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Neurologix, NPY gene therapy

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Tx name NPY gene therapy
Description In vivo NPY gene transfer allows for high level expression and functional efficacy limited to target region in the temporal lobe
Company Neurologix
Status Primate model testing
Overview NPY, a 36 aa, is the most abundant neuropeptide in the brain
Animals deficient in NPY show an epileptic phenotype
Overexpressing NPY in the hippocampus via either germline (transgenic) or via somatic cell gene transfer (NRGX, AAV-NPY) has consistent effects on suppressing seizures in both acute (kindling, drug induced) as well as chronic spontaneous epilepsy models
Acts via direct agonism on NPY Y2 receptors, which are upregulated in both animal models and human TLE
Y2 receptors largely presynaptic, inhibit glutamate release
Y2 receptors expressed widely and have pleiotropic actions so systemically administered agonists likely to have unwanted effects.
PK  
indications Treatment-resistant epilepsy
preclin data  
preclin tox  
Phase 1  
Phase 2  
Phase 3  
Adverse effects clinical experience with AAV gene transfer shows not a single gene therapy related adverse event after 35 surgeries
publications  
IP 3 key patents for AAV-Mediated Delivery of DNA to Cells of the Nervous System, Helper Functions for Recombinant Vector Production
Potential problems Difficult regulatory environment. Staff turnover at the FDA leading to a more conservative approach (data supporting our PD product was much less than for NPY/Epilepsy which has 16 supporting preclinical studies)
IRBs also often have a knee jerk reaction that gene therapy is both unproven and unsafe
Funding two programs in parallel with limited resources and staff
Epilepsy doesn’t engender as much as interest as neurodegenerative disorders from potential investors
Timeline Completion of preclinical (by 2H 2010); Phase I (2010-2011)
Financial Status  
Partnerships  
ETP Support grant

President
President
Created by President 07/23/2010 at 02:40pm
admin
admin
Edited by admin 09/2/2010 at 01:16pm

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