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Program History
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 AS. In late 2017, the Dindot laboratory identified an investigational 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 program moved forward in the most expeditious, safe, and detailed manner possible, with the primary goal of moving 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.
Key Researchers:
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Timeline:
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 AS.
2016
FAST created a for-profit company called GeneTx Biotherapeutics LLC specifically for one purpose: to accelerate the development of an investigational antisense oligonucleotide drug for the potential treatment of AS.
2020
The clinical trial was initiated, making this FAST-backed program the first potential disease-modifying therapy in human clinical trials for Angelman syndrome.
2022
FAST announced that the company Ultragenyx Pharmaceuticals had exercised its option and closed on its acquisition of GeneTx Biotherapeutics LLC, a biotechnology company founded by FAST.