Taysha Gene Therapies Stock Is Trading Higher After Buying Rights To New Gene Therapy Candidate For Neurodegenerative Disease

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  • Taysha Gene Therapies Inc TSHA has unveiled a deal for global rights to the AAV9 gene therapy program, now known as TSHA-120, to treat giant axonal neuropathy (GAN)
  • Under the terms of the agreement, the GAN patient advocacy group will receive an upfront payment of $5.5 million. It will be eligible to receive up to $19.3 million in milestone payments, as well as sales-based royalty.
  • GAN is a rare inherited genetic disorder that affects both the central and peripheral nervous systems, caused by mutations in the gene coding for gigaxonin that controls protein degradation and is essential for neuronal function and survival. 
  • Typical symptoms of GAN include clumsiness and muscle weakness. 
  • TSHA-120 has the potential to address a significant unmet need for an estimated 2,400 patients with GAN across the U.S. and in Europe, potentially representing a near-term commercial opportunity of greater than $2 billion." said RA Session II, President, Founder, and CEO of Taysha.
  • So far, 14 patients have been dosed with the AAV9 candidate in an open-label dose-escalation study by the NIH and the patient advocacy group.
  • Researchers observed a halt in disease progression at the second-highest dose level one year post-treatment, and six patients have shown dose-dependent improvements for more than three years, according to Taysha.
  • Additional data are expected later this year, including results from the highest dose cohort.
  • Price Action: TSHA shares increased 17.8% at $24.96 during the market trading session on the last check Monday.
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Posted In: BiotechNewsHealth CareSmall CapFDAGeneralBriefsgene therapyneurodegenerative disease
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