YolTech Therapeutics, a biotech company developing in vivo gene editing therapies to treat rare genetic diseases, announced the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) has officially  approved the YOLT-201 investigational new drug (IND) application for a Phase I study, marking that the drug candidate has officially entered the registration clinical development stage.. The first patient has been dosed with YOLT-201 in Investigator initiated trial(IIT)  by the end of 2023, achieving promising preliminary efficacy and safety results.

ATTR is a debilitating genetic disease, caused by misfolded transthyretin protein (TTR) forming amyloid fibrils and depositing in various organs and tissues in the body such as myocardium in the heart and peripheral nerves in the limbs. Depending on the mutation involved, hATTR can occur in people in their teens and 20s, though other forms are typically diagnosed in people over 50 years of age.

YOLT-201 is a world leader in the development of in vivo gene editing therapies and lipid nanoparticle (LNP) delivery systems. A single dose infusion of YOLT-201 has been shown to safely and durably knock down TTR protein level in the serum in preclinical NHP models, potentially providing effective and lifelong clinical benefits to patients.

"Clinical approval granted for YOLT-201, marks important progress in our team's efforts in the field of in vivo gene editing." Dr. Yuxuan Wu, Founder and CEO of YolTech, expressed that, "Previously, YOLT-201 has achieved promising preliminary efficacy and safety results in the Investigator-Initiated Trial (IIT), patients treated with YOLT-201 showed a significant decrease in TTR levels, with no Grade 2 above adverse reactions observed throughout the entire treatment and follow-up process. We are confident to advance YOLT-201 clinical trials and committed to providing innovative treatment solutions for patients."

YOLT-201 leads the first clinical approval of an in vivo gene editing drug mediated by lipid nanoparticles (LNP) in China. This breakthrough will further promote research and development in the field of gene editing and lay a solid foundation for the development of innovative drugs in the future. We look forward to YOLT-201 achieving more exciting achievements in clinical trials and bringing good news to patients around the world in the near future.