YolTech Therapeutics, a leading biotechnology company specializing in in vivo gene editing therapies for rare genetic diseases, has achieved a significant milestone with the approval of the YOLT-201 investigational new drug (IND) application by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA). This marks the beginning of the registration clinical development phase for the drug candidate.

YOLT-201 targets ATTR, a debilitating genetic disease characterized by the accumulation of misfolded transthyretin protein (TTR) in various organs. The disease can manifest at different ages, with some forms appearing in individuals as young as their teens and twenties. YOLT-201, which utilizes lipid nanoparticle (LNP) delivery systems for in vivo gene editing therapies, has shown promising results in preclinical studies by safely reducing TTR protein levels in non-human primate models.

Dr. Yuxuan Wu, Founder and CEO of YolTech, expressed optimism about the clinical approval of YOLT-201, citing promising efficacy and safety results from an Investigator-Initiated Trial (IIT). Patients treated with YOLT-201 experienced a significant decrease in TTR levels without observing adverse reactions above Grade 2 throughout the treatment and follow-up period.

YOLT-201's approval marks a significant milestone as the first clinical approval of an in vivo gene editing drug mediated by lipid nanoparticles (LNP) in China. This achievement is expected to drive further advancements in gene editing research and development, laying a solid foundation for innovative drug development in the future.

The YT-YOLT-201-101 trial is a multicenter, open-label, single-dose phase I/IIa study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of YOLT-201 in patients with ATTR polyneuropathy (ATTR-PN) and ATTR cardiomyopathy (ATTR-CM). The trial comprises two stages: a dose-escalation study to determine the optimal biological dose (OBD) followed by a dose-expansion study to assess safety and preliminary efficacy at the OBD.

YOLT-201 Injection utilizes lipid nanoparticles (LNP) to encapsulate mRNA and sgRNA, which are then delivered intravenously into the body. The mechanism involves plasma ApoE protein binding to LNP particles, facilitating their uptake by liver cells expressing the LDLR receptor. Within the cells, mRNA translates editor protein, guided by sgRNA to the TTR gene sequence, leading to the modification of the target gene and inhibition of TTR protein production. This innovative approach offers the potential for a one-time administration to comprehensively treat ATTR diseases.

 

Source: prnewswire.com

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