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. Dis­cov­ery

  2. Pre­Clin­i­cal

  3. Phase 1

  4. Phase 2

  5. Phase 3

    • An illustrated depiction of a human liver, targeting Hemophilia A, PreClinical 50% completed

    • An illustrated depiction of a human liver, targeting Primary Hyperoxaluria Type 1, PreClinical 50% completed, in partnership with Moderna

    • An illustrated depiction of a human liver, targeting Transthyretin Amyloidosis, PreClinical 50% completed, in partnership with Ionis

    • An illustrated depiction of a human liver, targeting Cardiovascular Disease, Discovery 100% completed, in partnership with Ionis

    • An illustrated depiction of a human liver, targeting Alpha-1 Antitrypsin Deficiency, Discovery 50% completed

    • An illustrated depiction of a human liver, targeting Wilson's Disease, Discovery 50% completed

    • An illustrated depiction of a human brain, targeting Familial ALS, Discovery 50% completed

    • An illustrated depiction of a human brain, targeting Spontaneous ALS, Discovery 50% completed

    • An illustrated depiction of a human brain, targeting Charcot-marie-tooth disease type 1a, Discovery 50% completed

    • An illustrated depiction of a human brain, targeting Duchenne Muscular Dystrophy, Discovery 50% completed

    • An illustrated depiction of a human lung, targeting Cystic Fibrosis, Discovery 50% completed

Ex vivo

  1. Dis­cov­ery

  2. Pre­Clin­i­cal

  3. Phase 1

  4. Phase 2

  5. Phase 3

    • An illustrated depiction of a human cell, targeting Immuno-oncology (TCR; CAR-T Cells), PreClinical 100% completed, in partnership with Affini-T

    • An illustrated depiction of a human cell, targeting Autoimmune (Car-T Cells), Discovery 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.