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

Image is a large table with pipeline illustrations. The highlights are: Broad pipeline built on our metagenomics platform. Hemophilia A/ALB is in Lead Optimization. Undisclosed secreted protein diseases is in Discovery. Transthyretin Amyloidosis/TTR is in Lead Optimization. Refactory Hyperteension / AGT is in Lead Optimization. Two undisclosed cardiovascular diseases are in Lead Optimization. Note: Other program, Primary Hyperoxaluria Type 1 / HAO1 with no pipeline yet. Alpha 1 Antitrypsin Deficiency / SERPINA1 is in Discovery. Wilsons Disease / ATP7B is in Discovery. Solid tumor indications / TCR T Cells is in IND-Enabling. Note: Multiplex editing Undisclosed cell therapy applications with no pipeline yet. Note: Programs in research: Familial ALS, Duchenne Muscular Dystrophy, Charcot Marie Tooth Disease with no pipeline yet. Programs in Research Undisclosed renal diseases, Cyctic Fibrosis with no pipeline yet.

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.