Le Lézard
Classified in: Health
Subject: TRI

Sangamo Announces European Medicines Agency Recommendation Of Orphan Medicinal Product Designation For Investigational Genome Editing Treatments For MPS I And MPS II



RICHMOND, Calif., Dec. 7, 2017 /PRNewswire/ -- Sangamo Therapeutics, Inc. (NASDAQ: SGMO) announced today that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has issued a positive opinion on the application for orphan medicinal product designation (OMPD) for SB-318 and SB-913, Sangamo's genome editing product candidates for the treatment of rare lysosomal storage disorders Mucopolysaccharidosis Type I (MPS I) and MPS II, respectively. 

Sangamo Therapeutics, Inc. (PRNewsFoto/Sangamo BioSciences, Inc.) (PRNewsFoto/)

The EMA's OMPD is granted to medicines intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating conditions that are rare and affect less than five in 10,000 persons in the European Union (EU). The designation provides incentives to advance the development and commercialization of orphan medicines, which include access to the EU centralized authorization procedure and potential for market exclusivity for a period of up to ten years.

MPS I and MPS II are caused by mutations in the genes encoding alpha-L-iduronidase (IDUA) and iduronate 2-sulfatase (IDS) enzymes, respectively. Using Sangamo's zinc finger nuclease (ZFN) genome editing technology, SB-318 (for MPS I) and SB-913 (for MPS II) are designed as a single treatment strategy intended to provide stable, continuous production of the IDUA or IDS enzyme for the lifetime of the patient.

SB-318 and SB-913 have already received Orphan Drug, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). The Phase 1/2 clinical trials for these programs, evaluating SB-318 and SB-913 in adults with MPS I and MPS II, respectively, are open and enrolling subjects.

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. The design of these programs is ultimately to target a population that includes 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 conducting Phase 1/2 clinical trials in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has an exclusive, global collaboration and license agreement with Pfizer Inc. for gene therapy programs for Hemophilia A, 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 www.sangamo.com.

 

SOURCE Sangamo Therapeutics, Inc.


These press releases may also interest you

at 14:00
The "Global Reusable Icepacks Market Size, Market Share, Application Analysis, Regional Outlook, Growth Trends, Key Players, Competitive Strategies and Forecasts, 2018 To 2026" report has been added to ResearchAndMarkets.com's offering. The global...

at 14:00
"The tube of my CPAP machine gets cold overnight which is uncomfortable, and then I cannot sleep," said an inventor from Pa. "I came up with this idea to ensure that the tube stays insulated so that it does not keep me awake at night."...

at 14:00
SUNRISE, Fla., Sept. 18, 2018 /PRNewswire-PRWeb/ -- Interim HealthCare Inc., a leading national franchisor of home care, hospice and healthcare staffing, was named to Staffing Industry Analysts (SIA) 2018 Largest Healthcare Staffing Firms and Largest...

at 14:00
WASHINGTON, Sept. 18, 2018 /PRNewswire-PRWeb/ -- In August, Hingez Therapeutics Inc. received a grant totaling more than 1.8 million from the National Institutes of Health (NIH) to develop oral drugs to treat high cholesterol, with the goal of...

at 13:58
September is Pain Awareness Month and Omron Healthcare is encouraging those seeking effective drug-free pain relief to explore TENS (transcutaneous electrical nerve stimulation), a trusted pain therapy regularly administered by physical therapists...

at 13:57
We continue to see an unprecedented number of opioid-related overdoses in Canada. The loss of life is tremendous and this national public health crisis continues to devastate the health and lives of many Canadians, their families and their...




News published on 7 december 2017 at 16:22 and distributed by: