Transforming Base Editing with the Novel Pin-point Platform

The Pin-point™ Base Editing platform, with its novel three-part design and strong safety profile, enhances base editing's capabilities by enabling simultaneous modification of several genes at once, expanding its application in human disease modeling. Base editing is a CRISPR-Cas9-based technology that allows researchers to make precision base changes in genomic DNA. Editing with such precision can be used to silence disease-causing genes, correct disease-associated mutations, and optimize cell therapies.

Pin-point base editing requires three key components:

The guide RNA directs the modified Cas9 to the precise location on the targeted DNA
The guide RNA directs the modified Cas9 to the precise location on the targeted DNA
Modified Cas9 nicks a single strand of the targeted DNA
Modified Cas9 nicks a single strand of the targeted DNA
The recruited deaminase performs the base edit on the opposite strand
The recruited deaminase performs the base edit on the opposite strand

The gRNA guides the modified Cas9 to a specific location in the genome where a single strand 'nick' is made in the DNA. Meanwhile, the deaminase enzyme is recruited to the site and modifies bases on the non-nicked strand, for example, C >T conversions. The modified DNA strand is then used as a repair template for the nicked strand to complete the base pair conversion.

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The Pin-point platform's novel three-part design offers unparalleled flexibility to "mix and match" individual system components, allowing full optimization of each targeted gene edit.

Pin-point impact target

Facilitating the widespread use of next-generation Gene Editing

Compared to traditional CRISPR technologies that create double-stranded breaks in the DNA, base editing only nicks one strand of the DNA making it safer for therapeutic use. In addition to reducing the risk of insertions, deletions, and other rearrangements, this single nick increases the base conversion efficiency.

We offer a range of Pin-point base editing products and services, as well as traditional licensing to the underlying patents and patent applications, to provide broad access to base editing across the research and therapeutic market. By democratizing access to base editing, the Pin-point platform is positioned to accelerate the development of novel treatments and possibly cures for genetic disorders.

Key applications include:

  • Creation of gene knock-out/SNP correction
  • Proprietary concurrent knock-out and knock-in demonstrated
  • Multiplexed gene editing for complex cell models
  • Ability to edit cells sensitive to the DNA damage response pathway
  • Mutagenesis for functional gene characterization
  • Orthogonal validation (RNAi, CRISPRi, traditional CRISPR/Cas9)

Benefits of the Pin-point platform include:

  • MODULAR SYSTEM for optimized research
  • HIGH EFFICIENCY gene editing platform
  • MULTIPLEX EDITING across several targets
  • IMPROVED SAFETY over standard CRISPR-Cas9
  • VERSATILE TECHNOLOGY for targeted editing
  • VALIDATED PERFORMANCE in T cells and iPSCs

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