Precision BioSciences, Inc. — Biomedical Watch V2

Traitements2programmes

Essais1liés

Publications0liées

SourceDBlocale

Traitements

2

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
PBGENE-DMD (IV)The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.	Myopathies	Phase 1/2	À vérifier	United States	À vérifier
PBGENE-DMD (IV)The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.	Myopathies	Phase 1/2	À vérifier	United States	À vérifier

Essais cliniques

1

Molécule	Indication / population	Phase	NCT	Titre	Statut
PBGENE-DMD (IV)	Myopathies	Phase 1/2	NCT07429240	PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)	RECRUITING

Publications

0

Molécule	Indication / population	Titre	Journal	Date
Aucune publication.