Genetic Disorders; Hard-to-Treat Diseases with Existing Therapeutics
A genetic disorder is a disease caused by mutation(s) in the DNA sequence away from the normal sequence. There have been several therapeutic ideas for the treatment of genetic disorders for which no curative therapies exist. The present enzyme replacement therapy is a good example, however, it requires regular treatment for the rest of one's life as blood levels rise with each dose and then fall. The other good example is a gene therapy that can maintain stable blood levels for an extended period with a single dose and is expected to improve patient's quality of life.
In the last decade, multiple approvals of in-vivo gene therapies using AAV and ex-vivo gene therapies using lentivirus and retrovirus were achieved, mainly in the US and Europe, but many challenges such as safety and therapeutic efficacy issues remain.
Our Platform, Novel Ex-vivo Gene Therapy Using GMAC
The GMAC platform is fusion innovation that leverages the excellent properties of adipocytes such as long lifetime and non-tumorigenesis, and viral gene vectors to treat patients with safety and low treatment burden. See more details to Platform.