At the core of biology is the flow of information from genes to proteins. Protein therapeutics, such as monoclonal antibodies, apply this principle outside the body: genes encoding therapeutic proteins are introduced into producer cells, manufactured at scale, purified, and administered as injectable medicines.
Science
Why AAV Gene Therapy
Why AAV Gene Therapy
Limitations of Conventional Protein Therapies: The Burden of Repeat Dosing
01
Repeat dosing to sustain effect
Because protein drugs are cleared over time, maintaining benefit typically requires ongoing administration.
02
Persistent burden in sensitive tissues
For sensitive tissues such as the eye or spinal canal, repeated injections over a lifetime impose significant physical discomfort and clinical burden.
03
Quality of life and infection risk
Beyond physical burden, repeated treatments disrupt daily life, increase infection risk, and place a lasting emotional and practical strain on both patients and caregivers.
AAV Gene Therapy: A Paradigm Shift
Gene therapy addresses this limitation at its root by delivering therapeutic genes directly into the body, enabling patients’ own cells to produce therapeutic proteins—effectively creating an <i class="--italic">in vivo</i> factory. Central to this approach is the vector, which delivers the gene safely and efficiently to target cells.
A single dose, lasting impact
Among various viral vectors, AAV(Adeno-associated virus) is one of the most widely studied delivery systems due to its low pathogenicity, minimal immune response, and strong capacity for long-term gene expression. AAV can enable sustained therapeutic protein expression for years after a single administration. This unique property positions AAV as a potential game changer for the treatment of chronic and rare diseases such as macular degeneration. To free patients from the burden of continuous treatment through a single administration that delivers lasting therapeutic benefit. This is the future that AAV gene therapy aims to create.
