Taysha Gene Therapies, Inc. Stock Price, News & Analysis

Taysha Gene Therapies (TSHA) has received orphan drug designation from the European Commission for TSHA-101, a gene therapy targeting GM2 gangliosidosis (Tay-Sachs disease). This therapy is currently the only bicistronic vector in clinical development and is expected to provide a life-changing treatment option, as no approved therapies exist for this fatal neurodegenerative disorder. Preliminary clinical safety and biomarker data from ongoing trials at Queen's University are anticipated by year-end 2021.

Taysha Gene Therapies (NASDAQ: TSHA) will participate in Chardan’s 5th Annual Genetic Medicines Conference on October 5, 2021, at 1:30 pm ET, featuring a fireside chat with its CEO. Additionally, the company will be showcased during the Meeting on the Mesa on October 12, 2021, with a panel discussion at 7:15 am PT and a corporate presentation at 2:30 pm PT. These events are crucial for discussing advancements in gene therapy, focusing on the treatment of monogenic CNS diseases. Webcasts will be available for attendees.

Taysha Gene Therapies (TSHA) has received orphan drug designation from the European Commission for TSHA-102, a gene therapy targeting Rett syndrome. Preclinical data show significant survival benefits in mouse models, indicating its potential for human application. The company plans to file an IND/CTA in the second half of 2021 and begin a Phase 1/2 trial by year-end. With an estimated 25,000 patients in the U.S. and Europe, TSHA-102 represents a multi-billion-dollar market opportunity. This development follows previous FDA designations for the therapy.

Taysha Gene Therapies has published promising preclinical data for TSHA-104, a gene therapy targeting SURF1-associated Leigh syndrome. The study, featured in Molecular Therapy: Methods & Clinical Development, shows that TSHA-104 can restore normal blood lactate levels and enhance COX1 activity in a dose-dependent manner in SURF1 knockout mice. No toxicity or severe tissue damage was observed. Notably, there are currently no approved therapies for this condition, highlighting the significance of TSHA-104's potential.

Taysha Gene Therapies, Inc. (Nasdaq: TSHA) will host a virtual KOL webinar on September 22, 2021, from 10:00 a.m. to 12:30 p.m. ET, focusing on TSHA-102 for Rett syndrome.

Key speakers include Dr. Jeffrey Neul, who will overview Rett syndrome, and Monica Coenraads from RSRT, who will discuss the disease's burden. Steven Gray will present preclinical data, while Suyash Prasad will cover the clinical development strategy and regulatory updates for TSHA-102. Register for the event on Taysha's website.

Taysha Gene Therapies (NASDAQ: TSHA) announced its participation in multiple investor healthcare conferences in September 2021, including Citi’s 16th Annual BioPharma Conference and the Morgan Stanley Global Healthcare Conference. Key dates include September 8 for Citi, September 9 at 5:00 pm ET for Morgan Stanley, and several others leading up to September 27. The company's leadership will participate in various formats such as 1x1 meetings and fireside chats, showcasing their commitment to advancing gene therapies for monogenic CNS diseases.

Taysha Gene Therapies (Nasdaq: TSHA) has received orphan drug designation from the European Commission for its leading gene therapy candidate, TSHA-105, aimed at treating SLC13A5-related epilepsy. This rare genetic condition lacks approved treatments, affecting approximately 1,900 patients across the U.S. and Europe. The designation highlights the urgent need for effective therapies and provides Taysha with regulatory advantages, including market exclusivity and reduced fees. TSHA-105 also holds orphan designation from the FDA, emphasizing its potential to address this debilitating condition.

Taysha Gene Therapies (Nasdaq: TSHA) is hosting a virtual key opinion leader webinar on TSHA-118, aimed at treating CLN1 disease, on August 30, 2021, from 10:00 a.m. to 12:30 p.m. ET. The event will feature Dr. Angela Schulz, who will present an overview of CLN1 disease, along with patient perspectives and preclinical data for TSHA-118. Notable speakers include Sharon King and Steven Gray, with a Q&A session to follow. Registration is available online, and an archived version will be accessible for 60 days post-event.

Taysha Gene Therapies, Inc. (Nasdaq: TSHA) reported its Q2 2021 financial results, showcasing significant advancements in developing gene therapies for CNS diseases. A non-dilutive financing of up to $100 million was secured, enhancing cash reserves to $197.4 million. Positive feedback from regulators for multiple programs allows for anticipated IND filings later this year. R&D expenses increased to $30.6 million, and net loss was $40.9 million. Upcoming milestones include clinical trial data releases and regulatory updates, reflecting a robust pipeline.