Zafgen(ZFGN)$ sage/zfgn/agio/blue都是三石风投孵化的公司，个个表现都很好，。三石一共投资了30多个生物技术公司，有一些还没有上市就给大公司收购了。看来要好好重视三石投资准备上市的公司。
Manufacturing Operations, Lentiviral Vector Sr. Scientist
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2015年4月24日 - At the heart of bluebird bio's product creation efforts is its broadly applicable gene therapy platform for the development of new treatments for ...
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2015年4月24日 - At the heart of bluebird bio's product creation efforts is its broadly applicable gene therapy platform for the development of new treatments for ...常見基因治療之病毒載體(Viral Vector)-下
病毒載體需要病毒表面的蛋白質與宿主細胞表面的蛋白質有良好的反應決定是否能順利進入宿主細胞，於是許多載體被研發出具有內生性外套膜蛋白（endogenous viral envelope proteins）來取代其他病毒的外套膜蛋白（envelope proteins）或融合蛋白（chimeric proteins）。這種病毒蛋白與宿主蛋白作用後結合形成嵌合體(Chimera virus）。而這種有外套膜蛋白被取代之病毒稱之為假性病毒（pseudotyped virus）。
例如最常使用在基因治療之反轉錄病毒是慢病毒猴免疫缺陷病毒（Lentivirus Simian immunodeficiency viruses，SIV）上所塗層的外套膜蛋白-- G 蛋白（G proteins）--來自口腔泡疹病毒（Vesicular stomatitis virus，VSV）。這種病毒又稱為口腔泡疹G假性慢病毒 （VSV G-pseudotyped lentivirus），可普遍感染許多宿主細胞。
5.慢病毒載體（Lentiviral vector）：利用反轉錄病毒科慢病毒屬作為載體，此種載體可由宿主細胞直接感染周圍細胞，即利用包裝細胞（packaging cell）內的慢病毒直接感染周圍的靶細胞（target cell），不必經由形成病毒顆粒。
參考文獻：1.科學人。2008。基因治療如何發揮作用？科學人。 79期9號。
2.胡育誠。2003。基因治療的過去與展望。科學發展。372期。
3.DNA疫苗 http://www.biochemlab.cn/jiaoxue/terms/20798.html
4.DNA疫苗及其特點 http://www.biochemlab.cn/jiaoxue/lilun/20800.html
5.Gene therapy using an adenovirus vector http://ghr.nlm.nih.gov/handbook/illustrations/therapyvector
6.基因治療的展望 http://www.cbt.ntu.edu.tw/General/BioMed/Biomed3/Biomed3-4.htm
Founded in 1992, the company was formerly known as Genetix Pharmaceuticals, Inc. The company's name was changed to bluebird bio, Inc. in September 2010. In addition to its headquarters in Cambridge, bluebird bio has operations in San Francisco, California, and Paris, France.
Pipeline
bluebird bio's pipeline includes:- Lenti-D to treat patients with childhood cerebral adrenoleukodystrophy (CCALD), a hereditary neurological disorder, as well as for the treatment of adult cerebral adrenoleukodystrophy (ALD);
- LentiGlobin vector for the treatment of ß-thalassemia, a hereditary blood disorder; and sickle cell diseases (SCD); and
- A preclinical oncology program in the chimeric antigen receptor T cells (CAR T) field under a collaboration with Celgene Corporation (NASDAQ:CELG).
Gene Therapy Platform and Process
Gene therapy is cutting edge science that strives to introduce a functional copy of a defective gene into a patient's cells through the gene transfer process. In this process, a functional gene is delivered and incorporated into a patient's cells through a delivery system called a vector. These vectors are usually based on viruses that have been altered to utilize the virus's natural ability to introduce genes into cells, but are modified to be non-replicating by deleting the part of the viral genome responsible for replication and disease.bluebird bio's gene therapy platform is based on viral vectors that utilize a modified, non-replicating version of the human immunodeficiency virus type 1 (HIV-1), that has been stripped of all of the components necessary for it to mutate and infect additional cells. Since HIV-1 is part of the lentivirus family of viruses, the company refers to its vectors as lentiviral vectors. bluebird bio's lentiviral vectors are used to introduce a functional copy of a gene to a patient's hematopoietic stem cells (HSCs). HSCs are capable of differentiating into many cell types. Since HSCs are dividing cells, bluebird bio scientists have found that this approach allows for sustained expression of the modified gene because it takes advantage of the lifetime replication of gene-modified HSCs.
bluebird bio has also developed a proprietary cell-based vector manufacturing process. The company believes its innovations in viral vector design and manufacturing processes are important steps towards advancing the field of gene therapy to a commercial scale.
Although its initial focus is in childhood cerebral adrenoleukodystrophy (CCALD), ß-thalassemia and sickle cell disease (SCD), bluebird bio believes its gene therapy platform has broad therapeutic potential for many indications.
For beta-thalassemia, SCD, and adrenoleukodystrophy, a functional copy of the malfunctioning gene is inserted into a patient's HSCs, with the goal of genetically modifying a patient's cells to correct or address the genetic basis underlying the disease.
In oncology, the gene therapy process targets T cells. In these cases, genetic sequences are inserted into a patient's own T cells, which are intended to program the T cells to recognize and attack cancer cells. T cells, also known as lymphocytes, are a type of white blood cell that help the body fight diseases.
In October 2003, Shenzhen SiBiono GeneTech made history by becoming the first company approved to market a gene therapy. China's State Food and Drug Administration approved Genedicine for treatment of head and neck squamous cell carcinoma. but the company believes that continued clinical trials will prove Genedicine to be a safe and effective treatment for other types of cancer.
On November 2, 2012, the European Commission approved Uniqure's Glybera (alipogene tiparvovec), for the treatment of lipoprotein lipase deficiency. Glybera is the first gene therapy approved in the Western world. The treatment costs approximately $1.6 million per patient. Uniqure, a small Dutch company, has a product pipeline with several gene therapies for hemophilia B, acute intermittent porphyria, Parkinson's disease and Sanfilippo B. The Amsterdam, Netherlands-based company plans to meet with the FDA with the aim of a U.S. launch in 2014.
According to the industry intelligence advisor, RNCOS, the global gene therapy market is estimated to have reached approximately $485 million in 2011, and is forecast to grow significantly with the introduction of new products in developed countries. The research firm predicts the gene therapy market reach the mark of $650 million by the end of 2015.
RNCOS noted that adenovirus vectors hold a leading position in the ongoing gene therapy clinical studies. Its usage rate is around 24% in the gene therapy treatment trials being pursued by industry participants.
Companies, such as Advantagene, Applied Genetic Technologies, Ceregene, Sanofi/Genzyme (NYSE:SNY), GenVec (NASDAQ:GNVC), are conducting trials for gene therapy-based products using adenovirus vectors.
Other companies developing gene therapies include AnGes MG Inc., ANI Pharmaceuticals, Inc./BioSante Pharmaceuticals (NASDAQ:ANIP), GlaxoSmithKline (NYSE:GSK), Oxford BioMedica (OTC:OXBDF), Transgene, Urigen Pharmaceuticals Inc. (OTC:URGP), and Vical, Inc. (NASDAQ:VICL).
Big pharma has renewed its interest in gene therapy.
In October 2010, GlaxoSmithKline (Glaxo), Fondazione Telethon and Fondazione San Raffaele announced a new strategic alliance to research and develop novel treatments to address rare genetic disorders, using gene therapy carried out on stem cells taken from the patient's bone marrow (ex vivo). The alliance capitalizes on research performed at the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), a joint venture between Fondazione Telethon and Fondazione San Raffaele established in 1995.
Under the terms of the agreement, Glaxo gained an exclusive license to develop and commercialize an investigational gene therapy, for ADA Severe Combined Immune Deficiency (ADA-SCID), a rare and life-threatening immune deficiency, which affects approximately 350 children worldwide. Phase 1/Phase 2 studies have demonstrated the potential of this treatment option to restore long-term immune function and protect against severe infections in children with ADA deficiency.
Glaxo is co-developing with Fondazione Telethon and Fondazione San Raffaele six other applications of ex vivo stem cell therapy using a new gene transfer technology developed by HSR-TIGET scientists with the potential to treat a range of rare disorders, including metachromatic leukodystrophy and Wiskott-Aldrich Syndrome, beta-thalassemia, mucopolysaccharoidosis type I, globoid leukodystrophy, and chronic granulomatous disorder. .
Fondazione Telethon received an upfront 10 million euro payment from Glaxo and is eligible to receive further payments upon successful completion of a number of predetermined development milestones.
In September 2011, Merck & Co (NYSE:MRK) announced that it issued an exclusive license to FKD Therapies Oy, a recently formed company in Finland specializing in advanced gene based medicines to develop and commercialize its gene therapy portfolio.
On August 26, 2013, Sangamo BioSciences, Inc. (NASDAQ:SGMO) announced that it signed a definitive agreement to acquire Ceregene, Inc., a privately held biotechnology company focused on developing adeno-associated virus gene therapies.
On May 1, 2013, Oxford BioMedica, a British company, announced that it agreed to produce clinical trial material using its LentiVector gene delivery technology for Novartis's (NYSE:NVS) use as part of its CTL019 leukemia program, a collaboration with scientists at the University of Pennsylvania.
Through this production agreement, Oxford BioMedica will be responsible for manufacturing a lentiviral vector-encoding CTL019 technology, which Novartis et al. will use to transduce patients' T-cells ex vivo before they are reinfused. In its announcement, Oxford BioMedica noted that CTL019 targets the protein CD19, which is associated with a number of B-cell malignancies, including chronic lymphocytic leukemia, B-cell acute lymphocytic leukemia, and diffuse large B-cell lymphoma.
Under the terms of the agreement, Oxford BioMedica stands to make $3.9 million to $6.2 million during the first year of collaboration.
The US National Institutes of Health (NIH) clinicaltrials.gov website lists over 3,000 gene therapy trials, of which 1,300 are listed as recruiting. The vast majority of these are being run by academic investigators.
The excitement surrounding gene therapy was tempered on August 12, 2013 when shares of Vical plunged after the company announced that it was terminating its Allovectin (velimogene aliplasmid) program and focusing its resources on its infectious disease vaccine program. In a Phase 3 trial of patients with metastatic melanoma, Allovectin failed to demonstrate a statistically significant improvement versus first-line chemotherapy for either the primary endpoint of objective response rate at 24 weeks or more after randomization or the secondary endpoint of overall survival.
Lenti-D
bluebird's most advanced product candidate is Lenti-D, which the company is developing initially to treat patients with childhood cerebral adrenoleukodystrophy (CCALD).CCALD is a rare, hereditary neurological disorder affecting young boys that is often fatal. CCALD is caused by mutations in the ABCD1 gene. bluebird bio scientists believe the disease can be treated with the ex vivo insertion of a functional copy of the ABCD1 gene into a patient's own HSCs to correct the aberrant expression of ALDP in patients with CCALD. HSCs derived from the patient's own body are called autologous HSCs. bluebird bio has named the autologous HSCs that have been modified to carry the functional copy of the ABCD1 gene as the final Lenti-D drug candidate.
bluebird bio performed a non-interventional retrospective data collection study. Comprised of 136 CCALD patients, the study assessed the course of the disease in patients who were left untreated and patients who received allogeneic HSCT. Researchers conducted a non-interventional retrospective data collection study that examined the historical clinical records from patients in the study to assess the typical course of the condition and the efficacy and safety of treatment options. The company believe the results of this study supported its approach of using autologous, gene-modified HSCs to treat CCALD, especially in light of several significant safety concerns commonly associated with the current standard of care, allogeneic HSCTs. Allogeneic HSCTs are the HSCTs from donors other than the patient.
The company believes that the results from a Phase 1/2 study in four patients with CCALD conducted by the company's scientific collaborators in France with an earlier generation lentiviral vector were useful in the design of its own trials to evaluate the efficacy and safety of Lenti-D.
Early clinical proof-of-concept results for two patients in a French study sponsored by the Institut National de la Santé et de la Recherche Médicale (National Institute of Health and Medical Research) (INSERM) produced promising results that were published in the November 2009 issue of Science.
In this pilot study, blood stem cells were removed from patients and genetically modified in the laboratory using a lentiviral vector to introduce a working copy of the ALD gene into the cells. The patients' modified cells were then infused back into the patients after they received a treatment to allow engraftment of the corrected cells. Initial results from this study demonstrated disease stabilization and no further disease progression in the two patients monitored for two years.
On May 20, 2010, Nathalie Cartier-Lacave, MD, study investigator and a director of research at the National Institute of Health and Medical Research at INSERM, presented additional data from the pilot study. Based on the continued follow-up of the first two patients as well as 20-month data on a third patient, the new data found that at three years, the two patients' functional ALD proteins remained stably expressed at 10% to 15% of circulating monocytes. Both continued to show neurological stabilization with no adverse effects noted. In the third patient, functional ALD protein remained stably expressed at 20 months. The new findings also demonstrated that the third patient's cognitive functioning was within normal range at 16 months. Although these results appear to be extremely promising, the researchers warned that the current data was insufficient to determine that the disease was stabilized. The researchers reported that the safety and therapeutic benefits of this procedure have been maintained for more than five years, and two additional patients have subsequently been treated.
Based on these promising early clinical proof of concept results, bluebird bio plans to initiate a larger Phase 2/3 clinical trial in CCALD in the United States and Europe in 2013 to evaluate Lenti-D's safety and efficacy in subjects with CCALD. The estimated completion date of the study (final data collection date for primary outcome measure) is August 2018.
In this clinical trial, up to 15 patients will be enrolled in order to obtain at least 12 subjects who will be followed over a 24-month period to assess the onset of major functional disabilities (MFDs), and other key assessments of disease progression. bluebird bio expects to initiate this study in the United States in late 2013. If successful, the company believes the results of this study could support submission of a Biologics License Application (BLA) and a Marketing Authorization Application (MAA) filing for Lenti-D.
On June 19, 2012, bluebird announced that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) granted an orphan drug designation to its investigational gene therapy product for the treatment of ALD.
"Receiving orphan drug designation is a positive step forward in our efforts to bring hope to ALD patients and their families," said David Davidson, M.D., chief medical officer of bluebird bio. "We believe our lentiviral technology has the potential to be a one-time transformative therapy for patients suffering from rare genetic disorders like ALD for whom there are limited treatment options. bluebird is committed to advancing the clinical and commercial development of our gene therapy platform because of the dramatic benefit it may have on the lives of patients."Orphan drug designation, which is intended to facilitate drug development for rare diseases, provides benefits to the sponsor, including the potential for funding for certain clinical studies, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.
In April 2013, the FDA informed bluebird bio that the Investigational New Drug application (IND) the company filed in March 2013 for a Phase 2/Phase 3 clinical study to evaluate Lenti-D's effect in preserving neurological function and stabilizing cerebral demyelination in subjects with CCALD was active.
Adrenoleukodystrophy (ALD) is a rare X-linked, inherited neurological disorder. In its most severe form, ALD causes a breakdown of the myelin sheath in the brain (a protective and insulating membrane that surrounds nerve cells) and progressive dysfunction of the adrenal glands. This damage can result in decreased motor coordination and function, visual and hearing disturbances, the loss of cognitive function, dementia, seizures, adrenal dysfunction and other complications, including death.
Also known as "Lorenzo's Oil disease," named after the 1993 film starring Susan Sarandon and Nick Nolte, ALD is estimated to affect approximately one in every 20,000 boys worldwide. ALD, is identified in about 120 young boys in the United States each year. In the childhood cerebral form (CCALD), symptoms usually occur between the ages of 3 and 15 years. Symptoms often progress rapidly and can lead to a vegetative state and death.
Currently, the only effective treatment option is allogeneic hematopoietic stem cell transplant (HSCT), in which the patient is treated with HSCs containing the properly functioning copy of the gene, which is contributed by a donor other than the patient. Unfortunately, allogenic HSCT has significant morbidity and mortality, particularly in patients who undergo non-sibling matched allogeneic HSCT. Sibling matched donors are only typically available for less than 30% of patients. Yet even in these patients, severe graft versus host disease (GVHD) is the lead cause of mortality after transplantation. It is estimated that even at the best transplant centers, approximately 15% of patients suffer transplant related mortality, and approximately 30% experience GVHD.
bluebird bio is developing Lenti‐D, a lentivirus based autologous cell therapy, as a safer alternative to allogeneic HSCT. bluebird bio scientists have designed Lenti-D to restore functional ALDP production in a patient's own hematopoietic stem cells, eliminating the risk of GVHD.
LentiGlobin
bluebird bio is also developing LentiGlobin to treat patients with ß-thalassemia and sickle cell disease (SCD).ß-thalassemia is a rare hereditary blood disorder caused by a genetic abnormality of the ß-globin gene resulting in defective red blood cells. Symptoms of ß-thalassemia include severe anemia, splenomegaly, marrow expansion, bone deformities and iron overload in major organs. According to Thalassemia International Federation, about 288,000 patients with ß-thalassemia major are alive and registered as receiving regular treatment around the world, of which it is estimated that about 15,000 live in the United States and Europe.
Patients with beta-thalassemia major (the most severe form of beta-thalassemia) receive chronic blood transfusions aimed at maintaining a steady hemoglobin levels to treat their severe anemia. Chronic blood transfusions can be effective at preventing many symptoms of childhood beta thalassemia major, but chronic transfusions introduce a large iron overload, which over time may lead to mortality through iron-associated heart and liver toxicity. In order to prevent iron overload-associated risks, patients must adhere to daily iron chelation regimens. Poor compliance with chelation regimens remains a key challenge, and even with transfusion and iron chelation therapies, overall survival is significantly reduced.
The only potentially curative therapy for beta-thalassemia is allogeneic hematopoietic stem cell transplant (HSCT). Due to the significant risk of transplant related morbidity and mortality, transplants are offered primarily only to pediatric patients with matched sibling donors, which occurs in less than 25% of all cases. Allogeneic HSCT carries a significant risk of morbidity and mortality related to serious infection, graft failure and graft-versus-host-disease.
SCD is a hereditary blood disorder resulting from a mutation in the ß-globin gene that causes polymerization of hemoglobin proteins and abnormal red blood cell function. SCD is characterized by anemia, vaso-occlusive crisis (a common complication of SCD in which there is severe pain due to obstructed blood flow in the bones, joints, lungs, liver, spleen, kidney, eye, or central nervous system), infections, stroke, overall poor quality of life and early death in a large subset of patients. The global incidence of SCD is estimated to be 250,000 to 300,000 births annually, and the global prevalence of the disease is estimated to be about 20 million to 25 million.
According to GBI Research, the SCD market will be the fastest growing of the three markets during the forecast period with a Compound Annual Growth Rate (OTCPK:CAGR) of 9% seeing it reach $70 million in 2019. The thalassemia market will grow at a lower CAGR of 7% to reach $59 million in 2019.
bluebird's approach involves the insertion of a single codon variant of the normal ß-globin gene, referred to as T87Q, into the patient's own HSCs via an HIV-1 based lentiviral vector to restore expression of the ß-globin protein required for hemoglobin production. The codon variant is also used as a biomarker to quantify expression levels of ß-globin protein derived from the vector, and provides strong anti-sickling properties in the context of SCD. bluebird bio refers to the gene-modified HSCs as the final LentiGlobin drug product.
On September 15, 2010, bluebird bio announced promising Phase 1/Phase 2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia that was published in the September 2010 issue of Nature,
The patient, who had been transfusion dependent since early childhood, became transfusion independent for the past 21 months, more than two years after treatment with the LentiGlobin vector. The study also identified a subset of cells with the corrected beta-globin gene that overexpressed a truncated form of a gene called HMGA2. The patient has not experienced any adverse events. The data show that while early on, the HMGA2 clone was a significant portion of the corrected cells, the clone levels had declined at the time the paper was prepared, and further follow up indicates the decline is continuing.
"Although based on the first treated patient, we believe these results are impressive and illustrate for the first time the significant potential for treatment of beta hemoglobinopathies using lentiviral beta-globin gene transfer in the context of autologous stem cell transplant," said Philippe Leboulch, MD, senior author of the study and head of the Institute of Emerging Diseases and Innovative Therapies of CEA and INSERM; professor of medicine, University of Paris and visiting professor, Harvard Medical School. "For beta-thalassemia, we have worked intensely for almost 20 years to design, develop and manufacture LentiGlobin to provide a sustained high level hemoglobin production, resulting in a major clinical benefit. It has been very rewarding to follow this patient as his life has dramatically improved since receiving our treatment."On March 16, 2011, bluebird bio announced that it had entered into an agreement with the French Muscular Dystrophy Association (AFM), a French non-profit entity, whereby the company will receive an initial amount of approximately $1.4 million in cash to support the development of LentiGlobin. As part of the research agreement, bluebird bio has the option to draw upon an additional amount of up to $2.8 million in credit toward the manufacturing of cGMP clinical trial material at Généthon. In December 2010, bluebird bio entered into an agreement with Généthon designed to enable substantial advances in the existing manufacturing process of lentiviral vectors for the benefit of both partners.
"For the first time, a patient with severe beta-thalassemia is living without the need for transfusions over a sustained period of time," said Marina Cavazzana-Calvo, MD, first co-author of the study and professor of hematology, University of Paris and chief of Cell and Gene Therapy Department, Necker-Enfants Malades Hospital in Paris. Salima Hacein-Bey-Abina, PhD, professor of immunology, University of Paris, added, "These results are not only important due to the tremendous medical need that exists for thalassemia patients around the world, but also represents a significant step forward for the field of autologous stem cell therapy as an emerging therapeutic modality."
In a Phase 1/Phase 2 study of patients with ß-thalassemia major being conducted by the company's scientific collaborators in France with an earlier generation of its LentiGlobin vector called HPV569, data have provided initial evidence of transfusion independence following treatment with gene modified HSCs. Now bluebird bio plans to use its new LentiGlobin vector for studies based on higher transduction efficiency and expression of ß-globin protein in target cells as compared to the HPV569 vector. bluebird bio initiated a study in France using a revised clinical protocol based on the use of LentiGlobin instead of HPV569. This Phase 1/Phase 2 continuation study, known as the HGB-205 Study, will enroll up to seven additional subjects with ß-thalassemia major or SCD to evaluate transfusion requirements post-transplant, as well as the number of hospitalization days post-transplant discharge. In SCD patients only, efficacy will also be measured based on the number of vaso-occlusive crises or acute chest syndrome events
The company also plans to initiate a Phase 1/Phase 2 clinical study in the United States to evaluate its LentiGlobin product candidate in increasing hemoglobin production and eliminating or reducing transfusion dependence in patients with ß-thalassemia major, which the company refers to as the HGB-204 Study. Up to 15 adults will be enrolled to evaluate production of hemoglobin containing ßA-T87Q-globin for the six-month period between 18 and 24 months post-transplant, followed by long-term monitoring to assess safety and efficacy beyond the initial 24 months. bluebird bio expects to submit an IND with the FDA in 2014 to evaluate LentiGlobin in patients with SCD
In October 2012, bluebird bio announced that the California Institute for Regenerative Medicine (CIRM) approved an award to the company for $9.3 million for the first round of its new Strategic Partnership Awards initiative. The award is to support a Phase 1/2 study to evaluate the safety and efficacy of LentiGlobin for the treatment of beta-thalassemia.
CAR T
On March 21, 2013, bluebird announced the formation of a broad, global strategic collaboration with Celgene to discover, develop and commercialize novel disease- altering gene therapies in oncology.CAR T-cell therapy represents a promising, emerging approach to treating cancer. Blood is withdrawn from a patient and the T-cells are then extracted from a patient's blood. These cells are then genetically modified to recognize and attack cancer cells and then re-introduced into the patient's blood. The patient's genetically modified cells are intended to bind to and kill the target cancer cells.
Celgene has also entered into a separate strategic collaboration in the CAR T-cell field with the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital and The Methodist Hospital, Houston, led by Malcolm Brenner, M.D., Ph.D., professor, Department of Molecular and Human Genetics and the director, Center for Cell and Gene Therapy. bluebird bio, Celgene and Dr. Brenner's team will work collaboratively to advance and develop existing and new products and programs in the CAR T-cell field.
The collaboration is applying gene therapy technology to genetically modify a patient's own T-cells, known as chimeric antigen receptor (CAR) T-cells, to target and destroy cancer cells. The multi-year research and development collaboration has the potential to lead to the development and commercialization of multiple CAR T-cell products.
Under the agreement, Celgene has an option to license any products resulting from the collaboration after the completion of a Phase 1 clinical study for each such product. bluebird is responsible for research and development activity through Phase 1 studies.
"The genetic manipulation of autologous T-cells is a new frontier in oncology, one that shows early promise in emerging clinical trials," said Tom Daniel, president, research & early development at Celgene. "We see strong prospects for this collaboration between Celgene, bluebird bio and Baylor College of Medicine's experienced leaders in this emerging field, led by Dr. Brenner, to advance this innovative approach to intractable problems in oncology."Financial terms of the agreement include an upfront payment and up to $225 million per production potential option fees and clinical and regulatory milestones. bluebird also has the right to participate in the development and commercialization of any licensed products resulting from the collaboration through a 50/50 co-development and profit share in the United States in exchange for a reduction of milestones. Royalties would also be paid in regions where there is no profit share including in the United States if bluebird bio declines to exercise their co-development and profit sharing rights.
Finances
On August. 14, 2013, bluebird bio reported financial results and operational highlights for the quarter ended June 30, 2013.Revenues were $6.3 million during the quarter ended June 30, 2013 compared to $0.1 million for the quarter ended June 30, 2012 and include amounts allocated to research and development services from the Celgene collaboration.
Net cash provided by operating activities during the six months ended June 30, 2013 was $57.8 million. bluebird bio held $228.8 million in cash and cash equivalents as of June 30, 2013.
Total operating expenses for the quarter ended June 30, 2013 were $10.5 million as compared to $4.7 million for the same period in 2012.
bluebird bio reported a net loss of $4.6 million, or $2.13 per share, for the quarter ended June 30, 2013, as compared to a net loss of $4.6 million, or $23.21 per share, for the quarter ended June 30, 2012. The net loss applicable to basic and diluted common shareholders was $4.6 million for the quarter ended June 30, 2013, as compared to $5.8 million for the quarter ended June 30, 2012.
Conclusion
Most analysts are positive about bluebird bio. Motley Fool readers gave the stock a three star rating. Zacks has a 3 or Hold rating on the stock.On July 30, 2013, Cowen and Company initiated coverage of bluebird bio with an Outperform rating and a $44 price target.
On July 15, 2013, both Wedbush and JPMorgan initiated coverage of bluebird bio with Outperform ratings. Wedbush put a $32.25 price target on the stock. Both Bank of America and Canaccord Genuity began coverage of bluebird bio with a Buy rating. Canaccord set a $45 price target, and Bank of America put a $42 price target on the stock. JPMorgan gave bluebird bio a $44 price target.
Top institutional holders of bluebird bio stock are Ameriprise Financial Inc., Camber Capital Management, LLC, and Citadel Advisors LLC. Top mutual fund holders are Fidelity Contrafund, Prudential Jennison Health Sciences Fund, American Funds Insurance Ser-Global Small Capitalization Fund, Fidelity Growth Company Fund, Fidelity Growth Company Fund, Waddell & Reed Advisors Funds-Science & Technology Fund, Ivy Science & Technology Fund, Fidelity Advisor New Insights Fund, Fidelity Blue Chip Growth Fund, and Franklin Strategic Series-Franklin Biotechnology Discovery Fund.
Nick Leschly has served as the company's president and chief executive officer since September 2010. Previously, he served as the company's interim chief executive officer from March 2010 to September 2010. Formerly a partner of Third Rock Ventures, L.P. since its founding in 2007, Leschly played an integral role in the overall formation, development and business strategy of several of Third Rock's portfolio companies, including Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) and Edimer Pharmaceuticals, Inc.
bluebird bio's gene therapy platform holds the promise of delivering a one-time treatment for a wide variety of diseases. The company's initial focus is on genetic diseases caused by a known mutation in a single gene. Through the application of gene therapy, bluebird bio is developing product candidates designed to insert a functional gene into a patient, potentially correcting their underlying disease. In addition to genetic diseases, bluebird bio and its partners are also utilizing this technology to develop product candidates intended to treat various cancers, by targeting a patient's own immune system to seek out and kill cancer cells in the body, offering a new approach to cancer treatment.
Although gene therapy shows great promise for a number of currently incurable diseases, the technique remains risky and is still under study to ensure that it is safe and effective. Currently, gene therapy is only being tested for the treatment of diseases that have no other cures.
bluebird bio is well positioned to drive the continued advancement of gene therapy technology for the treatment of severe genetic and orphan diseases. The company has assembled extensive expertise in viral vector design, manufacturing and gene transfer, a broad intellectual property estate, an experienced management team and a high caliber group of scientific advisors and key opinion leaders.
Disclosure: I am long CELG, MRK. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article
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