Regeneron Pharmaceuticals and Tessera Therapeutics have entered into a major global collaboration to advance TSRA-196, an investigational in vivo Gene Writing therapy designed to correct the genetic mutation underlying alpha-1 antitrypsin deficiency (AATD). The partnership positions the two biotechnology companies at the forefront of next-generation genetic medicine as they work toward a potentially one-time, curative treatment for a disease that affects an estimated 200,000 people in the United States and Europe.
AATD is a hereditary monogenic disorder that can damage the lungs, liver, or both. The disease stems from mutations in the SERPINA1 gene, which impair the body’s ability to produce functional alpha-1 antitrypsin (AAT) protein. Current treatment options are limited, often requiring chronic protein replacement therapy or management of organ damage. TSRA-196 seeks to directly address the source of the condition by precisely rewriting the faulty gene and restoring normal AAT protein production.
Tessera plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration and multiple Clinical Trial Applications in global markets by the end of the year. These filings would clear the way for first-in-human studies, signaling a critical milestone for the company’s Gene Writing platform.
The agreement leverages Regeneron’s extensive experience in human genetics and genetic medicine development and Tessera’s pioneering Gene Writing technology, along with its proprietary non-viral lipid nanoparticle delivery system. Under the terms of the collaboration, both companies will share worldwide development costs and future potential profits from TSRA-196 equally. Tessera will receive $150 million upfront, comprising a cash payment and an equity investment, and is eligible for an additional $125 million in near- and mid-term development milestone payments.
Tessera will lead the initial clinical trial, while Regeneron will assume responsibility for later-stage global development and commercialization.
George D. Yancopoulos, Regeneron’s Board co-Chair, President, and Chief Scientific Officer, described AATD as a disease for which existing therapies remain inadequate. “Alpha-1 antitrypsin deficiency is particularly well suited for Tessera’s gene editing approach,” he said, noting Regeneron’s commitment to advancing innovative genetic medicines. “Together with Tessera, we have an opportunity to pioneer new frontiers in genetic medicine and redefine what is possible for AATD patients.”
Tessera CEO Michael Severino emphasized the transformative potential of a single-administration, intravenously delivered therapeutic. “This collaboration underscores what we believe is a medically and commercially important opportunity to deliver transformative outcomes for patients living with alpha-1 antitrypsin deficiency,” he said. “We are on the cusp of a critical inflection point as we prepare to enter the clinic.”
The scientific promise of TSRA-196 is supported by recent preclinical results presented at the American Society of Gene & Cell Therapy Annual Meeting, where Tessera reported durable, high-fidelity genome editing in mice and non-human primates. The data showed precise editing of SERPINA1 with no germline or off-target effects, strong liver specificity, and favorable safety, all achieved with a single dose delivered through Tessera’s lipid nanoparticle platform.
These findings underscore the therapy’s potential to correct the root cause of AATD rather than simply treating symptoms—a significant ambition in the gene-editing field.
The collaboration remains subject to customary closing conditions, including regulatory clearances under the Hart-Scott-Rodino Antitrust Improvements Act. If approved, the partnership could mark a pivotal step toward bringing a durable gene-correcting therapy to AATD patients worldwide.