Science

Our Science

DNA Medicine Designed for Long-Term Safety

Infirmacea’s scientific approach is centered on the use of plasmid DNA for in vivo protein production. Plasmid offers advantages in safety, stability, and manufacturability, making it attractive for long-term therapeutic use.

However, conventional plasmid DNA has been limited by inefficient gene expression in vivo.

Overcoming a Key Expression Barrier

Through systematic investigation, Infirmacea identified a previously underappreciated intracellular bottleneck that limits protein expression after DNA delivery — distinct from cellular uptake and endosomal escape.

To address this, we developed gResQ™, a proprietary gene expression helper that improves intracellular processing and transcriptional efficiency.

gResQ™ Technology

gResQ™ rescues gene expression, which was repressed by cellular defense mechanism, across multiple genetic modalities, including:

• Plasmid DNA
• mRNA
• Viral vectors such as AAV

Its modular and tunable design allows adaptation to different genes, tissues, and therapeutic contexts without increasing DNA dose.

Delivery Strategy

Infirmacea’s DNA medicines are delivered primarily to skeletal muscle using chemical or physical delivery technology. Skeletal muscle provides a large, stable tissue reservoir capable of sustained protein production and systemic release.

By combining efficient expression with low DNA dosing, our approach is designed to minimize inflammatory and immune responses associated with gene delivery.

Design Principles

Our platform development is guided by three core principles:

• Physiological restoration of protein expression to support efficacy and safety
• Sustained protein expression to reduce dosing frequency
• Patient-friendly delivery to improve adherence and quality of life

These principles inform all exploratory applications of our technology.

トップへ戻る