Busy Months Ahead For Taysha Gene

(RTTNews) - Taysha Gene Therapies Inc. (TSHA), a pivotal-stage gene therapy company, has a couple of clinical, regulatory and preclinical milestones lined up for the remainder of this year.

The company has 26 AAV-based gene therapies under development for the treatment of monogenic diseases of the central nervous system, including neurodegenerative diseases, neurodevelopmental disorders, and genetic epilepsies. Monogenic disorders are caused by variation in a single gene.

The most advanced program in the pipeline is TSHA-120, an *intrathecally dosed AAV9 gene therapy, currently being evaluated in a phase I/II clinical trial for the treatment of giant axonal neuropathy (GAN). (*Intrathecal delivery refers to injection of the drug into the cerebrospinal fluid within the intrathecal space of the spinal column).

Giant axonal neuropathy is a rare and severe neurodegenerative disease. Currently, there are no approved treatments for GAN, which often results in death for patients in their late teens or early twenties. The clinical trial of TSHA-120 is being conducted by the National Institutes of Health (NIH) in close collaboration with a patient advocacy group focused on finding treatments and cures for GAN.

Human proof-of-concept data for TSHA-120 has demonstrated clear arrest of disease progression and long-term durability at therapeutic dose levels in patients with giant axonal neuropathy, according to the company.

The company expects to report clinical data from a high dose cohort of TSHA-120 in the GAN trial in the second half of this year and provide a regulatory update by year-end.

Another clinical program is TSHA-101, which is under an investigator-sponsored phase I/II trial for the treatment of infantile GM2 gangliosidosis. This trial is being conducted by Queen's University in Ontario, Canada.

Preliminary safety and biomarker data in the phase I/II trial for TSHA-101 are expected in the second half of this year. The company also expects to initiate a phase I/II clinical trial of TSHA-101 in the U.S. in the second half of 2021.

Next in the pipeline is TSHA-118 to potentially treat CLN1, a rapidly progressing rare lysosomal storage disease with no approved treatments.

The company expects to initiate a phase I/II clinical trial of TSHA-118 in the second half of this year. TSHA-118 has been granted orphan drug designation, rare pediatric disease designation and fast track designation from the FDA and orphan medicinal product designation from the EMA for the treatment of CLN1 disease.

In addition to the above, Taysha has a number of programs in preclinical stage, i.e., TSHA-102 in Rett syndrome, TSHA-104 in SURF1-associated Leigh syndrome, TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN and TSHA-111-MALIN in two forms of Lafora disease, TSHA-112 in APBD, TSHA-119 in GM2 AB variant and TSHA-103 in SLC6A1 haploinsufficiency disorder.

Cash position:

As of June 30, 2021, Taysha had $197.4 million in cash and cash equivalents.

The company made its debut on the Nasdaq Global Select Market on September 24, 2020, offering its shares at a price of $20 each. Taysha Gene will mark one year as a public company tomorrow (Sep.24, 2021).

The stock has thus far hit a low of $15.50 and a high of $33.35. TSHA closed Wednesday's trading at $18.98, up 2.54%.