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Shares of Taysha Gene Therapies, Inc. (TSHA) rallied 49% by Thursday morning after the U.S. Food and Drug Administration granted Breakthrough Therapy designation to its gene therapy TSHA-102 for the treatment of Rett syndrome.
Rett syndrome is a rare genetic neurological and developmental disorder that affects the way the brain develops. The disorder, which primarily occurs in females, is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities, and shortened life expectancy.
According to Taysha, there are currently no approved disease-modifying therapies that treat the genetic root cause of the disease, which affects between 15,000 and 20,000 patients in the U.S., the EU, and the U.K.
The FDA on Thursday granted Taysha’s TSHA-102 Breakthrough Therapy designation. It is intended to expedite the development and regulatory review of an investigational therapy intended to treat a serious condition. The designation follows the agency’s review of available clinical data from the 12 patients treated in a part of its early-to mid-stage trials.
On Stocktwits, retail sentiment around TSHA stock jumped from ‘bearish’ to ‘extremely bullish’ territory over the past 24 hours, while message volume improved from ‘normal’ to ‘extremely high’ levels.
Taysha also said on Thursday that it has finalized alignment with the FDA on the protocol for the trial intended to support its application for approval of TSHA-102. The company is now on track to begin patient enrollment in the trial in the fourth quarter of 2025. In the trial, a single intrathecal administration of high-dose TSHA-102 will be evaluated in 15 females between the ages of six and below 22 years with Rett syndrome.
Following the news, Needham analyst Gil Blum raised the firm's price target on Taysha Gene Therapies to $10 from $8 while keeping a ‘Buy’ rating on the shares. Needham views the developments as "derisking" for TSHA-102, putting the program well ahead of Neurogene’s gene therapy NGN-401, which is also being evaluated for Rett syndrome.
TSHA stock has risen 174% this year and by about 131% over the past 12 months.
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