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Avenue Therapeutics, Inc.

IV Tramadol

Scott, L. et al. Tramadol: A Review of its Use in Perioperative Pain. Adis International Limited, Drugs 2000 Jul; 60 (1): 139-176.

Caelum Biosciences, Inc.

Edward, 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.

Checkpoint Therapeutics, Inc.


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).

Cellvation, Inc.

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.

Escala Therapeutics, Inc.


Argov Z, Mitrani-Rosenbaum S. The hereditary inclusion body myopathy enigma and its future therapy. Neurotherapeutics 2008; 5(4): 633-7.

Celeste FV, Vilboux T, et al. Mutation update for GNE gene variants associated with GNE myopathy. Hum Mutat 2014; 35(8): 915-26.

Eisenberg I, Avidan N, et al. The UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase gene is mutated in recessive hereditary inclusion body myopathy. Nat Genet 2001; 29(1): 83-7.

Galeano B, Klootwijk R, et al. Mutation in the key enzyme of sialic acid biosynthesis causes severe glomerular proteinuria and is rescued by N-acetylmannosamine. J Clin Invest 2007; 117(6): 1585-94.

Hinderlich S, Berger M, et al. Biosynthesis of N-acetylneuraminic acid in cells lacking UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase. Biol Chem 2001; 382(2): 291-7.

Huizing M, Carrillo-Carrasco N, et al. GNE myopathy: new name and new mutation nomenclature. Neuromuscul Disord 2014; 24(5): 387-9.

Huizing M, Krasnewich DM, Manoli I, Carrillo-Carrasco N. GNE Myopathy. In: Valle D BA, Vogelstein B, Kinzler KW, Antonarakis SE, Ballabio A, Gibson K, Mitchell G, editor. OMMBID – The Online Metabolic and Molecular Bases of Inherited Diseases. New York, NY: McGraw-Hill; 2013.

Huizing M, Krasnewich DM. Hereditary inclusion body myopathy: a decade of progress. Biochim Biophys Acta 2009; 1792(9): 881-7.

Malicdan MC, Noguchi S, et al. Prophylactic treatment with sialic acid metabolites precludes the development of the myopathic phenotype in the DMRV-hIBM mouse model. Nat Med 2009; 15(6): 690-5.

Malicdan MC, Noguchi S, et al. Peracetylated N-acetylmannosamine, a synthetic sugar molecule, efficiently rescues muscle phenotype and biochemical defects in mouse model of sialic acid-deficient myopathy. J Biol Chem 2012; 287(4): 2689-705.

Niethamer TK, Yardeni T, et al. Oral monosaccharide therapies to reverse renal and muscle hyposialylation in a mouse model of GNE myopathy. Mol Genet Metab 2012; 107(4): 748-55.

Nishino I, Carrillo-Carrasco N, et al. GNE myopathy: current update and future therapy. J Neurol Neurosurg Psychiatry 2015; 86(4): 385-92.

Sparks S, Rakocevic G, et al. Intravenous immune globulin in hereditary inclusion body myopathy: a pilot study. BMC Neurol 2007; 7: 3.

Varki A. Sialic acids in human health and disease. Trends Mol Med 2008; 14(8): 351-360.

Fortress Biotech, Inc.


CNDO-109-Activated Allogeneic Natural Killer Cells in Patients with High Risk Acute Myeloid Leukemia in First Complete Remission (CR1): A Phase 1 Study. Innate Killer Summit 2016, May 17, 2016. (Oral Presentation).

Helocyte, Inc.


Nakamura, R., et al (2016). Viraemia, immunogenicity, and survival outcomes of cytomegalovirus chimeric epitope vaccine supplemented with PF03512676 (CMVPepVax) in allogeneic haemopoietic stem-cell transplantation: Randomised phase 1b trial. The Lancet Haematology, 3(2). doi:10.1016/s2352-3026(15)00246-x.


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]. In: 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.


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.

Mustang Bio, Inc.

MB-101 (IL13Rα2-specific CAR)

2016 Abstract Presentations

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.

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.

Reference Articles

Brown, C. et al. Regression of Gliobastoma after Chimeric Antigen Receptor T-Cell Therapy. New England Journal of Medicine December 2016; 375: 2561-2569.

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.

MB-102 (CD123 CAR)

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.