AccuLNP® is a lipid mediated, multi-component system for the delivery of nucleic acids. We have a versatile and diverse library of over several hundred proprietary ionizable lipids. Which serves as the major component of the delivery system. AccuLNP® can be applied for a variety types of therapeutic applications in the field of genetic medicine. Our preclinical studies have shown that the composition of the delivery vehicle can be tailored for the encapsulation of different types of nucleic acids (e.g., mRNA, siRNA, self-amplifying RNA and oligonucleotides) to achieve high payload encapsulation efficiency, superior product stability and desirable biological performance. Ionizable Lipids and LNP Formulations: Rational lipid design to establish structure-activity relationships; Use artificial intelligence (AI) based techniques to predict biological performance and to accelerate lipid screening; High-throughput screening for efficient formulation evaluation; Proprietary library with hundreds of novel ionizable lipids with desirable biological performance; Tailored formulation composition to fit specific applications of genetic medicine. Organ/Tissue Targeted Delivery: Organ/tissue targeting moieties are incorporated on LNP surface to improve delivery specificity; Endogenous organ targeting empowered by proprietary ionizable lipids. Manufacturing Efficiency: Clinical validated equipment for LNP production; Established manufacturing process with comprehensive analytical methods to support drug product development. AccuLNP® Mediated Delivery of Nucleic Acid: Step 1 AccuLNP® reaches a target cell of interest and then is taken up by an endogenous cellular transport pathway to deliver nucleic acids intracellularly; Step 2 Upon entering the cytoplasm, the AccuLNP® then is trapped within the endosome; Step 3 As the endosome matures, the acidity increases activating the pH responsive ionizable lipid within AccuLNP® enabling the release of the payload (nucleic acids) into the cytoplasm, which subsequently exerts its desired therapeutic function. Applications of AccuLNP® Delivery Platform: Protein Therapy & mRNA Vaccines - AccuLNP® delivers mRNA For the following therapies: (1) Protein replacement to restore single protein function (2) Cell reprogramming to modulate cell behavior by expressing growth factors (3) Therapeutic antibodies; Modulating the immune system through mRNA-LNP vaccines. Genome Editing - AccuLNP® delivers CRISPR components (CAS9 mRNA and sgRNA) to cell for gene editing. CAR T cell Therapy - AccuLNP® generates transient CAR (chimeric antigen receptor) expression on T cells for cancer immunotherapy (autologous cell therapies). Targeting the Tumor Tissue - AccuLNP® can target the tumor tissue through the well known EPR effect and deliver genetic medicine for cancer treatment; Antibody-targeted LNPs (AccuLNP-T®) can reach cancer cells with the complementary receptors
