da Luca Bonadei | Dic 4, 2019 | Business
https://www.fiercebiotech.com Astellas Pharma is the latest on a growing list of companies to see gene therapies in their future, as it has agreed to acquire Audentes Therapeutics for $3 billion. Audentes uses adeno-associated viruses (AAVs) to deliver DNA, primarily...
da Luca Bonadei | Set 4, 2019 | Clinical
https://hemophilianewstoday.com UniQure has achieved its target patient enrollment goal ahead of schedule for its ongoing Phase 3 HOPE-B trial; the trial is assessing the safety and efficacy of AMT-061, the company’s investigational gene therapy for the treatment of...
da Luca Bonadei | Set 4, 2019 | Clinical
https://www.globenewswire.com Ultragenyx Pharmaceutical, a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, announced positive data from the second dose cohort of the ongoing Phase 1/2 study of...
da Luca Bonadei | Set 4, 2019 | Clinica
https://www.globenewswire.com Ultragenyx Pharmaceutical, azienda biofarmaceutica focalizzata nello sviluppo di terapie innovative per il trattamento di patologie genetiche ultra rare, ha annunciato l’ottenimento di dati positivi dalla seconda coorte di uno studio...
da Luca Bonadei | Set 4, 2019 | Regulatory
https://pharmaphorum.com Orchard Therapeutics said it planned to file data from its OTL-200, gene therapy for the life-threatening neurodegenerative rare disease metachromatic leukodystrophy, with regulators early next year in Europe after results from a late-stage...