The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...

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 ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...

Results from MUSIC-HFpEF Phase 1/2a clinical trial show favorable safety profile and early clinical benefits with SRD-002 gene therapy BOSTON, May 19, 2025 (GLOBE NEWSWIRE) -- Medera Inc. (“Medera”), ...

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 ...

Researchers engineered lipid nanoparticles to deliver a full CFTR gene into human airway cells, restoring near-normal ...

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 ...

Bayer AG (BAYRY) announced an update on their ongoing clinical study. Study Overview The PROGRESS-GT LOPD trial is an early-stage clinical study sponsored by AskBio, with Bayer as a collaborator, to ...