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EP 33

Modern Therapeutic Modalities

The podcast discusses the new therapeutic toolkit, including engineered cells, genetic instructions, and digital design. It highlights the shift in market power towards biologics and the role of artificial intelligence in linking different approaches together. The episode also explores the diversification of therapeutic modalities, including gene editing, cell therapy, and RNA therapies.

gene editingcell therapyRNA therapiesimmunotherapypeptides
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Key Concepts

Gene Editing

  • Gene editing is a precise technology that allows for the modification of genes.
  • CRISPR-based gene editing is a major breakthrough, with the first approval of a CRISPR-based therapy in 2023.
  • Base editing is a new technology that allows for the direct conversion of one DNA base to another without making a double-strand cut.

Cell Therapy

  • Cell therapy involves the use of living cells to treat diseases.
  • The first approval of a TIL therapy for advanced melanoma was in 2023.
  • Cell therapy is also being used to treat other diseases, including type 1 diabetes and sickle cell anemia.

RNA Therapies

  • RNA therapies involve the use of RNA molecules to modulate gene expression.
  • ASOs and siRNAs are two types of RNA therapies being used to treat diseases.
  • RNA therapies have the potential to treat a wide range of diseases, including genetic disorders and cancer.

Immunotherapy

  • Immunotherapy involves the use of the immune system to treat diseases.
  • Checkpoint inhibitors and cell therapies are two types of immunotherapies being used to treat cancer.
  • Immunotherapy has the potential to revolutionize cancer treatment and provide long-term remissions and potential cures.

Peptides

  • Peptides are short chains of amino acids that can be used to treat diseases.
  • GLP-1 receptor agonists are a type of peptide being used to treat type 2 diabetes and obesity.
  • Peptides have the potential to treat a wide range of diseases, including cancer and autoimmune disorders.

Episode Summary

  • check_circleThe complexity of drug development has exploded, with a new therapeutic toolkit including engineered cells, genetic instructions, and digital design.
  • check_circleThe market is shifting towards biologics, with biologics accounting for 42% of global pharma sales in 2023.
  • check_circleArtificial intelligence is acting as a connective tissue, linking different approaches together and enabling the development of new therapies.
  • check_circleGene editing, cell therapy, and RNA therapies are becoming increasingly important, with CRISPR-based gene editing being a major breakthrough.
  • check_circleThe first CRISPR-based gene editing therapy, CAS-GEBI, was approved in 2023 for sickle cell anemia.
  • check_circleCell therapy is also showing promise, with the first approval of a TIL therapy for advanced melanoma in 2023.
  • check_circleRNA therapies, including ASOs and siRNAs, are being used to modulate gene expression and treat diseases.
  • check_circleThe use of peptides, such as GLP-1 receptor agonists, is also becoming increasingly popular.
  • check_circleImmunotherapy, including checkpoint inhibitors and cell therapies, is revolutionizing cancer treatment.
  • check_circleThe future of therapy development lies in the integration of different modalities, including small molecules, biologics, and gene editing.

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