Taysha Gene Therapies Raises $150 Million in Private Placement

Rare Daily Staff

Taysha Gene Therapies said it has entered into a securities purchase agreement for a private placement financing that is expected to result in gross proceeds of approximately $150 million, before deducting placement agent commissions and offering expenses.

New investor RA Capital Management led the PIPE with participation from a large institutional investor, PBM Capital, RTW Investments, LP, Venrock Healthcare Capital Partners, TCGX, Acuta Capital Partners, Kynam Capital Management, Octagon Capital, Invus, GordonMD Global Investments, and B Group Capital.

“We expect that the net proceeds from the PIPE, together with our existing cash and cash equivalents, will extend our cash runway into the third quarter of 2025 to primarily support the clinical development of TSHA-102 in Rett syndrome and provide support for TSHA-120 program activities in GAN, working capital and other general corporate purposes,” said Sean Nolan, chairman and CEO of Taysha. “With this capital infusion, we believe we are well positioned to continue to execute across key program milestones.”

Taysha also reported that data from the first patient dosed in the REVEAL phase 1/2 trial evaluating TSHA-102 for the treatment of Rett syndrome in adults showed that it was well-tolerated with no treatment-emergent serious adverse events (SAEs) as of six-week assessment and improvement in key efficacy measures, including Clinical Global Impression—Improvement (CGI-I), Clinical Global Impression—Severity (CGI-S) and Rett Syndrome Behavior Questionnaire (RSBQ), four weeks post-treatment.

The principal Investigator observed clinical improvement in multiple domains, including autonomic function (sleep and breathing), vocalization, as well as gross motor skills (gained ability to sit unassisted for three minutes) and fine motor skills (gained ability to hold objects), which were supported by initial clinical data and video evidence.

Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene. The disorder is characterized by intellectual disabilities, loss of communication, seizures, slowing and/or regression of development, motor and respiratory impairment, and shortened life expectancy. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the United States, European Union, and United Kingdom.

“We are highly encouraged by the initial data for TSHA-102 and are focused on continuing to explore its therapeutic potential, with the dosing of the second patient expected in the third quarter,” said Nolan. “We also received FDA clearance to initiate clinical development of TSHA-102 in pediatric patients in the U.S. and have submitted a CTA to the MHRA for TSHA-102 in pediatric patients with Rett syndrome, which will expand our clinical evaluation to children with earlier stages of disease progression.”

Photo: Sean Nolan, chairman and CEO of Taysha