YolTech Receives U.S. FDA ODD for YOLT-203 in Treating PH1

SHANGHAI, September 23 — YolTech Therapeutics, a pioneering gene editing company focused on developing in vivo gene editing medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to YOLT-203 for the treatment of Primary Hyperoxaluria Type 1 (PH1). Earlier this month, YOLT-203 received Rare Pediatric Disease Designation (RPDD) from the FDA.

After reviewing YolTech's submission, the FDA determined that the therapy is appropriate for treating Primary Hyperoxaluria (PH) and noted that the proposed PH1 designation falls within this broader disease category. Consequently, the FDA officially granted the ODD&RPDD to YOLT-203. 

Primary hyperoxaluria is a rare autosomal recessive genetic disorder characterized by hepatic overproduction of oxalate, which leads to the formation of kidney stones and progressive renal failure. PH1, the most common and severe subtype of the disease, is caused by mutations in the AGXT gene, resulting in the absence or dysfunction of the enzyme alanine-glyoxylate aminotransferase (AGT). Without treatment, most patients eventually progress to end-stage renal disease (ESRD), necessitating intensive hemodialysis and often dual liver-kidney transplants. 

YOLT-203, a first-in-class in vivo gene editing therapy, is designed to specifically target and correct the genetic mutations underlying PH1 using YolTech’s proprietary YolCas12™ technology. This innovative therapy is delivered via lipid nanoparticles (LNPs) to liver cells, where it aims to reduce oxalate overproduction at its source, potentially offering a curative approach to PH1. YOLT-203 is the first in vivo gene editing therapy developed for PH1 and is currently undergoing clinical trials. It also represents a significant milestone as the first-ever in vivo gene editing clinical trial globally for a pediatric rare disease. 

The investigator-initiated clinical study of YOLT-203 commenced on August 5, 2024, with the first adult patient dosed, followed by the first pediatric patient on August 20, 2024. Early data has shown promising reductions in oxalate levels, offering hope to patients who have had limited treatment options. 

The ODD program is designed to encourage the development of treatments for rare diseases affecting fewer than 200,000 people in the United States. This designation qualifies YolTech for various development incentives, including potential eligibility for tax credits, exemption from FDA application fees, and seven years of market exclusivity upon approval.

About YOLT-203

YOLT-203 is an innovative in vivo gene editing therapy designed using YolTech's proprietary YolCas12™ system, a novel CRISPR/Cas tool developed through the High-Throughput Evolution Platform (HEPDONE®). This therapy is encapsulated within lipid nanoparticles (LNPs) that facilitate targeted delivery to the liver, where it addresses the underlying genetic mutations responsible for primary hyperoxaluria type 1 (PH1). Upon intravenous administration, the LNPs are recognized and internalized by liver cells. Once inside, the YolCas12™ editor protein, guided by the corresponding guide RNA (gRNA), targets and corrects mutations in the AGXT gene, aiming to reduce harmful oxalate levels in the blood, thereby offering a potential one-time, curative treatment for PH1.

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.