Spark Therapeutics
Spark Therapeutics, Inc. is a biotechnology company focused on developing gene therapy products aimed at treating debilitating genetic diseases. Headquartered in Philadelphia, Pennsylvania, the company was founded in 2013 and has established itself as a leader in the field of gene therapy, with a robust pipeline that includes LUXTURNA for retinal dystrophy, as well as several candidates for hemophilia and choroideremia. Spark is also exploring therapies for neurodegenerative diseases and inherited retinal diseases. The company holds a collaboration agreement with Pfizer for the development of hemophilia B treatments and has partnered with Novartis for the commercialization of its lead product outside the United States. Spark Therapeutics leverages a proprietary manufacturing platform and a team with extensive expertise in research, clinical development, and regulatory affairs, building on a legacy of innovation in gene therapy from its origins at The Children’s Hospital of Philadelphia. As of December 2019, Spark operates as a subsidiary of Roche Holding AG.
Genable Technologies
Acquisition in 2016
Genable Technologies Limited is a bio-pharmaceutical company based in Dublin, Ireland, focused on developing gene therapies for autosomal dominant genetic diseases. The company's primary product, RhoNova, targets rhodopsin-linked autosomal dominant retinitis pigmentosa, a progressive genetic condition leading to blindness caused by mutations in the RHO gene. Genable employs a unique approach that utilizes adeno-associated virus (AAV) vectors to deliver RNA interference molecules, effectively suppressing both faulty and normal gene copies while replacing them with a modified gene that produces a normal protein. This method, known as suppression and replacement, addresses the challenge of mutation variability in dominant diseases. Genable Technologies is backed by a strong portfolio of patents in the United States, Europe, and worldwide, reflecting its innovative contributions to the field of gene therapy.
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