Genethon — Biomedical Watch V2

Traitements3programmes

Essais1liés

Publications1liées

SourceDBlocale

Traitements

3

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
GNT0004 (1x10e13 vg/kg)GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin. Duchenne muscular dystrophy (DMD) is a neuromuscular disorder caused by dystrophin gene mutations leading to absence of functional dystrophin, a key protein that prevents muscle cell damage during physiological contractions. To address the underlying cause of the disease, GNT0004 delivers an optimized dystrophin gene that is capable to produce a shortened but functional dystrophin protein in target tissues (heart and skeletal muscles) in DMD subjects. It is expected to significantly delay or slow disease progression. GNT0004 will be given as a one-time intravenous (IV) infusion to help it reach muscles throughout the body.	Myopathies	Phase 2/3	Thérapie génique + Ralentissement de la progression	Belgium, France, Spain, United Kingdom	À vérifier
GNT0004 (3x10e13 vg/kg)GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin. Duchenne muscular dystrophy (DMD) is a neuromuscular disorder caused by dystrophin gene mutations leading to absence of functional dystrophin, a key protein that prevents muscle cell damage during physiological contractions. To address the underlying cause of the disease, GNT0004 delivers an optimized dystrophin gene that is capable to produce a shortened but functional dystrophin protein in target tissues (heart and skeletal muscles) in DMD subjects. It is expected to significantly delay or slow disease progression. GNT0004 will be given as a one-time intravenous (IV) infusion to help it reach muscles throughout the body.	Myopathies	Phase 2/3	Thérapie génique + Ralentissement de la progression	Belgium, France, Spain, United Kingdom	À vérifier
GNT0004 (1x10e13 vg/kg)GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin. Duchenne muscular dystrophy (DMD) is a neuromuscular disorder caused by dystrophin gene mutations leading to absence of functional dystrophin, a key protein that prevents muscle cell damage during physiological contractions. To address the underlying cause of the disease, GNT0004 delivers an optimized dystrophin gene that is capable to produce a shortened but functional dystrophin protein in target tissues (heart and skeletal muscles) in DMD subjects. It is expected to significantly delay or slow disease progression. GNT0004 will be given as a one-time intravenous (IV) infusion to help it reach muscles throughout the body.	Myopathies	Phase 2/3	Thérapie génique + Ralentissement de la progression	Belgium, France, Spain, United Kingdom	À vérifier

Essais cliniques

1

Molécule	Indication / population	Phase	NCT	Titre	Statut
GNT0004 (1x10e13 vg/kg)	Myopathies	Phase 2/3	NCT07856329	A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy	RECRUITING

Publications

1

Molécule	Indication / population	Titre	Journal	Date
GNT0004 (3x10e13 vg/kg)		Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy.	Molecular therapy : the journal of the American Society of Gene Therapy