A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

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

Clinical and commercial quality controls are eased as the FDA looks to expedite therapy approvals in areas of unmet need.

Universal gene‑edited CAR‑T therapies turn donor T cells into off‑the‑shelf cancer treatments, cutting delays and costs while ...

Scientists from UC Davis Center for Surgical Bioengineering, the MIND Institute and UC Berkeley’s Murthy Lab are developing ...

OCU410 controlled lesion growth 46% better than placebo in dAMD-related GA.

GEN’s first virtual event of 2026—we present a group of outstanding researchers and thought-leaders to discuss the latest advances and challenges in delivering genetic therapies.

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

Join the ELRIG conference to hear from experts on cell and gene therapy. Discover advancements shaping the future of medicine.

Neurodegenerative diseases (NDDs) represent a profound challenge to global health, characterized by progressive neuronal dysfunction and loss, leading to ...

Techne, bioMerieux, Roche, Lonza Group, Miltenyi Biotec India and More Cell and Gene Therapy Manufacturing QC Market Cell and Gene Therapy Manufacturing QC Market Dublin, Jan. 23, 2026 (GLOBE NEWSWIRE ...