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Asklepios, galanin gene therapy

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Philanthropic Funding Provided by: Epilepsy Therapy Project

Tx name Galanin
Description Gene therapy
Company Asklepios
Status Pre-clinical
Overview Infusion of galanin into the CNS significantly attenuates seizure activity in a number of animal models.
Galanin knockout mice exhibit a significantly greater seizure sensitivity while galanin overexpressing mice exhibit a reduced seizure sensitivity. Galanin exhibits neuroprotective actions. AAV delivery method Efficiently mediates stable long term gene expression in the CNS. Excellent safety profile (Pre-clinical & Clinical.  Potential seizure reduction without loss of cognitive function; Neuro-protective & anticonvulsant benefits with single treatment
PK N/A
indications Mesial temporal lobe epilepsy
preclin data  
preclin tox Small animal POC - Completed
Phase I SBIR Awarded - 9/2008
Top level toxicity - Completed
Small animal model dose response - Completed
Preliminary Primate Studies - Completed
Pre pre-IND Interaction ~ Q1/2010
Phase II SBIR Submission ~ Q2/2010
Phase 1 Phase I Clinical trial at Yale & Emory, $2.5M required, 2012 Initiation. Demonstrate safety of Galanin gene delivery. This will establish Dosing of Galanin gene delivery, and demonstrate the initial efficacy of Galanin gene delivery.
Phase 2  
Phase 3  
Adverse effects  
publications  
IP Biological Nano Particle (BNP) is a platform vector technology that has been developed over the last 35 years based on parvovirus research and engineering by Dr. Jude Samulski at the University of North Carolina at Chapel Hill. Over $35 M has been invested in BNP development to date. The technology was exclusively licensed to AskBio in 2003.  
Mature IP Portfolio - >22 issued patents, >50 active applications. IP licensed by GSK, Merck, Medtronic & others. Unique IP improves upon efficiency and safety and has been validated in the clinic
Multiple POC and clinical studies support CNS gene delivery system. Epilepsy program builds on IP for anti-epileptic neuropeptide gene expression.
Potential problems Rodent models - early risk mitigation: Preliminary behavioral and cognitive testing; Optimization of expression cassette; Top level toxicity
Primate studies: Proof of principle in cortically robust species; Accurate test of Clinical Approach
Comparison of Galanin Delivery and Resection procedures
Timeline  
Financial Status  
Partnerships  
ETP Support Grant Spring 2010

President
President
Created by President 07/23/2010 at 02:38pm
admin
admin
Edited by admin 09/2/2010 at 01:15pm

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