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YolTech’s Gene Editing Therapy Shows Promise for Alpha-1 Antitrypsin Deficiency

YolTech Therapeutics Reports Promising Interim Data for YOLT-202 in Alpha-1 Antitrypsin Deficiency SHANGHAI, February 19, 2026 – YolTech Therapeutics, a late-clinical-stage biotechnology company specializing in in vivo gene editing therapies, today announced positive interim data from an investigator-initiated…

YolTech’s Gene Editing Therapy Shows Promise for Alpha-1 Antitrypsin Deficiency

YolTech Therapeutics Reports Promising Interim Data for YOLT-202 in Alpha-1 Antitrypsin Deficiency

SHANGHAI, February 19, 2026 – YolTech Therapeutics, a late-clinical-stage biotechnology company specializing in in vivo gene editing therapies, today announced positive interim data from an investigator-initiated trial (IIT) evaluating YOLT-202 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The data demonstrate a favorable safety profile and significant increases in AAT levels in patients treated with 35 mg and 45 mg doses.

What is Alpha-1 Antitrypsin Deficiency (AATD)?

AATD is a genetic disorder caused by mutations in the SERPINA1 gene . The most common severe form of the disease is associated with the PiZZ genotype, leading to misfolding and polymerization of alpha-1 antitrypsin (AAT) .

About YOLT-202 and its Mechanism

YOLT-202 is an investigational in vivo base editing therapy designed to correct the PiZ to PiM mutation, the most common and severe disease-causing variant in AATD . It utilizes YolTech’s proprietary adenine base editor, YolBE, engineered from a novel deaminase derived from Hafnia paralvei . YolBE is optimized for precise correction of the mutation with minimal off-target effects .

Key Findings from the Investigator-Initiated Trial

  • AAT Level Increases: Administration of YOLT-202 led to rapid, robust, and dose-dependent increases in AAT levels .
  • Normalization of AAT Levels: In patients receiving the 45 mg dose, AAT levels reached normal values (> 20 μM) .
  • Functional AAT Production: The newly produced AAT proteins were structurally corrected (M-AAT) and functional, with over 95% correction in the 45 mg dose group .
  • Safety and Tolerability: YOLT-202 demonstrated a favorable safety and tolerability profile, with adverse events considered controllable . No serious adverse reactions or treatment discontinuations were reported, and all adverse events were Grade 1 .

Regulatory Progress and Future Plans

YOLT-202 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of AATD . YolTech is preparing to submit an Investigational Novel Drug (IND) application to the FDA to support global clinical development .

“These interim findings mark an exciting and vital milestone for YolTech and for patients living with severe AATD,” said Yuxuan Wu, M.D., Founder and CEO of YolTech Therapeutics .

About YolTech Therapeutics

YolTech Therapeutics is a late clinical-stage biotechnology company developing in vivo gene editing therapies using its HEPDONE™ Novel Editor platform and non-viral lipid nanoparticle (LNP) technologies . The company’s pipeline targets genetic, metabolic, cardiovascular, and autoimmune diseases.

About the author: Dr Natalie Singh - Health Editor

Board‑certified internal‑medicine physician and MPH. Natalie authored peer‑reviewed studies on infectious disease and served as medical editor. “Dr. Natalie Singh delivers evidence‑based health news, medical breakthroughs, and expert wellness guidance.”