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EB-201

Development stage: Preclinical
Lead developer: Abeona Therapeutics
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intramuscular

Overview

EB-201 is a preclinical gene therapy candidate developed by Abeona Therapeutics for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). It utilizes an AAV DJ vector to deliver the COL7A1 gene, aiming to correct the genetic defect responsible for the disease. Abeona Therapeutics licensed the underlying technology for EB-201 from Stanford University in 2016 as part of a broader collaboration for gene therapies for Epidermolysis Bullosa.

Other names: AAV DJ COL7A1

Targets

COL7A1 (Collagen type VII alpha 1 chain)

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