The centerpiece of the deal is the in vivo editor TSRA-196, which in preclinical studies has shown robust editing at SERPINA1, the locus linked to alpha-1 antitrypsin deficiency.
Regeneron has forged a global collaboration with Tessera Therapeutics, looking to advance an in vivo gene editor for alpha-1 antitrypsin deficiency.
The partnership, announced Monday, will involve a $150 million upfront cash payment and equity investment from the pharma, as well as up to $125 million in near- and mid-term development milestones. The companies will split worldwide development costs and future profits born from the collaboration equally.
At the heart of the agreement is TSRA-196, Tessera’s lead gene editor that the company claims can “precisely correct the genetic mutation underlying AATD.” TSRA-196 is designed to be a one-time therapy that restores the production of functional alpha-1 antitrypsin, a protein that under healthy conditions protects the lungs from autoimmune damage.
At the 28th annual conference of the American Society of Gene and Cell Therapy in May this year, Tessera presented pre-clinical data for TSRA-196, touting “robust levels of genome editing” in non-human primates. Unintended edits were rare, and genetic changes were not detected in germline tissues. The therapy had a “favorable safety and tolerability” profile, the company said.
Tessera will take charge of the gene editor’s first-in-human trial, according to Monday’s release, after which Regeneron will assume responsibility for subsequent global development and collaboration. The companies expect to file an investigational new drug application for TSRA-196 before the end of the year.
With the Tessera partnership, Regeneron continues to expand its genetic toolbox. The pharma is currently advancing DB-OTO, a gene therapy for congenital hearing loss that in October elicited “clinically meaningful” hearing improvements in 11 of 12 patients treated in a Phase I/II study. Regeneron at the time said it was building up to an approval application before the year’s end.
In the editing space specifically, Regeneron in April 2024 joined hands with Mammoth Biosciences—paying $100 million upfront and promising up to $370 million in milestones—to leverage the biotech’s “ultracompact” CRISPR editing system, targeting several tissues and cell types. Regeneron is also partnered with Intellia Therapeutics on a similar CRISPR-based gene editing approach to neurological and muscular diseases.
