Taysha Gene Therapies develops intrathecally delivered AAV gene-replacement therapies targeting rare monogenic central nervous system (CNS) diseases. Its pipeline includes TSHA-102 (a self-complementary AAV9 therapy for Rett syndrome using the miRARE auto-regulatory element) and TSHA-120 (an AAV9 program for giant axonal neuropathy, in Phase 1/2). TSHA-102 is described as the first-and-only gene therapy in clinical development for Rett syndrome; both programs have received multiple Orphan Drug designations. Taysha has partnered with the UT Southwestern Gene Therapy Program and emphasizes a fully integrated AAV platform to translate treatments from bench to bedside. The company is publicly listed (Nasdaq: TSHA) and announced a strategic equity investment from Astellas to support advancement of its clinical-stage programs. Taysha’s stated mission is to eradicate monogenic CNS disease by rapidly developing curative medicines. Taysha Gene Therapies develops gene therapies. The company announced it entered into a loan and security agreement with Silicon Valley Bank. The agreement provides Taysha with up to $100 million of borrowing capacity. The financing is described as a non-dilutive term loan. The facility is structured as debt under a term loan instrument to bolster the company's liquidity. Taysha Gene Therapies is a patient-centric company developing AAV-based gene therapies to treat monogenic CNS diseases in both rare and larger patient populations. It was founded in partnership with the UT Southwestern Gene Therapy Program and has assembled a pipeline of 17 product candidates with exclusive options to acquire four additional programs. Lead programs include TSHA-101 for GM2 Gangliosidosis, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder and TSHA-104 for SURF1 deficiency, with TSHA-101 expected to enter clinical studies later this year. Taysha expects to file INDs for these four candidates by the end of 2021. The company plans to use financing proceeds to advance initial cohorts into the clinic, accelerate IND submissions and build a commercially scalable GMP manufacturing facility. Taysha emphasizes rapid translation of preclinical programs into the clinic to develop potentially curative therapies for CNS disease. Taysha Gene Therapies was launched to eradicate severe and life-threatening monogenic CNS diseases by advancing a pipeline of AAV gene therapies. The company currently has 15 AAV programs with options to an additional four programs; TGTX-101, a gene replacement for GM2-Gangliosidosis, is expected to enter clinical development later this year. Taysha plans to file four IND applications by the end of 2021, including indications for SURF1 deficiency, SLC6A1 haploinsufficiency and Rett syndrome. It has a strategic partnership with the UT Southwestern Gene Therapy Program, which will conduct discovery, preclinical research, IND-enabling studies, clinical GMP manufacturing and natural history studies. Taysha will lead clinical development, regulatory strategy, commercial manufacturing and commercialization. The company is developing a novel AAV capsid platform using machine learning, DNA shuffling and directed evolution, as well as an AAV redosing platform that targets the vagus nerve. Taysha launched with $30 million in seed financing and brings together former AveXis investors and executives alongside UT Southwestern expertise.

Total raised

$275M

Funding rounds

4

Latest round

Equity

Latest activity

Oct 2022