YolTech Therapeutics, a late clinical-stage biotechnology company developing in vivo gene-editing therapies, today announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for YOLT-202, the company’s investigational in vivo base-editing therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD).

The FDA approval enables the initiation of an open-label, single-dose expansion Phase 2/3 clinical study to evaluate the efficacy and safety of YOLT-202 in adult patients with Alpha-1 Antitrypsin Deficiency (AATD). The study is designed as a multiregional clinical trial (MRCT) to be conducted at clinical sites in the U.S. and other countries.

YOLT-202 is currently being investigated in a first-in-human investigator-initiated trial (IIT) (NCT07193615) designed to evaluate the safety, tolerability, and preliminary efficacy of YOLT-202 in patients with AATD. As of the date of this release, two patients had been enrolled and completed dose. Following administration of YOLT-202, both patients showed rapid, robust and dose-dependent increases in AAT level as early as in Week 1. AAT levels in both patients reached above the protective threshold of 11 μM. Additionally, AAT levels increased to normal range (>20 μM) in the 45 mg dose group. These newly produced AAT proteins were both structurally corrected (M-AAT) and functional, with the proportion of corrected M-AAT increasing to >95% in the 45 mg dose group.

“The FDA clearance, together with the encouraging early clinical data, highlights the transformative potential of YOLT-202 and our in vivo base-editing platform,” stated Yuxuan Wu, M.D., Co-Founder and CEO of YolTech Therapeutics. “We are excited to advance this Phase 2/3 study and bring a potential ‘one and done’ therapy to patients with Alpha-1 Antitrypsin Deficiency.”