The U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy Designation to NS Pharma Inc.’s NS-089/NCNP-02, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy. The breakthrough designation is based on results from a first-in-human, investigator-initiated clinical trial conducted in Japan. In June 2023, NS-089/NCNP-02 was granted Rare […]
DTx Pharma, a preclinical stage biotechnology company addressing the delivery challenges of oligonucleotide therapeutics with its fatty acid ligand conjugated oligonucleotide (FALCON) platform, has been acquired by Novartis. The FALCON platform enables the delivery and activity of small interfering RNA (siRNA) therapeutics to tissues beyond the liver, enhancing biodistribution and cellular uptake. DTx Pharma’s lead […]
Sarepta Therapeutics, Inc. has announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an Adeno-associated virus (AAV)-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]