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 ...

By Karen Roman Biopharmaceutical company Medera Inc. said it completed patient enrollment for Cohort B of its clinical trial ...

Results from MUSIC-HFpEF Phase 1/2a clinical trial show favorable safety profile and early clinical benefits with SRD-002 gene therapy The MUSIC-HFpEF trial is investigating SRD-002, a one-time gene ...

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to ...

Study supports favorable safety and tolerability profile and clinically meaningful functional and structural benefits in Stargardt disease ...

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 ...

The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source([1]) . The FDA approved three ...

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 ...

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology.

Experiments in mice reveal an early postnatal window of opportunity for the effective transfer of genes to blood-cell-producing haematopoietic stem cells by injecting mice with gene-carrying ...

There have been hundreds of human gene-therapy trials for many diseases, from hemophilia to cancer, in the past 18 years. But nearly all failed because of the difficulties of getting a working gene ...