Inhaled Messenger RNA Treatment Fast-Tracked for Cystic Fibrosis
The Food and Drug Administration has granted Fast Track designation to MRT5005 (Translate Bio), an inhaled messenger RNA (mRNA)...
CFTR Amplifiers May Be Potent CF Mono or Combo Therapy, Study Says
Amplifiers, a potential new class of therapies that modify CFTR — the protein whose defects cause cystic fibrosis (CF) — do so by...
FDA Grants Fast Track Status to Translate Bio’s RNA-Based Therapy MRT5005
The U.S. Food and Drug Administration (FDA) has granted fast track designation to MRT5005, an investigational RNA-based treatment for...
New Gene Therapy Strategy May Reverse Effects of Disease-related Mutations, Study Shows
A new strategy to genetically prevent disease-related mutations from occurring shows the potential to treat several genetic diseases,...
Inhalable RNA could breathe new life into lung cancer treatments
Lung epithelial cells that have taken up the mRNA particles (yellow dots), which makes them glow green(Credit: Asha Patel) In the body,...
Vertex and Treating CF: High Hopes for Triple Combinations and Gene Therapy Possibilities
Editor’s note: This is third in a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we look at...
New Lung Cell Type Linked to Cystic Fibrosis Emerges From Mouse, Human Cell Atlas Studies
NEW YORK (GenomeWeb) – Two new studies that used single-cell RNA sequencing to profile tracheal or bronchial tissue from mice or humans...
Arcturus’ RNA Therapy for CF Shows Promise in Preclinical Studies, Company Announces
Arcturus Therapeutics’ RNA-based approach enables the production of healthy CFTR— the protein whose dysfunction is key in cystic fibrosis...
