Taysha will collaborate with
TSHA-120 is the first and only gene therapy to demonstrate clinical-stage arrest of disease progression at the 1.2x1014 and 1.8x1014 total vg dose
Clinical data for high dose 3.5x1014 total vg cohort expected in the second half of 2021 with regulatory feedback by year-end 2021
Estimated prevalence of patients with GAN is 2,400 in
GAN is a progressive neurodegenerative disease that affects both the central and peripheral nervous systems leading to motor weakness, sensory impairment, and cognitive dysfunction. Currently, there are no approved treatments for GAN, which results in death for patients often in their late teens or early twenties. Although no symptoms are present in the first few months of life, many children with early onset GAN develop symptoms and features before the age of three. A more recently identified later onset phenotype of GAN is often mischaracterized as Charcot-Marie-Tooth. The estimated prevalence for GAN is 2,400 patients, but the GAN population is anticipated to be larger than previously appreciated.
In the natural history study, Motor Function Measure 32 (MFM32), a validated and well-known scale to measure strength and motor function, is the primary endpoint. A four-point change on the MFM32 score is considered clinically meaningful, which has been validated across numerous similar neuropathies. Natural history decline in patients with GAN is eight points per year. Treatment with TSHA-120, Taysha’s intrathecally dosed AAV9 gene therapy currently being evaluated in a Phase 1/2 clinical trial led by Carsten Bönnemann, M.D., Chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section and Senior Investigator, Neurogenetics Branch of the
“Rare diseases like GAN are often mischaracterized, particularly when there are overlapping symptoms with other diseases with higher awareness,” said
Taysha will sponsor the new initiative which will make genetic testing available free of charge to individuals at risk for or suspected of having GAN. The initiative is designed to increase access to genetic testing for patients.
As the later onset phenotype of GAN is sometimes miscategorized as Charcot-Marie-Tooth or Charcot-Marie-Tooth Type 2, Taysha plans to collaborate with the
“Through this initiative, patients will have access to no-charge genetic testing, eliminating one of the biggest barriers,” said
All testing will be ordered through a clinician. Sponsored testing programs have previously been shown to increase access to genetic testing, confirm diagnosis and enable participation in clinical trials for patients.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-120, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, TSHA-120’s eligibility for accelerated approval in
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