YOLT-201 / YolTech Therap - LARVOL DELTA

Clinical study on the safety and efficacy of YOLT-201 in the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) (ChiCTR) - P=N/A | N=9 | Completed | Sponsor: The FIrst Affiliated Hospital, College of Medicine, Zhejiang University; The FIrst Affiliated Hospital, College of Medicine, Zhejiang University

New trial • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

YOLT-201_2023_IIT_01: Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy (clinicaltrials.gov) - P1 | N=7 | Recruiting | Sponsor: Zhejiang University | N=14 ➔ 7 | Trial completion date: Oct 2025 ➔ Feb 2026 | Trial primary completion date: Oct 2024 ➔ Jul 2025

Enrollment change • Trial completion date • Trial primary completion date • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular

YolTech Therapeutics Announces Successful Completion of Dose Escalation Phase in Phase I Trial of YOLT-201 for ATTR (PRNewswire) - P1/2a | N=31 | NCT06539208 | Sponsor: YolTech Therapeutics Co., Ltd | 'YolTech Therapeutics today announced updated data from its ongoing Phase I/IIa clinical trial of YOLT-201, a first-in-class CRISPR/Cas9-based in vivo gene-editing therapy for ATTR amyloidosis. The trial has completed dosing in eight participants, including six patients with ATTR amyloidosis with polyneuropathy (ATTR-PN) and two patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM), across two dose cohorts. No Grade 3 adverse events (AEs), dose-limiting toxicities (DLTs), or serious adverse events (SAEs) leading to treatment discontinuation have been observed. All ATTR-PN participants across both dose cohorts have completed dosing and follow-up. Preliminary data indicate that participants in the higher dose cohort achieved over 90% reductions in circulating TTR protein levels, with the therapy demonstrating robust safety and tolerability."

P1/2 data • Amyloidosis

A Phase I/IIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) (clinicaltrials.gov) - P1/2 | N=31 | Recruiting | Sponsor: YolTech Therapeutics Co., Ltd

New P1/2 trial • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Pain

First Patient Dosed in Phase I Clinical Trial of YOLT-201 (PRNewswire) - "YolTech Therapeutics...today the successful enrollment of the first patient in the Phase I clinical trial of YOLT-201, its independently developed in vivo gene editing therapy. This marks a significant milestone in the clinical development of this therapeutic candidate...The first enrolled patient received their first dose on June 28, 2024. Two weeks post-dosing, the patient was in good health with no significant drug-related adverse events observed...On March 1, 2024, YolTech Biotech announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) had approved the clinical trial application (IND) for YOLT-201."

Trial status • Amyloidosis • Cardiomyopathy

Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy (clinicaltrials.gov) - P1 | N=14 | Recruiting | Sponsor: Zhejiang University

New P1 trial • Amyloidosis • Cardiac Amyloidosis • Cardiomyopathy • Cardiovascular