Gene therapy start-up GeneMedicine Inc. announced today thatit has received a $50,000 small business innovation (SBIR)grant from the National Institute of Arthritis andMusculoskeletal and Skin Diseases (NIAMD) to develop genemedicines for skin diseases.
The Phase I grant will cover research on the ability of aparticular DNA expression vector to actually direct theexpression of therapeutic products in normal and hyperplasticskin when injected directly into the epidermis. The vector, HK1,has already been shown to direct high-level tissue-specificexpression from keratinocytes in the epidermis.
It was developed by GeneMedicine founding scientist DennisRoop, a professor of cell biology and dermatology at BaylorCollege of Medicine in Houston.
In transgenic animal models, the HK1 vector (which containstranscription and translation control elements for the humanK1 gene) is able to "direct high levels of gene expressionspecifically in the proliferative basal cells, as well as thedifferentiated supra-basal cells of the epidermis," explainedEric Tomlinson, president and chief executive officer of theHouston company.
"We believe that by using the HK1 vector," he said, "we candeliver therapeutic products to treat a variety of inflammatoryand infectious diseases."
Privately held GeneMedicine was founded in 1992 with $8.5million in venture capital from investors D. Blech & Co. Inc.,Abingworth, Kleiner Perkins Caufield & Byers, New EnterpriseAssociates, Schroder Ventures, Ventures Medical, SED Venturesand Community Technology Fund.
Those investors are betting that GeneMedicine will developproprietary gene therapy technologies that enable therapeuticgenes to be directly administered to patients usingconventional methods.
"Our site-specific vector technologies will enable us to developthe first controllable gene therapeutics," Tomlinson said. "Wewill be able to target our therapeutic genes to specific celltypes for maximum effectiveness, ultimately directing whichcells express the genes."
-- Jennifer Van Brunt Senior Editor
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