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ASGCT 2024 comes at pivotal time for gene and cell therapy sector

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A record 8,000 attendees are expected at this week’s 27th Annual Meeting of the American Society of Gene & Cell Therapy in Baltimore to discuss the opportunities and challenges facing the industry. Billed as the largest gathering of gene and cell therapy professionals, ASGCT 2024 starts tomorrow and will run through the end of the week, bringing together stakeholders from therapeutic developers and investors to patient advocates, policymakers and regulators.

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FDA’s accelerated approval of Sarepta DMD gene therapy comes under fire

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The FDA’s authorization of therapies that fail their efficacy endpoints casts doubt on the regulator’s accelerated approval pathway, according to an article from David Rind, chief medical officer of the drug pricing non-profit Institute for Clinical and Economic Review, published in the Journal of the American Medical Association. In his viewpoint piece, Rind specifically called out Sarepta Therapeutics’ gene therapy Elevidys, which won the FDA’s accelerated approval in June 2023—despite falling short of its primary efficacy endpoint in two studies.