Regeneron Inks $275M Gene Editing Deal with Tessera Therapeutics for Alpha-1 Antitrypsin Deficiency Treatment

Regeneron Pharmaceuticals has announced a significant partnership with gene editing startup Tessera Therapeutics, marking a major investment in the development of novel treatments for alpha-1 antitrypsin deficiency (AATD). The collaboration, centered on Tessera's innovative gene writing technology, represents a $275 million commitment from Regeneron and underscores the growing interest in genetic medicine approaches to rare diseases.

Deal Structure and Financial Terms

Under the terms of the agreement, Regeneron will make an upfront payment of $150 million to Tessera, comprising cash and an equity investment. The deal also includes the potential for an additional $125 million in near- and mid-term development milestone payments. Both companies will equally share worldwide development costs and future profits resulting from the collaboration.

The centerpiece of this partnership is TSRA-196, Tessera's lead gene editor designed to correct the genetic mutation underlying AATD. This experimental treatment aims to restore the production of functional alpha-1 antitrypsin (AAT), a protein crucial for protecting the lungs from autoimmune damage.

TSRA-196: A Promising Approach to AATD Treatment

TSRA-196 represents a potentially groundbreaking approach to treating AATD, a rare genetic condition that can lead to serious lung and liver diseases. The therapy is designed as a one-time treatment that precisely targets and corrects mutations in the SERPINA1 gene, which is responsible for producing AAT.

Preclinical data presented by Tessera at the 28th annual conference of the American Society of Gene and Cell Therapy earlier this year demonstrated "robust levels of genome editing" in non-human primates. The company reported rare unintended edits and no detected genetic changes in germline tissues, along with a favorable safety and tolerability profile.

George Yancopoulos, Regeneron's chief scientific officer, emphasized the potential of this approach, stating, "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."

Development Timeline and Future Prospects

Tessera will lead the initial first-in-human trial for TSRA-196, with Regeneron taking over subsequent global development and potential commercialization efforts. The companies expect to file an investigational new drug (IND) application for TSRA-196 before the end of 2025, marking a significant step towards clinical trials.

This collaboration aligns with Regeneron's growing focus on genetic medicine. The company has been actively expanding its portfolio in this area through partnerships with various biotechnology firms, including Intellia Therapeutics, Decibel Therapeutics, and Mammoth Biosciences. Notably, Regeneron is also advancing DB-OTO, a gene therapy for congenital hearing loss that has shown promising results in recent clinical trials.