An investigator-initiated trial examining YolTech Therapeutics’ in vivo gene editing-based ‘life-long cure’ for patients living with a rare genetic disorder has seen the treatment able to normalise the chemicals responsible.
The YOLT-203 in-vivo gene editing therapy was developed to treat patients living with primary hyperoxaluria type 1 (PH1), a condition caused by a genetic deficiency in the liver enzyme alanine-glyoxylate aminotransferase (AGT). This results in the formation of crystals in the liver, leading to kidney failure. The condition typically manifests in childhood with symptoms such as kidney stones and blood in urine.
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Results from the early Phase I (formerly known as Phase 0) trial (NCT06511349) saw YOLT-203 normalise urinary oxalate levels whilst being safe and well tolerated in all seven patients enrolled on the single-arm trial.
The 52-week trial, run by Renji Hospital in Shanghai, China, saw a nearly 70% drop in 24-hour urinary oxalate levels through to week 16, a marker of PH1. At the same time, there were no reported serious adverse events (AEs). The company added that the therapy was designed using the company’s proprietary CRISPR/Cas gene-editing system, YolCas12HF.
YolTech Therapeutics founder Yuxuan Wu said: “The clinical results of YOLT-203 represent a breakthrough in the field of in vivo gene-editing therapies. In this clinical trial, we have proven the potential efficacy of our proprietary gene-editing system YolCas12HF, a system with enhanced editing precision and efficiency compared to conventional platforms.
“It marks the first full-cycle innovation by a Chinese team, spanning from discovery and optimisation to clinical translation of next-generation in vivo gene-editing drugs. We fully understand the urgent need for patients with rare diseases to have a treatment addressing the root of their diseases. We believe one-time treatments for lifelong cure will redefine the standard of care for rare diseases globally.”
YOLT-203 received both orphan drug and rare pediatric disease designations from the US Food and Drug Administration (FDA) in September 2024.
In terms of competition, YolTech is entering a relatively empty market with only two options available, Novo Nordisk’s Rivfloza (nedosiran) and Alnylam’s Oxlumo (lumasiran). Research by GlobalData found that the international market for PH1 brought in approximately $167m last year. By the end of 2030, the market is set to bring in $693m annually, with Oxlumo set to bring in $381m of that amount while Rivfloza will earn $242m.
GlobalData is the parent company of Clinical Trials Arena.
Elsewhere in the field of gene therapies, Boehringer Ingelheim has initiated the Phase I/II LENTICLAIR 1 trial of its inhaled treatment for cystic fibrosis. Meanwhile, Solid Biosciences’ gene therapy candidate for patients with Duchenne muscular dystrophy (DMD) has elicited a 110% expression of the microdystrophin gene.
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