YolTech Therapeutics has received clearance from the US Food and Drug Administration (FDA) to begin a Phase II/III clinical study of its in vivo gene-editing therapy, YOLT-202, for alpha-1 antitrypsin deficiency (AATD).
The FDA’s approval for the company’s investigational new drug (IND) application allows it to initiate a single-dose expansion, open-label trial to assess the safety and efficacy of YOLT-202 in adults with AATD.
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The study will be conducted as a multiregional clinical trial (MRCT) at sites in the US and additional countries.
YOLT-202 is currently under investigation in a first-in-human investigator-initiated trial, which is assessing the tolerability, preliminary efficacy, and safety in patients with AATD. Two patients have so far been enrolled and completed dosing.
After receiving the YOLT-202 doses, these patients experienced swift and dose-dependent rises in AAT levels beginning in the first week. Each patient achieved AAT concentrations exceeding the protective threshold of 11μM.
In the 45mg dose group, AAT levels increased to the normal range, above 20μM. The newly produced proteins were structurally corrected (M-AAT) and functional, with more than 95% reporting corrected M-AAT in the 45mg group.
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By GlobalDataYolTech Therapeutics co-founder and CEO Dr Yuxuan Wu said: “The FDA clearance, together with the encouraging early clinical data, highlights the transformative potential of YOLT-202 and our in vivo base-editing platform. We are excited to advance this Phase II/III study and bring a potential ‘one and done’ therapy to patients with alpha-1 antitrypsin deficiency.”
AATD is an inherited genetic disorder caused by mutations in the SERPINA1 gene, most frequently from the Z (Glu342Lys) and S (Glu264Val) alleles.
In January 2025, YolTech Therapeutics commenced a trial of its in vivo gene-editing therapy, YOLT-204, aimed at treating transfusion-dependent beta-thalassemia (TDT).
