MPS II Trial Recruiting - NCT03041324

According to the Clinicaltrials.gov website Sangamo's MPS II clinical trial is recruiting.
https://clinicaltrials.gov/ct2/show/NCT03041324?term=sangamo&rank=1

ASGCT 2017 PR

RICHMOND, Calif., April 24, 2017 /PRNewswire/ -- Sangamo Therapeutics, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced that data from the Company's therapeutic and research programs will be presented at the 20^th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) to be held in Washington, D.C. from May 10-13, 2017.

Sangamo scientists or collaborators will deliver ten oral and nine poster presentations during the conference. These presentations will detail data from therapeutic and research programs for lysosomal storage disorders and other monogenic diseases, cancer immunotherapy, and central nervous system disorders, as well as advancements in genome editing technology and novel delivery modalities. Sangamo scientists and their collaborators have also been invited to participate in scientific symposia and educational sessions focused on clinical and research applications of genome editing.
"Sangamo once again has a very strong presence at ASGCT, with 19 oral and poster presentations," said Dr. Sandy Macrae, Sangamo's chief executive officer. "These data highlight the breadth of our clinical and early stage pipeline across genome editing, gene therapy, gene regulation and cell therapy. With our focus now on the translation of our groundbreaking science into new genomic therapies that transform patients' lives, our research and technology programs will continue to provide new assets for therapeutic development."
The following presentations are scheduled at the ASGCT Meeting sessions:
Invited Presentations at Scientific Symposia

• C-Suite Executive Panel: Sandy Macrae, M.B., Ch.B., Ph.D., Sangamo TherapeuticsSession: Commercialization WorkshopPanel Discussion – Tuesday, May 9; 3:15PM
• Preclinical Studies Evaluating Zinc Finger Nuclease-Driven Genome Editing – Michael C. Holmes, Ph.D., Sangamo TherapeuticsSession: Clinical Trials Training CourseInvited Talk – Tuesday, May 9; 9:50AM
• Educational Session Co-Chair: Thomas Wechsler, Ph.D., Sangamo TherapeuticsSession: 100. Getting Started in Genome Editing Panel Discussion – Wednesday, May 10; 8:00AM
• Scientific Symposium Co-Chair: Michael C. Holmes, Ph.D., Sangamo TherapeuticsSession: 204. Therapeutic Editing of the Human Genome and EpigenomePanel Discussion – Thursday, May 11; 8:00AM
• Scientific Symposium Co-Chair: Kathleen Meyer, M.P.H., Ph.D., D.A.B.T., Sangamo TherapeuticsSession: 300. Clinical Advancement of Gene Editing – Moving New Science to the ClinicPanel Discussion – Friday, May 12; 8:00AM

Lysosomal Storage Disorders

• Liver-Based Expression of the Human alpha-Galactosidase A Gene in a Murine Fabry Model Results in Continuous High, Therapeutic Levels of Enzyme Activity and Effective Substrate Reduction – Abstract #27Session: 113. Genome Editing and Integration Analysis in Metabolic and Endocrine DisordersOral Presentation – Wednesday, May 10; 10:45AM
• ZFN-Mediated In Vivo Genome Editing Results in Phenotypic Correction in MPS I and MPS II Mouse Models – Abstract #30Session: 113. Genome Editing and Integration Analysis in Metabolic and Endocrine DisordersOral Presentation – Wednesday, May 10; 11:30AM

Central Nervous System Disorders

• Sustained Tau Reduction via Zinc Finger Protein Transcription Factors as a Potential Next-Generation Therapy for Alzheimer's Disease and Other Tauopathies – Abstract #24Session: 112. Genome Editing: Transcriptional Regulation and SpecificityOral Presentation – Wednesday, May 10; 12:00PM

Monogenic and Infectious Diseases

• In Vivo ZFN-Mediated Editing of the Mutant SERPINA1 Gene Results in Spontaneous Liver Repopulation by the Gene-Edited Hepatocytes and Greatly Decreased Fibrosis in the PiZ Mouse Model of alpha-1 Antitrypsin Deficiency Liver Disease – Abstract #511Session: 341. In Vivo Gene EditingOral Presentation – Friday, May 12; 4:15PM
• Targeted Genome Editing of Recombination Activating Gene 1 to Potentially Treat Severe Combined Immunodeficiency – Abstract #654Session: Gene Targeting and Gene Correction IIIPoster Presentation – Friday, May 12; 5:45PM
• Evolution of HIV-1 Resistance to the Fusion Inhibitor CD34-CXCR4 and Potential Fitness Costs in Consideration of a Phase 1 Clinical Trial – Abstract #657Session: Hematologic & Immunologic Diseases IIIPoster Presentation – Friday, May 12; 5:45PM

Technology and Delivery Developments

• New Zinc Finger Nuclease Architectures for Highly Efficient Genome Engineering in Primary Cells at Large Scale with No Detectable Off-Target Effects – Abstract #23Session: 112. Genome Editing: Transcriptional Regulation and SpecificityOral Presentation – Wednesday, May 10; 11:45AM
• In Vivo Genome Editing via Non-Viral Delivery of Zinc Finger Nucleases Results in Supraphysiological Levels of Therapeutic Proteins in Adult Mice – Abstract #509Session: 341. In Vivo Gene EditingOral Presentation – Friday, May 12; 3:45PM
• Non-Viral Delivery of Zinc Finger Nucleases Enable Greater Than 90% Protein Knockdown of Multiple Therapeutic Gene Targets In Vivo – Abstract #510Session: 341. In Vivo Gene EditingOral Presentation – Friday, May 12; 4:00PM
• Ex Vivo Protein Replacement Using Homology Driven Genome Editing in Human B Cells by Combining Zinc Finger Nuclease mRNA and AAV6 Donor Delivery – Abstract #750Session: 412. Ex Vivo Gene EditingOral Presentation – Saturday, May 13; 11:30AM 
• A New, Reversed Zinc-Finger Nuclease Structure for High-Precision Therapeutic Genome Engineering – Abstract #170Session: Gene Targeting and Gene Correction IPoster Presentation – Wednesday, May 10; 5:30PM
• Improved In Vitro Assay to Assess Human Serum Neutralization of AAV Vectors Yields Cell Line-Dependent Results – Abstract #396Session: Immunological Aspects of Gene Therapy and Vaccines IIPoster Presentation – Thursday, May 11; 5:15PM
• Development of a Qualifiable MiSeq Assay for Precise and Accurate Quantitation of Small Insertions and Deletions (Indels) in the Human Genome Induced by Sequence-Specific Zinc Finger Nucleases – Abstract #644Session: Gene Targeting and Gene Correction IIIPoster Presentation – Friday, May 12; 5:45PM

Applications of Gene Editing in Stem Cells

• In Vivo Selection of Engineered Human CD34⁺ HSPCs Using Targeted Gene Integration – Abstract #512Session: 341. In Vivo Gene EditingOral Presentation – Friday, May 12; 4:30PM
• Correction of SCID-X1 by Targeted Genome Editing of Hematopoietic Stem/Progenitor Cells (HSPC) in a Humanized Mouse Model – Abstract #747Session: 412. Ex Vivo Gene EditingOral Presentation – Saturday, May 13; 10:45AM
• A Novel Gene Therapy Approach of Fanconi Anemia Hematopoietic Stem Cells Based on NHEJ-Mediated Gene Editing – Abstract #165Session: Gene Targeting and Gene Correction IPoster Presentation: Wednesday, May 10; 5:30PM
• Towards Clinical Translation of Hematopoietic Stem Cell Gene Editing for the Correction of SCID-X1 Mutations – Abstract #163Session: Gene Targeting and Gene Correction IPoster Presentation: Wednesday, May 10; 5:30PM
• HSPC Expansion Drugs Enhance Gene Editing Efficiency in Long Term Hematopoietic Stem Cells – Abstract #378Session: Gene Targeting and Gene Correction IIPoster presentation: Thursday, May 11; 5:15PM
• Molecular Evidence of Ex Vivo Genome Editing in a Mouse Model of Immunodeficiency – Abstract #656Session: Hematologic & Immunologic Diseases IIIPoster Presentation – Friday, May 12; 5:45PM

All abstracts for the ASGCT meeting are available online at 2017 ASGCT Annual Meeting Abstracts.
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 www.sangamo.com.

uniQure Shelves Glybera

uniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe

-- Marketing Authorization for Glybera® to Expire on October 25, 2017 --

-- Company Maintains Focus on Core Programs in Hemophilia B, Huntington's Disease and Congestive Heart Failure --

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 20, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will not pursue the renewal of the Glybera®(alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017.

"The decision to not pursue marketing authorization renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern," stated Matthew Kapusta, chief executive officer of uniQure.  "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."

Mr. Kapusta added, "In line with our previously announced strategy, we will focus our resources on advancing our hemophilia B program into a pivotal trial, moving our Huntington's disease program into a clinical proof-of-concept trial, and progressing our research and development collaboration with Bristol-Myers Squibb."   

In October 2012, the European Commission granted a five-year marketing authorization for Glybera under exceptional circumstances as a treatment for a small subset of patients with familial lipoprotein lipase deficiency (LPLD), an ultra-rare genetic disorder. As part of Glybera's approval, uniQure was required to establish a global registry for the long-term surveillance of patients, conduct a post-approval clinical study, submit for annual regulatory reassessments and implement additional risk management procedures.  All of these activities required a significant infrastructure for uniQure that included the Company bearing the full costs of maintaining commercial manufacturing capabilities, managing development and validation of numerous assays and supporting regulatory interactions and inspections.

uniQure has initiated discussions with the European Medicines Agency (EMA) to discuss steps to wind down these various activities and review plans for ongoing patient monitoring.

Under the terms of the agreement between uniQure and Chiesi Group, which has exclusive rights for the commercialization of Glybera in Europe and other selected countries, uniQure will continue to make product available to Chiesi to treat any patients that are approved for treatment prior to October 25, 2017, and will also be responsible for terminating the Phase IV post-approval study. 

As a result of the withdrawal of Glybera, uniQure expects to reduce future expenses related to the product by approximately $2 million annually, beginning in 2018 and net of any payments to Chiesi.  These cost savings will be in addition to those previously announced by the Company related to the consolidation of manufacturing into the Company's Lexington facility.  uniQure continues to expect its existing cash resources will be sufficient to fund operations into 2019.

UniQure - busy April

Sangamo....... not so much.

uniQure Announces Presentations at Upcoming April Conferences

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 03, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced presentations at the following conferences taking place in April:

World Orphan Drug Congress USA, April 19 - 21 2017, at the Washington Marriott Wardman Park in Washington D.C.

• Matt Kapusta, Chief Executive Officer, will be presenting a keynote address: Adeno-associated virus (AAV)-based gene therapies for rare, chronic and degenerative diseases, on Friday April 21st, 2017, 9.40 a.m. EDT.

12th Annual HD Therapeutics Conference by CHDI, April 24 - 27 2017, at the Westin Dragonara Resort in St. Julian's, Malta.

• A presentation new preclinical data on huntingtin gene silencing in a mini-pig model of Huntington's Disease, using allele-specific and non-selective miRNAs, will be presented during the CHDI conference.

Gene Therapy for Rare Disorders Conference, April 25 - 26 2017, at the Sheraton Boston Hotel, in Boston MA.

• Lance Weed, Vice President of US Operations, will be presenting: Optimizing the Scalability of Gene Therapy Manufacturing, on Tuesday April 25th, 2017, 2.00 p.m. EDT.
• Eileen Sawyer, Director of Global Medical Affairs, will be presenting: Bridging Gaps Through Early Integration of the Internal Medical Affairs Function, on Wednesday April 26th, 2017, 11.00 a.m. EDT.
• Daniel Leonard, Director of Global Patient Advocacy, will be presenting: The Intersection of Patient Advocacy and Gene Therapy: A Case Study in Hemophilia, on Wednesday April 26th, 2017, 2.00 p.m. EDT.

Alliance for Regenerative Medicines (ARM)'s 5th Annual Cell & Gene Therapy Investor Day, April 27th 2017, at The State Room, in Boston MA.

• Matthew Kapusta, chief executive officer, will be participating in a fireside chat on Thursday, April 27th, 2017, 2.15 p.m. EDT.

The Company also announced its participation in the following conferences taking place in April:

Hemophilia Federation of America (HFA)'s Annual Symposium, April 6 - 9 2017, at the Rhode Island Convention Center in Providence, RI.

The International Liver Congress 2017 by EASL, April 19 - 23 2017 at the RAI in Amsterdam, The Netherlands.

Spark Therapeutics Presents Updated Preliminary Data from Hemophilia B

Sangamo........... not so much.

Spark Therapeutics Presents Updated Preliminary Data from Hemophilia B Phase 1/2 Trial Suggesting Consistent and Sustained Levels of Factor IX Activity at the Hemostasis and Thrombosis Research Society (HTRS) 2017 Scientific Symposium

As of data cutoff, the annualized bleeding rate (ABR) has been reduced by 96 percent and the annualized infusion rate (AIR) reduced by 99 percent

Both participants who began a tapering course of steroids have completed their regimen

PHILADELPHIA, April 06, 2017 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE) announced updated preliminary data today from 10 infused participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for hemophilia B. All participants have experienced consistent and sustained increases in factor IX activity following administration of the investigational therapy. These data will be presented at the Hemostasis and Thrombosis Research Society (HTRS) 2017 Scientific Symposium in Scottsdale, Arizona on Friday, April 7, by Adam Cuker, M.D., assistant professor of medicine at the Perelman School of Medicine of the University of Pennsylvania and a clinical investigator at Children’s Hospital of Philadelphia.
Data as of March 24, 2017 will be presented on 10 participants in the study, who were dosed with a single administration of 5 x 10¹¹ vector genomes (vg)/kg body weight. All participants have discontinued routine infusions of factor IX concentrates. Based on individual patient history prior to the study, ABR was reduced by 96 percent to a mean of 0.39 annual bleeds, compared with 9.2 bleeds before SPK-9001 administration. AIR was reduced 99 percent to a mean of 0.98 annual infusions, compared with 68.5 infusions before SPK-9001 administration.
As of the data cutoff, nine of the 10 infused participants have not taken factor IX concentrates to prevent or control bleeding events since vector administration. As previously reported, one participant with severe joint disease has self-administered precautionary infusions for persistent knee pain. The mean steady-state factor IX activity level post 12 weeks treatment for the 10 participants was a sustained 33 percent (range as of the data cutoff: 14 to 81 percent). In the study to date, no serious adverse events have been reported, including no factor IX inhibitors and no thrombotic events. These data represent more than 2,400 cumulative patient days of exposure from the start of the trial.
Two of the 10 participants experienced an asymptomatic, transient elevation in liver enzymes, or decline in FIX activity, potentially indicative of an immune response to the Spark100 vector capsid, that occurred several weeks post infusion. Both participants received a tapering dose of oral corticosteroids, after which their alanine aminotransferase (ALT) levels returned to baseline. The activity level of one of these participants has stabilized at approximately 15 percent for more than nine weeks post corticosteroid use. The other participant had a factor IX activity level between 70 to 80 percent at completion of steroid use.
“The additional preliminary data continue to support our initial observations that a single intravenous administration of SPK-9001 has resulted in consistent and sustained levels of factor IX activity for trial participants,” said Katherine A. High, M.D., president and chief scientific officer at Spark Therapeutics.  “Notably, all participants to date have consistently achieved our targeted therapeutic range of FIX activity. As we continue to glean more insights from these preliminary data, our analysis suggests that a tapering course of oral corticosteroids has been well-tolerated and may help control potential capsid immune responses following SPK-9001 infusion.”

These data from the Phase 1/2 clinical trial of SPK-9001 will be presented during a poster session on Friday, April 7, from 5:15-6:15 p.m. MST.