FEATURE STORY ● The Alliance for Regenerative Medicine outlines recommendations to increase the number of European-based ATMP clinical trials GENE THERAPY ● Long-term correction of copper metabolism in WD mice with AAV8 vector delivering truncated ATP7b ● Enhancing the therapeutic potentiel of sulfamidase for the treatment of MPSIIIA DISRUPTIVE TECHNOLOGIES…..
How to increase the number of European-based ATMP clinical trials? The latest report published by the Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader advanced therapies sector, shows that Europe has become less competitive than other regions in attracting new advanced…..
New R&D agreement to expand Ziopharm's TCR-T Program Ziopharm Oncology, an immuno-oncology company focused on developing end-to-end cost-effective solutions using its non-viral Sleeping Beauty platform for TCR and CAR T-cell therapies and immune-stimulating gene therapy, has concluded a new R&D agreement with MD Anderson Cancer Center. This agreement is related…..
Transforming DNA repair errors into assets A new bioinformatics tool, MHcut, developed by researchers in Kyoto, Japan, and Montreal, Canada, reveals that a natural repair system for DNA damage, microhomology-mediated end joining, is probably far more common in humans than originally assumed. Using MHcut and commercial genome-editing technology, the researchers created…..
Accumulation of DNA mutations found in healthy liver leads to disease New insights into the journey from health to disease in the human liver have been made by scientists at the Wellcome Sanger Institute, Cancer Research UK Cambridge Institute, the University of Cambridge and their collaborators. In the largest study…..
Fourth FDA approval for Vertex's cystic fibrosis medicines The FDA has approved Trikafta™ (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of…..
New weapons against superbugs The rapid emergence of antimicrobial resistance is a matter of global concern. According to the WHO, particularly Gram-negative bacteria like Acinetobactebaumannii, Pseudomonas aeruginosa and Enterobacteriaceae that are resistant to the carbapenem and cephalosporin antibiotics, pose a growing threat to human health. These pathogens can cause severe and often life-threatening…..
BioPharmAnalyses and OctopusyX BioConsulting are proud to announce the launch of their up-dated and enriched 2019 edition of their report entitled “Landscape… in Gene Therapy Companies”. This report provides you with an in-depth analysis of more than 230 companies involved in this expanding field. (UPDATED SEP 2019) In…..
300 million people living with a rare disease worldwide An open-access article, written by Orphanet (coordinated by INSERM, the French National Institute of Health and Medical Research), EURORDISRare Diseases Europe and Orphanet Ireland (based at the National Rare Diseases Office, Mater Misericordiae University Hospital, Dublin, Ireland), confirms the number of…..
Convergence of digital technology and healthcare – digital health – has become a key driver of change for healthcare systems globally, with the mass adoption of digital technologies set to revolutionise healthcare and patient wellbeing. Payors and healthcare and life science companies must learn from the mistakes made in other…..