A Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia

NCTID	NCT06185673 (View at clinicaltrials.gov)
Description	Subjects who have enrolled in the oculopharyngeal muscular dystrophy (OPMD) natural history study (Study BNTC-OPMD-NH-001) and have completed at least 6 months of follow up in Study BNTC-OPMD-NH-001 may be eligible to participate in this study, where all subjects will be treated with a single dose of BB-301. BB-301 will be injected directly into the middle pharyngeal constrictor muscle and the inferior pharyngeal constrictor muscle of the throat through the use of an open surgical procedure conducted under general anesthesia. The primary objectives of the study are to evaluate the safety of BB-301, to identify the best dose of BB-301 to administer to patients, and to characterize how well BB-301 works to improve the symptoms of dysphagia in patients with OPMD. (Show More)
Development Status	Active
Indication	Oculopharyngeal Muscular Dystrophy
Disease Ontology Term	DOID:11719
Compound Name	BB-301
Compound Description	modified AAV9 capsid that expresses a bifunctional construct under the control of a single muscle specific Spc5-12 promoter to achieve co-expression of both the codon-optimized PABPN1 mRNA and two shmiR molecules directed against wild type and mutant PABPN1
Sponsor	Benitec Biopharma, Inc.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	30
Results Posted	Not Available

Target Gene/Variant	PABPN1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement/gene inactivation
Route of Administration	Intramuscular (pharyngeal constrictor muscles)
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type
Dose 1	1.2E13 vg
Dose 2	3.6E13 vg
Dose 3	5.4E13 vg
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	5th subject was treated in February 2025