YolTech Therapeutics Initiates Phase I Clinical Trial for Gene Editing Therapy YOLT-201 to Treat Rare Genetic Diseases
YolTech Therapeutics, a China-based company, recently announced the dosing of the first patient in its Phase I clinical trial for the gene editing therapy YOLT-201. The therapy is designed to treat rare genetic diseases by using therapeutic lipid nanoparticles to encapsulate mRNA and sgRNA. The multicenter, open-label, single-dose Phase I/IIa clinical study, YT-YOLT-201-101, aims to assess the tolerability, safety, pharmacokinetics, and pharmacodynamic parameters of YOLT-201 in patients suffering from transthyretin amyloidosis polyneuropathy (ATTR-PN) and transthyretin amyloidosis cardiomyopathy (ATTR-CM).
The Clinical Trial
The trial consists of two stages. The first stage is an open-label, single-dose, dose-escalation study aimed at determining the optimal biological dose of YOLT-201. The second stage is an open-label, single-dose, dose-expansion study designed to evaluate the safety and preliminary efficacy of YOLT-201 at the optimal biological dose.
A debilitating genetic disease, ATTR occurs due to the misfolded transthyretin protein (TTR) forming amyloid fibrils that deposit in various organs and tissues in the body, such as the myocardium in the heart and peripheral nerves in the limbs. ATTR can manifest in people in their teens and 20s, while other types are usually detected in people aged above 50 years. Preclinical models have shown that YOLT-201 may safely and effectively reduce TTR protein levels with a single dose infusion.
The clinical trial application for YOLT-201 was approved by China’s Center for Drug Evaluation of the National Medical Products Administration in March 2021. The Phase I trial is recruiting ATTR patients in China.
YolTech’s Investigator-Initiated Trial of YOLT-101 for Familial Hypercholesterolemia
In April 2024, YolTech dosed the first subject in an investigator-initiated trial (IIT) of YOLT-101. The in vivo genome editing candidate is specifically designed for familial hypercholesterolemia (FH), an inherited condition that causes high levels of low-density lipoprotein (LDL) cholesterol. YolTech’s IIT aims to test the safety and efficacy of YOLT-101 in reducing cholesterol in FH patients.
Conclusion
Gene editing therapies such as YOLT-201 and YOLT-101 hold tremendous promise for treating a wide range of genetic diseases. Although still in the early stages of development, the successful completion of clinical trials for these therapies could help pave the way for more effective treatments for patients suffering from rare, inherited conditions.
Originally Post From https://www.clinicaltrialsarena.com/news/yoltech-phase-i-yolt201-therapy/
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