Regeneron Partners with Tessera in $275M Gene Editing Deal for Alpha-1 Antitrypsin Deficiency Treatment
核心洞察
Regeneron Pharmaceuticals has entered a global collaboration with Tessera Therapeutics (搜索) worth up to $275 million to develop TSRA-196 (搜索), an in vivo gene editor for alpha-1 antitrypsin deficiency (搜索).
The partnership includes $150 million upfront in cash and equity investment, plus up to $125 million in development milestones, with equal sharing of worldwide development costs and future profits.
TSRA-196 (搜索) is designed as a one-time therapy that precisely corrects the genetic mutation underlying AATD and restores functional alpha-1 antitrypsin protein production.
Regeneron Pharmaceuticals has announced a major gene editing collaboration with Tessera Therapeutics (搜索), committing up to $275 million to advance TSRA-196 (搜索), an investigational in vivo gene editor targeting alpha-1 antitrypsin deficiency (搜索) (AATD). The partnership represents Regeneron's continued expansion into genetic medicine technologies.
Partnership Structure and Financial Terms
Under the global collaboration announced Monday, Regeneron will provide $150 million upfront through a combination of cash payment and equity investment in Tessera. The biotech company is eligible to receive an additional $125 million in near- and mid-term development milestone payments. The companies will equally share worldwide development costs and split future profits from TSRA-196 (搜索).
Tessera will lead the initial first-in-human trial for TSRA-196 (搜索), after which Regeneron will assume responsibility for subsequent global development and commercialization activities. The companies expect to file an investigational new drug application with the FDA before the end of the year.
TSRA-196 Gene Editor Technology
TSRA-196 (搜索) represents Tessera's lead Gene Writing program, designed to "precisely correct the genetic mutation underlying AATD." The therapy is engineered as a one-time treatment that restores production of functional alpha-1 antitrypsin, a protein that normally protects the lungs from autoimmune damage under healthy conditions.
At the 28th annual conference of the American Society of Gene and Cell Therapy in May, Tessera presented preclinical data demonstrating "robust levels of genome editing" in non-human primates. The study results showed that unintended edits were rare, and genetic changes were not detected in germline tissues. According to the company, TSRA-196 (搜索) exhibited a "favorable safety and tolerability" profile in these preclinical studies.
Regeneron's Expanding Genetic Medicine Portfolio
The Tessera partnership adds to Regeneron's growing genetic toolbox. The pharmaceutical company is currently advancing DB-OTO, a gene therapy for congenital hearing loss (搜索) that demonstrated promising clinical results in October. In a Phase I/II study, DB-OTO produced "clinically meaningful" hearing improvements in 11 of 12 patients treated. Regeneron indicated at the time that it was preparing for an approval application before the end of the year.
The collaboration with Tessera positions Regeneron to potentially address alpha-1 antitrypsin deficiency (搜索) through a novel gene editing approach, expanding the company's capabilities in treating genetic disorders through direct genetic correction rather than traditional protein replacement therapies.