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AAV2-shmTOR
- Development stage
- Unknown
- Lead developer
- Kubota Vision
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies, Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
- Administration
- Intravitreal, Subretinal
01
Overview
**AAV2-shmTOR** is an investigational gene therapy designed to target and silence the mammalian target of rapamycin (mTOR) pathway in retinal cells. It utilizes an adeno-associated virus serotype 2 (AAV2) vector to deliver a short hairpin RNA (shRNA) specifically targeting mTOR mRNA, leading to its knockdown via RNA interference. The primary therapeutic goal is neuroprotection and preservation of vision in degenerative retinal diseases, such as geographic atrophy secondary to age-related macular degeneration (AMD). By inhibiting mTOR signaling, the therapy aims to reduce cell death and slow disease progression.
- Other names
- AAV2-shmTORAAV-2-shmTORAAV 2-shmTOR
02
Targets
mTOR (Mammalian target of rapamycin kinase)
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