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uniQure NV is a gene therapy company. It develops treatments and platforms for patients suffering from genetic and other devastating diseases. Its products and services are focused on hemophilia, Huntington's disease, and cardiovascular diseases. The company is focused on the development of the pipeline of gene therapies with the collaboration of Bristol Myers Squibb for cardiovascular diseases.
Krystal Biotech Inc is engaged in the healthcare sector in the United States. It is focused on the development of easy-to-use, redosable gene therapies to improve the lives of patients living with debilitating diseases. The company used its proprietary gene therapy platform, STAR-D to develop treatments for rare or orphan dermatological indications caused by the absence of or a mutation in a single gene. It plans to leverage its platform to expand its pipeline to include other dermatological indications in the future. The company has one operating segment, which is the business of developing and commercializing pharmaceutical products.