USA flag logo/image

An Official Website of the United States Government

Preclinical Development of AAV-Galanin for Epilepsy

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
89431
Program Year/Program:
2008 / SBIR
Agency Tracking Number:
NS063611
Solicitation Year:
N/A
Solicitation Topic Code:
N/A
Solicitation Number:
N/A
Small Business Information
ASKLEPIOS BIOPHARMACEUTICAL, INC.
ASKLEPIOS BIOPHARMACEUTICAL, INC. 45 North Chatham Parkway CHAPEL HILL, NC 27517
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2008
Title: Preclinical Development of AAV-Galanin for Epilepsy
Agency: HHS
Contract: 1R43NS063611-01
Award Amount: $282,210.00
 

Abstract:

DESCRIPTION (provided by applicant): This proposal seeks to leverage proprietary technical advances in Adeno-Associated Virus (AAV) vector mediated gene therapy to develop an AAV based Galanin gene delivery for the treatment of epilepsy. Asklepios Biopharm aceutical, Inc. (Askbio) has obtained intellectual property rights to develop and commercialize an anti-epileptic biologic containing three key technologies: 1.) a gene cassette directing secretion of galanin, 2.) use of a self complementary genome, and 3. ) the specifically evolved chimeric AAV2.5 capsid. The incorporation of these technologies into a viable alternative gene therapy for epilepsy is a novel component of this proposal, and on that offers a great deal of hope to the estimated 350-700K patients in the U.S. for whom anti-epileptic drugs fail. The feasibility experiments detailed in this Phase I proposal will determine an efficacious dose range for the chimeric AAV2.5 containing a self-complementary FIB-Galanin transgene cassette (dsAAV2.5 FIB-GAL ), as well as examine the efficacy and safety of intracranial administration of dsAAV2.5 FIB-GAL to treat Mesiotemporal lobe epilepsy (MTLE) using two different mouse models of limbic seizure, focal electrical kindling and peripheral kainic acid administra tion. We fully expect the results of the proposed studies to support advancing to a Phase II study consisting of definitive preclinical studies, in both rodent and large animal models, as well as pivotal safety and toxicology studies in support of an IND s ubmission. The proposed initial clinical study will be undertaken in subjects with MTLE, who are already candidates for surgical resection, and will employ a study design involving gene delivery prior to scheduled resection of epileptogenic/dsAAV2.5 FIB-GA L infused tissue. PUBLIC HEALTH RELEVANCE: Asklepios Biopharmaceutical, Inc. is developing a therapeutic to treat people with Mesiotemporal lobe epilepsy (MTLE). Current treatment strategies are limited to anti-epileptogenic drugs, most of which are severa l decades old. For the 350-700K patients in whom drug therapies fail to resolve seizures, surgical resection offers a potential resolution. The proposed therapeutic, an AAV-mediated delivery of Galanin, is designed to provide an alternative therapy to meet this longstanding medical need.

Principal Investigator:

Business Contact:


resadmin@askbio.com
Small Business Information at Submission:

ASKLEPIOS BIOPHARMACEUTICAL, INC.
ASKLEPIOS BIOPHARMACEUTICAL, INC. 45 North Chatham Parkway CHAPEL HILL, NC 27517

EIN/Tax ID: 200267834
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No