On February 16th, the Cambridge, MA-based company announced a temporary suspension of its Phase 1/2 (HGB-206) and Phase 3 (HGB-210) studies of LentiGlobin … Found inside – Page 339Ongoing preclinical and clinical trials target globin chain imbalance, ... in studies sponsored by Novartis, Apopharma, bluebird bio, Agios, and Terumo BCT. In other words, when all other treatments have failed. Thus far, they have only delivered data on seven CTX001 recipients in a phase 1/2 trial. The data from this clinical trial could provide proof-of-concept for MGTA-145, in combination with plerixafor, as the preferred mobilization regimen for patients with SCD. Found inside – Page 424... in three clinical trials sponsored by the biotechnology company bluebird bio (clinicalTrial.gov identifiers, NCT02151526, NCT01745120, and NCT02140554). On January 11, 2021, we announced our intent to separate our severe genetic disease and oncology businesses into differentiated and independent publicly traded companies. Talk with your doctor and family members or friends about deciding to join a study. In addition, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-307) for people who have participated in bluebird bio sponsored clinical studies of … Bluebird is now working with regulators to resume clinical trials in beta-thalassemia and sickle cell disease. Shares plunged 34% at the news.. HGB-206 is the company’s ongoing Phase I/II trial of LentiGlobin for SCD. FDA placed a clinical hold on two gene therapy clinical trials for sickle cell disease after two patients developed blood diseases. Brian Orelli, PhD (TMFBiologyFool) Sep 14, 2015 at 2:06AM Author Bio… Choosing to participate in a study is an important personal decision. The data from this clinical trial could provide proof-of-concept for MGTA-145, in combination with plerixafor, as the preferred mobilization regimen for patients with SCD. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. Clinical Trials. by Gareth Macdonald Thursday, February 18, 2021 6:38 am. Cancel Continue. In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, ... Found inside – Page 726... Party Clinical Trials.gov # X-adrenoleukodystrophy MND retroviral LTR U3, ALDP NM_000033.3 Aubourg P NCT01801709 Bluebird bio NCT01896102 Metachromatic ... bluebird bio appoints Ramy Ibrahim, M.D., Leader in Cancer Immunotherapy, to Board of Directors. Reprogrammed homing endonucleases and megaTALs are novel enzymes that provide a highly specific and efficient way to potentially treat a variety of diseases by … With things looking grim for the company, Bluebird Bio made a bold move by announcing they would split into 2 companies by the end of 2021. W e believe that the stock price of bluebird bio (NASDAQ:BLUE), a biotechnology company focused on … You’ll be responsible for oversight, planning, operational implementation, and execution of clinical laboratory functions to support clinical trials. Bluebird bio has suspended two clinical trials of its sickle cell disease gene therapy after participants developed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). This book exemplifies experience across the globe in banking of cord blood, mesenchymal, embryonic and induced pluripotent stem cells for clinical use from the United States, Canada, the European Union, Switzerland and Japan to Iran, India ... Found inside – Page 316One such treatment (using a lentiviral vector, Zynteglo; Bluebird Bio), ... with clinical trials in progress for treatment of Usher syndrome (hearing and ... Found inside – Page vPLANNING COMMITTEE ON SHARING CLINICAL TRIAL DATA: CHALLENGES AND A WAY ... National Multiple Sclerosis Society PATRICK CULLINAN, bluebird bio SONALI ... Found inside – Page 121These events prompted the development of more academic trials in more ... MA, the USA-based Bluebird Bio, which were found in the 90's (bluebird was created ... Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with SKYSONA … Found inside – Page 262... Zynteglo (ClinicalTrials, 2020) bluebird bio (Netherlands) B.V. Patients up to 12 years old with beta thalassemia who require regular blood transfusions ... This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD. Found inside – Page 163There's good reason to be optimistic about such clinical trials with ex vivo ... The biotechnology company Bluebird Bio has been developing a product that ... In Mercies in Disguise, acclaimed New York Times science reporter and bestselling author Gina Kolata tells the story of the Baxleys, an almost archetypal family in a small town in South Carolina. Pioneering biotech bluebird bio (NASDAQ: BLUE) has resumed clinical trials for its blood disorder gene therapies after getting clearance from the … This allows the company to restart the Phase I/II HGB-206 and Phase III HGB-210 trials of LentiGlobin for sickle cell disease (SCD) gene therapy (bb1111) in adult and paediatric SCD patients. Found inside... Alexander Astrakhan. bluebird bio, Seattle, WA. ... Positive results in recent clinical trials exploiting T cells engineered to express chimeric antigen ... Found inside – Page 154Data on gene therapy for thalassaemia were presented by Bluebird Bio, Inc., ... However, as yet there are no clinical trials addressing this issue. bluebird bio (NASDAQ:BLUE) has lost ~25.8% in premarket after the company announced the temporary suspension of two clinical trials for its gene therapy candidate, LentiGlobin, and … From our Cambridge, Mass., headquarters, we’re developing gene and cell therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Found inside – Page 241Product name Clinical trials ID Structure and mechanism of action Therapeutic ... Developer/ sponsor Bluebird Bio BioMarin Pharmaceutical PTC Therapeutics ... First results from the two studies, disclosed last November, were "taking the promise of CRISPR and turning that into a reality," said Samarth Kulkarni, CRISPR Therapeutics' CEO, in an interview. PSIOXUS PRE-CLINICAL DATA - On April 14, 2021, bluebird bio and PsiOxus Therapeutics presented preclinical data at the American Association for Cancer Research (AACR) Annual Meeting 2021. Typically, red blood cells are flexible and round allowing them to move easily through blood vessels. Found inside – Page 17It was sponsored by bluebird bio. Expected success awaits these clinical trials (Figure 2) [9]. 1.2.2 Gene editing A newer approach employs genome editing ... Found insideIt has completed clinical trials as a treatment for heart attack and ... adult stem cell lines. bluebird bio (NASDAQ: BLUE) is a Cambridge, Massachusetts, ... by Barry Teater, NCBiotech Writer — June 10, 2021. Found insideOne involved both serious clinical and regulatory violations. ... volunteered to participate in a clinical study being conducted by researchers at Genovo, ... Bluebird bio, a gene therapy company with manufacturing facilities in Durham, is resuming clinical trials of potential therapies for two inherited blood disorders after clearance from the U.S. Food and Drug Administration.. A participant's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy … Skysona's unclear potential. Pioneering biotech bluebird bio (NASDAQ:BLUE) has resumed clinical trials for its blood disorder gene therapies after getting clearance from the … Join bluebird bio's enthusiastic and collaborative Clinical Development Operations (CDO) team by contributing to the overall success of our novel gene therapy approach in beta-thalassemia. bluebird bio (NASDAQ:BLUE) stock has fallen on hard times due to seemingly never-ending issues with its gene therapy, LentiGlobin. bluebird bio (NASDAQ:BLUE) ... Then they also have more recent issue, they had a patient that had been in their clinical trial who developed cancer. Found inside – Page 45Concerning clinical trials with LV, three companies are worth mentioning. Bluebird Bio (formerly Genetix Pharmaceuticals) in the USA, recently completed 2 ... In immune-oncology, bluebird bio comprises bb2121 and bb21217; CAR T products both for multiple myeloma (MM). Process 2 has been successfully scaled up for clinical manufacturing, and all LentiGlobin clinical trials moving forward will use manufacturing Process 2, including the Phase 3 HGB-207 (Northstar-2) trial and the Phase 1 HGB-206 clinical trial. Bluebird Bio already had a multiple myeloma CAR T-cell therapy, Abecma, approved earlier this year, and now it has not one, but two gene therapy approvals for rare genetic diseases in the EU. The FDA has lifted clinical holds on studies evaluating Bluebird Bio’s LentiGlobin gene therapy for sickle cell disease (SCD) as well as trials of a treatment for a blood disorder that uses the same viral vector. Found insideThis book will be of interest to stakeholders across the spectrum of research-from funders, to researchers, to journals, to physicians, and ultimately, to patients. Bluebird bio is a pioneer that develops gene and cell therapies for severe genetic diseases and cancer. Found inside – Page 5Translational Pathways to Clinical Adoption Hematti Peiman, Sowmya Viswanathan ... Bluebird Bio, and Kite Pharma ($USD 5.6 B and $USD 2.5 B market caps ... bluebird bio is conducting a long-term safety and efficacy follow-up study, LTF-303 for people who have participated in bluebird bio-sponsored clinical studies of ZYNTEGLO. bluebird bio (NASDAQ:BLUE) stock has fallen on hard times due to seemingly never-ending issues with its gene therapy, LentiGlobin. The company is now working closely with study investigators and clinical trial sites to … The New York Times and Boston Herald recently released featured articles that tell the personal stories of patients enrolled in a clinical trial conducted by bluebird bio. Dive Brief: Executives at Bluebird bio on Thursday gave investors listening to a conference hosted by bank SVB Leerink an update on their investigation into two recent cancer cases in a study of the company's gene therapy for sickle cell disease. Clinical Trials Our Products Patients & Advocacy Patient Advocacy ... By providing your information, you agree to allow bluebird bio to collect the name and email address provided and to be contacted by bluebird bio and its partners using this information. bluebird bio. Gene therapy developer bluebird bio suspended clinical trials using its LentiGlobin product for sickle cell disease due to reported cases of hematologic malignancies among patients who … Bluebird bio announced that it has placed its Phase I/II and Phase III trials of LentiGlobin gene therapy for sickle cell disease (SCD) on temporary suspension. This book is a completely revised new edition of the definitive reference on disorders of hemoglobin. The company, uniQure, planned to … The company, uniQure, planned to … The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as … bluebird bio presented preclinical data on the TCR candidate targeting the tumor antigen MAGE-A4, which is expressed by a variety of solid tumor types.. Roivant/ Cytovant RESEARCH TRIANGLE PARK – Bluebird bio, a gene therapy company with manufacturing facilities in Durham, is … Bluebird bio on Wednesday said its experimental gene therapy for sickle cell disease is "very unlikely" related to a recently diagnosed case of leukemia in the one of the company's clinical trials, citing findings from an investigation into the genetic make-up of the study volunteer's tumor. Immunotherapy is a form of cancer therapy that harnesses the body's immune system to destroy cancer cells. What's Next For bluebird bio After The U.S. FDA Lifts Hold On Clinical Trials. Bluebird Bio has temporarily suspended two clinical trials of its betibeglogene autotemcel (LentiGlobin) gene therapy to treat sickle cell disease (SCD) after reports of cancer diagnoses in two patients who participated in the trial and received the investigational treatment. Anne-Virginie Eggimann, M.Sc. The FDA has lifted clinical holds on trials for bluebird bio's (NASDAQ:BLUE) LentiGlobin, a gene therapy for sickle cell disease and Zynteglo for β-Thalassemia. Bluebird Bio said it has temporarily and voluntarily suspended two clinical trials assessing its LentiGlobin gene therapy for sickle cell disease (SCD; bb1111) following reports that two participants in the earlier-phase study developed blood cancers—one of acute myeloid leukemia (AML), the other of myelodysplastic syndrome (MDS). Found inside – Page 43Clinical trials conducted at 3 different centers have reported exciting results to date. Bluebird Bio (Cambridge, MA) is treating patients with SCD on an ... FDA lifts clinical hold on bluebird bio’s LentiGlobin studies Company previously put trials on hold after suspected unexpected serious adverse reactions The US Food and Drug Administration (FDA) has lifted the clinical holds on numerous studies evaluating its gene therapy LentiGlobin for sickle cell disease (SCD) and beta-thalassemia. bluebird bio accepts no responsibility for the content of linked sites. And could its lead in sickle cell disease clinical trials translate into massive gains for investors? So with the gene therapy headed for likely regulatory approval, bluebird bio has turned some of its resources to developing a price for the therapy. Bluebird Bio has paused two clinical trials of LentiGlobin, its gene therapy candidate for sickle cell disease (SCD), after learning that a patient who received treatment more than five years ago now has developed acute myeloid leukemia (AML), a type of blood cancer.. Bluebird suspends studies of sickle cell gene therapy following cancer diagnoses in two more treated patients. Found inside – Page 225A phase I dose-escalation study using CAR T-cells targeting CD30 included ... Bluebird Bio's bb2121 cell therapy product (4-1BB costimulatory domain) ... The cause is a Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML). For more information, follow the links below to … Found inside – Page 54... DCVax® 2001: Manufacturing and clinical trials partnership whereby trials ... Penn Bellicum Pharmaceuticals Bluebirdbio and Celgene LUMC 2015: Research ... And could its lead in sickle cell disease clinical trials translate into massive gains for investors? Found inside – Page 14Several stem cell trials are, however, under way for a number of rare diseases ... The biotechnology company Bluebird Bio has clinical stage assets based on ... Links to sites outside of this website are provided as a resource to the viewer. The data from this clinical trial could provide proof-of-concept for MGTA-145, in combination with plerixafor, as the preferred mobilization regimen for patients with SCD. The suspected serious adverse reaction resulted in the FDA instructing the company to halt its trial in late February. In this book, Hank Greely, a leading authority on law and genetics, tells the fascinating story of this human experiment and its consequences. Found inside – Page 254Novartis, Juno, Kite and Bluebird Bio were among the tech companies seeking to develop the therapies ... Since then, Juno has discontinued clinical trials. About bluebird bio… bluebird bio has eight programs in clinical trials, with most of them being in phase two or phase three. Found inside – Page 155... biotech company Bluebird Bio was running a successful clinical trial for the ALD ... More companies were sniffing around clinical trials in so - called ... Found inside – Page 395Table 25.4 Current gene therapy trials for SCD in the USA Clinical trial # Phase LV/nuclease Site/sponsor NCT02140554 1/2 BB305 LV (HGB206) BlueBird bio ... The FDA lifted a clinical hold it placed on bluebird bio’s sickle cell gene therapy in February, making way for the company to get a phase 1/2 trial and phase 3 … News broke today that Bluebird Bio had to halt its sickle cell clinical trial as 2 participants got blood cancers. We will retain focus on severe genetic disease (SGD) and will launch our oncology business (“Oncology Newco”) as a new entity, with the completed transition expected by the end of 2021. Found inside – Page 95ACT's product is currently in US clinical trials for Stargardt's macular ... is the use of a lentiviral-based integration by BluebirdBio (Cambridge, MA, ... Bluebird suspends studies of sickle cell gene therapy following cancer diagnoses in two more treated patients. The book's richly experienced authors detail methods utilizing a wide variety of the latest analytical techniques, including X-ray crystallography, high performance liquid chromatography, electrophoresis, and nuclear magnetic resonance. Found insideThis book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). i. The results showed synergistic activity between PsiOxus’ T-SIGn vector and bluebird bio’s CAR-T therapy in primary and metastatic solid tumors. The U.S. Food and Drug Administration (FDA) has pushed bluebird bio’s voluntary pause to a regulator-demanded clinical hold for all LentiGlobin gene therapy trials, following last week’s announcement that two enrolled sickle cell disease (SCD) patients developed acute myeloid leukemia (AML) and myelodysplastic syndrome, respectively. For general information, Learn About Clinical Studies. Found inside – Page 563... 3 Biosciences $31M 06/04/13 EBR Bluebird Bio $116M 06/18/13 NASDAQ Capricor ... the 55 organizations profiled on TRAIN have supported a clinical trial. bluebird bio (NASDAQ:BLUE) ... Then they also have more recent issue, they had a patient that had been in their clinical trial who developed cancer. In addition, bluebird is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for transfusion-dependent β-thalassemia (TDT) and SCD. Found insideNonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products. CAMBRIDGE, Mass.--(BUSINESS WIRE)-- bluebird bio (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin for sickle cell disease (SCD) gene therapy (bb1111) for adult and pediatric patients with SCD, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) … The purpose of this manual is to provide an educational genetics resource for individuals, families, and health professionals in the New York - Mid-Atlantic region and increase awareness of specialty care in genetics. In May 2019, bluebird bio announced that it intends to start clinical development in 2020 of the first therapeutic TCR candidate arising from the collaboration. The bb2121 is in four clinical trials for: first, second, third and fourth line therapy for MM. Thus far, they have only delivered data on seven CTX001 recipients in a phase 1/2 trial. bluebird bio: ClinicalTrials.gov Identifier: NCT01745120 Other Study ID Numbers: HGB-204 : First Posted: December 7, 2012 Key Record Dates: Results First Posted: May 8, 2019: Last Update Posted: May 8, 2019 Last Verified: April 2019 Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with SKYSONA … Study Description. Bluebird Bio Finds Lentiviral Vector “Unlikely” Cause of Clinical Trial’s Adverse Events. Found inside – Page 124... than 200 CAR-T cell clinical trials have been initiated. Multiple programs are showing promising clinical results. For example, the Bluebird Bio/Celgene ... bluebird bio is conducting clinical studies of investigational gene therapies in four diseases: cerebral adrenoleukodystrophy, relapsed/refractory multiple myeloma, sickle cell disease, and transfusion-dependent β-thalassemia. Bluebird Bio is set to resume trials of LentiGlobin, its investigational gene therapy for sickle cell disease, after the U.S. Food and Drug Administration (FDA) lifted its clinical hold. The LentiGlobin gene therapy alone can garner … Bluebird bio said its investigations have shown that it’s “very unlikely” that LentiGlobin, the company’s investigational gene therapy for sickle cell disease, caused the case of acute myeloid leukemia (AML) reported recently during LentiGlobin’s phase 1/2 trial. -- (BUSINESS WIRE)--Dec. 1, 2018-- bluebird bio, Inc. (Nasdaq: BLUE) announced new long-term data from the completed Phase 1/2 Northstar (HGB-204) study of investigational LentiGlobin ™ gene therapy in patients with transfusion-dependent β-thalassemia (TDT) and from the ongoing Phase 1/2 HGB-206 study of LentiGlobin in patients with sickle cell disease (SCD) today at the 60 th Annual … After about four months, the U.S. Food and Drug Administration has given the green light for Cambridge-based bluebird bio to move forward with two clinical trials… Bluebird Bio’s suspension of trials of its sickle cell gene therapy LentiGlobin has put the vector used to make it in the spotlight. The FDA lifted a clinical hold it placed on bluebird bio’s sickle cell gene therapy in February, making way for the company to get a phase 1/2 trial and phase 3 … Found inside – Page 649Several pilot and phase I, II, or III clinical trials are currently in progress ... Pharmaceuticals (Mountain View, CA) and Bluebird Bio (Cambridge, MA). The current data came from a clinical trial for fourth line MM treatment. CAMBRIDGE, Mass. The European Medicine Agency’s human medicines committee, the CHMP, has delivered a positive opinion on Bristol Myers Squibb Company/bluebird bio’s CAR T-cell therapy, Abecma (idecabtagene vicleucel), the first cell-based gene therapy for patients with multiple myeloma.. At its latest monthly meeting on 21-24 June, the CHMP recommended Abecma for conditional EU marketing … About bluebird bio, Inc. bluebird bio is pioneering gene therapy with purpose. Bluebird Bio already had a multiple myeloma CAR T-cell therapy, Abecma, approved earlier this year, and now it has not one, but two gene therapy approvals for rare genetic diseases in the EU. Bluebird Bio saw its share price plunge by more than one-third after temporarily and voluntarily suspending two clinical trials assessing its LentiGlobin gene therapy for … The trial uses gene therapy in combination with hematopoietic (blood) stem cells (HSCs) to give rise to normal red blood cells in SCD patients. We are focused on homing endonuclease and megaTAL gene editing technologies in a variety of potential applications and disease areas. Found inside – Page 168A US biotech company, Bluebird Bio, was given conditional EU authorisation for their gene therapy, ... Clinical trials are ongoing, but certainly promising. Bluebird Bio investigating potential vector role in AML and MDS cases. Things went from bad to catastrophic, in 2021, when the company announced it was temporarily suspending two of its clinical trials for LentiGlobin, due to concerns the treatment caused cancer. bluebird’s expert gene editing team is expanding our discovery research efforts in this emerging field. Found inside – Page 189Bluebird Bio Inc. was awarded $9.3 million to fund a Phase I/II study of ... positive results in early clinical trials, and the recent approval in Canada of ... Found inside – Page 333... with severe inherited globin disorders (NCT01639690 at clinicaltrials.gov). ... Blue Bird Bio (BBB; Cambridge, MA) subsequently bought the b87 vector, ... This edition includes new chapters such as signaling and antigen presentation by B-lymphocytes, molecular oncogenesis and more! Last month, Bluebird Bio halted their Phase 1/2 and Phase 3 studies of LentiGlobin gene therapy for sickle cell disease after a patient was diagnosed with acute myeloid leukemia (AML). The bluebird bio news comes on the heels of a December 2020 report that a patient in a gene therapy trial for hemophilia had developed a liver tumor. Found inside[14] Genethon is conducting four Phase I/II clinical trials as a sponsor or partner and is leading a dozen projects at various stages of preclinical ... Found inside – Page 470Table 1 Current human clinical trials for sickle cell gene therapy Identifier/Study ... Bluebird Bio (France) NCT02140554/HGB-206 Bluebird Bio (Multicenter, ... This trial will assess the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of cerebral adrenoleukodystrophy (CALD). 3 Clinical Trials to Watch As Bluebird Bio Rounds Out 2015 Circle Dec. 5-8 on your calendar. In a press release, the firm gave the details on the severe adverse events, termed “Suspected Unexpected Serious Adverse Reactions.” The company suspended its Phase 1/2 and Phase 3 trials of LentiGlobin gene therapy for sickle cell disease. Found inside – Page 462HSV vectors have certainly promising perspectives in clinic trials but detailed ... A clinical trial sponsored by Bluebird Bio used LentiGlobin BB305, ... Skysona's unclear potential. Found inside – Page 205Strategy for a multicenter phase I clinical trial to evaluate globin gene ... Bluebird bio I. Bluebird bio reports rapid transfusion independence in ... joined bluebird bio, Inc. to lead the Regulatory Science function in 2011. In both trials, LentiGlobin allowed patients to stop chronic blood transfusions. bluebird bio (NASDAQ: BLUE) stock has fallen on hard times due to seemingly never-ending issues with its gene therapy, LentiGlobin. Bluebird bio is on track to file gene therapy Skysona for cerebral adrenoleukodystrophy with the U.S. FDA mid-2021, while U.K. regulators are already reviewing its application. A gene therapy developed by Bluebird Bio has shown similar potential. During the … On Feb. 16, 2021, bluebird bio, Inc. suspended its clinical trial exploring the curative potential of genetic therapy for sickle cell disease using its lentiviral vector (gene therapy delivery system), because two research participants in this trial developed myeloid neoplasms following gene … Found inside – Page 5050 NOVEL CLINICAL TRIAL DESIGNS FOR GENE-BASED THERAPIES can have many causes having ... Work done with bluebird bio (a biotechnology company based in ... The US Food and Drug Administration (FDA) has lifted the clinical hold placed on bluebird bio’s gene therapy trials in February this year. Found inside – Page iThis book provides a comprehensive and up-to-date review of all aspects of childhood Acute Lymphoblastic Leukemia, from basic biology to supportive care. The bluebird bio news comes on the heels of a December 2020 report that a patient in a gene therapy trial for hemophilia had developed a liver tumor. 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