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

At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for the agency to ...

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

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

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

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

The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.

Fred Hutchinson researchers are testing a simple idea with surprisingly big potential: a special type of foam that makes gene therapy more efficient.

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

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

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

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 ...