Taysha Gene Therapies has received a regenerative medicine advanced therapy (RMAT) designation for its gene therapy candidate TSHA-102 from the US Food and Drug Administration (FDA) to treat Rett syndrome. 

TSHA-102 is a gene therapy that involves using an AAV9 virus to deliver the MECP2 gene directly into the central nervous system to treat the underlying cause of Rett syndrome, a rare genetic disorder caused by mutations in the MECP2 gene that affects brain development. It primarily occurs in girls and results in severe mental and physical disability.  

The RMAT designation programme is intended to expedite the development and review of regenerative therapies to treat, modify, reverse, or cure a serious condition. 

The FDA’s decision to grant the designation to Taysha is supported by initial results from the ongoing Phase I/II REVEAL clinical trials in adults (NCT05606614), and paediatric (NCT06152237) patients. The FDA reviewed safety and efficacy data from the first three patients dosed with the low dose of TSHA-102 across the two trials. Initial safety and efficacy data from the high-dose cohorts from both trials is expected in the second half of 2024, as per a 29 February update. 

In October 2022, Astellas announced plans to make a strategic investment to back the development of Taysha’s development programmes for Rett syndrome and giant axonal neuropathy (GAN). The pharma giant gained an exclusive option for licensing two clinical-stage programmes of Taysha—TSHA-102 and TSHA-120 for the treatment of GAN. 

Things however did not go to plan as about a year later, Taysha discontinued its TSHA-120 development programme following a Type C meeting with the US Food and Drug Administration (FDA), where the agency suggested the company conduct a placebo-controlled study and do a long-term follow-up trial. Following this, Astellas chose not to exercise its right to obtain an exclusive licence for the drug. 

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After this, Taysha said that it would shift its focus to the TSHA-102 programme. According to GlobalData’s Pharma Intelligence Center, TSHA-102 is forecast to generate $650m in sales in 2030. 

GlobalData is the parent company of Pharmaceutical Technology. 

In the announcement accompanying the designation, Taysha’s president and head of R&D Sukumar Nagendran said: “We believe receiving RMAT designation reinforces the high unmet medical need in Rett syndrome and the therapeutic potential of TSHA-102 to change the treatment paradigm.” 

In April 2023, Acadia commercialised the first US Rett syndrome drug Daybue (trofinetide). Daybue is a synthetic analog of N-terminal tripeptide of insulin-like growth factor 1. Still, there is currently no disease-modifying treatment that treats the genetic root of the disease.  

Cell and gene therapy coverage on Pharamceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.