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rAAV5-hCNGB3 Gene Therapy for Achromatopsia: Efficacy in a Dog Model
- Small Business Information
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APPLIED GENETIC TECHNOLOGIES CORPORATIONAPPLIED GENETIC TECHNOLOGIES CRP 12085 RESEARCH DR, STE 110 ALACHUA, FL 32615-0683View profile »Woman-Owned: NoMinority-Owned: NoHUBZone-Owned: No
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Fiscal Year: 2010Title: rAAV5-hCNGB3 Gene Therapy for Achromatopsia: Efficacy in a Dog ModelAgency: HHSContract: 1R43EY020069-01Award 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.Small Business Information at Submission:APPLIED GENETIC TECHNOLOGIES CORPORATION
APPLIED GENETIC TECHNOLOGIES CRP 11801 RESEARCH DR, SUITE D ALACHUA, FL 32615EIN/Tax ID: 159355371DUNS: N/ANumber of Employees: N/AWoman-Owned: NoMinority-Owned: NoHUBZone-Owned: No
Award Information
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Agency: Department of Health and Human Services |
Branch: N/A |
Award ID: 96144 |
Program Year/Program: 2010 / SBIR |
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Agency Tracking Number: EY020069 |
Solicitation Year: N/A |
Solicitation Topic Code: NEI |
Solicitation Number: N/A |