USA flag logo/image

An Official Website of the United States Government

rAAV5-hCNGB3 Gene Therapy for Achromatopsia: Efficacy in a Dog Model

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
96144
Program Year/Program:
2010 / SBIR
Agency Tracking Number:
EY020069
Solicitation Year:
N/A
Solicitation Topic Code:
NEI
Solicitation Number:
N/A
Small Business Information
APPLIED GENETIC TECHNOLOGIES CORPORATION
APPLIED GENETIC TECHNOLOGIES CRP 12085 RESEARCH DR, STE 110 ALACHUA, FL 32615-0683
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2010
Title: rAAV5-hCNGB3 Gene Therapy for Achromatopsia: Efficacy in a Dog Model
Agency: HHS
Contract: 1R43EY020069-01
Award Amount: $143,082.00
 

Abstract:

DESCRIPTION (provided by applicant): Complete achromatopsia is an inherited retinal disorder characterized by severely reduced visual acuity, nystagmus, severe photophobia, a small central scotoma, eccentric fixation, and complete loss of color discrimina tion. In 50% of patients with achromatopsia the disease is caused by mutations in the cyclic nucleotide gated channel beta subunit (CNGB3) gene. Preliminary studies indicate that gene therapy using a recombinant adeno-associated virus serotype 5 (rAAV5) ve ctor expressing a human CNGB3 gene can restore cone photoreceptor function in a dog model of achromatopsia caused by mutations in the CNGB3 gene. The objectives of the studies proposed in this application are to confirm and extend these findings using a rA AV5-CNGB3 vector produced using a commercially relevant manufacturing method. This will be accomplished by producing and purifying a rAAV5-hCNGB3 vector and evaluating the safety and efficacy of subretinal administration of a range of vector concentrations (1 x 1010, 1 x 1011, and 1 x 1012 vg/mL) of the rAAV5-CNGB3 vector in a dog model of achromatopsia caused by mutations in the CNGB3 gene. Results of these studies will be important for future advanced development of rAAV-CNGB3 gene therapy for evaluation in patients with CNGB3-related achromatopsia. PUBLIC HEALTH RELEVANCE: Complete achromatopsia is an inherited retinal disease characterized by severely reduced visual acuity and complete loss of color discrimination. In 50% of patients, the disease is caused by mutations in the CNGB3 gene. No treatment for achromatopsia is currently available. This project will evaluate a novel, CNGB3 gene therapy product for treatment of achromatopsia in a dog model.

Principal Investigator:

Jeffrey D. Chulay
3864622204
JCHULAY@AGTC.COM

Business Contact:

Jeffrey D. Chulay
jchulay@agtc.com
Small Business Information at Submission:

APPLIED GENETIC TECHNOLOGIES CORPORATION
APPLIED GENETIC TECHNOLOGIES CRP 11801 RESEARCH DR, SUITE D ALACHUA, FL 32615

EIN/Tax ID: 159355371
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No