YolTech Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation to YOLT-202 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

SHANGHAI - May 14, 2026 - 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 granted Regenerative Medicine Advanced Therapy (RMAT) designation to YOLT-202, the company's investigational in vivo gene-editing therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD).

The RMAT designation follows FDA clearance of YolTech's Investigational New Drug (IND) application to initiate an open-label, single-dose expansion Phase 2/3 clinical study evaluating the efficacy and safety of YOLT-202 in adult patients with AATD. The study is designed as a multiregional clinical trial (MRCT). In addition, YOLT-202 has received Orphan Drug Designation (ODD) from the FDA.

YOLT-202 is also being investigated in a first-in-human investigator-initiated trial (IIT) (NCT07193615) designed to evaluate its safety, tolerability, and preliminary efficacy in patients with AATD. Previously announced early clinical observations showed rapid and dose-dependent increases in AAT levels following administration of YOLT-202, with AAT levels reaching above the protective threshold in the first two dosed patients and evidence of structurally corrected, functional AAT protein production.

"RMAT designation for YOLT-202 marks another important regulatory milestone for YolTech and highlights the continued progress of our in vivo gene-editing platform," said Yuxuan Wu, Co-Founder and CEO of YolTech Therapeutics. "We remain focused on advancing YOLT-202 as a potential one-time, durable treatment option for patients with AATD and on exploring differentiated therapeutic approaches across serious genetic diseases."

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 adenine base editor, YOLT-202 is engineered to achieve on-target editing with minimal bystander activity.

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.