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Karyopharm Therapeutics Inc., a commercial-stage pharmaceutical company, discovers, develops, and commercializes drugs directed against nuclear export for the treatment of cancer and other diseases in the United States. The company discovers, develops, and commercializes novel and small molecule Selective Inhibitor of Nuclear Export (SINE) compounds that inhibit the nuclear export protein exportin 1 (XPO1). Its lead compound, include XPOVIO in combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma; in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma; and for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). It also developing Selinexor for treating hematological and solid tumor malignancies, including multiple myeloma, endometrial cancer, myelofibrosis, and DLBCL; and ELTANEXOR for treating Myelodysplastic Neoplasms, as well as verdinexor, KPT-9274, and IL-12 compounds. The company has license agreement with Menarini Group to develop and commercialize selinexor for human oncology indications; license agreement with Antengene Therapeutics Limited to develop and commercialize selinexor, eltanexor, and KPT-9274 for the treatment and/or prevention of human oncology indications, as well as verdinexor for the diagnosis, treatment, and/or prevention of human non-oncology indications; and distribution agreement for the commercialization of XPOVIO with FORUS Therapeutics Inc. Further, it has a collaboration with Bristol Myers Squibb company to evaluate novel cereblon E3 ligase modulator agent mezigdomide in combination with Selinexor in patients with relapsed/refractory multiple myeloma. Karyopharm Therapeutics Inc. was incorporated in 2008 and is headquartered in Newton, Massachusetts.

Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.

Stemline Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, acquisition, development, and commercialization of oncology therapeutics in the United States and internationally. The company offers ELZONRIS, a targeted therapy directed to the interleukin-3 receptor (CD123) for patients with blastic plasmacytoid dendritic cell neoplasm in adults, and in pediatric patients two years and older. It also developing CD123 that is in Phase I/II clinical trials for the treatment of chronic myelomonocytic leukemia and myelofibrosis, as well as acute myeloid leukemia and multiple myeloma in combination with other therapies. In addition, the company is developing SL-801, a novel oral small molecule reversible inhibitor of nuclear transport protein, which is in Phase I clinical trial for the treatment of solid and hematologic cancers; and SL-701, an immunotherapy that has completed Phase II clinical trials to treat brain cancer. Its preclinical pipeline products include SL-501, a CD123 targeted therapy; SL- 1001, an oral, selective small molecule RET kinase inhibitor targeting multiple oncology indications; and SL-901, a small molecule kinase inhibitor. Stemline Therapeutics, Inc. was founded in 2003 and is based in New York, New York.

Synthorx, Inc., a biopharmaceutical company, focuses on the development of cytokine Synthorin programs for the treatment of cancer and autoimmune disorders in the United States. The company's lead product candidate is THOR-707, a variant of recombinant human IL-2 that is in the development in various solid tumor types as a single agent and in combination with an immune checkpoint inhibitor. It also develops IL-2 Synthorin for autoimmune indications; IL-10 Synthorin, a naturally occurring immune cell growth factor in humans for the treatment of immuno-oncology (IO); and IL-15 Synthorins, an immunoregulatory cytokine to treat IO. In addition, the company develops other Synthorin programs targeting undisclosed cytokines that play critical roles in the orchestration of anti-tumor responses by innate and adaptive immune cells. The company was formerly known as Alinos, Inc. and changed its name to Synthorx, Inc. in March 2014. Synthorx, Inc. was founded in 2014 and is headquartered in La Jolla, California.