ARSA Human, SF9

The recombinant human Arylsulfatase A (ARSA) is produced in Sf9 Baculovirus cells. The enzyme is a single, glycosylated polypeptide chain containing 498 amino acids, with a molecular mass of approximately 53.0 kDa . The recombinant ARSA is expressed with a 9 amino acid His tag at the C-terminus and is purified using proprietary chromatographic techniques .

The ARSA protein solution is formulated in Phosphate Buffered Saline (pH 7.4) with 10% glycerol . It is a sterile, filtered clear solution with a purity greater than 95.0% as determined by SDS-PAGE . The specific activity of ARSA is greater than 2,500 pmol/min/µg, defined as the amount of enzyme that hydrolyzes 4-Nitrocatechol at pH 5.0 at 37°C .

For short-term storage (2-4 weeks), ARSA should be kept at 4°C. For long-term storage, it is recommended to store the enzyme at -20°C with the addition of a carrier protein (0.1% HSA or BSA) to prevent multiple freeze-thaw cycles .

ARSA plays a crucial role in the degradation of sulfatides, which are essential for maintaining the normal function of the nervous system. Deficiency in ARSA activity leads to the accumulation of sulfatides, causing a lysosomal storage disorder known as Metachromatic Leukodystrophy (MLD) . Recombinant human ARSA is in clinical development for the treatment of patients with MLD .

Nonclinical comparability studies have been conducted to address manufacturing process changes in recombinant human ARSA. These studies have shown that changes in the manufacturing process did not affect the pharmacodynamic, pharmacokinetic, or safety profiles of the enzyme . Additionally, ARSA has been studied as a genetic modifier in Parkinson’s disease, indicating its potential role in neurodegenerative diseases .