Taysha Gene Therapies Raises Funds, Advances Rare Disease Treatments.

Taysha Gene Therapies has priced a share and pre-funded warrant offering to advance its adeno-associated virus-based gene therapies for severe monogenic diseases of the central nervous system. The company's lead program, TSHA-102, is in development for Rett syndrome, and it is also evaluating TSHA-120 for giant axonal neuropathy. Taysha has received orphan drug designation from the European Commission for TSHA-120.

Taysha Gene Therapies, Inc. (TSHA) has priced a secondary share and pre-funded warrant offering to bolster its financial resources for developing adeno-associated virus (AAV)-based gene therapies targeting severe monogenic diseases of the central nervous system. The company's lead program, TSHA-102, is in development for Rett syndrome, while TSHA-120 is being evaluated for giant axonal neuropathy. TSHA-120 has received orphan drug designation from the European Commission [3].

The stock price of Taysha Gene Therapies climbed 13% to $2.76 on Wednesday after the company announced details of a planned pivotal Part B trial for TSHA-102 and filed for a secondary share offering [1]. The biotechnology company expects to initiate trial activities in the third quarter of 2025, following written alignment with the Food and Drug Administration (FDA) [2]. The trial will assess if patients gain or regain developmental milestones over 12 months and includes a six-month interim analysis.

The company also announced it has commenced a public offering of shares of its common stock, as well as pre-funded warrants to purchase shares of its common stock. The underwriters of the offering include Jefferies, BofA Securities, Piper Sandler, and Barclays. The offering's details, including the expected price and amount of shares, were not disclosed [1].

Taysha Gene Therapies has made significant strides in its clinical development program. As of the first quarter of 2025, the company has treated six high-dose patients without any reported safety concerns, building upon earlier data from low-dose cohorts that demonstrated encouraging efficacy signals across multiple functional domains in both pediatric and adult patients [3].

The market opportunity for an effective Rett syndrome treatment is substantial, with conservative estimates pointing to a prevalent patient population of around 25,000 in the United States and European Union combined. Analysts project that this could translate to over $2 billion in potential revenues for Taysha if TSHA-102 proves successful [3].

Taysha's progress on the regulatory front has been noteworthy, with the company successfully aligning with the FDA on key elements of the pivotal Part B trial design for TSHA-102. This alignment suggests the possibility of a single-arm study using natural history comparisons, which could streamline the path to potential approval [3].

The upcoming data release, expected between June 9-11, 2025, is anticipated to provide crucial insights into the efficacy of the high-dose treatment and could serve as a major catalyst for the company’s stock.

References:
[1] https://www.marketwatch.com/story/taysha-gene-therapies-stock-rises-on-trial-design-secondary-share-offering-bb8a9908
[2] https://www.marketscreener.com/quote/stock/TAYSHA-GENE-THERAPIES-INC-112942431/news/Taysha-Gene-Therapies-Announces-Pivotal-Part-B-Trial-Design-Details-for-TSHA-102-in-Rett-Syndrome-En-50094092/
[3] https://ng.investing.com/news/swot-analysis/taysha-gene-therapies-swot-analysis-promising-gene-therapy-stock-faces-pivotal-year-93CH-1936807