Mardin (Ashgar) Fallah

Our research focuses on the development of next-generation RNA platforms for therapeutic protein expression and regenerative medicine. The lab aims to harness RNA replication mechanisms inspired by alphaviruses to enable potent, durable, and controllable gene expression in target tissues. This approach allows for high-level protein production at low doses, paving the way for safer and more cost-effective genetic medicines.

Leveraging artificial intelligence (AI), we design and optimize RNA sequences and delivery systems to enhance stability, reduce immunogenicity, and improve therapeutic performance.

A key area of interest is age-related diseases such as osteoporosis and Age-related Macular Degeneration (AMD). Our work integrates RNA engineering, delivery systems, and in vivo modeling to create therapies that promote bone regeneration and preserve retinal function.

We are particularly interested in understanding and overcoming the innate immune barriers that limit RNA-based therapies. By combining RNA structure optimization with delivery innovations, our goal is to enhance stability, reduce immunogenicity, and achieve tissue-specific expression.

 Current Projects

1. RNA-Expressed Therapeutic Proteins (Monoclonal Antibodies & Fusion Proteins)
Development of RNA constructs that encode long-acting therapeutic proteins, including Denosumab analogs for osteoporosis and anti-VEGF fusion proteins for age-related macular degeneration. These RNA-based biologics are designed to provide sustained protein expression, reducing or eliminating the need for frequent injections.

2. RNA for iPSC Generation & Direct Cellular Reprogramming
Engineering of RNA systems encoding key transcription factors to induce pluripotency (iPSC generation) or directly reprogram somatic cells into osteoblasts and other therapeutic lineages. This platform leverages RNA-driven cell fate conversion for regenerative medicine applications.

3. Targeted Delivery of RNA to Bone and Retina:
Collaboration with local biotech partners to develop lipid nanoparticle (LNP) formulations coated with peptide or antibody ligands for tissue-specific RNA delivery to bone and retina, enhancing therapeutic precision and minimizing systemic exposure.