Press Release

Dec 06, 2023

YolTech Announces NMPA Acceptance of IND Application for YOLT-201 to Treat hATTR


Shanghai, China, December 6, 2023 - YolTech Therapeutics, a biotech company developing in vivo gene editing therapies to treat rare genetic diseases, today announced the National Medical Products Administration (NMPA) has accepted the YOLT-201 investigational new drug (IND) application for a Phase I study to evaluate the safety and efficacy of YOLT-201 in the treatment of hereditary Transthyretin Amyloidosis Polyneuropathy (hATTR-PN) and Transthyretin Amyloidosis Cardiomyopathy (hATTR-CM).

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.

Dr. Yuxuan Wu, Founder and CEO of YolTech, highlighted the significance of this milestone that we only took two years to accomplish IND filing from drug discovery to clinic, confirming YolTech’s expertise in gene editing technology and execution excellence in clinical translation. In vivo gene editing represents a revolutionary breakthrough, offering precise therapy for various genetic and rare diseases. We are confident to advance YOLT-201 clinical trials and committed to providing innovative treatment solutions for patients.

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 base editor protein. The base editor protein, in combination with sgRNA, enters the cell nucleus. sgRNA specifically locates the base editor to the TTR gene sequence, and the base editor protein modifies the bases on 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. Leading asset will enter phase I trial by end of 2023.

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