Klotho’s ALS candidate gains US orphan drug designation

July 10, 2025

The U.S. FDA has granted orphan drug designation to Klotho Neurosciences Inc.’s secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202 or s-KL-AAV.myo) for the treatment of amyotrophic lateral sclerosis (ALS).

BioWorld Science Regulatory Neurology/psychiatric Gene therapy FDA Orphan drug

Today's news in brief

BioWorld

BioWorld briefs for Dec. 4, 2025.

Today's news in brief

BioWorld MedTech

BioWorld MedTech briefs for Dec. 4, 2025.

UK Biobank delivers largest metabolomics dataset of 500K profiles

BioWorld Science

It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its...

Epigenetic technology could eliminate misfolded prion proteins

BioWorld Science

The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal...

Mesothelin is biomarker, potential target in arthritic bone damage

BioWorld Science

In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences...