Pune, India | December 02, 2025
Regeneron Pharmaceuticals and Tessera Therapeutics have announced a strategic collaboration to develop TSRA‑196. A one-time gene-writing therapy designed to treat Alpha-1 Antitrypsin Deficiency (AATD). Under this agreement, Regeneron will provide $150 million upfront, combining cash and equity to support Tessera’s research and development. Additionally, Tessera may receive up to $125 million through potential near- and mid-term milestone payments as TSRA‑196 progresses through clinical and regulatory milestones. Both companies will equally share development costs and future profits, ensuring a balanced approach to advancing this promising therapy.
Tessera will lead the initial first-in-human clinical trials, while Regeneron will oversee global development and commercialization. This collaboration strategically leverages Tessera’s expertise in gene-writing technologies and Regeneron’s deep experience in clinical development, regulatory affairs, and worldwide commercialization. Consequently, this partnership establishes a strong pathway for delivering TSRA‑196 to patients globally. The therapy specifically targets the underlying genetic mutation in SERPINA1, which causes AATD, and aims to restore functional Alpha-1 Antitrypsin protein production. If successful, it could replace current weekly augmentation therapies with a single, durable treatment addressing the disease’s root cause.
Preclinical studies have shown encouraging results for TSRA‑196. Experiments conducted in mice and non-human primates demonstrated that a single dose can achieve high-fidelity, long-lasting editing of the SERPINA1 gene. Furthermore, the therapy predominantly restricted gene editing to the liver, which serves as the primary site of AAT production. Importantly, no off-target or germline modifications were observed, highlighting the therapy’s precision and safety profile. These promising preclinical results support advancing TSRA‑196 into human clinical trials, marking a significant step toward therapeutic innovation.
Alpha-1 Antitrypsin Deficiency is a rare inherited disorder affecting both the lungs and liver. Mutations in the SERPINA1 gene lead to the accumulation of misfolded AAT protein in the liver, which can cause inflammation, fibrosis, and cirrhosis. Simultaneously, insufficient functional AAT in the bloodstream leaves the lungs vulnerable to progressive damage, often resulting in COPD or emphysema. Current treatments primarily alleviate symptoms and require frequent intravenous infusions. It is placing a considerable burden on patients without addressing the disease’s genetic cause. TSRA‑196 offers the potential to fundamentally transform this treatment landscape.
The companies aim to submit an Investigational New Drug application to the U.S. FDA and multiple Clinical Trial Applications internationally by the end of 2025. Upon approval, the first-in-human trial will evaluate safety, tolerability, and preliminary efficacy in adults with severe AATD. If successful, further trials will expand enrollment to larger patient populations and investigate long-term outcomes. Ultimately, these efforts will lay the groundwork for global commercialization and broader patient access.
Executives from both firms have expressed optimism regarding the collaboration’s potential. Regeneron emphasized genetic medicine’s ability to revolutionize patient care and treat previously untreatable diseases. Meanwhile, Tessera highlighted that TSRA‑196 could provide transformative outcomes with a single intravenous dose. It represents a major advance for AATD treatment. Together, the collaboration demonstrates a shared commitment to patient-centered innovation and next-generation genetic therapies.
Although the agreement signifies a key milestone, it remains subject to standard closing conditions, including regulatory approvals and antitrust review. The companies will invest further in research, clinical infrastructure, and manufacturing scale-up to ensure TSRA‑196 can reach patients efficiently. This partnership is expected to accelerate development timelines and enhance the therapy’s impact through shared expertise and resources.
The collaboration marks a critical step toward developing the first gene-writing therapy for AATD. If TSRA‑196 proves safe and effective, it could provide a durable, one-time solution that targets the genetic cause rather than the symptoms, offering hope to thousands worldwide. Both companies also plan to explore additional applications of Tessera’s gene-writing technology beyond AATD, potentially creating therapies for other genetic disorders and expanding the technology’s global impact.
By combining innovative science with global development capabilities, Regeneron and Tessera aim to improve outcomes for patients with rare genetic diseases. This collaboration represents a flagship program in genetic medicine, demonstrating how shared innovation, rigorous clinical development, and patient-focused strategies can bring transformative therapies to reality. TSRA‑196 has the potential to redefine AATD treatment by addressing the root cause, reducing long-term treatment burdens, and ultimately enhancing patients’ quality of life globally.
