Rhythm Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the development and commercialization of therapeutics for the treatment of rare genetic diseases of obesity.

Solid Biosciences Inc. engages in developing therapies for duchenne muscular dystrophy in the United States. The company's lead product candidate is SGT-001, a gene transfer candidate, which is in a Phase I/II clinical trial to drive functional dystrophin protein expression in patients' muscles; and SGT-003, a ext-generation gene transfer candidate for the treatment of duchenne muscular dystrophy.

MeiraGTx Holdings plc, a clinical stage gene therapy company, focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited blindness, as well as Xerostomia following radiation treatment for head and neck cancers; degenerative diseases; neurodegenerative diseases, such as amyotrophic lateral sclerosis; and Parkinson's diseases.

Vaxcyte, Inc., a clinical-stage biotechnology vaccine company, develops novel protein vaccines to prevent or treat bacterial infectious diseases. Its lead vaccine candidate is VAX-24, a 24-valent investigational pneumococcal conjugate vaccine that is in Phase 1/2 clinical trials to treat invasive pneumococcal disease and pneumonia.

Larimar Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing treatments for rare diseases using its novel cell penetrating peptide technology platform.

Syndax Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapies for the treatment of cancer. Its lead product candidates are SNDX-5613, which is in phase 1/2 clinical trial targeting the binding interaction of Menin with the mixed lineage leukemia 1 protein for the treatment of MLL-rearranged (MLLr) and nucleophosmin 1 mutant acute myeloid leukemia (NPM1c AML); and SNDX-6352 or axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1, or CSF-1 receptor for the treatment of patients with chronic graft versus host disease (cGVHD).

Merus N.V., a clinical-stage immuno-oncology company, engages in the discovery and development of bispecific antibody therapeutics in the Netherlands. Its bispecific antibody candidate pipeline includes Zenocutuzumab (MCLA-128), which is in a phase 2 clinical trials for the treatment of patients with metastatic breast cancer, as well as in Phase 1/2 clinical trials for the treatment of solid tumors that harbor Neuregulin 1.

Sutro Biopharma, Inc. operates as clinical stage drug discovery, development, and manufacturing company. It focuses on creating protein therapeutics for cancer and autoimmune disorders through integrated cell-free protein synthesis and site-specific conjugation platform, XpressCF+.The company's product candidates include STRO-001, an antibody-drug conjugate (ADC) directed against the cancer target CD74 for patients with multiple myeloma and non-Hodgkin lymphoma that is in Phase 1 clinical trials; and STRO-002, an ADC directed against folate receptor-alpha for patients with ovarian and endometrial cancers, which is in Phase 1 clinical trials.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops novel antisense oligonucleotide (ASO) medicines to treat the underlying causes of severe genetic diseases in the United States.

Pliant Therapeutics, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes novel therapies for the treatment of fibrosis and related diseases in the United States.

Dyne Therapeutics, Inc., a muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States.

Cytokinetics, Incorporated, a late-stage biopharmaceutical company, focuses on discovering, developing, and commercializing muscle activators and inhibitors as potential treatments for debilitating diseases.

Revolution Medicines, Inc., a clinical-stage precision oncology company, focuses on developing therapies to inhibit frontier targets in RAS-addicted cancers. The company is developing RMC-4630, an inhibitor of SHP2, which is in Phase 1/2 clinical trial for the treatment of solid tumors, such as gynecologic and colorectal cancer tumors.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins or antibodies that are intended to impact disease.

Day One Biopharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops and commercializes targeted therapies for patients with genetically defined cancers. Its lead product candidate is DAY101, an oral brain-penetrant type II pan-rapidly accelerated fibrosarcoma kinase inhibitor that is in Phase II clinical trial for pediatric patients with relapsed/progressive low-grade glioma.

Replimune Group, Inc., a biotechnology company, develops oncolytic immuno-gene therapies to treat cancer. It uses its proprietary Immunotherapy platform to design and develop product candidates that are intended to activate the immune system against cancer.

Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases.

Viridian Therapeutics, Inc., a biotechnology company, develops treatments for patients suffering from serious diseases. It develops VRDN-001, a humanized monoclonal anti-IGF-1R antibody that is in Phase 1/2 clinical trial for the treatment of thyroid eye disease (TED); VRDN-002, an IGF-1R antibody, which is in Phase 1 clinical trial; and VRDN-003, a therapeutic antibody targeting IGF-1R for the treatment of TED.

Inozyme Pharma, Inc., a rare disease biopharmaceutical company, engages in developing therapeutics for treating abnormal mineralization impacting the vasculature, soft tissue, and skeleton diseases.