Avirmax Biopharma has launched Investigational New Drug (IND)-enabling studies for ABI-201, marking a significant advancement in the treatment of dry age-related macular degeneration (AMD). The novel gene therapy represents a potential breakthrough in addressing one of the leading causes of blindness in older adults.
Innovative Triple-Gene Approach
ABI-201 distinguishes itself through a unique triple-action mechanism, delivering three therapeutic genes designed to:
- Correct complement activation dysregulation
- Provide anti-inflammatory protection for retinal pigment epithelia and photoreceptors
- Block retinal neovascularization
The therapy achieves these effects through a single intravitreal injection, designed to provide durable gene expression specifically within the eye. Preclinical studies across multiple species, including Cynomolgus monkeys, have validated the biological functions of the therapeutic genes.
Addressing a Critical Unmet Need
Age-related Macular Degeneration affects over 200 million people globally, with dry AMD accounting for approximately 90% of cases. Despite its prevalence, there are currently no effective long-term treatment options available.
"We are excited to reach this critical milestone in ABI-201 development," stated Shengjiang Shawn Liu, Ph.D., Chief Executive Officer of Avirmax Biopharma. "ABI-201 has the potential to revolutionize the treatment for dry AMD and many other retinal disorders."
Advanced Delivery Technology
The therapy leverages Avirmax's proprietary AAV.N54 capsid technology, which enables efficient delivery of therapeutic genes to the macular retina through intravitreal injection. This same delivery platform is currently being evaluated in Phase 1/2a trials for ABI-110, targeting wet AMD including PCV.
Comprehensive Development Program
The ongoing IND-enabling studies focus on several critical aspects:
- Safety and tolerability assessment
- Vector biodistribution analysis
- Transgene expression evaluation
- Pharmacokinetic profiling
These studies are being conducted in monkey models to ensure robust preclinical data before human trials.
Development Timeline
Avirmax Biopharma has outlined a clear development pathway for ABI-201:
- IND application submission planned for Q4 2025
- Phase 1/2a clinical trials expected to commence following regulatory clearance
- Initial focus on patients with dry AMD
The therapy aims to address multiple aspects of disease progression, including reducing geographic atrophy growth and preventing progression from dry to wet AMD, with the ultimate goal of preserving patients' vision.
