Regeneron Pharmaceuticals has teamed up with Tessera Therapeutics to develop and commercialise Tessera’s rare disease in vivo gene writing programme, TSRA-196.
Regeneron will now hand over $150m in equity investment and upfront cash payments in exchange for half of the global development and commercialisation rights to TSRA-196, which is being developed as a one-time treatment for the rare genetic disorder, alpha-1 antitrypsin deficiency (AATD).
Tessera could also be eligible to receive $125m in milestone payments, so the total deal value may reach $275m.
Under the agreement, Regeneron and Tessera will share the development costs for TSRA-196 equally, while subsequent profits associated with the therapy will be split 50:50 if the drug were to reach the market.
TSRA-196 has already shown potential in preclinical studies, with durable SERPINA1 locus editing observed through the drug’s administration in both mice and non-human primates following a single dose.
SERPINA1 is the gene encoding the alpha-1 antitrypsin (AAT) protein, so its defectiveness or inactivity is directly responsible for AATD’s disease pathology. AATD is commonly characterised by degeneration of the respiratory and hepatic systems.
Tessera hypothesises that replacing inactive SERPINA1 with a functioning version of the gene could halt the development of AATD.
To test this theory, the company will conduct the initial first-in-human study on the gene therapy. If the first-in-human study shows potential, Regeneron will take the reins on the subsequent global development and commercialisation efforts for TRSA-196.
Tessera is therefore in the process of completing an investigational new drug (IND) and clinical trial application (CTA) for TSRA-196, which are both set to be submitted to the US Food and Drug Administration (FDA) by the end of 2025.
If TSRA-196 is proven effective and safe for use in patients with AATD, it could become the first-ever disease-modifying treatment for the disease.
According to a paper published in the Journal of Internal Medicine, the illness currently impacts between zero and 30 in 100,000 individuals worldwide.
AATD landscape broadens
While there are no approved curative treatments for AATD, there are currently five different augmentation therapies available to patients with the disease.
While these therapies can protect a patient from further AATD-driven respiratory damage, they do not address the root cause of the condition, which is the body’s inability to produce the protein.
Tessera and Regeneron hope to change this dynamic with TSRA-196, though they will not be the only company looking to achieve this goal.
Beam Therapeutics is betting on its RNA candidate, BEAM-302. Thus far, the candidate has shown promise, with a Phase I/II trial in AATD finding that one dose of the therapy triggered durable increases in the production of the corrected form of AAT while diminishing the prevalence of aberrant AAT by up to 78%.
Meanwhile, CRISPR Therapeutics is also looking to take its gene therapy, CTX460, to clinical trials in mid-2026.
Analysts at GlobalData valued the AATD market at $1.2bn across the two major markets (2MM: the US and Germany) in 2023. The indication’s worth is also forecast to grow significantly, with analysts predicting the market will reach a value of $3.48bn by 2031 – exhibiting a compound annual growth rate (CAGR) of 11.2% over this time period.
GlobalData is the parent company of Pharmaceutical Technology.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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