Benitec Biopharma, Inc.
Benitec Biopharma Inc. is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines. They utilize a proprietary platform called DNA-directed RNA interference (ddRNAi), which combines RNA interference with gene therapy to create medicines that enable sustained silencing of disease-causing genes after a single administration. The company is developing ddRNAi-based therapeutics for chronic and life-threatening human conditions, including Oculopharyngeal Muscular Dystrophy (OPMD). Their mission is to change treatment paradigms of human disease through innovative gene silencing technologies.
Industries
Nr. of Employees
small (1-50)
Benitec Biopharma, Inc.
3940 Trust Way, Hayward, CA 94545, United States
Products
AAV-delivered ddRNAi gene therapy candidate targeting mutant PABPN1 for OPMD (early-stage clinical candidate)
A gene therapy candidate delivered by AAV vectors that uses DNA-directed shRNA expression to reduce expression of a mutant PABPN1 gene and, where appropriate, provide gene replacement to address Oculopharyngeal Muscular Dystrophy (OPMD).
AAV-delivered ddRNAi gene therapy candidate targeting mutant PABPN1 for OPMD (early-stage clinical candidate)
A gene therapy candidate delivered by AAV vectors that uses DNA-directed shRNA expression to reduce expression of a mutant PABPN1 gene and, where appropriate, provide gene replacement to address Oculopharyngeal Muscular Dystrophy (OPMD).
Services
Collaborative and licensing arrangements to advance development and commercialization of ddRNAi-based therapeutics and associated vector constructs.
Collaborative projects to design, validate and translate ddRNAi constructs and vector delivery approaches for disease-targeted programs.
Execution of early-phase clinical trials and preparation of clinical data for scientific and regulatory communication.
Collaborative and licensing arrangements to advance development and commercialization of ddRNAi-based therapeutics and associated vector constructs.
Collaborative projects to design, validate and translate ddRNAi constructs and vector delivery approaches for disease-targeted programs.
Execution of early-phase clinical trials and preparation of clinical data for scientific and regulatory communication.
Expertise Areas
- Genetic medicine development
- RNA interference (RNAi) therapeutics
- Gene therapy delivery and vector design
- Early-phase clinical trial management (Phase 1b/2a)
Key Technologies
- DNA-directed RNA interference (ddRNAi)
- RNA interference (RNAi) and siRNA mechanisms
- shRNA expression from DNA constructs
- Adeno-associated virus (AAV) and non-replicating viral vectors