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AGTC-001
- Development stage
- Discontinued
- Lead developer
- Syncona
- Modality
- Gene Therapies
- Administration
- Intravitreal
01
Overview
AGTC-001 is a gene therapy developed by Applied Genetic Technologies Corporation (AGTC) in collaboration with Biogen for the treatment of X-linked retinoschisis (XLRS). This adeno-associated virus (AAV)-based therapy was designed to deliver a functional copy of the RS1 gene to retinal cells, aiming to correct the genetic defect responsible for the disease. The program was terminated in December 2018 after Phase I/II clinical data showed a lack of efficacy.
- Other names
- Recombinant adeno associated virus vector expressing retinoschisinX-linked retinoschisis gene therapy-Beacon TherapeuticsXLRS gene therapy-Beacon Therapeutics
02
Targets
RS1 (Retinoschisin)
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