OrasomeTM Technology Platform

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OrasomeTM Technology 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
Designing a programmable and scalable approach for oral administration of nucleic acids and other biotherapeutics

PureTech is developing a versatile and programmable oral biotherapeutics platform, OrasomeTM, 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. 

PureTech’s Orasome technology platform was inspired by the in vivo trafficking of ubiquitous, naturally occurring vesicles, which are often referred to as exosomes, and PureTech has engineered them for transport through the gastro-intestinal tract. Exosomes are a type of extracellular vesicle approximately 100nm in diameter that are produced in the endosomal compartment and secreted from most types of eukaryotic cells. Human cell-derived exosomes have attractive promise as vehicles for systemic drug delivery due to their tolerability over synthetic polymer-based delivery technologies. However, the fragile nature of exosomes derived from human cells limits their usage for oral administration and the type of post-isolation manipulations that can be applied in order to optimize such vesicles for exogenous drug cargo loading and storage. 

PureTech’s Orasome technology platform utilizes multiple vesicle components, including those isolated from milk. PureTech has engineered these vesicles, building on the naturally evolved architecture in mammals, to remain stable following oral consumption and transit through the upper GI tract. Orasome vesicles are readily amenable to manufacturing at scale and relatively low cost based on the easily accessible and engineerable components.

We are advancing a broad pipeline of programs to treat cognitive deficiency and improve symptoms associated with medical conditions across neurology and psychiatry, including attention-deficit hyperactivity disorder (ADHD), major depressive disorder (MDD), autism spectrum disorder (ASD), multiple sclerosis (MS) and various other neuroinflammatory diseases. We are also developing associated clinical monitors and measurement-based care applications. Our lead, patented technology platform is exclusively licensed from the lab of Dr. Adam Gazzaley at the University of California, San Francisco (UCSF), and augmented by proprietary adaptive algorithms developed by our team.

  • Patient Need & Market Potential
    • PureTech’s proprietary Orasome technology platform has the potential to transform the treatment paradigm for diseases, such as rheumatoid arthritis, other autoimmune diseases, diabetes and cancer for which the standard of care often requires intravenous infusion or subcutaneous injection of monoclonal antibodies (e.g. anti-PD1, anti-TNF) or therapeutic proteins/peptides (e.g., GLP-1, insulin, GCSF, Factor VIII and IX, cytokines, and erythropoietin, among others.
  • Innovative Approach for Solving the Problem
    • PureTech’s Orasome vesicles are currently constructed to transport macromolecular medicines to selected mucosal cell types of the intestinal tract where the therapeutics act either directly in the GI tract, transit through the mucosa to the underlying lymphatic vascular network or, in the case of cargos that yield mRNAs, enable the body to produce its own therapeutic proteins and peptides, such as antibodies within mucosal cells that are secreted into the mucosal lymphatic vascular network for subsequent systemic distribution. Using PureTech’s Orasome technology platform, it may be possible for a patient to take an oral drug product that will permit their own GI tract cells to make virtually any type of therapeutic protein. This approach also has the potential to provide a more convenient and significantly less expensive means to deliver biological medicines. 
    • Within the context of the current COVID-19 pandemic, PureTech’s Orasome technology platform has the potential to support oral administration of anti-SARS CoV-2 monoclonal antibodies or antibody combinations and vaccines to supply passive immune therapies for infected individuals and passive immune protection for health care and first responder professionals.
    • Thus, whether combating emerging epidemic/pandemic pathogens or other diseases where monoclonal antibody therapeutics or vaccines offer significant clinical benefit, the Orasome technology platform has the potential to transform the range of biotherapeutics clinical indications, while also lowering costs and simplifying administration. 
  • Intellectual Property
    • PureTech has broad intellectual property coverage for its Orasome technology platform. PureTech’s 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 oral biotherapeutics, which include nucleic acid-based therapeutics (such as messenger RNA, nucleic acid expression systems, short interfering RNA and antisense oligonucleotide-based approaches), small molecules, biologics (such as peptides, proteins and antibodies) and other therapeutics for use in the treatment of a wide range of diseases and disorders, including various cancers, inflammatory diseases, autoimmune diseases, infectious diseases and orphan diseases.
  • Expected milestones
    • PureTech expects preclinical proof-of-concept data in 2021 and anticipates results from a non-human primate proof-of-concept study in 2021. The proof-of-concept studies are designed to document the presence of therapeutic serum levels of biotherapeutics (peptides and proteins, such as antibodies) produced by the body following the oral administration designer payloads. 
    • This work could lay the foundation for IND-enabling clinical studies for one or more additional product candidates to be included in the company’s Wholly Owned Pipeline.
PureTech is well-positioned to unleash the potential of oral biotherapeutics