Volume 11.36 | Sep 20

Cell Therapy News 11.36, September 20, 2010.
In this issue:  Science  |  Policy  |  Business  |  NIH  |  CBER  |  Regulatory  |  Events Subscribe  |  Unsubscribe


Promising Results on Treatment of First Patient in Bluebird Bio’s Phase I/II Beta-Thalassemia Study
Bluebird Bio announced publication of its promising Phase I/II data highlighting positive results of LentiGlobin™ gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases. [Press release from Bluebird Bio discussing online prepublication in Nature]



Novartis JAK Inhibitor Provides Marked and Durable Clinical Benefits in Patients with Myelofibrosis
Results from a Phase I/II study of the Novartis Janus kinase (JAK) inhibitor with the investigational name INC424 (also known as INCB018424 and INCB18424) demonstrate marked and durable clinical benefits in patients with myelofibrosis. [Press release from Novartis discussing online prepublication in the New England Journal of Medicine]

Data Shows PLX4032 Selectivity Enables Substantial Pathway Inhibition and Significant Clinical Response
Plexxikon announced publication of key data demonstrating that the high degree of selectivity of PLX4032 (RG7204) enables substantial RAF/MEK/ERK pathway inhibition, which may be necessary to achieve significant tumor shrinkage and clinical response in patients with BRAF mutant melanoma. [Press release from Plexxikon discussing online prepublication in Nature]

‘Key Player’ Genes Indentified That Could Lead to Type 1 Diabetes
Scientists published new research identifying an entire network of genes associated with type 1 diabetes. [Press release from Diabetes UK discussing online prepublication in Nature]

Scientists Identify Molecules Involved in Touch and Other Mechanically Activated Systems
Scientists have identified two proteins with potential to be important targets for research into a wide range of health problems, including pain, deafness, and cardiac and kidney dysfunction. [Press release from the Scripps Research Institute discussing online prepublication in Science]

Lung Cancer Culprit Could Offer Target for Therapy, Researchers Report
A tiny molecule that spurs the progression of non-small-cell lung cancer could become a player in fighting the disease. [Press release from the University of Texas Southwestern Medical Center discussing online prepublication in Cancer Cell]

Cloned Virus
A scientist has successfully cloned a human virus, offering new hope for the development of treatments for life-threatening diseases. [Press release from Cardiff University discussing online prepublication in the Journal of Clinical Investigation]

Your Body Recycling Itself — Captured on Film
Proteins are made up of a chain of amino acids, and scientists have known since the 1980s that the first one in the chain determines the lifetime of a protein. Researchers have finally discovered how the cell identifies this first amino acid — and caught it on camera. [Press release from McGill University discussing online prepublication in Nature Structural and Molecular Biology]

Scientists Unravel Genetic Risk of Bowel Cancer
An international study has shown how a single variant in a person’s DNA can promote bowel cancer development. [Press release from The Institute of Cancer Research discussing online prepublication in PLoS Genetics]

Researchers Identify Genetic Marker of Aggressive Alzheimer’s Disease
An international team of Alzheimer’s disease experts has uncovered a gene variation that appears to predict the rate at which Alzheimer’s disease will progress. [Press release from the Washington University School of Medicine in St. Louis discussing online prepublication in PLoS Genetics]

First Trial of Bone-Marrow Stem Cells for Heart Attack Proves Safe
The first randomized, placebo-controlled U.S. clinical trial to assess the use of bone marrow-derived mononuclear cells in patients after a ST-elevation myocardial infarction demonstrated a strong safety profile for this cell therapy, based on Phase I results. [Press release from EurekAlert! discussing online prepublication in the American Heart Journal]

Researchers Create iPS Cells from Human Gums
A team of researchers has succeeded in creating induced pluripotent stem (iPS) cells from human gums. [Press release from The Mainichi Daily News discussing online prepublication in PLoS One]

Scientists Improve Biomarker Detection Technique
Scientists have developed a new strategy to enable quicker and more precise detection of biomarkers. [Press release from the National Physical Laboratory discussing online prepublication in Molecular BioSystems]

Researchers Build ‘Artificial Ovary’ to Develop Oocytes into Mature Human Eggs
Researchers have invented the first artificial human ovary, an advance that provides a potentially powerful new means for conducting fertility research and could also yield infertility treatments for cancer patients. [Press release from Brown University discussing online prepublication in the Journal of Assisted Reproduction and Genetics]

Scientists Find Genes Related to Body Mass
Scientists who specialize in unconventional hunts for genetic information outside nuclear DNA sequences have bagged a weighty quarry — 13 genes linked to human body mass. [Press release from Johns Hopkins University discussing online prepublication in Science Translational Medicine]

Cells Restore Ovary Function in Rat Study
Scientists have found that injecting a particular type of stem cells into infertile female rats can restore the function of their ovaries, and say their findings could pave the way for a similar treatment for humans. [Press release from Reuters discussing research presented at the 20th World Congress on Fertility and Sterility]

CUPID: AAV1/SERCA2a Found Safe, Beneficial in Patients with Advanced Heart Failure
New data from a Phase II trial showed favorable results, including group and individual improvements, for patients with heart failure (HF) who were treated with AAV1/SERCA2a, a genetically targeted enzyme replacement therapy for advanced HF. [Press release from CardiologyToday discussing research presented at the Heart Failure Society of America 14th Annual Scientific Meeting]

Data from Clinical Trials Show Pluristem’s Placenta-Derived Cell Therapy is Safe and Improves Quality of Life and Efficacy Measurements in Patients with Critical Limb Ischemia
Pluristem Therapeutics Inc. reported that data from clinical trials show the Company’s placenta-derived cell therapy, PLX-PAD, is safe and improves quality of life as well as other efficacy measurements in patients with Critical Limb Ischemia. [Pluristem Therapeutics Inc. Press Release]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Safety and Efficacy of INCB018424, a JAK1 and JAK2 Inhibitor, in Myelofibrosis
INCB018424 was associated with marked and durable clinical benefits in patients with myelofibrosis for whom no approved therapies existed. [N Engl J Med]

Transfusion Independence and HMGA2 Activation After Gene Therapy of Human Beta-Thalassemia
Researchers show that, 33 months after lentiviral beta-globin gene transfer, an adult patient with severe betaE/beta0-thalassaemia dependent on monthly transfusions since early childhood has become transfusion independent for the past 21 months. [Nature]

Clinical Efficacy of a RAF Inhibitor Needs Broad Target Blockade in BRAF-Mutant Melanoma
Data demonstrate that BRAF-mutant melanomas are highly dependent on B-RAF kinase activity. [Nature]

A Trans-Acting Locus Regulates an Anti-Viral Expression Network and Type 1 Diabetes Risk
Data implicate IRF7 network genes and their regulatory locus in the pathogenesis of type 1 diabetes. [Nature]

Piezo1 and Piezo2 Are Essential Components of Distinct Mechanically Activated Cation Channels
Expression profiling and RNAi knockdown of candidate genes identified Piezo1 (Fam38A) to be required for mechanically activated currents in a mouse neuroblastoma cell line. [Science]

Modulation of K-Ras-Dependent Lung Tumorigenesis by MicroRNA-21
Researchers show that overexpression of microRNA-21 (miR-21) enhances tumorigenesis and that genetic deletion of miR-21 partially protects against tumor formation. MiR-21 drives tumorigenesis through inhibition of negative regulators of the Ras/MEK/ERK pathway and inhibition of apoptosis. [Cancer Cell]

Reconstruction of the Complete Human Cytomegalovirus Genome in a BAC Reveals RL13 to Be a Potent Inhibitor of Replication
To generate a genetically intact source of human cytomegalovirus, researchers cloned strain Merlin into a self-excising BAC. [J Clin Invest]
Structural Basis of Substrate Recognition and Specificity in the N-End Rule Pathway
Researchers report the crystal structures of the UBR boxes of the human N-recognins UBR1 and UBR2, alone and in complex with an N-end rule peptide, Arg-Ile-Phe-Ser. [Nat Struct Mol Biol]
Allelic Variation at the 8q23.3 Colorectal Cancer Risk Locus Functions as a Cis-Acting Regulator of EIF3H
Data provide evidence for a functional basis for the non-coding risk variant rs16888589 at 8q23.3 and provides novel insight into the etiological basis of colorectal cancer. [PLoS Genet]
SNPs Associated with Cerebrospinal Fluid Phospho-Tau Levels Influence Rate of Decline in Alzheimer's Disease
Researchers identified a genetic factor associated with the rate of decline in Alzheimer's Disease patients and present a methodology for identification of other such factors. [PLoS Genet]

Personalized Epigenomic Signatures That Are Stable Over Time and Covary with Body Mass Index
Four variably methylated regions showed covariation with body mass index consistently at two study visits and were located in or near genes previously implicated in regulating body weight or diabetes. [Sci Transl Med]



Senate Panel Weighs in on Threat to Stem Cell Research
In a Senate hearing on the ongoing legal tussle over human embryonic stem cell research, lawmakers and expert witnesses lamented the disruption to this promising research. Congress may have to act soon to fix the problem, two Democratic members said. [Senate, United States]

Oral Hearing Scheduled in Stem Cell Injunction Appeal
In an unexpected move, the United States Court of Appeals for the D.C. Circuit scheduled a 30-minute oral hearing on 24 September to hear arguments on whether Chief Judge Royce Lamberth's injunction that blocked National Institutes of Health funding for work with the cells should be lifted while the case is being litigated. [Court of Appeals, United States]

'Sound Science' Code Draws Fire
The first draft policy on scientific integrity from a federal agency, released by the Department of the Interior, has drawn a storm of criticism from watchdogs including the Union of Concerned Scientists, based in Cambridge, Massachusetts. [Union of Concerned Scientists, United States]

Mixing Animals and Human Tissues? Go Ahead If It Cures, Public Tells Researchers
The majority of the general public is in favor of research using animals containing human material if doing so helps to improve human health and cure diseases, says a study. [Academy of Medical Sciences, United Kingdom]

'Very Significant' Cuts to Spanish Science Funding Coming: Minister
Spanish science has been shaken by a series of tremors of ill omen, say online reports. Word that current Spanish Science and Innovation Minister Cristina Garmendia may leave the government, that president Prime Minister José Luis Rodríguez Zapatero could dissolve her ministry and that the science budget will suffer yet another drastic cut have spread uncertainty about the future of science in Spain. [Ministry of Science and Innovation, Spain]

Government Plans Policy Options to Boost Pharma
The government is looking at policy options to strengthen India’s thriving pharmaceutical industry and check growing instances of takeovers by foreign players. [Commerce Department, India]

Government Sets Up Panel to Regulate Use of Stem Cells
The health ministry has approved and notified a committee to look at therapies related to stem cells and genes. [Ministry of Health, India]



B.C. Researchers Gain $3.1M for Rare Cancer Research
A team of researchers from the University of British Columbia, B.C. Cancer Agency and Vancouver Coastal Health Research Institute has received a $3.1-million grant from Terry Fox Foundation to study genetic aspects of rare cancers. [University of British Columbia Press Release]

Cellular Dynamics International, ACEA Biosciences, and Roche Applied Sciences Cooperate to Evaluate iCell Cardiomyocytes on the xCELLigence System
Cellular Dynamics International announced their partnership with ACEA Biosciences to bring early and accurate prediction of potential cardiotoxic side effects to the drug discovery process. [Cellular Dynamics International Press Release]

Genmab and Seattle Genetics Enter into Antibody-Drug Conjugate Research Collaboration
Genmab A/S and Seattle Genetics, Inc. announced that the companies have entered into an antibody-drug conjugate research collaboration agreement. [Seattle Genetics, Inc. Press Release]

Merck and BGI Sign Statement of Intent to Collaborate to Advance the Application of Genomics in Medicine
Merck and BGI announced the signing of a statement of intent to initiate and develop a working relationship. Under the agreement, Merck and BGI intend to explore areas of mutual interest in healthcare research and discovery with the common goal of creating value from the massive output of genomic information enabled by next-generation, high throughput DNA sequencing and analysis technologies. [BGI Press Release]

Johnson & Johnson and Crucell in Advanced Negotiations for an All Cash Public Offer of EUR24.75 per Ordinary Share of Crucell
Johnson & Johnson and Crucell N.V. announced that they are in advanced negotiations for a potential public offer by Johnson & Johnson or an affiliate for all outstanding ordinary shares of Crucell not already held by Johnson & Johnson and its affiliates. [Crucell N.V. Press Release]

Genzyme Announces Agreement to Sell Genetic Testing Business to LabCorp
Genzyme Corporation announced that it has entered into an asset purchase agreement under which Laboratory Corporation of America Holdings (LabCorp) will acquire Genzyme Genetics for $925 million in cash. [Genzyme Corporation Press Release]

Okairos Raises EUR16 Million in Series B Financing
Okairos announced the completion of a Series B private financing round totaling EUR16 million. [Okairos Press Release]

Third Rock Ventures Raises $426 Million Fund II
Third Rock Ventures, LLC announced the final closing of Fund II, raising $426 million in an oversubscribed fund. With Fund II, Third Rock will continue to execute on its strategy and build on its successful track record of launching and supporting transformative life sciences companies. [Third Rock Ventures, LLC Press Release]

Scripps Research Team Wins $5.1 Million to Develop DNA Sequencing Technology
Scripps Research Institute Professor Reza Ghadiri has been awarded a four-year, $5.1 million grant as part of a National Institutes of Health initiative to spur the development of the next generation of DNA sequencing technologies, which could enable biomedical researchers and health care workers to routinely sequence a person's DNA. [Scripps Research Institute Press Release]

Life Technologies Signs Strategic Out-Licenses for Recombinant Protein Bioproduction
Life Technologies Corporation announced that it has finalized cell line license agreements with a number of companies to provide rights to Life Technologies' proprietary CHO (Chinese hamster ovary) cell lines for the production of recombinant proteins used as therapeutic agents and vaccines. [Life Technologies Corporation Press Release]


NHGRI Funds Development of Third Generation DNA Sequencing Technologies
More than $18 million in grants to spur the development of a third generation of DNA sequencing technologies was announced today by the National Human Genome Research Institute (NHGRI).

Program Projects for Collaborative Research on the Basic Biology of Pluripotency and Reprogramming (P01) (RFA-GM-11-005)

Innovative Emerging Molecular Analysis Technologies (SBIR R43/R44) (RFA-CA-10-013)

Prospective Grant of Exclusive License: The Development of Immunotoxins/Targeted Toxins for the Treatment of Human Cancer (FR Doc. 2010-22844)

Prospective Grant of Exclusive License: Development of AAV5 Based Therapeutics to Treat Human Diseases (FR Doc. 2010-22833)

National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. 2010-23027)
National Cancer Institute; Notice of Closed Meeting (FR Doc. 2010-23144)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-23143)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2010-23275)



Agenda for the Public Workshop on Cell and Gene Therapy Clinical Trials in Pediatric Populations

Complete List of Licensed Products and Establishments




European Medicines Agency and U.S. Food and Drug Administration Extend Confidentiality Arrangements Indefinitely
The European Medicines Agency and the U.S. Food and Drug Administration have extended their confidentiality arrangements related to medicinal products for human and veterinary use, following the positive experience gained since the initial arrangements were signed in September 2003.

FDA to Review Medical Products Alongside Medicare
A new process to review medical products at the U.S. Food and Drug Administration in tandem with the nation's Medicare insurance program could help speed up coverage decisions.

Genentech Provides Update on Avastin for Advanced Breast Cancer
Genentech, Inc. announced that information submitted by the company to the U.S. Food and Drug Administration during the review of the supplemental Biologics License Applications for Avastin® (bevacizumab) for previously untreated advanced HER2-negative breast cancer has been deemed a major amendment.

Draft Guidance for Industry on Chronic Hepatitis C Virus Infection: Developing Direct-Acting Antiviral Agents for Treatment; Availability (Docket No. FDA-2010-D-0462)

Fee for Using a Priority Review Voucher in Fiscal Year 2011 (Docket No. FDA-2010-N-0463)


Information for Wholesale Dealers and Manufacturers: Advertising of Unlicensed Medicines
A change was made to the way in which appropriately authorized wholesale dealers and manufacturers can make healthcare professionals aware of the unlicensed medicines they have available.

EVENTS (Listed by Date)

First Annual BIO India International Partnering Conference
September 21-22, 2010
Hyderabad, India

Third Annual Maryland Stem Cell Research Symposium
September 22, 2010
Gaithersburg, United States

Cellular Therapy of Cancer Symposium – ATTACK Project Meeting
September 24-27, 2010
Montpellier, France

International Society for Cellular Therapy 2nd Annual Symposium on Stem Cell Translation: Best Practices & Regulatory Considerations
September 27-28, 2010
San Francisco, United States

International Society for Cell and Gene Therapy of Cancer (ISCGT) Congress
September 27-29, 2010
Doha, Qatar

4th American Association of Cancer Research International Conference on Molecular Diagnostics in Cancer Therapeutic Development
September 27-30, 2010
Denver, United States

Select Biosciences 6th Annual Ion Channel Targets Conference
September 28-29, 2010
Washington, DC, United States

NEW 5th Annual Meeting of the German Society for Stem Cell Research
September 30-October 2, 2010
Lubeck, Germany

Cytokines 2010: Cytokines in Infectious Diseases, Autoimmune Disorders and Cancer
October 3-7, 2010
Chicago, United States

2010 World Stem Cell Summit
October 4-6, 2010
Detroit, United States

9th Annual BIO Investor Forum
October 5-6, 2010
San Francisco, United States

5th World Congress on Preventive & Regenerative Medicine (WCRM)
October 5-7, 2010
Hannover, Germany

17th Annual Meeting German Society for Gene Therapy (DG-GT e.V.)
October 6-9, 2010
Munich, Germany

COGEM Symposium: GM Viruses as Medicine: Panacea or Pandora's Box?
October 7, 2010
Amsterdam, The Netherlands

NEW Induced Pluripotent Stem Cells: Production and Utility in Regenerative Medicine
October 7, 2010
Hertfordshire, United Kingdom

NEW Functional Stem Cell and Regeneration
October 9-10, 2010
Kansas City, United States

American Association of Blood Banks (AABB) Annual Meeting & Cellular Therapy and Transfusion Medicine Expo (CTTXPO) 2010
October 9-12, 2010
Baltimore, United States

18th Annual BioPartnering Europe
October 10-12, 2010
London, United Kingdom

3rd Biobridge “Generation Regeneration” Conference
October 11, 2010
Geneva, Switzerland

New York Stem Cell Foundation 5th Annual Translational Stem Cell Research Meeting
October 12-13, 2010
New York, United States

NEW 4th International Symposium Stem Cells, Development and Regulation
October 12-14, 2010
Amsterdam, Netherlands

Translational Cancer Research for Basic Scientists
October 17-22, 2010
Boston, United States

2010 NanoMedicine Summit
October 18-19, 2010
Cleveland, United States

BIO Intellectual Property Counsels Committee Fall Conference and Committee Meeting
October 18-20, 2010
Boston, United States

NEW Stem Cells and Regenerative Medicine – New Solutions to Old Problems
October 18-20, 2010
San Antonio, United States

6th Modern Drug Discovery & Development Summit (M3D)
October 20-22, 2010
San Francisco, United States

European Society of Gene and Cell Therapy (ESGCT) 18th Annual Congress
October 22-25, 2010
Milan, Italy

3rd Annual Dubai Congress on Anti-Aging and Aesthetic Medicine (DCAAAM)
October 26-27, 2010
Dubai, United Arab Emirates

NEW International Consensus Conference on Risk-Based Decision Making for Blood Safety
October 26-28, 2010
Toronto, Canada

World Stem Cells & Regenerative Medicine Congress Asia 2010
October 26-29, 2010
Singapore City, Singapore

Select Biosciences 3rd Annual Molecular Diagnostics World Congress
October 28-29, 2010
San Diego, United States

Fraunhofer Life Science Symposium 2010
October 29-30, 2010
Leipzig, Germany

NEW Public Workshop on Cell and Gene Therapy Clinical Trials in Pediatric Populations
November 2, 2010
Bethesda, United States

Stem Cell Society Singapore Symposium 2010: Translating Science to Therapy
November 2-3, 2010

BioMalaysia 2010 Conference and Exhibition
November 2-4, 2010
Kuala Lumpur, Malaysia

World Cord Blood Congress
November 4-7, 2010
Marseille, France

Select Biosciences 5th Annual European Biomarkers Summit
November 9-10, 2010
Florence, Italy

Select Biosciences International Forum on Stem Cells
November 12-13, 2010
Tianjin, China

Select Biosciences 2nd Annual RNAi Asia Conference
November 15-16, 2010
Singapore City, Singapore

BIO-Europe International Partnering Conference
November 15-17, 2010
Munich, Germany

NEW The 16th International Conference of the ISD (International Society of Differentiation)
November 15-18, 2010
Nara, Japan

9th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases
November 17-19, 2010
Sonoma, United States

8th Annual Commercial Translation of Regenerative Medicine Conference
November 18-19, 2010
London, United Kingdom

Stem Cell Network 10th Annual Scientific Meeting
November 22-24, 2010
Calgary, Canada

HealthTech and Medicines Knowledge Transfer Network 7th Annual BioProcessUK Conference
November 24-25, 2010
Manchester, United Kingdom

NEW Berlin-Brandenburg School for Regenerative Therapies (BSRT) PhD Symposium: Stem Cells — Hopes, Fears and Realities
December 1-3, 2010
Berlin, Germany

2nd European Association for Cardio-Thoracic Surgery (EACTS) Meeting on Cardiac and Pulmonary Regeneration
December 2-3, 2010
Vienna, Austria

Tissue Engineering & Regenerative Medicine International Society (TERMIS) – North America 2010
December 5-8, 2010
Orlando, United States

NEW American Society for Cell Biology 50th Anniversary Annual Meeting
December 11-15, 2010
Philadelphia, United States

Select Biosciences 5th Annual Stem Cells & Regenerative Medicine World Congress
January 24-25, 2011
San Diego, United States

BIO Asia International Partnering Conference
January 24-25, 2011
Tokyo, Japan

Phacilitate 7th Annual Cell & Gene Therapy Forum 2011
January 24-26, 2011
Washington DC, United States

NEW Keystone Symposia-Stem Cells in Development, Tissue Homeostasis and Disease
January 30-February 4, 2011
Santa Fe, United States

Visit our events page to stay up to date with the latest events in the cell, gene and immunotherapy community.



Cell Therapy Technologist (Opexa Therapeutics)

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