Regeneron(US:REGN) Globenewswire·2025-12-01 12:00Core Insights - Regeneron Pharmaceuticals and Tessera Therapeutics have announced a global collaboration to develop and commercialize TSRA-196, a gene therapy for alpha-1 antitrypsin deficiency (AATD), which affects approximately 200,000 people in the U.S. and Europe [1][5][6] - TSRA-196 aims to correct the genetic mutation causing AATD, providing a one-time treatment option to restore functional alpha-1 antitrypsin protein production [1][6] - Tessera plans to file an Investigational New Drug application and multiple Clinical Trial Applications with the FDA by the end of the year [1][6] Collaboration Details - The collaboration will involve shared worldwide development costs and potential future profits, with both companies splitting these equally [2] - Tessera will receive $150 million from Regeneron, which includes an upfront cash payment and equity investment, along with potential milestone payments totaling $125 million [2][6] - Tessera will lead the initial first-in-human trial, while Regeneron will oversee subsequent global development and commercialization efforts [2] Scientific Background - AATD is caused by mutations in the SERPINA1 gene, leading to insufficient alpha-1 antitrypsin protein, which protects lung tissue from damage [5][7] - Current treatment options are limited, primarily consisting of weekly intravenous augmentation therapy for patients with lung disease [7] - Preclinical data for TSRA-196 has shown durable and high-fidelity genome editing in animal models, indicating its potential effectiveness and safety [3][6] Company Profiles - Regeneron is a leading biotechnology company focused on developing life-transforming medicines for serious diseases, leveraging its expertise in genetics and clinical development [8][9] - Tessera Therapeutics specializes in genome engineering through its Gene Writing platform, aiming to create transformative genetic medicines [11]