BB-301

DB17011InvestigationalGene Therapies

BB-301 is a modified AAV serotype 9 (AAV9) capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs... Developed by Benitec Biopharma, BB-301 is being investigated for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD).

Code names: BB-301

What you can answer from here — as of September 25, 2025

1Clinical trialPhase, status and sponsor resolved per trial

2+ReferencesStructured and connected to the statements they support

Which companies are running trials of BB-301, in which indications and phases, and which of those programs are still active?

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