Regeneron partners with Tessera Therapeutics in USD275M deal to develop gene writing therapy for AATD

USA—Regeneron Pharmaceuticals has joined forces with Tessera Therapeutics to develop and commercialize Tessera’s in vivo gene writing program, TSRA-196, aimed at treating alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder.

As part of this collaboration, Regeneron is investing USD 150 million in equity and upfront payments to acquire half of the global development and commercialization rights for TSRA-196.

This partnership allows both companies to share the development costs for TSRA-196 equally.

Following potential market approval, the therapy’s profits will be split evenly between Regeneron and Tessera.

Beyond the initial investment, Tessera may receive an additional USD125 million upon achieving specific milestones, bringing the total deal value to USD275 million.

TSRA-196 has demonstrated promising results in preclinical studies, showing durable editing of the SERPINA1 gene locus after a single dose in both mice and non-human primates.

Since the SERPINA1 gene produces the alpha-1 antitrypsin (AAT) protein, which is deficient or malfunctioning in AATD patients, repairing or replacing this gene could directly address the underlying cause of the disease.

AATD primarily causes damage to the respiratory and liver systems.

Tessera hypothesizes that substituting the inactive SERPINA1 gene with a functional one could halt the progression of AATD.

To evaluate this approach, Tessera plans a first-in-human clinical trial for TSRA-196.

Upon positive results, Regeneron will take the lead in further global development and commercialization efforts.

Currently, Tessera is finalizing the submission of an investigational new drug (IND) application and clinical trial authorization (CTA) for TSRA-196, which are expected to be submitted to the US Food and Drug Administration (FDA) by the end of 2025.

Successful clinical outcomes could position TSRA-196 as the first disease-modifying treatment available for patients with AATD.

According to research published in the Journal of Internal Medicine, AATD affects up to 30 individuals per 100,000 worldwide.

While no curative treatments exist yet, five augmentation therapies help protect patients from worsening respiratory symptoms but do not target the root cause of the disease.

Both Tessera and Regeneron aim to change this treatment landscape by addressing the genetic cause of AATD with TSRA-196.

However, they are not alone in this effort. Beam Therapeutics is developing an RNA-based candidate, BEAM-302, which has shown encouraging results in a Phase I/II trial by increasing production of the correct form of AAT and reducing faulty protein levels by as much as 78%.

Additionally, CRISPR Therapeutics plans to begin clinical trials for its gene therapy candidate, CTX460, by mid-2026.

Market analysts at GlobalData estimated the AATD market at USD1.2 billion in 2023 across the US and Germany.

This market is expected to more than double to 3.48 billion by 2031, growing at a compound annual rate of 11.2%, driven by advances in gene therapies and increasing disease awareness.