Regeneron Pharmaceuticals has entered into a global partnership with Tessera Therapeutics to develop an in vivo gene editing therapy for alpha-1 antitrypsin deficiency (AATD). The agreement includes a $150 million upfront payment and equity investment from Regeneron, as well as up to $125 million in potential development milestone payments. Both companies will share worldwide development costs and future profits equally.
Central to the collaboration is TSRA-196, Tessera’s lead gene editor. According to Tessera, TSRA-196 can “precisely correct the genetic mutation underlying AATD.” The therapy is intended as a one-time treatment that restores the production of functional alpha-1 antitrypsin, a protein important for protecting lung tissue.
At the 28th annual conference of the American Society of Gene and Cell Therapy in May, Tessera presented pre-clinical data on TSRA-196. The company reported “robust levels of genome editing” in non-human primates. Unintended edits were rare, and no genetic changes were found in germline tissues. The therapy showed a “favorable safety and tolerability” profile, according to Tessera.
Tessera will lead the first-in-human trial for TSRA-196. After this stage, Regeneron will take over responsibility for further global development. The companies plan to file an investigational new drug application before the end of the year.
This partnership adds to Regeneron's ongoing efforts in genetic therapies. In October, Regeneron reported positive results from its DB-OTO gene therapy for congenital hearing loss, with most patients showing improvement in early trials. The company indicated plans to seek regulatory approval by year-end.
Regeneron has also expanded its gene editing capabilities through other collaborations. In April 2024, it partnered with Mammoth Biosciences on CRISPR-based technology targeting multiple tissues and cell types, committing $100 million upfront and additional milestone payments. Another partnership with Intellia Therapeutics focuses on CRISPR-based approaches for neurological and muscular diseases.
