Taysha Gene Therapies has obtained global rights to TSHA-120, an intrathecally dosed AAV9 gene therapy designed to treat giant axonal neuropathy. A patientthe advocacy group will receive an upfront $5.5 million payment from Taysha in exchange for Taysha exclusive worldwide rights to TSHA-120.The patient advocacy group will also be eligible to receive additional clinical, regulatory and commercial milestones ringing in at up to $19.3 million. The terms of the agreement state the group will also be eligible to receive low, single-digit royalties on net sales following the product’s commercialization.
Giant axonal neuropathy is a rare genetic disorder caused by loss-of-function mutations in the gene coding for gigatons. The disorder affects the central and peripheral nervous systems, and symptoms related to these effects typically show before the age of five. Nearly 2,400 people in the United States and Europe have this disease. Common symptoms and features of the disease include contractures, progressive scoliosis, spinal cord atrophy as well as white brain matter abnormalities. The disorder usually leads to death during patients’ late teens or early twenties.
Taysha Chief Executive Officer, President and Founder R.A. Session II said that the chief scientific advisor works on the GAN program was the catalyst for all the other translational research initiatives in his lab and they are pleased to continue this important and meaningful work that has had a considerable impact across the entire gene therapy landscape.
The National Institute of Health ongoing open-label, dose-escalation trial of TSHA-120 is evaluating the therapy’s safety. A secondary endpoint includes efficacy, as assessed by different pathologic, physiologic, functional as well as clinical markers. The Motor Function Measure 32 score is of particular importance regarding the trial’s efficacy assessment, as the MFM32 score helps researchers evaluate the severity and progression of patients’ motor function abilities. Currently, 14 patients have received one of the four TSHA-120 dose levels.
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