According to a story from finanznachrichten.de, the biopharmaceutical company Abeona Therapeutics, Inc. recently announced that its experimental medical product ABO-101 has earned Fast Track designation from the US Food and Drug Administration (FDA). This therapy is in development as a treatment for mucopolysaccharidosis IIIB, also known as Sanfilippo syndrome. ABO-101 is a single use AAV mediated gene therapy.
About Sanfilippo Syndrome
Sanfilippo syndrome, which is also known as mucopolysaccharidosis III, is rare genetic lysosomal storage disease. It is linked to a deficiency in the enzyme responsible for the breakdown of heparan sulfate. There are four different types of Sanfilippo syndrome and each one is caused by a different genetic mutation. In type A, the mutation affects the SGSH gene. The mutation caused is the only defining characteristic of the different types, which otherwise present similarly. Symptoms include behavioral abnormalities, dementia, sleep disturbances, difficulty speaking, developmental delays, deafness, and loss of movement. There are currently no disease modifying therapies available for this disorder. However, bone marrow replacement can be useful if implemented early. Most patients do not survive beyond their teenage years, but some can survive into their 30s. To learn more about Sanfilippo syndrome, click here.
About Fast Track Designation
Fast Track designation is a program offered by the FDA that is intended to help accelerate the review of experimental treatments that have the potential to fulfill currently unmet medical needs in serious, life threatening medical conditions and diseases. When a product earns Fast Track designation it will benefit from more frequent interactions and collaboration between the developer and agency officials. The product may also be eligible for accelerated approval and priority review if other criteria are satisfied. ABO-101 has also earned Orphan Drug designation.
ABO-101 is a single use gene therapy that has the potential to revolutionize Sanfilippo syndrome treatment. This experimental therapy is intended to deliver functional copy of the affected gene, which in type B is the NAGLU gene. This replacement should theoretically allow the patient to produce the enzyme that is normally deficient in those with the disease. Future progress of trials with this therapy should be watched closely, as success would make this therapy a game changer for Sanfilippo syndrome type B.