Regeneron and Tessera Therapeutics have entered into a global collaboration agreement to develop and commercialize TSRA-196, Tessera’s lead investigational in vivo (in-body) gene writing program designed for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). About Alpha-1 Antitrypsin Deficiency (AATD): AATD is a rare inherited monogenic disease that affects the lungs, liver, or both organs, impacting an estimated 200,000 people in the U.S. and Europe. TSRA-196 was engineered to precisely correct the genetic mutationunderlying AATD, aiming to restore the production of functional alpha-1 antitrypsin (AAT) protein through a one-time, durable treatment option. Regeneron’s representative highlighted that AATD is a serious disease with limited current treatment options, making it particularly well suited for Tessera’s gene editing approach. Financial and Development Collaboration Terms: Upfront Payment: Tessera is set to receive $150 million, inclusive of a cash upfront payment and an equity investment from Regeneron. Milestone Payments: Tessera will also be eligible to receive an additional $125 million in near and mid-term development milestone payments. Development Responsibilities: Tessera is expected to lead the initial first-in-human clinical trials. Regeneron is slated to lead subsequent global development and commercialization. Both companies will equally share worldwide development costs and potential future earnings related to TSRA-196. Encouraging Preclinical Data: The collaboration comes as TSRA-196 advances, with preclinical data presented at the American Society of Gene & Cell Therapy 28th Annual Meeting reinforcing the therapy's potential to correct the underlying genetic cause of AATD. The preclinical findings highlighted durable, high-fidelity genome editing of the Serpina1 locus (responsible for AATD) in both mice and non-human primates after a single dose of TSRA-196 administration. Results also showed high liver editing specificity, no germline or off-target editing, and a favorable safety and tolerability profile using Tessera’s proprietary lipid nanoparticle delivery vehicle. Source: https://www.pharmexec.com/view/regeneron-announces-150-million-collaboration-tessera-therapeutics-gene-editing-therapy
