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 ...
A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
Precision DNA editing gene therapy achieves deep remissions in aggressive leukemia previously considered incurable.
GlobalData on MSN
FDA increases manufacturing flexibility for cell and gene therapies
Clinical and commercial quality controls are eased as the FDA looks to expedite therapy approvals in areas of unmet need.
In November, six months after KJ’s debut, top Food and Drug Administration officials Marty Makary and Vinay Prasad published ...
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.
The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.
"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...
Clinical Trials Arena on MSN
Genespire to take rare disease gene therapy to clinic in 2026
GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.
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