Latest news with #BioCentury
Boston Globe
18-07-2025
- Health
- Boston Globe
Patient dies in Sarepta gene therapy trial, adding to safety concerns
Two teenage boys died after receiving Elevidys, Sarepta's gene therapy for Duchenne muscular dystrophy, forcing the company to take the drug off the market for older patients. The 51-year-old man was in a clinical trial for limb girdle muscular dystrophy, a collection of rarer and generally slower-moving conditions. Advertisement BioCentury first reported the man's death. The death will likely further stoke safety concerns around Sarepta's gene therapies, as well as raise questions around the company's transparency. Although they deliver different genes, Elevidys and the company's experimental limb girdle gene therapies rely on the same engineered virus to ferry the gene to patients' muscles. In all three cases, patients suffered acute liver failure after treatment. Sarepta did not disclose the patient's death Wednesday, when it announced that it was restructuring and laying off 500 employees. The company revealed it was discontinuing all but one of its limb girdle programs — it was developing treatments for six different subtypes — but described it as a financially motivated decision, as all subtypes are quite rare. Asked on a Wednesday investor call if the decision was driven by any safety concerns, CEO Doug Ingram said the company had made 'a risk-adjusted net present value' calculation and determined the financial opportunity didn't justify the costs of developing the limb girdle treatments. Advertisement A Sarepta spokesperson said Thursday the company 'prioritized disclosing to clinicians, regulators, and the community.' Sarepta informed the patient community about the fatal case earlier Thursday, weeks after the man's death. It's not clear when regulators and investigators were informed. 'Our deepest condolences go to the family and all those involved in his care,' the spokesperson said. 'Advancing the science of genetic medicine is incredibly challenging — especially in ultra-rare diseases like the limb-girdle subtypes. While we do everything possible to ensure patient safety, there is inherent risk in clinical trials, and we are grateful to the courageous patients and families who participate.' The news will likely be devastating to limb girdle patients and advocates. Their conditions have long been overlooked because they are generally regarded as less deadly than other types of muscular dystrophy, even though they are debilitating and some subtypes can significantly shorten life. They are also too rare to interest most for-profit drug developers. A few gene therapy companies are targeting individual subtypes, but Sarepta had promised to develop a suite of different treatments, building on the approval of its Duchenne therapy. The company said it still plans to file for approval later this year for one subtype, called LGMD 2E. But the three deaths could give regulators pause. Sarepta has said it will present data from its LGMD 2E therapy in October, at the World Muscle Society conference. The company also detailed on Wednesday plans to test, in a six-month trial, whether a new immune-modulating drug can mitigate the risk of liver toxicity from Elevidys. For now the drug remains off the market for all Duchenne patients who rely on wheelchairs — most patients over the age of 12. Advertisement
Reuters
18-07-2025
- Business
- Reuters
Sarepta shares slump after third patient death this year
July 18 (Reuters) - Shares of Sarepta Therapeutics (SRPT.O), opens new tab declined 17% in early trading on Friday after a patient who had received one of the company's experimental gene therapies died, deepening investor concern over the use of its treatments. Sarepta said a 51-year-old man enrolled in an early-stage trial of gene therapy, SRP-9004, for the treatment of limb-girdle muscular dystrophy (LGMD) died from acute liver failure last month. This is the third death this year for Sarepta after two teenage boys who had received Elevidys, a gene therapy approved to treat a rare condition called Duchenne muscular dystrophy, died of liver failure. Sarepta said the patient was the last to be dosed in that study. LGMD includes a group of genetic disorders that primarily weaken the hip and shoulder muscles. The news of the death was first reported by pharma website BioCentury on Thursday evening. On Wednesday, Sarepta announced a restructuring plan that included 500 job cuts and halting development of several gene therapies for limb-girdle muscular dystrophy. It said then the cuts were a financial decision and that it hopes to find partners for these programs. The company said it plans to host a call with investors later on Friday.
Reuters
18-07-2025
- Business
- Reuters
Sarepta shares slump on reports of third patient death this year
July 18 (Reuters) - Shares of Sarepta Therapeutics (SRPT.O), opens new tab declined 20% in early trading on Friday after media reports that a patient who had received one of its experimental gene therapies has died, deepening investor concern over the use of the company's treatments. This is the third death this year for Sarepta after two teenage boys who had received Elevidys, a gene therapy approved to treat a rare condition called Duchenne muscular dystrophy, died of liver failure. A 51-year-old man enrolled in an early-stage trial of Sarepta's gene therapy, SRP-9004, for the treatment of limb-girdle muscular dystrophy (LGMD) died from acute liver failure last month, according to several media reports. LGMD includes a group of genetic disorders that primarily weaken the hip and shoulder muscles. The news of the death was first reported by pharma website BioCentury on Thursday evening. On Wednesday, Sarepta announced a restructuring plan that included 500 job cuts and halting development of several gene therapies for limb-girdle muscular dystrophy.
Business Wire
14-05-2025
- Business
- Business Wire
BioCentury to Host Grand Rounds in Chicago: Where Translational Science Meets Investors and Dealmakers
CHICAGO--(BUSINESS WIRE)--Top decision-makers from across the biopharma ecosystem will convene in Chicago for the 2 nd BioCentury Grand Rounds – U.S., at the intersection of research, investment and drug development. Designed to drive practical progress in R&D, the conference connects academic innovators with biopharma executives and early-stage investors who are building the next wave of biotech breakthroughs. BioCentury is bringing together VCs, biopharma R&D decision-makers and academic innovators to discuss critical bottlenecks in the translation of scientific discoveries into the next wave of biotech breakthroughs. #BioCenturyGrandRounds Share Taking place June 4 – 6 at the Four Seasons Hotel Chicago, the program will address critical bottlenecks in three themes: Disease biology Therapeutic modalities Enabling technologies Featured sessions include a plenary with Marty Burke, Professor of Chemical Innovation at the University of Illinois Urbana-Champaign, and a fireside chat with Jonathon Sedgwick, SVP and Global Head of Discovery Research at AbbVie. In a special Horizon session, Luciana Borio, Venture Partner at ARCH Ventures, will lead a panel on the future of research funding. View the full agenda. Speakers include R&D, investment and academic leaders from AUTM, Biogen, Chan Zuckerberg Biohub Chicago, Daiichi Sankyo, Deerfield Management, Eli Lilly, Genmab, Johnson & Johnson Innovation, Merck & Co., Northwestern University, Novartis Venture Fund, Takeda, The University of Chicago and many other leaders in the industry. 'At this complex and politically fraught moment, bringing industry and academia together to enable the future of translational research is more critical than ever,' said Simone Fishburn, VP & Editor in Chief of BioCentury. 'BioCentury continues its 30-year tradition of fostering the future of medicine, bringing innovators and investors to delve into the science that can write the next stories in biotech.' 'We are hugely pleased the Chicago Biomedical Consortium and their Midwest colleagues have joined the Grand Rounds community,' said BioCentury Co-Founder and CEO David Flores. 'This important biomedical ecosystem will be a foundational link in the global network BioCentury is building to collectively identify solutions for translating great science into transformational cures.' Flores added: 'The Grand Rounds network, begun last year with our launch partners Vanderbilt University and Life Science Tennessee, will grow further with our first Grand Rounds - Europe meeting in the U.K. in September, led by Cambridge University Health Partners, Cambridge Innovation Capital and AstraZeneca.' The importance of Grand Rounds in Chicago is demonstrated by its collaborators. Insights Partner McKinsey & Company is joined by Founding Investors ARCH Venture Partners, Iaso Ventures, Lightspeed Venture Partners, MPM BioImpact, Third Rock Ventures and Xontogeny; and Sponsors Chan Zuckerberg Biohub Chicago, Collaborative Drug Discovery, inThought Research and Xentria. Regional Hosts advancing innovation in the Midwest include Chicago Biomedical Consortium, iBIO, Northwestern Memorial Healthcare, Northwestern University, P33, Portal Innovations, The University of Chicago, University of Illinois Chicago, and the University of Illinois Urbana-Champaign.
