YolTech Therapeutics to Initiate a Clinical Trial for YOLT-204, a First-in-Class Bone Marrow-Targeted In Vivo Gene Editing Therapy for β-Thalassemia

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 [[email protected]].

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 

SOURCE YolTech Therapeutics