Innorna’s mRNA therapy granted US orphan drug designation for Wilson disease

April 28, 2025

Innorna Co. Ltd.’s investigational mRNA therapy, IN-013, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Wilson disease.

BioWorld Science Regulatory Endocrine/metabolic Genetic/congenital FDA Orphan drug

Today's news in brief

BioWorld

BioWorld briefs for May 21, 2025.

Today's news in brief

BioWorld MedTech

BioWorld MedTech briefs for May 21, 2025.

Neurobiogen divulges new MAO-B inhibitors for Parkinson’s disease

BioWorld Science

Neurobiogen Co Ltd. has synthesized monoamine oxidase-B (MAO-B) inhibitors reported to be useful for the treatment of Parkinson’s disease.

Scorpion Therapeutics patents p53 Y220 mutant activators

BioWorld Science

Work at Scorpion Therapeutics Inc. has led to the identification of cellular tumor antigen p53 (TP53) (Y220C mutant) activators reported to be useful for the...

Pfizer bets $6B+ on 3Sbio’s bispecific PD-1/VEGF antibody

BioWorld

Pfizer Inc. is paying $1.25 billion up front and up to $4.8 billion in milestone payments to gain global, ex-China rights to SSGJ-707, a PD-1/VEGF bispecific...