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.
Regulatory
Advancing Oligonucleotide Drug Development with Ligand Binding Assays in the Preclinical Stage
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.
Bioanalysis of GLP-1 Antagonist Drugs by Multiple Analytical Platforms
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.
Overcoming nonspecific binding challenges in PK assays
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.
Bioanalytical Strategies for Peptide-Drug Conjugates (PDCs): Optimized Approaches for the Pharmaceutical Industry
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.
Validating Flow Cytometry-Based Micronucleus Assays in Alternative Species
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.
Preclinical Strategies for Safety Evaluation of Oligonucleotide Drugs
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.
Tackling Development and Regulatory Challenges for Gene Therapies
Gene therapies have been one of the fastest-growing fields of drug development. These therapies hold the potential to treat cancers and other “undruggable” diseases and could hold the key to saving millions of lives. But they also have unique characteristics that make...
Dose Range-Finding Studies for Safety Assessment: 3 Reasons Not to Skip Them
Are you ready for your GLP toxicology studies? Not without dose range-finding studies, you aren’t. Here are 3 reasons why these can’t be skipped. A well-planned preclinical toxicology program always starts with the end in mind. In the case of dose levels, then, you...
4 Reasons Non-GLP Bioanalysis Matters for DMPK Testing
DMPK testing helps drug developers investigate how a drug compound is absorbed, distributed, metabolized, and eliminated (ADME) by the body. The earlier these properties can be understood, the better – which is why many drug developers start the process with non-GLP...
A New Method to Improve Identification of the Payload-Containing Catabolites of ADCs
Antibody-drug conjugates (ADCs) are a relatively new medicine with the potential to target cancer cells and deliver a toxic payload, all while minimizing any damage to otherwise healthy body parts. ADCs have a number of strengths, but perhaps chief among them is...
Improving PROTAC Drugs with MetID Studies
Most drugs undergo some level of transformation once they enter the body. This metabolism process allows a drug's active pharmaceutical ingredient (API) to achieve its desired effect and eliminate it from the body. But metabolism can also be tricky. Metabolizing a...