Regeneron Pharmaceuticals and Tessera Therapeutics announced that the U.S. Food and Drug Administration has granted Fast‑Track and Orphan Drug designations to Tessera’s lead in‑vivo gene‑editing program, TSRA‑196, for the treatment of adults with alpha‑1 antitrypsin deficiency (AATD) who are homozygous for the PiZ allele.
The Fast‑Track status enables the FDA to facilitate development through more frequent interactions and potential priority review, while the Orphan Drug designation provides market exclusivity and tax credits for a disease that affects fewer than 200,000 people in the United States. Together, these designations reduce regulatory hurdles and can shorten the time to market for a therapy that could transform care for a rare, under‑diagnosed condition affecting roughly 1 in 2,500–5,000 people in the U.S. and Europe with the ZZ genotype.
Regeneron’s collaboration with Tessera is part of a broader gene‑editing strategy that includes partnerships with Intellia Therapeutics and Mammoth Biosciences. Tessera’s proprietary “Gene Writing” technology delivers precise, in‑vivo correction of genetic mutations without double‑strand breaks, positioning TSRA‑196 as a potentially one‑time, durable treatment. The partnership also includes a $150 million upfront payment from Regeneron, underscoring confidence in the platform and providing Tessera with additional resources to advance the program.
Regeneron’s Q4 2025 financial results provide a strong backdrop for the regulatory milestone. The company reported revenue of $3.9 billion, up 3 % year‑over‑year, and GAAP net income of $845 million, down 8 % YoY. Non‑GAAP earnings per share reached $11.44, beating analyst estimates of $10.84. Management highlighted robust demand for core products such as Dupixent, which grew 34 % in Q4 2025, while noting headwinds in the broader Eylea franchise, which declined 28 % YoY. Guidance for 2026 projects GAAP R&D expenses of $6.45–$6.68 billion and non‑GAAP R&D expenses of $5.9–$6.1 billion, reflecting continued investment in pipeline expansion.
George D. Yancopoulos, M.D., Ph.D., Board Co‑Chair, President and Chief Scientific Officer of Regeneron, said, “Alpha‑1 antitrypsin deficiency is a serious disease with limited treatment options today and is particularly well suited for Tessera’s gene editing approach. Together with Tessera, we have an opportunity to pioneer new frontiers in genetic medicine and redefine what is possible for AATD patients.” Michael Severino, M.D., CEO of Tessera Therapeutics, added, “This collaboration underscores what we believe is a medically and commercially important opportunity to deliver transformative outcomes with a one‑time, intravenously delivered genetic treatment for patients living with alpha‑1 antitrypsin deficiency. Tessera is on the cusp of a critical inflection point as we prepare to enter the clinic in the near term. 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.”
The Fast‑Track and Orphan Drug designations for TSRA‑196 represent a significant regulatory milestone that accelerates development and positions Regeneron to expand its gene‑editing portfolio into a high‑need, rare‑disease market. Combined with strong Q4 2025 earnings and a clear investment plan for 2026, the announcement signals confidence in both the company’s current commercial performance and its future pipeline potential.
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