IV Tramadol

• Gritti, G. et al. Multicenter trial comparing tramadol and morphine for pain after abdominal surgery. Drugs Exp Clin Res. 1998;24(1):9-16.
• Lu, L., et al. Comparing the Pharmacokinetics of 2 Novel Intravenous Tramadol Dosing Regimens to Oral Tramadol: A Randomized 3-Arm Crossover Study. Clinical Pharmacology in Drug Development
• Magrini, M. et al. Analgesic activity of tramadol and pentazocine in postoperative pain. Int J Clin Pharmacol Res. 1998;18(2):87-92.
• Scott, L. et al. Tramadol: A Review of its Use in Perioperative Pain. Adis International Limited, Drugs 2000 Jul; 60 (1): 139-176.
• Scott, L.J., Perry, C.M. Tramadol: a review of its use in perioperative pain. Drugs. 2000 Jul;60(1):139-76.
• Tarradell, R. et al. Respiratory and analgesic effects of meperidine and tramadol in patients undergoing orthopedic surgery. Methods Find Exp Clin Pharmacol. 1996 Apr;18(3):211-8.
• Pain Management in the Postoperative Environment

CUTX-101

• Kaler S. G., et al. Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome Aggregation Database (gnomAD). Molecular Genetics and Metabolism Reports. June 2020.
• Parad, R. B., et al. Targeted next generation sequencing for newborn screening of Menkes disease. Molecular Genetics and Metabolism Reports. July 2020.
• Kaler, S et al. Neurodevelopment and brain growth in classic Menkes disease is influenced by age and symptomatology at initiation of copper treatment. J Trace Elem Med Biol. 2014 Oct; 28(4): 427–430. Published online 2014 Aug 28. doi: 10.1016/j.jtemb.2014.08.008
• Haddad, M et al. Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model. Mol Ther Methods Clin Dev. 2018 Sep 21; 10: 165–178. Published online 2018 Jul 9. doi: 10.1016/j.omtm.2018.07.002
• Kaler, S. et al. Neonatal Diagnosis and Treatment of Menkes Disease New England Journal of Medicine. February 7, 2008; N Engl J Med 2008; 358:605-614 DOI: 10.1056/NEJMoa070613.
• Kaler, S. ATP7A-related copper transport diseases —emerging concepts and future trends. Nat Rev Neurol. 2011 Jan; 7(1): 15–29. doi: 10.1038/nrneurol.2010.180

MB-107 (Lentiviral Gene Therapy for XSCID)

• Zhou, S. et al. A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells. Blood. 2010;116(6):900–908. doi:10.1182/blood-2009-10-250209.
• Greene, M. et al. Transduction of Human CD34+ Repopulating Cells with a Self-Inactivating Lentiviral Vector for SCID-X1 Produced at Clinical Scale by a Stable Cell Line. Hum Gene Ther Methods. Hum Gene Ther Methods. 2012 Oct; 23(5): 297–308. Published online 2012 Sep 11. doi: 10.1089/hgtb.2012.150.
• Ravin, S et al. Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. June 2016;8(341):341er5]. Sci Transl Med. 2016;8(335):335ra57. doi:10.1126/scitranslmed.aad8856
• Mamcarz, E. et al. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. New England Journal of Medicine. April 18, 2019; N Engl J Med 2019; 380:1525-1534 DOI: 10.1056/NEJMoa1815408.

CAEL-101

• Hrncic, R., Wall, JS. et al. Antibody-Mediated Resolution of Light Chain-Associated Amyloid Deposits. Am J Pathol 2000; 157(4): 1239-1246.
• Bhutani, D. et al. Improvement in Global Longitudinal Strain (GLS) Correlates with NT-ProBNP Response in Patients with Cardiac Amyloidosis Treated on a Phase 1b Study of Anti-Amyloid mAb CAEL-101. 60th Annual American Society of Hematology; 2018. Abstract 958.
• Fu, J. et al. Personalizing Amyloidosis Therapy with Real Time PET Imaging of Fibril-Reactive Monoclonal Antibody CAEL-101. 60th Annual American Society of Hematology; 2018. Abstract 1003.
• O’Nuallain, B. et al. Localization of a Conformational Epitope Common to Non-Native and Fibrillar Immunoglobulin Light Chains. Biochemistry 2007; 46(5): 1240-1247.
• Edwards, C. et al. Final Analysis of the Phase 1a/b Study of Fibril-Reactive Monoclonal Antibody 11-1F4 (CAEL-101) in Patients with AL Amyloidosis. 16th International Symposium on Amyloidosis (ISA); 2018.
• Solomon, A., Weiss, D.T., Wall, JS. Immunotherapy in Systemic Primary (AL) Amyloidosis Using Amyloid Reactive Monoclonal Antibodies. Cancer Biother Radiopharm 2003; 18(6): 853-60.
• O’Nuallain, B. et al. Phage Display and Peptide Mapping of an Immunoglobulin Light Chain Fibril-Related Conformational Epitope. Biochemistry 2007; 46(45): 1349-58.
• Langer, A. et al. Results of Phase I Study of Chimeric Fibril-Reactive Monoclonal Antibody 11-1F4 in Patients with AL Amyloidosis. 57th Annual American Society of Hematology; December 2015. Abstract 188.
• Edwards, C. et al. Analysis of the Phase 1a/b Study of Chimeric Fibril-Reactive Monoclonal Antibody 11-1F4 in Patients with AL Amyloidosis. 58th Annual American Society of Hematology; December 2016. Abstract 643.
• Edwards, C. et al. Final Analysis of the Phase 1a/b Study of Chimeric Fibril-Reactive Monoconal Antibody 11-1F4 in Patients with Relapsed or Refractory AL Amyloidosis. 59th Annual American Society of Hematology; 2017. Abstract 509.
• Wall, J.S., Kennel, S.J. et al. Radioimaging of Light Chain Amyloid with a Fibril Reactive Monoclonal Antibody. J Nucl Med 2006; 47(12): 2016-2024.
• Wall, J.S., Solomon, A., Kennel, S.J. Development and Evaluation of Agents for Targeting Visceral Amyloid. Tijdschr Nucl Geneeskd 2011; 33(4): 807-814.
• Valent, J, et al. CAEL-101 is Well-Tolerated in AL Amyloidosis Patients Receiving Concomitant Cyclophosphomide-Bortezomib-Dexamethasone (CyborD): A Phase 2 Dose-Finding Study (NCT04304144). 62nd Annual American Society of Hematology; December 2020. Abstract 2277.
• Valent, J. et al. Safety and Tolerability of CAEL-101 in Combination With Cyclophosphamide-Bortezomib-Dexamethasone and Daratumumab in Patients With AL Amyloidosis European Hematology Association 2021. June 2021. eposter 1018.
• Valent, J. et al. Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis In a Phase 2 Study for a Median of 49 Weeks. European Hematology Association 2021. June 2021. eposter 1017.
• Khouri, J, et al. Safety, Tolerability and Efficacy of CAEL-101 in AL Amyloidosis Patients Treated on a Phase 2, Open-Label, Dose Selection Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients with AL Amyloidosis. 62nd Annual American Society of Hematology; December 2020. Abstract 729.
• Wall, J.S. et al. Radioimmunodetection of Amyloid Deposits in Patients with AL Amyloidosis. Blood 2010; 116(13): 2241-4.

Cosibelimab

• Harris, D., et al. Cosibelimab, an anti-PD-L1 antibody: Preliminary Safety and Efficacy Results from a Global, Multicohort Phase 1 Clinical Trial. Society for Immunotherapy of Cancer (SITC) 35th Annual Meeting. 2020-11-09 Poster 399.
• Clingan, P., et al. Safety, Efficacy and Pharmacokinetic Profile of Cosibelimab, an Anti-PD-L1 Antibody, in Patients with Advanced Cancers. European Society for Medical Oncology (ESMO) Congress. 2019-09-28 Poster 469P.
• Gorelik, L. et al. Preclinical characterization of a novel fully human IgG1 anti-PD-L1 mAb CK-301. American Association for Cancer Research Annual Meeting (AACR). 2016-04-04 Abstract 4606.
• Lin, L., et al. Semi-mechanistic PK and target-occupancy modeling to support dose justification for anti-PD-L1 clinical candidate CK-301 (cosibelimab) in oncology patients. Society for Immunotherapy of Cancer (SITC) 34th Annual Meeting. 2019-11-08 Abstract P431

MB-102 (CD123 CAR T)

2017 Abstracts

• Budde, L. et al. Remissions of Acute Myeloid Leukemia and Blastic Plasmacytoid Dendritic Cell Neoplasm Following Treatment with CD123-Specific CAR T Cells: A First-in-Human Clinical Trial. 2017; Abstract #811.

Reference Articles

• Mardiros, A. et al. T cells expressing CD123-specific chimeric antigen receptors exhibit specific cytolytic effector functions and antitumor effects against human acute myeloid leukemia. Blood 2013;122: 3138–3148.
• Wang, X. et al. A transgene-encoded cell surface polypeptide for selection, in vivo tracking, and ablation of engineered cells. Blood 2011;118(5): 1255-1263.

• Mardiros, Armen et al. T cells expressing CD123 chimeric antigen receptors for treatment of acute myeloid leukemia. Current opinion in hematology vol. 22,6 (2015): 484-8. doi:10.1097/MOH.0000000000000190

Olafertinib

• Qian, X., et al. CK-101 (RX518), a mutant-selective inhibitor of EGFR that overcomes T790M-mediated resistance in NSCLC. American Association for Cancer Research Annual Meeting (AACR). 2016-04-03 Abstract 2078.
• Johnson, M., et al. CK-101 (RX518), a Third Generation Mutant-Selective Inhibitor of EGFR in NSCLC: Results of an Ongoing Phase I/II Trial. International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer. 2018-09-24 Abstract.

Triplex

• Aldoss, I. et al. Multi-Antigen Vaccine (Triplex) Based on Attenuated Poxvirus Prevents Cytomegalovirus Viremia in a Multi-Center Placebo-Controlled, Double Blinded, Randomized Phase 2 Clinical Trial in Allogeneic HCT Recipients.
• Diamond, D.J. et al. Safety, Maximum Tolerated Dose and Immunogenicity of CMV-MVA-Triplex in Healthy Volunteers with or without Prior Immunity to CMV and Vaccinia [abstract]. American Society of Hematology 57th Annual Meeting & Exposition; 2015 Dec 5-8; Orlando, FL. ASH; 2015. Abstract nr 3108.
• Diamond, D et al. 3108 Safety, Maximum Tolerated Dose and Immunogenicity of CMV-MVA-Triplex in Healthy Volunteers with or without Prior Immunity to CMV and Vaccinia American Society of Hematology 57th Annual Meeting & Exposition; 2015 Dec 5-8, Orlando, FL. ASH; 2015. Abstract nr 3108.
• La Rosa, C. et al. MVA vaccine encoding CMV antigens safely induces durable expansion of CMV-specific T-cells in healthy adults. Blood 2016: blood-2016-07-729756. doi: 10.1182.

CEVA-101 (Pediatric)

• Liao, GP., Harting, MT., Hetz ,RA., Walker, PA., Sh,a SK., Corkin,s CJ. et al. Autologous Bone Marrow Mononuclear Cells Reduce Therapeutic Intensity for Severe Traumatic Brain Injury in Children Pediatric Crit Care Med. 2015;16(3):245-55. Pmid:25581630

CEVA-101 (Adult)

• Cox Jr., C. S. et al. Treatment of Severe Adult Traumatic Brain Injury Using Bone Marrow Mononuclear Cells. STEM CELLS 2016. DOI: 10.1002/stem.2538.

MB-101 (IL13Rα2-specific CAR T)

2016 Abstracts

• Alizadeh, D. et al. Development of Murine IL13Rα2-Targeted CAR T Cells (mIL13BBz) for Assessment of CAR T Cell Therapy in Syngeneic Glioma Models. 21st Annual Meeting and Education Day of the Society for Neuro-Oncology, November 18, 2016. Abstract IMST-36.
• Brown, C. et al. Phase I Study of Chimeric Antigen Receptor-Engineered T Cells Targeting IL13Ra2 for the Treatment of Glioblastoma. 21st Annual Meeting and Education Day of the Society for Neuro-Oncology, November 18, 2016. Abstract ATIM-13.
• Brown, C. et al. Phase I Study of Second Generation Chimeric Antigen Receptor–Engineered T cells Targeting IL13Rα2 for the Treatment of Glioblastoma. 2016 American Society of Gene and Cell Therapy 19th Annual Meeting, May 4-7, 2016. Abstract #247.
• Aguilar, B. et al. Optimization of IL13Rα2-specific CAR T cells for Clinical Development Using Orthotopic Human Glioblastoma Models in NSG Mice. 2016 American Society of Gene and Cell Therapy 19th Annual Meeting, May 4-7, 2016. Abstract #275.

Reference Articles

• Brown, C et al. Optimization of IL13Ra2-Targeted Chimeric Antigen Receptor T Cells for Improved Anti-tumor Efficacy against Glioblastoma. Molecular Therapy. October 5, 2017; http://dio.org/10.1016/j.ymthe.2017.10.002.
• Jonnalagadda, M. et al. Chimeric Antigen Receptors With Mutated IgG4 Fc Spacer Avoid Fc Receptor Binding and Improve T Cell Persistence and Antitumor Efficacy. Mol Ther. 2015;23: 757–768.
• Brown, C. et al. Regression of Gliobastoma after Chimeric Antigen Receptor T-Cell Therapy. New England Journal of Medicine December 2016; 375: 2561-2569.

MB-103 (HER2 CAR T)

• Priceman, S. et al. Regional Delivery of Chimeric Antigen Receptor-Engineered T Cells Effectively Targets HER2+ Breast Cancer Metastasis to the Brain. Clinical Cancer Research. January 2018; 10.1158/1078-0432.CCR-17-204.

MB-104 (CS1 CAR T)

• Wang, X. et al. Lenalidomide Enhances the Function of CS1 Chimeric Antigen Receptor–Redirected T Cells Against Multiple Myeloma. Clinical Cancer Research. January 2018; 10.1158/1078-0432.CCR-17-0344.

MB-106 (CD20 CAR T)

• Shadman, M. et al. Third Generation CD20 targeted CAR T-Cell therapy (MB-106) for Treatment of Patients with Relapsed/Refractory B-NHL. American Society of Hematology Annual Meeting 2020; Abstract #1143.
• Shadman, M. et al. Immunotherapy Using a 3rd Generation CD20 CAR T-Cell (MB-106) for B-NHL and CLL. European Hematology Association 2021 Virtual Congress, June 9, 2021. Abstract EP731.
• Till, B. et al. Preclinical Optimization of a CD20-specific Chimeric Antigen Receptor Vector and Culture Conditions. Blood. 2012;119:3940-3950; doi: https://doi.org/10.1182/blood-2011-10-387969
• Till, B. et al. CD20-specific adoptive immunotherapy for lymphoma using a chimeric antigen receptor with both CD28 and 4-1BB domains: pilot clinical trial results. Blood. 2012;119:3940-3950; doi: https://doi.org/10.1182/blood-2011-10-387969.

MB-108 (Oncolytic Virus C134)

• γ134.5-Deleted HSV-1 Expressing Human Cytomegalovirus IRS1 Gene Kills Human Glioblastoma Cells as Efficiently as Wild-type HSV-1 in Normoxia or Hypoxia. Friedman, G.K. et al. Gene Ther. 2015 Apr; 22(4): 348–355. Published online 2014 Nov 27. doi: 10.1038/gt.2014.107.
• Cassady, K.A. et al. Pre-clinical Assessment of C134, a Chimeric Oncolytic Herpes Simplex Virus, in Mice and Non-human Primates. Molecular Therapy Oncolytics. June 2017; https://doi.org/10.1016/j.omto.2017.02.001
• Ghonime M.G. et al. Chimeric HGMV/HSV-1 and Δγ134.5 oncolytic herpes simplex virus elicit immune mediated antigliomal effect and antitumor memory. Translational Oncology. 2018;11(1):86–93. doi:10.1016/j.tranon.2017.10.005

MB-105 (PSCA CAR T)

• Priceman, S. et al. Co-stimulatory signaling determines tumor antigen sensitivity and persistence of CAR T cells targeting PSCA+ metastatic prostate cancer. Taylor & Francis Online. DOI: 10.1080/2162402X.2017.1380764

CK-103 BET Inhibitor

• Normant, E., et al. TG-1601 is a novel BET inhibitor with strong binding affinity and long-lasting effect in pre-clinical models. American Association for Cancer Research Annual Meeting (AACR). 2018-04-18 Abstract 5790.

ConVax (fka Pentamer)

• Wussow, F. et al (2014). Human Cytomegalovirus Vaccine Based on the Envelope gH/gL Pentamer Complex. PLoS Pathog PLoS Pathogens, 10(11). doi:10.1371/journal.ppat.1004524.
• Chiuppesi, F. et al. Multiantigenic Modified Vaccinia Virus Ankara Vaccine Vectors to Elicit Potent Humoral and Cellular Immune Reponses against Human Cytomegalovirus in Mice. DOI: 10.1128/JVI.01012-18.

CEVA-D

• Diaz, M. F. et al. Biomechanical Forces Promote Immune Regulatory Function of Bone Marrow Mesenchymal Stromal Cells. Stem Cells 35, 1259–1272, (2017).
• Diaz, M.F. et al. Biomechanical Forces Promote Immune Regulatory Function of Bone Marrow Mesenchymal Stromal Cells. Stem Cells 35, 1259-1272, (2017).

Anti-CAIX

• Xu, C. et al Unique biological properties of catalytic domain directed human anti-CAIX antibodies discovered through phage-display technology. PLoS One 5, e9625 (2010).
• Chang, D.-K. et al. Human anti-CAIX antibodies mediate immune cell inhibition of renal cell carcinoma in vitro and in a humanized mouse model in vivo. Mol. Cancer 14, 119 (2015).

Oncogenuity

• Obstruction of BRAFV600E Transcription by Complementary PNA Oligomers As A Means to Inhibit BRAF-mutant Melanoma Growth September 22, 2017