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inherited retinal disease

This page shows the latest inherited retinal disease news and features for those working in and with pharma, biotech and healthcare.

Freeline raises £88m to accelerate haemophilia gene therapy

Freeline raises £88m to accelerate haemophilia gene therapy

Last week it launched SwanBio, focused on the Central Nervous System, and Orbit Biomedical, which is based on a leading sub-retinal surgical delivery platform acquired from Janssen Biotech. ... This adds to Freeline and its focus on chronic systemic

Latest news

  • Novartis negotiates ex-US rights to Spark gene therapy Novartis negotiates ex-US rights to Spark gene therapy

    Luxturna is the first gene therapy to be approved in the US where it has a similar indication - biallelic RPE65-mediated inherited retinal disease (IRD) - and is being launched at a ... No otherwise healthy child should have to go blind due to this

  • Spark sets Luxturna price, igniting US affordability debate Spark sets Luxturna price, igniting US affordability debate

    Luxturna is used to treat a sight-robbing disorder called biallelic RPE65-mediated inherited retinal disease (IRD), which affects somewhere between 1, 000 and 2, 000 people in the US with

  • Spark’s Luxturna becomes US’s first approved gene therapy Spark’s Luxturna becomes US’s first approved gene therapy

    The FDA has chalked up another first for 2017, approving Spark Therapeutics’gene therapy Luxturna for a sight-robbing disorder called biallelic RPE65-mediated inherited retinal disease (IRD). ... a policy framework - including the development of disease

  • Spark closes on first US gene therapy approval Spark closes on first US gene therapy approval

    Spark closes on first US gene therapy approval. FDA starts review of Luxturna for patients with inherited retinal disease. ... Luxturna (voretigene neparvovec) is under review for a condition known as biallelic RPE65-mediated inherited retinal disease

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