Taysha Gene Therapies (TSHA) Announces Late-Breaking Abstract and Poster Presentation on Positive Preclinical Data For TSHA-105

December 6, 2021 4:11 PM EST
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Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced a late-breaking abstract and poster presentation by Dr. Rachel Bailey, Assistant Professor at UT Southwestern Medical Center on positive preclinical data for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy at the American Epilepsy Society Annual Meeting on December 6th 2021.

“SLC13A5-related epilepsy results from a mutation in the SLC13A5 gene that prevents citrate from being taken up into neurons in the brain. Affected children present with seizures beginning within a few days of birth and as they continue to grow, have motor progression and difficulty with speech and language development. Some children never achieve walking independently,” said Rachel M. Bailey, Ph.D., Assistant Professor with the Center for Alzheimer's and Neurodegenerative Diseases and Pediatrics at UT Southwestern. “Currently there are no treatments for SLC13A5 deficiency that target the underlying cause of disease. In knockout mouse models of SLC13A5 deficiency, treatment with TSHA-105 resulted in normalized citrate levels, reduced seizure activity and improved survival regardless of age. We are highly encouraged by the positive therapeutic response and absence of toxicity in these preclinical models and look forward to further exploring the possible utility of TSHA-105 as a treatment for SLC13A5 deficiency.”

Dr. Bailey, whose research is supported in part by Taysha, and UT Southwestern maintain financial interests in Taysha Gene Therapies due to their development of the intellectual property that serves as the basis for TSHA-105.

“These preclinical results suggest that TSHA-105 can demonstrate functional improvements with intervention at any age in a potentially safe and tolerable manner which would be expected to translate into a meaningful benefit to patients with SCL13A5 deficiency,” said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. “These highly encouraging preclinical data further support our plan to submit an IND/CTA filing in 2022.”

TSHA-105 is a self-complementary vector encoding a codon-optimized human SLC13A5 gene that was evaluated in an SLC13A5 knockout (KO) mouse model recapitulating the increased plasma citrate levels, electroencephalogram (EEG) abnormalities and an increased susceptibility to seizure induction seen in patients. CSF-delivered TSHA-105 significantly decreased plasma citrate levels in SLC13A5 KO mice and reduced epileptic activity. Increased seizure susceptibility in SLC13A5 KO mice measured by the Racine scale, a well-established methodology for assessing seizure severity in preclinical models, was attenuated with TSHA-105 treatment in both age groups. No adverse findings were detected following CSF delivery of TSHA-105. An IND/CTA filing for TSHA-105 for the treatment of SLC13A5-related epilepsy is expected in 2022. TSHA-105 previously received orphan drug designation from the U.S. Food and Drug Administration and from the European Commission.

SLC13A5 deficiency is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. As an autosomal recessive disorder, two copies of the mutated gene must be inherited for an infant to be affected. This type of epilepsy manifests as developmental delay, and seizures beginning within the first few days of life. SLC13A5 deficiency is a rare disorder, with an estimated prevalence of 1,900 patients in the United States and in Europe. Current standards of care include anti-seizure medications which only target the symptoms and do not address the underlying cause of the disease.



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