PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders. Its portfolio pipeline includes commercial products and product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focuses on the development of treatments for multiple therapeutic areas, such as rare diseases.

Krystal Biotech, Inc., a clinical stage biotechnology company, engages in the field of redosable gene therapy to treat serious rare diseases in the United States.

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases.

Madrigal Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapeutic candidates for the treatment of cardiovascular, metabolic, and liver diseases.

CureVac 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, respiratiory syncytial virus, rotavirus, malaria, and universal influenza.

Apellis 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.

Ocean Biomedical, Inc., a biopharmaceutical company, focuses on discovering and developing therapeutic products in oncology, fibrosis, infectious diseases, and inflammation. The company is developing mono-specific and bi-specific humanized monoclonal antibodies (mAb) product candidates targeting Chi3l1 for the treatment of non-small cell lung cancer and glioblastoma multiforme; and a small molecule product candidate targeting Chit1 for the treatment of Idiopathic Pulmonary Fibrosis and Hermansky-Pudlak Syndrome.

Hepion Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development of drug therapy treatment for chronic liver diseases in the United States.