A Free DNA Software
AAV-based gene therapies are often constrained by poor transduction efficiency in target cells and off-target delivery, necessitating higher doses that escalate both cost and toxicity. VectorBuilder’s innovative capsid engineering technology provides a powerful solution: novel capsids with enhanced specificity and optimal delivery, enabling safer, more efficient, and more effective AAV-based therapeutics.
VectorBuilder’s advanced DeepCap™ capsid engineering platform integrates several proprietary technologies spanning design, packaging, and screening to develop next-generation AAV capsids with enhanced tissue targeting, transduction efficiency, and manufacturability.
Validated for performance and manufacturability, our novel capsids enhance the therapeutic potential of cell and gene therapies across multiple target tissues.
Our ocular capsids, C-PR4 and C-PR5, boast excellent pan-retinal penetration and peripheral spread, with superior transduction efficiency in the retinal pigment epithelium (RPE).
Our novel capsid, C-M1, markedly improves tissue targeting and transduction efficiency in cardiac and skeletal muscle, including the diaphragm. Additionally, C-M1 also demonstrates robust transduction efficiency in the dorsal root ganglion, enabling development of therapies for neuromuscular and restorative nerve applications.
VectorBuilder’s C-B1 capsid is engineered for optimal efficiency, enabling streamlined delivery with robust penetration of the blood-brain barrier and broad targeting of the central nervous system (CNS) and motor neurons.
Figure 1. VectorBuilder’s novel ocular capsids achieve superior transduction across all retinal layers in NHPs. CAG>mCherry constructs were packaged in four separate capsids and administered to 2-year old crab-eating macaques by intravitreal injection (C-PR4, C-PR5, Capsid R: 7.5 × 1011 GC/eye; AAV2.7m8: 1.5 × 1012 GC/eye). Samples were harvested four weeks post-injection. (A) Representative macular sections were imaged (200X, 300 ms) and (B) mCherry expression was quantified across retinal layers.
Figure 2. VectorBuilder’s ocular capsids exhibit superior pan-retinal penetration and peripheral spread in mice. CAG>mCherry constructs were packaged in four separate capsids and administered to 6-8 week old C57BL/6J mice by intravitreal injection (8 × 109 GC/eye). Samples were harvested 4 weeks post-injection and representative fluorescence images were taken (40X, 2000 ms; 200X, 800 ms).
Figure 3. VectorBuilder’s muscular capsid enables specific, efficient muscle transduction in NHPs. CAG>mCherry constructs were packaged in separate capsids and administered to 2-year old crab-eating macaques by intravenous injection (7.5 × 1013 GC/kg). Samples were harvested 4 weeks post-injection and representative fluorescence images were taken (tibialis anterior muscle, diaphragm, cardiac muscle: 10X, 400 ms; liver: 10X, 500 ms).
Figure 4. VectorBuilder’s novel capsid exhibits significantly higher transduction efficiency in dorsal root ganglion (DRG). CAG>mCherry constructs were packaged in separate capsids and administered to 2-year old crab-eating macaques by intravenous injection (7.5 × 1013 GC/kg). Samples were harvested 4 weeks post-injection and representative fluorescence images were taken (10X, 800 ms).
Figure 5. VectorBuilder’s C-B1 capsid demonstrates superior targeting and transduction efficiency of the CNS. CAG>EGFP constructs were packaged in separate capsids and administered to 5-week old Balb/c mice by intravenous injection (1.5 × 1012 GC/mouse). Samples were harvested 3 weeks post-injection and representative fluorescence images were taken (motor cortex, hippocampus, thalamus and hypothalamus, cerebellum: 10X, 50 ms; lumbar spine, liver: 10X, 200 ms).
Figure 6. VectorBuilder’s C-B1 capsid demonstrates superior targeting and transduction efficiency of the CNS. CAG>EGFP constructs were packaged in separate capsids and administered to 5-week old C57BL/6 mice by intravenous injection (1.5 × 1012 GC/mouse). Samples were harvested 3 weeks post-injection and representative fluorescence images were taken (motor cortex, hippocampus, thalamus and hypothalamus, cerebellum: 10X, 50 ms; lumbar spine, liver: 10X, 200 ms).
