Category Archives: Cardiomyopathies

Phase 3 randomized withdrawal trial for cardiomyopathy in Barth planned ReCLAIM Phase 1 data presented at ARVO 2021 supports ReCLAIM-2 Phase 2 geographic atrophy trial design; data from fully enrolled ReCLAIM-2 trial expected second quarter of 2022 Type C Meeting with the Division of Rare Disease and Medical Genetics (DRDMG) to discuss Phase 3 trial in patients with mitochondrial disease caused by nuclear DNA mutations (nPMD) scheduled during the third quarter of 2021; trial initiation expected by end of 2021 Clinical expansion efforts underway for elamipretide in Duchenne and Friedreich's ataxia; pipeline development ongoing with SBT-272, SBT-550, and other early-stage compounds Additional funding committed under Development Funding Agreement; Hercules term loan extension Management to host conference call today at 8:30am ET BOSTON, May 18, 2021 /PRNewswire/ -- Stealth BioTherapeutics Corp (Nasdaq:MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today reported financial results for the quarter ended March 31, 2021 and announced recent business highlights. Stealth BioTherapeutics Logo (PRNewsFoto/Stealth BioTherapeutics) (PRNewsfoto/Stealth BioTherapeutics) "Our team is energized by the potential of both elamipretide and our deep pipeline of novel mitochondrial-targeted compounds to address the devastating and typically progressive disease burden associated with diseases of mitochondrial dysfunction and we are thrilled to have secured additional financial resources to expand these development efforts," said Reenie McCarthy, Chief Executive Officer at Stealth. Continue reading →

Alison Laurie-Chalmers. Continue reading →

Orange County has joined a five-county Southeast Texas group in an effort to get more COVID-19 vaccines into the arms of its citizens. John Gothia, Orange County judge, said the goal is to establish one or more permanent vaccination hub sites and possibly a mobile vaccine unit for use in Orange, Hardin, Jasper, Newton, and Jefferson Counties. By combining their public health resources and populations, the resulting "consortium" covering a population of nearly a half-million residents would be able to guarantee the state that it has the ability to give out 22,000 vaccinations per week, which Gothia explained is a requirement to be considered a permanent hub Continue reading →

BENSHEIM, Germany and PATRAS, Greece, December 16, 2020 / B3C newswire / -- Relaxera Pharmazeutische Gesellschaft mbH & Co. KG and CBL Patras (Chemical and Biopharmaceutical Laboratories) announce the signing of an exclusive contract for human relaxin-2. This cooperation agreement designates CBL as the exclusive manufacturing partner for synthetic human relaxin-2 (GMP and non-GMP) and Relaxera as the exclusive sales executive partner for relaxin. Continue reading →

Aims:Amyloid cardiomyopathy is an underappreciated cause of morbidity and mortality. Recent evidence suggests that ATTR wild-type cardiomyopathy (ATTRwt-CM) is probably much more common than widely appreciated. So far, no data are available on comparison of mortality from ATTRwt-CM and other heart failure aetiologies Continue reading →

The market has been high on Myokardia Inc (MYOK) stock recently. MYOK gets a Bullish score from InvestorsObserver's Stock Sentiment Indicator. Continue reading →

More than 35 million people around the world have tested positive for the novel coronavirus that causes Covid-19, according to data compiled by Johns Hopkins University. And even if that figure is an enormous undercount of the people who have had the disease as the World Health Organization and other experts say it is it means tens of millions of people have had the disease and then been able to leave isolation Continue reading →

BOSTON, Sept. Continue reading →

SOUTH SAN FRANCISCO, Calif., May 11, 2020 /PRNewswire/ --Tenaya Therapeutics, Inc., a company with a mission to discover, develop, and deliver potentially curative therapies that target the underlying causes of heart disease, today announced that it will present preclinical data from four studies as postersat the 23rd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), being held May 12-15, 2020, in a virtual format. Three posters illustrate advances made with Tenaya's Cellular Regeneration platform using direct reprogramming of resident cardiac fibroblasts to create new cardiomyocytes.The fourth poster illustrates advances made with the Company's Gene Therapy platform to identify novel adeno-associated virus (AAV) variants with superior attributes to parental serotypes, that can be used develop best-in-class therapies for several undisclosed genetic cardiomyopathies under development. Faraz Ali, Chief Executive Officer, stated, "These data highlightimportant advances in our science, our capabilities, and our intellectual property since Tenaya was founded in 2016. Continue reading →

BioMarin has just struck a deal to back a Swiss biotech startup with some deep ties to top research institution UCL in London as it beefs up the swelling gene therapy portion of the pipeline. We dont have any terms to deal with, just the knowledge that BioMarin CEO JJ Bienaim saw enough of DiNAQORs work to invest in the company as it licenses their lead preclinical program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy, while collaborating on the rest of the pipeline. Continue reading →