Regeneron And Tessera Therapeutics Establish Global Collaboration Accelerate Development Of TSRA-196, A One-Time Gene Correction Therapy For Alpha-1 Antitrypsin Deficiency

Regeneron Pharmaceuticals and Tessera Therapeutics have entered into a global partnership to develop and commercialise TSRA-196, Tessera’s leading in vivo Gene Writing candidate aimed at treating alpha-1 antitrypsin deficiency (AATD). The rare genetic disorder, affecting roughly 200,000 people across the U.S. and Europe, can lead to serious lung and liver complications. TSRA-196 is designed to directly correct the genetic mutation that causes AATD, potentially enabling the body to resume production of healthy AAT protein through a one-time, long-lasting therapeutic approach. Tessera plans to submit an Investigational New Drug application to the FDA, along with several Clinical Trial Applications, by year-end.

“At Regeneron, we are strong believers in the power of genetics and genetic medicines to transform patients’ lives, and we have a robust portfolio of potential treatments to do just this,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer of Regeneron. “Alpha-1 antitrypsin deficiency is a serious disease with limited treatment options today and is particularly well suited for Tessera’s gene editing approach. Together with Tessera, we have an opportunity to pioneer new frontiers in genetic medicine and redefine what is possible for AATD patients.”

The collaboration combines Regeneron’s extensive experience in genetic research and drug development with Tessera’s cutting-edge Gene Writing technology and proprietary non-viral delivery systems. Both companies will jointly fund global development and share future profits equally. As part of the agreement, Tessera will receive $150 million in upfront and equity-based payments from Regeneron, along with eligibility for up to $125 million in additional milestone payments. Tessera will manage the first human clinical trial, after which Regeneron will take over global development and commercial activities.

“This collaboration underscores what we believe is a medically and commercially important opportunity to deliver transformative outcomes with a one-time, intravenously delivered genetic treatment for patients living with alpha-1 antitrypsin deficiency,” said Michael Severino, M.D., Chief Executive Officer of Tessera Therapeutics. “Tessera is on the cusp of a critical inflection point as we prepare to enter the clinic in the near term. We are excited to partner with Regeneron, a global leader in innovative biotechnology and genetic medicine, to accelerate the development of TSRA-196 and broaden its potential impact to patients in need.”

This partnership follows promising preclinical results for TSRA-196, presented at the ASGCT Annual Meeting, which demonstrated durable and highly accurate gene correction of the SERPINA1 gene in mice and non-human primates after a single dose. The study showed strong liver-targeted editing, no off-target or germline effects, and a favourable safety profile using Tessera’s lipid nanoparticle platform. These data further validate the program’s potential to address the root cause of AATD and advance it toward clinical evaluation.

The agreement remains subject to standard closing requirements, including regulatory clearance under the Hart-Scott-Rodino Antitrust Improvements Act in the United States.