Long-term gene therapy study in dogs with hemophilia A; plus a comparison of HBV vaccine adjuvants and mAb that alleviates chemotherapy side effects

Long-term study of AAV gene therapy in dogs finds genome integration, clonal expansion
A team led by University of Pennsylvania scientists showed in Nature Biotechnology that adeno-associated viral (AAV8 or AAV9) gene therapy corrected factor VIII (FVIII) deficiency to 1.9-11.3% of normal FVIII levels and reduced bleeding events in nine dogs with hemophilia A at a follow-up of two to 10 years. The researchers analyzed integration sites in liver samples from six treated dogs and identified 1,741 unique AAV integration events in the genome and expanded cell clones in five dogs; 44% of the integrations were near genes involved in cell growth. No dogs showed evidence of tumors or altered liver function. 

GSK team compares immunogenicity of HBV vaccine adjuvants
A Science Translational Medicine study by GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) researchers revealed the cellular transcriptional profiles from subjects that received one of four HBV vaccines with different adjuvants, and found that three adjuvants induced a common gene signature after the second vaccination characterized by positive regulation of genes associated with interferon-related responses and by negative regulation of NK cell-associated genes. The adjuvant AS01_B stimulated the highest immunogenicity, attributed to its ability to induce the signature after the first vaccination in most recipients. AS03, the adjuvant used in the protein-based COVID-19 vaccine in development by GSK and Sanofi (Euronext:SAN; NASDAQ:SNY), induced the common signature but showed the highest interindividual response heterogeneity...