Gene Therapy

Gene therapy offers opportunities for treating a wide range of diseases that were historically considered untreatable. This includes various types of cancers, hematopoietic disorders, diabetes, and hereditary conditions including rare diseases. In vivo genetic modification or gene altering therapy works by replacing, inactivating, or introducing genes into patients using viral vectors such as adeno-associated virus (AAV) or nanoparticles. Viral vector optimization and selection of the best AAV serotypes is critical for a successful gene therapy program.

Advances in gene editing technologies have opened new avenues for more precise genetic engineering to treat monogenic disorders. By correcting the aberrant gene sequence at the core of the ailment, these therapies are guiding the way toward precision medicine.

Viral Vector Development and Manufacture

Viral vectors are complex biological drug delivery vehicles comprising the capsid which surrounds the engineered DNA payload. Viral vector designs allow for optimized tissue tropism and delivery to the target tissue, improving transduction rates and appropriate gene expression levels, while at the same time reducing immunogenicity and enabling high yield production. Viral targeting can further be enhanced by redirecting the vector capsid to specific cellular receptors using molecular adaptors, such as bispecific antibodies or nanobodies.

  • Engineer and validate the best-in-class viral vectors to elevate your gene therapy program with service support from discovery to regulatory approvals while addressing key clinical requirements
  • Quickly and easily pioneer novel approaches to deliver viral vectors with consistent performance, optimal dose, and drug efficacy using our proprietary solutions for vector development and manufacture
  • De-risk cell and gene therapy programs using our robust platform processes for manufacturing high quality and high titre preclinical batches for up to 50L

Viral Vector Quantification and Characterization

Critical Quality Attributes (CQAs) for viral vectors require robust methods to enable successful phase-to-phase transition during the drug development cycle. To minimize lot-to-lot variability in upstream manufacturing of recombinant viral vectors, extensive characterization, quality control measures, and empty capsid reference standards need to be implemented.     

  • Evaluate and document CQAs by measuring viral identity, potency, purity, safety, and stability
  • Rapidly separate, characterize and quantify viral proteins and detect contaminants using our automated, microfluidic, capillary electrophoresis system for protein analysis
  • Accelerate vector optimization with our range of high-speed, homogeneous, no-wash capsid detection immunoassay platforms 

Genome Editing

The discovery of the CRISPR-Cas9 gene editing system has redefined our approach to gene therapy and revolutionized biotherapeutic development. Genome editing has since undergone rapid enhancements and become an essential tool for modern therapeutics. 

The recent advent of base-editing technology, which avoids double stranded breaks, resulting in less off-target editing, and reduced frequency of genomic rearrangement, has paved the way for a new generation of safer, more potent, and efficacious biotherapeutics.

  • Modify DNA sequences with a high degree of precision and accuracy with tools and services such as our base editing platform
  • Create the optimal DNA payload cassette that is sufficiently encapsulated in a compact viral capsomere 
  • Conduct functional genomics screening and target identification with gene editing and RNAi platforms. 

Safety and Efficacy

In gene therapy applications, the viral vector load strongly affects the severity of an undesirable immune response. High viral titers can potentially result in unwanted vector uptake by healthy cells in multiple organs. By increasing the specificity, sensitivity, and efficacy of viral vectors we can mitigate safety and toxicity outcomes by lowering the required viral concentration.  

  • Streamline the design, development, and delivery of your viral vectors for discovery and preclinical applications
  • Access a range of AAV serotypes with tissue-specific promoters and surface modified particles to enable precise gene expression and tissue targeting  
  • Accurately measure viral titers using p24 analysis to control the lentiviral load

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