Cell Therapy News Volume 12.49 | Dec 12 2011

May 12, 2022

Issue 12.49 December 12, 2011

TOP STORY

Gene Therapy Achieves Early Success Against Hereditary Bleeding Disorder
A study on gene therapy offers first proof that adults with hemophilia B benefit from treatment, reducing need for injections with clotting factor to prevent bleeds. [Press release from University College London discussing online prepublication in The New England Journal of Medicine] Press Release | Abstract

PUBLICATIONS (Ranked by Impact Factor of the Journal)

Polycomb-Repressed Genes Have Permissive Enhancers that Initiate Reprogramming
Using the well-characterized enhancer/promoter pair of MYOD1 as a model, researchers have identified a critical role for enhancers in reprogramming. [Cell] Abstract | Press Release

Biodegradable Poly(Amine-Co-Ester) Terpolymers for Targeted Gene Delivery
Here investigators describe the synthesis of a series of high molecular weight terpolymers with low charge density, and show that they exhibit efficient gene delivery, some surpassing the efficiency of the commercial transfection reagents Polyethylenimine and Lipofectamine 2000. [Nat Mater] Abstract | Press Release

Oncogene-Targeting T Cells Reject Large Tumors while Oncogene Inactivation Selects Escape Variants in Mouse Models of Cancer
Researchers established mouse cancer models, which allowed targeting of an oncogene by drug-mediated inactivation or monospecific CD8⁺ effector T cells. [Cancer Cell] Abstract

Nonmalignant Late Effects and Compromised Functional Status in Survivors of Hematopoietic Cell Transplantation
Researchers’ objective was to describe the incidence of nonmalignant late complications and their association with health and functional status in a recent cohort of hematopoietic cell transplantation survivors. [J Clin Oncol] Abstract

Long-Term Restoration of Cardiac Dystrophin Expression in Golden Retriever Muscular Dystrophy Following rAAV6-Mediated Exon Skipping
In this study, researchers sought to optimize restoration of cardiac dystrophin expression in the golden retriever muscular dystrophy model using percutaneous transendocardial delivery of recombinant AAV6 (rAAV6) to deliver a modified U7 small nuclear RNA carrying antisense sequence to target the exon splicing enhancers of exons 6 and 8 and correct the disrupted reading frame. [Mol Ther] Abstract

Enhanced Extravasation, Stability and In Vivo Cardiac Gene Silencing via In Situ siRNA-Albumin Conjugation
This study aimed to bypass the endothelial barrier by harnessing the extravasation capability of the serum protein albumin to allow siRNA to reach cardiomyocytes. [Mol Pharm] Abstract

Mechanistic Insights into LDL Nanoparticle-Mediated siRNA Delivery
Here, researchers examine purified low density lipoprotein (LDL) nanoparticles as carriers for small interfering RNAs (siRNAs). [Bioconjug Chem] Abstract

Early Gestational Gene Transfer with Targeted ATP7B Expression in the Liver Improves Phenotype in a Murine Model of Wilson’s Disease
Here, scientists demonstrate that prenatal gene transfer of human Atp7b reduces liver pathology and improves biochemical markers in Atp7b^-/- mice, a murine model of Wilson’s disease. [Gene Ther] Abstract

SCIENCE NEWS

Studies Investigate New Methodologies to Improve Recovery and Survival Strategies for Patients Undergoing Hematopoietic Cell Transplantation
Hematopoietic cell transplantation is the primary option for treatment for many patients who suffer from various hematologic disorders. The studies presented demonstrate the major advances underway in the field of hematopoietic cell transplantation. [Press release from the American Society of Hematology discussing research presented at the 53rd Annual Meeting of the American Society of Hematology, San Diego] Press Release

Study Points to Novel Way to Improve Outcomes from Umbilical Cord Blood Transplants
A new method to boost the number of immune cells in umbilical cord blood prior to cord blood transplants for cancer patients appears to lead to a quicker rebuilding of a new immune system in the patient’s body than with a conventional cord blood transplant procedure, according to new research. [Press release from the Perelman School of Medicine at the University of Pennsylvania discussing research presented at the 53rd Annual Meeting of the American Society of Hematology, San Diego] Press Release

Update on TK Clinical Benefit Presented at ASH and Expansion of Phase III Trial in Europe
MolMed S.p.A. presented an update on the clinical benefit following treatment with its investigational cell-based therapy TK for high-risk leukemias in adult patients: patients treated, including the first patients enrolled in the ongoing Phase III trial (TK008), show restoration of a fully functional immune system. [Press release from MolMed S.p.A. discussing research presented at the 53rd Annual Meeting of the American Society of Hematology (ASH), San Diego] Press Release

Researchers Use Stem Cells to Gain New Insight into Severe Childhood Form of Epilepsy
A team of researchers investigating the fundamental cause of Dravet syndrome have reprogrammed fibroblasts from Dravet patients and generated patient-specific neurons – which could help determine new therapies or better medications for the syndrome. [Press release from the University of Michigan Health System discussing research presented at the American Epilepsy Society’s 65th Annual Meeting, Baltimore] Press Release

POLICY

New Chief for HHS’s Research Misconduct Office
The federal office that guards against scientific misdeeds in biomedical research has a new director. David E. Wright will take the helm of the Department of Health and Human Services (HHS) Office of Research Integrity in January. [Department of Health and Human Services, United States] Press Release

UK Cancer Charity Slashes Research Budget
One of Britain’s largest biomedical funders, Cancer Research UK, will slash research spending because of falling donations, the charity announced. [Cancer Research UK, United Kingdom] Press Release

German Science Organizations Slam European Court Over Stem-Cell Ruling
The powerful Alliance of German Scientific Organizations has now publicly criticized a ruling from the European Court of Justice that bans patenting of inventions involving human embryonic stem (ES) cells and says that research using human ES cell lines is immoral. [Alliance of German Scientific Organizations, Germany] Press Release

BUSINESS

Stem Cell Research Milestone
Stem cell scientists announced that they have submitted to the UK Stem Cell Bank their first clinical grade human embryonic stem cell lines that are free from animal-derived products. [King’s College London] Press Release

CIRM Approves $27 Million for Initiatives to Accelerate Promising Stem Cell Research Projects
The California Institute for Regenerative Medicine (CIRM) enhanced its efforts to proactively discover and fund innovative research and to provide continuous support to successful research programs, speeding the development of new stem cell-based therapies. [California Institute for Regenerative Medicine] Press Release

£50 Million Technology and Innovation Centre in Cell Therapy to Come to London
The Technology Strategy Board has announced further details about its Technology and Innovation Centre in Cell Therapy. The new centre will support the development and commercialization of cell therapies and advanced therapeutics, as well as the underpinning technologies for manufacturing, quality control, safety and efficiency. [Technology Strategy Board] Press Release

Pediatric Clinical Trial Investigating Vaccine-Based Therapy for Solid Tumors
Recruitment is ongoing at Texas Children’s Cancer Center for a Phase I clinical trial in children with cancer using a novel immunotherapy approach called oncolytic virus therapy. [Baylor College of Medicine] Press Release

HSCI Receives Approval to Market Neovasculgen – The First Russian Gene-Therapy Drug for Treatment of Peripheral Arterial Disease
The Human Stem Cells Institute (HSCI) announced that it has received state approval for Neovasculgen® – the first Russian gene-therapy drug to treat Peripheral Arterial Disease. [The Human Stem Cells Institute] Press Release

Silence Therapeutics Announces Positive Outcome from Oral Hearing at the European Patent Office
Silence Therapeutics plc (Silence) announced a positive outcome from the oral hearing at the European Patent Office over opposition to Silence’s granted European Patent EP 1 536 827 “Further use of protein kinase N beta“. [Silence Therapeutics plc] Press Release

VistaGen Therapeutics and Synterys Sign Strategic Medicinal Chemistry Collaboration Agreement for Drug Rescue
The collaboration will further VistaGen’s stem cell technology-based drug rescue initiatives with the support of Synterys’ medicinal chemistry expertise. [VistaGen Therapeutics, Inc.] Press Release

Collaboration Aimed at Automating Manufacturing of Regenerative Medicine Therapies
Wake Forest Baptist Medical Center’s Institute for Regenerative Medicine is teaming up with North Carolina State University’s Edward P. Fitts Department of Industrial and Systems Engineering with the goal of applying the latest advances in industrial engineering to the new field of regenerative medicine. [Wake Forest Baptist Medical Center] Press Release

NeoStem Appoints a Biopharmaceutical Business Leader and a Cellular Therapy Expert to its Board of Directors
NeoStem, Inc. announced two appointments to its Board of Directors. These appointments are Martyn D. Greenacre and Dr. Andrew L. Pecora. [NeoStem, Inc.] Press Release

Season's Greetings: Wishing You a Joyous Holiday Season and a Happy New Year

NIH

Center for Scientific Review; Notice of Closed Meeting (FR Doc No: 2011-31391)

Government-Owned Inventions; Licensing and Collaborative Research Opportunity: Chemotoxins for Targeted Treatment of Diseased Cells (FR Doc No: 2011-31554)

Notice of Termination of PAR-10-278, “Limited Competition for the Global Research Initiative Program, Basic/Biomedical Sciences (R01)” (NOT-TW-12-003)

Notice of Termination of PAR-11-037, “Limited Competition: Fogarty International Research Collaboration – Basic Biomedical (FIRCA-BB) Research Award (R03)” (NOT-TW-12-004)

Limited Competition: Comprehensive Partnerships to Advance Cancer Health Equity (CPACHE) (U54) (PAR-12-055)

CBER

2011 Biological License Application Approvals

2011 Biological License Application Supplement Approvals

2011 Biological Device Application Approvals

Approval History, Letters, Reviews and Related Documents – Hemacord

REGULATORY

Food and Drug Administration (United States)

GOP Sen. Coats Calls for FDA Mission Reform
Sen. Dan Coats introduced an “FDA Mission Reform Act”, aiming to clarify the mission of the federal watchdog agency and clarify the regulatory environment. Press Release

Regulatory Site Visit Training Program (Docket No. FDA-2011-N-0824)

European Medicines Agency (European Union)

European Medicines Agency and United States Food and Drug Administration to Share Manufacturing Site Inspections
The European Medicines Agency and the United States Food and Drug Administration are launching an initiative to share work on inspections of manufacturing sites in each other’s territories. Press Release

Therapeutic Goods Administration (Australia)

Government to Improve Regulation of Therapeutic Goods
The Australian government will introduce a comprehensive package of reforms for Australia’s Therapeutic Goods Administration to ensure the regulation of medicines and medical devices is more effective and transparent. Press Release

TGA Reforms: A Blueprint for TGA’s Future
The Australian Government has released its response to several major reviews of therapeutic goods regulation that have been undertaken over the past 18 months. Press Release

Risk Management Plan Guideline

TGA Committee Members Must Declare Conflict of Interest