Regulatory Expertise

Next-generation AAV (adeno-associated virus) therapies have recently emerged as a significant area of therapeutic innovation to address complex or inherited diseases. They offer a potential pathway to treat conditions once deemed untreatable through methods like replacement, inactivation, or introduction of new materials. Viral vectors are essential for delivering these advanced therapies, as they efficiently transport therapeutic material into target cells. Once inside the cell, the vector incorporates into the host’s natural machinery, enabling protein expression and facilitating treatment outcomes.

The development of oligonucleotide drugs has seen remarkable progress, driven by innovations in chemical modifications and delivery systems. These advancements have significantly enhanced drug stability, extended half-life, and improved targeting capabilities, increasing therapeutic efficacy and reducing dosage frequency. However, these benefits come with unique challenges in preclinical bioanalysis, particularly in pharmacokinetics (PK), drug metabolism, and tissue distribution studies.

Diabetes is classified into Type 1 diabetes mellitus (TIDM) and Type 2 diabetes mellitus (TIIDM) based on whether the patient has a deficiency in insulin secretion. TIDM is, on the one hand, characterized by destroying pancreatic beta cells that secrete insulin, mainly during childhood and adolescence. On the other hand, TIIDM is characterized by an imbalance between insulin levels and insulin sensitivity, resulting in persistent hyperglycemia. TIIDM is primarily found in middle-aged and elderly individuals. Globally, 90% of diabetes patients have TIIDM. Whether it is TIDM or TIIDM, if patients do not treat their hyperglycemia through appropriate medical interventions, they may face a series of severe consequences like blindness, kidney failure, or amputation.

Pharmacokinetic (PK) assays are a crucial stage of drug development. They help ensure the safety and effectiveness of new therapeutics by establishing how drugs are absorbed, distributed, metabolized, and excreted in the body.

Genotoxicity testing is an essential tool for drug developers, as it can ensure patient safety in new and established drugs. Genotoxic events can cause irreversible damage and particularly severe health events, which makes it imperative these tests are conducted accurately and efficiently.

Genotoxicity testing is an essential tool for drug developers, as it can ensure patient safety in new and established drugs. Genotoxic events can cause irreversible damage and particularly severe health events, which makes it imperative these tests are conducted accurately and efficiently.

Oligonucleotide drugs (ONs) are synthetic molecules ranging from 12 to 30 nucleotides in length and typically made up of single or double strands of nucleotides. Through Watson-Crick base pairing, these drugs use target messenger RNA (mRNA), which results in the inhibition of gene expression and the prevention of erroneous protein production.

Ensuring the medications we rely on are safe and effective from the moment they leave the manufacturer to when they reach the patient involves rigorous stability testing, a critical process that safeguards drug product integrity under diverse environmental conditions. This testing is crucial in biopharmaceutical development to maintain the safety, efficacy, and quality of drug products (DP) and drug substances (DS). It accounts for potential degradation and the stability of different formulations and packaging configurations, providing data to inform labeling, storage, transport, and handling guidelines.