GeneTx Biotherapeutics launches to take on Angelman syndrome

GeneTx has a worldwide license agreement and research collaboration with Texas A&M, under which it hopes to develop and commercialize an antisense treatment for Angelman syndrome. (Dominik Moser)

The Foundation for Angelman Syndrome Therapeutics (FAST) has launched a new company, GeneTx Biotherapeutics, to develop an antisense drug for the treatment of Angelman syndrome, a rare genetic disease that affects the nervous system.

FAST Chairperson Paula Evans will take the helm as the initial CEO, while Allyson Berent, FAST’s chief scientific officer, will sign on as COO. Both are serving on an unpaid basis, the foundation said in a statement.

Launching GeneTx is a natural step for FAST, which has been working on technologies to treat the root cause and symptoms of Angelman syndrome, Berent said.


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The disorder is caused by a faulty UBE3A gene, inherited from a patient’s mother. Its symptoms include delayed development, intellectual disability, severe speech impairment and problems with movement and balance.

“Our aggressive research agenda and collaboration with Texas A&M AgriLife Research has identified and characterized a promising therapeutic to address this devastating disorder,” she said. “Angelman syndrome has such a significant unmet need, for which there are no approved therapeutics, and the landscape for bringing meaningful treatments to the community is significantly expanding.”

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The promising therapeutic, GTX-101 is in a class of medication called antisense drugs, which silence genes responsible for genetic disorders by binding to the messenger RNA produced by that gene.

GeneTx isn’t the only one looking for a treatment—Ovid, which went public last May, is using part of its IPO proceeds to drive its lead candidate through clinical trials for Angelman syndrome and fragile X syndrome.

Ovid acquired the GABA agonist, OV101, from Lundbeck, which took it into a phase 3 trial for insomnia with partner Merck before ultimately dumping it in 2007. The drug is currently in a phase 2, proof-of-concept trial in adults with Angelman syndrome, as well as a phase 1 study in adolescents with either Angelman or fragile X.

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