技术创新

IP Outlicensing GVP

At VectorBuilder, we push the boundaries of innovation to provide cutting-edge solutions that propel research and development in cell and gene therapy through solving every bottleneck in gene delivery. Our innovations are centered around the three cornerstones of cell and gene therapeutics: Efficacy, Safety, and Manufacturability. De-risk drug development with our time- and cost-effective solutions that boost therapeutic efficacy and efficiency, improve safety profiles, and are primed for clinical and commercial use.

Precision Capsids

AAV-based gene therapies are often constrained by poor transduction efficiency and off-target delivery, necessitating higher doses that escalate both cost and toxicity. VectorBuilder’s innovative capsid technology provides a powerful solution: engineered capsids with enhanced targeting specificity and maximum gene delivery performance, enabling safer, more effective, and more efficient AAV-based therapeutics.

AAV Precision Capsids
Brain

Optimized for maximum efficiency and streamlined delivery across the blood-brain barrier.

Highlights
  • Delivery via systemic injection
  • Efficiently penetrates blood-brain barrier
  • Validated in BALB/c and C57BL/6 mouse models
  • Substantially reduced off-target expression in liver
Ocular

Our ocular capsid boasts excellent pan-retinal penetration and peripheral spread, with superior transduction efficiency in the retinal pigment epithelium (RPE).

Highlights
  • Delivery via intravitreal injection
  • Exceptional penetration and efficiency across all retinal layers, especially in RPE
  • Validated in non-human primates (NHPs)
Muscular

VectorBuilder’s novel capsids significantly enhance targeting and efficiency to skeletal, cardiac, and diaphragm muscles.

Highlights
  • Delivery via systemic injection
  • Improved transduction efficiency in muscle
  • Minimized off-target expression

Proprietary Delivery Systems

IP outlicensing Proprietary Delivery Systems

The potential of next-generation therapeutics is restrained by traditional gene delivery approaches, which face limitations such as limited payload capacity, unwanted immune responses, complicated scale-up processes, and especially for AAV-based systems, vector instability. Our cutting-edge technologies overcome these challenges with advanced gene delivery systems engineered for enhanced efficacy, improved versatility, and robust scalability, opening up unlimited possibilities for therapeutic drug delivery.

MiniVec™

VectorBuilder's proprietary MiniVec™ plasmid offers a miniaturized backbone with higher effective yields and translation efficiency. This antibiotic- and supplement-free system streamlines drug development, providing remarkable efficacy, safety, and manufacturability for cell and gene therapies.

MuteFree™ AAV

Our novel MuteFree™ AAV backbone has been designed, built, and validated to generate AAV vectors with highly stable ITRs. This results in therapeutic gene products with maximum stability for consistent and high-yield manufacturing.

MegaAAV™

Breaking through boundaries of traditional AAV systems, VectorBuilder’s innovative and highly efficacious MegaAAV™ system allows for expression of large genes or complex multi-component systems.

Enhanced Vector Components

Effective gene delivery is often hindered by fundamental vector limitations, including payload capacity (particularly in AAV vectors), suboptimal gene expression, and poor recombination efficiency. Our innovative vector components address these through shortened promoters that enable more efficient use of limited vector space and optimized coding sequences that drive more robust, consistent transgene expression and enable precise control of gene expression. Leverage our technologies to unlock the full potential of your research for precise, high-performance gene therapies.

AAV Enhanced Vector Components

Shortened Promoters

Photoreceptor-specific

Our shortened photoreceptor promoters are not only significantly smaller, but also boast higher specificity to target cells. All promoters have been validated in mice and/or NHPs. Promoters include high specificity to cone cells (<270 bp), rod cells, and all photoreceptors (200-500 bp).

Beta-globin-specific

Supporting development of gene therapies targeting the human beta-globin (HBB) gene with our novel HBB specific promoter (shortened to <1100 bp).

Ubiquitous

Ideal for use in vector backbones with smaller carrying capacity, our shortened ubiquitous promoter (200-400 bp) allows for design of vectors with larger ORFs and markers without sacrificing viral titer or gene expression levels.

Optimized Coding Sequences

mCherry

Providing even more robust expression of mCherry for a wide variety of applications in vitro and in vivo.

Cas9

Our codon-optimized HiExpress™ hSpCas9 IVT mRNA ensures consistent, high expression for quick and efficient gene editing.

Cre/Lox

Achieve precise control of gene expression using our novel optimized Cre/Lox alongside HiExpress™ Cre for efficient recombination. HiExpress™ Cre is available in two delivery formats:

Explore our full HiExpress™ lineup of codon-optimized RNA products, designed to elevate your discoveries.

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