Latest news

• 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

  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

  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

  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

  biallelic RPE65-mediated inherited retinal disease (IRD). ... a policy framework - including the development of disease-specific guidance - “for the evaluation and review of gene therapy.”.