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New gene therapy for inherited blindness could cost patients up to $1m per treatment

A new gene therapy for the treatment of Leber’s congenital amaurosis (LCA) is predicted to be priced at somewhere between $650,000 and $1,000,000 per patient per treatment, sparking considerable debate in the gene therapy market. According to Wall St. analysts, the new gene therapy for LCA may earn up to $200M in revenues in its […]

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New location for European Medicines Agency (EMA) may have a significant impact for patients and industry

Following on from the UK government decision to trigger Article 50 of the Treaty on European Union, and thereby provide notice of the country’s intention to leave the European Union (EU), one near-term impact will be the requirement for the European Medicines Agency (EMA) to move from the UK to a new location. The EMA, […]

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Spark Therapeutics announces additional efficacy outcomes from first randomised US trial in gene therapy

Spark Therapeutics (NASDAQ:ONCE), a gene therapy company based in Philadelphia, USA, has announced announced that the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended (16-0) approval of “LUXTURNA” (voretigene neparvovec), an investigational, “potential one-time gene therapy, for the treatment of patients with vision loss due to confirmed […]

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