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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.

Roche moving closer to approval of Spark takeover; report

Roche moving closer to approval of Spark takeover; report

neparvovec) for a rare inherited retinal disease – as well as four other candidates in clinical trials headed by haemophilia A therapy SPK-8011.

Latest news

  • Roche buys gene therapy specialist Spark for $4.3bn Roche buys gene therapy specialist Spark for $4.3bn

    The $114.50 per share deal will give the Swiss pharma giant rights the approved therapy Luxturna (voretigene neparvovec) for a rare inherited retinal disease, as well as four candidates in ... Also in Spark’s pipeline is SPK-7001 for choroideremia in

  • J&J bolsters gene therapy unit with $100m MeiraGTx deal J&J bolsters gene therapy unit with $100m MeiraGTx deal

    The companies will also work together on new targets for other inherited retinal diseases and on adeno-associated virus (AAV) manufacturing technology. ... The US market developed a little later, with Spark Therapeutics’ Luxturna (voretigene neparvovec

  • Freeline raises £88m to accelerate haemophilia gene therapy Freeline raises £88m to accelerate haemophilia gene therapy

    additional testing of its lead haemophilia B gene therapy, and take another for Fabry disease into the clinic. ... This adds to Freeline and its focus on chronic systemic disease, Nightstar ( specialising in inherited retinal diseases), and Gyroscope,

  • 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

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