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Taysha Gene Therapies, Inc. (Nasdaq: TSHA) is a pioneering clinical-stage biotechnology company headquartered in Dallas, Texas. Founded in 2019, Taysha is dedicated to developing and commercializing transformative gene therapies using adeno-associated viruses (AAV) to treat severe monogenic diseases of the central nervous system (CNS).
Taysha focuses on eradicating monogenic CNS diseases, addressing severe unmet medical needs, and dramatically improving the lives of patients and their caregivers. With a robust pipeline, including leading clinical program TSHA-102, the company aims to develop curative medicines. TSHA-102, currently in clinical evaluation for Rett syndrome, employs miRNA-Responsive Auto-Regulatory Element (miRARE) technology to deliver a functional MECP2 gene, aiming to mediate MECP2 levels in the CNS without the risk of overexpression.
Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the MECP2 gene, leading to severe intellectual disabilities and various neurological impairments. TSHA-102 has received multiple designations, including Fast Track, Orphan Drug, and Rare Pediatric Disease from the FDA, and Orphan Drug designation from the European Commission, underscoring its potential to address significant medical needs.
Taysha has also made strategic advances with its partnerships, particularly with the University of Texas Southwestern Medical Center. This collaboration focuses on developing and commercializing AAV-based gene therapies, leveraging Taysha's integrated platform and the university's Gene Therapy Program.
Recently, Taysha achieved numerous milestones, including significant updates to the TSHA-102 program. The Independent Data Monitoring Committee (IDMC) approved dose escalation in the REVEAL Phase 1/2 trials, indicating promising initial safety and efficacy data. Taysha also expanded these trials into the United States, enhancing their ability to evaluate TSHA-102 across broader patient demographics.
Furthermore, Taysha terminated an existing loan agreement and entered a new one to facilitate the transfer of intellectual property (IP) for several programs to third parties. This strategic move extended their cash runway into 2026, demonstrating financial prudence and long-term planning.
The company is actively progressing clinical trials for TSHA-102, including both adolescent/adult and pediatric cohorts, which are crucial for regulatory and clinical development. Taysha's focus on innovative gene therapy solutions, combined with a patient-centric approach, positions it as a significant player in the biotech landscape.
For continuous updates and detailed information, visit the company’s website at www.tayshagtx.com.
Taysha Gene Therapies, Inc. (NASDAQ: TSHA) will participate in a fireside chat at the Jefferies Gene Therapy & Editing Summit on October 28, 2021, at 11:00 am ET. This pivotal-stage company focuses on developing AAV-based gene therapies for monogenic diseases affecting the central nervous system. Key participants include RA Session II, President and CEO; Dr. Suyash Prasad, Chief Medical Officer; Kamran Alam, CFO; and Dr. Kimberly Lee, SVP of Corporate Communications. Taysha aims to rapidly translate innovative therapies from research to clinical application.
Taysha Gene Therapies has launched a partnership with GeneDx to provide free genetic testing for Giant Axonal Neuropathy (GAN) to individuals at risk. This initiative aims to enhance disease awareness and improve diagnostic accessibility, addressing the estimated 2,400 patients with GAN in the US and Europe. The company's gene therapy, TSHA-120, has shown promising results in halting disease progression in clinical trials. Data from the high-dose cohort is expected in late 2021.
Taysha Gene Therapies will host a virtual webinar on October 26, 2021, from 10:00 a.m. to 1:00 p.m. ET, focusing on TSHA-106 for treating Angelman syndrome. The event will feature expert presentations, including Dr. Ben Philpot on gene replacement strategies, Dr. Kimberly Goodspeed on the syndrome overview, Dr. Allyson Berent discussing disease burden, Dr. Ryan Butler on UBE3A-ATS approaches, and Dr. Suyash Prasad reviewing clinical development strategies. Registration and live viewing are available on Taysha's website.
Taysha Gene Therapies (NASDAQ: TSHA) has announced advancements in its CLN7 gene therapy program. Currently in Phase 1 clinical trials, preliminary data is expected by year-end 2021. The company has partnered with UT Southwestern to develop an improved next-generation construct aimed at enhancing potency and safety. Data from preclinical studies show significant improvements in motor function and life expectancy in affected mice. Taysha plans to initiate a pivotal trial in 2022, with an estimated 4,000 CLN7 patients globally and no current approved therapies.
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.
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