Category Archives: Gene Medicine

FRESNO, Calif. (KFSN) --

Six-year-old Andy Moorhead is learning how to read. But instead of using his eyes, he's using his fingers. Andy told ABC30, "Well, I read the letters with my fingers."

Andy is blind. Andy's Mother, Heather Ingram-Moorhead explained, "He was around nine months, and we started to notice his eyes were twitching."

Andy has leber congenital amaurosis, or LCA. It's the most common type of childhood blindness and is caused by genetic mutations.

"It is just very hard. It's taken us a while to really understand the condition and do everything to help Andy," Heather told ABC30.

Andy's whole family is hands-on. Even his sister Valerie gives him guidance. But despite their efforts, his mom says gene therapy is their only hope.

University of Florida scientist Shannon E. Boye, PhD, is using a $900,000 grant to perfect a gene therapy that could restore vision.

"It's not an attempt just to slow the progression of the disease. It's actually an attempt to halt the progression and make the patient better by delivering them the gene they don't have," Boye told ABC30.

Boye says the therapy has worked in animals. "We're able to show, via what's called an electra retinal gram, that the retinal function has been restored to the mice," she explained.

Gene therapy is still an investigational treatment with risks and only available for those in a clinical trial. Right now there are hundreds of studies underway to treat conditions like LCA, cancer and HIV. It's hope that one day Andy could put down his cane and see his family for the first time.

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Gene therapy: Hope for the blind?

Researchers at the Veterans Affairs San Diego Healthcare System and University of California, San Diego School of Medicine, with colleagues in New York and the United Kingdom, have identified genetic markers, derived from blood samples that are linked to post-traumatic stress disorder (PTSD). The markers are associated with gene networks that regulate innate immune function and interferon signaling.

The findings, published in the March 10 issue of the journal Molecular Psychiatry, offer novel insights into the pathophysiology of PTSD. In clinical terms, researchers say they could lead to new ways to not just improve diagnosis and treatment of persons with the mental health condition, but predict who might be more susceptible.

Previous genomic studies of PTSD have focused upon identifying differences in gene expression between persons with PTSD relative to a control group. The new study takes a broader "systems-level approach," using whole transcriptome RNA sequencing, said first author Michael S. Breen, PhD, at the University of Southampton in England.

"By comparing U.S. Marines who develop PTSD symptoms to those who do not, we can measure differences in genes, but also take into consideration the dynamic relationships between and among them, their connectivity," Breen said. "Because PTSD is thought to be such a complex disorder, measuring these dynamic relationships is crucial to better understanding the PTSD pathology."

The researchers analyzed blood samples from 188 U.S. Marines, taken before and after deployment to conflict zones. They identified modules of co-regulated genes involved in innate immune response - the body's first line of defense against pathogens - and interferon signaling, that were also associated with PTSD. Interferons are proteins released by host cells in response to the presence of pathogens and in this study are also shown to partake in the pathology PTSD.

The results were replicated with a second, completely independent group of 96 U.S. Marines.

"What's interesting is that molecular signatures of innate immunity and interferon signaling were identified both after developing PTSD as well as before developing PTSD," said Dewleen G. Baker, MD, MRS-II principal investigator, research director at the VA Center of Excellence for Stress and Mental Health, and professor in the Department of Psychiatry at UC San Diego.

The work, a sub-study of MRS-II, was co-led by Caroline M. Nievergelt, PhD, associate chief of the Neuroscience Unit at the VA Center of Excellence for Stress and Mental Health and assistant adjunct professor in the Department of Psychiatry at UC San Diego and the late Daniel T. O'Connor MD, departments of Medicine and Pharmacology at UC San Diego.

"The question to ask is what's stimulating an interferon response prior to PTSD development," said Baker. "The answer could be any number of factors, ranging from a simple explanation of increased anticipatory stress prior to deployment or more complex scenarios where individuals may have a higher viral load. It's a question for future studies."

Experts say what makes PTSD different - and more challenging to study - than other psychiatric disorders is the presence or trigger of a traumatic event, such as serving in a combat zone.

Continued here:
Gene networks for innate immunity linked to PTSD risk

New mechanism for engineering traits governed by multiple genes paves the way for various advances in genomics and regenerative medicine

(BOSTON) - When it comes to gene expression - the process by which our DNA provides the recipe used to direct the synthesis of proteins and other molecules that we need for development and survival - scientists have so far studied one single gene at a time. A new approach developed by Harvard geneticist George Church, Ph.D., can help uncover how tandem gene circuits dictate life processes, such as the healthy development of tissue or the triggering of a particular disease, and can also be used for directing precision stem cell differentiation for regenerative medicine and growing organ transplants.

The findings, reported by Church and his team of researchers at the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard Medical School in Nature Methods, show promise that precision gene therapies could be developed to prevent and treat disease on a highly customizable, personalized level, which is crucial given the fact that diseases develop among diverse pathways among genetically-varied individuals. Wyss Core Faculty member Jim Collins, Ph.D., was also a co-author on the paper. Collins is also the Henri Termeer Professor of Medical Engineering & Science and Professor in the Department of Biological Engineering at the Massachusetts Institute of Technology.

The approach leverages the Cas9 protein, which has already been employed as a Swiss Army knife for genome engineering, in a novel way. The Cas9 protein can be programmed to bind and cleave any desired section of DNA - but now Church's new approach activates the genes Cas9 binds to rather than cleaving them, triggering them to activate transcription to express or repress desired genetic traits. And by engineering the Cas9 to be fused to a triple-pronged transcription factor, Church and his team can robustly manipulate single or multiple genes to control gene expression.

"In terms of genetic engineering, the more knobs you can twist to exert control over the expression of genetic traits, the better," said Church, a Wyss Core Faculty member who is also Professor of Genetics at Harvard Medical School and Professor of Health Sciences and Technology at Harvard and MIT. "This new work represents a major, entirely new class of knobs that we could use to control multiple genes and therefore influence whether or not specific genetics traits are expressed and to what extent - we could essentially dial gene expression up or down with great precision."

Such a capability could lead to gene therapies that would mitigate age-related degeneration and the onset of disease; in the study, Church and his team demonstrated the ability to manipulate gene expression in yeast, flies, mouse and human cell cultures.

"We envision using this approach to investigate and create comprehensive libraries that document which gene circuits control a wide range of gene expression," said one of the study's lead authors Alejandro Chavez, Ph.D., Postdoctoral Fellow at the Wyss Institute. Jonathan Schieman, Ph.D, of the Wyss Institute and Harvard Medical School, and Suhani Vora, of the Wyss Institute, Massachusetts Institute of Technology, and Harvard Medical School, are also lead co-authors on the study.

The new Cas9 approach could also potentially target and activate sections of the genome made up of genes that are not directly responsible for transcription, and which previously were poorly understood. These sections, which comprise up to 90% of the genome in humans, have previously been considered to be useless DNA "dark matter" by geneticists. In contrast to translated DNA, which contains recipes of genetic information used to express traits, this DNA dark matter contains transcribed genes which act in mysterious ways, with several of these genes often having influence in tandem.

But now, that DNA dark matter could be accessed using Cas9, allowing scientists to document which non-translated genes can be activated in tandem to influence gene expression. Furthermore, these non-translated genes could also be turned into a docking station of sorts. By using Cas9 to target and bind gene circuits to these sections, scientists could introduce synthetic loops of genes to a genome, therefore triggering entirely new or altered gene expressions.

The ability to manipulate multiple genes in tandem so precisely also has big implications for advancing stem cell engineering for development of transplant organs and regenerative therapies.

The rest is here:
Activating genes on demand

FRESNO, Calif. (KFSN) --

Five-year-old Elizabeth Eastham has just finished a round of chemotherapy to treat kidney cancer. It's a tough battle, but Elizabeth's mom knows today's discoveries may bring tomorrow's hope.

"With what they find with your child can help another child later on would be fantastic," Elizabeth's mother, Angela Eastham told ABC30.

Dr. Hao Zhu is researching how pediatric cancers develop on the genetic level. He's pinpointed a gene that contributes to childhood cancers like neuroblastoma, Wilms tumor and liver cancer.

"And what we hope to do is to discover specific genetic targets for novel drugs to kill cancers without hurting the rest of the body," Hao Zhu, M.D., Assistant Professor Children's Research Institute at UT Southwestern told ABC30.

In laboratory mice, Dr. Zhu has found that the lin28 gene, which normally contributes to embryonic growth, also plays a role in cancer formation in fully developed juveniles.

Dr. Zhu explained, "We hope one day to be able to treat and diagnose cancers better in children."

"You don't want this for your child," said Eastham. "But you know that everything is in God's plan, and you know he saw us through this entire process and kept our strength up, and everyone's strength up around us to keep going and face one day at a time."

It may be years from the laboratory to the patient, but this discovery, published in the journal Cancer Cell, gives researchers hope that understanding how cancer works will lead to better treatments for children in the future.

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Cancer gene: Medicine's next big thing?

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Cancer medicine is entering a new era, using drugs that target specific mutations identified by gene sequencing of cancerous tumors.

WILLMAN: A different way of delivering medicine

The new techniques pose special challenges for cancer centers in small states like New Mexico, where patient numbers are small, said Dr. Cheryl Willman, director and CEO of the University of New Mexico Cancer Center.

The real challenge for our patients is, how do you get your hands on those drugs, because you are going to need a whole cabinet of targeted agents, Willman said.

Part of the answer involves collaborating with other institutions to pool the genetic data from large numbers of patients, she said.

UNM Cancer Center announced recently that it has joined a research collaboration with five other U.S. cancer centers to pool genetic data of cancerous tumors and more quickly match patients to targeted treatments and drug trials.

For New Mexico, the collaboration is expected to help attract partnerships with drug companies that require large numbers of cancer patients to validate the results of drug trials, Willman said.

Cancer medicine is going through a huge transformation, which is to do comprehensive sequencing of each patients tumor, identify the mutations that are present, then pick the drug that really is targeting those mutations, she said.

UNM Cancer Center also plans this year to begin a nationwide study of leukemia patients. UNM will genetically sequence cancerous tumors for each of some 4,000 U.S. patients diagnosed with the blood cancer each year in search of mutations that can be targeted for drug therapies, Willman said.

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Cancer centers facing new challenges, NM doctor says

When it comes to gene expression -- the process by which our DNA provides the recipe used to direct the synthesis of proteins and other molecules that we need for development and survival -- scientists have so far studied one single gene at a time. A new approach developed by Harvard geneticist George Church, Ph.D., can help uncover how tandem gene circuits dictate life processes, such as the healthy development of tissue or the triggering of a particular disease, and can also be used for directing precision stem cell differentiation for regenerative medicine and growing organ transplants.

The findings, reported by Church and his team of researchers at the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard Medical School in Nature Methods, show promise that precision gene therapies could be developed to prevent and treat disease on a highly customizable, personalized level, which is crucial given the fact that diseases develop among diverse pathways among genetically-varied individuals. Wyss Core Faculty member Jim Collins, Ph.D., was also a co-author on the paper. Collins is also the Henri Termeer Professor of Medical Engineering & Science and Professor in the Department of Biological Engineering at the Massachusetts Institute of Technology.

The approach leverages the Cas9 protein, which has already been employed as a Swiss Army knife for genome engineering, in a novel way. The Cas9 protein can be programmed to bind and cleave any desired section of DNA -- but now Church's new approach activates the genes Cas9 binds to rather than cleaving them, triggering them to activate transcription to express or repress desired genetic traits. And by engineering the Cas9 to be fused to a triple-pronged transcription factor, Church and his team can robustly manipulate single or multiple genes to control gene expression.

"In terms of genetic engineering, the more knobs you can twist to exert control over the expression of genetic traits, the better," said Church, a Wyss Core Faculty member who is also Professor of Genetics at Harvard Medical School and Professor of Health Sciences and Technology at Harvard and MIT. "This new work represents a major, entirely new class of knobs that we could use to control multiple genes and therefore influence whether or not specific genetics traits are expressed and to what extent -- we could essentially dial gene expression up or down with great precision."

Such a capability could lead to gene therapies that would mitigate age-related degeneration and the onset of disease; in the study, Church and his team demonstrated the ability to manipulate gene expression in yeast, flies, mouse and human cell cultures.

"We envision using this approach to investigate and create comprehensive libraries that document which gene circuits control a wide range of gene expression," said one of the study's lead authors Alejandro Chavez, Ph.D., Postdoctoral Fellow at the Wyss Institute. Jonathan Schieman, Ph.D, of the Wyss Institute and Harvard Medical School, and Suhani Vora, of the Wyss Institute, Massachusetts Institute of Technology, and Harvard Medical School, are also lead co-authors on the study.

The new Cas9 approach could also potentially target and activate sections of the genome made up of genes that are not directly responsible for transcription, and which previously were poorly understood. These sections, which comprise up to 90% of the genome in humans, have previously been considered to be useless DNA "dark matter" by geneticists. In contrast to translated DNA, which contains recipes of genetic information used to express traits, this DNA dark matter contains transcribed genes which act in mysterious ways, with several of these genes often having influence in tandem.

But now, that DNA dark matter could be accessed using Cas9, allowing scientists to document which non-translated genes can be activated in tandem to influence gene expression. Furthermore, these non-translated genes could also be turned into a docking station of sorts. By using Cas9 to target and bind gene circuits to these sections, scientists could introduce synthetic loops of genes to a genome, therefore triggering entirely new or altered gene expressions.

The ability to manipulate multiple genes in tandem so precisely also has big implications for advancing stem cell engineering for development of transplant organs and regenerative therapies.

"In order to grow organs from stem cells, our understanding of developmental biology needs to increase rapidly," said Church. "This multivariate approach allows us to quickly churn through and analyze large numbers of gene combinations to identify developmental pathways much faster than has been previously capable."

Continued here:
Activating genes on demand: Possible?

DUBLIN, Mar. 03, 2015 /PRNewswire/ --Research and Markets

(http://www.researchandmarkets.com/research/rcv4lq/gene_therapy) has announced the addition of the "Gene Therapy Market, 2015 - 2025" report to their offering.

The "Gene Therapy Market, 2015-2025" report provides an extensive study on the marketed and pipeline gene therapies. A lot of research has been carried out in this field for over a decade but there are only five approved therapies (four available in Asian markets; one approved in the EU). There are many promising therapies which are currently being developed worldwide; the approach is likely to result in several commercial success stories in the foreseen future. The report covers various aspects, such as key players, marketed gene therapy products, products in clinical / pre-clinical research, associated ethical issues, likely future developments and upcoming opportunities for a variety of stakeholders.

Several disorders that arise inside the body are a result of either a direct genetic aberration or a dysfunctional/non-functional protein. The attempt to use nucleic acids to correct or delete the genes causing a particular disease is known as gene therapy. Although gene therapy has not contributed significantly to the global pharmaceutical market yet, it is anticipated to grow at a fast pace over the next decade.

Gendicine, developed by SiBiono GeneTech, was the foremost gene therapy that entered market in 2004 in China. Since then four more therapies have received approval in China, Philippines, Russia and the EU. This number for approved / marketed therapies seems weak at present; however, the strong and highly populated pipeline holds tremendous potential. There are 12 gene therapies in late stage of clinical development for the treatment of cancer, ocular and cardiovascular disorders.

There are several concerns that remain to be answered; examples include insertional mutagenesis, treatment of multigene disorders, curbing the risk of immune reactions, eugenics, high cost of therapy and ethical concerns related to making alterations at the genetic level. Despite this, gene therapy does offer a ray of hope for patients who either have no treatment options or show no benefits with drugs that are currently available. Such a benefit far outweighs any disadvantages that may be associated with this upcoming therapeutic field.

Key Topics Covered:

1. Preface

2. Executive Summary

3. Introduction

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Global Gene Therapy Market Report 2015-2025 - Extensive Study on the Marketed and Pipeline Gene Therapies

DUBLIN, Mar. 03, 2015 /PRNewswire/ --Research and Markets

(http://www.researchandmarkets.com/research/gxqhg9/gene_therapy) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2014, over 2050 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the geographical areas and applications.

Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2014-2024. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

The voluminous literature on gene therapy was reviewed and selected 750 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 75 tables and 15 figures.

Profiles of 181 companies involved in developing gene therapy are presented along with 223 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade.

Key Topics Covered:

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Gene Therapy Market Report 2014-2024 - Technologies, Markets and Companies

Mason, OH (PRWEB) March 03, 2015

The combinatorial, multi-gene GeneSight test has been found to better predict antidepressant treatment outcomes for patients with depression, and their use of health care resources, than any of the individual genes that comprise the test, according to a peer-reviewed analysis by investigators from the Mayo Clinic and Assurex Health, and published online by The Pharmacogenomics Journal i.

The proprietary technology of the GeneSight Psychotropic test is based on combinatorial pharmacogenomics (CPGx), the study of how variations in multiple genes collaborate to influence an individuals response to medications, and evidence-based medicine and the known clinical pharmacology of various drugs.

This new publication shows that the combinatorial GeneSight test predicts which patients are likely to experience poorer antidepressant outcomes and use more health care services, whereas single gene diagnostics mostly did not, said lead author and Assurex Health Senior Vice President, C. Anthony Altar, Ph.D. The robust evidence from these analyses reinforce the advantage of the combinatorial GeneSight test in helping clinicians guide antidepressant and anti-anxiety treatment decisions. This and other features of GeneSight distinguish our pharmacogenomic products from all others.

The GeneSight Psychotropic test helps inform clinicians treatment selection for commonly prescribed medications including those for depression, post-traumatic stress disorder (PTSD), anxiety, bipolar disorder and schizophrenia. The test is covered by Medicare, the U.S. Department of Veterans Affairs, and a growing number of commercial payers.

Evaluating Drug Metabolism with Genetic Data

The CPGx approach that generates the GeneSight report examines DNA variations of multiple genes since these variations can change the efficacy, metabolism, and adverse effects of many psychiatric drugs. Using a patients unique genetics, the GeneSight Psychotropic test creates a personalized report that places 38 U.S. Food and Drug Administration (FDA)-approved medications for depression and other mental health conditions into three color-coded categories for clinicians to review: Use as Directed in green, Use with Caution in yellow, or Use with Increased Caution and with More Frequent Monitoring in red. The GeneSight report also alerts healthcare providers to the implications of the patients genetic information to a drugs dosage, and FDA-approved package insert information. Most single gene tests have high variability and are less accurate in predicting patient responses to psychotropic medications. The GeneSight approach compensates for these limitations by aggregating predictions by the drug metabolism and response genes to better predict patients responses.

Nearly 90 percent of antidepressant and antipsychotic medications are metabolized by at least two of the liver cytochrome P450 (CYP) enzymes, and many interact with the brain serotonin transporter (SLC6A4) or the serotonin 2A receptor (HTR2A), explained the authors. The GeneSight Psychotropic test accounts for this complexity by measuring and combining the DNA sequence variations within drug response and drug metabolism genes. This analysis looked at the GeneSight test that included the liver metabolism genes CYP2D6, CYP2C19, CYP2C9, and CYP1A2, and the two drug response genes, SLC6A4 and HTR2A.

Since these studies were conducted, Assurex Health has enhanced the GeneSight test to include two more genes, CYP3A4 and CYP2B6, making it the first and only psychiatric pharmacogenomic test to offer CYP3A4 analysis distinct and separate from CYP3A5. The CYP2B6 gene affects medications including bupropion (Wellbutrin), the third most commonly prescribed antidepressant.

GeneSight Outperforms Single Gene Tests in Predicting Patient Outcomes

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GeneSight Multi-Gene Combinatorial Pharmacogenomic (CPGx) Test is More Predictive of Antidepressant Response than ...

Edward Lanphier, President, Chief Executive Officer, Sangamo BioSciences; Jeffrey Walsh, Chief Operating Officer, bluebird bio; and Karen Kozarsky, Ph.D., Vice President, Research & Development, ReGenX Biosciences named as co-chairs

-- The Alliance for Regenerative Medicine and the American Society of Gene & Cell Therapy partner to support policies to advance novel gene therapies --

WASHINGTON, DC and SALT LAKE CITY, May 20, 2014 The Alliance for Regenerative Medicine (ARM) and the American Society of Gene & Cell Therapy (ASGCT) today announced their partnership. ARMs new Gene and GeneModified Cell Therapy Section (GTS) brings together the leading gene therapy companies and organizations in the U.S. and Europe to advocate for policies and programs to accelerate the development of new therapeutics to treat and cure a range of diseases for which no effective treatment options are available. ARMs focus on advocacy and clinical and commercial development will create a powerful alliance with the deep scientific and translational expertise resident in ASGCT.

The new ARM Gene Therapy Section is one of three technology sections that comprise the ARM membership. The other two sections focus on Cell Therapy and Tissue Engineering and Biomaterials. The Gene Therapy Section will dedicate its efforts to addressing regulatory, manufacturing, commercial and financial issues crucial to the success of the sector. In addition, the GTS will focus on building public awareness for this field of medicine and an appreciation for its potential to transform healthcare. The GTS will be led by Sarah Haecker, Ph.D., a member of ARMs senior staff. Sarah received her Ph.D. in Molecular Biology and Bioethics (with focus on gene transfer applications) and her postdoctoral scientific and business training in the Human Gene Therapy Program and the Center for Technology Transfer at the University of Pennsylvania. The three co-chairs of the group are Edward Lanphier, President, Chief Executive Officer, Sangamo BioSciences; Jeffrey Walsh, Chief Operating Officer, bluebird bio; and Karen Kozarsky, Ph.D., Vice President, Research & Development, ReGenX Biosciences.

As ARMs membership has grown, the organization has created specific technical sub-groups, such as the GTS, to focus on the unique development and commercialization needs of major sectors within advanced therapies said Lanphier. The addition of this new technology section is particularly exciting as the gene therapy field is making tremendous progress and holds great promise for transforming the lives of so many patients. It is our hope that gathering this group of technical, clinical and commercial experts in the field will help to accelerate product development and commercialization of these innovative technologies.

ASGCTs mission to bring together diverse stakeholders and advance the field of genetic and cellular therapies is closely aligned with ARMs goals, and we are looking forward to working with the organization, said Harry L. Malech, M.D., President-Elect of ASGCT. ARMs staff and members bring a wealth of knowledge involving regulatory and commercialization challenges in the field, and we feel this will nicely complement ASGCTs scientific and medical expertise.

ARM is the leading advocacy organization in the U.S. and Europe representing companies and organizations focused in the regenerative medicine and gene therapy field, and serves as an invaluable resource for all of its members. ASGCT, a nonprofit medical and scientific organization focused on genetic and cellular therapies, recently became a member of ARM and the two groups will work together to lead the advocacy and education efforts for the GTS of ARM.

Members of ARMs Gene Therapy Section: Abeona Therapeutics, AGTC, Alpha-1 Foundation, ALS Association, Association of Clinical Research Organizations (ACRO), American Society of Gene & Cell Therapy, Baxter/Chatham Therapeutics, Benitec Ltd., bluebird bio, Calimmune, Celgene Corporation, CIRM, Cornell University, Friends of Cancer Research, GenVec, Genzyme-Sanofi, Global Biotherapeutics, Juventas Therapeutics, MaxCyte, Memorial Sloan Kettering Cancer Center, National Multiple Sclerosis Society (NMSS), NeoStem, Oxford Biomedica, Parkinsons Action Network, Progenitor Cell Therapy (PCT), Pfizer, Prevent Cancer Foundation, ReGenX, Sangamo BioSciences, Shire, SironRX Therapeutics, Stop ALD, TissueGene, UniQure and Voyager Therapeutics

About The American Society of Gene & Cell Therapy: The American Society of Gene & Cell Therapy (ASGCT) is a professional nonprofit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website at http://www.asgct.org.

About the Alliance for Regenerative Medicine: The Alliance for Regenerative Medicine (ARM) is a Washington, DC-based multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, DC to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today ARM has more than 150 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

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Gene Therapy Section Formed Within the Alliance for ...