Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists and clinicians

Gene therapy is the delivery of DNA into a patient's cells to replace faulty or missing genes—or adds new genes—in an attempt to cure cancer or make changes so the body is better able to fight off disease. Scientists and clinicians have identified a number of different ways to do this, in an effort to correct malfunctioning or mutated genes. Many gene therapy clinical trials are ongoing to assess the safety and potential benefits in patients with rare diseases.

Researchers and clinicians at leading children's hospitals in Ohio believe that gene therapy is the key to addressing Sanfilippo (SF) Syndrome, a genetic metabolism disorder that prohibits the proper breakdown of the body's sugar molecules in a cell organelle called the lysosome. There are 4 types of MPS III (MPS III A, MPS III B, MPS III C, and MPS III D), each with a deficiency in one of four lysosomal enzymes. The disease affects the central nervous system, causing severe brain damage, and typically results in hearing loss, vision loss, organ damage, bone deformities, and eventual death. There is currently no approved treatment for SF, and approximately 70% of the children with the disease do not reach their 18th birthday.

Leading new treatments against SF is Abeona Therapeutics, a Cleveland-based biotechnology start-up created in March 2013, with the goal of developing therapies for patients with the rare Lysosomal Storage Disorder (LSD), Sanfilippo (SF) Syndrome or Mucopolysaccharidosis III (MPS III). Abeona Therapeutics, with financial support from multiple international patient advocacy groups, are collaborating to develop 2 gene therapy products, ABX-A and ABX-B, for children with SF Types A and B. Both products deliver the therapeutic product by intravenous injection, and preclinical studies have demonstrated robust delivery to the central nervous system and peripheral organs with the aim of replacing the malfunctioning gene to alleviate the underlying effects of the genetic errors that cause SF.

Abeona's CEO, Dr. Tim Miller can discuss the following:
•What causes Sanfilippo syndrome?
•What happens to children with Sanfilippo syndrome?
•What treatments are available?
•What is gene therapy?
•What types of gene therapy are there?
•How are gene therapies delivered to patients?
•How are Abeona's gene therapy programs advancing treatments for these diseases?
•How are Abeona's gene therapy programs different from other therapies in development?
•What are the next steps in developing these programs?