SHANGHAI, Jan. 20,
2025 /PRNewswire/ -- YolTech Therapeutics, a
clinical-stage gene editing company dedicated to delivering
lifelong cures, announced the initiation of a clinical trial for
YOLT-204, an investigational
therapy for the treatment of transfusion-dependent beta-thalassemia
(TDT). TDT is a severe genetic blood disorder where mutations in
the beta-globin gene leads to reduced or absence of hemoglobin, a
condition where regular blood transfusion is required to manage
anemia and prevent other complications.
YOLT-204 is a first-in-class
in vivo gene editing therapy leveraging YolTech's
proprietary lipid nanoparticles (LNP). The therapy edits the
regulatory region of hemoglobin to induce expression of fetal
hemoglobin, potentially alleviating the imbalance of hemoglobin
production and normalizing the number of red blood cells in TDT
patients. In pre-clinical models, YOLT-204 showed effective and sustained
induction of fetal hemoglobin, suggesting therapeutic potential for
TDT. YOLT-204 may also be an
effective treatment for patients with sickle cell disease (SCD), as
increased expression of fetal hemoglobin in these patients has been
associated with less polymerization of sickle hemoglobin as well as
reduced complications and mortality.
The clinical trial for YOLT-204
is a dose-escalation study to preliminarily examine the safety and
efficacy of a single-dose regimen with YOLT-204 in TDT. If successful, YOLT-204 may eventually provide an
off-the-shelf curative treatment for TDT patients without
conditioning chemotherapy and HSCT (Hematopoietic Stem Cell
Transplantation).
"The initiation of clinical trial for YOLT-204 represents a significant milestone
of gene editing therapy development for TDT and SCD," said Dr.
Yuxuan Wu, founder and Chief
Executive Officer of YolTech Therapeutics. "We are excited to
collaborate with our clinical investigators to bring this
innovative therapy to patients."
For further details about the trial or YolTech's ongoing
programs, please visit [www.yoltx.com] or contact [info@yoltx.com].
About YOLT-204
YOLT-204 is an off-the-shelf in
vivo gene editing therapy developed for TDT without
conditioning chemotherapy and HSCT. YOLT-204 delivers a gene editor
to hematopoietic stem cells through YolTech's proprietary lipid
nanoparticles. The editor together with a guide RNA targets the
hemoglobin regulatory region to induce expression of fetal
hemoglobin. The expression of fetal hemoglobin has the potential to
normalize hemoglobin composition and the red blood cell counts in
patients with transfusion-dependent beta-thalassemia, eventually
making them transfusion independent.
About YolTech
YolTech Therapeutics is a clinical-stage
in vivo gene editing company committed to pioneering the
next generation of precision genetic medicines. Our approach
combines innovative gene editing technologies with an advanced
lipid nanoparticle (LNP) delivery system, creating a versatile
platform designed to address a wide range of serious diseases.
Central to our mission is the development of internal capabilities,
including end-to-end manufacturing, to ensure the highest standards
of quality and scalability. Our lead candidate, targeting ATTR,
marks a significant milestone as China's first LNP-mediated in vivo gene
editing therapy to enter clinical development. With promising early
clinical outcomes, YolTech is also advancing therapies for familial
hypercholesterolemia (FH) and primary hyperoxaluria type 1 (PH1).
As a company dedicated to transforming the treatment landscape,
YolTech continues to push the boundaries of what is possible in
gene editing.
For more information, please visit: www.yoltx.com
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SOURCE YolTech Therapeutics