All CPD case study articles

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    CPD Supplement

    Qualification of stride velocity endpoint in Duchenne Muscular Dystrophy

    2021-10-01T10:39:00Z

    In April 2019, the Committee for Medicinal Products for Human Use (CHMP) issued a qualification opinion on stride velocity as an endpoint in clinical studies of medicines for the treatment of Duchenne Muscular Dystrophy (DMD).[1] Mutations in the dystrophin gene cause this inherited, x-linked disorder which causes progressive muscle weakness ...

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    CPD Supplement

    CTIS national pilot – Belgium

    2021-04-01T14:06:00Z

    Although the CTIS is still under development, several EU countries (eg, Belgium, France, Germany, Finland and the UK prior to Brexit) are running national pilots to test the new way of working introduced by the Clinical Trial Regulation 536/2014. Pilot conditions in the various countries are different. The Heads of ...

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    CPD Supplement

    Case Study- Luxturna

    2021-02-01T15:35:00Z

    The ATMP Luxturna (voretigene neparvovec) is a GTMP which utilises a recombinant adeno-associated viral vector serotype 2 (rAAV2) capsid as a delivery vehicle for the human retinal pigment epithelium 65 kDa protein (hRPE65) cDNA to the retina. Luxturna is approved for patients with retinal dystrophy associated with biallelic RPE65 mutations ...

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    Journal

    Gene therapy: regional assessment comparison

    2020-11-01T15:52:00Z

    Axicabtagene ciloleucel (tradename YESCARTA) is an autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets CD19 and is approved for the treatment of aggressive, relapsed or refractory forms of B-cell non-Hodgkins lymphoma (NHL). The active substance is composed of a patient’s cells that have undergone ex vivo modification, which causes ...

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    CPD Supplement

    Creating an integrated summary of immunogenicity report

    2020-05-01T13:43:00Z

    Introduction The purpose of this case study is to illustrate the creation of an integrated summary of immunogenicity report (ISI) that fulfils the recommendations described in current regulatory guidance for the scope of information needed to facilitate assessment of immunogenicity-related risks to support marketing authorisation of novel and biosimilar versions ...