Volume 12.25 | Jun 27

Cell Therapy News 12.25, June 27, 2011
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
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Cut-And-Paste Therapy Fixes Mouse Hemophilia
Scientists have developed a gene-repair kit that treats the blood-clotting disorder hemophilia in mice. The technique replaces genes in targeted organs without removing cells from the body, simultaneously correcting multiple mutations. [Press release from NatureNews discussing online prepublication in Nature]



Genes at the Flick of a Light Switch
Researchers have fitted human cells with a synthetic signaling cascade that can be used to switch on and regulate genes via blue light. [Press release from the Swiss Federal Institute of Technology Zurich discussing online prepublication in Science]

Molecular Glue Sticks it to Cancer
According to new research, the drug could force the proteins to the cell’s membrane and make the cancer cell more vulnerable to chemotherapy. [Press release from the University of Toronto, Mississauga discussing online prepublication in Angewandte Chemie]

Gold Nanoparticles Help Earlier Diagnosis of Liver Cancer
A research team has devised a new technique to spot cancerous tumors in the liver as small as 5 millimeters. [Press release from Brown University discussing online prepublication in Nano Letters]

Scientists Discover How to Control Fate of Stem Cells without the Risk of Developing Cancer Cells
Scientists have discovered how the body uses a single communication system to decide the fate of stem cells. [Press release from the Genome Institute of Singapore discussing online prepublication in PLoS Genetics]

Nanoparticles Disguised as Red Blood Cells Will Deliver Cancer-Fighting Drugs
Researchers have developed a novel method of disguising nanoparticles as red blood cells, which will enable them to evade the body’s immune system and deliver cancer-fighting drugs straight to a tumor. [Press release from the University of California, San Diego discussing online prepublication in the Proceedings of the National Academy of Sciences U S A]

Nanotubes to the Rescue: Brain Cells Rescued by Gene Silencing Offers New Options for Stroke
The use of carbon nanotubes to deliver siRNA and induce gene silencing of specific target areas in the brain responsible for motor functions has allowed scientists to ‘switch off’ proteins that contribute to neuronal tissue loss. [Press release from the University of London The School of Pharmacy discussing online prepublication in the Proceedings of the National Academy of Sciences U S A]

Next Generation Gene Therapy: Study Demonstrates Potential of New Gene Vector to Broaden Treatment of Eye Diseases
In the present study, the team compared the safety and efficiency of delivery in an animal model of two different types of adeno-associated viruses (AAVs) – AAV2 and AAV8. [Press release from the University of Pennsylvania Perelman School of Medicine discussing online prepublication in Science Translational Medicine]

Pigs Could Grow Human Organs in Stem Cell Breakthrough
Scientists have found they can create chimeric animals that have organs belonging to another species by injecting stem cells into the embryo of another species. [Press release from The Telegraph discussing research presented at the European Society of Human Genetics]

Curis Announces Presentation of Positive Vismodegib Pivotal Clinical Data in Advanced Basal Cell Carcinoma
Curis, Inc. announced that positive data was presented by its collaborator Genentech from a pivotal Phase II clinical trial conducted by Roche and Genentech of vismodegib (GDC-0449, RG3616) in patients with advanced basal cell carcinoma. [Press release from Curis, Inc. discussing research presented at the 7th European Association of Dermato-Oncology Congress]

Scientists Develop First Ever Drug to Treat ‘Celtic Gene’ in Cystic Fibrosis Sufferers
An international research team has developed a ground breaking treatment for cystic fibrosis sufferers. The new drug will benefit sufferers who have the ‘Celtic Gene’, a genetic mutation which is particularly common in Ireland. [Queen’s University Belfast Press Release]

Oxford BioMedica Announces Ocular Program Update
Oxford BioMedica plc announced that the first dose level of RetinoStat® is safe and well-tolerated at one month following treatment. [PR Newswire]

InnaVirVax Announces Promising Preclinical Results of its Therapeutic Vaccine VAC-3S for the Treatment of HIV Infections
InnaVirVax announced promising preclinical results of its therapeutic vaccine VAC-3S, which aims at preserving the immune system of patients infected with HIV-1. [Medical News Today]


(Ranked by Impact Factor of the Journal)

In Vivo Genome Editing Restores Hemostasis in a Mouse Model of Hemophilia
Researchers showed that zinc finger nucleases (ZFNs) are able to induce double-strand breaks efficiently when delivered directly to mouse liver and that, when co-delivered with an appropriately designed gene-targeting vector, they can stimulate gene replacement through both homology-directed and homology-independent targeted gene insertion at the ZFN-specified locus. [Nature]

A Synthetic Optogenetic Transcription Device Enhances Blood-Glucose Homeostasis in Mice
By functionally linking the signal transduction of melanopsin to the control circuit of the nuclear factor of activated T cells, investigators have designed a synthetic signaling cascade enabling light-inducible transgene expression in different cell lines grown in culture or bioreactors or implanted into mice. [Science]

Nanomaterials for X-Ray Imaging: Gold Nanoparticle Enhancement of X-ray Scatter Imaging of Hepatocellular Carcinoma
Scientists presented the development of a new imaging technique for the early diagnosis of hepatocellular carcinoma that utilizes surface-modified gold nanoparticles in combination with X-ray imaging. [Nano Letters]

Graded Nodal/Activin Signaling Titrates Conversion of Quantitative Phospho-Smad2 Levels into Qualitative Embryonic Stem Cell Fate Decisions
Researchers showed that any perturbation of endogenous signaling levels in mouse embryonic stem cells leads to their exit from self-renewal towards divergent differentiation programs. [PLoS Genet]

Erythrocyte Membrane-Camouflaged Polymeric Nanoparticles as a Biomimetic Delivery Platform
Scientists reported a top-down biomimetic approach in particle functionalization by coating biodegradable polymeric nanoparticles with natural erythrocyte membranes, including both membrane lipids and associated membrane proteins for long-circulating cargo delivery. [Proc Natl Acad Sci U S A]

Functional Motor Recovery from Brain Ischemic Insult by Carbon Nanotube-Mediated siRNA Silencing
Researchers studied whether silencing of caspase-3 using carbon nanotube-mediated in vivo RNA interference could offer a therapeutic opportunity against stroke. [Proc Natl Acad Sci U S A]

Stem Cell-Based Meniscus Tissue Engineering
To address the need for meniscus tissue engineering, a multiporous, multilamellar meniscus was formed using silk protein scaffolds and stem cells. [Tissue Eng Part A]



Bond Financier Elected to Chair California Stem Cell Agency
The California Institute for Regenerative Medicine has elected southern California investor Jonathan Thomas to be its next chair. [California Institute for Regenerative Medicine, United States]

Stem Cell Briefs Probe Whether Grants “Incentivize” Embryo Destruction
The two sides in a high-stakes legal battle over stem cells expanded on their arguments in briefs filed in a case being heard by Chief Judge Royce Lamberth of the U.S. District Court for the District of Columbia. [U.S. District Court for the District of Columbia, United States]

U.S. Patent Reform Survives a Shakedown in the House
A plan to make sweeping changes in the U.S. patent system passed the House of Representatives by a vote of 304-117. [House of Representatives, United States]

Diabetes Pill Deaths Prompts French Drug Regulation Reform
Saying he hopes to prevent a repeat of the Médiator scandal, French health minister Xavier Bertrand unveiled what he described as the most important overhaul to the drug regulation system ever undertaken in the country. [French Health Ministry, France]

And We’re Calling It: Horizon 2020
The European Commission announced that the successor to Framework Programme 7, slated to start in 2014, will be called Horizon 2020. [European Commission, European Union]

Insider To Take Over Troubled Spanish Cancer Institute
Spanish molecular biologist María Blasco will take over the leadership of the Spanish National Cancer Research Centre in Madrid, where she is now a vice-director. [Spanish National Cancer Research Centre, Spain]

Korea Is First to Approve Therapy
A Korean pharmaceutical company took a step closer to beginning commercial production of the world’s first stem cell therapy as the Korea Food and Drug Administration said the approval will be given. [Korea Food and Drug Administration, Korea]


ReNeuron Signs License Agreement with Leading US Eye Research Institute to Develop Stem Cell Therapies for Diseases of the Retina
ReNeuron Group plc announced that it has signed a patent and know-how license agreement with Schepens Eye Research Institute regarding the Company’s ReN003 stem cell therapy program focused on diseases of the retina. [ReNeuron Group plc Press Release]

BioFocus Extends Collaboration with Usher III Initiative
BioFocus announced that it has expanded a collaboration with the Usher III Initiative, Inc. [BioFocus Press Release]

MIT and Skolkovo Foundation Announce Collaboration
The Massachusetts Institute of Technology (MIT) and the Skolkovo Foundation announced the signing of a preliminary agreement to create the Skolkovo Institute of Science and Technology. [Massachusetts Institute of Technology Press Release]

Apeiron, CCRI and SIOPEN Join Forces Against Neuroblastoma
Apeiron Biologics AG will support the clinical development of an immune therapy against high-risk neuroblastoma that is being conducted by the Children’s Cancer Research Institute (CCRI) and the European Neuroblastoma Research Network (SIOPEN). [Apeiron Biologics Press Release]

UC Davis Clinical and Translational Science Center Receives Renewed Grant Funding
The University of California (UC) Davis Clinical and Translational Science Center received renewed grant funding of $20 million from the National Center for Research Resources. [University of California, Davis Press Release]

University to Develop Skin Cancer Detection Tool to Save Lives
De Montfort University, working in collaboration with the University Hospitals of Leicester NHS Trust’s Dermatology Department, has been awarded £75,000 to create a new device to help in the early detection of malignant melanoma skin cancer. [De Montfort University Press Release]

Children’s Hospital Los Angeles Researcher Wins Grant to Study Stem Cells’ Ability to Reverse Renal Fibrosis
Laura Perin, PhD, co-director of Research in the division of Pediatric Urology at Children’s Hospital Los Angeles, has been awarded a $100,000 grant from the Alport Syndrome Foundation for her novel study of amniotic fluid stem cells in reversing the fibrosis common to inherited kidney disorder. [Children’s Hospital Los Angeles Press Release]

Gene Therapy Faces Setback as EU Rebuffs AMT Drug
A novel gene therapy drug from Amsterdam Molecular Therapeutics (AMT) has been rejected by European regulators, underscoring the difficulty of getting gene-based treatments to market. [Reuters Press Release]

Korea Is First to Approve Therapy
A Korean pharmaceutical company took a step closer to beginning commercial production of the world’s first stem cell therapy as the Korea Food and Drug Administration said the approval will be given. [Korea JoonAng Daily Press Release]

Fibrocell Science, Inc. Announces FDA Approval for laVív® (azficel-T)
Fibrocell Science, Inc. announced that the U.S. Food and Drug Administration (FDA) approved the Company’s Biologics License Application for its lead product, laVív® (azficel-T). [Fibrocell Science, Inc. Press Release]

Threshold Pharmaceuticals Announces Completion of Patient Recruitment for Phase II TH-302 Pancreatic Cancer Study
Threshold Pharmaceuticals, Inc. announced the completion of patient enrollment into the Company’s Phase II pancreatic cancer study with TH-302, a novel small molecule tumor selective Hypoxia-Activated Prodrug that specifically targets tumor hypoxia and is applicable to a broad range of solid tumors. [Threshold Pharmaceuticals, Inc. Press Release]

Clavis Pharma Announces the Expansion by its Partner Clovis Oncology of the Observational Study to Determine Patient Stratification in its Pivotal LEAP Clinical Trial with CP-4126
Clavis Pharma ASA announces that Clovis Oncology, Inc, its development partner for CP-4126, has expanded the ongoing observational study to determine the patient stratification parameters in the pivotal LEAP clinical trial with CP-4126. [Clavis Pharma ASA Press Release]

CEL-SCI Announces Start of Multikine Phase III Study by Orient Europharma in Taiwan
CEL-SCI Corporation announced that its Taiwanese partner, Orient Europharma, has concluded the Site Initiation Visit for the first Taiwanese clinical center in CEL-SCI’s Phase III clinical trial for Multikine®, the Company’s flagship cancer immunotherapy. [Cel-Sci Corporation Press Release]

Bergen County Spine Clinic Spine & Joint Center Offers New Stem Cell Treatment Options for Chronic Joint Problems
A spine clinic Bergen County is now offering a revolutionary treatment for individuals with chronic joint problems using the patient’s own stem cells. Performed at Spine & Joint Center, the treatment utilizes adult stem cells harvested from the patient’s own fat cells, which are then injected into the injured joint to help stimulate the regeneration of healthy tissue. [PR Newswire Press Release]

Stowers Institute Recruits Top Scientists in Developmental and Regenerative Biology
Tatjana Piotrowski, Ph.D., and Alejandro Sánchez Alvarado, Ph.D., joined the Stowers Institute for Medical Research. [Stowers Institute for Medical Research Press Release]

Aastrom Establishes Independent Steering Committee for REVIVE Phase III CLI Clinical Program
Aastrom Biosciences, Inc. announced the formation of an independent steering committee to support the REVIVE Phase III clinical program for ixmyelocel-T. [Aastrom Biosciences, Inc. Press Release]

Two Hutchinson Center Researchers Receive Prestigious Awards from Damon Runyon Cancer Research Foundation
Two Fred Hutchinson Cancer Research Center researchers have received prestigious awards from the Damon Runyon Cancer Research Foundation. [Fred Hutchinson Cancer Research Center Press Release]

Leaders in Fight Against Cancer Join Historic Initiative Addressing Clinical Investigator Shortage
The Damon Runyon Cancer Research Foundation announced the formation of Accelerating Cancer Cures, a historic collaboration of the biopharmaceutical industry and academia with a strong commitment to achieving key breakthroughs in cancer treatments. [Damon Runyon Cancer Research Foundation Press Release]

Sanofi Pasteur Opens $100M R&D Centre in Canada
On June 22, Ontario Premier, Dalton McGuinty joined Mark Lievonen, President, Sanofi Pasteur Canada, to open a $101 million dollar vaccine research and development facility at Sanofi Pasteur’s Connaught Campus in north Toronto. [Sanofi Pasteur Press Release]


Legislator Asks NIH to Halt Search for Translational Center Director
The National Institutes of Health is again taking flak from Congress for its plan to create a center for translational research.

Center for Scientific Review; Notice of Closed Meetings [FR Doc No: 2011-15489]

Center for Scientific Review; Notice of Closed Meetings [FR Doc No: 2011-15717]

Center for Scientific Review; Notice of Closed Meetings [FR Doc No: 2011-15727]

National Cancer Institute; Notice of Closed Meetings [FR Doc No: 2011-16000]

National Cancer Institute; Notice of Closed Meeting [FR Doc No: 2011-15997]

National Cancer Institute; Amended Notice of Meeting [FR Doc No: 2011-15999]

National Center for Research Resources; Notice of Closed Meetings [FR Doc No: 2011-15634]

National Human Genome Research Institute; Notice of Closed Meeting [FR Doc No: 2011-15718]

National Institute of General Medical Sciences; Notice of Closed Meeting [FR Doc No: 2011-15635]

National Institute of General Medical Sciences; Notice of Closed Meeting [FR Doc No: 2011-15711]

National Institute of General Medical Sciences; Notice of Closed Meeting [FR Doc No: 2011-15995]

Office of the Director, National Institutes of Health; Notice of Meeting [FR Doc No: 2011-15998]


Final Rule – Medical Devices; Neurological Devices; Clarification of Classification for Human Dura Mater; Technical Amendment (Final Rule; Technical Amendment)

Proposed Rule – Amendments to Sterility Test Requirements for Biological Products, June 21, 2011



FDA Fires Warning Shot at Australian Vaccine Maker
A warning letter from the U.S. Food and Drug Administration to Australian vaccine maker CSL Biotherapies has shaken confidence in the country’s biotech superstar.

Medical Devices; Exception From General Requirements for Informed Consent [Docket No. FDA-2003-N-0212; (formerly Docket No. 2003N-0355)]|

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Guidance for Industry on Pharmacogenomic Data Submissions [Docket No. FDA-2010-N-0536]


European Medicines Agency and US Food and Drug Administration Set Up Biosimilar ‘Cluster’ and Publish First Report on Interactions
The European Medicines Agency and the United States Food and Drug Administration have set up a new ‘cluster’ on biosimilar medicines.

European Medicines Agency Holds First Meeting of Expert Group on the Replacement, Reduction and Refinement of the Use of Animals
The European Medicines Agency has established an ad-hoc expert group on the application of the ‘3Rs’ – replacement, reduction and refinement of animal testing – in the development of medicines.

European Medicines Agency Listens to Stakeholder Expectations for Pharmacovigilance Reforms
The European Medicines Agency has hosted a second workshop on the changes to the safety-monitoring of medicines in Europe, where it heard the expectations from patients, consumer groups, healthcare professionals and the pharmaceutical industry.

Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 20-23 June 2011
Positive opinions for new medicines adopted.

European Medicines Agency Recommends Restricting the Use of Dexrazoxane-Containing Medicines
The European Medicines Agency has recommended restricting the use of dexrazoxane to adult patients with advanced or metastatic breast cancer who have already received a certain amount of the anthracyclines doxorubicin and epirubicin to treat their cancer.


New MHRA Director of Communications
The MHRA welcomed its new Director of Communications, Rachel Bosworth.


Advanced Notice: Revised TGA Fees & Charges
The amendment regulations for amending the Therapeutic Goods Regulations 1990, the Therapeutic Goods (Charges) Regulations 1990 and the Therapeutic Goods (Medical Devices) Regulations 2002 have been approved by the Executive Council in its meeting on 16 June 2011 in order to increase most TGA fees and charges by 3.4%.

Australian Pharmacovigilance Guideline for Sponsors
This guideline is for the reporting of adverse reactions to registered and listed medicines.

Transparency Review of the TGA
Added reports from the 4th, 5th & 6th meetings of the TGA Transparency Review Panel.

Landmark Agreement to Achieve World Standards in Therapeutic Products


NEW Cold Spring Harbor Laboratory – Stem Cell Biology
September 20-24, 2011
Cold Spring Harbor, United States

NEW Screening Stem Cells 2011: From Reprogramming to Regenerative Medicine
September 26-27, 2011
Boston, United States

our events page to see a complete list of events in the cell, gene and immunotherapy community.


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Lab Technologist – Tissue Culture (Maternity Leave) (STEMCELL Technologies)

Research and Development Technologist, hPSC Media (STEMCELL Technologies)

Scientist (iPSC) (STEMCELL Technologies)

Director, Cell Processing Operations (Hospira Industry)

PhD Scholarship (Tasmania University)

Postdoctoral Fellow (Roswell Park Cancer Institute)

Industrial Postdoctoral Research Fellow (Regenerative Medicine Cell Therapy) (Tissue Regeneration Therapeutics Inc.)

Lab Technician (Tissue Regeneration Therapeutics, Inc.)

Research Laboratory Technician (Cambridge Centre for Brain Repair)

Postdoctoral Research Fellow (Tshwane University of Technology)

Senior Research Fellowship (Masaryk University)

Junior Research Fellowship (Masaryk University)

Reintegrating/Czech Nationality/ Research Fellowship (Masaryk University)

Microscopy and Image Analysis Specialist (Masaryk University)

PhD Positions in Stem Cell Research (Masaryk University)

PhD Positions in Medical Biology (Masaryk University)

Postdoctoral Fellow – Stem Cell Biology and Immunology (NIH Center for Biomedical Research Excellence)

Graduate Student – Laboratory for Equine and Comparative Orthopedic Research (Louisiana State University)

Post Doctoral Fellow (University of California, Los Angeles)

Staff Scientist Position (Spanish National Cancer Center)

Postdoctoral Research Fellows (Stem Cells) (A*Star Bioprocessing Technology Institute) 

Postdoctoral Positions in Immune Cell Activation (Institut de Recherches Cliniques de Montréal)

Postdoctoral Position in Developmental Neurobiology (Institut de Recherches Cliniques de Montréal)

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