and will have an exclusive option to further develop any new therapeutic compounds that result from the funded research programs. You can read further details here

Sarepta Therapeutics Inc. had a pretty favorable run when it comes to the market performance. The 1-year high price for the companys stock is recorded $175.00 on 07/20/20, with the lowest value was $78.06 for the same time period, recorded on 03/18/20.

Price records that include history of low and high prices in the period of 52 weeks can tell a lot about the stocks existing status and the future performance. Presently, Sarepta Therapeutics Inc. shares are logging -19.45% during the 52-week period from high price, and 95.66% higher than the lowest price point for the same timeframe. The stocks price range for the 52-week period managed to maintain the performance between $72.05 and $175.00.

The companys shares, operating in the sector of Healthcare managed to top a trading volume set approximately around 1160694 for the day, which was evidently higher, when compared to the average daily volumes of the shares.

When it comes to the year-to-date metrics, the Sarepta Therapeutics Inc. (SRPT) recorded performance in the market was 9.25%, having the revenues showcasing -3.27% on a quarterly basis in comparison with the same period year before. At the time of this writing, the total market value of the company is set at 11.21B, as it employees total of 743 workers.

During the last month, 19 analysts gave the Sarepta Therapeutics Inc. a BUY rating, 2 of the polled analysts branded the stock as an OVERWEIGHT, 1 analysts were recommending to HOLD this stock, 0 of them gave the stock UNDERWEIGHT rating, and 0 of the polled analysts provided SELL rating.

According to the data provided on Barchart.com, the moving average of the company in the 100-day period was set at 144.54, with a change in the price was noted +42.45. In a similar fashion, Sarepta Therapeutics Inc. posted a movement of +43.09% for the period of last 100 days, recording 852,747 in trading volumes.

Total Debt to Equity Ratio (D/E) can also provide valuable insight into the companys financial health and market status. The debt to equity ratio can be calculated by dividing the present total liabilities of a company by shareholders equity. Debt to Equity thus makes a valuable metrics that describes the debt, company is using in order to support assets, correlating with the value of shareholders equity The total Debt to Equity ratio for SRPT is recording 0.67 at the time of this writing. In addition, long term Debt to Equity ratio is set at 0.67.

Raw Stochastic average of Sarepta Therapeutics Inc. in the period of last 50 days is set at 6.23%. The result represents downgrade in oppose to Raw Stochastic average for the period of the last 20 days, recording 10.00%. In the last 20 days, the companys Stochastic %K was 21.79% and its Stochastic %D was recorded 31.90%.

Considering, the past performance of Sarepta Therapeutics Inc., multiple moving trends are noted. Year-to-date Price performance of the companys stock appears to be pessimistic, given the fact the metric is recording 9.25%. Additionally, trading for the stock in the period of the last six months notably improved by 13.70%, alongside a boost of 38.89% for the period of the last 12 months. The shares increased approximately by -11.34% in the 7-day charts and went up by -14.31% in the period of the last 30 days. Common stock shares were lifted by -3.27% during last recorded quarter.

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Analysts Mean recommendation for Sarepta Therapeutics Inc. (SRPT) was 1.70: Is this the key time? - The InvestChronicle

Newswise Irvine, CA August 21, 2020 A new study finds women with diabetes and significant levels of calcium in their coronary arteries have higher rates of death from cardiovascular disease and all causes than their male counterparts.

Published in the American Diabetes Association journal, Diabetes Care, researchers from the University of California, Irvine School of Medicine and Cedars-Sinai Medical Center compared the sex-specific impact of coronary artery calcium (CAC) levels in adults with diabetes. CAC was used to predict cardiovascular and all-cause mortality in patients with diabetes. The results of this comparison showed greater CAC predicts cardiovascular and total mortality more strongly in women.

We showed that coronary calcium scores of greater than 100 in a woman with diabetes was associated with higher death rates from cardiovascular diseases and all causes than similar calcium scores in women than in man with diabetes, said Nathan D. Wong, PhD, professor and director for UCIs Heart Disease Prevention Program, and the lead author for the study.

Wong and colleagues studied 4,503 adults with diabetes from a national registry of patients who received coronary calcium heart scans from computed tomography and were followed for causes of death over more than 11 years. Death rates from cardiovascular disease in those who had coronary calcium scores of 101-400 or more, were approximately twice as high in women compared to men. Total death rates in these patients were also higher in women than in men. In analyses adjusted for age and other potential confounders, compared to those with calcium scores of 0, women who had calcium scores of 101-400 and 401 or greater had cardiovascular deaths that were 3.7 and 6.3-fold greater, respectively, compared to men whose risks were 1.6 and 3.5-fold greater, respectively.

Our findings, showing significant levels of coronary calcium to predict mortality from cardiovascular causes more strongly in women than men with diabetes, might also help to explain the poorer prognosis for cardiovascular disease that has been observed for decades in women compared to men with diabetes, said Wong.

Conversely, very low death rates from coronary heart disease and cardiovascular disease seen in those with diabetes who had negative scans (calcium scores of 0), comprising 39 percent of women and 20 percent of men in our study, underscore the point that not all persons with diabetes are risk equivalents for cardiovascular disease, as has been the common belief for decades, noted Cedars-Sinai Medical Centers Daniel Berman, MD, senior author of the study.

Our findings suggest a call-to-action for even more aggressive risk factor management in a woman with diabetes found to have significant levels of coronary calcium to prevent future death from cardiovascular causes said Wong. Previous research conducted by Wong and colleagues, has shown rates of cardiovascular disease to be 60 percent lower in those who are well-controlled for blood sugar, cholesterol, and blood pressure.

The study population was part of the CAC Consortium, directed by Michael Joseph Blaha, MD, MPH, from Johns Hopkins School of Medicine. UCIs Amber Cordola-Hsu, PhD, co-led the study with Wong.

This study was funded in part by the National Institutes of Health and the American Heart Association.

About the UCI School of Medicine

Each year, the UCI School of Medicine educates more than 400 medical students, and nearly 150 doctoral and masters students. More than 700 residents and fellows are trained at UCI Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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UCI study finds women with diabetes and high levels of coronary artery calcium at greater risk of death than men - Newswise

1. Reduced physical activity as a result of COVID-19 related restrictions may be associated with higher levels of stress and anxiety.

2. Relationships between perceived activity level, stress, and anxiety are confounded by genetic and environmental factors in addition to age and sex.

Evidence Rating Level: 2 (Good)

Due to the Covid-19 pandemic, restrictions have been in place worldwide to limit the spread of the virus. Some of these restrictions also limit the opportunity for physical activity, such as the closure of athletic facilities and shelter-in-place orders. Since past research has associated lack of physical activity to poorer mental health, there is a growing need for empirical research regarding the restrictions effect on physical and mental health outcomes. The aim of this study was to investigate the association between perceived alterations in physical activity (due to the restrictions) and mental health. The study population was 3,971 adults taken from a twin registry in Washington State, including 909 same-sex twin pairs. Twins were used in this study to control for genetic and shared environmental factors, as changes in perceived physical activity were anticipated to be stemming from non-shared environmental factors. Participants completed an online survey which assessed perceived changes in physical activity (increased, decreased, or stayed the same), stress (using the 5-point Likert-type Perceived Stress Scale), and anxiety (using the Brief Symptom Inventory, also on a 5-point scale). The study found that there was no association between physical activity and mental health, in twins who reported an increase or no change in activity (stress: b = 0.089, SE = 0.060, p = 0.139; anxiety: b = 0.117, SE = 0.079, p = 0.141). For twins reporting a decrease or no change in activity, there was a significant association between activity and stress before controlling for the confounding variables (b = 0.036, SE = 0.010, p < 0.001). After controlling for genetics and shared environment though, the association was non-significant (b = 0.017, SE = 0.010, p = 0.090). For twins reporting decreased or no change in activity, the association with anxiety was significant before controlling (b = 0.143, SE = 0.039, p < 0.001), was still significant after controlling for genetics and shared environment (b = 0.134, SE = 0.042, p = 0.002), but was ultimately non-significant after controlling for age and sex, as older twins were more likely to report lower anxiety levels and females more likely to report higher anxiety levels (b = 0.150, SE = 0.106, p = 0.158). Overall, decreased perceived physical activity was linked to higher stress and anxiety, although the association with stress was confounded by genetics and shared environment, and anxiety with age and sex. This study demonstrates that the restrictions in place to protect public health could potentially be detrimental to physical and mental health, which has implications for potential interventions targeted at improving peoples well-being while restrictions are still in place.

Click to read the study in PlosONE

Image: PD

2020 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.

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Reduced physical activity from COVID-19 related restrictions may be linked to higher stress and anxiety levels - 2 Minute Medicine

Governor Roy Cooper announced today that Beam Therapeutics (Nasdaq; BEAM), a biotechnology company developing precision medicines through DNA base editing, plans to build a manufacturing facility in North Carolinas Research Triangle Park, creating 201 jobs. Over a period of 5 years, the company expects to invest $83 million in the facility, which will support clinical and commercial manufacturing for the companys novel base editing programs.

"North Carolina is a leader in biotechnology, from the research in our labs to the states biomanufacturers, said Governor Cooper. Companies like Beam Therapeutics work in developing precision medicines will help keep North Carolina on the cutting edge of this industry.

Beam Therapeutics, with headquarters in Cambridge, Massachusetts, develops precision genetic medicines through base editing. The foundational level of genetic information is a single base letter in DNA, and an error to a single letter, known as a point mutation, can cause disease. Base editors have the ability to rewrite just a single letter, and thereby intervene at the most foundational level. Beams proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs.

We believe investment in strategic manufacturing capabilities is an important component of fully realizing the power of our base editing technology and achieving our vision to provide life-long cures to patients suffering from serious diseases, said John Evans, CEO of Beam Therapeutics. Research Triangle Park is a thriving biopharmaceutical hub, providing significant access to the broad range of talent we will need to make this vision a reality.

Although wages will vary depending on position, the average salary for the new positions will be $102,654. The average wage in Durham County is $71,756. The state and local area will see a yearly economic impact of more than $20.6 million from this companys new payroll.

"North Carolina has been a world leader in biotechnology for many years, but were not resting on our past accomplishments, said North Carolina Commerce Secretary Anthony M. Copeland. Beam Therapeutics joins a host of gene therapy companies that are keeping North Carolina at the forefront of this new frontier of medicine.

Beam Therapeutics project in North Carolina will be facilitated, in part, by a Job Development Investment Grant (JDIG) approved by the states Economic Investment Committee earlier today. Over the course of 12 years, the project is estimated to grow the states economy by $1.36 billion. Using a formula that takes into account the new tax revenues generated by the new jobs, the agreement authorizes the potential reimbursement to the company of up to $3,237,750, spread over 12 years. Payments for all JDIGs only occur following performance verification by the departments of Commerce and Revenue that the company has met its incremental job creation and investment targets. JDIG projects result in positive net tax revenue to the state treasury, even after taking into consideration the grants reimbursement payments to a given company.

Because Beam Therapeutics chose a site in Durham County, classified by the states economic tier system as Tier 3, the companys JDIG agreement also calls for moving as much as $1,079,250 into the states Industrial Development Fund Utility Account. The Utility Account helps rural communities finance necessary infrastructure upgrades to attract future business. Even when new jobs are created in a Tier 3 county such as Durham, the new tax revenue generated through JDIG grants helps more economically challenged communities elsewhere in the state. More information on the states economic tier designations is available here.

In addition to the North Carolina Department of Commerce and the Economic Development Partnership of N.C., other key partners on this project were the the North Carolina Community College System, the North Carolina Biotechnology Center, Durham County, and the Greater Durham Chamber of Commerce.

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Governor Cooper Announces Genetic Medicine Company Will Create 201 Jobs in Durham County - NC Dept of Commerce

LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today reported financial results for the quarter ended June 30, 2020, provided a business update and announced the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for LB-001 for the treatment of methylmalonic acidemia in pediatric patients. LogicBio released a separate press release this morning providing further details on the planned Phase 1/2 clinical design for LB-001.

We are thrilled to have received clearance to move forward with this first-in-human clinical trial with our lead product candidate, LB-001, for the treatment of methylmalonic acidemia, a life-threatening congenital genetic disease with no current therapeutic treatment options. This represents a significant milestone in our goal of bringing a treatment to MMA patients as well as for our GeneRide platform. We have maintained continuous dialogue with the centers of excellence that are planned to participate in the Phase 1/2 clinical trial, and we look forward to activating these sites as quickly as possible, said Fred Chereau, CEO of LogicBio. We have instituted systems attempting to mitigate COVID-19 dynamics on our study start-up process and, based on our best estimates, we plan to enroll our first patient in early 2021.

Commenting on the Next Generation Capsid Program, Mr. Chereau said, We are very excited about the recent advances in our novel capsid program, which has generated liver-tropic capsids intended for use in gene editing technologies such as GeneRide and other gene therapy approaches. We are focused on executing across all of our programs and look forward to sharing further details on our novel capsids in early 2021.

Appointment of Daniel Gruskin, M.D. to SVP, Head of Clinical Development

Daniel Gruskin, M.D. was appointed as SVP, head of clinical development in August 2020. Dr. Gruskin has served as interim head of clinical development of LogicBio since June 2020. In April 2020, Dr. Gruskin started consulting with the Company as a special advisor. Previously, Dr. Gruskin served in roles of increasing responsibility at Sanofi Genzyme, most recently as vice president, head of global medical affairs, rare disease, in which capacity he oversaw medical affairs, life cycle management, scientific affairs and other medical and development activities related to metabolic, rare and/or genetic diseases. Prior to his role at Sanofi Genzyme, Dr. Gruskin served as assistant professor, human genetics and pediatrics at Emory University School of Medicine, where he was also the chief of the genetics section at Childrens Healthcare of Atlanta.

Daniel has been instrumental in leading LB-001 clinical development efforts including getting the IND cleared. His deep experience in genetic medicines and metabolic diseases will serve LogicBio well as we look to execute on our goals for both the GeneRide and Next Generation Capsid platforms in search of transformative medicines, said Mr. Chereau.

Anticipated Milestones for 2020 and 2021:

Second Quarter 2020 Financial Results

Three Months Ended June 30, 2020 and 2019

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms.

LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered inLexington, Mass. For more information, please visitwww.logicbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the Companys plans to initiate, advance and complete its planned SUNRISE Phase 1/2 clinical trial of LB-001 in MMA; the timing, progress and results of the Companys research and development activities, including those related to the GeneRide technology platform and Next Generation Capsid Program; its plans for LB-301 in Crigler-Najjar; and the sufficiency of its cash and cash equivalents to fund operating expenses and capital expenditure requirements. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts:

Investors:Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Media:Stephanie SimonTen Bridge CommunicationsStephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business UpdatesFDA Clears IND Application for LB-001 for the...

CAMBRIDGE, Mass. and GAINESVILLE, Fla., Aug. 11, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and the University of Florida today announced a strategic collaboration to enable cutting-edge research for novel genetic medicines. Through the agreement, Sarepta will fund multiple research programs at the University, and will have an exclusive option to further develop any new therapeutic compounds that result from the funded research programs.

We have developed a productive incubator approach to our pipeline development, partnering with the best and brightest in genetic medicine, including leading academic researchers like those at the University of Florida, to discover and translate into meaningful therapies genetic medicine for rare diseases, said Sarepta President and CEO Doug Ingram. Weare excited topartner with andsupportUF research that has the potentialto profoundlyimproveand extend the lives of patients with rare genetic-based diseases.

Through the collaboration, currently unique to UF, funding has been allocated for four innovative projects. These projects include exploratory research in novel gene therapy vectors, next-generation capsids and gene editing technologies as well as work in new therapeutic areas in degenerative genetic diseases. The goal is to foster early relationships with experts and accelerate the scientific advancements that lead to the development of transformational precision genetic medicines for patients in need.

Our researchers intend to find solutions for diseases that have no cure or limited therapeutic options. Their goal is to move these solutions from their labs to patients who need them to see their discoveries change lives. Because Sarepta has a focus and expertise in disease areas that coincide with the work of some of our scientists, its a match and collaboration that make sense and, we hope, will save lives, said Jim OConnell, assistant vice president of UF Innovate, the technology commercialization arm of the university. Sarepta has a bold vision for transforming genetic disease because the company, ultimately, serves patients. That end goal drives its willingness and ability to translate research into a medical reality. We want to be part of that.

University of Florida is a gene therapy powerhouse. UF researchers were the first to discover the life cycle of the adeno-associated virus (AAV), the smallest human virus. Using AAV as a benign delivery vehicle to carry therapeutics to a target, UF was first to reverse blindness in dogs with genetic disease, and UF researchers were integral in the first gene therapy approved by the FDA to treat an inherited genetic disease that can cause blindness. Today, UF is developing technologies in manufacturing, capsid design and therapies to address neuromuscular, cardiovascular, inflammatory, metabolic, pulmonary, skeletal, ophthalmic, and other disorders.

About SareptaAt 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 Statements 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 ability of the collaboration between Sarepta and UF to engage in cutting-edge research for novel genetic medicines; Sareptas commitment to fund multiple research programs at UF; Sareptas option to further develop any new therapeutic compounds that result from the funded research programs; Sareptas incubator approach to discover and translate into meaningful therapies genetic medicine for rare diseases; the collaborations potential to profoundly improve and extend the lives of patients with rare genetic-based diseases; the collaborations ability to foster early relationships with experts to accelerate the scientific advancements that lead to the development of transformational precision genetic medicines; and Sareptas vision to transform genetic disease and translate research into a medical reality.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and UF 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 the discovery of any new therapeutic compounds or 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; 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.

Contacts:

Sarepta Therapeutics Investors: Ian Estepan, 617-274-4052, iestepan@sarepta.comMedia: Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com

UF Innovate: Sara Dagen, 352-294-0998, saradagen@ufl.edu

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Sarepta Therapeutics and University of Florida Announce Collaboration to Accelerate the Discovery and Development of Therapies for Rare Genetic...

Conspiracy theories are everywhere and in the age of social media, theyre hard to escape. But why do they flourish so easily, even when we have so much information at our fingertips? According to Pulling The Thread, a new digital series on WORLD Channel, our brains have a lot to do with it.

The capacity of the human brain to be irrational is really remarkable, said Pulling The Thread filmmaker Kristin Pichaske about conspiracy theories. The six-part series dives into cognitive psychology to understand why were so susceptible to misinformation. The goal of the series is really to help us all think about how we think. And how can we, by understanding how our brains work, hope to be a little more rational and more accurate in terms of what we think, what information we take in, and, maybe most importantly, what sorts of media we share online.

Each episode dives into an area that is rife with conspiracy theories, from politics to science fiction to tragedies. Episode three explores the roots of medical conspiracy thought in the Black community, and the lingering healthcare challenges that this legacy perpetuates today. "Conspiracy theories about HIV are really understandable if you look at the context of how that played out, but have negative health outcomes, right? So people who think [HIV] was created in a lab somewhere on purpose are less likely to get tested and engage in safe behavior," says Pulling the Thread filmmaker Kristin Pichaske.

Here are five reasons conspiracy theories thrive in medicine, according to Pulling The Thread:

1. African Americans are more likely to mistrust doctors because of real historical events.

The Tuskegee Study, conducted between 1932 and 1972 by the United States Public Health Service, followed African American men with syphilis so that doctors could learn about the natural course of the disease. The men were offered free treatment for the disease, but were never actually given any medical treatment, even after a reliable cure for the disease was discovered. Not only did many of the patients die from a curable disease; they also unwittingly passed the disease to their spouses. The experiment continued for forty years, and was only stopped when a whistleblower made the study public. "This sounds like a prototypical conspiracy theory," says Rob Brotherton, author of Suspicious Minds: Why We Believe Conspiracy Theories, in the film. "This shady experiment that was put on by the government, which involved people's health and keeping important information from them." But it's not a conspiracy theory: it's 100% real.

2. The Tuskegee Study wasn't an isolated incident.

The study ended in 1972; well within living memory. For many young people in the Black community, stories of medical harm from parents and grandparents have a profound and lasting impact. "Automatically, in your brain you're going to be thinking 'I can't trust these people,'" says Alicia Williams, Community Outreach and Engagement Manager at Howard Brown Health, in the film.

Medical research in the United States has been an institutionalized system of inequality from its inception. One historical example is that of HeLa cells, an immortal cell line used for breakthrough cancer research, which were unwillingly and unknowingly taken from a Black woman, Henrietta Lacks. Lacks' family was never compensated, even though her genetic contributions led to major medical discoveries. And this is just one example: Black Americans have been abused by the medical system and used as unwilling or unknowing test subjects throughout the history of the United States, and are still recieving substandard medical care to this day.

3. Sometimes, paranoia is protection...

"When you talk about levels of mistrust, is it paranoia, or is it self care?" asks Dexter Voisin, University of Chicago Professor, in the film. Black women are 4 to 5 times more likely to die from childbirth than white women. Studies have found that Black patients are systematically undertreated for pain, and also that most health care professionals have an implicit bias against Black patients, which contributes to a lower quality of care and worse outcomes for Black patients. For Black Americans, knowing the long history of mistreatment from the medical establishment, being wary of doctors isn't just understandable it can be the safest choice.

4. ... and other times, Prudent Paranoia puts people at risk.

The HIV epidemic took hold only about 10 years after the Tuskegee Study concluded, and the mysterious new disease disproportionately affected marginalized communities, including Black Americans. It was a perfect candidate for conspiracy. For doctors and community advocates, this made effectively stemming the tide of transmission and treating the disease much more difficult, because the Black community was and is understandably hesitant to trust. Years of mistreatment left people vulnerable to conspiracy thought, despite the unlikelihood of a global conspiracy to hide a cure for HIV. Based on mathematical models, David Grimes, a Mathematician at Queens University, says that, "the idea of trying to sustain that many people for that long in a dangerous, unethical fiction? it just doesn't work when you put the numbers to it."

5. Facts are no match for fear and distrust.

Just because an algorithm exists to debunk conspiracy theories doesn't mean people will believe it. "I'm telling you, that pales to the strength of the human mind to believe whatever it wants," says Williams in the film. "The problem isn't the lack of trust in the African American community, it's the lack of trustworthiness in the medical community," says Dr. Maya Green, Medical Director at Howard Brown Health. The only antidote to conspiracy thought is building trust by respecting the autonomy of Black patients and working with medical personnel who are themselves members of the communities they seek to help.

Curious about how your own brain works in regard to conspiracy theories? Use the Discussion and Activity Guide to explore for yourself and watch the whole series here.

Chris Hastings, WORLD Channel Executive Producer and Editorial Manager, contributed to this interview.

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5 Reasons Why Conspiracy Theories Thrive In Medicine And Health - wgbh.org

Injections of a natural energy molecule prompted regrowth of almost half of the cartilage lost with aging in knees, a new study in rodents shows.

The study results revolve around the long-established idea that machines within animal and human cells turn the sugars, fats, and proteins we eat into energy used by the bodys millions of cells. The molecule most used to store that energy is called adenosine triphosphate, or ATP. Along with this central role in metabolism, adenosine also helps signal other cells and serves as a building block of genetic material, and so is central to the growth of human tissue.

Previous research had shown that maintaining supplies of adenosine, known to nourish the chondrocyte cells that make cartilage, also prevented osteoarthritis in similar animal models of the disease.

In the new NYU Grossman School of Medicineled study, researchers injected adenosine into the joints of rodents whose limbs had been damaged by inflammation resulting from either traumatic injury, such as a torn ligament, or from massive weight gain placing pressure on joints. The biological damage in these cases is similar, researchers say, to that sustained in human osteoarthritis.

Published online in the journal Scientific Reports on August 10, the study rodents received 8 weekly injections of adenosine, which prompted regrowth rates of cartilage tissue between 50 percent and 35 percent as measured by standard laboratory scores.

Our latest study shows that replenishing adenosine stores by injection works well as a treatment for osteoarthritis in animal models of the disease, and with no apparent side effects, says lead study author Carmen Corciulo, PhD, a postdoctoral fellow at NYU Langone.

Dr. Corciulo says it is too soon to use this experimental model as a therapy in people. Clinical trials must await a test drug that can be safely stored for days if not weeks, and experiments in larger mammals.

Study senior investigator Bruce N. Cronstein, MD, the Dr. Paul R. Esserman Professor of Medicine at NYU Langone Health, says the teams research is important because the few existing drug therapies for osteoarthritis such as acetaminophen and COX-2 inhibitor drugs, including naproxen and ibuprofen, only numb joint pain, or like hyaluronic acid just lubricate its tissues. None stall disease progression or reverse the damage. Painkillers, such as opioids, are often prescribed, but are also highly addictive, he cautions.

People with osteoarthritis desperately need more treatment options with fewer side effects, and our research advances that effort, says Dr. Cronstein, who also serves as the director of NYU Langones Clinical and Translational Science Institute. He notes that other experimental medications are being developed elsewhere, including parathyroid hormone to stimulate bone growth, WNT inhibitor drugs to block the bone and cartilage degradation, and growth factor chemicals to promote cartilage growth.

Dr. Cronstein, Dr. Corciulo, and NYU Grossman School of Medicine have a patent application pending for the use of adenosine and other agents that help with its binding to chondrocytes, called A2A receptor agonists, for the treatment of osteoarthritis.

Among the studys other key findings was that a cell-signaling pathway, known as transforming growth factor beta (TGF-beta) and involved in many forms of tissue growth, death, and differentiation, was highly active in cartilage tissue damaged by osteoarthritis, as well as in cartilage tissue undergoing repair after being treated with adenosine. Additional testing in lab-grown chondrocytes from people with osteoarthritis showed different chemical profiles of TGF-beta signaling during breakdown than during growth, providing the first evidence that the pathway switched function in the presence of adenosine (from assisting in cartilage breakdown to encouraging its repair.)

Developing treatments to halt or slow the disease is important, Dr. Cronstein says, because well over 100 million people worldwide are estimated to have osteoarthritis, which is tied to aging, especially in women. This figure, he says, is only expected to grow as more people live longer and obesity rates climb.

Right now, the only way to stop osteoarthritis is to have affected joints surgically replaced, which not only comes with pain and risk of infection, but is also quite costly, says Dr. Cronstein. If new therapies can delay or prevent disease onset and progression, then fewer joint replacements will save people from a lot of pain and expense.

The study was funded by National Institutes of Health grants R01 AR056672 and R01 AR068593, NYU-HHC Clinical and Translational Science Institute grant UL1 TR000038, and the Arthritis Foundation.

Dr. Corciulo and Dr. Cronstein have a patent for the methods and compositions for treating osteoarthritis and promoting cartilage formation (U.S. Patent 10,441,541), which has been assigned to NYU Grossman School of Medicine. They are cofounders of Regenosine Inc., a company that is developing new treatments for osteoarthritis, and in which they hold a financial interest. Dr. Cronstein has consulted for Eli Lilly, Horizon Pharmaceuticals, Bristol Myers Squibb, and Astrazeneca. He also has grants from Arcus Biopharma. All relationships are being managed in accordance with the policies and practices of NYU Langone.

Besides Dr. Cronstein and Dr. Corciulo, other NYU Langone investigators involved in this study are Cristina Castro, MD; Thomas Coughlin, PhD; Samson Jacob, MS; David Fenyo, PhD; Daniel B. Rifkin, PhD; and Oran Kennedy, PhD.

David MarchPhone: 212-404-3528david.march@nyulangone.org

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New Approach to Treating Osteoarthritis Advances | NYU Langone News - NYU Langone Health

Published on August 11, 2020

Researchers from MedUniVienna have successfully identified a blockade of compounds that activate a rare mutation found in malignant mesothelioma cells, thus halting their unrestricted growth. This highly specific approach makes clear that the best way to treat this rare and fatal form of cancer is through a highly tailored approach.

The mesothelioma investigators focused on the impact that telomerase has on the growth and spread of cancer cells. Telomerase is an enzyme that is active in rapidly dividing cells in our bodies. Though it plays a positive role in most instances, it also facilitates many types of cancer growth and is found in excessive amounts in mesothelioma patients.

The researchers determined that a particularly aggressive subgroup of malignant pleural mesothelioma cells have a distinct genetic mutation that serves to promote the production of the TERT gene, which plays an outsized role in the activation of telomerase. Through this discovery, they were able to identify a new treatment strategy for those suffering from the rare form of cancer and who test positive for the mutation.

Writing in the journalClinical Cancer Research, the researchers explained that the mutation they identified is found in the regulatory region of the cancer cell, which determines how much of a protein is produced. Previous cancer studies have shown that mutations activating the TERT gene are found in families with high genetic tendencies towards cancer, and are associated with other highly aggressive tumors like melanoma and glioblastoma.

Though the TERT gene mutation is only present in a small subgroup of patients diagnosed with malignant pleural mesothelioma, those that have it generally have an extremely poor prognosis. The researchers found that by inhibiting a specific protein linked to the activation of the mutated TERT gene, they were able to block its aggressiveness. According to Walter Berger, Member, Comprehensive Cancer Center, Institute of Cancer Research, Medical University of Vienna, We are currently investigating whether a pharmacological blockade of the ETS factors has potential as a new treatment option for patients with TERT promoter-mutated MPM. If this proves to be the case, the mutation would be both a biomarker for the selection of suitable patients and a therapeutic target an ideal combination for precision medicine.

Every person diagnosed with malignant pleural mesothelioma has a different story of exposure and a different experience with the disease. The Patient Advocates at Mesothelioma.net are here to help you make sense of your situation. Contact us today at 1-800-692-8608.

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Vienna Researchers' Mesothelioma Discovery Highlights Use of Precision Medicine - Mesothelioma.net Blog