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FAST Ventures: GeneTx Biotherapeutics

Beginning in early 2012, FAST began funding Scott Dindot’s laboratory at Texas A&M University to understand the mechanism by which the UBE3A-antisense transcript (UBE3A-AS) silences the paternal UBE3A allele, why it is only silenced in neurons, and how that process may be leveraged as a potential therapy for Angelman syndrome (AS).

In late 2017, the Dindot laboratory identified an investigational antisense oligonucleotide (ASO) that, through preliminary studies, was shown to interfere with the imprinting mechanism of the paternal UBE3A allele in neurons, and unsilenced the gene.

FAST wanted to ensure that the development of this treatment moved forward in the most expeditious, safe, and detailed manner possible, with the primary goal of moving this treatment into a human clinical trial if preclinical studies supported further investigation. To do this, FAST formed GeneTx Biotherapeutics LLC (GeneTx), a for-profit limited liability company, singularly focused to develop an investigational ASO for potential future use in a Phase 1/2 clinical trial for individuals living with AS.

The aim here was for GeneTx to advance this ASO program to bring this to a first-in-human clinical trial and help shepherd it through these early stages of development with the hope it would eventually be advanced by an experienced industry partner based on clinical trial results. It was also thought that if this approach to development and partnering were successful, this arrangement might generate revenue back to the patient advocacy group that could then be used to expand the scope of AS research and outreach funded by FAST to further advance numerous other priority research programs.

Very quickly, GeneTx attracted investment by Ultragenyx Pharmaceuticals, a company involved in the research and development of novel products for the treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and a high unmet medical need.

Together, GeneTx and Ultragenyx pioneered a Phase 1/2 clinical study of investigational GTX-102 in the U.S, the United Kingdom and Canada—the ASO in human clinical trials for AS. In 2022, FAST announced that the company, Ultragenyx Pharmaceuticals, had exercised its option and closed on its acquisition of GeneTx. At that point, GeneTx was folded into Ultragenyx, which assumed complete control of the GTX-102 program, including the ongoing clinical trial. For more detail on the acquisition, see here.

Angelman Syndrome News