MALVERN, Pa., April 27, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (OCGN) (Ocugen or the Company), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell ...
Ocugen, Inc. announced that the U.S. FDA has granted Rare Pediatric Disease Designation to its treatment OCU410ST for ABCA4-associated retinopathies, including Stargardt disease. This follows prior ...
Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon ...
SpliceBio, a clinical-stage genetic medicines company pioneering protein splicing to address diseases caused by mutations in large genes, today announced that the first patient has been dosed in the ...
Among the forms of monogenic macular degeneration, the most common is Stargardt disease. This disease is caused by mutations in the ATP-binding cassette transporter alpha 4 subunit (ABCA4) gene, ...
Please provide your email address to receive an email when new articles are posted on . A phase 1/2 trial will assess safety, tolerability and preliminary efficacy for VG801. The FDA also selected ...
A collaboration between scientists at the Institute of Molecular and Clinical Ophthalmology Basel (IOB) and Beam Therapeutics has resulted in a new base editing therapy for Stargardt disease, the most ...
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