BARCELONA, Spain I March 13, 2025 I SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations ...

SB-007 addresses the root cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations SB-007 is the first dual AAV gene therapy in clinical development for Stargardt ...

SB-007 addresses the root cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations SB-007 is the first dual AAV gene therapy in clinical development for ...

Stargardt disease is an inherited retinal disorder caused by mutations in the ABCA4 gene. It is the most common form of juvenile macular degeneration, leading to progressive central vision loss ...

AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company today announces the publication of two abstracts submitted to The Association for Research in Vision and Ophthalmology Annual ...

OCU410ST with a single subretinal injection has potential to treat Stargardt in all ABCA4-associated retinopathies and, in the fourth quarter, received orphan medicinal product designation from ...

for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD), and positive opinions from the EMA's ...

Stargardt disease occurs due to changes to a gene called ABCA4 that affects how your body uses Vitamin A. When fatty materials (lipofuscin) build up on the macula, ABCA4 is supposed to make a ...

SpliceBio’s lead in-house pipeline project is in Stargardt disease, an IRD with no approved therapies that is caused by changes in a gene called ABCA4 and results in fatty material building up ...

Meanwhile, OCU410ST targets Stargardt disease, a rare genetic condition stemming from ABCA4-related retinopathies. Current treatment options for GA are limited to two approved products in the U.S ...