YolTech Therapeutics Announces Positive Interim Data on YOLT-202 for the Treatment of Alpha-1 Antitrypsin Deficiency
Business Wire India
Single dose of YOLT-202 led to rapid, robust and dose-dependent increases in AAT levels to normal range
Single dose of YOLT-202 was well tolerated with a favorable safety profile
YolTech Therapeutics, a late clinical-stage biotechnology company developing in vivo gene editing therapies, today 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 Alpha-1 Antitrypsin Deficiency (AATD) that 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.
“These interim findings mark an exciting and important milestone for YolTech and for patients living with severe AATD. The rapid, robust, and dose‑dependent increases in functional AAT levels observed in this study—particularly among individuals with the PiZZ genotype—underscore the transformative potential of in vivo base editing as a one‑time treatment approach,” stated Yuxuan Wu, M.D., Founder and CEO of YolTech Therapeutics. “Equally encouraging is the favorable safety profile we have seen to date, which reinforces the precision and thoughtful engineering behind YOLT‑202. With these results in hand, we are more confident than ever in YOLT‑202’s potential to redefine the treatment paradigm for AATD, and we look forward to advancing this program toward an Investigational New Drug (IND) filing with the U.S. Food and Drug Administration (FDA) as we continue our mission to bring durable, life‑changing therapies to patients.”
YOLT-202 is being evaluated in a first-in-human, open-label, single dose escalation study in adult AATD patients, aiming to evaluate safety and tolerability. As of February 6th, two participants genetically confirmed as PiZZ genotype, were enrolled and dosed with YOLT-202 in both the 35 mg and 45 mg dose groups.
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.
YOLT-202 demonstrated favorable safety and tolerability with manageable adverse events (AEs). No severe AEs or AEs leading to discontinuation of YOLT-202 were reported, and all AEs were classified as Grade 1. The most common AE was infusion-related reaction (IRR). Elevation of alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were asymptomatic, mild and soon recovered without medication.
The ongoing IIT study (NCT07193615) is evaluating single doses administered via intravenous infusion of YOLT-202 at 35 mg, 45 mg and 55 mg dose levels. YolTech is actively preparing to file an IND with the FDA to support the global clinical development of YOLT-202 in AATD.
About YOLT-202
YOLT-202 is an in vivo gene-editing therapy that corrects PiZ mutation to PiM for the treatment of AATD. Utilizing YolTech’s proprietary adeneine base editor, YOLT-202 is engineered to achieve on-target editing with minimal bystander activity. YOLT-202 is currently being investigated in a first-in-human IIT study designed to evaluate safety and tolerability as well as an optimal biological dose of YOLT-202 in subjects with AATD. YOLT-202 has previously been granted Orphan Drug Designation by the U.S. FDA and the Company is currently preparing to file an IND with the U.S. FDA to support clinical development of YOLT-202.
About Alpha-1 Antitrypsin Deficiency (AATD)
AATD is an inherited, genetic, autosomal co-dominant disorder caused by mutations in the SERPINA1 gene, with the most frequent deficient variants coming from the Z (Glu342Lys) and S alleles (Glu264Val). The presence of Z alleles results in misfolding and polymerization of the AAT, leading to over 95% of severe AATD patients being PIZZ.
About YolTech
Built on HEPDONE™ Novel Editor Platform and non-viral LNP technologies, YolTech Therapeutics is pioneering in vivo gene-editing medicines with the potential for a one-time treatment that provides lifelong benefit. The company’s expanding clinical pipeline targets genetic, metabolic, cardiovascular, and autoimmune diseases, with initial results supporting the potential for durable and transformative therapeutic benefit.
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