AIDE Technology Platform

Our proprietary Autologous Intracellular Drug Encapsulation, or AIDE, technology platform is an innovative drug/device combination platform that uses an automated process to encapsulate a drug into a patient’s own red blood cells.

Red blood cells have several characteristics that make them a potentially ideal vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Our AIDE technology is designed to harness many of these benefits to allow for new and improved therapeutic options for patients living with high unmet medical needs. The AIDE technology platform may confer benefits over conventional therapies and if so, AIDE has the potential to be applied to a broad range of small or large molecule drugs and biologics. The use of autologous blood may minimize safety risks associated with the use of donor blood and may reduce the potential immunogenic risks associated donor cells and engineered cell therapies.

Our AIDE technology platform reflects more than 20 years of innovation and approximately $100 million of investment, which has resulted in innovation that creates high barriers to competitive entry. The RCL, EryKit, Syringe Kit, and process solutions are proprietary products and CE marked in the European Union, in accordance with the Medical Devices Regulation 2017/745 (MDR) and Medical Devices Directive (MDD).

AIDE – Autologous Intracellular Drug Encapsulation

Potential Benefits of Red Blood Cell Encapsulated Drug Delivery

Many efficacious drugs have limited therapeutic potential because of dose limiting toxicity, while other drugs may have efficacy ceilings due to suboptimal biodistribution, pharmacokinetics and pharmacodynamics. Our proprietary AIDE technology uses an automated process designed to encapsulate a drug into the patient’s own red blood cells to deliver a therapy in a potentially more effective and safer way. Autologous red blood cells have several characteristics that make them an ideal vehicle for drug delivery:

Potential for improved biodistribution as encapsulated drug in autologous red blood cells is designed to enable the slow release of the drug from the red blood cells traversing through the body, various tissue beds, and many capillaries for desired therapeutic effect.

Potential for altered pharmacokinetics and pharmacodynamics, including long circulating half-life, and altered or improved tissue distribution. The altered pharmacokinetics and pharmacodynamics of the encapsulated drug delivery enabled by autologous red blood cells may significantly increase the desired therapeutic effect and/or improve the safety profile of the therapy.

Potential for improved biocompatibility through the use of autologous red blood cells to, thereby avoiding issues with donor compatibility.

Potential for the encapsulation of small or large molecules, peptides, and proteins inside of autologous red blood cells to limit biodegradability, thereby altering the encapsulated drug’s metabolism and degradation.

Potential for significantly decreasing adverse effects of treatments.

Lead Asset EryDex and Mitigating the Limitations of Chronic Corticosteroid Administration

EryDex is the first product in development that leverages our AIDE technology and is composed of dexamethasone sodium phosphate (DSP) encapsulated in autologous red blood cells targeted for the treatment of patients with Ataxia-Telangiectasia, or A-T. DSP is a corticosteroid well known for its potent anti-inflammatory properties, as well as its dose-limiting toxicity due to adrenal suppression. EryDex is designed to provide the efficacy of corticosteroids while reducing or eliminating the significant adverse effects that accompany chronic corticosteroid treatment.

DSP is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. Our AIDE technology is designed to encapsulate DSP in a patient’s own red blood cells and to alter the biodistribution, pharmacokinetics, and pharmacodynamics of the DSP allowing for both effective and safe treatment.

The optimal efficacy of corticosteroids is the result of two pharmacokinetic characteristics: 1) an initial bolus to achieve a high Cmax that results in high levels of corticosteroid receptor occupation; and 2) sufficient sustained tissue concentrations that allow for continued receptor site occupancy over time.

In order for a conventional corticosteroid to achieve these characteristics, the drug must be dosed frequently, typically daily. Long-term daily dosing regimens sufficient to provide efficacy, lead to significant and debilitating long-term adverse effects associated with HPA axis suppression. Corticosteroid therapy without significant long-term safety liabilities would represent a major advancement in the treatment of many chronic diseases where corticosteroids are already known to be beneficial.

Pharmacokinetic Profile of Oral/IV Daily Administration of Dexamethasone

Note: Information represented does not reflect a completed comparative study of EryDex versus oral/IV administration of DSP, but rather provides a comparison of published corticosteroid pharmacokinetic information relative to company data regarding EryDex. IC50 and SC50 refer to pharmacodynamic parameters of which IC50 reflects drug concentration eliciting 50% of the maximum inhibition and SC50 reflects drug concentration eliciting 50% of the maximum stimulation. References: Company prior Phase 3 ATTeST clinical trial data ( ID: NCT02770807); Montanha et al, Frontiers in Pharmacology (2022) 13: 814134; Krzyzanski et al, Journal of Pharmacokinetics and Pharmacodynamics (2021) 48: 411-438; Aljebab et al, PLOS ONE (2017) 10: 1371.

Highly Differentiated and Proprietary Drug/Device Combination

Our proprietary AIDE technology platform is a novel drug/device combination that uses an automated process designed to encapsulate a drug into the patient’s own red blood cells. The AIDE technology drug/device combination consists of a specialized automated equipment including the RCL, a sterile single-use consumable treatment kit called EryKit, Syringe Kit, drug, and process solutions.

RCL Machine

Red Cell Loader (RCL)

The RCL is a proprietary CE marked non-invasive device that allows blood processing at the point of care. It automates the AIDE process technology by handling the blood, drug, and processing solutions with the use of EryKit. The system has a user-friendly touch screen interface made up of three microprocessor circuit boards, two main boards, and an independent protective board as control.

EryKit Treatment Consumables

EryKit is a CE marked medical device. It provides the essential single-use components for loading human red blood cells (RBCs) with variety of therapeutics, ranging from small to large molecules, as well as biologics, by using the RCL. The EryKit contains a sterile, single use, pyrogen-free disposable blood tubing system equipped with a centrifuge, hemoconcentrator filter, bags, organizer cassette, and other accessories.

EryKit Consumables

AIDE Technology Fully Automated at Patient Point-of-Care

The automated AIDE process and treatment is designed to be completed at the point-of-care and includes a series of steps which take approximately two hours from start to finish. This process includes:

AIDE Technology at Point-of-Care

Collection of 50mL of a patient’s blood using our proprietary Syringe Kit.

Processing the patient's collected blood in the RCL using our proprietary EryKit.

Autologous red blood cells in the RCL are swollen and their pores are “opened” in two steps using two sequential hypotonic process solutions.

Drug is added to the RCL and enters into the opened red blood cells.

Physiological osmotic conditions are then restored by adding a hypertonic solution that “reseals” the red blood cells.

Drug that is not encapsulated during the process is removed by extensive washing with an injectable saline solution.

Upon completion of the AIDE process, the drug encapsulated red blood cells are infused into the patient.