Opus Genetics, Inc. Common Stock
Opus Genetics, Inc., a clinical-stage ophthalmic biopharmaceutical company, focuses on developing and commercializing therapies for the treatment of inherited retinal diseases (IRDs). It develops OPGx-LCA5, an early-onset retinal degeneration that is in Phase 1/2 clinical trial for the treatment of LCA5-associated IRD; and OPGx-BEST1 to treat BEST1-associated retinal disease. The company also develops various pre-clinical IRD programs, including OPGx-RHO, a gene therapy that targets autosomal dominant retinitis pigmentosa caused by RHO mutations; OPGx-RDH12 that is designed to restore protein expression and halt functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase gene; OPGx-MERTK, an AAV vector for the treatment of MERTK IRD; OPGx-NMNAT1, a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 gene; and OPGx-CNGB1, an AAV gene therapy for retinitis pigmentosa due to mutations in the CNGB1 gene. In addition, it develops Phentolamine Ophthalmic Solution for reversal of mydriasis, as well as for the treatment of presbyopia and dim light or night vision disturbances; and APX3330, a small-molecule inhibitor of Ref-1 for the treatment of diabetic retinopathy. The company was formerly known as Ocuphire Pharma, Inc. Opus Genetics, Inc. was founded in 2018 and is headquartered in Durham, North Carolina.
Open IRD dashboard ↗Karyopharm Therapeutics Inc.
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. In addition, 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, the company 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.
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