Published On: Mon, Feb 17th, 2020

KIK-AS clinical trial : Angelman syndrome drug candidate GTX-102 to be evaluated by GeneTx Biotherapeutics

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GeneTx Biotherapeutics is set to start the KIK-AS clinical trial for evaluating GTX-102 for the treatment of Angelman syndrome (AS), a rare, neurogenetic disorder resulting from the loss-of-function of the maternally inherited allele of the UBE3A gene.

In this connection, the biotech startup has secured institutional review board (IRB) approval from Rush University Medical Center in , which will be the first center to enroll patients for the KIK-AS clinical study. More clinical sites are planned for Boston, Cincinnati, , Denver, New , and Ottawa, Canada, said GeneTx Biotherapeutics.

The KIK-AS trial, which expands to Knockdown of UBE3A-antisense in Kids with Angelman Syndrome, is a phase 1/2 clinical study.

Scott Stromatt – Chief Medical Officer of GeneTx Biotherapeutics said: “A tremendous amount of basic research over decades, and intense research and development over the last couple of years has now brought us to the cusp of testing a novel drug in individuals with Angelman syndrome.

“This is truly an exciting time for patients, families, and medicine.”

KIK-AS clinical trial : GTX-102 to be evaluated in Angelman syndrome by GeneTx Biotherapeutics

KIK-AS clinical trial : GTX-102 to be evaluated in Angelman syndrome by GeneTx Biotherapeutics. Photo courtesy of Free-Photos from Pixabay.

The goal of the phase 1/2 open-label, multiple-dose, dose-escalating trial is to assess the safety, tolerability, and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102 in pediatric patients having Angelman syndrome.

Nearly 20 patients of both the sexes aged ≥ 4 and ≤ 17 years of age with a genetically confirmed diagnosis of full maternal UBE3A gene deletion will be enrolled in the KIK-AS clinical trial.

GeneTx Biotherapeutics had partnered with Ultragenyx Pharmaceutical to develop GTX-102, which is an investigational antisense oligonucleotide designed to target and block expression of UBE3A-AS. According to GeneTx Biotherapeutics, nonclinical studies had shown that GTX-102 brought down the levels of UBE3A-AS and reactivated expression of the paternal UBE3A allele in neurons of the central nervous system.

Camille L. Bedrosian – Chief Medical Officer of Ultragenyx Pharmaceutical said: “The GeneTx team continues to make significant progress in advancing the GTX-102 program for patients with Angelman Syndrome, and we look forward to continuing our work with the team to move this program into the clinic.”

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