Somerville, Massachusetts — Regeneron Pharmaceuticals and Tessera Therapeutics have entered into a global partnership to develop and commercialize TSRA-196, an investigational in vivo gene editing therapy designed to correct the genetic mutation that causes alpha-1 antitrypsin deficiency (AATD). The rare inherited condition affects roughly 200,000 people across the U.S. and Europe and can lead to lung disease, liver disease, or both.
TSRA-196, Tessera’s lead Gene Writing program, is intended as a one-time treatment aimed at restoring production of functional alpha-1 antitrypsin protein. Tessera plans to file an Investigational New Drug application and multiple Clinical Trial Applications with the U.S. Food and Drug Administration by year-end.
Under the collaboration, the companies will share global development costs and any eventual profits on a 50:50 basis. Tessera will receive $150 million in a combination of upfront cash and an equity investment from Regeneron, and may receive up to $125 million in additional near- and mid-term development milestone payments. Tessera will oversee the first-in-human study, while Regeneron will take the lead on later-stage clinical development and global commercialization.
“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.”
“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.”
Recent preclinical studies presented at the American Society of Gene & Cell Therapy’s annual meeting showed durable, high-fidelity genome editing of SERPINA1 — the gene responsible for AATD — in mice and non-human primates after a single dose. The research highlighted strong liver-targeting specificity, no detectable germline or off-target editing, and favorable safety using Tessera’s lipid nanoparticle delivery technology. These data support advancing TSRA-196 into clinical development.
The agreement is subject to customary closing conditions, including regulatory clearance under the Hart-Scott-Rodino Antitrust Improvements Act.
