Pharming Group N.V. is a biopharmaceutical company dedicated to the creation and commercialization of protein replacement therapies and precision medicines. The firm concentrates its efforts on addressing rare diseases and critical unmet medical needs across the United States, Europe, and other international markets. Its primary commercialized product is Ruconest, a recombinant human C1 esterase inhibitor utilized for the acute treatment of hereditary angioedema. Pharming's development pipeline also includes rhC1INH, which is being investigated for conditions such as pre-eclampsia, acute kidney injury, and COVID-19. Additionally, the company is developing leniolisib, a phosphoinositide 3-kinase delta (PI3K delta) inhibitor designed for patients with activated PI3K delta syndrome, as well as an alpha-glucosidase therapy targeting Pompe and Fabry diseases. The company has established significant collaborations, including a development and licensing agreement with Novartis. It also maintains a strategic partnership with Orchard Therapeutics plc for the research, development, manufacturing, and commercialization of OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy intended for hereditary angioedema. Pharming Group N.V. is headquartered in Leiden, the Netherlands.
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Pharming Group N.V. is a pharmaceuticals company headquartered in Leiden, Netherlands. It is publicly listed (PHAR) with a market capitalisation of $834.5M. Xcout tracks 13 named people at Pharming Group N.V., including Fabrice Chouraqui (Chief Executive Officer & Executive Director).
7 more named people on record for Pharming Group N.V., with roles, board committees and tenure — see the full roster →
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Pharvaris N.V., a late-stage biopharmaceutical company, focuses on the development and commercialization of therapies for rare diseases with unmet needs covering angioedema and other bradykinin-mediated diseases. The company develops deucrictibant, a small molecule bradykinin B2-receptor antagonist to treat attacks due to bradykinin-mediated angioedema, including hereditary angioedema (HAE) and acquired angioedema due to C1-inhibitor deficiency (AAE-C1INH), which is in phase 3, as well as in phase 3 trials for treatment and prophylaxis of HAE attacks; and extended-release tablet and immediate-release capsule formulation of deucrictibant. Pharvaris N.V. was incorporated in 2015 and is headquartered in Zug, Switzerland.
NewAmsterdam Pharma Company N.V. is a biopharmaceutical firm currently in the clinical development phase, committed to enhancing therapeutic options for individuals living with metabolic diseases. The company's primary experimental treatment, obicetrapib, is an innovative, selective agent designed to inhibit Cholesteryl Ester Transfer Protein (CETP). Clinical trials have shown this compound to be effective in both lowering "bad" low-density lipoprotein cholesterol (LDL-C) and significantly elevating "good" high-density lipoprotein cholesterol (HDL-C) simultaneously. Founded in 2019, this Dutch company maintains its headquarters in Naarden.
Chiesi Farmaceutici is a global biopharmaceutical group that researches, develops, and commercializes therapeutic solutions for patients. The company focuses primarily on respiratory health, rare diseases, and specialty care.
Alnylam Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the exploration, advancement, and marketing of innovative therapies rooted in RNA interference (RNAi) technology. The company's research pipeline for RNAi treatments targets a diverse array of medical conditions, encompassing inherited disorders, cardiovascular and metabolic diseases, hepatic infections, and illnesses affecting the central nervous system (CNS) and eyes. Alnylam's commercialized products include ONPATTRO (patisiran), an injectable lipid complex prescribed for adults experiencing polyneuropathy due to hereditary transthyretin-mediated amyloidosis. Also marketed are GIVLAARI, used to treat acute hepatic porphyria (AHP) in adult patients, and OXLUMO (lumasiran), indicated for primary hyperoxaluria type 1 (PH1). In terms of its developmental programs, the company is progressing several candidates: Givosiran, for the treatment of AHP in adolescent patients. Patisiran, being investigated for transthyretin amyloidosis (ATTR amyloidosis) when accompanied by cardiomyopathy. Cemdisiran, aimed at complement-mediated diseases. ALN-AAT02, for liver disease associated with AAT deficiency. ALN-HBV02, targeting chronic hepatitis B virus infection. Zilebesiran, for the management of hypertension. ALN-HSD, for non-alcoholic steatohepatitis (NASH). Fitusiran, addressing hemophilia and various rare bleeding disorders. Inclisiran, developed to treat hypercholesterolemia. Lumasiran, for advanced PH1 and recurrent kidney stones. Vutrisiran, currently in Phase 3 clinical trials for ATTR amyloidosis. Alnylam Pharmaceuticals maintains strategic alliances with several key players in the industry. It collaborates with Regeneron Pharmaceuticals, Inc. on the discovery, development, and commercialization of RNAi therapeutics for ocular and CNS targets. Another significant partnership exists with Sanofi Genzyme, focusing on the broader development and commercialization of RNAi treatments. Furthermore, the company holds licensing and collaboration agreements with Novartis AG, Vir Biotechnology, Inc., Dicerna Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., and PeptiDream, Inc. The company was established in 2002 and operates from its headquarters in Cambridge, Massachusetts.
Curatis Holding AG specializes in the entire lifecycle of pharmaceutical products, including the procurement, advancement, and market release of medications specifically designed for the diagnosis, prevention, and management of rare and specialized medical conditions. The company's extensive product offerings span a broad array of therapeutic areas. These encompass treatments for antiplatelet conditions, metastatic hormone receptor-positive breast cancer, anti-arrhythmic disorders, nasal oil applications, Wilson's disease, antidotes for cyanide poisoning and digoxin toxicity, antihypotensive agents, therapies for bladder pain syndrome/interstitial cystitis, antibiotics, diagnostic tools for ophthalmology, antivirals, ophthalmic/antibiotic combinations, gentamycin collagen sponges, solutions for benign prostatic hyperplasia, immunomodulators, treatments for epilepsy and essential tremor, thyreostatics, therapies for hereditary tyrosinemia type 1, gastroenterological products, additional immunomodulators and thyreostatics, tranquilizers, Helicobacter pylori tests, phosphate binders, specialized nutritional support for dialysis patients, dermatological corticosteroids, antidotes for lead and mercury poisoning, corticostimulants, anti-fog agents, treatments for chorea associated with Huntington's disease, and antiepileptics. In addition to its commercialized portfolio, Curatis is actively engaged in developing new pharmaceutical compounds. Its pipeline includes C-PTBE-01, currently in Phase IIb clinical trials for treating peritumoral brain edema in pediatric patients; C-AM-01, undergoing Phase IIa trials for preventing severe migraine with aura; and C-MOH-01, also in Phase IIa for the treatment and prevention of medication overuse headache. Furthermore, KIN001 is in Phase I clinical trials, targeting rare inflammatory and fibrotic diseases. The company maintains its headquarters in Liestal, Switzerland.
Omeros Corporation is a biopharmaceutical firm with commercialized products, actively engaged in the discovery, development, and market introduction of both small-molecule and protein-based therapies, including treatments for rare diseases. The company's therapeutic endeavors primarily focus on inflammatory conditions, illnesses mediated by the complement system, cancers linked to immune system dysregulation, and disorders characterized by addiction or compulsion. Its robust clinical pipeline features several promising candidates. Narsoplimab (also known as OMS721 or MASP-2), a leading asset, has successfully concluded pivotal trials for hematopoietic stem-cell transplant-associated thrombotic microangiopathy (HSCT-TMA). This drug is also currently undergoing Phase III clinical evaluation for immunoglobulin A nephropathy (IgAN) and atypical hemolytic uremic syndrome (aHUS), in addition to a Phase II trial investigating its potential for treating COVID-19. Further expanding its clinical portfolio, Omeros is developing PPAR? (OMS405) in Phase II for opioid and nicotine dependence. PDE7 (OMS527) is progressing through Phase I studies for addiction, compulsive disorders, and movement disorders. Additionally, MASP-3 (OMS906) is currently in Phase I trials for paroxysmal nocturnal hemoglobinuria (PNH) and other conditions involving the alternative complement pathway. Beyond its clinical efforts, the company maintains a dynamic preclinical program. This includes small-molecule inhibitors targeting MASP-2, which are being explored for applications in aHUS, IgAN, HSCT-TMA, and age-related macular degeneration. Also in development is a next-generation, longer-acting antibody that targets MASP-2, alongside MASP-3 small-molecule inhibitors designed to address PNH and other alternative pathway disorders. Omeros's preclinical research further encompasses GPR174 inhibitors, as well as Chimeric Antigen Receptor (CAR) T-Cell and Adoptive T-Cell Therapies for various oncological indications. The company is also investigating G protein-coupled receptor targets for a broad spectrum of diseases, including immunological, immuno-oncological, metabolic, central nervous system (CNS), cardiovascular, musculoskeletal, and other conditions. Established in 1994, Omeros Corporation is headquartered in Seattle, Washington.
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