Research
With a belief that knowledge is power, we connect our patients directly with their results so they have valuable health information when they need it most, care about our people and are committed to excellence in our work.
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Gene Therapy & Genetic Medicine
Genetic & Genomic Research
Metabolic Research
Neuromuscular Disease Research
Paediatric Rare Disease Research
AAV9 Delivery Technology - Vector Delivery System
AAV9 is the backbone of HaliGene’s delivery architecture due to its unmatched ability to transduce neuromuscular and CNS tissues. Its safety profile and established clinical use make it one of the most validated vectors for systemic genetic therapy.
- Selective Tissue Tropism:
AAV9 naturally targets skeletal muscle, heart, and nervous tissue, aligning with CHKB disease pathology. - Efficient Gene Transfer:
Once internalized, AAV9 delivers the therapeutic gene to the nucleus, allowing for stable episomal expression. - Broad Biodistribution:
Systemic administration enables correction across distributed muscle groups and cardiac tissue.
- Clinically established vector with strong safety profile
- Enables correction of multi-system rare diseases
- Supports durable gene expression
- Suitable for infants, children, and adults due to flexibility in dosing approaches
Join us in advancing transformative gene therapies for ultra-rare neuromuscular diseases. Connect with our team to explore collaboration opportunities.
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