No more on again, off again?: RheoGene licenses gene switch to Chemicon

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NORRISTOWN, Penn.—February 9, 2006—RheoGene announced it has granted a commercial license for its RheoSwitch Therapeutic System to Ichor Medical Systems, based in San Diego, which will combine the system with its proprietary TriGrid Delivery System to develop a gene therapy product for multiple sclerosis. Ichor also has an option for RheoSwitch on two other indications.
ATLANTA–A new licensing agreement between RheoGene Inc., and Chemicon International Inc., should help bring new research tools to stem cell scientists in 2006.
RheoGene is licensing out its RheoSwitch technology, which turns gene function on and off, to Chemicon under a semi-exclusive agreement. Financial details were not released.

Dennis Harris, vice president of research and development at Chemicon, says the company is prioritizing potential new products that use RheoSwitch. The simplest items, he says, are generic kits for cloning genes of interest. "People would essentially use that to create cell lines with a gene that's either completely on or completely off," he says. Such kits, he adds, could be expanded "to products that would contain genes that we know would be of generic interest to people." Chemicon has already identified genes involved in regulating stem cell differentiation pathways.
RheoSwitch, says Thomas Tillett, president and CEO of RheoGene, is a molecular switch that allows precise regulation of genes. A protein receptor binds next to a target gene and is then "turned on with a small molecule," referred to as an activator drug or ligand.
RheoSwitch exists in versions for research and therapeutics, with compounds and switches varying slightly because use in humans requires "an even greater degree of control," says Tillett. RheoGene's library has thousands of compounds that can be used with hundreds of receptors, and Tillett calls RheoSwitch "the tightest" gene regulation system available, citing control as "a critical benefit that both researchers and patients need."
Switch performance is one reason Chemicon chose RheoSwitch, says Harris. Chemicon, he says, was "very impressed with the quality of the switch" after evaluating its use at sister company Celliance, which, with Chemicon, is part of Serologicals Corp. Another Serologicals company, Upstate, signed a licensing agreement in 2004 for using RheoSwitch with RNA interference and antibody research kits.
Chemicon, says Harris, will promote new products with RheoSwitch to its traditional markets, which are about 70 percent academic labs and 30 percent pharmaceutical companies. Harris calls Chemicon a leader in stem cell work, noting "we are the first commercial licensee of the [Wisconsin Alumni Research Foundation] patent families." That agreement, covering use of WARF's complete patent portfolio of stem cell technologies, was announced in July 2005.
RheoGene has licensed RheoSwitch to numerous other companies and noncommercial research institutions, but only Chemicon and New England Biolabs may sell the ligand. RheoSwitch uses vary broadly and include research with transgenic animals and production of therapeutic proteins.
Licensing RheoSwitch, says Tillett, has benefited RheoGene by bringing in short-term revenues that support its research program. Licensing also helped "establish the RheoSwitch system as the best gene regulation technology available today," he says, raising interest in the switch among licensees developing advanced therapies. The small molecules used to trigger the switch, says Tillett, "are not pharmacologically active. So what's critical here is that in these kinds of applications, the competitors' systems are using compounds that are pharmacologically active."
The University of Pittsburgh Medical Center is a strategic investor in RheoGene, and the company enjoys "a wide array of collaborations that we have going in Pittsburgh that really help to synergize the relationship that we have with our strategic investor," says Tillett.
Chemicon's stem cell work complements RheoGene's therapeutic programs, which also involve stem cells, Tillett says. RheoGene currently has six candidates in preclinical trials; five use RheoSwitch. RheoGene's plans for 2006 include introducing RheoPlex, for regulating multiple genes within the same cell, enabling greater understanding of gene pathways rather than only single genes.

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