Regeneron, Tessera Therapeutics Partner to Develop & Commercialize TSRA-196

Regeneron Pharmaceuticals Inc. and Tessera Therapeutics Inc. have announced a global collaboration to develop and commercialize TSRA-196, Tessera’s lead investigational in vivo Gene Writing program for the treatment of alpha-1 antitrypsin deficiency (AATD), an inherited monogenic disease that can affect the lungs, liver, or both organs.

TSRA-196 is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional alpha-1 antitrypsin (AAT) protein through a one-time, durable treatment option for patients. Tessera expects to file an Investigational New Drug and multiple Clinical Trial Applications for TSRA-196 with the U.S. Food and Drug Administration (FDA) by the end of the year.

The collaboration brings together Regeneron’s capabilities in genetics and track record in advancing novel genetic medicines with Tessera’s innovative Gene Writing and proprietary non-viral delivery platforms. Under the terms of the agreement, the companies will share worldwide development costs and potential future profits relating to TSRA-196 equally. Tessera will receive $150 million, inclusive of a cash upfront payment and equity investment from Regeneron. Tessera is also eligible to receive additional near and mid-term development milestone payments totaling $125 million. Tessera will lead the initial first-in-human trial, while Regeneron will lead subsequent global development and commercialization.

George D. Yancopoulos, M.D., Ph.D., Board Co-Chair, President and Chief Scientific Officer of Regeneron, said, “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., CEO 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.”