Benitec Ltd
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 Ltd
Hayward, California, United States, North America
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
Partnering and licensing for genetic medicine development
Collaborative and licensing arrangements to advance development and commercialization of ddRNAi-based therapeutics and associated vector constructs.
Preclinical and translational research collaborations
Collaborative projects to design, validate and translate ddRNAi constructs and vector delivery approaches for disease-targeted programs.
Clinical development and data reporting support
Execution of early-phase clinical trials and preparation of clinical data for scientific and regulatory communication.
Partnering and licensing for genetic medicine development
Collaborative and licensing arrangements to advance development and commercialization of ddRNAi-based therapeutics and associated vector constructs.
Preclinical and translational research collaborations
Collaborative projects to design, validate and translate ddRNAi constructs and vector delivery approaches for disease-targeted programs.
Clinical development and data reporting support
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