Exosome Technologies: Recent Surge in R&D with Capricor, Codiak and Evox Therapeutics among Key Players
- GBI Research
- October 2018
- 93 pages
GBI Research’s latest report, Exosome Technologies: Recent Surge in R&D with Capricor, Codiak and Evox Therapeutics among Key Players, explores the application of exosome technologies within the pharmaceutical and healthcare industries. Exosomes are small cell-derived vesicles that are abundant in bodily fluids, including blood, urine and cerebrospinal fluid as well as in in vitro cell culture.
These vesicles are being used in a variety of therapeutic applications, including as therapeutic biomarkers, drug delivery systems and therapies in their own right. Research within this area remains in the nascent stages, although a number of clinical trials have been registered within the field. Exosomes have several diverse therapeutic applications, largely centering on stem cell and gene therapy. Exosomes have been identified as endogenous carriers of RNA within the body, allowing for the intercellular transportation of genetic material to target cells.
As such, developers have worked to engineer exosomes for the delivery of therapeutic miRNA and siRNA-based gene therapies. As RNA is highly unstable within the body, a number of different biologic vector systems have been developed to enhance their transport within the circulation, including viruses and liposomes. Similarly, exosomes derived from stem cells have also been identified for their therapeutic applications, particularly in the treatment of cancer and cardiovascular disease. Exosome technologies offer several advantages over existing biologic-based drug delivery systems.
They have a long circulatory half-life as a result of their high stability and ability to avoid breakdown by the mononuclear phagocyte system and reticuloendothelial systems. Moreover, exosomes have several functional properties that favor their use in therapeutic delivery. Exosomes can be engineered to incorporate targeting ligands, allowing them to deliver cargo selectively to cells. Their small size allows them to penetrate the blood-brain barrier for the delivery of central nervous system therapies, whereas in cancer they can accumulate within the tumor via enhanced permeability and retention effects.
Finally, clinical trials have shown relatively large-scale production to be possible and indicate that exosome therapies can be safely administered to humans. Additionally, exosomes are being investigated for their potential as prognostic and diagnostic biomarkers for several different disease indications. Exosomes make good candidates for biomarker research because of two unique characteristics: their presence in various accessible bodily fluids, and their resemblance to their parent cells of origin. R&D in exosome technologies has increased markedly in recent years. This report provides detailed information on the various healthcare applications of exosomes, and assesses the pipeline, clinical trial and company landscapes.
- What are the features of the exosome lifecycle?
- How are therapeutic exosomes prepared?
- How do exosome therapies in development differ in terms of stage of development, molecule type and therapy area?
- Which companies are investing in exosome technologies?
- How many clinical trials investigate exosomes as biomarkers, therapeutics and vectors?
Reasons to buy
- Develop a comprehensive understanding of exosome technologies and their potential for use within the healthcare sector.
- Analyze the pipeline landscape and gain insight into the key companies investing in exosomes technologies.
- Identify trends in interventional and observational clinical trials relevant to exosomes.
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Table of Contents
1 Table of Contents
1 Table of Contents 4
1.1 List of Tables 6
1.2 List of Figures 7
2 Exosomes in Healthcare 8
2.1 Overview of Exosomes 8
2.2 Drug Delivery Systems 9
2.2.1 Modified Release Drug Delivery Systems 9
2.2.2 Targeted Drug Delivery Systems 10
2.2.3 Liposomes 12
2.2.4 Viruses 14
2.2.5 Exosomes 17
2.3 The Exosome Lifecycle 18
2.4 Exosomes in Biology 18
2.5 Exosomes in Medicine 19
2.5.1 Biomarkers 19
2.5.2 Vaccines 20
2.6 Exosomes as a Therapeutic Target 20
2.7 Exosomes as Drug Delivery Vehicles 21
2.8 Therapeutic Preparation of Exosomes 21
2.8.1 Isolation and Purification 22
2.8.2 Drug Loading 22
2.8.3 Characterization 23
2.8.4 Bioengineering 23
2.8.5 Biodistribution and In Vivo Studies 23
2.8.6 Advantages of Exosome Therapies 24
2.8.7 Disadvantages of Exosome Therapies 24
2.9 Exosomes in Therapeutic Research 25
2.9.1 Exosome Gene Therapies 25
2.9.2 Exosome in Stem Cell Therapy 26
2.10 Exosomes in Oncology 27
2.10.1 Immunotherapy 27
2.10.2 Gene Therapy 28
2.10.3 Drug Delivery 29
2.10.4 Biomarkers 30
2.11 Exosomes in CNS Disease 30
2.11.1 Tackling the Blood-Brain Barrier 30
2.11.2 Exosomes in CNS Drug Delivery 31
2.11.3 Gene Therapy 32
2.12 Exosomes in Other Diseases 33
2.12.1 Cardiovascular Disease 33
2.12.2 Metabolic Disease 33
3 Assessment of Pipeline Product Innovation 36
3.1 Overview 36
3.2 Exosome Pipeline by Stage of Development and Molecule Type 36
3.3 Pipeline by Molecular Target 37
3.4 Pipeline by Therapy Area and Indication 38
3.5 Pipeline Product Profiles 38
3.5.1 AB-126 - ArunA Biomedical Inc. 38
3.5.2 ALX-029 and ALX-102 - Alxerion Biotech 39
3.5.3 Biologics for Autism - Stem Cell Medicine Ltd 39
3.5.4 Biologic for Breast Cancer - Exovita Biosciences Inc. 39
3.5.5 Biologics for Idiopathic Pulmonary Fibrosis and Non-alcoholic Steatohepatitis - Regenasome Pty 39
3.5.6 Biologic for Lysosomal Storage Disorder - Exerkine 39
3.5.7 Biologics for Prostate Cancer - Cells for Cells 40
3.5.8 CAP-2003 - Capricor Therapeutics Inc. 40
3.5.9 CAP-1002 - Capricor Therapeutics Inc. 41
3.5.10 CIL-15001 and CIL-15002 - Ciloa 42
3.5.11 ExoPr0 - ReNeuron Group Plc 42
3.5.12 MVAX-001 - MolecuVax Inc. 43
3.5.13 Oligonucleotides to Activate miR124 for Acute Ischemic Stroke - Isfahan University of Medical Sciences 44
3.5.14 Oligonucleotides to Inhibit KRAS for Pancreatic Cancer - Codiak BioSciences Inc. 44
3.5.15 Proteins for Neurology and Proteins for CNS Disorders and Oligonucleotides for Neurology - Evox Therapeutics Ltd 44
3.5.16 TVC-201 and TVC-300 - Tavec Inc. 45
4 Assessment of Clinical Trial Landscape 48
4.1 Interventional Clinical Trials 48
4.1.1 Clinical Trials by Therapy Type 48
4.1.2 Clinical Trials by Therapy Area 49
4.1.3 Clinical Trials by Stage of Development 50
4.1.4 Clinical Trials by Start Date and Status 50
4.2 Observational Clinical Trials 51
4.2.1 Clinical Trials by Therapy Type 51
4.2.2 Clinical Trials by Therapy Area 51
4.2.3 Clinical Trials by Stage of Development 52
4.2.4 Clinical Trials by Start Date and Status 53
4.2.5 List of All Clinical Trials 54
5 Company Analysis and Positioning 67
5.1 Company Profiles 67
5.1.1 Capricor Therapeutics Inc. 67
5.1.2 Evox Therapeutics Ltd 72
5.1.3 ReNeuron Group Plc 73
5.1.4 Stem Cell Medicine Ltd 77
5.1.5 Tavec Inc. 78
5.1.6 Codiak Biosciences Inc. 80
5.1.7 Therapeutic Solutions International Inc. 81
5.1.8 ArunA Biomedical Inc. 83
5.1.9 Ciloa 85
Table 1: Exosome Technologies, Global, Examples of Modified Release Dosage Systems, 2018 10
Table 2: Exosome Technologies, Global, Challenges and Opportunities in Drug Delivery, 2018 11
Table 3: Exosome Technologies, Global, Breakdown of Lipid-Based Drug Delivery Systems, 2018 13
Table 4: Exosome Technologies, Global, Types of Viral Vector for Gene Therapy Delivery, 2018 15
Table 5: Exosome Technologies, Global, Capricor Pipeline Products, 2018 69
Table 6: Exosome Technologies, Global, Capricor Clinical Trials, 2018 70
Table 7: Exosome Technologies, Global, Capricor Completed Deals, 2009-2018 72
Table 8: Exosome Technologies, Global, Evox Pipeline Products, 2018 73
Table 9: Exosome Technologies, Global, Evox, Completed Deals, 2018 73
Table 10: Exosome Technologies, Global, ReNeuron Pipeline Products, 2018 75
Table 11: Exosome Technologies, Global, ReNeuron Clinical Trials, 2010-2019 75
Table 12: Exosome Technologies, Global, ReNeuron Completed Deals, 2018 76
Table 13: Exosome Technologies, Global, Stem Cell Medicine Pipeline Products, 2018 78
Table 14: Exosome Technologies, Global, Stem Cell Medicine Completed Deals, 2018 78
Table 15: Exosome Technologies, Global, Tavec Pipeline Products, 2018 79
Table 16: Exosome Technologies, Global, Tavec Clinical Trials, 2018 79
Table 17: Exosome Technologies, Global, Tavec Completed Deals, 2018 79
Table 18: Exosome Technologies, Global, Codiak Biosciences Pipeline Products, 2018 80
Table 19: Exosome Technologies, Global, Codiak Biosciences Clinical Trials, 2018 81
Table 20: Exosome Technologies, Global, Codiak Biosciences Completed Deals, 2018 81
Table 21: Exosome Technologies, Global, Therapeutic Solutions International Pipeline Products, 2018 82
Table 22: Exosome Technologies, Global, Therapeutic Solutions International Clinical Trials, 2018 82
Table 23: Exosome Technologies, Global, Therapeutic Solutions International Completed Deals, 2018 83
Table 24: Exosome Technologies, Global, ArunA Biomedical Pipeline Products, 2018 84
Table 25: Exosome Technologies, Global, ArunA Biomedical Clinical Trials, 2018 84
Table 26: Exosome Technologies, Global, ArunA Biomedical Completed Deals, 2018 84
Table 27: Exosome Technologies, Global, Ciloa Pipeline Products, 2018 85
Table 28: Exosome Technologies, Global, Ciloa Completed Deals, 2018 85
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