A Gene Therapy Being Developed for Sanfilippo Syndrome Has Been Granted RMAT status in the US.

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.

The gene therapy, ABO-102, is currently undergoing a phase 1/2 trial in eleven patients with Sanfilippo syndrome. They have been given an injection of the medicine that will deliver a working form of the gene they are missing. It is hoped that the newly introduced gene will then fulfil the role of the non-functional gene by producing the missing enzyme, which will help to remove toxic build-ups and improve the disease symptoms. The patients undergoing the therapy are being followed up to see how their bodies are reacting to the therapy. More details about the outcome of the study are expected to be announced next month.
Receiving the Regenerative Medicine Advanced Therapy designation in the US is an encouraging sign for the researchers behind ABO-102. The treatment had previously received Rare Pediatric Disease Designation, Orphan Drug Designation, and Fast Track Designation in the United States, and Orphan Drug Designation from the European Union.

Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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