How our gene therapy works
Gene therapy offers the ability to permanently correct a disease at its most basic level, the genome, and could offer cures for many conditions that are considered incurable at this time. It is a flexible treatment option, gene therapy has the potential to treat a variety of illnesses by:
- Replacing missing or defective genes which can cause inherited or acquired disorders.
- Delivering genes that add needed proteins to the body.
- Delivering genes that enhance the body to resist disease and perform at higher levels.
- Introducing genes which stimulate cell growth and heal damaged tissue.
Gene therapy offers patient alternatives to regular daily maintenance. Patients suffering from life-threatening disease are often forced to endure swallowing pills or daily/weekly injections to treat and/or monitor their disease. Gene therapy could ease the burden of enduring the disease by offering the promise of 1-2 treatment injections in a patient’s lifetime with no need for long-term follow-up.
With the remarkable progress in genetics, we believe gene therapy will prove to play an increasingly prominent and transformative role in medicine. Gene therapy has the potential to treat and maybe cure monogenic diseases. Most monogenic diseases are ones for which a protein is defective or not being expressed. As such, gene therapy has the potential to address many of these congenital disorders. There is also a broad opportunity to apply gene therapy for acquired diseases as diverse as HIV, heart failure, Alzheimer’s and more.
Meet our Vectors
Adeno-Associated Virus (AAV): AAV vectors are not pathogenic to humans and cause limited and infrequent immune responses. These are the preferred vectors for disease in which the transgene does not need to integrate into the human genome (dividing cells cannot be targeted).
Advantages Of AAVs:
- Minimal immunogenicity/infectivity
- Not known to be pathogenic to humans
- Low immune response
- Long-term Gene Expression
- Lower Cost Production
- Multiple Serotypes
- Efficient Transduction
- Dividing/Non-Dividing Cells