Latest Updates
Stay informed on regulatory milestones, clinical trials, and breakthrough research
FDA
2025-09-15FDA Grants Rare Pediatric Disease Designation for PTC Therapy
Novel suppressor tRNA approach receives regulatory milestone for Duchenne muscular dystrophy treatment.
Read moreTrials
2025-09-01Phase 2 Clinical Trial Initiated for Cystic Fibrosis PTC Patients
Multi-center study enrolling 120 patients with nonsense mutations in CFTR gene.
Read morePubMed
2025-08-22Nature: AI-Designed tRNAs Show 40% Improved Suppression Efficiency
Machine learning models predict context-dependent suppressor tRNA performance in mammalian cells.
Read moreEMA
2025-08-10EMA Issues Draft Guidance on tRNA Therapeutics
European regulatory framework addresses safety, manufacturing, and clinical endpoints for genetic medicines.
Read morePubMed
2025-07-28Cell: Translation Small-World Networks Reveal Drug Targets
Systems biology approach identifies 23 novel intervention points in cellular translation machinery.
Read moreTrials
2025-07-15India Launches National Rare Disease Registry
Government initiative aims to catalog genetic variants across all states, enabling precision medicine.
Read more