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AAV9.hCLN2
- Development stage
- Unknown
- Lead developer
- Tern Therapeutics
- Modality
- Gene Therapies
- Administration
- Intracisternal, Subretinal
01
Overview
AAV9.hCLN2 is an investigational **AAV9-based in vivo gene therapy** designed to deliver a functional human **CLN2** transgene encoding **tripeptidyl peptidase 1** to restore lysosomal enzyme activity in CLN2 disease. Publicly described CNS and ocular programs indicate that this construct, or closely specified versions such as **AAV9.CB7.hCLN2**, uses an adeno-associated virus serotype 9 vector to drive sustained production of TPP1 after local administration, with the therapeutic goal of reducing lysosomal storage material accumulation and slowing neurodegeneration and retinal degeneration in **neuronal ceroid lipofuscinosis type 2**. The CNS-directed program has been identified as **RGX-181** and the ocular program as **RGX-381** or **TTX-381** for ocular manifestations of CLN2 disease.
- Brand names
- RGX-181RGX181RGX 181
- Other names
- human CLN2 gene therapyTPP1 gene therapyTPP-1 gene therapyTPP 1 gene therapy
02
Targets
TPP1 (Tripeptidyl peptidase 1)
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