Juvenile Macular Degeneration Companies

Latest Juvenile Macular Degeneration Companies Update

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  March 2023: Abeona Therapeutics Inc. announced three internally developed investigational preclinical gene therapy product candidates from its ophthalmology program. Abeona’s preclinical programs are investigating the use of novel adeno-associated virus (AAV) capsids in therapies for serious genetic eye diseases. In 2022, they evaluated the ability of their gene constructs and capsids to deliver and express the recombinant protein in target eye tissues and rescue mutant phenotypes in mouse disease models. Based on encouraging findings from these animal proof of concept experiments, they are looking forward to reporting new data from these programs at a scientific congress in the second quarter of 2023 and gaining alignment with the U.S. FDA on the clinical development plans for these programs. Abeona’s internal research and development team developed ABO-504, which is designed to efficiently reconstitute the full-length ABCA4 gene by implementing a dual AAV vector strategy using the Cre-LoxP recombinase system. 

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  June 2022: Kubota Vision Inc., a clinical-stage ophthalmology company and wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd., announced the completion of the phase 3 clinical trial investigating emixustat hydrochloride in patients with macular atrophy secondary to Stargardt disease.The primary objective of this study is to determine if emixustat reduces the rate of macular atrophy progression, in comparison to placebo, in subjects with Stargardt disease. Secondary objectives include assessing changes in visual function parameters such as BCVA (best-corrected visual acuity) letter score and reading speed. The database of this study will be locked in the third quarter 2022.

• Astellas Pharma Inc (Japan)


• Sanofi (France)


• Acucela Inc (Japan)


• Alkeus Pharma (US)


• Copernicus Therapeutics (US)


• Ophthotech Corporation (US)


• Bayer AG (Germany)


• Oxford BioMedica PLC (UK)


• Grupo Ferrer (Spain)