Science

Genetic medicines are comprised of both a payload and a delivery system.

Payload

Genetic medicine payloads include siRNA, antisense oligos (ASOs), mRNA, DNA and gene editing systems.

These technologies have advanced considerably over the last several years, offering the potential to revolutionize the treatment of human disease by addressing underlying disease causes with unprecedented specificity.

siRNA

ASO

mRNA

DNA

Gene Editor

Delivery System

Genetic medicines need to be precisely delivered into the cell; current delivery approaches include viral vectors, lipid nanoparticles (LNPs), exosomes, GalNAc-conjugates and others.

Each of these delivery approaches possess inherent limitations—ranging from safety and immunogenicity to payload constraints to the tissues that are currently addressable—that prevent their broad therapeutic application

Viral Vector

LNP

Exosome

GaINAc-conjugate

Our Technology

Aera’s proprietary protein nanoparticle (PNP) delivery platform is based on endogenous human proteins, leveraging the discovery of human proteins derived from retroelements that self-assemble to form capsid-like structures and which can package and transfer nucleic acid cargo. This platform combines the benefits of an endogenous, fully synthetic self-assembling system with the engineerability of a protein-based system and has the potential to enable various genetic medicine modalities.

Aera’s licensed technology also includes a proprietary therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes. The compact size of these enzymes may help overcome the packaging and delivery challenges of current gene editor systems.