Regeneron has entered into a global collaboration with Tessera Therapeutics to jointly develop and commercialise TSRA-196, a gene editing therapy for alpha-1 antitrypsin deficiency (AATD). The partnership leverages Regeneron's expertise in genetics and clinical development alongside Tessera's pioneering Gene Writing technology platform, aiming to deliver a potential one-time curative treatment for this rare genetic disorder. Under the agreement, Regeneron will pay Tessera USD 150 million in an upfront payment and equity investment. The companies will share global development costs and future profits equally, with Tessera also eligible for up to USD 125 million in near to mid-term development milestone payments. Tessera will lead the first-in-human clinical trial, while Regeneron will oversee subsequent global clinical development and commercialisation.

AATD is a monogenic disorder caused by mutations in the SERPINA1 gene, leading to insufficient functional alpha-1 antitrypsin protein and progressive damage to the lungs and liver. Current treatment is limited to weekly intravenous augmentation therapy, which does not address the underlying cause. TSRA-196 is designed to correct the causative gene mutation, enabling the body to continuously produce functional AAT protein. Preclinical data presented in May 2025 demonstrated durable and precise gene editing in animal models with a favourable safety profile. Tessera plans to submit an Investigational New Drug (IND) application to US regulators by the end of 2025.

PharmCube's NextBiopharm® database shows that the deal marks the 2025 recovery of deal-making activity in the gene editing sector. Click here to request a free trial for NextBiopharm®.