Regeneron has established a global collaboration with Tessera Therapeutics to advance TSRA-196, an in vivo gene-editing candidate intended to address alpha-1 antitrypsin deficiency (AATD). As part of the agreement, Regeneron will provide a $150 million upfront payment and equity investment, while Tessera is eligible for up to $125 million in near- and mid-term development milestones. The two companies will divide global development expenses and future profits equally.
TSRA-196 is designed as a one-time therapy aimed at correcting the genetic mutation that causes AATD. Preclinical data from studies in mice and non-human primates showed high editing precision in the liver without detectable off-target activity, according to Regeneron.
Additional findings presented at the 28th annual meeting of the American Society of Gene and Cell Therapy indicated strong levels of genome editing in non-human primates, few unintended edits, no germline changes detected, and a safety and tolerability profile the company described as favorable.
Under the partnership, Tessera will direct the first-in-human trial, and Regeneron will take responsibility for subsequent global development and commercialization. The companies plan to submit an investigational new drug application for TSRA-196 before the end of the year. Tessera CEO Michael Severino, M.D., said, “Tessera is on the cusp of a critical inflection point as we prepare to enter the clinic in the near term,” adding that the collaboration is intended to expedite progress toward clinical testing.
Regeneron’s chief scientific officer, George D. Yancopoulos, M.D., Ph.D., noted that the company views AATD as well aligned with Tessera’s gene-editing approach. He also indicated that the partnership creates an opportunity to expand work in genetic medicine for individuals affected by the condition.
AATD results from mutations in the serpina1 gene, which encodes alpha-1 antitrypsin, a liver-produced protein that protects the lungs and liver from damage. Approximately 200,000 people in the U.S. and Europe are affected. Misfolding of the protein can lead to liver inflammation and fibrosis, while insufficient circulating protein may contribute to progressive lung disease. There are currently no approved therapies specifically for AATD, and patients with related lung symptoms often rely on weekly intravenous augmentation therapy.
Tessera was founded in 2018 by Geoffrey von Maltzahn, Ph.D., Jacob Rubens, Ph.D., and Noubar Afeyan, Ph.D., and has secured more than $500 million in funding to support its gene-writing platform and tissue-targeted non-viral delivery system. According to Regeneron, the company’s platform can target single point mutations, insertions, and deletions, or add entire genes.
Regeneron’s investment in Tessera follows progress in its broader genetic medicine portfolio, including advancement of DB-OTO, a gene therapy for a rare form of genetic deafness. The company has also expanded its in vivo editing and gene therapy capabilities through partnerships with Mammoth Biosciences and Intellia Therapeutics.
Through this collaboration, Regeneron reinforces its position in the field of genetic therapies. By investing in TSRA-196, the company is not only advancing potential treatments for AATD but also expanding its platform capabilities to tackle other rare and serious genetic disorders. This strategic approach aligns with Regeneron’s long-term commitment to precision medicine and improving patient outcomes globally.
Regeneron’s partnership with Tessera marks a significant milestone in the development of one-time gene-editing therapies for rare diseases. With robust preclinical data, a clear clinical path, and substantial financial backing, Regeneron is poised to bring innovative solutions to patients living with AATD while solidifying its leadership in the genetic medicine field.
Regeneron’s investment in TSRA-196 also underscores the company’s focus on advancing gene-editing technology beyond conventional therapies. By partnering with Tessera, Regeneron gains access to Tessera’s proprietary gene-writing platform and tissue-targeted delivery system, which allows for precise correction of single-point mutations, insertions, deletions, or even full gene replacements.
This approach could pave the way for treating a variety of genetic diseases, positioning Regeneron at the forefront of next-generation therapies.
