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.

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

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 ...

Recently, GLP-1 (glucagon-like peptide-1) class obesity treatments such as ‘Wegovy’ and ‘Mounjaro’ have gained immense ...

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

"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.

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

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

Nanoscope Therapeutics has received Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations for ...