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Mucopolysaccharidosis Type I Treatment Receives Orphan Drug Designation

RDR Staff
Published Online: Wednesday, Jan 11, 2017
Another orphan drug receives Orphan Drug Designation so far this year. Sangamo Therapeutics’ genome editing product SB-318, for the treatment of the rare lysosomal storage disease Mucopolysaccharidosis Type I (MPS I), was designated. 
 
MPS I is a rare neurodegenerative disease caused by deficiency of the a-l-iduronidase (IDUA) gene. Over 1,000 individuals with MPS I are estimated to be born each year worldwide. Symptoms include excessive accumulation of fluid in the brain, spinal cord compression and cognitive impairment.
 
With genome editing technology, SB-318 is designed as a single treatment strategy intended to provide stable, continuous production of the IDUA enzyme for the lifetime of the patient.
 
This year, SB-318 will most likely be tested in vivo genome editing Phase 1/2 clinical trial involving pediatric patients. 
 
Sangamo’s in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic site that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein product.
 
SB-318 will also be tested in clinical trials for the treatment of MPS II and hemophilia B.

What is orphan designation?

According to the FDA, the Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

 
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