SHANGHAI, Sept. 4, 2024 — 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 treating primary hyperoxaluria type 1 (PH1) in September. Earlier that month, the FDA also granted YOLT-203 Rare Pediatric Disease Designation (RPDD).
Following review, the FDA concluded YOLT-203’s suitability extends beyond PH1 to encompass primary hyperoxaluria (PH) generally. Therefore, the designations cover PH, acknowledging the therapy’s broader potential. These designations highlight YOLT-203’s potential to revolutionize treatment for this rare, life-threatening genetic disorder.
About Primary Hyperoxaluria and PH1
Primary hyperoxaluria (PH) is a rare, autosomal recessive genetic disorder. Excessive liver oxalate production leads to kidney stones, progressive kidney damage, and systemic oxalosis. Primary hyperoxaluria type 1 (PH1), the most prevalent subtype, stems from alanine-glyoxylate aminotransferase (AGT) dysfunction due to AGXT gene mutations. Without intervention, most patients develop end-stage renal disease (ESRD), requiring hemodialysis and often a liver-kidney transplant.
About YOLT-203
YOLT-203 is a novel investigational in vivo gene editing therapy. It aims to offer a one-time, potentially curative treatment for PH1. YOLT-203 permanently reduces excessive oxalate production by deactivating the HAO1 gene using YolTech’s proprietary YolCas12
system, a novel CRISPR/Cas enzyme from the High-Throughput Evolution Platform (HEPDONE®). Administered intravenously, it uses liver-targeted lipid nanoparticles (LNPs) carrying YolCas12 mRNA and a guide RNA (gRNA) targeting HAO1.
YOLT-203 is also the first in vivo gene editing therapy globally to enter clinical trials for a pediatric rare disease.
Clinical Development and Early Results
The investigator-initiated YOLT-203 clinical study started on August 5, 2024, dosing the first adult patient, followed by the first pediatric patient on August 20, 2024. Early preclinical and clinical data show promising oxalate level reductions (over 90%), with no dose-limiting toxicities (DLTs) or severe adverse events (SAEs) causing treatment interruptions.
Significance of FDA Designations
The Rare Pediatric Disease Designation (RPDD) program promotes rare pediatric disease drug development. It makes YolTech eligible for a priority review voucher (PRV) upon potential FDA approval. The Orphan Drug Designation (ODD) program offers benefits including tax credits, fee exemptions, and seven years of market exclusivity after approval.
About YolTech Therapeutics
YolTech Therapeutics is a clinical-stage in vivo gene editing company focused on developing next-generation precision genetic medicines. Our approach combines innovative gene editing with advanced LNP delivery, creating a versatile platform for various serious diseases. We prioritize internal capabilities, including manufacturing, for quality and scalability. Our lead candidate (targeting ATTR) is China’s first LNP-mediated in vivo gene editing therapy in clinical development. With promising early results, YolTech is also developing therapies for familial hypercholesterolemia (FH) and PH1.
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Contacts
Colin LIN
Business Development & Investor Relations
+86 180 2100 7750
SOURCE YolTech Therapeutics