Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a multi-platform biotechnology company that focuses on developing gene therapies for rare and devastating diseases.

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.

Terns Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops small-molecule single-agent and combination therapy candidates for the treatment of non-alcoholic steatohepatitis (NASH) and obesity.

Amylyx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, engages in developing various therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases.

Acumen Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, discovers and develops therapies for the treatment of Alzheimer's disease. The company focuses on advancing a targeted immunotherapy drug candidate ACU193, a humanized monoclonal antibody that is in Phase I clinical-stage to target soluble amyloid-beta oligomers.

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

X4 Pharmaceuticals, Inc., a late-stage clinical biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare 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.

Kiniksa Pharmaceuticals, Ltd., a biopharmaceutical company, focuses on discovering, acquiring, developing, and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical needs worldwide.

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.

Structure Therapeutics Inc., a clinical stage global biopharmaceutical company, develops and delivers novel oral therapeutics to treat a range of chronic diseases with unmet medical needs.

Phathom Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing treatments for gastrointestinal diseases. The company has the rights in the United States, Europe, and Canada to vonoprazan, a potassium-competitive acid blocker (P-CAB) that blocks acid secretion in the stomach.

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.

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.

Avidity Biosciences, Inc., a biopharmaceutical company, engages in the development of oligonucleotide-based therapies. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat a range of serious diseases.

Protagonist Therapeutics, Inc., a biopharmaceutical company, discovers and develops peptide-based therapeutic drugs to address hematology and blood disorders, and inflammatory and immunomodulatory diseases.