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The Taysha Gene Therapies Inc (NASDAQ: TSHA) share price rose more than 3% during today’s intraday trading. That’s why it happened.

The share price of Taysha Gene Therapies Inc (NASDAQ: TSHA) – a patient-centered, pivotal stage gene therapy company focused on the development and commercialization of AAV-based gene therapies for the treatment of Monogenic central nervous system (CNS) diseases in both rare and large patient populations – increased by more than 3% during today’s intraday trading. Investors responded positively to the company by announcing that it had obtained an exclusive option from UT Southwestern (UTSW) to license the worldwide rights to a clinical-stage AAV9 gene therapy replacement program for the treatment of CLN7 disease.

The company has also entered into a research collaboration with UTSW to develop a next-generation construct for the treatment of CLN7 disease – which is expected to improve potency, safety profile, packaging efficacy and manufacturability compared to to first generation construction. And the next generation construction design is expected to be completed by the end of 2021 with commercial grade GMP materials expected in 2022.

The first generation construction of the CLN7 program was developed in the lab of Steven Gray, Ph.D., Associate Professor at UT Southwestern Medical Center and Chief Scientific Advisor to Taysha, with financial support from Mila’s Miracle and Batten Hope , the main CLN7 patient advocacy groups. And the CLN7 program is currently undergoing a Phase 1 proof-of-concept clinical trial conducted by UTSW, and Taysha expects preliminary clinical data for human proof of concept and efficacy of the first-generation construct are available by the end of 2021. Taysha plans to launch a planned pivotal trial using a next-generation construct in 2022, with reference to human proof-of-concept clinical data generated from the construct. first generation. And Taysha awarded a grant to Batten Hope to support patient awareness, disease education and newborn screening initiatives.

CLN7 disease is known as a rare, fatal and rapidly progressive neurodegenerative disease which is a form of Batten disease. And CLN7 is caused by autosomal recessive mutations in the MFSD8 gene which results in lysosomal dysfunction.

The onset of the disease occurs around the age of 2 to 5 years, and death often occurs in early adolescence. And patients experience gradual loss of nerve cells in parts of the brain and typically experience seizures, vision loss, speech disturbances, and mental and motor regression. Currently, there is no approved therapy for the treatment of CLN7 disease, which affects approximately 4,000 patients worldwide.

Preclinical data in rodents supported the advancement of the first generation construct in a phase 1 clinical proof of concept study in patients with CLN7 disease. And in an in vivo efficacy study, intrathecal (IT) administration of the first generation construct to MFSD8 knockout mice with high or low doses resulted in clear age and dose effects with high dose. early intervention and a high dose to achieve the best therapeutic benefits. A high IT dose of the first generation construct in younger knockout mice resulted in: 1) generalized expression of MFSD8 mRNA in all tissues tested; 2) almost complete normalization of altered free field and rotarod performance at 6 and 9 months after injection; 3) more than doubled the median life expectancy (16.82 months versus 7.77 months in untreated knockout mice); and 4) maintaining a healthy body weight for an extended period. Toxicology studies in wild-type rodents have demonstrated the safety and tolerability of computer administration of the first generation construct. These preclinical data will be presented by Xin Chen, Ph.D., assistant professor, department of pediatrics at UT Southwestern, during an oral presentation at the 17th Annual International Ceroid Neuronal Lipofuscinosis Congress on October 8, 2021.

UTSW is currently enrolling patients in a researcher-sponsored open-concept Phase 1 clinical trial at Children’s Hospital in Dallas for AAV9-based gene replacement therapy administered intrathecally for treatment of childhood disease CLN7. And the primary endpoint of the trial is safety and tolerability based on the incidence and severity of serious treatment-related adverse events.

Secondary efficacy endpoints include overall clinical impression, neuropsychological, ataxic and motor assessments, and quality of life. And the rationale for the design and a discussion of the outcome measures of this clinical trial will be presented in poster form at the upcoming 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis. To date, one patient has received a dose of 5 × 1014 total vg and a second patient has received a dose of 1 × 1015 total vg as measured by the qPCR method. UTSW continues to enroll patients in this phase 1 study at 1 × 1015 vg total and plans to assay additional patients in the short term. Preliminary safety and efficacy data are expected by the end of 2021.

With the addition of the CLN7 program, Taysha expects to have 5 clinical stage programs by the end of the year. And as such, the TSHA-104 program for the treatment of Leigh syndrome associated with SURF1 will pass to company collaborators at UTSW to complete studies enabling IND, followed by a planned clinical trial by the investigator by the end of 2022. Taysha is going to continue to support the SURF1 natural history study in partnership with UTSW.

Financial terms of the agreements were not disclosed.

KEY QUOTES:

“The CLN7 program is a strategic addition to our gene therapy pipeline focused on monogenic CNS diseases. Encouraging preclinical data generated in relevant rodent models suggests that the first generation construct has the potential to reduce overall disease pathology, preserve motor function, and ultimately prolong survival. The first-generation construct is currently in a Phase 1 proof-of-concept clinical trial with two patients treated to date, and we look forward to the availability of preliminary data by the end of the year. With human proof-of-concept clinical data to benchmark, we plan to advance a next-generation construct in a pivotal trial scheduled for 2022, which is expected to improve potency, safety, packaging efficacy and manufacturability by compared to the first generation construction. Importantly, we are also pleased to announce our grant to Batten Hope, the leading nonprofit patient advocacy organization for CLN7 disease, to support patient awareness, disease education and screening initiatives. neonatal. We believe that a gene therapy approach has the potential to address a significant unmet need in approximately 4,000 patients worldwide.

– RA Session II, President, Founder and CEO of Taysha

“Our mission is to support families with children suffering from terminal and rapidly progressive neurodegenerative diseases like CLN7. We are honored to receive Taysha’s support to raise awareness, increase newborn screening, and help patients access potentially life-changing treatments that offer hope and therapeutic advancements for conditions with significant unmet needs.

– Gina Hann, Founder, President and Treasurer of Batten Hope

Disclaimer: This content is intended for informational purposes. Before making any investment, you should do your own analysis.

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