News and Trends 28 Feb 2023
What’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…
genetic disorders
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News and Trends 10 Feb 2023
Rocket 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…
News and Trends 3 Feb 2023
AI boosts genome editing
Researchers at the University of Zurich have developed a new tool that uses artificial intelligence to predict the efficacy of…
News and Trends 26 Jan 2023
Milestone 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…
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News and Trends 14 Dec 2022
NMD 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…
News and Trends 8 Dec 2022
Metrion 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…
News and Trends 17 Nov 2022
New 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…
News and Trends 16 Nov 2022
Enzymes 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…
