Ultragenyx Announces Acquisition of Foundation for Angelman Syndrome Subsidiary GeneTx

Anna Osborne
16 November 2022
Collaborations, Mergers & Acquisitions
Gene Therapy
Biopharmaceutical company Ultragenyx has announced it has exercised its option to acquire the biotherapeutic company GeneTx for $75 million, excluding future milestones and royalty payments. 

Phacilitate’s Anna Osborne was joined by Advanced Therapies Week 2023 confirmed speaker, and Foundation for Angelman Syndrome CEO, Allyson Berent, as well as foundation Chairperson John Schlueter, to discuss what the news will mean for patients of Angelman syndrome. 

This news comes after both companies – who entered into a partnership in August 2019 to develop GeneTx’s product candidate GTX-102 – provided a positive program update of GTX-102 for the treatment of Angelman syndrome.

GeneTx Biotherapeutics was originally set up as a subsidiary of the Foundation for Angelman Syndrome Therapeutics, dedicated to developing and commercializing GTX-102 for the rare neurological disorder Angelman syndrome, a disorder caused by the loss of function of the maternal allele of the UBE3A gene.

Interim data from 11 patients in the open-label, dose-escalating Phase I/II trial of GXT-102 in pediatric patients with Angelman syndrome, demonstrated meaningful improvements in clinical disease.

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Emil D. Kakkis, CEO and President of Ultragenyx explained that, “These data combined with the excellent science of Dr. Scott Dindot, the inventor of GTX-102, have given us the confidence to exercise our option to acquire GeneTx at an earlier timepoint so that we can take the lead on advancing GTX-102 into late-stage development for Angelman syndrome.”


Results of the clinical trial are measured using the AS Change Scale and the AS Severity Scale. These clinical assessments measure sleep, behavior, communication, gross motor and fine motor skills.

For example, observations on day 128 showed in one cohort, from the UK and Canadian arm of the trial, that 6, out of a potential 9, patients displayed a decrease in AS severity. 7 patients in the same cohort demonstrated an improvement from baseline level on the AS Change Scale.

Of the 14 patients to receive GTX-102 so far, 10 under the UK and Canadian arm and 4 under the U.S. arm of the trial, 13 patients have been on treatment for over 147 days – with no serious treatment-related adverse effects reported during this time. Cerebrospinal fluid (CSF) protein levels also remained stable with an absence of inflammation in all participants over the course of the study to date.

“I am convinced we are seeing evidence of a therapeutic effect with GTX-102 though it’s still early in the study. I am also encouraged by the improvements in quality of life that most of the families at my center are consistently reporting and believe that this could be a promising treatment for patients with Angelman syndrome,” said Erick Sell, Primary Investigator on the Study.

Patients with Angelman syndrome display developmental delays, loss of speech and motor function, and, epilepsy.

Source: Ultragenyx Press Release

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