Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

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 ...

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

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

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

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

"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

ELRIG, a not-for-profit, volunteer-led organisation dedicated to the global drug discovery community, today announced the ...

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

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 ...