Delivery

Lipid Nanoparticle Technology

Delivering on the promise of Gene Writing™

Delivery Process

We deliver our Gene Writers as all RNA products using proprietary lipid nanoparticles or LNPs.
 

LNPs can be used to deliver nucleic acids to cells, with the potential to avoid eliciting a robust immune response often associated with viral delivery vectors.

Man­u­fac­ture Gene Writer mRNA and tem­plate RNA

mRNA-encod­ed Gene Writer and tem­plate RNA are pack­aged into a lipid nanoparticle

RNA-LNP com­po­si­tion of mat­ter is admin­is­tered to the patient, and enters the cell type of interest

After enter­ing the tar­get cell, the Gene Writer mRNA is trans­lat­ed into pro­tein and binds the tem­plate RNA

The Gene Writer-tem­plate com­plex is shut­tled to the nucle­us and effects the desired alter­ation into the genome

We believe LNPs are the optimal delivery vehicle for Gene Writers
 

Potential Advantages of LNPs

We believe LNPs constitute an advantaged delivery vehicle over viral methods to dispatch targeted genetic instructions.

  • Scalable, non-viral delivery

    Our LNP delivery is synthetically manufactured, scalable, and not constrained by viral packaging limits or pre-existing antiviral immunity

  • In vivo cell engineering

    Targeted LNP delivery to HSCs and T ells is designed to eliminate the need for complex and toxic ex vivo procedures, such as stem cell mobilization, chemotherapy, or individualized manufacturing 

  • Multiplex editing capability

    Modular Gene Writer architecture and flexible RNA payloads allow for multiple genetic modifications in a single step, enabling more sophisticated cell engineering

  • Durable yet flexible treatment approach 

    Gene Writers are designed to make permanent DNA changes, enabling potentially durable, one-time treatments, although we expect that LNP delivery may support repeat dosing if needed

Limitless Potential

Advancing delivery capabilities with our proprietary LNP technology

Existing therapeutic solutions to many genetic diseases involve the liver, where conventional LNPs naturally accumulate. We are advancing our proprietary technology to direct LNPs to go not only to the liver but beyond. Targeted delivery of our Gene Writers to tissues beyond the liver is essential to writing in the code of life precisely where a therapeutic change is needed.

Delivery to the liver designed to ensure that our Gene Writers may address genetic diseases that involve this organ.
Through effectively writing DNA in HSCs, we could cure serious blood-based diseases.
Through writing DNA in T cells, we could effectively eliminate tumors and treat autoimmune diseases.
Liver
Hematopoietic Stem Cells
T cells
Making the broadest possible impact for patients
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