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.

Adagene Inc., a clinical stage biopharmaceutical company, engages in the research, development, and production of monoclonal antibody drugs for cancers. The company's product candidates include ADG106, a human ligand-blocking agonistic anti-CD137 monoclonal antibodies (mAbs) that is in Phase 1b/2 clinical trials for the treatment advanced solid tumors and non-Hodgkin's lymphoma; ADG126, a fully-human anti-CTLA-4 mAb that is in Phase 1 clinical trial for the treatment advanced/metastatic solid tumors; and ADG116, a human ligand-blocking anti-CTLA-4 mAb, which is in Phase I clinical trial for the treatment of advanced metastatic solid tumors.

Acrivon Therapeutics, Inc., a clinical stage biopharmaceutical company, engages in developing oncology medicines for the patients whose tumors are predicted to be sensitive to each specific medicine by utilizing its proteomics-based patient responder identification platform.

Rezolute, Inc., a clinical stage biopharmaceutical company, develops transformative therapies for metabolic diseases associated with chronic glucose imbalance in the United States.

AN2 Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing treatments for rare, chronic, and serious infectious diseases. It is developing epetraborole, a once-daily oral treatment for patients with chronic non-tuberculous mycobacterial lung disease.

Passage Bio, Inc., a genetic medicines company, develops transformative therapies for central nervous system diseases. It develops PBGM01, which utilizes a proprietary, AAVhu68 capsid to deliver to the brain and peripheral tissues a functional GLB1 gene encoding lysosomal acid beta-galactosidase for infantile GM1; PBFT02, which utilizes an AAV1 capsid to deliver to the brain a functional granulin (GRN) and gene encoding progranulin (PGRN) for the treatment of FTD-GRN; and PBKR03, which utilizes a proprietary, AAVhu68 capsid to deliver to the brain and peripheral tissues a functional GALC gene encoding the hydrolytic enzyme galactosylceramidase for infantile Krabbe disease.

Absci Corporation, a drug and target discovery company, provides biologic drug candidates and production cell lines using integrated drug creation platform for partners in the United States.

Kezar Life Sciences, Inc., a clinical-stage biotechnology company, engages in the discovery and development of novel small molecule therapeutics to treat unmet needs in immune-mediated diseases and cancer in the United States.

Kodiak Sciences Inc., a clinical stage biopharmaceutical company, researches, develops, and commercializes therapeutics to treat retinal diseases. Its lead product candidate is KSI-301, an anti-vascular endothelial growth factor antibody biopolymer that is in Phase IIb/III clinical study to treat wet age-related macular degeneration (AMD), as well as for the treatment of diabetic macular edema, naïve macular edema due to retinal vein occlusion, and non-proliferative diabetic retinopathy.

Galmed Pharmaceuticals Ltd., a clinical-stage biopharmaceutical company, focuses on the development of therapeutics for the treatment of liver diseases. It develops Aramchol, an oral therapy, which is in Phase III study for the treatment of non-alcoholic steato-hepatitis (NASH) in patients with overweight or obesity and who are pre-diabetic or type-II-diabetes mellitus.

Affimed N.V., a clinical-stage biopharmaceutical company, focuses on discovering and developing cancer immunotherapies in the United States, Germany, and rest of Europe.

Mereo BioPharma Group plc, a biopharmaceutical company, develops and commercializes therapeutics for the treatment of oncology and rare diseases in the United Kingdom and internationally.

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.