Taysha Gene Therapies (TSHA) Investor Presentation - Slideshow

GAN Program Acquisition Highlights - The GAN program transforms Taysha into a pivotal-stage gene therapy company[13, 103] - TSHA-120 has shown clear arrest of disease progression and long-term durability in patients treated for 3+ years[13, 68, 103] - TSHA-120 is well-tolerated with efficacy established at multiple doses in an ongoing clinical trial[13, 61, 103] - The estimated prevalence of GAN is 2,400 patients in the US & EU, representing a potentially greater than $2 billion near-term commercial opportunity[13, 21, 25, 31, 103] TSHA-120 Clinical Data - Clinical data demonstrated arrest of disease progression[16] - Bayesian analysis confirmed documented natural history data of an 8-point decline in the MFM32 total % score per year[70] - TSHA-120 dose of 1.8x10^14 total vg resulted in an arrest of disease progression that was statistically significant[70, 71] - 1.8x10^14 total vg dose confirmed nearly 100% probability of clinically meaningful slowing of disease compared to natural history decline of GAN patients[83, 84] - 1.2x10^14 total vg dose confirmed approximately 85% probability of clinically meaningful slowing of disease and 100% probability of any slowing of disease[83, 84] Vagus Nerve Redosing Platform - There is an opportunity to achieve human POC for vagus nerve redosing platform with previously treated low dose patients[13, 89, 103]