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AAV-Ntf3
- Development stage
- Preclinical
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intracochlear, Intramuscular, Intrathecal
01
Overview
AAV-Ntf3 is an experimental gene therapy that utilizes an adeno-associated viral (AAV) vector to deliver the neurotrophin-3 (NTF3) gene. Neurotrophin-3 is a critical neurotrophic factor that supports the survival, development, and differentiation of existing neurons and promotes the growth of new neurons and synapses. The therapy is being investigated in preclinical models for several conditions, most notably hearing loss and deafness, where it aims to preserve spiral ganglion neurons and encourage the regrowth of peripheral auditory fibers to improve the efficacy of cochlear implants. It has also shown potential in research for multiple sclerosis and peripheral neuropathy. Currently, AAV-Ntf3 is in the early research and preclinical stages, primarily within academic settings, and has not been associated with a specific commercial developer.
- Other names
- AAV-Neurotrophin-3AAV-Neurotrophin3AAV-Neurotrophin 3AAV-NT3AAV-NT-3AAV-NT 3
02
Targets
NTRK3 (Tropomyosin receptor kinase C)NGFR (p75NTR)
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