Oral Biotherapeutics Platform

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Oral Biotherapeutics Platform

A milk exosome-based technology being developed to enable oral administration of macromolecule therapeutic payloads, including antisense oligonucleotides, short interfering RNA, messenger RNA (mRNA), peptides and nanoparticles that are otherwise administered exclusively by injection.

Phase completedPhase in progress
Harnessing milk exosomes for oral administration of nucleic acids and other biologics

PureTech is developing a milk exosome-based technology to enable oral administration of macromolecule therapeutic payloads, including antisense oligonucleotides, short interfering RNA, messenger RNA (mRNA), peptides and nanoparticles that are otherwise administered exclusively by injection. 

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 the type of post-isolation manipulations that can be applied in order to optimize such vesicles for exogenous drug cargo loading, administration and storage. This contrasts with milk-derived exosomes, which form the basis of PureTech’s technology and have evolved in all mammals to remain stable following oral consumption and transit through the upper GI tract.

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.

  • Innovative Approach for Solving the Problem
    • PureTech’s platform utilizes bovine-derived milk exosomes. Bovine milk is a rich, readily available and inexpensive source of exosomes harboring approximately 1011 to 1012 purifiable exosomes per milliliter. By comparison, serum or plasma contains approximately 1,000-fold fewer exosomes (108 to 109 exosomes) per milliliter. The concept for utilizing bovine milk-derived exosomes for drug delivery is based on earlier research conducted in the laboratory of Ramesh Gupta, PhD, at the University of Louisville. PureTech has inlicensed the underlying foundational intellectual property derived from this research. Subsequently, PureTech has expanded and industrialized this technology developing easily scalable processes for low cost exosome purification, efficient universal cargo loading and formulations for oral administration. 
    • PureTech’s milk-derived exosome platform is 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, produce complex biologics such as antibodies within mucosal cells that are secreted into the mucosal lymphatic vascular network for subsequent systemic distribution. Using PureTech’s milk exosome technology, 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. This proprietary milk-derived exosome technology has the potential to alter the treatment paradigm for diseases, such as rheumatoid arthritis, diabetes and cancer for which the standard of care requires intravenous infusion or subcutaneous injection of monoclonal antibodies (e.g. anti-PD1, anti-TNF) or protein/peptides (e.g. GLP-1, β-glucocerebrosidase, Factor IX, Erythropoietin). Within the context of the current COVID-19 pandemic, PureTech’s milk-derived exosome platform has the potential to support oral administration of anti-SARS CoV-2 monoclonal antibodies or antibody combinations 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 offer significant clinical benefit, the milk exosome 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 milk exosome platform technologies, which includes both exclusively licensed and company-owned patents and patent applications. PureTech’s milk exosome 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 milk-exosome formulated therapeutics, 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 and inflammatory diseases. 
    • As of 31 December 2019, PureTech’s milk exosome patent portfolio consists of eight patent families comprising three issued U.S. patents, five U.S. patent applications, two international PCT applications and five foreign patent applications. Of these, company-owned IP consists of eight U.S. and foreign patent applications and one pending international PCT application in four patent families. PureTech exclusively licensed a patent portfolio consisting of two patent families from 3P Biotechnologies, Inc. based on technology originating from the University of Louisville. In addition, PureTech exclusively licensed a patent portfolio consisting of two patent families from NuTech Ventures based on technology originating from the University of Nebraska. PureTech’s issued patents and any patents issuing from their related filings are expected to expire in 2034 and 2037. Any issued patents from the other patent applications are expected to expire in 2037 through 2041, exclusive of possible patent term adjustments or extensions or other forms of exclusivity.

This figure depicts milk exosomes enabling the oral intake of large molecules via the gut and the lymphatic system.