The Massachusetts-based biotech plans to use the funds to push its candidates into mid-stage clinical trials in a space ...
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 ...
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 ...
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 ...
Grossman, J.M. Wilson, L.G. Johnson, R.C. Boucher, "A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis," New England Journal of ...
Figure 3: Characterization of the split AAV vector heterodimerization by RT ... hemophilia A and cystic fibrosis.
"The paper focuses on the use of an adenoviris vector to transfer a normal CFTR cDNA [complementary DNA] to the respiratory epithelium [inner lining] of individuals with CF," explains Ronald G.