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Best-in-class delivery technology

WHAT ARE NUCLEIC ACID THERAPEUTICS?

Nucleic acid therapeutics: A new paradigm in medicine.

Nucleic acid therapeutics are based on the same nucleic acids that form genes (DNA) or that act as the messengers (messenger RNA or mRNA) that direct our cells to make specific proteins. Proteins, in turn, are the building blocks of cells, tissues and organs.

Nucleic acid therapeutics are revolutionizing medicine in the treatment of a wide range of diseases and to provide protection against serious infections. New therapeutics address genetic diseases, improve medicines to treat cancer and inflammatory conditions, and enhance vaccines. This includes vaccines to protect against more challenging infectious diseases such as malaria, tuberculosis and HIV, as well as vaccines to treat cancer.

WHAT ARE LIPID NANOPARTICLES (LNP)?

Lipid nanoparticles: Groundbreaking delivery systems.

Nucleic acid therapeutics require an effective delivery technology to realize their potential to improve human health. For example, messenger RNA (mRNA) is very fragile and would be rapidly broken down if administered by itself. In addition, it is a very large, charged molecule and would not be able to enter cells (where it acts) without help. We provide the delivery technology that enables nucleic acid therapeutics to work.

LNP are tiny spheres (about 1/300th of the width of a human hair) composed of special lipids (fats). The nucleic acid therapeutic is encapsulated inside this sphere. Our LNP delivery systems use specially designed, proprietary, ionizable lipids and PEG-lipids (which stabilize the LNP) to efficiently load and protect the nucleic acid therapeutic and then effectively deliver it to the cell interior.

A scientist wearing a lab coat, safety goggles, gloves, and a patterned headscarf works in a laboratory, handling samples inside a fume hood. The lab is well-lit with natural light from large windows in the background.
HOW DO LNP WORK?

Enabling delivery.

The Acuitas team has identified key mechanisms for LNP delivery of nucleic acid therapies to cells, showcasing the adaptability and efficacy of our delivery systems. This allows applications such as gene editing, gene modulation or RNA interference (RNAi) requiring systemic (intravenous) delivery as well as vaccine applications requiring local (intramuscular) administration. We have shown that the ionizable cationic lipid component, in addition to the biophysical properties of the LNP itself, critically determines in vivo performance.

Following administration of our LNP carriers, they shed the PEG-lipid required to ensure stability during storage and then bind a specific protein that circulates in our blood called apolipoprotein E (Apo E). This then allows the Apo E-decorated LNP to be recognized by specific receptors on our cells and taken into the cell, localizing within organelles termed endosomes. Our LNP are designed to allow efficient release of the nucleic acid from the endosome into the cytoplasm where the nucleic acid therapeutic then acts to provide a clinical benefit.

The Power of Our Science

This video illustrates how our LNP enter the cells and release their payload (mRNA) into the cell cytoplasm, allowing the instructions on the mRNA to be read by other components in the cell to make new proteins.

HOW OUR LNP ARE USED

Our LNP are clinically validated.

Our proprietary LNP technology enables the RNAi therapeutic ONPATTRO® for the treatment of a rare genetic disorder, hereditary transthyretin amyloidosis (hATTR), and the mRNA vaccine COMIRNATY® to protect against COVID-19.

Logos displaying branding for Onpattro and Comirnaty. Onpattro is labeled as "patisiran, lipid complex injection, 2 mg/mL." Comirnaty is labeled as "COVID-19 Vaccine, mRNA.

In addition to these two approved clinical products, we are also supporting our partners in advancing new vaccines and therapeutics into clinical development. This includes vaccines to protect against common viral infections such as influenza and RSV as well as historically challenging diseases such as malaria, HIV and tuberculosis.

We continue to work with our partners to develop vaccines to harness our immune system to fight cancer. Our LNP technology is also enabling gene editing and gene modulation therapeutics to address serious genetic diseases, including the first human proof of concept for a base editing treatment for familial hypercholesterolemia.

ENABLING VARIOUS NUCLEIC ACID MODALITIES

Our LNP delivery systems are payload-agnostic and customizable.

 

Our LNP delivery technology is versatile, allowing efficient delivery of a variety of different payloads, including siRNA, DNA and guide RNA.

While much of our work with partners uses mRNA, we are also working with partners developing gene-editing therapeutics, these payloads may also include guide RNA to direct the gene-editing protein to a specific genome location.

 

 

A scientist wearing safety goggles and ear protection works in a laboratory. He is seated at a bench and dressed in a white lab coat and blue gloves, using a pipette. Various lab equipment and supplies can be seen in the background.
DELIVERING BEYOND THE LIVER

Extra-hepatic delivery

Acuitas is evaluating opportunities for mRNA LNP medicines to address clinical indications outside of the liver. Recognizing the significant challenge for intravenously administered particulate carriers, such as LNP, to escape the circulation and reach these non-liver targets, Acuitas is focused on delivery to cell targets that are directly accessible from the blood compartment including immune cells and hematopoietic stem cells.

Acuitas is also developing technology for local LNP delivery, such as to the lung via aerosolization and to the eye via direct administration. In particular, Acuitas has identified LNP compositions that are stable following exposure to aerosolization technologies and allow deep lung penetration. Similarly, we have identified LNP systems that are uniquely suitable for clinical indications requiring delivery to the eye and are supporting partners advancing exciting applications in this area to address unmet patient needs.

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Our clinical applications

Our LNP delivery technology offers a flexible platform to better treat a wide array of diseases. This includes applications in prophylactic infectious disease and therapeutic cancer vaccines, gene editing/gene modulation, and therapeutic protein delivery, showcasing the extraordinary potential of our technology.

Our Clinical Applications