Cabaletta Bio
NASDAQ:CABACabaletta Bio, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases. The company's lead product candidate is CABA-201, a fully human anti-CD19 binder for the treatment of Phase 1/2 clinical trials in dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, lupus nephritis, non-renal systemic lupus erythematosus, systemic sclerosis, and generalized myasthenia gravis. It also develops DSG3-CAART, which is in Phase I/II clinical trial for the treatment of mucosal pemphigus vulgaris; and MuSK-CAART, an investigational cell therapy that is in Phase I/II clinical trial for treating patients with anti- muscle-specific kinase antibody positive myasthenia gravis. It has a collaboration with the University of Pennsylvania and the Children's Hospital of Philadelphia; Nanjing IASO Biotherapeutics Co., Ltd; Oxford Biomedica; and WuXi Advanced Therapies, Inc. The company was formerly known as Tycho Therapeutics, Inc. and changed its name to Cabaletta Bio, Inc. in August 2018. Cabaletta Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.
LogicBio Therapeutics
NASDAQ:LOGCLogicBio Therapeutics, Inc., a genetic medicine company, focuses on developing and commercializing genome editing and gene therapy treatments using its GeneRide and sAAVy platforms. The company's GeneRide technology is a new approach to precise gene insertion harnessing a cell's natural deoxyribonucleic acid; and gene delivery platform, sAAVy is an adeno-associated virus, which is designed to optimize gene delivery for treatments in a range of indications and tissues. Its lead product candidate is LB-001 that is in Phase I/II clinical trials for the treatment of methylmalonic acidemia. The company has a collaboration with Children's Medical Research Institute to develop next-generation capsids for gene therapy and gene editing applications in the liver, as well as additional tissues; and a collaboration agreement with Takeda Pharmaceutical Company Limited to develop LB-301, an investigational therapy for the treatment of Crigler-Najjar syndrome. The company also has a research collaboration, license, and option agreement with CANbridge Care Pharma Hong Kong Limited; and collaboration agreement with Daiichi Sankyo Company. The company was incorporated in 2014 and is headquartered in Lexington, Massachusetts.
Solid Biosciences
NASDAQ:SLDBSolid Biosciences Inc., a life science company, develops therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, a gene transfer candidate for the treatment of Duchenne muscular dystrophy; and SGT-501 to treat Catecholaminergic polymorphic ventricular tachycardia. It is also developing AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy; SGT-601 to treat a rare cardiac disease characterized by mutations in the gene that codes for cardiac troponin T protein, which helps coordinate contraction of the heart muscle; SGT-701 for treatment of rare inherited disease characterized by mutations in the RBM20 gene, a cardiac splicing factor that regulates alternative splicing, and codes for RNA binding motif protein 20; and AVB-202-TT to treat Friedreich's ataxia, as well as other drugs for the treatment of cardiac and other diseases. In addition, the company is involved in developing platform technologies, including capsid libraries and dual gene expression, a technology for packaging multiple transgenes into one capsid. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize new gene therapies for Duchenne; and a license agreement with the University of Washington, University of Missouri, and University of Florida. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.
Scholar Rock
NASDAQ:SRRKScholar Rock Holding Corporation, a biopharmaceutical company, focuses on the discovery, development, and delivery of medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. The company develops Apitegromab, an inhibitor of the activation of myostatin that is in Phase 3 clinical trial for the treatment of spinal muscular atrophy; and SRK-181, which has completed Phase 1 clinical trials for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies. It is developing a pipeline of product candidates to deliver novel therapies to treat a range of serious diseases, including neuromuscular disorders, cardiometabolic disorders, cancer, fibrosis, and iron-restricted anemia. Scholar Rock Holding Corporation was founded in 2012 and is headquartered in Cambridge, Massachusetts.
Sutro Biopharma
NASDAQ:STROSutro Biopharma, Inc. operates as a clinical-stage oncology company. The company develops site-specific and novel-format antibody drug conjugates (ADCs) that enables its proprietary integrated cell-free protein synthesis platform, XpressCF and XpressCF+. Its product candidates include STRO-002, an ADC directed against folate receptor-alpha, which is in Phase II/III clinical trials for patients with ovarian and endometrial cancers; VAX-24 and Vax-31 pneumococcal conjugate vaccine candidates that is in Phase II/III clinical trials for the treatment of invasive pneumococcal disease; and MK-1484, a distinct cytokine derivative molecule that is in Phase I clinical study for the treatment of cancer. The company's pre-clinical product candidates include STRO-003, an ADC directed against an anti-receptor tyrosine kinase-like orphan receptor 1 (ROR1) for the treatment of solid tumors and hematological cancers; and STRO-004, a tissue factor (TF) targeting ADC for the treatment of TF-expressing solid tumors, including cervical, lung, and breast cancer. Sutro Biopharma, Inc. has collaboration and license agreements with Merck Sharp & Dohme Corporation to develop research programs focusing on cytokine derivatives for cancer and autoimmune disorders; Vaxcyte to discover and develop vaccine candidates for the treatment or prophylaxis of infectious diseases; Tasly Biopharmaceuticals Co., Ltd. to develop and commercialize STRO-002 in Greater China; EMD Serono to develop ADCs for multiple cancer targets; and Astellas Pharma Inc. to develop immunostimulatory ADC. The company was formerly known as Fundamental Applied Biology, Inc. Sutro Biopharma, Inc. was incorporated in 2003 and is headquartered in South San Francisco, California.