Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

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 ...

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.

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) ...

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

February 25, 2026 marks one year since KJ, an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the world's first person to receive a personalized CRISPR-based gene ...

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 ...

What Is Itvisma, and Why Does It Matter? Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that the FDA has approved to treat spinal muscular atrophy (SMA) in adults, teens, and children 2 or ...

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