Regeneron Enters Collaboration with Tessera for AATD Gene Editing Therapy

Rare Daily Staff

Regeneron Pharmaceuticals has entered into a global collaboration with Tessera Therapeutics to develop and commercialize TSRA-196, Tessera’s lead experimental in vivo gene writing program for the treatment of alpha-1 antitrypsin deficiency, a rare genetic disease that can affect the lungs and liver.

Under the terms of the agreement, the companies will share worldwide development costs and potential future profits related 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.

Alpha-1 antitrypsin deficiency (AATD) is most often caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a protein produced in the liver and secreted into the bloodstream to protect lung tissue from enzymes such as neutrophil elastase. In individuals with severe AATD, mutations in the Z allele cause AAT protein to misfold and accumulate in the liver, leading to toxic effects such as inflammation and fibrosis. At the same time, insufficient circulating AAT leaves the lungs vulnerable to progressive damage consistent with chronic obstructive pulmonary disease and emphysema.

There are currently no FDA-approved therapies that address the underlying genetic cause of AATD, and treatment options remain limited to weekly intravenous augmentation therapy for patients with lung disease.

TSRA-196 is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional AAT protein through a one-time, durable treatment option. Tessera expects to file an investigational new drug application and multiple clinical trial applications for TSRA-196 with the U.S. Food and Drug Administration by the end of the year.

The collaboration brings together Regeneron’s industry-leading capabilities in genetics and proven track record in advancing novel genetic medicines with Tessera’s innovative gene writing technology and proprietary nonviral delivery platforms.

“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, CEO of Tessera. “Tessera is on the cusp of a critical inflection point as we prepare to enter the clinic in the near term.”

Photo: Michael Severino, CEO of Tessera