Patients with TDT need blood transfusions regularly for anaemia management to avoid complications in the future. Credit: JOURNEY STUDIO7 / Shutterstock.

YolTech Therapeutics is set to commence a trial of its in vivo gene editing therapy, YOLT-204, aimed at treating transfusion-dependent beta-thalassemia (TDT).

TDT is a genetic blood disorder characterised by beta-globin gene mutations, resulting in a decrease or absence of haemoglobin. Affected patients need blood transfusions regularly for anaemia management to avoid complications in the future.

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The dose-escalation trial is designed to assess the efficacy and safety of a single-dose regimen with the therapy, which may offer an off-the-shelf treatment for individuals with this condition. This can potentially eliminate the need for conditioning chemotherapy and haematopoietic stem cell transplantation (HSCT), on the success of the trial.

YolTech Therapeutics CEO and founder Dr Yuxuan Wu said: “The initiation of the clinical trial for YOLT-204 represents a significant milestone of gene editing therapy development for TDT and SCD [Stearoyl-CoA Desaturase]. We are excited to collaborate with our clinical investigators to bring this innovative therapy to patients.”

YOLT-204 utilises the company’s lipid nanoparticles (LNP) and edits the haemoglobin regulatory area for inducing foetal haemoglobin expression.

With this process, it may be possible to correct the haemoglobin imbalance and red blood cell deficiency in individuals with TDT. Pre-clinical models suggest that the therapy has exhibited ‘sustained’ and ‘effective’ foetal haemoglobin induction.

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YolTech develops precision genetic medicines. Combining gene editing with an LNP delivery system, the company aims to tackle serious conditions. It is also focused on building internal capabilities, including end-to-end manufacturing, to maintain scalability.

The company is also advancing therapies for primary hyperoxaluria type 1 (PH1) and familial hypercholesterolemia (FH).

Last July, the company dosed the first subject in a Phase I trial of YOLT-201, which targets rare genetic diseases.