Rare & Genetic Diseases

Passage Bio plans Phase 1/2 trial of gene therapy in humans based on preclinical results

RNA-targeting gene therapy reverses molecular and functional features of DM1 in mice

Normal cell and tissue physiology is complex, even without pathologies that arise from myriad sources and impact multiple pathways. Rather than focusing on individual molecular entry points, phenotypic imaging enables a broader understanding of disease outcomes. This webinar will examine advances in imaging and high-content screening modalities to facilitate such understanding.

Recent advances push the gene-editing platform from analytic resource to therapeutic solution.

Healx and Muscular Dystrophy UK join forces to find new treatments for rare muscle-wasting diseases

QED Therapeutics doses first child in Phase 2 clinical trial of the investigational medicine infigratinib in achondroplasia

Catalyst Biosciences begins pivotal Phase 3 study design for MarzAA in individuals with Hemophilia A or B with inhibitors

Start Codon joins Cancer Research UK’s Entrepreneurial Programmes Initiative

Regeneron reports encouraging garetosmab Phase 2 results in patients with ultra-rare bone disease

ACADIA Pharmaceuticals and Neuren Pharmaceuticals announce Rare Pediatric Disease designation for trofinetide for the treatment of Rett syndrome