Therapeutics

Therapeutic cures through CRISPR-based precision editing

Leveraging novel CRISPR-Cas enzymes from our protein discovery platform.

Image representing Cas14 and CasPhi cutting DNA as part of the genome editing process
  • Ultra-small and Flexible Delivery

    Expand delivery options

    The small sizes of our Cas14 and CasΦ nucleases overcome the limitations to viral and non-viral delivery methods experienced with Cas9.

  • Precise Edits

    Edit more of the genome

    With alternative and shorter PAM requirements, newly discovered nucleases can direct edits to an expanded set of DNA sequences.

  • Cleaner Intellectual Property

    Simpler and cleaner IP

    IP protection for each novel system and method.

Cas14 and CasΦ Families

Enabling new possibilities.

Our novel Cas nucleases enable genome editing results that might not be possible with other Cas nucleases.

Newly discovered CRISPR-Cas systems are strong candidates for genome editing due to their small size and unique PAM (protospacer adjacent motif) requirements. Using our protein discovery workflow, we have and continue to identify variants with flexibility to cut single or double-stranded DNA or RNA or to be used with novel DNA or RNA modifying modalities.

Domain organization of Cas14a and CasΦ-2 compared to commonly used variants of Cas9 and Cas12 with the nuclease domains (RuvC and HNH) indicated. Protein lengths are drawn to scale.

Length distribution of Cas14a–c and CasΦ systems compared to Cas12a-e and Cas9.

Therapeutic Applications

CRISPR has been used to address many needs in a number of therapeutic applications.

  • Immuno-Oncology

  • Autoimmune Diseases

  • Liver-targeted Diseases

  • Hematology

  • Ophthalmology

  • Neurology

  • Neuromuscular Diseases

  • Cardiovascular

Partnerships

Let’s work together

Don’t miss out on the next generation of CRISPR-based therapeutics as we just started collaborations with Vertex Pharmaceuticals and Bayer AG. Please contact us to learn more about our groundbreaking CRISPR gene editing technologies.

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