Taysha IPO Presentation Deck slide image

Taysha IPO Presentation Deck

Deep pipeline of gene therapies targeting neurodegenerative diseases TSHA-104 GRT SURF1 Deficiency IND-enabling studies TSHA-112 GRT/miRNA APBD Preclinical TSHA-111 GRT/miRNA LaFora Preclinical TSHA-113 miRNA Tauopathies Preclinical TSHA-115 miRNA GSDs Preclinical SURF1 protein implicated in mitochondrial translation and assembly of cytochrome C oxidase complex; most frequent cause of Leigh syndrome Patients develop difficulty in swallowing and subsequent failure to thrive. Death ensues through respiratory failure by approximately age 10 Estimated prevalence of 300 patients in the US and EU GBE1 is responsible for the creation of branches during glycogen synthesis and a reduction yields elongated glycogen changes that form poorly soluble aggregates Prime of life disease, with onset between 40-50 years; patients have sensory loss in the legs, progressive muscle weakness, gait disturbances, and cognitive impairment Estimated prevalence of 1,000 patients in the US and EU • EPM2A and EPM2B genes are involved in forming of glycogen metabolism, and without the proteins, poorly branched Lafora bodies aggregate Adolescents typically present with myoclonus, progressive dementia, refractory epilepsy, cerebellar ataxia and respiratory failure, and generally results in death within about a decade Estimated prevalence of 700 patients in the US and EU Tau accumulation is thought to underpin several neurodegenerative diseases, including Alzheimer's, frontotemporal dementia, and parkinsonism linked to chromosome 17 We are employing tau-specific miRNA shuttles that have been designed to target mRNA for all six isoforms of tau found in the human brain and/or mouse brain Estimated prevalence of 40,000 patients in the US and EU miRNA targeting GYS1 to inhibit glycogen synthase in the brain to decrease abnormal glycogen formation This approach may enable the treatment of several glycogen storage disorders Estimated prevalence of 20,000 patients in the US and EU * 26
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