Alnylam Pharmaceuticals is a commercial-stage biopharmaceutical company developing therapeutics based on RNA interference (RNAi). RNAi is a biological pathway within cells for sequence-specific silencing and regulation of gene expression. The company has two RNAi-based medicines, ONPATTRO? (patisiran) and GIVLAARI? (givosiran). ONPATTRO is approved by the United States Food and Drug Administration (FDA) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults and has also been approved in the European Union, Japan, Canada and Switzerland. GIVLAARI is approved by FDA for the treatment of adults with acute hepatic porphyria.

Amicus Therapeutics is a biotechnology company focused on the discovering, developing, and delivering medicines for diseases. The company's Commercial Product and Product Candidates include Galafold? (Migalastat HCl), which is for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant based on in vitro assay data; Enzyme Replacement Therapy for Pompe disease; and Cyclin-dependent kinase-like 5 (CDKL5) Deficiency Disorder, which is a gene on the X-chromosome encoding the CDKL5 protein that regulates the expression of several essential proteins for normal brain development.

Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing therapies for people with rare, debilitating, chronic neuromuscular and neurological diseases. The company has licensed the North American rights to Firdapse?, a proprietary form of amifampridine phosphate, or chemically known as 3,4-diaminopyridine phosphate, from BioMarin Pharmaceutical Inc. for the treatment of patients with Lambert-Eaton Myasthenic Syndrome, Congenital Myasthenic Syndromes and Myasthenia Gravis. In addition, the company has launched Firdapse? in the United States, selling through a field force in neurologic, central nervous system or rare disease products consisting of field personnel.

Merck is a global science and technology company based in Germany. Co. diversifies its activities into four business divisions: Merck Serono, Consumer Health, Performance Materials and Merck Millipore. The Merck Serono develops, manufactures and markets prescription drugs to treat cancer, multiple sclerosis, infertility, growth disorders, and selected cardiovascular and metabolic diseases. The Consumer Health manufactures and markets over-the-counter drugs addressing health themes such as mobility, women's and children's health, cough and cold. Performance Materials is a specialty chemicals business. The Merck Millipore is the supplier of products and services for the life science industry.

REGENXBIO is a clinical-stage biotechnology company. The company's product candidates include: RGX-314, which is being developed as a gene therapy for the treatment of wet age-related macular degeneration, diabetic retinopathy, and other additional chronic retinal conditions which cause total or partial vision loss for patients in the U.S., Europe and Japan; RGX-121, which is for the treatment of Mucopolysaccharidosis Type II; RGX-111, which is for the treatment of Mucopolysaccharidosis Type I; and RGX-181, which is for the treatment of late infantile neuronal ceroid lipofuscinosis type II disease; and RGX-501, which is for the treatment of homozygous familial hypercholesterolemia.

Synthetic Biologics is a clinical-stage company utilizing the microbiome to develop therapeutics designed to treat gastrointestinal (GI) diseases. The company's clinical and preclinical programs include: SYN-004 (ribaxamase) (oral enzyme) for prevention of microbiome damage, Clostridioides difficile infection, overgrowth of pathogenic organisms, antimicrobial resistance, and acute graft-versus-host-disease in allogeneic hematopoietic cell transplant recipients; SYN-010 (oral modified-release lovastatin lactone) for treatment of irritable bowel syndrome with constipation; and SYN-020 (oral intestinal alkaline phosphatase enzyme) to treat local GI inflammation, and restore gut microbiome.