SHANGHAI, July 11,
2024 /PRNewswire/ -- YolTech Therapeutics, a biotech
company developing in vivo gene editing therapies to treat rare
genetic diseases, announced today the successful enrollment of
the first patient in the Phase I clinical trial of YOLT-201, its
independently developed in vivo gene editing therapy. This marks a
significant milestone in the clinical development of this
therapeutic candidate.
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.
Eligible ATTR patients will be recruited nationwide for this
clinical trial. The first enrolled patient received their first
dose on June 28, 2024. Two weeks
post-dosing, the patient was in good health with no significant
drug-related adverse events observed.
"The completion of the first patient dosing and safety
assessment is a critical milestone in the clinical development of
YOLT-201." Dr. Yuxuan Wu, Founder
and CEO of YolTech, expressed that, "The early clinical trials have
demonstrated excellent safety and efficacy for YOLT-201, and we
believe it has the potential to transform the global clinical
treatment landscape for ATTR diseases.".
On March 1, 2024, YolTech Biotech
announced that the Center for Drug Evaluation (CDE) of the National
Medical Products Administration (NMPA) had approved the clinical
trial application (IND) for YOLT-201. This is the first in vivo
gene editing drug mediated by lipid nanoparticles (LNP) to receive
clinical trial approval in China.
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.
For more information, please visit: www.yoltx.com
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SOURCE YolTech Therapeutics