FDA Grants Tessera Fast Track, Orphan Drug Designations for AATD Gene Writing Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Tessera Therapeutics Fast Track and Orphan Drug designations for TSRA-196, the company’s in vivo gene editing program for the rare condition alpha-1 antitrypsin deficiency.

Alpha-1 antitrypsin deficiency (AATD) is an inherited monogenic disorder that can affect the lungs, liver, or both. The condition is most commonly caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a protein produced in the liver that protects lung tissue from destructive enzymes. Mutations can cause the AAT protein to misfold and accumulate in the liver, leading to inflammation and fibrosis. At the same time, low levels of circulating AAT leave lung tissue vulnerable to progressive damage, often resulting in chronic obstructive pulmonary disease and emphysema.

TSRA-196 is designed to precisely repair the genetic mutation at the root of AATD, offering the potential for a one-time, durable treatment that restores normal production of functional AAT protein. It is being developed in collaboration with Regeneron.

“Despite a clear understanding of the mutation that drives severe AATD, options are limited to lifelong treatments that fail to address the underlying cause of the disease,” said David Davidson, chief development and medical officer for Tessera. “Based on compelling evidence to date, we believe TSRA-196 has the potential to permanently correct the underlying mutation responsible for this debilitating condition and address a high unmet need for a durable therapy.”

Tessera recently initiated a phase 1/2 trial evaluating the safety, tolerability, and efficacy of TSRA-196 in adults with AATD. The open-label, multinational study will administer a single intravenous dose of the therapy and follow participants over time for safety signals and biomarkers relevant to AAT expression and function.

The FDA’s Fast Track designation is designed to facilitate the development and expedite the review of therapies for serious or life-threatening conditions with unmet medical needs. It provides more frequent FDA interactions, Rolling Review eligibility, and potential for Priority Review or Accelerated Approval at the time of a Biologics License Application.

Orphan Drug designation supports the development of treatments for rare conditions affecting fewer than 200,000 people in the United States. It offers incentives such as tax credits for clinical trials, exemption from user fees, and the potential for seven years of market exclusivity upon approval.

Photo: David Davidson, chief development and medical officer for Tessera