News and Trends 9 May 2023 Fabry disease treatment gets EU marketing authorization Chiesi Global Rare Diseases and Protalix BioTherapeutics, Inc. have announced that the European Commission (EC) has granted marketing authorization to PRX-102 (pegunigalsidase alfa) in the European Union (EU) for the treatment of adult patients with Fabry disease. “People living with Fabry disease often perceive their disease as burdensome and still experience unmet medical needs,” said […] May 9, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 21 Apr 2023 Orphalan announces U.S. launch of drug to treat Wilson disease Orphalan SA, an international orphan drug development and commercialization company, has announced the commercial launch in the U.S. for Cuvrior. Cuvrior is a new trientine tetrahydrochloride (TETA-4HCl). It is now available for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to D-penicillamine. Cuvrior was approved by the United States […] April 21, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
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 people around the world living with a rare disease. More than 6,000 rare diseases are characterized by a diversity of disorders and symptoms that vary not only from disease to disease, but also from patient […] February 28, 2023 - 8 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
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 RP-A501. RP-A501 is Rocket Pharmaceuticals’ investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. RMAT designation was […] February 10, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
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 various genome-editing repair options. Unintentional errors in the correction of DNA mutations of genetic diseases can thus be reduced. Genome editing technologies offer great opportunities for treating genetic diseases. Methods such as the widely used […] February 3, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
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 of exa-cel for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The submission is supported by two global phase 3 studies investigating exa-cel as a potential one-time therapy for people with […] January 26, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 9 Jan 2023 Selecta and Astellas sign LOPD treatment agreement Selecta Biosciences, Inc. and Astellas Pharma Inc. have announced an exclusive licensing and development agreement for IdeXork (Xork). Xork is being studied as a potential next generation immunoglobulin G (IgG) protease that will be developed by Astellas for use with AT845, an investigational, adeno-associated virus (AAV)-based treatment for late-onset Pompe disease (LOPD) in adults. Pompe […] January 9, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
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 (FDA) to advance NMD670 into a phase II clinical study in patients of spinal muscular atrophy (SMA) type 3. NMD Pharma is a Danish clinical stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for […] December 14, 2022 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
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 Foundation is a not-for-profit organization focused on the development of a treatment for an ultra-rare genetic disorder. Variants of the KCNC1 gene impact Kv3.1 potassium channel function, resulting in neurodevelopmental disorders, which can include progressive […] December 8, 2022 - 2 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
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 constantly fuse together and split apart. They contain a small amount of genetic information, mitochondrial DNA (mtDNA). The mtDNA, organized into dot-like structures called nucleoids, also moves around inside the mitochondria. The method of distribution […] November 17, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
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 spontaneously mutate – causing potentially permanent genetic errors, according to new research from the University of Surrey in the U.K. Using quantum chemical calculations, researchers from Surrey’s Quantum Biology Doctoral Training Centre have found that […] November 16, 2022 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 10 Aug 2022 Aro Biotherapeutics gets FDA orphan drug designation for treatment of Pompe Disease Aro Biotherapeutics says the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in the muscle, for the treatment of Pompe disease. “We are pleased to have received this designation and are gratified by the FDA’s recognition of the potential of ABX1100 […] August 10, 2022 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email