Tim Miller, Ph.D. is co-Founder, President and CEO of Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases.             


BioEnterprise: What does Abeona Therapeutics do?

Tim: Our lead programs include gene therapies for Sanfilippo Syndromes type A and B, deadly genetic diseases that attack children between the ages of 3 and 6 years of age. We develop genetically-modified viruses to deliver the functioning version of the defective gene that caused the child’s metabolism to go awry.

Sanfilippo kids can’t break down certain kinds of sugar. Over time, these sugars build up in their cells resulting in a cruel neurological and cognitive decline, where 70% don’t reach age 18 – and there’s currently no approved therapy.

BioEnterprise: What is unique about Abeona?

Tim: Abeona is a rare disease company developing gene therapies that are delivered as a one-time injection rather than surgical interventions.

Abeona was formed in 2013 through a venture philanthropy model: the company was founded and funded by a dozen international foundations that, collectively, represent the global Sanfilippo community. Their support came with a directive to find and accelerate the development and global access of the most promising therapies for Sanfilippo Syndrome (Types A and B).

Essentially, we are explorers developing something no one else has tried to do, and our role is to help bridge the gap between the scientific and the clinical to develop products that can be manufactured large-scale and quickly.

BioEnterprise: What have been your recent company milestones?

Tim:  Last November, we raised $42 million in a common stock offering. We recently received FDA Breakthrough Therapy designation for EB-101, the leading gene therapy for patients with Recessive Dystrophic Epidermolysis Bullosa, and are initiating a pivotal Phase 3 trial to advance our therapy for patients with this debilitating disease.

And I am proud to share that we’ve created the Elisa Linton Center for Rare Disease Therapies. This Cleveland-based manufacturing facility will be a global resource for production of gene therapies with the potential to bring new treatments to rare disease patients around the world.

BioEnterprise: What does “Abeona” mean?

Tim: Abeona is named after the Roman goddess who protected children as they took their first steps away from home.

BioEnterprise: Why are you doing this work?

Tim: Time is the enemy for kids with rare genetic diseases such as Sanfilippo Syndrome. The disease usually presents between ages three and six, and the children spend the rest of their lives regressing. Even something as small as a fever can cause the loss of the power of speech – forever.

We believe in hope. When we meet the families and see how desperate they are, and what they are willing to do for their children, we gain an understanding of real courage. It is the reason for us to get up in the morning – to help accelerate development of these promising treatments.

We may be working on the only therapy they will ever get.