Development of Novel Alternative Serotype Adenoviral Vectors for Gene Delivery in

Award Information
Agency:
Department of Health and Human Services
Branch
n/a
Amount:
$138,699.00
Award Year:
2009
Program:
SBIR
Phase:
Phase I
Contract:
1R43DC010291-01
Award Id:
93660
Agency Tracking Number:
DC010291
Solicitation Year:
n/a
Solicitation Topic Code:
n/a
Solicitation Number:
n/a
Small Business Information
GENVEC, INC., 65 W WATKINS MILL RD, GAITHERSBURG, MD, 20878
Hubzone Owned:
N
Minority Owned:
N
Woman Owned:
N
Duns:
806729547
Principal Investigator:
DOUGLAS BROUGH
(240) 632-5540
DBROUGH@GENVEC.COM
Business Contact:
WEST
() -
dbrough@genvec.com
Research Institute:
n/a
Abstract
DESCRIPTION (provided by applicant): Myriad studies have examined the potential of gene therapy in the inner ear. For the most part they have focused on adenoviral vectors and delivery into the cochlea. Most studies have emphasized looking at expression o f marker genes driven by a CMV promoter and have used first generation adenoviral constructs based on the adenovirus serotype Ad5. The main advantages of the adenoviral delivery system are efficient gene delivery to most target tissues, the ease of synthes is and the ability to redirect adenoviral vectors to a variety of cell types and the advanced use of the vector system in the clinic. The main disadvantages of the current adenoviral delivery system is that gene delivery is not targeted for entry into spec ific cells in the inner ear and the Ad5 based vectors are strongly immunologically recognized in the human population. The proposed studies will test the hypothesis that adenoviral vectors based on non- Ad5 serotypes incorporating cell type specific promot ers can be used to efficiently and specifically deliver proteins to the sensory cells and supporting cells of the inner ear. These new adenovirus vectors will overcome the limitations to the current Ad5 based system. During SBIR phase I we will first chara cterize and select novel adenoviral vector platforms that efficiently deliver genes to the inner ear and second test preferred vectors for selective expression in sensory cells and in supporting cells using specific promoter control elements. With these re sults in hand and the ever increasing genomic findings molecular therapeutics can be designed and tested that may treat inner ear disease. PUBLIC HEALTH RELEVANCE: This SBIR Phase 1 proposal tests the hypothesis that adenoviral vectors based on non - Ad5 serotypes incorporating cell type specific promoters can be used to efficiently and specifically deliver proteins to the sensory cells and supporting cells of the inner ear. These new adenovirus vectors will overcome the limitations to the current Ad 5 based system. We will first characterize and select novel adenoviral vector platforms that efficiently deliver genes to the inner ear and second test preferred vectors for selective expression in sensory cells and in supporting cells using specific promo ter control elements.

* information listed above is at the time of submission.

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