Epigenome Editing by Induced Proximity to Durably Restore Paternal UBE3A Expression
Epigenome Editing by Induced Proximity to Durably Restore Paternal UBE3A Expression
Knowing that Angelman Syndrome (AS) is a brain disorder caused by a missing or nonfunctional gene called UBE3A, many treatments attempts are trying to turn on the silent copy of this gene. Some require frequent injections, while others use complicated gene therapies using a virus to carry the payload, which are expensive. This team is developing a new
treatment strategy that works with the body’s natural processes to switch a silent gene back on in a precise and long-lasting way. The hope is that this could lead to a safer, easier, and more effective treatment option for individuals living with AS.
Principle Investigator
Jon Ark, PhD
Dr. Jonathan Ark is the founder and CEO of Junipero Therapeutics, where he is working on a new class of genetic medicines based on induced proximity. He earned his PhD from Duke University, with training in gene therapy, delivery, and translational biotech, and his graduate work helped support the launch of Azalea Therapeutics. Before starting Junipero, he worked across venture and biotech settings, which gave him experience in both early science and company building. He cares deeply about turning strong science into treatments that can help patients.
