Regeneron announced that it has entered into a global collaboration agreement with Tessera Therapeutics to commercialize and develop TSRA-196, Tessera’s lead investigational in vivo gene writing program for the treatment of AATD.

Along with the collaboration agreement, Tessera is expected to file an Investigational New Drug Application, along with several Clinical Trial Applications for TSRA-196 to the FDA by the end of the year.¹

What is Alpha-1 Antirypsn Deficiency?

AATD, an inherited monogenic disease affecting the lungs, liver, or both organs, impacts an estimated 200,000 people in both the U.S. and Europe.¹

TSRA-196 was designed to precisely correct the genetic mutation underlying AATD, aiming to restore production of functional alpha-1 antitrypsin (AAT) protein through a one-time, durable treatment option for patients.¹

“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.”

What are the terms of the collaboration agreement?

Per the terms of the agreement, Tessera is expected to lead initial first-in-human trials, while Regeneron is expected to lead subsequent global development and commercialization. Both companies are set to equally share worldwide development costs and potential future earnings relating to TSRA-196.¹ Additionally, Tessera is set to receive $150 million, inclusive of a cash upfront payment and equity investment from Regeneron and will also be eligible to receive additional near and mid-term development milestone payments valued at a total of $125 million.¹

The collaboration agreement expands Tessera’s recent progress advancing TSRA-196 which includes preclinical data presented at the American Society of Gene & Cell Therapy 28th Annual Meeting, reinforcing TSRA-196’s potential to correct the underlying genetic cause of AATD along with supporting its case for clinical trials.¹

The presentation highlighted durable, high-fidelity genome editing of Serpina1, the locus responsible for AATD, in both mice and non-human primates after administration of a single dose of TSRA-196, with high liver editing specificity, no germline or off-target editing, and favorable safety and tolerability using Tessera’s proprietary lipid nanoparticle delivery vehicle.¹

“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.”

Sources

1. Regeneron and Tessera Therapeutics to Jointly Develop TSRA-196, an Investigational Gene Editing Therapy for Alpha-1 Antitrypsin Deficiency (AATD) Regeneron December 1, 2025 https://investor.regeneron.com/news-releases/news-release-details/regeneron-and-tessera-therapeutics-jointly-develop-tsra-196