Inherited retinal degenerations are a diverse group of genetic disorders that result in progressive vision loss. Advances in ...

Casgevy is the first gene therapy approved for sickle cell disease and transfusion-dependent beta-thalassemia in patients aged 2 and older.

When injected into mice with retinal disease, the special retinal blood cells integrated into the damaged tissue to ...

Successful Type B RDEP Meeting confirms FDA alignment on pivotal Phase 3 study designFDA indicates Opus Genetics may submit a BLA based on ...

Belite Bio, Inc (NASDAQ: BLTE) ('Belite Bio®” or the 'Company”), a clinical-stage drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have ...

Life Biosciences has received FDA clearance to begin a first-in-human Phase 1 clinical trial of ER-100, a gene therapy ...

Casgevy sickle cell gene therapy is now FDA-approved for children as young as two, opening access for 5,500 more U.S. kids.

Quantified preferences favored higher lesion growth rate reduction (10%→50% at 18 months) as the primary driver across all ...

Daniel Cressy, 23, has become the first person in the Gulf South to become functionally cured of sickle cell disease using ...

REGENXBIO Inc., a biotechnology company on a mission to improve lives through the curative potential of gene therapy, announced the first patient has been dosed in the phase IIb/III NAAVIGATE clinical ...

The retina, as a highly specialized and metabolically demanding neural tissue, is vulnerable to a wide spectrum of debilitating disorders — including ...

Biomedical engineers use iPSCs to grow specialized retinal endothelial cells, successfully regenerating damaged eye blood ...