Regeneron Pharmaceuticals and Tessera Therapeutics have entered a global collaboration to jointly develop and commercialize TSRA-196, an investigational gene editing therapy designed as a one-time treatment for alpha-1 antitrypsin deficiency (AATD).
Under the agreement, the companies will split worldwide development costs and future profits for TSRA-196 equally. Tessera will receive $150 million from Regeneron, comprising an upfront cash payment and an equity investment.
Tessera is also eligible for up to $125 million in additional development milestone payments. The company will lead the initial human trial, while Regeneron will helm subsequent global development and commercialization efforts.
The Science Behind TSRA-196
TSRA-196 utilizes Tessera’s proprietary “Gene Writing” platform and non-viral delivery technology. It aims to precisely correct the mutation in the SERPINA1 gene that causes AATD, potentially restoring the body’s production of functional alpha-1 antitrypsin protein. Preclinical data presented earlier this year showed durable genome editing in animal models with high specificity for the liver and a favorable safety profile.
An Investigational New Drug application to initiate clinical trials is anticipated to be submitted with the US Food and Drug Administration (FDA) by the end of the year.
AATD is an inherited disorder that can lead to severe lung and liver disease, affecting an estimated 200,000 people in the US and Europe. Current treatment is primarily limited to weekly intravenous protein augmentation for lung symptoms, with no approved therapies that address the underlying genetic cause. “Alpha-1 antitrypsin deficiency is a serious disease with limited treatment options today,” said George D. Yancopoulos, Board co-chair, President and Chief Scientific Officer of Regeneron.
Regeneron’s Push for Gene Therapies
This collaboration between Regeneron and Tessera arrives amid intensifying competition and significant investment in the gene therapy and editing sector, as major pharmaceutical firms seek to secure next-generation technological platforms.
Regeneron has been actively expanding its genetic medicine footprint through partnerships, most notably a longstanding, multi-product alliance with Intellia Therapeutics utilizing CRISPR/Cas9 technology. In 2023, it acquired Decibel Therapeutics in a deal worth $213 million for gene therapy expertise in hearing disorders.
Recent deals and funding in the gene therapy space include AAVantgarde Bio closing a $141 million (€122 million) Series B financing round. The funds will be used to accelerate the company’s gene therapy clinical programs targeting Stargardt disease and Usher syndrome type 1B, both inherited retinal diseases (IRDs). Last month, Danish biopharmaceutical BOOST Pharma announced a SEK 34 million ($3.55 million) investment from Sound Bioventures. The funding forms part of a seed round extension and will support continued development of the company’s stem cell-based therapy, BT-101.
