SHANGHAI, Sept. 4, 2024 /PRNewswire/ — YolTech Therapeutics, a clinical-stage in vivo gene editing company, announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to YOLT-203 for the treatment of primary hyperoxaluria type 1 (PH1) in September. Earlier that month, YOLT-203 received Rare Pediatric Disease Designation (RPDD) from the FDA.
After reviewing YolTech’s submission, the FDA determined that the therapy is not only appropriate for primary hyperoxaluria type 1 (PH1), but also applicable to the broader category of primary hyperoxaluria (PH). As a result, the designations were granted for PH, reflecting the expanded scope of the therapy’s potential impact. These dual designations underscore the transformative potential of YOLT-203 to address this rare and life-threatening genetic disorder.
About Primary Hyperoxaluria and PH1
Primary hyperoxaluria (PH) is a rare autosomal recessive genetic disorder in which excessive oxalate production in the liver causes kidney stone formation, progressive renal damage, and systemic oxalosis. The most common subtype of PH, primary hyperoxaluria type 1 (PH1), results from dysfunction of alanine-glyoxylate aminotransferase (AGT) due to mutations in the AGXT gene. In the absence of intervention, most patients progress to end-stage renal disease (ESRD), requiring intensive hemodialysis and often a dual liver-kidney transplant to correct the metabolic defect and address organ failure.
About YOLT-203
YOLT-203 is an innovative investigational in vivo gene editing therapy designated to provide a one-time, potentially curative treatment for primary hyperoxaluria type 1 (PH1). YOLT-203 permanently reduces excessive oxalate production in PH1 by deactivating the HAO1 gene through YolTech’s proprietary YolCas12™ system, a novel CRISPR/Cas enzyme identified through the High-Throughput Evolution Platform (HEPDONE®). YOLT-203 is administered through intravenous infusion of liver-targeted lipid nanoparticles (LNPs) encapsulating YolCas12 mRNA and a guide RNA (gRNA) targeting the HAO1 gene.
YOLT-203 also represents a historic milestone as the first in vivo gene editing therapy to enter clinical trial globally for a pediatric rare disease.
Clinical Development and Early Results
The investigator-initiated clinical study of YOLT-203 began on August 5, 2024, with the first adult patient dosed, followed by the first pediatric patient on August 20, 2024. Early data from preclinical and initial clinical studies have demonstrated promising reductions of over 90% in blood oxalate levels, with no dose-limiting toxicities (DLTs) or severe adverse events (SAEs) leading to treatment interruptions observed to date.
Significance of FDA Designations
The Rare Pediatric Disease Designation (RPDD) program encourages the development of new drugs and biologics for rare pediatric conditions. This designation makes YolTech eligible to receive a priority review voucher (PRV) upon potential FDA approval of YOLT-203. Similarly, the Orphan Drug Designation (ODD) program provides benefits including tax credits, exemption from certain FDA fees, and seven years of market exclusivity upon regulatory approval.
About YolTech Therapeutics
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, visit: www.yoltx.com or follow YolTech on LinkedIn: YolTech Therapeutics.
Contacts
Colin LIN
Business Development & Investor Relations
[email protected]
+86 180 2100 7750
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SOURCE YolTech Therapeutics
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