Full Breakdown
Regeneron Partners with Tessera Therapeutics on Gene Therapy for AAT Deficiency
12/2/2025, 5:43:23 PM
Overview of the Collaboration
Regeneron Pharmaceuticals, Inc. has entered into a significant partnership with Tessera Therapeutics, Inc. to develop TSRA-196, an innovative gene therapy targeting alpha-1 antitrypsin deficiency (AATD). This collaboration involves a $150 million upfront payment and equity investment from Regeneron, with the potential for Tessera to earn an additional $125 million in milestone payments. The agreement stipulates that both companies will share the worldwide development costs and future profits equally.
Details of TSRA-196
TSRA-196 is designed as a one-time treatment aimed at correcting the genetic mutation associated with AATD, a rare inherited disorder affecting approximately 200,000 individuals in the U.S. and Europe. AATD results from mutations in the SERPINA1 gene, leading to insufficient production of the alpha-1 antitrypsin protein, which is crucial for protecting the lungs and liver. Current treatment options are limited, primarily involving weekly intravenous augmentation therapy.
Recent preclinical studies presented by Tessera have demonstrated promising results, showing high liver editing specificity and no off-target effects in animal models. Tessera plans to file an Investigational New Drug application with the U.S. Food and Drug Administration (FDA) by the end of the year, with the aim of initiating first-in-human trials.
Key Figures in the Partnership
Tessera Therapeutics was founded in 2018 by Geoffrey von Maltzahn, Ph.D., Jacob Rubens, Ph.D., and Noubar Afeyan, Ph.D. The company is recognized for its pioneering gene-writing platform, which utilizes RNA gene writers to target specific genetic mutations. Regeneron’s Chief Scientific Officer, George D. Yancopoulos, M.D., Ph.D., emphasized the potential of this collaboration to redefine treatment possibilities for AATD patients.
Implications of the Collaboration
The partnership is seen as a critical step in advancing gene therapy for AATD, a condition that currently lacks approved therapies. Regeneron’s investment follows its own advancements in gene therapy, particularly with its investigational drug DB-OTO, which targets a mutation causing a rare genetic form of deafness. This collaboration not only enhances Tessera’s capabilities but also positions Regeneron to expand its portfolio in genetic medicine.
Criticism & Opposition
While the collaboration has been largely welcomed, some industry observers express caution regarding the long-term efficacy and safety of gene therapies. Concerns about the scalability of such treatments and the regulatory hurdles they may face have been raised, particularly in light of the complexities involved in gene editing technologies.
Verbatim Quotes
- “Tessera is on the cusp of a critical inflection point as we prepare to enter the clinic in the near term,” — Michael Severino, M.D., CEO of Tessera Therapeutics
- “Together with Tessera, we have an opportunity to pioneer new frontiers in genetic medicine and redefine what is possible for AATD patients,” — George D. Yancopoulos, M.D., Ph.D., Chief Scientific Officer of Regeneron
This partnership marks a significant advancement in the field of genetic medicine, with the potential to address a critical unmet need for patients suffering from AATD.
