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

by Dr Natalie Singh - Health Editor
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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.

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