Therapeutic Focus

We are developing potentially curative therapies for patients by deploying our genome editing toolbox with a stepwise approach

Effectively addressing disease

Our ther­a­peu­tic indi­ca­tions were cho­sen based on our con­vic­tion in the under­ly­ing biol­o­gy, exis­tence of val­i­dat­ing pre­clin­i­cal and clin­i­cal data, avail­abil­i­ty of phar­ma­co­dy­nam­ic and trans­la­tion­al tools to assess ear­ly proof-of-con­­cept, rel­e­vant val­ue sup­port­ing out­come mea­sures, and, most impor­tant­ly, ongo­ing clin­i­cal unmet need. These pro­grams are select­ed to both address dev­as­tat­ing dis­eases and to estab­lish new stan­dards in tar­getabil­i­ty, pre­ci­sion, effi­cien­cy, and scope of edit­ing capa­bil­i­ties. Using our tool­box, we aim to match the opti­mal genome edit­ing tool to each indi­ca­tion to ensure last­ing effects on the lives of patients.

Therapeutic Focus

In vivo

  1. Research
  2. Lead Opti­miza­tion
  3. IND-enabling
  4. Clin­i­cal
    • An illustrated depiction of a human liver, targeting Undisclosed Metabolic Disease Target, Lead optimization 75% completed, in partnership with Moderna

    • An illustrated depiction of a human liver, targeting Hemophilia A, Lead optimization 75% completed

    • An illustrated depiction of a human liver, targeting Undisclosed Disease Target, Research 75% completed

    • An illustrated depiction of a human liver, targeting Undisclosed Cardiovascular Disease Target 1, Research 75% completed

    • An illustrated depiction of a human liver, targeting Undisclosed Cardiovascular Disease Target 2, Research 50% completed

    • An illustrated depiction of a human brain, targeting Undisclosed CNS Disease Target, Research 50% completed

    • An illustrated depiction of a human lung, targeting Cystic Fibrosis, Research 25% completed

Ex vivo

  1. Research
  2. Lead Opti­miza­tion
  3. IND-enabling
  4. Clin­i­cal
    • An illustrated depiction of a human cell, targeting Immuno-oncology (TCR), Lead optimization 25% completed, in partnership with Affini-T

    • An illustrated depiction of a human cell, targeting Immuno-oncology (Modular CAR-T), Research 50% completed

Lever­ag­ing next-gen­er­a­­tion gene edit­ing tools to gen­er­ate a broad and dif­fer­en­ti­at­ed ther­a­peu­tic approach

With our tool­box, we can edit any tar­get in the human genome.