Ligand Modified Poly (Beta Amino) Ester Based Polymer and Nano-Complex for Muscle Targeted Gene Delivery

This technology provides an advanced non-viral delivery platform that combines safety, specificity and scalability.

Key value drivers:

• Muscle-targeted delivery of therapeutic nucleic acids
• Non-viral alternative to AAV vectors
• High transfection efficiency with low cytotoxicity
• Biodegradable polymer backbone
• Reduced immunogenicity
• Stable nanocomplex formation
• Lower manufacturing complexity than lipid nanoparticle systems
• Compatible with different types of nucleic acid therapeutics
• Potential for repeat dosing
• Flexible chemistry enabling disease-specific customization
• Platform Technology: A single delivery platform adaptable to multiple muscle disorders and future nucleic acid therapeutics.
• Scalable Manufacturing Potential: Straightforward polymer synthesis enables scalable and cost-effective manufacturing.

The platform is suitable for a broad range of therapeutic and research applications in future including:

• Gene therapy applications in different inherited neuromuscular disorders: Muscle directed chemistry can be exploited
• Gene replacement, RNA therapeutics and gene editing applications: Versatility of cargo choice can be exploited
• Platform technology for other gene therapy applications: Modular chemistry can be exploited for applications beyond muscular disorders.
• Application as laboratory tool in muscle regeneration and other allied research.

Competitive Advantages

Additional demonstrated advantages include:

• Better nucleic acid condensation than conventional polymers of similar chemistry
• Stable nanocomplexes
• Reduced non-specific organ distribution
• Lower anticipated therapeutic dose
• Potential reduction in systemic toxicity