AAVantgarde Bio, Corp USA
AAVantgarde is a clinical stage biotechnology company dedicated to developing next-generation therapies for inherited retinal diseases (IRDs). Their mission is to retain and rejuvenate sight for patients with IRDs by addressing the root causes of these diseases through innovative gene therapy platforms. The company originated from research at TIGEM and was co-founded by Professor Alberto Auricchio, focusing on treatments for diseases like Stargardt and Usher 1B.
Industries
Nr. of Employees
small (1-50)
AAVantgarde Bio, Corp USA
Products
Clinical-stage gene therapy program targeting MYO7A for Usher 1B
A clinical-stage gene replacement program using AAV-based delivery strategies aimed at treating Usher syndrome type 1B by restoring MYO7A expression in the retina.
Clinical-stage gene therapy program targeting ABCA4 for Stargardt disease
A clinical-stage program developed to deliver functional ABCA4 coding sequences to the retina using expanded-capacity AAV strategies for Stargardt disease.
Clinical-stage gene therapy program targeting MYO7A for Usher 1B
A clinical-stage gene replacement program using AAV-based delivery strategies aimed at treating Usher syndrome type 1B by restoring MYO7A expression in the retina.
Clinical-stage gene therapy program targeting ABCA4 for Stargardt disease
A clinical-stage program developed to deliver functional ABCA4 coding sequences to the retina using expanded-capacity AAV strategies for Stargardt disease.
Services
Early-phase clinical trial conduct for ophthalmic gene therapies
Design and execution of Phase I/II clinical studies for inherited retinal diseases, including patient recruitment through clinical sites and adherence to GCP.
Natural history study design and execution
Implementation of observational natural history studies to characterize disease progression and support endpoint selection for clinical trials.
Early-phase clinical trial conduct for ophthalmic gene therapies
Design and execution of Phase I/II clinical studies for inherited retinal diseases, including patient recruitment through clinical sites and adherence to GCP.
Natural history study design and execution
Implementation of observational natural history studies to characterize disease progression and support endpoint selection for clinical trials.
Expertise Areas
- Gene therapy for inherited retinal diseases
- AAV vector engineering and large-gene delivery
- Preclinical large-animal ophthalmic studies
- Early-phase clinical trial management (Phase I/II)
Key Technologies
- AAV-based large-gene delivery
- Dual AAV vector strategies
- Intein-mediated protein trans-splicing
- Viral vector engineering