Endo International
NASDAQ:ENDPEndo International plc, a specialty pharmaceutical company, manufactures and sells generic and branded pharmaceuticals in the United States and internationally. Its Branded Pharmaceuticals segment provides branded prescription products, including XIAFLEX to treat adult patients with Dupuytren's contracture; SUPPRELIN LA to treat central precocious puberty in children; NASCOBAL nasal spray to treat vitamin B12 deficiency; AVEED to treat hypogonadism; QWO, an injectable treatment for moderate to severe cellulite in the buttocks of adult women; PERCOCET to treat moderate-to-moderately-severe pain; TESTOPEL an implantable pellet indicated for TRT in conditions associated with a deficiency or absence of endogenous testosterone; EDEX to treat erectile dysfunction; LIDODERM a topical patch product containing lidocaine for the relief of pain; and products for the pain management and urology. The company's Sterile Injectables segment manufactures VASOSTRICT, a vasopressin injection; ADRENALIN, a non-selective adrenergic agonist; and APLISOL, which is a sterile aqueous solution, as well as generic sterile injectable products, including ertapenem for injections and ephedrine sulfate injections. Its Generic Pharmaceuticals segment offers solid oral extended-release, solid oral immediate-release, liquids, semi-solids, patches, powders, ophthalmic products, and sprays. The company's International Pharmaceuticals segment offers specialty pharmaceutical products in various therapeutic areas comprising attention deficit hyperactivity disorder, pain, women's health, oncology, and transplantation. The company sells its branded pharmaceuticals and generics to specialty physicians, retailers, clinics, government agencies, doctors, retail and specialty pharmacies, and specialty distributors. Endo International plc was founded in 1920 and is headquartered in Dublin, Ireland.
Generation Bio
NASDAQ:GBIOGeneration Bio Co. develops non-viral genetic medicines for the treatment of rare and prevalent diseases. The company develops cell-targeted lipid nanoparticle (ctLNP) platform, a modular delivery system for nucleic acids to avoid off-target clearance by the liver and spleen that enables ctLNPs to persist in systemic circulation, which allows for highly selective and potent ligand-driven targeting to specific tissues and cell types; and novel immune-quiet DNA (iqDNA) to enable long-lasting high levels of gene expression from non-integrating episomes and avoids innate immune sensors that have long prevented DNA from use in non-viral systems. It uses its platform for developing a portfolio of programs for treating cancer, autoimmune, hematologic disorders, prioritizing sickle cell, beta-thalassemia, and hemophilia A diseases, as well as for other tissues and cell types, including retina, skeletal muscle, and central nervous system. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.
NRx Pharmaceuticals
NASDAQ:NRXPNRx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops novel therapeutics based on N-methyl-D-aspartate platform for the treatment of central nervous system disorders, including suicidal depression, post-traumatic stress disorder, and chronic pain. Its products include NRX-101 (D-cycloserine/Lurasidone), an oral, fixed dosed combination of D-cycloserine and lurasidone that earned FDA-designated investigational breakthrough therapy for suicidal treatment-resistant bipolar depression and chronic pain; and NRX-100 (ketamine), which has been awarded FDA fast track designation for the treatment of acute suicidality in depression. The company has a partnership with Alvogen and Lotus to develop and commercialize NRX-101 for the suicidal bipolar depression treatment, as well as an agreement with Nephron Pharmaceuticals, Inc. to develop ketamine. The company was founded in 2015 and is based in Wilmington, Delaware.
uniQure
NASDAQ:QUREuniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease. In addition, it is developing AMT-162, which is in preclinical trial for the treatment of superoxide dismutase 1- amyotrophic lateral sclerosis; AMT-260 that is in preclinical trial to treat temporal lobe epilepsy; AMT-191, which is in preclinical trial for the treatment of fabry disease; AMT-161 that is in preclinical trial to treat amyotrophic lateral sclerosis caused by mutations; AMT-240, which is in preclinical trial to for the treatment of autosomal dominant Alzheimer's disease; and AMT-210 that is in preclinical trial to treat Parkinson's disease. The company was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
Stoke Therapeutics
NASDAQ:STOKStoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.