Meet Tim Miller, founder of Abeona Therapeutics LLC
, which develops gene therapy for potential cures for children with rare genetic diseases. Abeona’s lead therapies target the deadly childhood diseases Sanfilippo (SF) Syndrome Type A and Type B. Abeona’s two lead products, ABX-A and ABX-B, deliver the therapies to the central nervous system with the aim of reversing the effects of the genetic errors that cause the disease.
In a nutshell, what is Sanfilippo Syndrome and who does it affect?
Sanfilippo Syndrome is a rare, devastating lysosomal storage disease, caused by a single gene defect that results in cells being unable to degrade certain sugars. Infants with Sanfilippo appear normal at birth. However, the disease is relentlessly progressive with progressive deterioration of social and adaptive abilities, high mortality and premature death. There is no treatment currently available for the disease.
How did you become interested in lysosomal storage diseases and Sanfilippo disease?
I’ve worked in startup biotechnology companies that have focused on novel regenerative medicines aimed at improving patient quality of life for the past few years.
I heard about the research being conducted on Sanfilippo syndrome and the devastating toll it takes on the children and families born with the disease. I found their stories very inspiring, and wanted to help develop new therapies to give these kids a chance at growing up. After seeing the promising initial data, and speaking to the foundations and researchers leading the charge, my co-founder and I formed Abeona to help bring these therapies into clinical trials.
Why is Cleveland an ideal location for Abeona Therapeutics’ research?
Cleveland has become a focal point in the Midwest for new companies to develop exciting gene and stem cell therapies. The past few years have seen a half dozen companies emerge as potential leaders in their fields, and Ohio has been very supportive for helping fund startup biotechnology/pharmaceutical companies.
What is the Champions of Hope Award that Abeona Therapeutics received last July?
There are nearly 7,000 rare diseases, and 95 percent of them do not have a single FDA-approved drug treatment. The Champions of Hope award by the Global Genes Project
recognized Abeona and its collaborators for their work as patient advocates in the search for a cure for Sanfilippo Syndrome. It was truly an honor to be recognized so early as a startup, and the award strongly recognizes the potential inherent in the research of Abeona’s scientific founders.
What do you like most about being an entrepreneur in the biomedical field?
Working as an entrepreneur in biotechnology can be very challenging -- in one day you can experience both great success and devastating loss. But you’re at the forefront of clinical medicine, that point where science and medicine can be developed to help people suffering who do not have any alternative.