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Olenasufligene relduparvovec
- Development stage
- Discontinued
- Lead developer
- Lysogene
- Modality
- Gene Therapies
- Administration
- Intracerebral
01
Overview
Olenasufligene relduparvovec is an investigational gene therapy developed for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A). It uses a recombinant adeno-associated virus serotype rh.10 (AAVrh.10) vector to deliver a functional copy of the human N-sulfoglucosamine sulfohydrolase (SGSH) gene directly into the brain via intracerebral injection. The therapy aims to restore SGSH enzyme activity, which is deficient in patients with MPS IIIA, thereby reducing pathological accumulation of heparan sulfate and related neurodegeneration. Olenasufligene relduparvovec has demonstrated biological activity in clinical trials by reducing disease biomarkers in cerebrospinal fluid and serum[1][3][5].
- Brand names
- LYS-SAF302LYS-SAF-302LYS-SAF 302
- Other names
- dna (recombinant adeno-associated virus 2 vector lys-saf302 human n-sulfoglucosamine sulfohydrolase cdna-specifying)UNII-7DU50HR4GBUNII7DU50HR4GBUNII 7DU50HR4GB
02
Targets
HS (Heparan sulfate)
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