"Sangamo Therapeutics" from_date:2012

as aconsultant for BioMarin Pharmaceutical Inc. D.D.K. hasserved as a consultant for Sangamo Therapeutics, Inc;Sanofi; Amicus Therapeutics; Takeda; and Vertex Pharma-ceuticals; has received grant support from Viking Thera-peutics, Genzyme Sanofi, Roivant Rare Diseases, andAmicus Therapeutics; and has equity in Askbio, which isdeveloping gene therapy for Pompe disease. All other au-thors declare no conflicts

and Uniqure. Steven Pipe, MD, Pediatric Medical Director, Hemophilia and Coagulation Disorders Program, University of Michigan Dr. Steven Pipe has received consulting fees from Apcintex, Bayer, BioMarin, Catalyst Biosciences, CSL Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sangamo Therapeutics, Sanofi, Takeda, Spark Therapeutics, uniQure. Margaret Ragni, MD, MPH, Professor

to access all of BMJ Best PracticeTreatment algorithmINITIALlow riskintermediate riskhigh riskACUTElaboratory or clinical TLSLog in or subscribe to access all of BMJ Best PracticeContributorsVIEW ALLAuthorsTariq Mughal, MD, FRCP, FACP, FRCPathClinical Professor of MedicineTufts University Cancer CenterBostonMADisclosuresTM has consulted for Roche Foundation Medicine, Sangamo Therapeutics, Stemline

), the National Institute of Mental Health (NIMH), and the National Institute of Neurological Disorders and Stroke (NINDS). Sangamo Therapeutics also provided funding for these studies.

, neurological, and physical symptoms; however, some animals have developed unexplained liver tumors. If safety issues can be resolved, gene therapy may provide an alternative human treatment for MPS disorders in the future.[11]Sangamo Therapeutics, headquartered in Richmond, California, is currently conducting a clinical trial involving gene editing using Zinc Finger Nuclease (ZFN) for the treatment of MPS I

therapy[edit]In February 2019, medical scientists working with Sangamo Therapeutics, headquartered in Richmond, California, announced the first "in body" human gene editing therapy to permanently alter DNA - in a patient with MPS II.[9] Clinical trials by Sangamo involving gene editing using zinc finger nuclease are ongoing as of February 2019.[10]Prognosis[edit]Earlier onset of symptoms is linked

our disclaimer for details. ClinicalTrials.gov Identifier: NCT02225665 Recruitment Status : Completed First Posted : August 26, 2014 Results First Posted : February 9, 2021 Last Update Posted : March 19, 2021 Sponsor: Sangamo Therapeutics Information provided by (Responsible Party): Sangamo Austin, Texas, United States, 78705 Dallas, Texas, United States, 75246 Sponsors and Collaborators Sangamo Therapeutics Study Documents (Full-Text) Documents provided by Sangamo Therapeutics: Study Protocol: Protocol Amendment 2 Signature page[PDF] October 15, 2014 Study Protocol: Protocol Amendment 2[PDF] October 10, 2014 Statistical Analysis Plan[PDF] December 21

sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. ClinicalTrials.gov Identifier: NCT03432364 Recruitment Status : Active, not recruiting First Posted : February 14, 2018 Last Update Posted : July 7, 2022 Sponsor: Sangamo Therapeutics Collaborator: Sanofi Information provided by (Responsible Party): Sangamo Therapeutics * Study Details * Tabular View * No Results Posted * Disclaimer * How to Read a Study RecordStudy DescriptionGo to Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information Brief Summary: This is a single-arm, multi-site, single-dose

disclaimer for details. ClinicalTrials.gov Identifier: NCT01543152 Recruitment Status : Completed First Posted : March 2, 2012 Results First Posted : April 21, 2021 Last Update Posted : May 24, 2021 Sponsor: Sangamo Therapeutics Information provided by (Responsible Party): Sangamo Therapeutics * Study Dallas, Texas, United States, 75246 Gordon Crofoot, MD, PA Houston, Texas, United States, 77098 Puerto Rico Clinical Research Puerto Rico San Juan, Puerto Rico, 00909 Sponsors and Collaborators Sangamo Therapeutics Study Documents (Full-Text

not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. ClinicalTrials.gov Identifier: NCT02695160 Recruitment Status : Terminated First Posted : March 1, 2016 Last Update Posted : March 23, 2022 Sponsor: Sangamo Therapeutics Information provided by (Responsible Party): Sangamo Therapeutics * Study , 20007 Sponsors and Collaborators Sangamo Therapeutics Investigators Study Director: Medical Monitor Sangamo Therapeutics, Inc. More InformationGo to Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information Responsible Party: Sangamo Therapeutics

-Term Follow-up Study IVPRP-LT01 (NCT04628871)) First Posted : March 8, 2016 Last Update Posted : December 16, 2021 Sponsor: Sangamo Therapeutics Information provided by (Responsible Party): Sangamo Therapeutics * Study Details * Tabular View * No Results Posted * Disclaimer * How to Read a Study RecordStudy DescriptionGo to Top of Page Study Description Study Design Arms and Interventions and Collaborators Sangamo Therapeutics Investigators Study Director: Medical Monitor Sangamo Therapeutics More InformationGo to Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number): Ou L, Przybilla MJ, Ahlat O, Kim S

Follow-up Study IVPRP-LT01 [NCT04628871]) First Posted : February 2, 2017 Last Update Posted : June 8, 2021 Sponsor: Sangamo Therapeutics Information provided by (Responsible Party): Sangamo Therapeutics * Study Details * Tabular View * Results Submitted * Disclaimer * How to Read a Study RecordStudy DescriptionGo to Top of Page Study Description Study Design Arms and Interventions Outcome University of North Carolina Chapel Hill, North Carolina, United States, 27514 United States, Ohio Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States, 45229 Sponsors and Collaborators Sangamo Therapeutics Investigators Study Director: Medical Monitor Sangamo Therapeutics