Apellis Pharmaceuticals
NASDAQ:APLSApellis Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases. It offers EMPAVELI for the treatment of paroxysmal nocturnal hemoglobinuria, C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, and hematopoietic stem cell transplantation-associated thrombotic microangiopathy; and SYFOVRE for treating geographic atrophy secondary to age-related macular degeneration and geographic atrophy (GA). The company also develops APL-3007, a small interfering RNA, or siRNA, which is in a Phase 1 clinical trial, as well as an oral complement inhibitor that is in preclinical development. It has a collaboration and license agreement with Swedish Orphan Biovitrum AB (publ) for development and commercialization of pegcetacoplan; and a collaboration with Beam Therapeutics Inc. focused on the use of Beam's base editing technology to discover new treatments for complement-driven diseases. The company was incorporated in 2009 and is based in Waltham, Massachusetts.
Arvinas
NASDAQ:ARVNArvinas, Inc., a clinical-stage biotechnology company, engages in the discovery, development, and commercialization of therapies to degrade disease-causing proteins. The company engineers proteolysis targeting chimeras (PROTAC) targeted protein degraders that are designed to harness the body's own natural protein disposal system to degrade and remove disease-causing proteins. Its product pipeline includes Bavdegalutamide and ARV-766, investigational orally bioavailable PROTAC protein degraders for the treatment of men with metastatic castration-resistant prostate cancer, which are in Phase 1/2 clinical trials; and ARV-471, an orally bioavailable estrogen receptor degrading PROTAC targeted protein degrader for the treatment of patients with locally advanced or metastatic estrogen receptor+/human epidermal growth factor receptor 2-breast cancer, which is Phase 3 clinical trial. Arvinas, Inc. has collaborations with Pfizer Inc., Genentech, Inc., F. Hoffman-La Roche Ltd., Carrick Therapeutics Limited, and Bayer AG. The company was founded in 2013 and is based in New Haven, Connecticut.
CureVac
NASDAQ:CVACCureVac N.V., a clinical-stage biopharmaceutical company, focuses on developing various transformative medicines based on messenger ribonucleic acid (mRNA). It is developing prophylactic vaccines, such as mRNA-based vaccine candidates CV2CoV, which is in Phase 1 clinical trial against SARS-CoV-2; CV7202, a prophylactic mRNA-based vaccine, which has completed Phase 1 clinical trial for rabies virus glycoprotein; and CVSQIV, which is in Phase 1 clinical trial against influenza, as well as vaccine for lassa fever, yellow fever, respiratory syncytial virus, rotavirus, malaria, and universal influenza. The company also develops RNA-based cancer therapies, including CV8102, which is in Phase 1 clinical trial for treating cutaneous melanoma and adenoidcystic carcinoma, as well as squamous cell cancer of skin, head, and neck. CureVac N.V. was founded in 2000 and is headquartered in Tübingen, Germany.
Dicerna Pharmaceuticals
NASDAQ:DRNADicerna Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to selectively silence genes that cause or contribute to disease. The firm uses its proprietary GalXC RNAi technology platform to build a pipeline in these therapeutic areas. The company was founded by Douglas M. Fambrough III, Mark A. Behlke, Roberto Guerciolini, James Craig Jenson, and John J. Rossi in October 2006 and is headquartered in Lexington, MA.
Karuna Therapeutics
NASDAQ:KRTXKaruna Therapeutics, Inc., a clinical-stage biopharmaceutical company, creates and delivers transformative medicines for people living with psychiatric and neurological conditions. Its lead product candidate includes KarXT, an oral modulator of muscarinic receptors for the treatment of acute psychosis in patients with schizophrenia; and for the treatment of central nervous system disorders, such as negative and cognitive symptoms of schizophrenia and psychosis, as well as for the treatment of various peripheral tissues and dementia-related psychosis, including Alzheimer's disease. The company also focuses on developing other muscarinic-targeted drug candidates; and intends to develop lead candidature TRPC4/5 and KAR-2618 for the treatment of depressive disorder. Karuna Therapeutics, Inc. has a license agreement with Eli Lilly and Company, and Zai Lab (Shanghai) Co., Ltd; patent license agreement with PureTech Health LLC; and drug discovery partnership with Charles River Laboratories, as well as drug discovery collaboration with PsychoGenics, Inc. The company was formerly known as Karuna Pharmaceuticals, Inc. and changed its name to Karuna Therapeutics, Inc. in March 2019. Karuna Therapeutics, Inc. was incorporated in 2009 and is headquartered in Boston, Massachusetts.