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 (TSHA) reported positive initial data for TSHA-101, a gene therapy for GM2 gangliosidosis. Patient 1 with Sandhoff disease achieved Hex A enzyme activity at 190% of normal by Month 1 and 288% by Month 3, translating to 58-fold above the asymptomatic level. Patient 2 with Tay-Sachs saw a 5-fold increase at Month 1. This is the first support for a bicistronic vector in humans, aiming to normalize enzyme activity. The therapy was well-tolerated, with further clinical updates expected by year-end.
Taysha Gene Therapies (Nasdaq: TSHA) has announced that RA Session II, its President and CEO, will present a corporate overview at the 40th Annual J.P. Morgan Healthcare Conference on January 13, 2022, at 9:00 AM ET. The presentation will focus on Taysha's mission to develop AAV-based gene therapies aimed at treating monogenic diseases of the central nervous system. A webcast of the presentation will be accessible in the 'Events & Media' section of Taysha's website, with archived access available for 60 days.
Taysha Gene Therapies, Inc. (Nasdaq: TSHA) announces participation of RA Session II, President and CEO, in two panels at the LifeSci Partners 11th Annual Corporate Access Event on January 6, 2022. The first panel, titled Gene Therapy: Reaching its Full Potential, occurs at 11:00 AM ET, while the second, Patient Advocacy: Honing the Skill for Crossover Communications, takes place at 2:00 PM ET. Viewers can access the webcasts through the company's website, which also offers archived versions for 60 days.
Taysha Gene Therapies (NASDAQ: TSHA) announced plans for clinical data updates in January 2022, focusing on TSHA-101 for GM2 gangliosidosis and TSHA-120 for giant axonal neuropathy (GAN). The virtual investor calls will cover preliminary clinical safety data and enzyme activity for TSHA-101, as well as safety and efficacy results for the highest dose of TSHA-120. Taysha aims to discuss the program's status, potential regulatory strategies, and commercialization efforts to improve the lives of patients with monogenic CNS diseases.
Taysha Gene Therapies (NASDAQ: TSHA) has been added to the ICE Biotechnology Index (NYSE: ICEBIO), effective December 20, 2021. This inclusion recognizes its value and platform in the biotechnology sector, focusing on gene therapies for central nervous system disorders. CEO RA Session II emphasized the importance of this validation as the company works towards clinical and regulatory milestones to enhance visibility within the investment community. The ICE Biotechnology Index tracks U.S.-listed biotech companies engaged in therapeutic research and development.
Taysha Gene Therapies (TSHA) reported preliminary clinical safety data for its first-generation CLN7 construct, demonstrating a favorable safety profile with no major adverse events observed. The Data Safety Monitoring Board supported dose escalation to the highest dose of 1.0x1015 viral genomes in humans. Upcoming results will be presented at the 18th Annual WORLDSymposium in February 2022. Additionally, Taysha has designed a next-generation construct aimed at enhancing potency and manufacturability while reducing immunogenicity risks.
Taysha Gene Therapies (NASDAQ: TSHA) has initiated the clinical development of TSHA-118 for treating CLN1 disease, following a recently approved Clinical Trial Application. The first clinical site is Queen’s University in Ontario, Canada, under Dr. Jagdeep Wahlia. Preclinical studies show that TSHA-118 can significantly improve survival and behavior in models of CLN1 disease, with preliminary clinical safety and enzyme activity data expected in H1 2022. This gene therapy holds promise as a disease-modifying treatment for CLN1 disease, which affects approximately 1 in 138,000 live births globally.
Taysha Gene Therapies, Inc. (Nasdaq: TSHA) has been officially included in the Nasdaq Biotechnology Index effective December 20, 2021. This inclusion aims to enhance the company's visibility and attract investors as it continues to advance its AAV-based gene therapy pipeline focused on treating monogenic diseases of the CNS. The Nasdaq Biotechnology Index tracks biotechnology and pharmaceutical securities that meet specific eligibility criteria, reflecting Taysha's commitment to developing curative treatments for patients.
Taysha Gene Therapies (NASDAQ: TSHA) presented promising preclinical data for TSHA-105 at the American Epilepsy Society Annual Meeting on December 6, 2021. The AAV9-based gene therapy showed the ability to normalize electroencephalogram (EEG) and citrate levels, improving survival rates in mouse models with SLC13A5 deficiency. No adverse effects were noted following cerebrospinal fluid delivery. The therapy received orphan drug designation from both the U.S. FDA and European Commission, with an IND/CTA filing expected in 2022.
Taysha Gene Therapies (Nasdaq: TSHA) reported significant advancements in its clinical pipeline during Q3 2021. The company anticipates clinical safety and functional data for TSHA-120 in GAN, TSHA-101 in GM2 gangliosidosis, and preliminary data for the first-ever dosed patient in the CLN7 program, all expected in December 2021. Notably, Taysha completed five GMP campaigns to support its clinical programs and received Orphan Drug Designation for multiple therapies. However, net losses increased to $51.2 million, or $1.35 per share, from $15 million, or $1.28 per share, a year earlier.