Friday , May 25 2018
Home / FDA News / Sangamo Therapeutics Receives Rare Pediatric Disease Designation from the FDA for its MPS I Candidate

Sangamo Therapeutics Receives Rare Pediatric Disease Designation from the FDA for its MPS I Candidate

RICHMOND, Calif., Feb. 27, 2017 /PRNewswire/ — Sangamo Therapeutics, Inc. (Nasdaq: SGMO), the leader in therapeutic genome editing, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for SB-318, the Company’s in vivo genome editing product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I), a lysosomal storage disorder.

Rare pediatric disease designation is granted to drugs and biologics intended to treat orphan diseases affecting less than 200,000 patients in the U.S., primarily age 18 years or younger. The designation provides incentives to advance the development of rare disease drugs, including access to the FDA’s expedited review and approval programs. In addition, under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval for a new drug application or biologics license application for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the sponsor or sold or transferred.

“MPS I is a progressive and potentially fatal inherited disease with limited treatment options,” said Sangamo Chief Executive Officer Sandy Macrae, M.B., Ch.B., Ph.D. “New treatments for MPS I are needed, and we are pleased that the FDA has granted rare pediatric disease designation to SB-318, an experimental genome editing treatment designed to correct for the genetic mutation that causes the disease.”

MPS I is caused by a mutation in the gene encoding the alpha-L-iduronidase (IDUA) enzyme. SB-318 is designed as a single treatment strategy, based on Sangamo’s zinc finger nuclease (ZFN) genome editing technology and intended to provide stable, continuous production of the IDUA enzyme for the lifetime of the patient. In 2017, Sangamo is conducting a Phase 1/2 clinical trial evaluating SB-318 for MPS I.

The FDA previously granted SB-318 with orphan drug designation (ODD). Sangamo has also submitted applications to the FDA for ODD and rare pediatric disease designation for SB-913, the Company’s in vivo genome editing program for MPS II, another rare lysosomal storage disorder, being evaluated in a Phase 1/2 clinical trial in 2017.


Sangamo’s In Vivo Genome Editing Approach¬†

Sangamo’s ZFN-mediated in vivo genome editing approach makes use of the endogenous albumin gene locus, a highly expressing and liver-specific 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. The ability to permanently integrate the therapeutic gene in a highly specific, targeted fashion significantly differentiates Sangamo’s in vivo genome editing approach from conventional AAV cDNA gene therapy. Ultimately, the target population for these programs will include pediatric patients, and it will be important in this population to be able to produce stable levels of therapeutic protein for the lifetime of the patient.

About Sangamo Therapeutics 

Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients’ lives using the company’s industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is advancing Phase 1/2 clinical programs in hemophilia A and hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has a strategic collaboration with Bioverativ, Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington’s disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company’s website at

Check Also

FDA Grants Priority Review for Prophylactic Pediatric Use of Coagadex for Treatment of Hereditary Factor X Deficiency

ELSTREE, England and DURHAM, N.C., May 23, 2018 /PRNewswire/ — Bio Products Laboratory Limited (BPL), …