Targeted constant-region pI shifts separate heterodimers from homodimers by ion exchange while helping extend antibody serum half-life.
A chimeric FVIII-VWF protein uses a short XTEN linker and selected VWF domains to extend half-life and reduce dosing frequency in hemophilia A.
Liver-targeted AAV delivery of codon-optimized ATP7B lowers circulating copper and may provide lasting Wilson's disease correction.
A covalently linked FVIII and VWF fragment blocks endogenous VWF binding to extend FVIII half-life and reduce dosing frequency in hemophilia A.
Specific FVIII mutations such as N2118Q and B-domain deletion reduce immunogenicity while improving expression, secretion, and vector packaging.
Charge-shifted IgG1 constant regions create pI gaps that separate heterodimers from homodimers and can also extend serum half-life.
Specific Factor VIII amino acid substitutions boost secretion and clotting activity, enabling lower vector doses for hemophilia A treatment.
A TRIS-free buffer with sucrose, surfactant, and salt helps lentiviral vectors resist freeze-thaw, agitation, and temperature stress.
Cell-free synthesis from nicked closed-ended DNA enables strand-specific single-stranded AAV vectors with higher purity and expanded transgene capacity.
A two-step affinity and anion exchange purification route improves chimeric FVIII purity and activity despite low expression and pH sensitivity.
Single-domain antibodies targeting the VWF D′D3 domain help clotting factor chimeras stay in circulation longer and reduce dosing frequency.
Engineered IDLVs use nuclease sites and homology arms to insert multi-kb sequences precisely with lower toxicity in stem cells.
Codon-optimized liver-targeted lentiviral FVIII vectors enable stable hepatocyte integration and higher plasma activity at lower doses.
ECM-binding peptides retain cytokines in tumor tissue, improving local therapeutic effect while limiting systemic toxicity and side effects.
Distinct constant-region pIs let charge-based methods separate IgG1 heterodimers from homodimers while supporting longer serum half-life.
Genetically modified CALLAR T cells target B cells producing Factor VIII inhibitors to sustain titer reduction and lower bleeding risk.
Cell-targeted promoter sequences sustain therapeutic FVIII expression while limiting immune responses and anti-FVIII antibodies in hemophilia A.