Columbia Technology Ventures

Modified prime editor for gene therapy optimization

This technology is a DNA editing platform with a reduced payload size to allow adeno-associated virus (AAV) and nanoparticle packaging for gene therapy.

Unmet Need: Size-compatible genomic prime editing system

Mutations in short sequences or single bases in the human genome account for 89% of known pathogenic variants, implying a need for efficient and directable genomic editing. Current methods to edit small regions of DNA through a process called “prime editing” are contained within a DNA payload that is too large to facilitate many gene therapies. Reducing the size of the prime editing platform would permit packaging into a single adeno-associated virus (AAV), which would allow for safer and more efficient delivery strategies and expand the application of gene therapy to treat a broader variety of conditions.

The Technology: Smaller prime editing platform for improved gene therapy opportunities

This technology enables enhanced modification of a target nucleic acid by using a single subunit of the reverse transcriptase. By presenting a smaller size than conventional prime editors, this technology can be packed and delivered in a single adeno-associated virus (AAV). The smaller payload allows for more effective delivery of genetic therapeutics and could also be used in other genetic assays, such as RT-PCR. Delivery of the technology enhances the ability of researchers and clinicians to design, optimize, and test novel gene therapies for adaptation into next-generation therapeutics.

This technology has been validated with in vitro studies with different cell lines.

Applications:

  • AAV-compatible gene therapy platform
  • Nanoparticle-based gene therapy platform
  • Research tool for prime editing of cells in cellular experiments
  • Germline editing for generation of animal models
  • Research tool for development of new prime editing strategies

Advantages:

  • Size-compatible with AAV and nanoparticles
  • Expanded range of gene therapy opportunities
  • Expression of a single nickase gene rather than multiple
  • Opportunity to expand thermostability, activity, and interactivity with template

Lead Inventor:

Peter Quinn, Ph.D.

Patent Information:

Patent Pending (US20250171810)

Related Publications:

Tech Ventures Reference: