Alzheimer's Matters Blog

Closing in on a Cure: Oryzon Genomics and ORY-2001

January 17, 2017

Category: Developing Drugs

Dr. Tamara Maes


The Alzheimer’s Drug Discovery Foundation is proud to have supported over 20% of all the treatments for Alzheimer’s now in clinical trials. One of the most innovative of these is ORY-2001, which is being developed by Oryzon Genomics.

Oryzon is a biopharmaceutical company founded in 2000 in Barcelona, Spain, that has emerged as a global leader in epigenetic therapies. Epigenetics is a new area for drug development. Rather than trying to change actual genes, epigenetic drugs change how, and how much, those genes are expressed.

Researchers have identified numerous genes involved in Alzheimer’s disease. With initial support from the ADDF in 2000, Dr. Tamara Maes and her team at Oryzon developed ORY-2001. It works by inhibiting a protein that “turns down” the expression of several genes that are beneficial to the brain. By helping these genes express more, ORY-2001 may slow cognitive impairment and restore memory deficits in patients with Alzheimer’s and other disorders. In fact, preclinical testing has shown that ORY-2001 also holds promise for patients with multiple sclerosis.

The ADDF funded Oryzon again in 2015 to help prepare for human clinical trials. ORY-2001 became the first epigenetic therapy for any neurodegenerative disease to make it to clinical trials last year when it initiated a phase 1 study in 40 volunteers. That trial successfully demonstrated the drug’s safety. Another phase 1 clinical trial is currently underway to determine its optimal dose. And phase 2 clinical trials are being planned for later this year.

Thanks to the ADDF, this revolutionary treatment for Alzheimer’s disease is advancing. When you support the ADDF, 100% of your contribution funds drug discovery research.