SOMERVILLE, Mass., Feb. 23, 2026 (GLOBE NEWSWIRE) — Tessera Therapeutics, the biotechnology company pioneering a new approach in genetic medicine known as Gene Writing™, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to TSRA-196, its lead in vivo gene editing program. TSRA-196 is being jointly developed with Regeneron for the treatment of adults with alpha-1 antitrypsin deficiency (AATD) who are homozygous for the PiZ allele (PiZZ).
TSRA-196 is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional alpha-1 antitrypsin (AAT) protein through a one-time, durable treatment option for patients.
“Despite a clear understanding of the mutation that drives severe AATD, options are limited to life-long treatments that fail to address the underlying cause of the disease,” said David Davidson, M.D., Chief Development and Medical Officer. “Based on compelling evidence to date, we believe that TSRA-196 has the potential to permanently correct the underlying mutation responsible for this debilitating disease and address a high unmet need for a durable therapy. Fast Track and Orphan designations allow us to accelerate the development of TSRA-196 to dramatically improve the lives of people living with AATD, and we look forward to translating this promising mechanism into meaningful outcomes in our ongoing global first-in-human trial.”
Tessera recently initiated a Phase 1/2 trial to evaluate the safety, tolerability, and efficacy of TSRA-196 in adults with AATD. In this first-in-human, open-label, multi-national study, trial participants will receive a single intravenous administration of TSRA-196 and be followed longitudinally for safety and key biomarkers relevant to AAT expression and function.
Fast Track designation is intended to facilitate the development and expedite the review of new drugs or biologics that are intended to treat serious or life-threatening conditions and demonstrate the potential to address an unmet medical need. The designation provides more frequent FDA interactions and eligibility for Rolling Review. In addition, at the time of Biologics License Application (BLA) submission and review, TSRA-196 may be considered for Priority Review and/or Accelerated Approval if the applicable criteria are met. Orphan Drug designation provides certain development incentives for products that treat rare diseases or conditions affecting fewer than 200,000 people in the United States. The designation provides development incentives including tax credits for qualified clinical trials, exemption from FDA user fees, and the potential for seven years of market exclusivity upon approval in the U.S.
About Alpha-1 Antitrypsin Deficiency (AATD)
AATD is an inherited monogenic disease that can affect the lungs, liver, or both organs. It is most often caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a protein produced in the liver and secreted into the bloodstream to protect lung tissue from enzymes such as neutrophil elastase. In individuals with severe AATD, mutations in the Z allele cause AAT protein to misfold and accumulate in the liver, leading to toxic effects such as inflammation and fibrosis. At the same time, insufficient circulating AAT leaves the lungs vulnerable to progressive damage consistent with chronic obstructive pulmonary disease (COPD) and emphysema. An estimated 200,000 people in the U.S. and Europe carry two copies of the Z allele (PiZZ genotype), typically resulting in only about 15 percent of normal serum AAT levels. There are currently no FDA-approved therapies that address the underlying genetic cause of AATD, and treatment options remain limited to weekly intravenous augmentation therapy for patients with lung disease.
About Tessera Therapeutics
Tessera Therapeutics is pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, with the aim to unlock broad new therapeutic frontiers. Our Gene Writing platform is designed to write therapeutic messages into the genome by efficiently changing single or multiple DNA base pairs, precisely correcting insertions and deletions, or adding exon-length sequences and whole genes. Our proprietary lipid nanoparticle delivery platform is designed to enable the in vivo delivery of RNA to targeted cell types. We believe our Gene Writing and delivery platforms will enable transformative genetic medicines to not only cure diseases that arise from errors in a single gene, but also modify inherited risk factors for common diseases and create engineered cells to treat cancer and potentially autoimmune and other diseases. Tessera Therapeutics was founded in 2018 by Flagship Pioneering, a life sciences innovation enterprise that conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability.
For more information about Tessera, please visit www.tesseratherapeutics.com.
Contact
Jonathan Pappas
LifeSci Communications, LLC
jpappas@lifescicomms.com
