Regeneron and Tessera partner to develop gene therapy for AATD

Regeneron Pharmaceuticals and Tessera Therapeutics are collaborating to advance a promising in vivo gene editing therapy for alpha-1 antitrypsin deficiency (AATD).

The companies will jointly develop and commercialise Tessera Therapeutics’ lead investigational candidate TSRA-196. The drug is designed to restore production of functional alpha-1 antitrypsin (AAT) protein in patients with the inherited monogenic disease.

The collaboration unites Regeneron’s capabilities in genetics and track record in advancing novel genetic medicines with Tessera’s gene writing and proprietary non-viral delivery platforms.

Tessera will receive $150 million from Regeneron and is eligible for $125 million in development milestone payments.

Preclinical data shows TSRA-196 offers durable, high-fidelity genome editing of SERPINA1, the locus responsible for AATD. This backs the treatment’s potential to correct the underlying genetic cause of AATD and advancing it into clinical development.

Dr George Yancopoulos, PhD, 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.”

Advancing a one-time gene editing therapy for AATD

Under the agreement, Tessera will lead the initial first-in-human trial, with Regeneron managing global development and commercialisation; the companies equally sharing related global development costs and potential future profits.

Dr Michael Severino, Chief Executive Officer of Tessera Therapeutics said: “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.

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

This collaboration between Regeneron and Tessera is subject to customary closing conditions.