Delivery

Novel Lipid Nanoparticle Technology

Delivering on the promise of Gene Writing™

Delivery Process

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

LNPs can safely and effectively deliver nucleic acids to cells without eliciting a robust immune response as is common 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

LNPs are the optimal delivery vehicle for Gene Writers

Benefits of LNPs

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

  • Established safety track record

    The widespread use of mRNA vaccines has demonstrated the broad safety profile of LNP delivery of RNA therapeutics.

  • Scalable and cost-effective to manufacture

    Viral vector manufacturing is often costly and a bottleneck for therapeutic delivery.

  • Re-dosable, if needed

    Unlike viral vectors, LNPs are not recognized as foreign by the human immune system.

  • Transient expression of engineering machinery

    Gene Writing machinery will be short-lived inside the cell, reducing the risk of genotoxicity.

Limitless Potential

Solving delivery 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 ensures that our Gene Writers can address the plethora of genetic diseases that involve this organ.
Through effectively rewriting DNA into HSCs, we could cure serious blood-based diseases.
Through writing DNA in T cells, we could effectively eliminate tumors and treat autoimmune diseases.
<p>Liver</p>
<p>Hematopoietic Stem Cells</p>
<p>T cells</p>
Making the broadest possible impact for patients
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