In Vivo
Delivery
Our in vivo delivery offers a simpler alternative to complex ex vivo gene therapies. Instead of modifying cells outside the body, we deliver gene-editing tools directly to cells within the patient. This in vivo approach avoids the challenges and limitations of traditional cell-based manufacturing and transplantation.
Pipeline
Protein Delivery Vehicles (PDVs) delivered in vivo CRISPR therapy
Ocular
Stromal corneal dystrophy
Undisclosed
Undisclosed
Lipid nanoparticles (LNPs) delivered in vivo CRISPR therapy
Rare disease
ATTR Amyloidosis
Cardiovascular
High Lp(a)
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Program |
Discovery |
Preclinical |
IND-enabling |
Clinical |
Approval |
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Provide affordable precision DNA surgery for genetic diseases with unmet needs
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