Gendyne Therapeutics

The Company:

Ithaca, NY based Gendyne Therapeutics is an early-stage biopharmaceutical company developing nucleic acid delivery systems to serve defined niches in both the cell transfection and the rapidly emerging RNA Interference (RNAi) therapeutic markets. Founded by nanobiotechnology classmates at Cornell University John P. Reilly (MS) and Adam T. Hammond (PhD) the company is focused on the development of novel non-viral hybrid encapsulation platforms for clinically relevant RNAi delivery. Rights to their silica and polymer nanoparticle technology have been attained from the Cornell Center for Technology, Enterprise, and Commercialization.

Business Model:

The company is working to license its lead product, NanoFect® to top-tier life science reagent companies both in the US and Europe. Using its proprietary technology (Polycomp™) and drug delivery expertise, Gendyne intends to enter into development and commercialization partnerships with RNAi drug discovery companies. A fee for service/contract manufacturing model during pre-clinical feasibility studies will evolve into its own R&D efforts on RNAi approaches to CNS/neurodegenerative diseases. “In order to take advantage of the inherent efficacy and safety benefits of an RNAi based therapy, challenges of cellular insertion and degradation must be overcome. The RNAi must not only enter cells to access its eventual targets but also evade natural cellular degradation mechanisms” says Hammond.

Technology:

Both NanoFect and Polycomp were invented by Dr. Dan Luo while a post-doctoral associate in the School of Chemical Engineering at Cornell. The simple advance in the NanoFect^TM procedure is to create dense complexes with very high DNA/siRNA concentrations. These dense transfection complexes sediment much more rapidly than other standard transfection products, thereby increasing the transfection efficiency by delivering more DNA/siRNA more rapidly.

For Polycomp™, surface modified PLA/PLGA nanospheres targeting specific receptors maintain a significant advantage over other vector delivery systems mainly because of their ease of large-scale production, non-immunogenicity, and relative safety.

Prospects:

Gendyne has received funding from the New York State Center for Advanced Technology, Cornell University (Center for Life Science Enterprise) and private investors. Several NSF/NIH SBIR grants are pending. It is seeking to raise $1.5M in order to complete its database of transfected cells with NanoFect® and to expand its in vivo RNAi targeting studies in animal models.