Preclinical models have demonstrated its potential to safely and effectively reduce TTR protein levels with a single dose infusion. Credit: Mongkolchon Akesin/Shutterstock. 

China-based YolTech Therapeutics has dosed the first patient as part of its Phase I clinical trial for gene editing therapy YOLT-201 to treat rare genetic diseases.

YOLT-201 Injection is formulated with ionisable lipids among other lipid components to encapsulate mRNA and sgRNA, creating therapeutic lipid nanoparticles (LNPs).

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YT-YOLT-201-101 is a multicentre, open-label, single-dose Phase I/IIa clinical study. It aims to assess the tolerability, safety, pharmacokinetics, and pharmacodynamic parameters of YOLT-201 in patients with transthyretin amyloidosis polyneuropathy (ATTR-PN) and transthyretin amyloidosis cardiomyopathy (ATTR-CM).

The trial consists of two stages: the first stage is an open-label, single-dose, dose-escalation study to determine the optimal biological dose (OBD) of YOLT-201; the second stage is an open-label, single-dose, dose-expansion study to preliminarily assess the safety and preliminary efficacy of YOLT-201 at the OBD.

A debilitating genetic disease, ATTR is caused due to misfolded transthyretin protein (TTR) forming amyloid fibrils and depositing in several organs and tissues in the body, such as myocardium in the heart and peripheral nerves in the limbs.

Based on the mutation involved, ATTR can manifest in people in their teens and 20s, though other types are usually detected in people aged above 50 years. 

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Preclinical models have demonstrated its potential to safely and effectively reduce TTR protein levels with a single dose infusion.

Phase I trial is recruiting ATTR patients in China.

The clinical trial application for YOLT-201 was approved by China’s Center for Drug Evaluation of the National Medical Products Administration in March this year.

In April 2024, YolTech dosed the first subject in an investigator-initiated trial (IIT) of YOLT-101, an in vivo genome editing candidate for familial hypercholesterolemia (FH).