Nobelpharma Presents Preclinical Results of Sanfilippo Syndrome Type A Treatment at Global Symposium
Rare drug specialist bio-venture Novelpharma announced on the 13th that it presented preclinical results of the Sanfilippo Syndrome Type A (MPS IIIA) treatment, co-developed with GC Green Cross, at the WORLDSymposium 2024.
WORLDSymposium 2024 is an international forum where medical professionals and experts in lysosomal diseases from around the world gather to share the latest treatments and clinical status related to lysosomal storage diseases (LSD). This year, it was held in San Diego, USA, from the 4th to the 9th (local time).
At this symposium, Dongkyu Jin, founder and Chief Scientific Officer (CSO) of Novelpharma and emeritus professor at Sungkyunkwan University School of Medicine, presented a poster on the preclinical results of NP3011 (substance name GC1130A), an MPS IIIA treatment currently under development. The presentation detailed the application of intracerebroventricular (ICV) recombinant human heparan N-sulfatase enzyme replacement therapy in a mouse disease model, confirming the reduction effect of heparan sulfate and improvement in brain lesions.
MPS IIIA is an autosomal recessive genetic disorder caused by gene defects that lead to the accumulation of heparan sulfate in the body, with severe brain damage as the main symptom. It is a severe rare disease where most patients die around the age of 15, and there is currently no approved treatment. Novelpharma, in collaboration with GC Green Cross, is developing an enzyme replacement therapy (ERT) by directly administering heparan N-sulfatase enzyme, which is not expressed in patients’ bodies, into the brain ventricles (ICV) to treat brain lesions in MPS IIIA patients.
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A Novelpharma official stated, "There is currently no developed treatment for Sanfilippo Syndrome Type A, so the unmet medical needs of patients are very high," adding, "We will accelerate the clinical development of NP3011, co-developed with GC Green Cross, to open the path for treating patients with Sanfilippo Syndrome Type A."
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