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Catch up on new trial readouts, trends in hepatitis B birth dose vaccination, updates in a rare hepatic disease in honor of Rare Disease Day, and more.
February 2026 spotlighted a wave of innovation in genetic and translational hepatology, particularly in alpha-1 antitrypsin deficiency (AATD). Interim data for YOLT-202 demonstrated that a single in vivo base-editing dose could meaningfully increase functional AAT levels, while TSRA-196 earned both Fast Track and Orphan Drug designations from the US Food and Drug Administration (FDA), reinforcing growing regulatory support for one-time gene-editing strategies aimed at correcting the underlying PiZZ mutation.
Beyond gene editing, the month reflected broader advances across metabolic, viral, and cholestatic liver disease. Preclinical data using a human Liver-on-a-Chip platform suggested disease-modifying potential for Thykamine in metabolic dysfunction-associated steatohepatitis (MASH), offering a translational bridge between animal models and human systems. At the population level, emerging data showing declining hepatitis B birth dose vaccination rates raised concerns about long-term liver cancer risk, while new research challenged persistent hesitancy around statin use in primary biliary cholangitis (PBC). Rounding out February, Rare Disease Day brought renewed attention to biliary atresia, underscoring the urgency of early recognition in altering lifelong outcomes.
Check out this February 2026 hepatology month in review for a recap of HCPLive’s coverage of the top hepatic news and research from the past few weeks:
On February 19, 2026, YolTech Therapeutics announced positive interim data from an investigator-initiated trial (IIT) of YOLT-202, the Company’s investigational in vivo base editing therapy, for the treatment of AATD. Findings demonstrated positive safety and tolerability as well as meaningful increases in AAT levels in evaluated patients treated with the 35 mg and 45 mg dose levels.
On February 19, 2026, Devonian Health Group announced positive results from a follow-up preclinical study evaluating Thykamine™ in a human MASH model using PhysioMimix® Liver-on-a-Chip platform. Findings suggest Thykamine exerts dose-dependent effects on key pathological hallmarks of MASH, including fibrosis and inflammation, in a physiologically relevant human liver system, representing a notable translational bridge between Devonian’s previously disclosed efficacy in the mouse STAM® MASH model and a predictive human microphysiological model.
On February 23, 2026, the FDA granted Fast Track and Orphan Drug designations to TSRA-196, an in vivo gene editing program from Tessera Therapeutics, for the treatment of adults with AATD who are homozygous for the PiZ allele (PiZZ). TSRA-196 is being jointly developed with Regeneron and is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional AAT protein through a one-time, durable treatment option for patients.
Emerging research suggests hepatitis B birth dose vaccination is on the decline, posing important implications for rising chronic infection and future liver cancer risk. The study found that rates of the newborn hepatitis B vaccine declined by > 10% over the past 2 years in the United States. Of note, this decline occurred before updated recommendations from the Advisory Committee on Immunization Practices (ACIP) in December 2025 and subsequent updated guidance from the Centers for Disease Control and Prevention (CDC) in January 2026.
For many years, concerns about hepatotoxicity have contributed to hesitancy around prescribing statins in patients with chronic liver disease. While recent research has largely addressed and dispelled these concerns, a lack of studies specific to cholestatic conditions like PBC have resulted in continued hesitancy. A recent study from Jonggi Choi, MD, PhD, and colleagues points to a reduced risk of hepatic decompensation and major liver events in patients with PBC taking statins, supporting a potential protective effect in this population.
In honor of Rare Disease Day, celebrated on February 28 each year, the editorial team of HCPLive spoke with families and clinicians to highlight the race against time in biliary atresia, a rare hepatic condition where early recognition can change the course of a child’s life.