Spruce Biosciences Introduces Breakthrough TA-ERT for the Treatment of Sanfilippo Syndrome Type B
Spruce Biosciences has introduced Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) as an investigational therapy that has received FDA Breakthrough Therapy Designation for the treatment of Sanfilippo Syndrome Type B (MPS IIIB), a rare genetic disorder causing progressive neurodegeneration in children.
TA-ERT is a fusion protein comprising recombinant human alpha-N-acetylglucosaminidase (rhNAGLU), designed to restore enzyme activity in the central nervous system. The therapy is engineered to target lysosomes effectively, promoting clearance of accumulated heparan sulfate and preserving neuronal health, with the aim of slowing or halting neurological decline in patients with MPS IIIB.
The FDA Breakthrough Therapy Designation recognises the potential of TA-ERT to deliver meaningful clinical benefits for patients with this ultra-rare condition. The therapy is the first disease-modifying approach under investigation for Sanfilippo Syndrome Type B and is being evaluated in multiple clinical studies, which have shown encouraging results in safety, enzyme activity restoration, and impact on neurological function.
Sanfilippo Syndrome Type B (MPS IIIB) is a severe and fatal lysosomal storage disorder caused by deficiency of NAGLU enzyme. The disease leads to progressive brain atrophy, cognitive and motor impairment, hyperactivity, delayed speech, hearing loss, and other somatic symptoms. Life expectancy is typically between 15 and 19 years, and current management is limited to palliative care.
TA-ERT is now the first investigational therapy to receive U.S. FDA Breakthrough Therapy designation for MPS IIIB, representing a significant advancement in treatment for children affected by this devastating disorder.
