What is Gene Therapy?


Gene therapy aims to address diseases at the most fundamental level: the genes that lead to the disease or its symptoms. There are several different approaches employed by gene therapy, including:

Turning off damaged, disease-causing gene(s)

Adding a functioning gene(s) to do the work of a missing or defective gene(s)​

Replacing a damaged gene(s) with a
functioning one​

Fixing a damaged gene(s)
so that it functions again

Gene therapies need to enter cells to do this work of manipulating genes and they do so by leveraging a virus’ natural ability to enter cells and express genes, but using a non-disease-causing virus, called a vector. These vectors can be designed to target certain cells better than others, or they can be delivered directly to the cells that are most relevant to the disease.

The vector carries a “payload,” including a gene or genes, which is inserted into the nucleus of a cell so the cell can now produce the functioning protein or stop producing a defective protein This approach is expected to result in long-lasting effects from a one-time treatment.

Each of Axovant’s gene therapies are specifically designed to address the underlying biological cause of each disease we tackle:

GM1 Gangliosidosis, Tay-Sachs and Sandhoff Diseases


GM1 gangliosidosis, Tay-Sachs and Sandhoff diseases are a set of diseases caused by defects in the GLB1, HEXA or HEXB genes, respectively. These defects cause compounds called gangliosides to build up in neurons and cause severe neurological damage. AXO-AAV-GM1 and AXO-AAV-GM2, our gene therapies for these diseases, introduce a functioning copy of the affected gene to cells in the nervous system.

Parkinson’s Disease


Parkinson’s disease results in a loss of dopamine in the brain. Axovant’s gene therapy for Parkinson’s disease, AXO-Lenti-PD adds three genes that help to produce dopamine into the part of the brain, the putamen, that is most impacted by the loss of dopamine in Parkinson’s disease.

Visit www.asgct.org/education/parkinsons-disease to learn more. Click here for a Parkinson's disease gene therapy infographic. 

Learn More about GM1 Gangliosidosis, Tay-Sachs and Sandhoff Diseases

Learn More about Parkinson’s Disease


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