This page shows the latest inherited retinal disease news and features for those working in and with pharma, biotech and healthcare.
neparvovec) for a rare inherited retinal disease – as well as four other candidates in clinical trials headed by haemophilia A therapy SPK-8011.
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
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
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,
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
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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