Sangamo Therapeutics Announces Presentations At 2019 Annual Meeting Of The American Society Of Gene & Cell Therapy
Abstracts accepted for presentation highlight data from the Company's gene therapy program in Fabry disease; ex vivo gene-edited cell therapy programs in hemoglobinopathies being developed in partnership with
"Sangamo's strong scientific presence at ASGCT demonstrates the breadth of our therapeutic pipeline and our expertise in innovative genomic medicines research and development," said
ASGCT Annual Meeting presentations
Gene Therapy
- Liver-Targeted AAV Gene Therapy Vectors Produced at Clinical Scale Result in High, Continuous Therapeutic Levels of α-GalA Enzyme Activity and Effective Substrate Reduction in a Mouse Model of Fabry Disease – Abstract #794
Session: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases II
Poster Presentation –Wednesday, May 1 ;5:00PM ET
Ex Vivo Gene-Edited Cell Therapy
- Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer in Human Hematopoietic Stem and Progenitor Cells Results in Enriched Bialleleic Editing with Highly Replicable and Precise On-Target Small Indels and Allele-Additive Increases in Fetal Hemoglobin – Abstract #972
Session: Gene Editing for Red Blood Cell Disorders
Oral Presentation –Thursday, May 2 ;11:00AM ET
Zinc Finger Nuclease Engineering
- Optimizing Nuclease Specificity via Catalytic Domain Engineering Enables Complete Gene Modification with No Detectable Off-Targets – Abstract #133
Session: Measuring and Mitigating Genotoxicity of Genome Editing
Oral Presentation –Monday, April 29 ;4:00PM ET
Gene Regulation
- Evaluation of Programmable Zinc Finger Protein Transcription Factors for the Efficient Reduction of Tau in the Nonhuman Primate Brain – Abstract #62
Session: Gene Silencing Approaches
Oral Presentation –Monday, April 29 ;10:45AM ET - Repression of mHTT Expression in Huntington's Disease Mouse Models by AAV-Mediated Expression of Zinc-Finger Protein-Repressor Transgene – Abstract #63
Session: Gene Silencing Approaches
Oral Presentation –Monday, April 29 ;11:00AM ET - Selective Repression of C90RF72 Repeat Expansion-Containing Transcripts for the Treatment of ALS – Abstract #555
Session: Neurologic Diseases II
Poster Presentation –Tuesday, April 30 ;5:00PM ET
Invited Presentations at Scientific Symposia
Sangamo's research and development team and clinical collaborators have additionally been invited to participate in a variety of special workshops and scientific symposia. Dr.
- Therapeutic Genome Editing and Gene Regulation Using Designed Zinc Finger Proteins –
Ed Rebar , Ph.D.,Sangamo Therapeutics
Session:Gene Editing Workshop
Invited Talk –Sunday, April 28 ;5:00PM ET - Clinical Trials of Genome-editing (ZFN) for MPS I and MPS II –
Paul Harmatz , M.D., UCSF Benioff Children's Hospital Oakland
Session: Novel Strategies for Lung and GI Tract-directed Genetic Therapies – Organized by the Respiratory & GI Tract Gene & Cell Therapy Committee
Invited Talk –Monday, April 29 ;9:00AM ET - Gene Modification of Hematopoietic Stem Cells for Research and Treatment of Hemoglobinopathies –
Ed Rebar , Ph.D.,Sangamo Therapeutics
Session: Genetic Modification of Stem Cells – Organized by the Stem Cell Committee
Invited Talk –Wednesday, May 1 ;9:30 AM ET - IRB and Patient Safety 5 Things You Must Know to Start a Human Gene Therapy Trial –
Sagar Vaidya , M.D., Ph.D.,Sangamo Therapeutics
Session: Getting to theFinish Line : Market Success – Organized by the Clinical Trials and Regulatory Affairs Committee
Invited Talk –Thursday, May 2 ;8:30AM ET
All abstracts for the ASGCT Annual Meeting are available online at 2019 ASGCT Annual Meeting Abstracts.
About Sangamo Therapeutics
Forward-Looking Statements
This press release contains forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references relating to presentation of data from various therapeutic and research programs and the potential of these programs to transform the lives of patients. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the dependence on third parties for clinical trial supply and conduct of clinical trials, the lengthy and uncertain regulatory approval process, uncertainties related to the timing of initiation, enrollment and completion of clinical trials, and whether clinical trial results will validate and support the safety and efficacy of our product candidates. There can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable therapeutics. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K and its Current Report on Form 8-K as filed with the
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SOURCE
Investor Relations - United States, McDavid Stilwell, 510-970-6000, x219, mstilwell@sangamo.com; Varant Shirvanian, 510-970-6000, x205, vshirvanian@sangamo.com; Media Inquiries - United States, Aron Feingold, 510-970-6000, x421, afeingold@sangamo.com, Investor Relations and Media Inquiries - European Union, Caroline Courme, 33 4 97 21 27 27, ccourme@sangamo.com