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SKIP-NMD

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A phase I IIa clinical trial in Duchenne muscular dystrophy using systemically d...

A phase I IIa clinical trial in Duchenne muscular dystrophy using systemically delivered morpholino antisense oligomer to skip exon 53 Duchenne muscular dystrophy (DMD) is a progressive, lethal muscle degenerative condition arising from the absence of dystrophin in skeletal and cardiac muscles. 65% of DMD boys have out-of-frame deletions. Modulation of pre-mRNA s... ver más

30/04/2016

UNIVERSITY COLLEGE...

8M€

Presupuesto del proyecto: 8M€

Líder del proyecto

UNIVERSITY COLLEGE LONDON No se ha especificado una descripción o un objeto social para esta compañía.

Fecha límite participación Sin fecha límite de participación.

Ver 9 Participantes

Financiación concedida El organismo FP7 notifico la concesión del proyecto el día 2016-04-30 No tenemos la información de la convocatoria

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Información adicional privada

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9 Participantes

UNIVERSITY COLLEGE LONDON

1.15M€ | Lider

C.R.I.S

N.D. | Participante

UNEW

N.D. | Participante

GOSH

N.D. | Participante

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Líder del proyecto

UNIVERSITY COLLEGE LONDON No se ha especificado una descripción o un objeto social para esta compañía.

Presupuesto del proyecto 8M€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto Duchenne muscular dystrophy (DMD) is a progressive, lethal muscle degenerative condition arising from the absence of dystrophin in skeletal and cardiac muscles. 65% of DMD boys have out-of-frame deletions. Modulation of pre-mRNA splicing by exon skipping is the most promising molecular intervention in DMD. 2 Phase Ib and 2 Phase IIa clinical trials (MDEX Consortium in collaboration with Sarepta Therapeutics; a Dutch Consortium) demonstrated that delivery of antisense oligonucleotides (AOs) to mediate exon skipping of exon 51 were able to return specific DMD mutations in-frame (~13% of all mutations) leading to new dystrophin protein expression after intramuscular and systemic delivery. The Dutch study of repeated 2-O-methylated phosphorothioate (2OMe) AO administration suggested limited efficacy after 5 weeks of treatment. Our MDEX Consortium study using a morpholino (PMO) AO demonstrated a clear dose response, robust dystrophin restoration and reduction of muscle inflammation after 12 weeks at doses up to 20mg/Kg, with no drug related adverse events. This, and preclinical studies focused on level of protein expression, clearly indicate that PMO have a superior therapeutic index compared to 2OMe. New PMOs are needed to target other DMD mutations. We will develop a PMO to skip exon 53 and perform a clinical trial in DMD boys using a world leading pan-European consortium. This will allow us to advance this class of PMO therapy in DMD by i. assessing the safety and efficacy of targeting another exon; ii. exploring the use of non-invasive techniques to monitor dystrophin restoration. This new PMO will be administered over 12 weeks in 3 groups, each of 4 DMD boys, receiving between 4 to 30mg/kg or placebo. If well tolerated, all boys will be treated for another 24 weeks at a dose of 30mg/kg. Safety and dystrophin restoration in a muscle biopsy at the end of this period will be the study endpoints. MRI, MRS and serum miRNA will be used to monitor muscle pathology non-invasively.

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