According to a story from publicnow.com, the drug development company Sarepta Therapeutics recently announced that is has completed negotiations for a licensing agreement with Lysogene, a biopharmaceutical company. This agreement is related to an experimental gene therapy called LYS-SAF302, which is in development to treat the rare genetic disorder Sanfilippo syndrome type A. This arrangement allows Sarepta commercial rights to the drug in the US and outside of Europe, while Lysogene will retain commercial rights in Europe.
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 LYS-SAF302
LYS-SAF302 could potentially become the first treatment that could rectify the serious neurological manifestations that occur with Sanfilippo syndrome. Common treatments like enzyme replacement therapy are of limited utility because they are unable to pass the blood-brain barrier, which means that developmental delays and dementia cannot be addressed. The next clinical trial which will test the drug’s effectiveness is slated to begin before the end of 2018.
LYS-SAF302 is a gene therapy which functions by replacing the SGSH gene, which is mutated in patients with the syndrome, with a normal copy. It is classified as an AAV mediated gene therapy, which means that it uses a virus vector in order to deliver the genetic material. Proof-of-concept trials have established that this therapy is capable of producing the deficient enzyme and it also does not appear to cause serious adverse events.
