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INSM vs IRD

Insmed, Inc. and Opus Genetics, Inc. Common Stock

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Insmed, Inc.

Insmed Incorporated develops and commercializes therapies for patients with serious and rare diseases in the United States, Europe, Japan, and internationally. The company offers ARIKAYCE for the treatment of refractory nontuberculous mycobacterial lung infections, as well as is in phase 3 clinical trial for the treatment of mycobacterium avium complex lung disease as part of a combination antibacterial drug regimen for adult patients. It also devlops brensocatib, an oral reversible inhibitor of dipeptidyl peptidase 1(DPP1) that is in phase 3 clinical trial for the treatment of bronchiectasis; and in phase 2 clinical trial for the treatment of chronic rhinosinusitis without nasal polyps and hidradenitis suppurativa. In addition, the company is developing treprostinil palmitil inhalation powder, an inhaled formulation of a treprostinil prodrug treprostinil palmitil, which is in phase 3 clinical trial for the treatment of pulmonary hypertension associated with interstitial lung disease; and phase 2 clinical trial for the treatment of pulmonary arterial hypertension. Further, it develops gene therapy, a microdystrophin adeno-associated virus gene replacement therapy which is in phase 1 clinical trial for the treatment of Duchenne muscular dystrophy, as well as is in pre-clinical development for gene therapy, next generation DPP1 inhibitor, deimmunized therapeutic protein, and synthetic rescue. The company was founded in 1988 and is headquartered in Bridgewater, New Jersey.

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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.

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