Orasome™ and Other Technologies Platform for Oral Administration of Therapeutics

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Preclinical
Phase 1
Phase 2
Phase 3
OrasomeTM and Other Technologies Platform

A versatile and programmable oral biotherapeutics platform being developed to enable administration of macromolecule therapeutic payloads, including antisense oligonucleotides, short interfering RNA, messenger RNA (mRNA), modular expression vector systems, peptides and nanoparticles that are otherwise administered exclusively by injection.

Phase completedPhase in progress
Programmable and scalable approach for oral administration of engineered expression systems to generate therapeutic proteins

The PureTech Orasome platform allows for oral administration of a new generation of engineered expression systems that selectively infect small intestine enterocytes and produce therapeutic proteins. The virus-encoded proteins are secreted into the mesenteric lymphatic system leading to systemic distribution of the therapeutic entity. The expression vectors are localized to GI tract enterocytes and do not enter the circulation. The expression vectors can be multi-cistronic and have the capacity to encode even large therapeutic proteins such as immunoglobulins. It is anticipated that the Orasome platform will have potentially broad therapeutic applications including hormone generation, protein/enzyme replacement, immunoglobulin-based treatments and vaccines. We believe this approach has the potential to provide a more convenient and less expensive means to administer biological medicines.

Key advantages of our Orasome platform include:

  • Oral administration (flexible repeat dosing)

  • Body manufactures the therapeutic proteins

  • Low immune & cell toxicity (protein synthesis in GI tract enterocytes)

  • Low dose requirement for protein production

Validation of this technology:

  • We have achieved initial platform proof of concept in rodents by generation of physiologically relevant levels of human erythropoietin following a single administration and are currently evaluating expression profiles of additional peptide hormones such as GLP-1, checkpoint blockade immunoglobulins and secreted locked trimer SARS CoV-2 spike protein.

 

  • Key Points of Innovation & Differentiation
    • Our proprietary oral administration technologies, including Orasome and Other Technologies Platform, have the potential to transform the treatment paradigm for diseases such as rheumatoid arthritis, diabetes, other autoimmune diseases and cancer for which the standard of care often requires intravenous infusion or subcutaneous injection of monoclonal antibodies (e.g., anti-PD-1, anti-tumor necrosis factor) or therapeutic proteins/peptides (e.g., glucagon-like peptide-1, insulin, granulocyte colony-stimulating factor GCSF, Factor VIII and IX, cytokines and erythropoietin), among others.

     

  • Program Discovery Process by the PureTech Team
    • We sought out different approaches to enable the oral administration of macromolecule therapeutic payloads that are otherwise administered exclusively by injection. We have independently developed our Orasome and Other Technologies Platform and have generated data and intellectual property supporting oral administration of macromolecule therapeutic payloads. We are also developing other oral administration technologies and intellectual property.
  • Milestones Achieved & Development Status
    • In 2021, we established preclinical proof-of-concept supporting the potential of our Orasome and Other Technologies Platform to achieve production of therapeutic proteins in the gut of an animal following simulated oral administration of expression systems and transport of these proteins from the gut into systemic circulation. Proof-of-concept was observed with multiple formulations involving Orasome and Other Technologies Platform, which are being further optimized to achieve a range of expression profiles for therapeutic proteins.
  • Expected Milestones
    • We continue to generate additional data in preclinical models to optimize and validate the Orasome and Other Technologies Platform. We intend to leverage our proprietary technology platforms, as well as our extensive network with major pharmaceutical companies and world-leading scientists in immunology and lymphatics, to generate additional novel therapeutic candidates.
  • Intellectual Property
    • We have broad intellectual property coverage for our Orasome technology platform. Our Orasome technology platform intellectual property portfolio covers compositions of matter, methods of use and methods of treatment spanning various platform-based technologies, as well as various broad classes of Orasome-formulated therapeutics, for use in the prevention and treatment of a wide range of diseases, including various immunological disorders, such as cancers, inflammatory diseases and emerging pathogenic viruses.
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