“This is exciting news for the Alpha-1 community, marking an important regulatory milestone for Tessera Therapeutics and also, in the field of in vivo genome editing. It is the first-ever IND clearance for a therapy based on target-primed reverse transcription (TPRT)-based genome editing. This approval allows Tessera to initiate its Phase 1/2 clinical trial, bringing the company closer to delivering an innovative therapy designed to permanently correct the root cause of Alpha-1. We look forward to the shared results of this first of its kind trial,” said Scott Santarella, A1F President & CEO.
“This technology represents a breakthrough in Alpha-1 research. Currently, there are no FDA-approved therapies that address the underlying genetic cause of Alpha-1. Existing treatments, such as weekly intravenous augmentation therapy, manage symptoms but do not cure the disease. TSRA-196 offers hope for a transformative therapy that could permanently correct the genetic root cause, potentially improve quality of life and reduce the long-term health impacts on Alpha-1 patients,” stated Andrew Wilson, MD, A1F Scientific Director.
“As an Alpha, I am very encouraged by this advancement in the trial. Correcting mutations in the SERPINA1 gene addresses the underlying genetic cause of Alpha-1 and has the potential to fundamentally change the trajectory of the disease,” said Jon Hagstrom, Chair of the Board of Directors, Alpha-1 Foundation.
PRESS RELEASE
SOMERVILLE, Mass., January 12, 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 cleared its Investigational New Drug (IND) application for TSRA-196, its lead in vivo gene editing therapy for alpha-1 antitrypsin deficiency (AATD). The Company has also received Australian Human Research Ethics Committee (HREC) approval to begin the Phase 1/2 clinical trial.
The Phase 1/2 trial is a first-in-human, open-label, multi-national study designed to evaluate the safety, tolerability, and efficacy of TSRA-196 in adults with AATD. Trial participants will receive a single intravenous administration of TSRA-196 and be followed longitudinally for safety and key biomarkers relevant to alpha-1 antitrypsin (AAT) expression and function.
“FDA clearance of our IND represents an important regulatory milestone for Tessera and for the field of in vivo genome editing,” said David Davidson, M.D., Chief Development and Medical Officer. “This is the f irst ever IND clearance for an in vivo target-primed reverse transcription (TPRT)-based genome editing therapy, and it reflects the rigor of our development approach and the strength of our data package. With regulatory clearances now in hand in both the US and Australia, we are well positioned to initiate our Phase 1/2 study and generate the first-in-human clinical data for TSRA-196. People living with AATD have limited effective treatment options today and we are now one step closer to delivering an innovative therapy designed to permanently correct the root cause of this devastating disease.”
“We are extremely encouraged by this progress, which underscores Tessera’s leadership in developing a new class of genetic medicines,” said Michael Severino, M.D., CEO. “This milestone builds on our broader mission to harness the power of our Gene Writing and delivery platforms to advance transformative medicines for patients. Alpha-1 antitrypsin deficiency remains a serious genetic disease with significant unmet medical need, and we believe TSRA-196 has the potential to offer a truly differentiated, best-in-class approach by addressing the underlying genetic cause.”
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 exonlength 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.
I am so thrilled that we have actual cures being worked on! I know so many precious Alphas that deserve this. Including my kids. If accepting MZ patients we will sign up because we want to help further the fight towards the cure if possible. Can’t wait to hear more from them.
I’m a ZZ taking augmentation therapy once a week. My brother received his Alpha Wings at 52. So, I am hopeful this is successful.
I hope at some point it will be tested in those of us with something other than MZ and ZZ. I’m SZ, and would be very happy to be turned into an MS…
Are they choosing people to test .
Hi. I-am an MZ and this is really exciting news. How can I be part of the trial? I’d do anything to be part of it and hopefully this treatment becomes available for those suffering from it. My 13 year old son is an MZ too.