Advancing the Frontiers of Gene & RNA-Based Medicine
Our Ethical Commitment
Multidisciplinary excellence spanning molecular biology, RNA chemistry, gene editing, and targeted delivery technologies
Precision RNA Therapeutics
siRNA, saRNA, mRNA, and antisense oligonucleotides development Proprietary aptamer-mediated targeting systems Advanced nucleic acid chemistry for enhanced stability
Gene Therapy & Editing
Lentiviral and AAV vectors with improved safety CRISPR/Cas-based and epigenetic editing systems GMP-compliant scalable vector manufacturing
Targeted Delivery Innovation
Tissue-specific targeting to brain, cartilage, bone, liver, muscle Multi-modal delivery approaches Imaging-guided administration for precision
Clinical Development
Rare disease trial design and adaptive protocols Global regulatory expertise (FDA, EMA, MHRA) Academic and CRO partnerships worldwide
Why Our R&D Stands Out
Multidisciplinary Leadership
Led by globally recognised experts in RNA therapeutics, gene therapy, and rare disease research
Innovative Platforms
Proprietary aptamer-targeted delivery systems for larger, more complex genetic payloads
Patient-Centred Focus
R&D roadmap driven by unmet clinical needs and diseases with limited treatment options
Global Collaboration
Strategic alliances with academic institutions, biotech companies, and patient advocacy groups
Therapeutic Areas of Focus
Our R&D pipeline spans 21 disease areas with transformative potential
Rare CNS Disorders
- • Huntington's disease
- • Parkinson's disease
- • Sanfilippo syndrome
Musculoskeletal
- • Huntington's disease
- • Parkinson's disease
- • Sanfilippo syndrome
Genetic Syndromes
- • Hurler syndrome (MPS I)
- • Hunter syndrome (MPS II)
- • Sly syndrome (MPS VII)
Global Access
- • Autism spectrum disorder
- • Developmental delays
- • Cognitive disorders
Highest Standards
- • NAFLD & NASH
- • Type 2 diabetes
- • Metabolic syndromes
Additional Areas
- • Oncology
- • Immunology
- • Ophthalmology