A global collaboration focused on the development and commercialization of TSRA-196, Tessera’s lead investigational in vivo Gene Writing program for Alpha-1 Antitrypsin Deficiency (AATD), was announced by Regeneron Pharmaceuticals& Tessera Therapeutics.

AATD is a serious inherited monogenic disease, affecting an estimated 200,000 individuals in the U.S. and Europe, caused by mutations in the SERPINA1 gene that result in both lung damage from protein deficiency and toxic protein accumulation in the liver.

TSRA-196 is designed as a potential one-time, durable treatment intended to precisely correct the genetic mutation to restore the production of functional alpha-1 antitrypsin (AAT) protein, a significant step beyond current augmentation therapies that do not address the genetic root of the condition.

This partnership strategically combines Regeneron’s established expertise in genetics, genetic medicines, and clinical development with Tessera’s pioneering Gene Writing and proprietary non-viral delivery platforms. Financially, Tessera will receive $150 million, which includes an upfront cash payment and an equity investment from Regeneron, and is eligible for an additional $125 million in near and mid-term development milestone payments. Furthermore, the two companies have agreed to share all worldwide development costs and future profits equally (50:50). Building on preclinical data that showed durable, high-fidelity genome editing of the target SERPINA1 locus with favorable safety and no off-target editing in non-human primates, an Investigational New Drug (IND) filing with the U.S. FDA is expected by the end of the year. As part of the development plan, Tessera will lead the initial first-in-human trial, while Regeneron will assume responsibility for subsequent global development and commercialization efforts.