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AAV-ABCA4
- Development stage
- Preclinical
- Lead developer
- MeiraGTx
- Modality
- Gene Therapies
- Administration
- Ophthalmic
01
Overview
AAV-ABCA4 is an investigational gene therapy being developed by MeiraGTx for the treatment of Stargardt's disease, the most common form of inherited juvenile macular degeneration. The therapy utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the ABCA4 gene directly to photoreceptor cells in the retina. Stargardt's disease is caused by mutations in the ABCA4 gene, which encodes a flippase transporter protein responsible for clearing vitamin A derivatives from photoreceptors. Defective ABCA4 function leads to the accumulation of toxic bisretinoids in the retinal pigment epithelium, causing progressive vision loss. AAV-ABCA4 aims to restore or augment the transporter's function, thereby addressing the root genetic cause of the disease and preventing further retinal degeneration. The program is currently in preclinical development.
- Other names
- AAV-ABCA4-MeiraGTx-ABCA4-gene therapy-Stargardt's diseaseMeiraGTx ABCA4 gene therapy
02
Targets
ABCA4
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