First participant to be dosed in Rett gene therapy TSHA-102 trial
12 women expected to be enrolled in Phase 1/2 REVEAL study
The Phase 1/2 REVEAL study, which is testing the investigational gene therapy TSHA-102 in women with Rett syndrome, is expected to dose its first participant in the next month or two, according to Taysha Gene Therapies, its developer.
“Screening is completed, and dosing is now scheduled for our first potential patient in the adult Rett syndrome study,” Sean Nolan, Taysha’s chairman and CEO, said in a company press release.
Initial trial data, primarily concerning the therapy’s safety, are expected by the end of June, according to Taysha. The company plans to provide updates on the clinical program every three months.
The REVEAL trial (NCT05606614) is expected to enroll about 12 women with Rett syndrome. Recruitment began late last year in Montréal, Canada.
All participants will be given a low or high dose of TSHA-102, though the timing of the treatment will be delayed in some patients. The main goals are to evaluate safety and the treatment’s effect on patients’ clinical condition, motor behavior, and hand function.
Upcoming plans for TSHA-102
TSHA-102 is administered via a single injection through the spine, or intrathecal injection. It’s designed to deliver to cells a miniaturized version of the MECP2 gene, mutations of which cause most cases of Rett syndrome.
Before this year ends, Taysha plans to submit an investigational new drug application, or IND, for TSHA-102 to the U.S. Food and Drug Administration (FDA). If it’s accepted, it would grant permission to begin clinical testing in the U.S.
The company also is planning to ask the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) for permission to launch a clinical trial of TSHA-102 in children with Rett syndrome.
“We continue to make significant progress … remain on track to deliver on multiple key milestones, including the generation of first-in-human clinical data for TSHA-102 in adult patients with Rett syndrome, the submission of a [clinical trial application] to the MHRA to initiate expansion of TSHA-102 in pediatric patients, the submission of an IND application to the FDA for TSHA-102,” Nolan said.
New preclinical data on TSHA-102 will be presented at the annual meeting of the American Society of Gene and Cell Therapy (ASGCT). Taysha is also developing a gene therapy, dubbed TSHA-120, to treat giant axonal neuropathy, or GAN, another genetic neurological condition.
“We believe that the clinical and preclinical data generated to date across our Rett syndrome and GAN programs reinforce our gene therapy approach, and the therapeutic potential to address severe unmet needs in monogenic central nervous system disease,” said Sukumar Nagendran, MD, president, and head of R&D at Taysha.
Monogenic conditions are those caused by a mutation in a single gene.
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