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Paris, France

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Paris, France
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Science will have a bigger voice in the next French government. Newly elected President Emmanuel Macron announced yesterday that a molecular geneticist–turned–university administrator will head the new ministry of higher education, research, and innovation, while a highly respected physician-scientist is France’s new health minister. Both are women—as is fully half of the new cabinet. But perhaps the biggest surprise was the appointment of the immensely popular green activist Nicolas Hulot at the new Ministry of “Ecological and Solidarity-based Transition.” Hulot—who has called Donald Trump’s retreat from the Clean Power Plan “a crime against humanity” and who wants to phase out nuclear energy—is credited with major changes in French environmental policy in the past decade—but always from outside the government. Frédérique Vidal, 53, the new research minister for science, spent most of her career at the University of Nice Sophia Antipolis, where she increasingly focused on education and climbed through the administrative ranks until becoming university president in 2012. The fact that Vidal “knows the sector … is a good thing,” says Patrick Monfort, secretary general of SNCS-FSU, a trade union for researchers based near Paris. The French Conference of University Presidents (CPU) welcomed Vidal’s appointment in a public statement yesterday. CPU says it's also an “excellent signal to the university community” that research and higher education once again have a full-fledged ministry, after being relegated to the level of secretary of state by former President François Hollande in 2014. Macron, who has promised to kick-start the economy through science, also added the word “innovation” to the ministry’s title. The other researcher in the new administration is Agnès Buzyn, who will head the health ministry—a post Macron had promised to give to a physician. Aged 54, Buzyn spent most of her career as a clinical hematologist studying leukemia and bone marrow transplantation at Paris Descartes University and the Necker Hospital. A former president of the French National Cancer Institute, Buzyn has played many high-level science policy roles and is highly respected among her peers. But as French newspaper reported yesterday, some have criticized her for questioning the need for scientists working with the pharmaceutical industry to declare their conflicts of interest. And as it happens, she has a bit of a conflict herself: Buzyn is married to Yves Lévy, who leads the €1 billion French National Institute of Health and Medical Research (INSERM), which is jointly overseen by the research and health ministries. Le Monde reports that Buzyn will not handle any issues related to INSERM, but details of the arrangement are unclear. As the paper points out, the ministry interacts with the institute on an almost daily basis. Meanwhile, the appointment of Hulot, 62, has excited French friends of the environment. A former nature documentary maker, Hulot arrived at the first cabinet meeting today in an electric car and without a tie. He instigated the inclusion of an environmental charter in the French constitution in 2005 and triggered a national policy debate that led to two new environmental laws in 2008 and 2010 that seek to drastically reduce greenhouse gas emissions, promote renewable energy, and better regulate pesticides. As a special envoy of the French president for the protection of the planet, he helped prepare the 2015 Paris climate conference. According to French newspaper yesterday, Hulot hopes to reform the tax system to make production and consumption more sustainable, and to set in motion a transition toward sustainable energy. He also wants to start a national debate on sustainable food production. Hulot toyed with the idea of running for president himself in the last three presidential elections, but has repeatedly declined positions in previous governments. The big question is how much of his agenda he can deliver in Macron’s centrist administration, however. Monfort—who studies the impact of climate change on pathogenic water bacteria at the University of Montpellier—hopes Hulot’s nomination means that he “has secured some guarantees” about what he will be able to do. Update, 19 May, 6.30 a.m.:  The paragraph about Buzyn's conflict of interest has been updated.


News Article | May 15, 2017
Site: www.businesswire.com

Genomic Vision (Paris:GV) (Euronext: FR0011799907 – GV), a company specialized in the development of diagnostic tests for the early detection of cancers and genetic diseases, today reports of its first R&D Day who took place on May 10, to the Imagine Institute (Necker Hospital) in Paris, in front of a panel of individual and institutional investors, financial analysts and journalists. This first event was aimed at presenting a global overview of the IVD activities of the Company with its historical industrial partner Quest Diagnostics and with several academic teams. During an inaugural speech, Stanislas Lyonnet, Ph.D., head of Imagine Institute, directeur de l’Institut Imagine, called back the commitment of the Institute in the research on genetic pediatric diseases and the outlines of the partnership with Genomic Vision. “Our Institute is a reference center for the molecular combing technology use since almost two years. This technology can meet our stringent requirements: establish a precise and early diagnosis of the pathology, identify the genes and the mechanisms involved, evaluate the different therapeutic options and transform the patient’s healthcare.” Subsequently, Jay Wohlgemuth, M.D., Senior VP, CMO of Quest Diagnostics and Edward Ginns, M.D, Ph.D, Medical director - Neurology of Quest Diagnostics, presented the move of Quest strategy from a lab company to an added value diagnostic service provider and reminded the importance for Quest of investing in new products and technologies. He therefore insisted on the strong link between both companies by using the molecular combing technology as well as co-developing diagnostic tests. Jay Wohlgemuth declared: “We at Quest Diagnostics have been collaborating with GV for over seven years and we are highly committed to our collaboration through providing samples and data to develop applications for the DNA combing technology. Applications take the form of biomarkers for pharmaceutical development and as a clinical diagnostics tool for genetic diseases. Our first success is the development of the FSHD combing testing as a standard in the U.S. I’m committed to continue our collaboration with GV.” About the development of the BRCA test in the breast and ovarian cancer early detection, Jay Wohlgemuth specified: “The DNA combing technology has been used to explore a BRCA test for hereditary breast and ovarian cancer. We are currently performing a clinical study with GV using Quest Diagnostic’s samples and that process is ongoing. When data is available, it will be made public.” Aaron Bensimon, CEO and co-founder of Genomic Vision, indicated: « For the development of such a predisposition test, the environment profoundly evolved during the last years. BRCA test was initiated in 2012 and we had focused our analysis on the large rearrangements of BRCA1 and BRCA2 genes. Today, we know that the screening tests for breast hereditary cancer are evaluating a wider panel of more than 30 genes. Our partner Quest Diagnostics and Genomic Vision have to reposition the BRCA test on this basis and this is on what we work at the moment.” On the SMA diagnostic test, the representatives of Quest Diagnosis explained: “SMA, spinal muscular atrophy, is an hereditary complex disease with a large portion of healthy carrier of the recessive gene responsible for one of the most common muscular dystrophies in the US and around the world. Through our collaboration with Genomic Vision we believe that we will be able to uncover biomarkers crucial to the detection of potential carriers of this disease. To achieve Quest Diagnostics are collaborating through the provision of samples to fully characterize the SMA genomic region. This work is ongoing and we hope to have results over the coming year.” Pr Nicolas Levy, Head of Medical Genetics department at the Children's Hospital La Timone (Marseille, France) was next to speak for its presentation on the diagnostic approaches in FSHD by molecular combing use. « Facioscapulohumeral Muscular Dystrophy, the 3rd most spread myopathy, is perfect to demonstrate the benefits of the molecular combing technology. This one allows to update the genetic complexity of this disease while the other technologies currently used, including Next-Generation sequencing ones, don’t answer all the expectations. Off course, these NGS will evolve in the future but in our department we study pathologies which remain undetectable by them. Moreover, the molecular combing allows research in other pathologies, as the children leukemia”. Finally, Dr Petr Janda, CEO of PCS (Prague Clinical Services), the CRO in charge of the HPV clinical trial in Czech Republic and Dr Anne Jacquet, Director of Biomedical Research of Genomic Vision, presented the interim results of EXPL-HPC-002 study (http://www.genomicvision.com/wp-content/uploads/CP_GV_10-mai_HPV_FINAL-1.pdf). Dr Petr Janda reminded: “The current diagnosis tests of the cervical cancer are limited in terms of sensibility or specificity.” Beyond the promising results presented by the Genomic Vision’s HPV test, both speakers explained: “The use of the molecular combing allowed, for the first time, to visualize, to characterize and to quantify the number of HPV genomes integrated in the DNA of the female patients. This opens a new way in the diagnosis and the follow-up of the patients having a risk of cervical cancer associated with HPV virus by allowing the selection between the patients who are infected by the HPV virus but who will naturally eliminate it without developing a cancer and those who will require an appropriate care considering the rates of virus integration.” Through the presentation of the different IVD programs of Genomic Vision, Aaron Bensimon concluded: “The use of molecular combing presents a new paradigm on a large range of applications. This potential encourages us to build closer relationships with the clinicians, who are at the heart of the diagnosis of the genetic diseases and facing its deadlocks. Thanks to closer relationships with clinicians, we will be able to develop new tests, like we are doing for the SMA test." The whole conference of the R&D Day will be available for consultation on the Genomic Vision’s website in a few days. ABOUT GENOMIC VISION GENOMIC VISION is a company specialized in the development of diagnostic solutions for the early detection of cancers and serious genetic diseases and tools for life sciences research. Through the DNA Molecular Combing, a strong proprietary technology allowing to identify genetic abnormalities, GENOMIC VISION stimulates the R&D productivity of the pharmaceutical companies, the leaders of the diagnostic industry and the research labs. The Company develops a robust portfolio of diagnostic tests (breast, ovarian and colorectal cancers, myopathies) and analysis tools (DNA replication, biomarkers discovery, gene editing quality control). Based near Paris, in Bagneux, the Company has approximately 60 employees. GENOMIC VISION is a public listed company listed in compartment C of Euronext’s regulated market in Paris (Euronext: GV - ISIN: FR0011799907). For further information, please visit www.genomicvision.com This press release contains implicitly or explicitly certain forward-looking statements concerning Genomic Vision and its business. Such forward-looking statements are based on assumptions that Genomic Vision considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the “Risk Factors” section in its Document de Reference filed with the French Autorité des Marchés Financiers (AMF) on March 28, 2017, under number R.17-009, available on the web site of Genomic Vision (www.genomicvision.com) and to the development of economic conditions, financial markets and the markets in which Genomic Vision operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Genomic Vision or not currently considered material by Genomic Vision. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Genomic Vision to be materially different from such forward-looking statements. This press release and the information contained herein do not constitute and should not be construed as an offer or an invitation to sell or subscribe, or the solicitation of any order or invitation to purchase or subscribe for Genomic Vision shares in any country. The distribution of this press release in certain countries may be a breach of applicable laws. The persons in possession of this press release must inquire about any local restrictions and comply with these restrictions.


News Article | July 13, 2017
Site: news.yahoo.com

After first lady Melania Trump touched down in Paris with her husband Thursday, she headed to a local children’s hospital to meet the patients. Read: What Does the Body Language of President Trump and Vladimir Putin Say About Their First Meeting? Melania visited Necker Hospital, which is the country’s largest and oldest pediatric infirmary. It was founded in 1778. The first lady, who speaks French, chatted with children in the Enfants Malades wing. During the visit, she also met and shook hands with doctors, nurses and other staff. She later shared an image of her meeting one of the children on Twitter. Thank U @hospital_necker for allowing me to visit your wonderful patients & inspirational staff. Continued prayers for good health for all. pic.twitter.com/3keri8EUC4 Necker Hospital has previous ties to America. The late street artist Keith Haring donated a large sculpture to the facility in 1987. Read: No Room at the Inn for President Trump as All Luxury Hotels in Hamburg Are Booked The Trumps are in France for a two-day visit to celebrate Bastille Day. On Thursday, while Melania spent time at the hospital, her husband partook in official duties for the French holiday. The first family was also in Europe last week for the G20 summit in Hamburg, Germany.


Diese Daten werden von Elisa Magrin, Ph.D., Necker Children’s Hospital, Assistance Publique-Hôpitaux de Paris, Paris, Frankreich, am Samstag, 24. Juni, in einer Postersession auf der Jahresversammlung der European Hematology Association (EHA) in Madrid, Spanien, präsentiert. Marina Cavazzana, M.D., Ph.D., Professor der Medizin an der Paris Descartes University und Research Director im Centre for Clinical Research in Biotherapy, Necker Hospital, und am Institute of Genetic Diseases, Imagine, Paris, Frankreich, ist leitende Studienärztin der HGB-205-Studie. „Mit der als Proof-of-Concept angelegten HGB-205-Studie sollte zunächst die Durchführbarkeit der LentiGlobin-Gentherapie bei Patienten mit TDT und schwerer SCD beurteilt werden. Die bisherigen Ergebnisse der Studie belegen potenziell dauerhafte Behandlungswirkungen von LentiGlobin bei stabiler HbAT87Q-Produktion über 3,5 Jahre der Nachuntersuchung und nachhaltigen klinischen Nutzen“, erklärte David Davidson, M.D., Chief Medical Officer bei bluebird bio. „Die beiden kürzlich behandelten Patienten mit SCD – beide mit erhöhter HbAT87Q-Produktion – verdeutlichen den potenziellen Nutzen einiger Protokollmodifikationen, die wir in unserer gesonderten HGB-206-Studie mit SCD vorgenommen haben. Wie bei Patient 1204, dem ersten in HGB-205 behandelten SCD-Patienten, erhielten diese beiden Patienten vor der Stammzellenentnahme eine striktere Busulfankonditionierung und regelmäßige Bluttransfusionen. Zur Bestimmung ihrer endgültigen HbAT87Q-Produktion und der klinischen Ergebnisse sind längere Nachuntersuchungen erforderlich, es ist jedoch ermutigend, dass die in vivo VCN beider Patienten eine frühe Stabilisierung auf höherem Niveau war als die der ursprünglichen Patientengruppe in der HGB-206-Studie. Bemerkenswert ist auch, dass diese Änderungen in unserer HGB-206-Studie durch Verbesserungen des Herstellungsverfahrens und den Einsatz von Plerixafor für die Mobilisierung von Stammzellen ergänzt wurden. Dies trug unserer Meinung nach zu einer weiteren Optimierung der Behandlungsergebnisse bei.“ Über bluebird bio, Inc. Mit seinen Lentivirus-basierten Gentherapien, Expertise im Bereich der T-Zellen-Immuntherapien sowie bei der Genomeditierung (“gene editing“) hat bluebird bio eine integrierte Produktplattform mit einer Vielzahl möglicher Anwendungen für schwerwiegenden Erbkrankheiten und Krebs geschaffen. Die klinischen Programme der Gentherapie von bluebird bio umfassen seinen Produktkandidaten Lenti-D™, derzeit Gegenstand einer Phase-2/3-Studie mit dem Namen Starbeam-Studie für die Behandlung von zerebraler Adrenoleukodystrophie, sowie seinen Produktkandidaten LentiGlobin™, derzeit Gegenstand von vier klinischen Studien zur Behandlung von transfusionsabhängiger β-Thalassemie und schwerer Sichelzellenanämie. Die Onkologie-Pipeline von bluebird bio baut auf der führenden Position des Unternehmens in der Übertragung von lentiviralen Genen und der T-Zellen-Technik auf, bei der der Schwerpunkt auf der Entwicklung neuartiger T-Zellen-basierter Therapien, einschließlich chimären Antigen-Rezeptor(CAR T)- und T-Zellen-Rezeptor(TCR)-Therapien, liegt. bb2121, das führende onkologische Programm von bluebird bio, ist ein Anti-BCMA-CAR T-Programm in Kooperation mit Celgene.. bb2121 wird zurzeit in einer Phase-1-Studie für die Behandlung des wiederkehrenden oder refraktären multiplem Myelom untersucht. bluebird bio verfügt zusätzlich über Forschungsprogramme zum Einsatz von MegaTAL/Homing-Endonuklease-Genbearbeitungstechnologien, die über das Potenzial verfügen, in der gesamten Produktpipeline des Unternehmens eingesetzt zu werden. Zukunftsbezogene Aussagen Diese Pressemitteilung enthält „zukunftsbezogene Aussagen“ im Sinne des Private Securities Litigation Reform Act von 1995, einschließlich Aussagen hinsichtlich der Forschungs-, Entwicklungs-, Herstellungspläne und der geplanten Beantragung einer behördlichen Zulassung für seinen Produktkandidaten LentiGlobin zur Behandlung von transfusionsabhängiger ß-Thalassämie und schwerer Sichelzellenanämie, sowie Aussagen darüber, ob Änderungen des Herstellungsverfahrens für LentiGlobin zu besseren Behandlungsergebnissen bei transfusionsabhängiger ß-Thalassämie und schwerer Sichelzellenanämie führen und die langfristige Wirksamkeit der Behandlung mit LentiGlobin erhöhen. Alle zukunftsbezogene Aussagen beruhen auf aktuellen Erwartungen des Managements im Hinblick auf zukünftige Ereignisse und unterliegen zahlreichen Risiken und Unwägbarkeiten, die dazu führen können, dass die zukünftigen Ergebnisse von den hier dargelegten oder in zukunftsbezogenen Aussagen implizierten Ergebnissen erheblich abweichen und diese nachteilig beeinflussen. Zu diesen Risiken und Ungewissheiten gehören unter anderen insbesondere die Gefahr, dass die vorläufigen positiven Wirksamkeits- und Sicherheitsergebnisse aus unseren vorherigen oder laufenden klinischen Studien mit LentiGlobin nicht fortgeschrieben oder in unseren laufenden, geplanten oder erweiterten klinischen Studien von LentiGlobin nicht wiederholt werden können, dass die von uns am Herstellungsverfahren von LentiGlobin oder am Protokoll der klinischen HGB-206-Studie vorgenommenen Änderungen nicht zu verbesserten Behandlungsergebnissen führen, dass die aktuellen oder geplanten klinischen Studien mit LentiGlobin nicht für behördliche Zulassungen oder Marktzulassungen in den USA und der EU ausreichen, die Gefahr einer verzögerten Aufnahme von Patienten in unsere klinischen Studien und die Gefahr, dass mindestens einer unserer Produktkandidaten nicht erfolgreich weiterentwickelt, zugelassen oder vermarktet wird. Zwecks Erörterung anderer Risiken und Unwägbarkeiten sowie anderer wichtiger Faktoren, die dazu führen können, dass unsere tatsächlichen Ergebnisse von den in den zukunftsbezogenen Aussagen dargelegten abweichen, lesen Sie den Abschnitt „Risk Faktors“ (Risikofaktoren) in unsere aktuellen Version des Formblatts 10-Q sowie unseren nachfolgenden Einreichungen bei der US-Börsenaufsichtsbehörde SEC (Securities and Exchange Commission). Alle Informationen in dieser Pressemitteilung gelten zum Zeitpunkt der Veröffentlichung, und bluebird bio übernimmt keine Verpflichtung die Informationen zu aktualisieren, sofern dies nicht gesetzlich vorgeschrieben ist.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, announced new data from the ongoing HGB-205 clinical study evaluating its LentiGlobin gene therapy product candidate in patients with transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD). These data will be presented by Elisa Magrin, Ph.D., Necker Children’s Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France in a poster session on Saturday, June 24 at the European Hematology Association (EHA) Annual Meeting in Madrid, Spain. Marina Cavazzana, M.D., Ph.D., Professor of Medicine at Paris Descartes University and Research Director at the Centre for Clinical Research in Biotherapy, Necker Hospital, and at the Institute of Genetic Diseases, Imagine, Paris, France, is the primary investigator of the HGB-205 study. “HGB-205 was designed as a proof-of-concept study to initially assess the feasibility of treatment with LentiGlobin gene therapy in patients with TDT and severe SCD. Results from this study to date demonstrate the potential for durable treatment effect of LentiGlobin, with stable HbAT87Q production through 3.5 years of follow-up and sustained clinical benefit,” said David Davidson, M.D., chief medical officer, bluebird bio. “The two most recently treated patients with SCD, both of whom show rising HbAT87Q production, illustrate the potential benefit of some of the protocol modifications that we have made in our separate HGB-206 study in SCD. As with Patient 1204, the first patient with SCD treated in HGB-205, these two patients received a more stringent busulfan conditioning regimen and regular blood transfusions prior to stem cell harvest. Longer follow-up will be required to determine their eventual HbAT87Q production and clinical outcome, but it is encouraging that their in vivo VCN shows evidence of early stabilization at a higher level compared to the initial cohort of patients in HGB-206. It is also important to note that in our HGB-206 study, these modifications are further supplemented with manufacturing process improvements and evaluation of plerixafor for stem cell mobilization, which we believe may further optimize patient outcomes.” “We are beginning to see evidence of the long-term durability of benefit from treatment with LentiGlobin, with some TDT patients even transitioning off of chelation therapy,” said Prof. Cavazzana. “It is exciting to see the outcome in the patient with TDT with the longest follow-up in HGB-205, who has gone from years of regular transfusions to 3.5 years without a single blood transfusion after a one-time treatment with LentiGlobin gene therapy.” Update on the First Patients with Severe Hemoglobinopathies Treated with LentiGlobin Gene Therapy (HGB-205) (Abstract P631) HGB-205 is an ongoing, open-label, single-center Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin drug product in the treatment of patients with TDT and severe SCD. Four patients with TDT and three patients with severe SCD have undergone infusion with LentiGlobin drug product in this study as of June 2, 2017. Webcast Information bluebird bio will host a live webcast at 8:00 a.m. ET on Friday, June 23, 2017. The live webcast can be accessed under "Calendar of Events" in the Investors and Media section of the company's website at www.bluebirdbio.com. Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States or (315) 625-3227 from outside the United States. Please refer to conference ID number 39917037. About TDT Transfusion-dependent β-thalassemia (TDT), also called β-thalassemia major or Cooley’s anemia, is an inherited blood disease that can be fatal within the first few years of life if not treated. Despite advances in the supportive conventional management of the disease, which consists of frequent and lifelong blood transfusions and iron chelation therapy, there is still a significant unmet medical need, including the risk for significant morbidity and early mortality. Currently, the only advanced treatment option for TDT is allogeneic hematopoietic stem cell transplant (HSCT). Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, graft vs. host disease and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT. About SCD Sickle cell disease (SCD) is an inherited disease caused by a mutation in the beta-globin gene that results in sickle-shaped red blood cells. Common complications include anemia, vaso-occlusive crisis, infections, stroke, overall poor quality of life and sometimes, early death. Where adequate medical care is available, common treatments for patients with SCD largely revolve around prevention of infection and management and prevention of acute sickling episodes. Chronic management may include hydroxyurea and, in certain cases, chronic transfusions. Given the limitations of these treatments, there is no effective long-term treatment. The only advanced treatment for SCD is allogeneic HSCT. Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, GvHD and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT. About the HGB-205 Study HGB-205 is an ongoing, open-label Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin drug product in the treatment of subjects with TDT and SCD. The study enrolled seven subjects who will be followed to evaluate safety and transfusion requirements post-transplant. Among patients with sickle cell disease only, efficacy will also be measured based on the number of vaso-occlusive crises or acute chest syndrome events. For more information on the HGB-205 study, please visit clinicaltrials.gov using identifier NCT02151526. About bluebird bio, Inc. With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin™ product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-thalassemia, and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bb2121 is currently being studied in a Phase 1 trial for the treatment of relapsed/refractory multiple myeloma. bluebird bio also has discovery research programs utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. Forward-Looking Statements This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s research, development, manufacturing and regulatory approval plans for its LentiGlobin product candidate to treat transfusion-dependent ß-thalassemia and severe sickle cell disease, including statements whether the manufacturing process changes for LentiGlobin will improve outcomes of patients with transfusion-dependent ß-thalassemia and severe sickle cell disease and the potential long-term durable treatment effect of LentiGlobin. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of LentiGlobin will not continue or be repeated in our ongoing, planned or expanded clinical trials of LentiGlobin, the risks that the changes we have made in the LentiGlobin manufacturing process or the HGB-206 clinical trial protocol will not result in improved patient outcomes, risks that the current or planned clinical trials of LentiGlobin will be insufficient to support regulatory submissions or marketing approval in the US and EU, the risk of a delay in the enrollment of patients in our clinical studies, and the risk that any one or more of our product candidates will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


Lapillonne A.,University of Paris Descartes | Lapillonne A.,Baylor College of Medicine | Lapillonne A.,Necker Hospital | Griffin I.J.,University of California at Davis
Journal of Pediatrics | Year: 2013

Preterm birth continues to contribute disproportionately to neonatal morbidity and subsequent physical and neurodevelopmental disabilities. Epidemiologic studies have described additional long-term health consequences of preterm birth such as an increased risk of hypertension and insulin resistance in adult life. It is not known whether the influence of infant and childhood growth rates and early nutrition on long-term outcomes is the same or different among preterm infants and neonates with intrauterine growth restriction. Our goal is to review the effects of fetal growth, postnatal growth, and early nutrition on long-term cardiovascular and metabolic outcomes in preterm infants. Present evidence suggests that even brief periods of relative undernutrition during a sensitive period of development have significant adverse effects on later development. Our review suggests that growth between birth and expected term and 12-18 months post-term has no significant effect on later blood pressure and metabolic syndrome, whereas reduced growth during hospitalization significantly impacts later neurodevelopment. In contrast, growth during late infancy and childhood appears to be a major determinant of later metabolic and cardiovascular well being, which suggests that nutritional interventions during this period are worthy of more study. Our review also highlights the paucity of well-designed, controlled studies in preterm infants of the effects of nutrition during hospitalization and after discharge on development, the risk of developing hypertension, or insulin resistance.


SAN DIEGO--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced the presentation of new data from the ongoing HGB-205 clinical study evaluating its LentiGlobin product candidate in patients with transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD) at the 58th American Society of Hematology Annual Meeting. The data from the HGB-205 study were highlighted today in a poster presentation by Marina Cavazzana, M.D., Ph.D., lead investigator of the HGB-205 study conducted in Necker Hospital, AP-HP and professor of hematology at Paris Descartes University, head of the department of Biotherapy Hospital, the clinical research center of Biotherapy at Necker Enfants Malades - Greater Paris University Hospitals, AP-HP and Inserm) and the Lymphohematopoiesis Laboratory, Institute of Genetic Diseases, Imagine, Paris, France. “We believe the enduring responses seen in this study - in the patients with TDT as well as the patient with SCD - demonstrate the continued promise of LentiGlobin gene therapy in both of these patient populations. We have seen nearly three years of transfusion independence in TDT in certain patients, providing important data on the long-term safety and durability of this therapy,” said David Davidson, M.D., chief medical officer, bluebird bio. “In addition, it is encouraging that the patient with SCD has remained free of acute SCD-related clinical events in the 21 months since treatment, when he previously required monthly blood transfusions to help control his SCD symptoms. This patient’s successful outcome not only offers hope for the potential of LentiGlobin to benefit other patients with SCD, but also provides important insights into this complex disease that we are applying to our ongoing HGB-206 study.” Abstract #2311: Update from the HGB-205 Phase 1/2 Clinical Study of LentiGlobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies HGB-205 is an ongoing, open-label, single-center Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin drug product in the treatment of patients with TDT and severe SCD. Four patients with TDT and one patient with severe SCD have undergone infusion with LentiGlobin drug product in this study as of September 9, 2016. The patients with TDT have between 11.6 and 33.5 months of follow-up, and the patient with SCD has 22.9 months of follow-up. “These data show a stable clinical and biological effect in patients with TDT or severe SCD who have received a one-time treatment with LentiGlobin,” said Professor Cavazzana. “We are now seeing the benefit of gene therapy with LentiGlobin beyond two years in TDT in certain patients, and clinical benefit continues to be realized in the patient with severe SCD after almost 24 months of follow-up. We are encouraged by these results and the potential benefit treatment with LentiGlobin can have on patients living with these debilitating diseases and without an HLA compatible sibling donor.” bluebird bio will host a live webcast at 8:30 p.m. PT (11:30 p.m. ET) on Monday, December 5, 2016. The live webcast can be accessed under "Calendar of Events" in the Investors and Media section of the company's website at www.bluebirdbio.com. With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin™ BB305 product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-thalassemia and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bb2121 is currently being studied in a Phase 1 trial for the treatment of relapsed/refractory multiple myeloma. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s research, development, manufacturing and regulatory approval plans for its LentiGlobin product candidate to treat transfusion-dependent ß-thalassemia and severe sickle cell disease. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks that the preliminary positive results from our prior and ongoing clinical trials of LentiGlobin, including HGB-205, will not continue or be repeated in our ongoing or planned clinical trials of LentiGlobin, the risks that the changes we have made in the LentiGlobin manufacturing process or the HGB-206 clinical trial protocol will not result in improved patient outcomes, risks that the current or planned clinical trials of LentiGlobin will be insufficient to support regulatory submissions or marketing approval in the US and EU, the risk of a delay in the enrollment of patients in our clinical studies, and the risk that any one or more of our product candidates will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law. About AP-HP: AP-HP - Greater Paris University hospitals - is a European world-renowned European university hospital. Its 39 hospitals treat 8 million people every year: in consultation, emergency, during scheduled or home hospitalizations. The AP-HP provides a public health service for everyone, 24 hours a day. This mission is a duty as well as a grzeat source of pride. The AP-HP is the leading employer un the Greater Paris area : 100 000 staff members – doctors, researchers, paramedical staff, administrative personnel and workers – work there. http://www.aphp.fr About the Imagine Institute: As the leading European center for research, care and teaching in genetic diseases, the Imagine Institute's primary aim is to understand and cure. The Institute's staff includes 850 of the best physicians, scientists and healthcare professionals housed in an innovative new building designed to realize synergies. This unprecedented continuum of expertise available in close proximity to patients allows Imagine to accelerate discoveries and their application at the bedside. www.institutimagine.org


CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, today announced the publication in the New England Journal of Medicine of a case study on Patient 1204, the first patient with severe sickle cell disease (SCD) to be treated with gene therapy. This patient, who was 13 years old at the time of treatment, was treated with LentiGlobin drug product in the HGB-205 clinical study conducted in Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. The data in the publication reflect 15 months of follow-up, and a brief summary of this patient’s outcomes with 21 months of follow-up was presented at the 58th American Society of Hematology Annual Meeting in December 2016. “We have managed this patient at Necker for more than 10 years, and standard treatments were not able to control his SCD symptoms. He had to receive blood transfusions every month to prevent severe pain crises,” said Professor Marina Cavazzana, M.D., Ph.D., principal investigator of this study and professor of hematology at Paris Descartes University, head of the department of Biotherapy Hospital, the clinical research center of Biotherapy at Necker Enfants Malades - Greater Paris University Hospital, AP-HP and INSERM, and of the Lymphohematopoiesis Laboratory, Imagine Institute of Genetic Diseases, Paris, France. “Since receiving the autologous stem cell transplant with LentiGlobin, he has been free from severe symptoms and has resumed normal activities, without the need for further transfusions.” “Since our initial publication of this therapeutic approach in mouse models in 2001, we are delighted to obtain such a clear proof-of-principle of its efficacy in a patient,” said Philippe Leboulch, M.D. Dr. Leboulch is professor of medicine at the University Paris-Sud and High Counselor and International Scientific Director at France’s CEA. He was a scientific founder of bluebird bio and serves as the co-chairman of its Scientific Advisory Board. Dr. Leboulch led the development of the anti-sickling T87Q globin vector used in LentiGlobin. “We are pleased to see this case study published in NEJM and shared with the broader research community. The successful outcome in Patient 1204 demonstrates the promise of treatment with LentiGlobin gene therapy in patients with severe SCD and serves as a guide for our efforts to optimize outcomes in future patients,” said David Davidson, M.D., chief medical officer, bluebird bio. “By analyzing this patient’s experience, we have identified key variables to optimize in our ongoing HGB-206 study of LentiGlobin gene therapy in severe SCD, and we are hopeful that these protocol changes will enable subsequent patients to achieve the transformative benefit seen in Patient 1204.” Clinical and Biological Outcomes for the First Patient with Sickle Cell Disease Treated with Gene Therapy Patient 1204, a male patient with βS/βS genotype, was enrolled in May 2014 at 13 years of age into the HGB-205 clinical study. The patient underwent a regular transfusion regimen for 4 years prior to this study. He had an average of 1.6 SCD-related events annually in the 9 years prior to initiating transfusions, and his complications from SCD included vaso-occlusive crises, acute-chest syndrome, bilateral hip osteonecrosis, and cerebral vasculopathy. The patient underwent two bone marrow harvests to collect hematopoietic stem cells (HSCs) for gene transfer and back-up (6.2×108 and 5.4×108 total nucleated cells/kg harvested). CD34+ cells were enriched from the harvested marrow and then transduced with LentiGlobin BB305 lentiviral vector. The vector copy numbers (VCN; vector copies per diploid genome) for the drug product lots manufactured were 1.0 and 1.2. The patient underwent myeloablation with intravenous busulfan (2.3 to 4.8 mg/kg per day for 4 days) with daily pharmacokinetic studies and dose adjustment. Total busulfan area under the curve (AUC) was 19,363 μmol*min. After a 2-day washout, Patient 1204 was infused with LentiGlobin drug product in October 2014 at a post-thaw total dose of 5.6×106 CD34+ cells/kg. RBC transfusions were to be continued after transplantation until a sufficient proportion of HbAT87Q (25-30% of total Hb) was detected. Neutrophil and platelet engraftment were achieved on Day +38 and Day +91 post-transplantation, respectively. HbAT87Q levels increased steadily and RBC transfusions were discontinued after the last transfusion on Day +88. HbAT87Q reached 5.5 g/dL (46% of total Hb) at Month 9 and continued to increase to 5.7 g/dL at Month 15 (48%), with a reciprocal decrease in HbS levels to 5.5 g/dL (46%) at Month 9, and 5.8 g/dL (49%) at Month 15. Total Hb levels have been stable between 10.6 and 12.0 g/dL since Month 6 post-transplant. HbF levels have remained below 1.0 g/dL. Adverse events (AEs) were consistent with busulfan conditioning, and no AEs related to LentiGlobin drug product have been observed to date. Over the 15 months since transplantation, no SCD-related clinical events or hospitalizations have occurred, contrasting favorably with the period before the patient began regular transfusions. All medications have been discontinued, including pain medication. The patient has resumed regular school attendance and reports full participation in normal physical activities. About SCD Sickle cell disease (SCD) is an inherited disease caused by a mutation in the β-globin gene that results in sickle-shaped red blood cells. The disease is characterized by anemia, vaso-occlusive crisis, infections, stroke, overall poor quality of life and, sometimes, early death. Where adequate medical care is available, common treatments for patients with SCD largely revolve around management and prevention of acute sickling episodes. Chronic management may include hydroxyurea and, in certain cases, chronic transfusions. Given the limitations of these treatments, there is no effective long-term treatment. The only advanced therapy for SCD is allogeneic hematopoietic stem cell transplantation (HSCT). Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, graft-versus-host disease, and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT. About bluebird bio, Inc. With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin™ BB305 product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-thalassemia and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR) and T cell receptor (TCR) therapies. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bb2121 is currently being studied in a Phase 1 trial for the treatment of relapsed/refractory multiple myeloma. bluebird bio also has discovery research programs utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. About AP-HP AP-HP - Greater Paris University hospitals - is a European world-renowned university hospital. Its 39 hospitals treat 8 million people every year: in consultation, emergency, during scheduled or home hospitalizations. The AP-HP provides a public health service for everyone, 24 hours a day. This mission is a duty as well as a great source of pride. The AP-HP is the leading employer in the Greater Paris area: 100,000 staff members – doctors, researchers, paramedical staff, administrative personnel and workers – work there. http://www.aphp.fr About the Imagine Institute As the leading European center for research, care and teaching in genetic diseases, the Imagine Institute's primary aim is to understand and cure. The Institute's staff includes 850 of the best physicians, scientists and healthcare professionals housed in an innovative new building designed to realize synergies. This unprecedented continuum of expertise available in close proximity to patients allows Imagine to accelerate discoveries and their application at the bedside. www.institutimagine.org Forward-Looking Statements This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s research and development plans for its LentiGlobin product candidate to treat severe sickle cell disease, including statements whether the manufacturing process changes for LentiGlobin will improve outcomes of patients with severe sickle cell disease and whether the planned changes to the HGB-206 clinical trial protocol will improve outcomes in patients with severe sickle cell disease. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of LentiGlobin will not continue or be repeated in our ongoing, planned or expanded clinical trials of LentiGlobin, the risks that the changes we have made in the LentiGlobin manufacturing process or the HGB-206 clinical trial protocol will not result in improved patient outcomes, risks that the current or planned clinical trials of LentiGlobin will be insufficient to support regulatory submissions or marketing approval in the US and EU, the risk of a delay in the enrollment of patients in our clinical studies, and the risk that any one or more of our product candidates will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent quarterly report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


Loupy A.,Necker Hospital | Hill G.S.,Georges Pompidou European Hospital | Jordan S.C.,Cedars Sinai Medical Center
Nature Reviews Nephrology | Year: 2012

Despite improvements in outcomes of renal transplantation, kidney allograft loss remains substantial, and is associated with increased morbidity, mortality and costs. Identifying the pathologic pathways responsible for allograft loss, and the attendant development of therapeutic interventions, will be one of the guiding future objectives of transplant medicine. One of the most important advances of the past decade has been the demonstration of the destructive power of anti-HLA alloantibodies and their association with antibody-mediated rejection (ABMR). Compelling evidence exists to show that donor-specific anti-HLA antibodies (DSAs) are largely responsible for the chronic deterioration of allografts, a condition previously attributed to calcineurin inhibitor toxicity and chronic allograft nephropathy. The emergence of sensitive techniques to detect DSAs, together with advances in the assessment of graft pathology, have expanded the spectrum of what constitutes ABMR. Today, subtler forms of rejection-such as indolent ABMR, C4d-negative ABMR, and transplant arteriopathy-are seen in which DSAs exert a marked pathological effect. In addition, arteriosclerosis, previously thought to be a bystander lesion related to the vicissitudes of aging, is accelerated in ABMR. Advances in our understanding of the pathological significance of DSAs and ABMR show their primacy in the mediation of chronic allograft destruction. Therapies aimed at B cells, plasma cells and antibodies will be important therapeutic options to improve the length and quality of kidney allograft survival. © 2012 Macmillan Publishers Limited. All rights reserved.

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