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AAV-CNGA3 + AAV-CNGB3
- Development stage
- Unknown
- Lead developer
- MeiraGTx
- Modality
- Gene Therapies
- Administration
- Ophthalmic
01
Overview
AAV-CNGA3 and AAV-CNGB3 are investigational adeno-associated virus (AAV) vector-based gene therapies developed by MeiraGTx for the treatment of achromatopsia (ACHM). Achromatopsia is a rare inherited retinal disorder characterized by the loss of cone photoreceptor function, leading to legal blindness, extreme light sensitivity, and total color blindness. These therapies utilize AAV2/8 vectors to deliver functional copies of the CNGA3 or CNGB3 genes, respectively, to the cone receptors at the back of the eye via subretinal injection. The goal is to restore the function of cyclic nucleotide-gated (CNG) cation channels, which are essential for cone phototransduction. MeiraGTx is developing AAV-CNGA3 in collaboration with Janssen Pharmaceuticals, while AAV-CNGB3 is being developed independently by MeiraGTx.
- Other names
- AAV-CNGA3 and AAV-CNGB3AAV-CNGA-3 and AAV-CNGB3AAV-CNGA 3 and AAV-CNGB3AAV-CNGA3 + AAV-CNGB3-MeiraGTx UK II Ltd-achromatopsia
02
Targets
CNGB3 (Cyclic nucleotide-gated channel beta 3)CNGA3 (Cyclic nucleotide-gated channel subunit alpha 3)
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