Regeneron Forges $275M Gene Editing Partnership with Tessera Therapeutics

Regeneron Pharmaceuticals has announced a significant collaboration with Tessera Therapeutics, marking a substantial investment in gene editing technology for the treatment of alpha-1 antitrypsin deficiency (AATD). The partnership, revealed on Monday, centers around Tessera's innovative in vivo gene editor TSRA-196, which has shown promising results in preclinical studies.

Deal Structure and Financial Terms

The collaboration involves a $150 million upfront payment from Regeneron, comprising cash and equity investment. Additionally, Tessera stands to receive up to $125 million in near- and mid-term development milestones. Both companies have agreed to share worldwide development costs and future profits equally, underscoring the strategic importance of this partnership.

TSRA-196: A Potential Breakthrough for AATD

At the core of this collaboration is TSRA-196, Tessera's lead gene editor designed to address AATD. This genetic condition is linked to mutations in the SERPINA1 locus, which TSRA-196 aims to correct precisely. The therapy is intended to be a one-time treatment that restores the production of functional alpha-1 antitrypsin, a crucial protein that protects the lungs from autoimmune damage.

Preclinical data presented 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 studies also reported rare unintended edits and no detected genetic changes in germline tissues, along with a favorable safety and tolerability profile.

Development Timeline and Responsibilities

Tessera will spearhead the first-in-human trial for TSRA-196, with plans to file an investigational new drug (IND) application before the end of the year. Following this initial phase, Regeneron will take the reins for subsequent global development efforts within the collaboration.

This partnership adds to Regeneron's expanding genetic medicine portfolio, which includes other notable projects such as DB-OTO, a gene therapy for congenital hearing loss that recently showed promising results in a Phase I/II study. The company's strategic focus on genetic editing is further evidenced by its April 2024 collaboration with Mammoth Biosciences and ongoing partnership with Intellia Therapeutics, both centered on CRISPR-based gene editing technologies.