Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene-writing program, inking a deal that lets it in on a prospect for alpha-1 antitrypsin deficiency (AATD) through a $150 million upfront payment and equity investment.
The deal covers Tessera’s TSRA-196, a potential one-time treatment meant to correct the gene mutation at the heart of inherited monogenic disease AATD. Recently presented findings of the candidate in mice and non-human primates showed "high liver editing specificity” and no off-target editing, supporting its upcoming move into the clinic, according to a Regeneron press release.
Under the terms of the partnership, the two companies will split worldwide development costs and potential future profits equally, while Tessera is in line for another potential $125 million in near- and mid-term development milestone payments. Tessera will take the lead on the first-in-human trial, with Regeneron in charge of subsequent global development and commercialization.
“Tessera is on the cusp of a critical inflection point as we prepare to enter the clinic in the near term,” CEO Michael Severino, M.D., explained. “We are excited to partner with Regeneron, a global leader in innovative biotechnology and genetic medicine, to accelerate the development of TSRA-196 and broaden its potential impact to patients in need.”
Regeneron, for its part, considers AATD a disease that is “particularly well-suited” for Tessera’s gene editing approach, its chief scientific officer George D. Yancopoulos, M.D., Ph.D., added in the release.
“Together with Tessera, we have an opportunity to pioneer new frontiers in genetic medicine and redefine what is possible for AATD patients,” Yancopoulos said.
AATD is caused by mutations in the serpina1 gene, which encodes a liver-produced protein called alpha-1 antitrypsin (AAT). AAT is meant to protect the lungs and liver from damage, meaning that the approximately 200,000 people across the U.S. and Europe who are impacted by the disease can be vulnerable to progressive lung disease and suffer inflammation and fibrosis as the protein misfolds and accumulates in the liver. There are currently no therapies specifically approved to treat AATD, with patients with related lung disease limited to weekly intravenous augmentation therapy.
Tessera, established in 2018 by Flagship Pioneering, says it is pioneering a new category in genetic medicine with its gene-writing platform, according to its website.
Paired with its tissue-targeted non-viral delivery technology, the company’s RNA gene writers can target single point mutations, insertions and deletions or add entire genes altogether, enabling "transformative genetic medicines" that can cure diseases based on errors in a single gene, modify inherited risk factors for common diseases and more, according to the Regeneron release notes.
Founded by Geoffrey von Maltzahn, Ph.D., Jacob Rubens, Ph.D., and Flagship co-founder and CEO Noubar Afeyan, Ph.D., the biotech is backed by more than $500 million in investor support, including a $300 million series C in 2022.
Regeneron’s investment in Tessera comes after its own gene therapy work earned the company a Commissioner’s National Priority Voucher (CNPV), streamlining the FDA review of its investigational DB-OTO. The drugmaker is positioning DB-OTO to address a mutation that causes a rare genetic form of deafness and could come to market early next year or sooner with the CNPV, as FDA commissioner Marty Makary, M.D., looks to “to get a decision out promptly using the standard scientific process, cutting any wasted time,” he said of DB-OTO on an October FDA podcast.