YolTech Therapeutics, a biotech company developing in vivo gene editing therapies to treat rare genetic diseases, today 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.

About YOLT-201-101 Study

YT-YOLT-201-101 trial is a multicenter, open-label, single-dose phase I/IIa clinical study evaluating the safety, tolerability, 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.

About YOLT-201

YOLT-201 Injection utilizes several lipid components including ionizable lipids as primary excipients to encapsulate mRNA and sgRNA raw materials, forming lipid nanoparticles (LNP). Upon intravenous injection into the body, plasma ApoE protein binds to the surface of LNP particles. Liver cells expressing the LDLR receptor recognize ApoE protein and engulf the LNP through endocytosis, forming endosomes. The decrease in pH within endosomes promotes electrostatic interactions between ionizable lipids and endosomal membranes, leading to membrane disruption and the release of mRNA and sgRNA. mRNA, in the cytoplasm, binds to ribosomes, translating the editor protein. The editor protein, in combination with sgRNA, enters the cell nucleus. sgRNA specifically locates the editor to the TTR gene sequence, and the editor protein modifies the target TTR gene, preventing its normal transcription into mRNA. This process stops the production of the TTR protein, achieving the goal of a one-time administration for a comprehensive cure of ATTR diseases.

About YolTech

YolTech Therapeutics is a pioneering gene editing company dedicated to develop a robust gene editing medicines to treat patients with serious diseases, which has built leading high-throughput evolution platform and innovative LNP deliver system. It possesses strong capability of novel Cas and base editor discovery and exceptional in-house LNP production capacity for GMP manufactory, with independent intellectual property rights and core patent protection globally. It has created a pipeline with 10+ genetic medicines focusing on cardiovascular diseases, metabolic diseases, infectious diseases as well as more common and rare diseases. Clinical trial clearance has been received for its leading asset.

https://en.prnasia.com/releases/global/landmark-clinical-approval-for-yolt-201-obtained-by-the-nmpa-438489.shtml