Table 9.3.
BEVs produced biomolecules as disease therapeutic agents
| Therapy type | Targeted for | Product type | Product name | Expressed product | Development stage | Company name, if any |
|---|---|---|---|---|---|---|
| Immunotherapy | Prostate cancer | Recombinant fusion protein | Provenge or sipuleucel-T | PAP-GM-CSF^ | Approved | Dendreon |
| Immunotherapy | Colorectal carcinoma | Monoclonal antibodies | Anti-GA733-2E | CO17-1A Mab (IgG2a) | Unapproved | |
| Immunotherapy | Haematolymphoid cells | Recombinant protein | Anti-Bcl-2-Mab | B-cell lymphoma leukaemia-2 (Bcl-2) protein | Unapproved | |
| Immunotherapy | Rotavirus | Single-domain antibodies (sdAbs) | 3B2 and 2KD1 antibodies | Anti-VP6 | Unapproved | |
| Immunotherapy | Breast cancer | Monoclonal antibodies | mAb-BR55/mAb-BR55K | HC and LC | Unapproved | |
| Immunotherapy | Antigen-presenting cells (APCs) | Adjuvant antibody | APCH1 antibody | Anti-MHC class II DR | Unapproved | |
| Immunotherapy | Immune cells | Cytokine | IL-2 | Human interleukin 2 | Unapproved | |
| Immunotherapy | Stem cells, macrophages | Cytokine | hGM-CSF | Human granulocyte-macrophages colony-stimulating factor | Unapproved | |
| Enzyme therapy | Purine salvage pathway | Enzyme | ADA | Human adenosine deaminase | Unapproved | |
| Hormone therapy | Hypoparathyroidism | Hormone | hPTH | Human parathyroid hormone | Unapproved | |
| Growth factor therapy | Wound healing | Growth factor | huEGF1 | Human epidermal growth factor | Unapproved | |
| Growth factor therapy | Growth factor | huFGF2 | Human fibroblast growth factor 2 | Unapproved | ||
| Growth factor therapy | Growth factor | huKGF1 | Human keratinocyte growth factor 1 | Unapproved | ||
| Growth factor therapy | Alzheimer’s disease | Growth factor | rhNGF | Human prepro (beta) nerve growth factor | Unapproved | |
| Enzyme-gene therapy | Familial lipoprotein lipase deficiency | Transgene | Glybera or LPLS447 × transgene | Lipoprotein lipase transgene | Approved | UniQure |
| Protein gene therapy | Haemophilia A | Transgene | AAV-FVIII | Factor VIII | Unapproved | |
| Protein gene therapy | Haemophilia B | Transgene | AAV8-hFIX19 | Factor IX | Phase I | Spark Therapeutics |
| AskBio009 (AAV8) | Phase I/II | Baxalta US Inc. | ||||
| scAAV 2/8-LP1-hFIXco | Phase I | St. Jude Children’s Research Hospital | ||||
| AAV2-hFIX16 | Phase I | Spark Therapeutics | ||||
| Enzyme-gene therapy | Leber congenital amaurosis | Transgene | AAV2-hRPE65v2 | Retinoid isomerohydrolase | Phase III | Spark Therapeutics |
| rAAV2-CB-hRPE65 | Phase I/II | Applied Genetic Technologies Corp | ||||
| tgAAG76 (rAAV 2/2.hRPE65p.hRPE65) | Phase I/II | University College, London | ||||
| rAAV2/4.hRPE65 | Phase I/II | Nantes University Hospital | ||||
| rAAV2-CBSB-hRPE65 | Phase I | University of Pennsylvania | ||||
| rAAV2-hRPE65 | Phase I | Hadassah Medical Organization | ||||
| Enzyme-gene therapy | Leber’s hereditary optic neuropathy | Transgene | AAV2-ND4 | NADH-ubiquinone oxidoreductase chain 4 | Phase I | John Guy, University of Miami |
| Enzyme-gene therapy | Age-related macular degeneration | Transgene | AAV2-soluble Flt1 | Soluble fms-like tyrosine kinase | Phase I | Genzyme, a Sanofi Company |
| Enzyme-gene therapy | Canavan disease | Transgene | AAV-ASAP | Aspartoacylase | Unapproved | |
| Growth factor-gene therapy | Alzheimer’s disease | Transgene | AAV-NGF or CERE-110 | Beta-nerve growth factor | Phase I | Sangamo Therapeutics (Ceregene) |
| Enzyme-gene therapy | Parkinson’s disease | Transgene | AAV2-GAD | Glutamic acid decarboxylase | Phase II | Neurologix, Inc. |
| Protein gene therapy | AAV2-NTN or CERE-120 | Neurturin | Phase II | Ceregene | ||
| Enzyme-gene therapy | AAV-hAADC-2 | Aromatic L-amino acid decarboxylase | Phase I | Genzyme, a Sanofi Company | ||
| AAV2-hAADC | Phase I | Voyager Therapeutics | ||||
| Protein gene therapy | AAV2-GDNF | Glial cell line-derived neurotrophic factor | Phase I | National Institutes of Health Clinical Center (CC) | ||
| Protein gene therapy | Duchenne muscular dystrophy | Transgene | rAAV2.5-CMV-minidystrophin | Mini-dystrophin | Phase I | Asklepios BioPharmaceutical, Inc. |
| Protein gene therapy | Becker muscular dystrophy | Transgene | rAAV1.CMV.huFollistatin344 | Follistatin | Phase I | Nationwide Children’s Hospital |
| Protein gene therapy | Limb girdle muscular dystrophy | Transgene | AAV1-gamma-sarcoglycan | Gamma-sarcoglycan | Phase I | Genethon |
| Protein gene therapy | Spinal muscular atrophy | Transgene | scAAV9.CB.SMN | Survival motor neuron | Phase I | AveXis, Inc. |
| Enzyme-gene therapy | Acute intermittent porphyria | Transgene | rAAV2/5-PBGD | Porphobilinogen deaminase | Phase I | Digna Biotech S.L. |
| Enzyme-gene therapy | Alpha 1-antitrypsin deficiency | Transgene | rAAV1-CBhAAT | Alpha 1-antitrypsin | Phase II | Applied Genetic Technologies Corp |
| Enzyme-gene therapy | rAAV2-CBhAAT | Phase I | University of Massachusetts, Worcester | |||
| Enzyme-gene therapy | AAVrh.10halpha1AT | Phase I | Adverum Biotechnologies, Inc. | |||
| Enzyme-gene therapy | Aromatic amino acid decarboxylase deficiency | Transgene | AAV2-hAADC | Aromatic L-amino acid decarboxylase | Phase I/II | National Taiwan University Hospital |
| Protein gene therapy | Choroideremia | Transgene | rAAV2.REP1 | Rab-escort protein 1 | Phase I | University of Oxford |
| Enzyme-gene therapy | Chronic heart failure | Transgene | AAV1-CMV-SERCA2a | Sarcoplasmic reticulum calcium ATPase | Phase II | Imperial College London, Assistance Publique—Hôpitaux de Paris and Celladon Corporation |
| Protein gene therapy | Gastric cancer | Transgene | AAV-DC-CTL | Carcinoembryonic antigen | Phase I | Tianjin Medical University Cancer Institute and Hospital |
| Enzyme-gene therapy | HIV | Transgene | AAV-2 HIV vaccine (tgAAC09) | Gag, protease and reverse transcriptase parts | Phase I | International AIDS Vaccine Initiative |
| Antibody-gene therapy | rAAV1-PG9DP | PG9 antibody | Phase I | International AIDS Vaccine Initiative | ||
| Receptor-gene therapy | Inflammatory arthritis | Transgene | tgAAC94 | TNFR:Fc fusion gene | Phase 1/II | Targeted Genetics Corporation |
| Protein gene therapy | Late infantile neuronal ceroid lipofuscinosis | Transgene | AAVrh.10CUCLN2 | Neuronal ceroid-lipofuscinosis 2 | Phase 1/II | Weill Medical College of Cornell University |
| Protein gene therapy | AAV2CUhCLN2 | Neuronal ceroid-lipofuscinosis 2 | Phase I | Weill Medical College of Cornell University | ||
| Trinucleotide-gene therapy | Pompe disease | Transgene | rAAV1-CMV-GAA | Normal GAA | Phase 1/II | University of Florida |