Taysha Gene Therapies, Inc. reports company developments as a clinical-stage biotechnology company advancing adeno-associated virus, or AAV, gene therapies for severe monogenic diseases of the central nervous system. Its lead program, TSHA-102, is an intrathecally delivered, self-complementary AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome.
Recurring updates cover TSHA-102 clinical and preclinical data, FDA regulatory interactions and designations, manufacturing and comparability topics, financial results, corporate updates, and employee inducement equity grants under Nasdaq listing rules. Company communications also describe the program’s MECP2 construct design, miRARE technology, and clinical trial activity in Rett syndrome.
Taysha Gene Therapies (Nasdaq: TSHA) has announced its participation in three significant healthcare conferences: the Stifel 2021 Virtual Healthcare Conference on November 15, 2021, the Jefferies London Healthcare Conference on November 17, 2021, and the 4th Annual Evercore ISI HealthCONx Virtual Conference on November 30, 2021. The company focuses on developing AAV-based gene therapies for monogenic CNS diseases. Key participants include CEO RA Session II, Chief Medical Officer Dr. Suyash Prasad, and CFO Kamran Alam.
Taysha Gene Therapies (NASDAQ: TSHA) announced it will report third-quarter financial results on November 10, 2021, at 8:00 AM Eastern Time. The company is focused on developing AAV-based gene therapies for central nervous system diseases, aiming to provide curative treatments for both rare and larger patient populations. The conference will include a corporate update and access to a webcast will be available for 30 days post-event.
Taysha Gene Therapies (NASDAQ: TSHA) has announced promising preclinical data on an AAV-mediated UBE3A gene replacement therapy for Angelman syndrome. This novel approach, developed by Drs. Ben Philpot and Steven Gray, successfully packages both short and long isoforms of the UBE3A gene, showing improved motor learning, behavior, and seizure outcomes in mouse models. The therapy is seen as a potential safe treatment for Angelman syndrome, impacting approximately 55,000 patients in the U.S. and Europe. Further insights and data will be shared during the upcoming investor day on October 26, 2021.
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.