YOLT-203
- Generic Name
- YOLT-203
YolTech's YOLT-203 Shows Promise in Primary Hyperoxaluria Type 1 with Positive Clinical Data
• YolTech Therapeutics reported positive clinical data for YOLT-203, an in vivo gene editing therapy, demonstrating excellent safety and pharmacodynamic profiles in PH1 patients.
• YOLT-203 achieved nearly 70% reduction in 24-hour urinary oxalate levels in high-dose patients, sustained through the 16-week primary observation period.
• The FDA previously granted YOLT-203 Orphan Drug and Rare Pediatric Disease Designations, highlighting its potential to address this rare and life-threatening genetic disorder.
• YOLT-203 is the first in vivo gene-editing therapy to show positive clinical data for PH1, marking a significant advancement in treating this rare genetic disorder.
Gene Editing Advances: Intellia, Poseida, YolTech, and KSQ Therapeutics Report Clinical Trial Updates
• Intellia Therapeutics has initiated a pivotal Phase 3 trial (HAELO) of NTLA-2002 for hereditary angioedema, evaluating its ability to reduce HAE attacks through plasma kallikrein activity reduction.
• Poseida Therapeutics announced positive interim Phase 1 data for P-BCMA-ALLO1 in heavily pre-treated multiple myeloma patients, demonstrating a 91% overall response rate.
• YolTech Therapeutics received Orphan Drug Designation for YOLT-203, an in vivo gene-editing therapeutic for primary hyperoxaluria type 1, aiming to correct AGXT mutations.
• KSQ Therapeutics received FDA clearance for its IND application for KSQ-004EX, a CRISPR-Cas9 engineered TIL therapy, to treat advanced solid tumors by targeting SOCS1 and Regnase-1.
First Patients Dosed in Phase 1 Trial of In Vivo CRISPR Therapy YOLT-203 for Primary Hyperoxaluria Type 1
• YolTech Therapeutics has dosed the first two patients in a Phase 1 trial of YOLT-203, a novel in vivo CRISPR gene-editing therapy for primary hyperoxaluria type 1 (PH1).
• YOLT-203 utilizes the YolCas12 system to target and correct mutations in the AGXT gene within liver cells, aiming to reduce oxalate production and provide a one-time curative treatment.
• The trial, sponsored by RenJi Hospital in Shanghai, will assess the safety, tolerability, and preliminary efficacy of YOLT-203 in Chinese individuals with PH1.
• Pre-clinical studies have demonstrated high gene-editing activity of YolCas12 in both prokaryotic and eukaryotic cells, as well as efficient in vivo editing in animal models.
Drug Development Updates
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