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— Pipeline report on Sickle Cell Disease - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Sickle Cell Disease (Hematological Disorders). The complete is analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. Browse the 54 Tables and 11 Figures, 36 Company Profiles, Spread across 191 Pages Report Available at http://www.reportsnreports.com/reports/992906-sickle-cell-disease-pipeline-review-h1-2017.html . Sickle Cell Disease - Companies Involved in Therapeutics Development - Acceleron Pharma Inc, Addex Therapeutics Ltd, Addmedica SAS, Advinus Therapeutics Ltd, Alnylam Pharmaceuticals Inc, Angiocrine Bioscience Inc, ArQule Inc, Bio Products Laboratory Ltd, Bioverativ Inc, bluebird bio Inc, Bristol-Myers Squibb Company, Calimmune Inc, CRISPR Therapeutics, CSL Ltd 35,Editas Medicine Inc, Errant Gene Therapeutics LLC, Gamida Cell Ltd, Genethon SA, Gilead Sciences Inc, Global Blood Therapeutics Inc, Incyte Corp, Johnson & Johnson, La Jolla Pharmaceutical Company, MaxCyte Inc, Merck & Co Inc, Modus Therapeutics Holding AB, Morphogenesis Inc, NKT Therapeutics Inc, Novartis AG, Orphagen Pharmaceuticals Inc, Pfizer Inc, Prolong Pharmaceuticals LLC, Protagonist Therapeutics Inc, ReveraGen BioPharma Inc, Sancilio & Company Inc, Sangamo Therapeutics Inc. Sickle cell anemia is a genetic (inherited) blood disorder in which red blood cells, which carry oxygen around the body, develop abnormally. Signs and symptoms include anemia, delayed growth, vision problems, pain and frequent infections. Treatment includes antibiotics, pain relievers, blood transfusion and stem cell transplant. The Sickle Cell Disease (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Sickle Cell Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 5, 5, 9, 27, 6 and 1 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 1, 5 and 2 molecules, respectively. Place Order to This Report at http://www.reportsnreports.com/purchase.aspx?name=992906 Sickle Cell Disease (Hematological Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Scope • The pipeline guide provides a snapshot of the global therapeutic landscape of Sickle Cell Disease (Hematological Disorders). • The pipeline guide reviews pipeline therapeutics for Sickle Cell Disease (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources. • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. • The pipeline guide reviews key companies involved in Sickle Cell Disease (Hematological Disorders) therapeutics and enlists all their major and minor projects. • The pipeline guide evaluates Sickle Cell Disease (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. • The pipeline guide encapsulates all the dormant and discontinued pipeline projects. • The pipeline guide reviews latest news related to pipeline therapeutics for Sickle Cell Disease (Hematological Disorders) About Us: ReportsnReports.com is your single source for all market research needs. Our database includes 500,000+ market research reports from over 95 leading global publishers & in-depth market research studies of over 5000 micro markets. With comprehensive information about the publishers and the industries for which they publish market research reports, we help you in your purchase decision by mapping your information needs with our huge collection of reports. For more information, please visit http://www.reportsnreports.com/reports/992906-sickle-cell-disease-pipeline-review-h1-2017.html


WASHINGTON, DC--(Marketwired - May 9, 2017) - The Alliance for Regenerative Medicine (ARM) today released the agenda for the inaugural Cell & Gene Exchange, the only partnering conference that brings together the regulatory, business and patient advocacy communities across the cell and gene therapy sectors. Co-produced by ARM and EBD Group and held in partnership with Global Genes, the event will take place May 22 and 23 in Washington, D.C. "Our industry is on the cusp of some truly remarkable achievements, including some of the first significant sector product approvals," said Morrie Ruffin, managing director of ARM. "With these anticipated near-term milestones in mind, this event provides attendees timely regulatory and commercial content, as well as numerous business development and partnering opportunities." This first-ever Cell & Gene Exchange provides access to more than 200 potential partners across the field, enabling attendees to connect directly with drug development companies, manufacturers, investors and patient groups. The event also features presentations by 16 leading therapeutic developers and patient advocacy organizations, as well as numerous panels and roundtables offering insights from industry leaders on current trends in dealmaking, critical development benchmarks, commercialization, regulatory and reimbursement updates and more. Presenting Companies: Abeona Therapeutics, Asymmetrex, bBHC-STRI, Be the Match, Biostage, BrainStorm Cellular Therapeutics, Calimmune, Cellular Dynamics International, Cesca Therapeutics, Fibrocell, Genethon, MEDIPOST, ORIG3N, Research Cancer Institute of America, Sigilon and Universal Cells The event's most up-to-date agenda is available here. Additional speakers and presenting companies will be added in the coming weeks. Registration includes access to the ARM Annual Dinner on May 23 at the Willard InterContinental Hotel in Washington, D.C. This intimate dinner celebrates the field's major accomplishments over the past year, recognizing key corporate, scientific and policy leaders in the sector, including members of Congress, patient advocates, disease-foundation executives and other industry champions. The Cell & Gene Exchange will take place May 22 & 23, 2017 at the Ronald Reagan Building, 1300 Pennsylvania Avenue NW, Washington, D.C. 20004. For more information about the Cell & Gene Exchange and to register, please visit the event website here. For members of the media interested in attending, please contact Lyndsey Scull, lscull@alliancerm.org. About The Alliance for Regenerative Medicine The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. 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, D.C. 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 250 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.


The present invention provides an expression vector for preventing or inhibiting HlV entry, fusion or replication in mammalian cells. In particular, the invention provides a recombinant retroviral vector that encodes an inhibitor of a HlV co-receptor, such as CCR5 or CXCR4, and a protein that inhibits HIV fusion to target cells and/or HIV replication. Pharmaceutical compositions comprising such constructs and methods of use thereof to prevent or treat HIV infection in a patient are also disclosed.


The present invention provides an expression vector for preventing or inhibiting HIV entry, fusion or replication in mammalian cells. In particular, the invention provides a recombinant retroviral vector that encodes an inhibitor of a HIV co-receptor, such as CCR5 or CXCR4, and a protein that inhibits HIV fusion to target cells and/or HIV replication. Pharmaceutical compositions comprising such constructs and methods of use thereof to prevent or treat HIV infection in a patient are also disclosed.


The present invention provides an expression vector for preventing or inhibiting HIV entry, fusion or replication in mammalian cells. In particular, the invention provides a recombinant retroviral vector that encodes an inhibitor of a HIV co-receptor, such as CCR5 or CXCR4, and a protein that inhibits HIV fusion to target cells and/or HIV replication. Pharmaceutical compositions comprising such constructs and methods of use thereof to prevent or treat HIV infection in a patient are also disclosed.


Trademark
Calimmune | Date: 2016-08-03

Gene therapy products, namely, treatments for diseases affecting the immune system or hematologic system; Pharmaceuticals for the treatment of diseases affecting the immune system or hematologic system. Promoting public interest and awareness of gene therapies and pharmaceuticals; Promoting the exchange of information and resources within the scientific research and medical communities to achieve advances in the field of gene therapy. Providing medical and scientific research information in the fields of pharmaceuticals and genetics.


Trademark
Calimmune | Date: 2016-08-03

Gene therapy products, namely, treatments for diseases affecting the immune system or hematologic system; Pharmaceuticals for the treatment of diseases affecting the immune system or hematologic system. Promoting public interest and awareness of gene therapies and pharmaceuticals; Promoting the exchange of information and resources within the scientific research and medical communities to achieve advances in the field of gene therapy. Providing medical and scientific research information in the fields of pharmaceuticals and genetics.


TUCSON, Ariz.--(BUSINESS WIRE)--Calimmune, Inc., a clinical-stage gene therapy company, today announced a license agreement with Cincinnati Children’s Hospital Medical Center (Cincinnati Children’s) to develop and commercialize gene therapies combining Calimmune’s Select+™ technology with Cincinnati Children’s proprietary gene therapy construct for the treatment of patients with sickle cell disease and beta thalassemia. Calimmune’s Select+™ platform aims to improve engraftment of stem cell gene therapies through a post-cellular infusion selection process. “The combination of Cincinnati Children’s proprietary lentiviral construct with Calimmune’s Select+ technology is a powerful innovative step in the fight against debilitating hematologic conditions such as sickle cell disease and beta thalassemia,” said Salim Yazji, M.D., chief medical officer of Calimmune. “We are applying our unique experience in ex vivo lentiviral vector gene therapy to make treatments for hemoglobinopathy more effective, less toxic, and ultimately more accessible to the patients that need them.” “Our current clinical vector has been optimized to efficiently correct hematopoietic stem cells in patients suffering from sickle cell disease, so that the cells are able to produce the hemoglobin our circulatory system requires to function properly,” said Punam Malik, M.D., the Marjory J. Johnson chair of gene and cell therapy and director of the Cincinnati Comprehensive Sickle Cell Program at Cincinnati Children’s. “This collaboration with Calimmune allows us to create long-term or permanent solutions for patients with these diseases who are facing shortened life expectancy and reduced quality of life - even with the benefit of current therapies.” Monogenic diseases such as hemoglobinopathies are prime targets for gene therapy due to their high prevalence, significant morbidity and mortality, and the resulting high cost of medical care that indicate that gene therapy can greatly improve patient outcomes and significantly reduce associated medical costs. Calimmune’s approach for the treatment of sickle cell disease involves introduction of the gamma-globin gene (known to have anti-sickling properties) into the patient’s own hematopoietic stem cells (HSCs) via a self-inactivating lentiviral vector. Following introduction of the gamma-globin gene, Calimmune’s Select+™ technology will be used to positively select for the modified HSCs, thus increasing the population of modified versus unmodified cells in the patient’s system. Sickle cell disease and beta thalassemia are among the most common genetic defects worldwide. In the United States alone it is estimated that at least 2 million people carry one gene encoding for sickle cell anemia. Lentiviral vector delivery of a gamma-globin gene therapy to autologous HSCs holds great promise in creating healthy, long-lived HSCs in patients suffering from these currently incurable genetic diseases. Calimmune’s proprietary Select+™ technology is a safe tool aimed at driving selection of the genetically modified stem cells once they are given back to patients, to improve potency and lower toxicity. One of the historical challenges for gene therapy is achieving a high enough engraftment of stem cells in the bone marrow to reach the relevant therapeutic window. Toxic conditioning regimens used to drive engraftment of gene modified cells cause a list of adverse events that often require hospitalization and have additional long-term risks. Calimmune has focused on and made significant investments to solve this issue with Select+™. By coupling Select+™ with lentiviral therapeutic applications, we aim to reduce the conditioning regimens, increase efficacy, and improve the patient experience, ultimately making stem cell gene therapy an out-patient modality. Calimmune is accelerating the promise of gene therapy to liberate patients from chronic and currently incurable diseases. To achieve our ambitious goal, we have built a suite of technologies to advance the delivery, manufacturing, and overall efficiency of these life-changing medicines. Calimmune’s lead development programs are novel ex vivo gene therapies for hematologic diseases.


News Article | November 17, 2016
Site: www.businesswire.com

TUCSON, Ariz.--(BUSINESS WIRE)--Calimmune, Inc., a clinical-stage gene therapy company, has made a key addition to its leadership team with the appointment of Salim Yazji, M.D. as executive vice president and chief medical officer. Dr. Yazji was most recently the vice president and global therapeutics head of oncology for Baxalta, Inc. “Salim’s depth of experience and proven leadership in shepherding innovative products from pre-clinical development through product approval will be vital to Cal

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