UBE3a Protein

UBE3a Protein


About GeneTx

GeneTx Biotherapeutics was setup as a subsidiary of the Foundation for Angelman Syndrome Therapeutics for the development and commercialization of an investigational antisense drug, GTX-102, for the treatment of Angelman syndrome (AS).

About Angelman Syndrome

Angelman syndrome (AS) is a rare genetic disorder that affects approximately one in 15,000 live births. AS is caused by a loss of function of the maternally inherited UBE3A gene. Symptoms of AS include developmental delay, impaired motor function, loss of speech, and epilepsy. GeneTx Biotherapeutics’ GTX-102 is designed to compensate for the deficiencies that result from the underlying genetic cause of the disorder. GeneTx entered into a worldwide license agreement with The Texas A&M University System and a research collaboration agreement with Texas A&M AgriLife Research, under which GeneTx hopes to further develop and commercialize this novel antisense oligonucleotide that could potentially serve as a targeted therapy for patients with the disorder.

Our Advisors

Two research pioneers joined GeneTx as scientific advisors. Arthur L. Beaudet, M.D., is a pioneer in Angelman syndrome research. His lab and the Wagstaff lab independently identified the Angelman gene as UBE3A in 1997. Dr. Beaudet was inducted into the Institute of Medicine, the Society of Scholars and the National Academy of Sciences. Dr. Beaudet is the Henry and Emma Meyer chair and professor, department of Molecular and Human Genetics, Baylor College of Medicine.

“Compared to 30 other pediatric neurological disorders, I would make the case that Angelman syndrome holds one of the single most optimistic possibilities for a cure,” Beaudet said. “Antisense oligonucleotide technology offers tremendous promise to treat the symptoms of Angelman syndrome.”

James M. Wilson, M.D., Ph.D., has been working for three decades to develop effective strategies to treat and cure genetic diseases. Dr. Wilson was recognized as one of 12 leading pioneers in cell and gene research with the Pioneer Award given by Human Gene Therapy, a peer-reviewed journal of the medical research community. Wilson directs the Orphan Disease Center (ODC) in the Perelman School of Medicine at the University of Pennsylvania, which focuses on making rare disease research a priority.

“Rare disease patient organizations are becoming increasingly integral to the development of novel therapeutics and gene therapies,” Dr. Wilson said. “The launch of GeneTx demonstrates a keen vision by FAST that I believe can be emulated by other rare disease nonprofits in advancing their research agendas.”