Primary hyperoxaluria (PH)

YOLT-203 Awarded EMA Orphan Drug Designation for...

Jul. 30, 2025

YolTech Therapeutics announced earlier this week that the European Medicines Agency has granted Orphan Drug Designation to YOLT-203, its...

Single-Dose Therapeutic Genome Editing Using Lipid...

CMN Interview

Mar. 17, 2025

Scientists used lipid nanoparticles to deliver CRISPR-Cas9 targeting Hao1 for the treatment of primary hyperoxaluria type 1. Proof-of-concept studies...

The Latest Updates From the Gene-Editing Clinical...

CMN Clinical

Feb. 26, 2025

YolTech Therapeutics reports positive clinical data for the first in vivo gene-editing therapy in primary hyperoxaluria type 1, Precision BioSciences...

FDA Grants Dual Designations to Arbor's Gene...

CMN Clinical

Feb. 12, 2025

The FDA has granted orphan drug and rare pediatric disease designations to Arbor Biotechnologies' ABO-101, a gene-editing therapeutic candidate for...

Non-viral CRISPR Therapy Alleviates Symptoms in...

CMN Briefs

Oct. 14, 2024

Researchers in China report pre-clinical in vivo data demonstrating the potential of a non-viral CRISPR-Cas9 therapy to treat primary hyperoxaluria 1....

The Latest Updates From the Gene-Editing Clinical...

CMN Clinical

Oct. 9, 2024

In this clinical update, we bring you the latest news from four gene-editing trials sponsored by RenJi Hospital (Shanghai), The University of Texas MD...

Dosing Underway in World’s First in Vivo CRISPR...

CMN Clinical

Aug. 28, 2024

YolTech Therapeutics announced last week that the first two patients have been dosed in a Phase 1 trial of YOLT-203, an in vivo CRISPR-based...