The negative control vector AV.AmpOri is similar to AV.SupEnh ... for rAAV delivery of therapeutic transgenes (such as cystic fibrosis transmembrane conductance) that are near the maximum ...

Figure 3: Characterization of the split AAV vector heterodimerization by RT ... hemophilia A and cystic fibrosis.

In January 2023, Asklepios BioPharmaceutical entered a research partnership with ReCode to explore a single-vector gene-editing platform. "ReCode to secure funds for cystic fibrosis gene ...

The focus of newly formed partnership will be on a novel approach using a replication-deficient lentiviral vector in an inhaled formulation, to introduce a healthy copy of the cystic fibrosis ...

Edited by: Stephen P. Hoffert M.R. Knowles, K.W. Hohneker, Z. Zhou, J.C. Olson, T.L. Noah, P. Hu, M.W. Leigh, J.F. Engelhardt, L.J. Edwards, K.R. Jones, M. Grossman ...

4DMT will look to begin the Phase II dose expansion stage in its AEROW clinical trial after the biopharma reported positive interim data for its gene therapy 4D710. 4DMT is evaluating its asset 4D ...