Nucleic Acid-based Therapy Development Services for Cardiovascular Diseases

Nucleic acid-based therapies now hold the promise of providing a solution to a number of complex cardiovascular diseases that cannot be addressed by conventional therapies. Ace Therapeutics provides comprehensive drug development services for nucleic acid-based therapies to help clients advance the process of novel drug discovery for cardiovascular diseases.

Current Status of Nucleic Acid-based Therapies for Cardiovascular Diseases

Nucleic acid-based therapies are considered to be a revolution in pharmacology as they can radically cure or alleviate a wide range of diseases. In the area of new drug development for cardiovascular disease, a number of ASO and siRNA drug development pipelines are currently in clinical trials. These nucleic acid-based therapies treat familial hypercholesterolemia, familial amyloidosis cardiomyopathy and peripheral arterial disease by targeting genes involved in lipid metabolism, inflammation and vascular function.

Fig. 1 Schematic representation of miRNA-based therapies to compensate the aberrant expressed miRNAs in heart diseases. (Zhang X, and Schulze PC, 2016)

Ace Therapeutics provides comprehensive solutions for the development of nucleic acid-based therapies to help clients advance the discovery and development of novel cardiovascular drugs.

Target Identification and Validation

We can identify genes involved in the pathogenesis of cardiovascular diseases, and then use bioinformatics tools to predict potential gene targets. By knocking down/out or overexpressing the predicted gene targets, we can also validate these targets at in vitro and in vivo levels to confirm their role in the pathogenesis of cardiovascular diseases.

Development of Oligonucleotide Drugs for Cardiovascular Diseases

• Sequence Design and Synthesis
  We can use bioinformatics tools to design siRNA or ASO sequences with high specificity and low off-target effects based on relevant cardiovascular targets, and chemically synthesize these designed nucleic acid fragments.
• Modification and Optimization
  We can improve the stability, cellular uptake efficiency and reduce the immunogenicity of siRNA or ASO by chemical modifications to achieve inhibition or silencing of target genes in cardiovascular diseases.
• Efficacy Study
  We can test the potency and specificity of siRNAs or ASOs in target cell lines to assess their inhibitory effect on target genes.

Development of mRNA Drugs for Cardiovascular Diseases

• Sequence Design and Synthesis
  We can synthesize mRNA fragments based on mRNA sequences encoding cardiovascular disease-related target proteins using in vitro transcription techniques.
• Modification and Optimization
  We can optimize the sequences of coding and non-coding regions and chemically modify mRNAs to improve mRNA stability and translation efficiency.
• Efficacy Study
  We can test the expression efficiency and function of mRNAs in cellular models to assess their expression of target proteins.

Drug Delivery System Development for Nucleic Acid-based Therapies

We can design ideal delivery systems for nucleic acid-based therapies, such as lipid nanoparticles (LNPs), polymer nanoparticles, viral vectors, and cell-penetrating peptides (CPPs) to ensure that the nucleic acid drugs can be efficiently delivered to target cells in the cardiovascular system.

Preclinical Evaluation for Nucleic Acid-based Therapies

We can evaluate the therapeutic potential of candidate nucleic acid drugs using appropriate animal models of cardiovascular disease to assess their pharmacokinetics, pharmacodynamics and safety.

Ace Therapeutics is a CRO that specializes in providing outsourced drug discovery and development services for cardiovascular diseases. With a specialized technology platform for new drug development and an experienced research team, we can provide nucleic acid-based drug discovery and development services according to the needs of our clients. If you are interested in our services, please do not hesitate to contact us.

1. Zhang, X.; Schulze, P.C. MicroRNAs in heart failure: Non-coding regulators of metabolic function. Biochim Biophys Acta. 2016, 1862(12):2276-2287.