Regeneron and Tessera Therapeutics to Jointly Develop TSRA-196, an Investigational Gene Editing Therapy for Alpha-1 Antitrypsin Deficiency (AATD)
Tuesday, December 02, 2025
Regeneron Pharmaceuticals and Tessera Therapeutics have entered into a global collaboration to develop and commercialise TSRA-196, Tessera’s lead in vivo Gene Writing therapy for alpha-1 antitrypsin deficiency (AATD). AATD is an inherited monogenic condition affecting the lungs, liver, or both, and impacts an estimated 200,000 people across the U.S. and Europe. TSRA-196 aims to correct the underlying genetic mutation to restore production of functional alpha-1 antitrypsin (AAT) through a one-time, long-lasting treatment. Tessera plans to submit an Investigational New Drug application and several Clinical Trial Applications to the U.S. FDA by the end of the year.
The partnership combines Regeneron’s expertise in genetic research and development of genetic medicines with Tessera’s Gene Writing technology and non-viral delivery systems. Both companies will share global development costs and potential future profits equally. Tessera will receive $150 million through an upfront cash payment and an equity investment, and may receive up to $125 million in additional development milestone payments. Tessera will oversee the first-in-human trial, while Regeneron will lead the subsequent global development and commercial activities.
Recent preclinical progress supports the potential of TSRA-196. Data presented at the American Society of Gene & Cell Therapy Annual Meeting showed durable and accurate editing of SERPINA1—the gene responsible for AATD—in mice and non-human primates after a single dose. The results also indicated strong liver-targeted editing, no detectable germline or off-target editing, and a favourable safety profile using Tessera’s lipid nanoparticle delivery technology. These findings reinforce the therapy’s suitability for clinical development.
The collaboration agreement is subject to standard closing conditions, including regulatory clearances under the Hart-Scott-Rodino Antitrust Improvements Act in the United States.
Source: globenewswire.com
