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tralesinidase alfa
- Development stage
- Phase 4
- Lead developer
- Spruce Biosciences
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intracerebroventricular
01
Overview
Tralesinidase alfa is a chimeric fusion protein comprised of recombinant human alpha-N-acetylglucosaminidase (NAGLU) and a modified human insulin-like growth factor 2. It is being developed as an enzyme replacement therapy (ERT) for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo syndrome type B, a rare lysosomal storage disorder caused by NAGLU deficiency. The drug is administered via intracerebroventricular (ICV) infusion to bypass the blood-brain barrier and deliver the enzyme directly to the central nervous system. Tralesinidase alfa has demonstrated efficacy in normalizing heparan sulfate levels in cerebrospinal fluid and plasma, reducing disease-associated glycosaminoglycan accumulation, stabilizing cortical gray matter volume, resolving hepatomegaly, and improving cognitive outcomes in preclinical models and clinical trials[1][5][6]. The therapy was originally developed by BioMarin but its development program was transitioned to Allievex[6].
- Brand names
- TA-ERT
- Other names
- tralesinidase alfarhNAGLU-IGF2rhNAGLU-IGF-2rhNAGLU-IGF 2
02
Targets
IGF2R (Cation-independent mannose-6-phosphate receptor)NAGLU (Alpha-N-acetylglucosaminidase)
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