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Delivery technologies

Getting an experimental treatment to the right cells, with enough control over its activity.

SchematicDelivery has several boundaries

Prepare the carrierDefine the payload and delivery system.
Reach the intended cellsMeasure distribution, uptake and payload activity.
Follow exposure and effectsCheck duration, function and unintended activity.

Reaching a tissue does not establish activity in every cell within it.

Programmes in this field

Each dossier describes the mechanism, studies and development history of a research programme.

2 programmes and 1 article in this edition.

Programme records

DeliveryPreclinical research

RJB-0402 liver-directed FGF21 gene therapy

Rejuvenate Bio

Rejuvenate Bio proposes using the liver to produce a circulating protein intended to affect diseased heart tissue. RJB-0402 is aimed at arrhythmogenic cardiomyopathy. Public sources document preclinical work and trial preparation, but do not verify a human intervention study start.

Cells · AnimalsRead dossier

DeliveryPreclinical research

Liver mRNA immune restoration

Broad Institute, MIT and academic collaborators

A mouse study uses liver-directed mRNA to supply immune signals. The research tests a temporary change in immune function.

AnimalsRead dossier

Research and explanations

A lipid-like carrier containing an RNA strand beside a cell membrane and an opened internal vesicle.

Explainer4 min read

Getting rejuvenation machinery into the right cells

A delivery system must reach the intended cells and control exposure. Lipid nanoparticles, viral vectors and local administration solve different parts of that problem.

Read article