The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
Fifteen-year-old Jayden Wilsey of Falmouth is the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease, following approval of the therapy in 2024 by the U.S ...
Researchers developed a gene therapy for LAMA2-related muscular dystrophy that restored muscle and nerve function in mice.
Biopharmaceutical company Medera Inc. said it completed patient enrollment for Cohort B of its clinical trial evaluating SRD-002 gene therapy for heart failure with preserved ejection fraction (HFpEF) ...
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 ...
Showcased 7 presentations at ISSCR 2025, including a featured oral presentation on first-in-human gene therapy for heart failure, powered by their proprietary human mini-Heart technology. FDA-backed ...
Gene Therapy Meets Regulatory Reality: The FDA–uniQure Clash Over a Promising Huntington’s Treatment
The FDA’s dispute with biotech firm uniQure over Huntington’s gene therapy highlights growing tensions between innovation, ...
UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.
Cure Rare Disease (CRD), a non-profit biotechnology organization, today announced a landmark partnership with the LGMD2L Foundation. This collaboration is backed by a generous commitment of $7.65 ...
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