FDA Meeting Gives Window Into Gene Therapy Field’s Angst

FDA reassures rare disease advocates that ‘being flexible’ is plan for gene therapy

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and speed of new drug approvals.

FDA meeting gives window into gene therapy field’s angst

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a regulatory forum Thursday.

FDA Grants Sarepta Therapeutics Landmark Platform Technology Designation for Gene Therapy Vector

Sarepta Therapeutics has achieved a significant milestone, receiving the U.S. FDA’s Platform Technology Designation for its rAAVrh74 viral vector. This designation, a first of its kind publicly documented for a gene therapy vector, is expected to accelerate the development and review of multiple gene therapies, particularly for rare diseases like Limb Girdle Muscular Dystrophy. FDA Recognizes Sarepta’s Gene Therapy Platform On June 4, 2025, Sare…

US leadership emphasizes support for rare disease drug development

CureDuchenne is pleased that one of the main themes at the recent US FDA Cell and Gene Therapy Roundtable was preserving incentives and leveraging regulatory flexibility and innovation to remove obstacles for rare disease treatment.Read more hereThe post US leadership emphasizes support for rare disease drug development appeared first on CureDuchenne.

Cell and Gene Therapy Leaders Tell FDA: “Believe in American Solutions”

Leaders from the world of cell and gene therapy, including molecular geneticists, immunotherapists, physicians, nonprofit directors, and patient advocates, shared their personal stories and policy recommendations with leaders of the FDA in an extraordinary roundtable.  The roundtable was conducted in front of the FDA’s recently appointed top officials—director Martin A. Makary, MD, and Vinayak (Vinay) Kashyap Prasad, MD, director of the Center f…

FDA grants orphan drug status to DMD cell therapy for heart issues

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart’s experimental stem cell therapy for heart problems in Duchenne muscular dystrophy (DMD), the company announced in an email sent to Muscular Dystrophy News Today. This FDA designation aims to accelerate the development of treatments for rare diseases, like DMD, that affect fewer than 200,000 individuals in the U.S. It provides certain benefits, incl…