YolTech Therapeutics Receives FDA Clearance to Initiate Phase 2/3 Study of In Vivo Gene-Editing Therapy YOLT-202 in Alpha-1 Antitrypsin Deficiency (AATD)

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SHANGHAI--(BUSINESS WIRE)--Mar 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 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.”

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. YOLT-202 has previously been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA).

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 advancing in vivo gene-editing medicines with the potential for a one-time treatment that provides lifelong benefit. The company’s expanding clinical pipeline addresses genetic, metabolic, cardiovascular, and autoimmune diseases, with early clinical results supporting the potential for durable and transformative outcomes.

Stay informed with the latest from YolTech Therapeutics:
LinkedIn: linkedin.com/company/yoltech-therapeutics
Website: www.yoltx.com

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CONTACT: company name: YolTech Therapeutics

media contact: Ally Yu

email address: [email protected]

KEYWORD: CHINA UNITED STATES NORTH AMERICA ASIA PACIFIC

INDUSTRY KEYWORD: HEALTH FDA GENETICS OTHER HEALTH CLINICAL TRIALS PHARMACEUTICAL BIOTECHNOLOGY

SOURCE: YolTech Therapeutics

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PUB: 03/14/2026 12:53 AM/DISC: 03/14/2026 12:53 AM

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