New Therapeutic Modalities
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At Concept life sciences we support many established and novel drug modalities. These alternative approaches to drug discovery and development go beyond traditional small molecule drugs. Examples include fragments, peptides, therapeutic proteins, antibodies, targeted protein degraders, gene therapies, RNA-based therapeutics, and antibody drug conjugates. Drug discovery for these therapeutics involves developing biologics which offer high specificity and unique mechanisms of action and are designed to target complex biological processes and "undruggable" targets, often with fewer off-target effects. Advances in biotechnology, genomics, and delivery systems have significantly expanded the potential and efficacy of non-small molecule therapeutics in treating a wide range of diseases.
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A more in-depth view
- Fragments: Fragment-based drug discovery (FBDD) involves identifying small chemical fragments that bind to biological targets, which are then optimised to develop potent drug candidates. This approach allows for the efficient exploration of chemical space and can yield high-affinity compounds through iterative cycles of design and testing.
- Peptides: Peptide therapeutics leverage short chains of amino acids to modulate biological pathways, offering high specificity and low toxicity. Advances in peptide synthesis and delivery technologies are expanding their potential applications in treating a wide range of diseases.
- Therapeutic Proteins: Therapeutic proteins, such as hormones, enzymes, and cytokines, are designed to replace deficient or abnormal proteins in patients. These biologics have revolutionized the treatment of conditions like diabetes, anemia, and autoimmune diseases.
- Antibodies: Monoclonal antibodies (mAbs) are engineered to target specific antigens, offering precise treatment options for diseases such as cancer, autoimmune disorders, and infectious diseases. Their high specificity and ability to recruit immune system components make them powerful therapeutic agents.
- Targeted Protein Degraders: Targeted protein degraders, including PROTACs (Proteolysis Targeting Chimeras), harness the cell's natural degradation machinery to selectively eliminate disease-causing proteins. This novel approach provides a strategy to tackle previously "undruggable" targets by marking them for destruction.
- Gene Therapies: Gene therapies involve modifying a patient’s genetic material to treat or cure diseases, often through the delivery of functional genes or gene-editing tools. These therapies hold promise for addressing genetic disorders, cancers, and viral infections with long-lasting effects.
- RNA-based Therapeutics: RNA-based therapeutics, such as mRNA vaccines and RNA interference (RNAi) therapies, manipulate RNA to treat or prevent diseases by regulating gene expression. These innovative treatments have gained prominence, particularly with the success of mRNA COVID-19 vaccines.
- Antibody Drug Conjugates (ADCs): ADCs are sophisticated molecules that combine the specificity of monoclonal antibodies with the potency of cytotoxic drugs, delivering targeted cancer therapy. This approach allows for direct delivery of the drug to cancer cells, minimizing systemic side effects and enhancing therapeutic efficacy.
