Spark Therapeutics
Spark Therapeutics, Inc. is dedicated to developing innovative gene therapy products aimed at treating severe genetic diseases. The company's portfolio includes LUXTURNA, specifically designed for patients with biallelic RPE65 mutation-associated retinal dystrophy. Additionally, Spark is advancing several gene therapy candidates such as SPK-8011 and SPK-8016 for hemophilia, SPK-7001 for choroideremia, and SPK-9001 for hemophilia B. It is also exploring liver-directed therapies for conditions like Pompe disease and investigating treatments for neurodegenerative diseases, including Huntington's disease and TPP1 deficiency, a form of Batten disease. The company collaborates with Pfizer for the development of hemophilia B treatments and has a licensing agreement with Novartis for the commercialization of LUXTURNA outside the United States. Founded in 2013 and based in Philadelphia, Spark Therapeutics operates as a subsidiary of Roche Holding AG and leverages a proprietary manufacturing platform to support its clinical programs across various therapeutic areas.
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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