For more information about the publication and the groundbreaking work being conducted at AAVantgarde and TIGEM, please visit www.aavantgarde.com and https://www.tigem.it/.
About SpliceBioSpliceBio is a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes. The Company's lead program, SB-007, targets ...
Belite Bio is a clinical-stage biopharmaceutical drug development ... that have significant unmet medical needs, such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced ...
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Ocugen Advances with Phase 1/2 Trial for Stargardt Disease Treatmentmarking the final stage of the Phase 1 dose-escalation study. Ocugen’s GARDian trial aims to offer a beacon of hope for patients with Stargardt disease by potentially providing a viable and long ...
The three main types of juvenile macular degeneration (JMD) include Stargardt disease, X-linked retinoschisis, and Best disease. These result from gene mutations. Early treatment may help preserve ...
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, ...
AAVantgarde Bio is a clinical stage, International biotechnology company that has ... to deliver large genes to the Company’s two lead programs in Usher 1B and Stargardt disease, two inherited retinal ...
and the Pioneer Drug Designation in Japan for Stargardt disease, for which there are no approved treatments SAN DIEGO, Feb. 27, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE ...
Stargardt disease is an inherited retinal disorder caused ... About SpliceBio SpliceBio is a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by ...
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