A gene therapy being developed as a treatment for Sanfilippo syndrome has just been awarded Regenerative Medicine Advanced Therapy (RMAT) status in the US, reports GlobalGenes.
Sanfilippo syndrome is a rare genetic condition that causes brain damage. Approximately 1 out of every 70,000 children born will be affected by it. The disease is also known as MPS III, and it occurs in four different forms, known as types A, B, C, and D. Of these, A and B are the most common. Each form is linked to a different genetic change that prevents a specific enzyme from being effectively produced. The enzymes affected would normally be involved in removing certain waste substances from cells, but the genetic change means that they are unable to do this efficiently and as a result substances build up to toxic levels in the body. This usually occurs in the central nervous system, and in particular the brain and spine, which results in progressive cognitive and physical disability.
There is currently no cure for Sanfilippo syndrome, but researchers are hoping that new gene therapy technology might lead to a breakthrough. The treatment that the U.S. Food and Drug Authority (FDA) has just given Regenerative Medicine Advanced Therapy status to is a gene therapy being developed to treat the disease. The therapy is made by Abeona Therapeutics and is called ABO-102. This is the first ever gene therapy to be awarded Regenerative Medicine status after the FDA expanded the scope of the designation to include gene therapy treatments in November 2017. The new status of ABO-102 will speed up the development process by increasing collaboration between the developers and FDA, and making the therapy a priority medicine for FDA review. The designation is awarded if a treatment is thought to have the potential to meet an unmet need for a life-threatening disease.