Regeneron Pharmaceuticals and Tessera Therapeutics have announced a major strategic collaboration to advance the development of a potential gene therapy targeting alpha-1 antitrypsin deficiency (AATD), a rare hereditary condition that causes severe, progressive lung and liver damage. The partnership, which was formally detailed in press releases and filings on both companies' websites, is structured to leverage Tessera's proprietary gene writing technology and Regeneron's experience in genetics and late-stage drug development.
Under the agreement, Tessera will receive $150 million from Regeneron, consisting of both an upfront cash payment and an equity investment. Tessera will also be eligible for up to $125 million in additional development milestone payments, bringing the partnership's total potential value to $275 million. Each company will share worldwide development expenses and, if successful, will split profits equally from the commercialization of Tessera’s lead investigational therapy.
The main focus of the collaboration is TSRA-196, Tessera's lead in vivo gene writing candidate for AATD. This candidate aims to offer a durable, potentially one-time treatment by directly correcting the underlying disease-causing mutation in the SERPINA1 gene, which results in dysfunctional production of the alpha-1 antitrypsin (AAT) protein. Current therapies, such as weekly intravenous augmentation, do not address the genetic root of the disease and only slow further lung injury.
Preclinical data, highlighted at the American Society of Gene & Cell Therapy 28th Annual Meeting, demonstrated that a single dose of TSRA-196 in mice and non-human primates led to persistent and precise editing of the SERPINA1 locus. Notably, these studies showed high gene editing fidelity in liver tissue, no detectable off-target effects, and encouraging safety profiles using Tessera’s proprietary lipid nanoparticle delivery system.
Tessera will spearhead the initial human clinical trial. These preparations include plans to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) and multiple Clinical Trial Applications (CTAs) in other territories by the end of 2025. Should early clinical results prove promising, Regeneron will assume responsibility for broader global development and regulatory actions. The hope is that, if approved, this therapy could become the first to directly address AATD's genetic root cause, unlike current standards of care that only alleviate symptoms.
AATD is caused by harmful mutations in the SERPINA1 gene. These genetic errors result in misfolded AAT protein, which accumulates in the liver and fails to protect the lungs from damage, significantly increasing the risk of chronic obstructive pulmonary disease and liver failure. The condition affects thousands of patients globally, and current available therapies are primarily supportive rather than curative.
The market opportunity for effective AATD treatments is substantial, with industry analysts estimating annual value in the billions of dollars, given anticipated growth as new genetic therapies become available. Several companies, such as Vertex Pharmaceuticals, are also pursuing genetic approaches to AATD, but the Regeneron-Tessera partnership aims to be among the first to bring a truly curative, gene-correcting therapy to patients.
Further coverage is available from the original article on Yahoo Finance.
In financial markets, Regeneron Pharmaceuticals, Inc. (REGN) has recently traded at $738.63 per share, reflecting a minor decrease of $11.48 (or -0.02%) following the partnership announcement. The company opened at $742.12, with an intraday high of $749.15 and a low of $730.93. The trading volume stood at 440,178 shares as of Tuesday, December 2, 09:41:50 PST.
This collaboration underscores a broader industry commitment to developing gene editing solutions for challenging and currently incurable genetic disorders. Should TSRA-196 prove successful in clinical settings, it could pave the way for expanded uses of gene writing technology in treating a wide range of genetic diseases and have a transformative impact on the lives of patients with AATD.