Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.

Pharvaris N.V., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapies for rare diseases. The company develops PHA121, a small molecule bradykinin B2-receptor antagonist for the treatment of hereditary angioedema (HAE). It also develops PHVS416, an on-demand, rapid exposure soft capsule for patients suffering from acute HAE attacks which is under Phase 2 clinical trial; and PHVS719, a prophylactic extended-release tablet for HAE patients which is under Phase 1 clinical trial. The company operates in the Netherlands, Switzerland, and the United States. Pharvaris N.V. was incorporated in 2015 and is based in Leiden, the Netherlands.

Pharming Group N.V., a biopharmaceutical company, develops and commercializes protein replacement therapies and precision medicines for the treatment of rare diseases in the United States, Europe, and internationally. The company offers RUCONEST, a recombinant C1 esterase inhibitor for the treatment of acute attacks in adult and adolescent patients with acute hereditary angioedema (HAE); and Joenja (leniolisib), an oral small molecule PI3K? inhibitor for the treatment of activated phosphoinositide 3-kinase delta syndrome. It also develops OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of HAE. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105. Pharming Group N.V. was incorporated in 1988 and is headquartered in Leiden, the Netherlands.

Neurogene Inc., a biotechnology company, develops genetic medicines for rare neurological diseases. The company's product candidates include NGN-401 which is packaged in an adeno-associated virus 9 that is in Phase 1/2 clinical trial for the treatment of Rett syndrome; and NGN-101, a conventional gene therapy candidate that is in Phase 1/2 clinical trial to treat CLN5 Batten disease. It has a license agreement with The University of North Carolina, the University of Edinburgh, Virovek, Inc., and Sigma-Aldrich Co. LLC. The company is headquartered in New York, New York.

ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.

Voyager Therapeutics, Inc., a biotechnology company, focuses on the treatment of gene therapy and neurology diseases. The company's lead clinical candidate is VY-TAU01, an anti-tau antibody program for the treatment of alzheimer's disease. Its product pipeline includes superoxide dismutase 1 silencing gene therapy, which is in preclinical trial for the treatment of amyotrophic lateral sclerosis; tau silencing gene therapy, which is in preclinical trial for the treatment of alzheimer's disease; and vectorized anti-amyloid antibody, a gene therapy targeting anti-amyloid for the treatment of alzheimer's disease and is in preclinical trial. In addition, the company develops VY-FXN01, which is in preclinical trial to treat friedreich's ataxia; and GBA1 gene replacement to treat parkinson's disease and is in preclinical trial. Further, it provides research program for the treatment of Huntington's disease. The company has collaboration and license agreements with Alexion; AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc; and Sangamo Therapeutics, Inc. Voyager Therapeutics, Inc. was incorporated in 2013 and is headquartered in Lexington, Massachusetts.

NeuBase Therapeutics, Inc., a preclinical-stage biopharmaceutical company, focuses on the development of therapies to treat rare genetic diseases and cancers caused by mutant genes. The company's proprietary modular peptide-nucleic acid antisense oligo (PATrOL) platform focuses on addressing Huntington's disease (HD) and myotonic dystrophy, as well as other genetic disorders, and cancer and oncology applications. Its pipeline includes NT0100 for Huntington's disease; NT0200 for myotonic dystrophy type 1; and NT0300 treatment targets the mutated KRAS gene. NeuBase Therapeutics, Inc. is headquartered in Pittsburgh, Pennsylvania.

AVROBIO, Inc., a clinical-stage gene therapy company, develops hematopoietic stem cell gene therapies to treat rare diseases in the United States. Its gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The company's pipeline includes AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and 3; AVR-RD-01, which is in phase 2 clinical trials for the treatment of Fabry disease; and AVR-RD-03 that is in preclinical stage for the treatment of Pompe disease. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.