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 ...

Precision DNA editing gene therapy achieves deep remissions in aggressive leukemia previously considered incurable.

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 ...

The Integrated Health Clinic at University Medical Center New Orleans with LSU Health New Orleans is one of only six teams ...

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

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

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

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

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.

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

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