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Yesterday (January 25), Novartis announced a partnership agreement with Spark to jointly develop and promote innovative gene therapies outside the UNITED States.
agreement, Novart will make an upfront payment of $105 million to Spark, which is expected to total $170 million.
Luxturna, which was approved last year, is expected to treat a genetic disease called hereditary retinal dystrophies.
more than 220 mutations in the human body, which can lead to the disease and ultimately a visual decline in progression. Among them, the disease caused by the RPE65 gene allethic mutation affects about 1,000 to 2,000 people in the United States. In the human body, the RPE65 gene encodes an enzyme that plays an important role in normal vision. As a result, a drop or loss of RPE65 enzyme levels due to genetic mutations can impair a patient's vision and eventually lead to blindness.
Luxturna, brought in by Spark, uses adeno-related virus technology to introduce healthy RPE65 genes directly into retinal cells, allowing them to produce normal RPE65 enzymes and restore vision. In clinical trials involving 41 patients, the ability of treated patients to avoid obstacles in dark light was significantly improved.
