Precision BioSciences has activated Arkansas Children’s Hospital as the first site and initiated patient enrolment for its Phase I/II FUNCTION-DMD clinical trial of PBGENE-DMD for Duchenne muscular dystrophy (DMD).
PBGENE-DMD is a first-in-class in vivo gene editing therapy developed using its ARCUS platform. It is aimed at a significant subset of boys with this genetic disorder.
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It aims to deliver sustained muscle function improvement for DMD patients with mutations between exons 45 and 55.
The Phase I/II FUNCTION-DMD study will involve ambulatory patients and primarily assess the tolerability, safety, dystrophin protein expression, and functional outcomes.
Arkansas Children’s is a certified Duchenne Care Centre by the Parent Project Muscular Dystrophy and is also a Muscular Dystrophy Association Care Centre.
This centre provides specialised, multidisciplinary care and supports the diagnosis and management of neuromuscular diseases, facilitating the planned trial.
The study is set to enrol ambulatory children aged two to seven with DMD caused by targeted mutations, which covers up to 60% of boys with the condition.
Designed to remove exons 45-55 using two ARCUS nucleases delivered by a single adeno-associated virus (AAV), PBGENE-DMD seeks to restore functional dystrophin.
The Phase I/II trial includes immune modulation and comprehensive safety monitoring.
PBGENE-DMD has orphan drug and fast track designations and is eligible for a paediatric rare disease priority review voucher.
Precision BioSciences CEO Michael Amoroso said: “The activation of Arkansas Children’s Hospital marks a critical step in advancing PBGENE-DMD into the clinic and expanding access for patients and families who are looking for novel options to address this devastating genetic disease.
“Dr Aravindhan Veerapandiyan and the team at Arkansas Children’s Hospital bring deep experience in running clinical trials and research in Duchenne. As the inaugural site for the FUNCTION-DMD study, we are pleased to open screening and enrolment at this centre while we continue to work to activate other trial sites for inclusion in the FUNCTION-DMD study.”
Earlier this month, Precision received clinical trial application approval to expand its global ELIMINATE-B study of PBGENE-HBV, an in vivo gene editing therapy for chronic hepatitis B.
