Healio

Stargardt disease landscape sees emerging treatments

Please provide your email address to receive an email when new articles are posted on . Baruch D. Kuppermann, MD, PhD, highlights six companies developing Stargardt disease therapies. Two are oral ...
Healio

VIDEO: Oral Stargardt’s disease treatment focuses on reducing vitamin A dimerization

Please provide your email address to receive an email when new articles are posted on . CHICAGO — In this Healio Video Perspective from Eyecelerator@AAO, Michel Dahan, CEO of Alkeus Pharmaceuticals ...
Rutland Herald

Kubota Vision Inc. Signs Supply and Licensing Agreement with Laboratoires KÔL for Stargardt Disease Treatment Candidate Under Compassionate Use

Kubota Vision Inc. (“Kubota Vision”), a wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Minato-ku, Tokyo, Japan) announced signing of a Supply and Licensing Agreement with ...
Nasdaq

Belite Bio, Inc. to Present Key Findings on Stargardt Disease at 2025 ARVO Annual Meeting

Belite Bio, Inc., a clinical-stage biopharmaceutical company focusing on treatments for degenerative retinal diseases, announced that it will present two posters at the upcoming ARVO Annual Meeting ...
Seeking Alpha

Ocugen Announces Rare Pediatric Disease Designation Granted for OCU410ST—Modifier Gene Therapy for the Treatment of Stargardt Disease

MALVERN, Pa., May 27, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (OCGN) (Ocugen or the Company), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the United ...
Seeking Alpha

Belite Bio Announces Completion of DRAGON, a 2-Year, Phase 3 Trial of Oral Tinlarebant in the Treatment of Stargardt Disease

Tinlarebant has been granted Breakthrough Therapy, Fast Track, and Rare Pediatric Disease Designations in the U.S.; Orphan Drug Designation in the U.S., Europe, and Japan; and Pioneer Drug Designation ...
Nasdaq

Ocugen, Inc. Reports Progress on Clinical Trials and FDA Alignment for OCU410ST in Stargardt Disease, Highlights Positive Long-Term Data for OCU400

Ocugen reported clinical trial progress and financial results, highlighting advancements in gene therapies for retinal diseases and recent FDA alignments. OCU410ST is a therapy for Stargardt disease, ...

Belite Bio Receives Orphan Drug Status for Tinlarebant in Stargardt Disease in Switzerland

Tinlarebant is the first therapeutic candidate to demonstrate clinical efficacy in Stargardt disease, having met the primary endpoint, reductions ...
BioWorld

Series B brings Splicebio $135M for Stargardt disease asset

Three months after dosing the first patient with its dual vector gene therapy, Splicebio SL has closed a $135 million series B to fund the phase I/II trial of SB-007 in the treatment of Stargardt’s ...