BB-301

This drug entry is a stub and has not been fully annotated. It is scheduled to be annotated soon.

Identification

Generic Name: BB-301
DrugBank Accession Number: DB17011
Background: 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 (siRNAs) against mutant PABPN1.¹ Developed by Benitec Biopharma, BB-301 is being investigated for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD).²
Type: Biotech
Groups: Investigational
Biologic Classification: Gene Therapies
Other gene therapies
Synonyms: Not Available

Indication: Not Available
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Pharmacodynamics: Not Available
Mechanism of action: Not Available
Absorption: Not Available
Volume of distribution: Not Available
Protein binding: Not Available
Metabolism: Not Available
Route of elimination: Not Available
Half-life: Not Available
Clearance: Not Available
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Toxicity: Not Available
Pathways: Not Available
Pharmacogenomic Effects/ADRs: Not Available

Drug Interactions: This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions: Not Available

General References

Strings-Ufombah V, Malerba A, Kao SC, Harbaran S, Roth F, Cappellari O, Lu-Nguyen N, Takahashi K, Mukadam S, Kilfoil G, Kloth C, Roelvink P, Dickson G, Trollet C, Suhy D: BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy. Mol Ther Nucleic Acids. 2021 Feb 18;24:67-78. doi: 10.1016/j.omtn.2021.02.017. eCollection 2021 Jun 4. [Article]
Globe Newswire News Release: Benitec Biopharma Receives FDA Clearance of the IND for BB-301 for the Treatment of Oculopharyngeal Muscular Dystrophy [Link]

External Links

Not Available

Clinical Trials

Phase	Status	Purpose	Conditions	Count

Drug created at September 19, 2022 20:46 / Updated at June 27, 2023 01:10