News and Trends 28 Feb 2023What’s happening on Rare Disease Day? Rare Disease Day 2023 is on February 28. The day is dedicated to raising awareness for the approximately 300 million… February 28, 2023 - 8 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 10 Feb 2023Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment Rocket Pharmaceuticals, Inc says the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to… February 10, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 3 Feb 2023AI boosts genome editing Researchers at the University of Zurich have developed a new tool that uses artificial intelligence to predict the efficacy of… February 3, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 26 Jan 2023Milestone for gene-edited therapy for sickle cell disease Vertex Pharmaceuticals (Europe) and CRISPR Therapeutics have announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application… January 26, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 14 Dec 2022NMD Pharma cleared for spinal muscular atrophy trial NMD Pharma A/S has received clearance for its investigational new drug (IND) application from the US Food and Drug Administration… December 14, 2022 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 8 Dec 2022Metrion and KCNC1 Foundation announce collaboration to identify therapeutic Metrion Biosciences Limited, and The KCNC1 Foundation are to collaborate to try and find a therapeutic for children. The KCNC1… December 8, 2022 - 2 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
News and Trends 17 Nov 2022New mitochondria study could pave way for disease treatments Mitochondria, the ‘powerhouses of the cell,’ are important cellular structures that are vital for their role of generating energy. They… November 17, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 16 Nov 2022Enzymes could be key to understanding ‘origin of DNA errors’ Enzymes, critical to controlling how cells replicate in the human body, could be the very ingredient that encourages DNA to… November 16, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email