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Before diving into the rigor and regulatory demands of Good Laboratory Practices (GLP)-compliant toxicology studies, most drug development programs begin with non-GLP studies. While not subject to the same regulatory constraints, these early investigations play a vital role in reducing risk, informing study design, and optimizing the use of resources.

Developing a novel non-oncology small molecule drug involves much more than discovering a promising compound. Before advancing to clinical trials, regulatory authorities require comprehensive safety data to support an Investigational New Drug (IND) application. For non-oncology indications, this data must be generated through a sequence of preclinical studies—each designed with a specific purpose, interdependent timelines, and clearly defined regulatory expectations.

Oligonucleotide therapeutics (oligos) have emerged as a groundbreaking class of drugs with the potential to treat previously untreatable diseases. By targeting RNA or modulating protein function, therapies such as antisense oligonucleotides (ASOs), small interfering RNA (siRNA), and aptamers offer new avenues for precision medicine. However, their development is not without hurdles, particularly in bioanalysis. Their unique properties demand advanced analytical methods to ensure accuracy, stability, and regulatory compliance.

Large molecule drug candidates face unique challenges in preclinical testing due to their complexity, but with the right strategies, researchers can navigate these hurdles and develop novel therapeutics. This comprehensive guide walks you through everything you need to know about large molecule preclinical testing, ensuring potential risks are minimized and the drug’s full potential is realized.

Proteolysis-targeting chimeras (PROTACs) are some of the most eagerly discussed therapies in the world due to their potential to treat conditions previously thought undruggable. Their ability to degrade rather than inhibit proteins means they can better treat complex diseases such as cancers and neurodegeneration, giving them advantages over traditional small molecule drugs.

Imagine a healthcare landscape where treatments are precisely tailored to an individual’s unique genetic profile, environment, and lifestyle. This vision is no longer a distant possibility—it is the reality of precision medicine.

Antibody Drug Conjugates (ADCs), which consist of antibodies, payload and linkers, are an innovative therapeutic approach targeting various types of tumors and cancers. It can improve the therapeutic parameters of payload and reduce systemic cytotoxicity. ADCs rely on highly targeted tumor antigen recognition and effective endocytosis to recognize and bind to specific tumor antigens on the cell surface.

Antisense oligonucleotides (ASOs) have shown great promise in targeting disease-causing genes, offering hope for patients suffering from genetic disorders that were previously difficult to address. ASOs bind to specific RNA sequences, preventing the production of harmful proteins or altering gene expressions.

Large molecule therapies hold immense promise for treating a range of diseases. However, developing large molecule therapies has unique challenges, particularly in preclinical testing. Toxicology studies are crucial because they provide essential data about the drug’s biological effects and safety profile.