Category Archives: Genetic medicine

SAN RAFAEL, Calif., Aug. 20, 2020 /PRNewswire/ --BioMarin Pharmaceutical Inc.(NASDAQ: BMRN) today announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. BioMarin recently announced that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for vosoritide on Aug. 13, 2020.

"We are grateful to the children and families who have participated in the clinical studies to evaluate the potentially first pharmacological treatment option for children with achondroplasia. We are proud of our team and clinical partners, who have conducted extensive scientific and clinical research to address the underlying cause of the condition," said Hank Fuchs, M.D., President, Global Research and Development at BioMarin. "This is an important step to bring a treatment where none have existed before. We also recognize a broad range of views about treatment options, and we look forward to providing children with achondroplasia and their families a treatment choice with this potential new therapy."

"This regulatory submission is an important scientific and medical milestone for children with achondroplasia and their families," said Julie Hoover-Fong, MD, PhD, FACMG, Professor, McKusick-Nathans Department of Genetic Medicine andDirector, Greenberg Center for Skeletal Dysplasias at Johns Hopkins University and an investigator in the vosoritide clinical program. "The extensive clinical program for vosoritide includes important natural history information, which has contributed to the body of scientific knowledge about achondroplasia and a potential treatment option for patients."

"This regulatory submission brings our community closer to accessing the first therapeutic choice for children and families," said Chandler Crews, Founder of The Chandler Project. "A well-researched drug treatment choice has the potential to be an important resource for the community and to increase our understanding of the scientific underpinnings of achondroplasia."

The Chandler Project is dedicated to the most common form of dwarfism and other congenital abnormalities and is a means for patients and parents of children with achondroplasia to find the correct resources for themselves and their child.

About Achondroplasia

Achondroplasia, the most common form of disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull.This condition is caused by a mutation in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in the need for invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, studies show increased mortality at every age.

More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. Vosoritide is being tested in children whose growth plates are still "open", typically those under 18 years of age.This is approximately 25% of people with achondroplasia. In the U.S.,Europe,Latin America, theMiddle East, and most ofAsia Pacific, there are currently no licensed medicines for achondroplasia.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visitwww.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: BioMarin's vosoritide development program generally and specifically about the Company's submission of an NDA for vosoritide to the FDA and the EMA's validation of the MAA for vosoritide, which began onAugust 13, 2020. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: our ability to successfully manufacture vosoritide; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning vosoritide; and those other risks and uncertainties detailed from time to time under the caption "Risk Factors" and elsewhere in the BioMarin's Securities and Exchange Commission (SEC) filings, including, without limitation, BioMarin's Quarterly Report on Form 10-Q for the quarter endedJune 30, 2020, and future SEC filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

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BioMarin Submits New Drug Application to US Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia - BioSpace

MONCTON, New Brunswick--(BUSINESS WIRE)--Organigram Holdings Inc. (NASDAQ: OGI) (TSX: OGI), the parent company of Organigram Inc. (the Company or Organigram), a leading licensed producer of cannabis, is pleased to announce it has partnered with Medical Cannabis by Shoppers(Shoppers) on Phase 2 of Shoppers Pilot Program powered by software partner TruTrace Technologies Inc. (CSE: TTT; OTCQB: TTTSF) (TruTrace).

The program is designed to genetically finger-print all participating cannabis products, tracking them throughout the supply chain, from genome to patient, in order to provide real-time information about the composition of each cannabis product used by Medical Cannabis by Shoppers customers.Organigram will provide cannabis products to Shoppers for use in the tracking program.

Standardized and validated testing of medical cannabis, ensuring consistent quality and efficacy, are critical to the products value as a viable treatment option. Likewise, product information such as strain composition and potency can help healthcare practitioners and patients make more informed and confident decisions about their medical cannabis treatment regimens.

Organigram is proud of our long-standing commitment to our medical cannabis community. From the development of innovative products to the support offered by our patient care team and programs, patients and their needs are at the heart of our medical cannabis business, says Greg Engel, CEO, Organigram. We also recognize how critical consistency is to patients and their healthcare providers so are pleased to partner with Shoppers, providing our products so that they can be followed from raw material to finished product, to offer them important, useable product insights.

Using Trutraces StrainSecure system, the program collects plant testing data and performs genomic verification in plant batches which are then registered in a blockchain-enabled database for intellectual property protection and strain validation. All information gathered from the plants, including their molecular and chemical makeup, can be tracked via the technology.

As jurisdictions around the world have begun to legalize and adopt cannabis as a medical treatment, there has been an influx ofnew breeders and growers and a profusion of new cannabis strains, each with a different representation of at least 500 known metabolites. Subtle changes in the chemical expression of various strains, whether by genetic structure or environmental conditions, may have significant clinical effects on the patients using this treatment option.With so many strains available, and with relatively limitedinformationon strain composition or genetic lineage and their relation to their chemical output, patients havelittleability to control what they aretaking over time.

In the absence of assigned drug identification numbers (DIN)for cannabis products, quantifying the genetics and metabolomics, as well as potency and equivalencies ofcannabis products is of interest to producers, distributors, shippers, government agencies, payers, clinicians and patients.

Maintaining an effective traceability ecosystem about these details throughout the supply chain is a component of providing consistent medicine, says Engel.

Using TruTrace technology, Shoppers has partnered with University Health Network in Toronto (UHN) to launch Medical Cannabis Real World Evidence (MCRWE), a new ground-breaking study on cannabis and health which will track outcomes with TruTrace validated product for the first time in history.

This novel observational study is targeting a minimum of 2,000 patients who will be followed over a 24-week period. Enrolled patients will have access to certain fully verified products on the Medical Cannabis by Shoppers platform, which have been tested for detailed cannabinoid and terpene profiles.More information about the study can be found here.

About Organigram Holdings Inc.

Organigram Holdings Inc. is a NASDAQ Global Select and TSX listed company whose wholly owned subsidiary, Organigram Inc., is a licensed producer of cannabis and cannabis-derived products in Canada.

Organigram is focused on producing high-quality, indoor-grown cannabis for patients and adult recreational consumers in Canada, as well as developing international business partnerships to extend the Company's global footprint. Organigram has also developed a portfolio of legal adult use recreational cannabis brands including The Edison Cannabis Company, AnkrOrganics and Trailblazer. Organigram's facilityis located inMoncton, New Brunswick and the Company is regulated by theCannabis Act and theCannabis Regulations(Canada).

This news release contains forward-looking information. Often, but not always, forward-looking information can be identified by the use of words such as plans, expects, estimates, intends, anticipates, believes or variations of such words and phrases or state that certain actions, events, or results may, could, would, might or will be taken, occur or be achieved. Forward-looking information involves known and unknown risks, uncertainties and other factors that may cause actual results, events, performance or achievements of Organigram to differ materially from current expectations or future results, performance or achievements expressed or implied by the forward-looking information contained in this news release. Risks, uncertainties and other factors involved with forward-looking information could cause actual events, results, performance, prospects and opportunities to differ materially from those expressed or implied by such forward-looking information include factors that change supply quantities; risks associated with international jurisdictions including regulatory risk; receipt of any required permits from Health Canada and other authorities; including general risks related to COVID-19 and risks as disclosed in the Companys most recent annual information form, managements discussion and analysis and other Company documents filed from time to time on SEDAR (see http://www.sedar.com) and filed or furnished to the Securities and Exchange Commission on EDGAR (see http://www.sec.gov). Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Although the Company believes that the assumptions and factors used in preparing the forward-looking information in this news release are reasonable, undue reliance should not be placed on such information and no assurance can be given that such events will occur in the disclosed time frames or at all. The forward-looking information included in this news release are made as of the date of this news release and the Company disclaims any intention or obligation, except to the extent required by law, to update or revise any forward-looking information, whether as a result of new information, future events or otherwise.

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Organigram Joins Medical Cannabis by Shoppers Inc. and TruTrace in Effort to Track Source and Genetics of Cannabis Used by Medical Patients - Business...

SALT LAKE CITY, Aug. 21, 2020 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a global leader in molecular diagnostics and precision medicine, announced today that the American Society of Clinical Oncology (ASCO) has exclusively included Myriads myChoice CDx test in its new recommendations on the use of PARP inhibitors for the treatment and management of certain patients with advanced ovarian cancer. The new recommendations, based on clinical trial results, were published in the Journal of Clinical Oncology.

The guideline, titled PARP Inhibitors in the Management of Ovarian Cancer: ASCO Guideline, where myChoice CDx was the only named commercial companion diagnostic, states that women with ovarian cancer and germline or somatic mutations in BRCA1 or BRCA2 genes and/or genomic instability as determined by Myriad myChoice CDx are recommended by ASCO for PARP inhibitor therapy. The guideline includes myChoice CDx guided management in both newly diagnosed and recurrent ovarian cancer.

We are thrilled to be a part of the rapidly changing landscape in guiding treatment for patients with ovarian cancer. The new ASCO guidelines highlight the large number of recent studies that have gone into improving ovarian cancer patient outcomes, said Thomas Slavin, M.D., FACMG, DABCC, senior vice president of Medical Affairs for Myriad Oncology.

According to the American Cancer Society, ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system. In the United States, it is estimated there will be 21,750 new cases diagnosed and around 13,940 deaths in 2020. A woman's risk of getting ovarian cancer during her lifetime is about one in 78 and the chance of dying from ovarian cancer is about one in 108.

About Myriad myChoice CDx Myriad's myChoice CDx is the most comprehensive homologous recombination deficiency test, enabling physicians to identify patients with tumors that have lost the ability to repair double-stranded DNA breaks, resulting in increased susceptibility to DNA-damaging drugs such as platinum drugs or PARP inhibitors. The myChoice CDx test comprises tumor sequencing of the BRCA1 and BRCA2 genes and a composite of three proprietary technologies (loss of heterozygosity, telomeric allelic imbalance and large-scale state transitions). For more information, visit: https://myriad-oncology.com/mychoice-cdx/

About Myriad GeneticsMyriad Genetics Inc., is a leading personalized medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on three strategic imperatives: transitioning and expanding its hereditary cancer testing markets, diversifying its product portfolio through the introduction of new products and increasing the revenue contribution from international markets. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice CDx, Vectra, Prequel, Foresight, GeneSight, riskScore and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to the new ASCO guideline titled PARP Inhibitors in the Management of Ovarian Cancer: ASCO Guideline and the Companys myChoice CDx testings place in such guideline; and the Companys strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties associated with COVID-19, including its possible effects on our operations and the demand for our products and services; our ability to efficiently and flexibly manage our business amid uncertainties related to COVID-19; the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decisions in Mayo Collab. Servs. v. Prometheus Labs., Inc., 566 U.S. 66 (2012), Assn for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013), and Alice Corp. v. CLS Bank Intl, 573 U.S. 208 (2014); risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2020, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

Media Contact:Jared Maxwell(801) 505-5027jmaxwell@myriad.com

Investor Contact:Scott Gleason(801) 584-1143sgleason@myriad.com

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American Society of Clinical Oncology Exclusively Cites myChoice CDx in New Recommendations for Patients with Advanced Ovarian Cancer - BioSpace

While pre- and post-test education on genetic testing helped to educate patients, recent study findings may also highlight the way that telemedicine is revolutionizing the space, according to Kelly Morgan, MS, CGC.

In a recent study, presented at the 2020 ASCO Virtual Scientific Program, the BRCA Founder OutReach (BFOR) offered pre-testing online education with posttest engagement of primary care providers, which appeared to be effective in educating both patients and providers alike.

In an interview with CancerNetwork, Morgan, a genetic counselor at Memorial Sloan Kettering Cancer Center, explained how telemedicine is changing the future of genetic testing.

Transcription:

The current focus is going to be continuing to survey our participants and then analyze and report out our findings. These are sort of our first set of early results. But the goal from there is, there's a lot of different ways in which this information can probably be used in terms of next steps.

One of the things I mentioned was the idea of new ways to engage primary care providers. So that's certainly an area of interest. One thing I didn't mention, but it's also kind of along the lines of where we want to go is we were very happy with the recruitment that we achieved, but at the same time, we think this tool could be used even more broadly, potentially. So finding ways to better distribute this information and engage a broader number of participants. And then lastly, the immediate context for this is thinking about how this model may work for population screening in the Ashkenazi Jewish group. But there are ways that we could pivot this to other situations as well. So, whether that be things like testing family members for an unknown mutation, or maybe there are other groups with predominant risk factors where this model could work as well. So, I think that once we better understand the long term medical outcomes of participants, we will be able to continue to think about different ways that we can improve and change the model and then apply it further in this context and others as well.

I think, now more than ever, is a time where there's almost a digital revolution, if you will, and a lot of technology in the forefront. So, from our perspective, our goal is to find a way to leverage technology in the context of medicine and use it for better. So there's always a lot going on in terms of many different ways to access testing direct to consumer testing. And, you know, if and when this sort of convenience can be combined with medicine, I think there's a lot of opportunities for improved patient care through that partnership.

Originally posted here:
Kelly Morgan, MS, CGC, the Future of Telemedicine and Genetic Testing - Cancer Network

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

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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...