Stem Cell Clinic

bluebird bio Reports Third Quarter 2019 Financial Results and Highlights Operational Progress – Business Wire

By Stell Cell Research

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (NASDAQ: BLUE) today reported financial results and business highlights for the third quarter ended September 30, 2019.

During the third quarter we advanced our country-by-country launch plans in Europe and, with the recent approval of the commercial drug product manufacturing specifications for ZYNTEGLO, we moved one step closer to our goal of treating patients suffering from TDT in early 2020, said Nick Leschly, chief bluebird. Also this quarter, we presented updated data from the Phase 2/3 Starbeam study in patients with CALD. To report that patients continued to be free of MFDs at up to five years of follow-up is something were tremendously proud to do for these families, and we look forward to advancing that program in the regulatory process next year. Looking ahead, we plan to provide clinical updates for ZYNTEGLO and across the rest of our portfolio, including LentiGlobin in sickle cell disease, bb21217 in multiple myeloma, and from our registration-enabling KarMMa study of ide-cel in patients with multiple myeloma by the end of this year. Id like to thank all the bluebirds around the globe for their tireless focus on doing the right thing for our patients weve seen amazing progress thus far in 2019 and I look forward to ending the year on a strong note.

Recent Highlights:

TDT

CALD

COMPANY

Upcoming Anticipated Milestones:

Third Quarter 2019 Financial Results

About bluebird bio, Inc.bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene 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. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent -thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking StatementsThis release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the companys financial condition, results of operations, as well as statements regarding the plans for regulatory submissions and commercialization for ZYNTEGLO and the companys product candidates, including anticipated regulatory milestones, planned commercial launches, planned clinical studies, as well as the companys intentions regarding the timing for providing further updates on the development and commercialization of ZYNTEGLO and the companys product candidates. Any forward-looking statements are based on managements 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 will not continue or be repeated in our ongoing or future clinical trials, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, risks that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, the risk that we will encounter challenges in the commercial launch of ZYNTEGLO in the European Union, including in managing our complex supply chain for the delivery of drug product or in the adoption of value-based payment models or in obtaining sufficient coverage or reimbursement for our products if approved, the risk that our collaborations, including the collaboration with Celgene, will not continue or will not be successful, 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.

bluebird bio, Inc.Consolidated Statements of Operations(in thousands, except per share data)(unaudited)

For the three months ended September 30,

For the nine months ended September 30,

2019

2018

2019

2018

Revenue:

Collaboration revenue

$

6,575

$

10,926

$

29,310

$

33,971

License and royalty revenue

2,335

602

5,367

1,365

Total revenues

8,910

11,528

34,677

35,336

Operating expenses:

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bluebird bio Reports Third Quarter 2019 Financial Results and Highlights Operational Progress - Business Wire

Global Cell Culture Protein Surface Coating Market: What it got next? Find out with the latest research available at ‘The Market Reports’ – Market…

By Stell Cell Research

Cell culture is a complex procedure in which cells are grown under controlled physical conditions outside the natural environment. These cells are used to develop model systems for research, study of cellular structure and functions, stem cell research, drug discovery and genetic engineering. Growing scope of cell culture and its applications has led to increased use of protein coated surfaces, as these provide better adhesion and proper nutrition for growth of the cells during cell culture.

Rising investment by government and market players in stem cell research and development activities is one of the major factors driving the cell culture protein surface coatings market. Becton, Dickinson and Company grants a total of USD 100,000 worth reagents every year to 10 scientists pursuing research activities in stem cells. Similarly, the European Union funded four stem cell research projects in its Seventh Framework Program for Research and Technological Development (2007 2013). High funding is leading to extensive stem cell research, resulting in increased use of cell culture protein surface coating products. Moreover, diverse applications of stem cells such as development of bone grafts and artificial tissue would fuel the demand for cell culture protein surface coatings during the forecast period. In addition, increasing cell culture applications in toxicology studies and cell-based assays would boost the demand for protein surface coating products. Currently, 2D cell culture is the most preferred technique by researchers worldwide due to lack of compelling data to switch to 3D cell culture.

The Americas accounted for the majority market share during 2016 and will continue to dominate the market during the forecasted period. The presence of highly developed healthcare infrastructure and the increasing demand for stem cell therapies and regenerative medicines for orthopedics, neurology, and autoimmune therapies are some of the major factors responsible for the markets growth in this region.

Access Report Details at: https://www.themarketreports.com/report/global-cell-culture-protein-surface-coating-market-research-report

The global Cell Culture Protein Surface Coating market is valued at xx million US$ in 2018 is expected to reach xx million US$ by the end of 2025, growing at a CAGR of xx% during 2019-2025.

This report focuses on Cell Culture Protein Surface Coating volume and value at global level, regional level and company level. From a global perspective, this report represents overall Cell Culture Protein Surface Coating market size by analyzing historical data and future prospect. Regionally, this report focuses on several key regions: North America, Europe, China and Japan.

Key companies profiled in Cell Culture Protein Surface Coating Market report are Corning, EMD Millipore, Thermo Fisher Scientific, Sigma-Aldrich and more in term of company basic information, Product Introduction, Application, Specification, Production, Revenue, Price and Gross Margin (2014-2019), etc.

Purchase this Premium Report at: https://www.themarketreports.com/report/buy-now/1412777

Table of Content

1 Cell Culture Protein Surface Coating Market Overview

2 Global Cell Culture Protein Surface Coating Market Competition by Manufacturers

3 Global Cell Culture Protein Surface Coating Production Market Share by Regions

4 Global Cell Culture Protein Surface Coating Consumption by Regions

5 Global Cell Culture Protein Surface Coating Production, Revenue, Price Trend by Type

6 Global Cell Culture Protein Surface Coating Market Analysis by Applications

7 Company Profiles and Key Figures in Cell Culture Protein Surface Coating Business

8 Cell Culture Protein Surface Coating Manufacturing Cost Analysis

9 Marketing Channel, Distributors and Customers

10 Market Dynamics

11 Global Cell Culture Protein Surface Coating Market Forecast

12 Research Findings and Conclusion

13 Methodology and Data Source

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Global Cell Culture Protein Surface Coating Market: What it got next? Find out with the latest research available at 'The Market Reports' - Market...

SCD, HIV Gene Therapy Efforts Get $200M from NIH, Gates Foundation – Sickle Cell Anemia News

By Stell Cell Research

The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale.

This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries, said NIH Director Francis S. Collins, MD, PhD.

Seventy-five percent of babies born with SCD live in sub-Saharan Africa. It is hoped that experimental gene therapies would advance to clinical trials in the United States and relevant African countries within the next seven to 10 years, and that safe, effective, and inexpensive gene therapies be made available globally, including in low-resource settings where the cost and complexity of these therapies make them inaccessible to many.

In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases, Trevor Mundel, MD, PhD, president of the global health program at the Bill & Melinda Gates Foundation said in a news release.

While these treatments are exciting, people in low- and middle-income countries do not have access to these breakthroughs. By working with the NIH and scientists across Africa, we aim to ensure these approaches will improve the lives of those most in need and bring the incredible promise of gene-based treatments to the world of public health, he added.

Hemoglobin is the protein in red blood cells that binds oxygen, allowing oxygen to be transported around the body. Mutations in the HBBgene, which encodes a component of hemoglobin, result in the formation of sickle hemoglobin that causes sickle cell anemia.

Currently, gene therapies for SCD involves altering the patients own hematopoietic stem cells (bone marrow cells that divide and specialize to produce blood cells including red blood cells). Genes are introduced into the cells using a modified, harmless virus (known as a viral vector). The cells are then transplanted back into the patient where they will produce healthy red blood cells. Gene therapy has an advantage over a bone marrow transplant, as it circumvents the complications associated with a bone marrow donation.

The first goal of the collaboration between the NIH and the Gates Foundation is to develop an easy-to-administer gene-based intervention to correct the mutations in the HBBgene or deliver a functional gene that will promote the production of normal levels of hemoglobin without the need to extract cells from patients and modify them in the lab before introducing the cells back. However, this strategy, known as in vivotreatment, requires the advancement of more efficient delivery systems that can deliver the gene therapy specifically to hematopoietic stem cells.

A second goal of the collaboration will be to work together with African partners and bring potential therapies to clinical trials.

Further research is required to understand the burden of SCD in sub-Saharan Africa and to screen newborns at high risk for the disease, a task that the National Heart, Lung and Blood Institute (NHLBI) has started to tackle by building the necessary infrastructure for clinical research.

The NIH and the Gates Foundation will help boost this infrastructure to allow point-of-care screening (for example, when infants receive vaccinations), and to initiate a standard of care. This will occur outside of the official collaboration.

Our excitement around this partnership rests not only in its ability to leverage the expertise in two organizations to reduce childhood mortality rates in low-resource countries, but to bring curative therapies for sickle cell disease and HIV to communities that have been severely burdened by these diseases for generations, said Gary H. Gibbons, MD, director of the NHLBI.

A persons health should not be limited by their geographic location, whether rural America or sub-Saharan Africa; harnessing the power of science is needed to transcend borders to improve health for all, he added.

Matshidiso Rebecca Moeti, the regional director for Africa at the World Health Organization said, We are losing too much of Africas future to sickle cell disease and HIV.

Beating these diseases will take new thinking and long-term commitment. Im very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africas greatest public health challenges, Moeti added.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 94

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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SCD, HIV Gene Therapy Efforts Get $200M from NIH, Gates Foundation - Sickle Cell Anemia News

Three UCLA scientists receive grants totaling more than $18 million – Newswise

By Stem Cell Medical Center

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Newswise Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.

The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.

Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.

Continued here:
Three UCLA scientists receive grants totaling more than $18 million - Newswise

Doheny and UCLA Stein Eye Institutes Welcome Kaustabh Ghosh, PhD, to the Scientific and Clinical Research Faculty – PRNewswire

By Stem Cell Medical Center

"I am tremendously proud and honored to begin this position at Doheny-UCLA," says Dr. Ghosh. "I entered the field of biomedical research from an engineering background, which perhaps gave me a new perspective to see things differently. As a biomedical engineer, it allowed me to think about diseases in a way that a typical biomedical researcher and clinician may not."

Dr. Ghosh states that also as a vascular biologist, one such perspective he was able to successfully introduce was the importance of "stiffness" of blood vessels in disease pathogenesis.

"Doheny will be the ideal place for me to realize the true translational potential for my work as it offers strength and resources in ophthalmic imaging," shares Dr. Ghosh. "Doheny also provides the perfect balance between basic science and clinical research."

He adds, "I look forward to developing strong, collaborative relationships with members of Doheny-UCLA engineering, biomedical sciences and clinical infrastructure. Our goal will be to discover effective treatment strategies from a multidisciplinary approach especially in the area of investigating the role of chronic vascular inflammation, a major determinant of various debilitating conditions including macular degeneration and diabetic retinopathy."

Dr. Ghosh was most recently Associate Professor of Bioengineering at University of California, Riverside (UCR) as well as Participating Faculty in the Division of Biomedical Sciences, Stem Cell Center and the Program in Cell, Molecular and Developmental Biology. The Ghosh Research Group at UCR focused on leveraging the principles of mechanobiology to examine and treat inflammationmediated vascular degeneration associated with diabetic retinopathy and agerelated macular degeneration, the leading causes of vision loss in the diabetic and aging population. In 2016, these studies were supported by two R01 grants from the National Eye Institute (NEI), and a macular degeneration grant from the BrightFocus Foundation. Dr. Ghosh has received numerous awards during his research career, including the Hellman Fellowship and the NIH Postdoctoral Training Grant, and has published 24 peer-reviewed papers in highly-regarded journals that include PNAS, The FASEB Journal, Science, and Nano Letters, among others.

In 2011, prior to joining UCR, Dr. Ghosh was a postdoctoral fellow in the laboratory of Donald Ingber, MD, PhD, part of the Vascular Biology Program at Boston Children's Hospital and Harvard Medical School. In 2006, Dr. Ghosh received his PhD in Biomedical Engineering from Stony Brook University, New York. He obtained his undergraduate degree in Chemical Engineering from National Institute of Technology, Warangal, India in 2001.

Dr. Ghosh's dedication to collaborative research and team building is evident in his numerous and illustrious achievements. His distinguished scientific leadership demonstrates an excellence that will contribute greatly to Doheny Eye Institute's research programs.

About Doheny Eye InstituteFor over 70 years, Doheny Eye Institute has been at the forefront of vision science. From seeking new ways to free blockages that prevent fluid drainage in glaucoma, to replacing retinal cells in age-related macular degeneration, to providing colleagues worldwide with standardized analyses of anatomical changes in the eyes of patients, Doheny clinicianscientists and researchers are changing how people see and also how they think about the future of vision. Please visit doheny.org for more information.

Doheny Eye Institute and UCLA Stein Eye Institute have joined forces to offer the best inpatient care, vision research and education. This affiliation combines the strength, reputation and distinction of two of the nation's top eye institutions to advance vision research, education and patient care in Southern California.

CONTACT INFORMATIONMedia Contact:Matthew RabinDirect: (323) 342-7101Email: mrabin@doheny.org

SOURCE Doheny Eye Institute

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Doheny and UCLA Stein Eye Institutes Welcome Kaustabh Ghosh, PhD, to the Scientific and Clinical Research Faculty - PRNewswire

bluebird bio Reports Third Quarter 2019 Financial Results and Highlights Operational Progress – Yahoo Finance

By Stem Cell Medical Center

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

- Continued progress towards 2022 vision of four marketed gene and cell therapy products with robust development pipeline -

- ZYNTEGLO commercial launch advancing with European Medicines Agency approval of refined commercial manufacturing process -

- Ended quarter with $1.41 billion in cash, cash equivalents and marketable securities

bluebird bio, Inc. (BLUE) today reported financial results and business highlights for the third quarter ended September 30, 2019.

During the third quarter we advanced our country-by-country launch plans in Europe and, with the recent approval of the commercial drug product manufacturing specifications for ZYNTEGLO, we moved one step closer to our goal of treating patients suffering from TDT in early 2020, said Nick Leschly, chief bluebird. Also this quarter, we presented updated data from the Phase 2/3 Starbeam study in patients with CALD. To report that patients continued to be free of MFDs at up to five years of follow-up is something were tremendously proud to do for these families, and we look forward to advancing that program in the regulatory process next year. Looking ahead, we plan to provide clinical updates for ZYNTEGLO and across the rest of our portfolio, including LentiGlobin in sickle cell disease, bb21217 in multiple myeloma, and from our registration-enabling KarMMa study of ide-cel in patients with multiple myeloma by the end of this year. Id like to thank all the bluebirds around the globe for their tireless focus on doing the right thing for our patients weve seen amazing progress thus far in 2019 and I look forward to ending the year on a strong note.

Recent Highlights:

TDT

ZYNTEGLO COMMERCIAL READINESS In October, bluebird bio announced that the European Medicines Agency (EMA) approved the refined commercial drug product manufacturing specifications for ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene), a one-time gene therapy for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. With this update, apceth is in the final stages of preparing to manufacture ZYNTEGLO for commercial use. The company continues to proceed with discussions on value-based payment agreements and Qualified Treatment Center contracts and expects to treat the first commercial patient in early 2020.

CALD

Story continues

DATA FROM STARBEAM STUDY (ALD-102) AND ALD-103 PRESENTED At the 13th European Pediatric Neurology Society (EPNS) Congress in September 2019, bluebird bio presented new data from the clinical development program for its investigational studies of Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy: updated data from the Phase 2/3 Starbeam study (ALD-102) in boys 17 years of age and under with CALD and updated data from the ongoing observational study (ALD-103) of allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys 17 years of age and under with CALD. Long-term follow-up data as of April 2019 showed that the 88% of patients treated in the Starbeam study (ALD-102) were free of major functional disabilities (MFDs) at two years, and continued to remain MFD-free at up to five years of follow-up.

COMPANY

FIRST PATIENT TREATED IN PHASE 1/2 TRIAL FOR MERKEL CELL CARCINOMA (MCC) In August 2019, Fred Hutchinson Cancer Research Center infused the first patient in their proof-of-concept phase 1/2 single-arm study evaluating Merkel Cell Polyomavirus (MCPyV) TCR-engineered autologous T cells in combination with avelumab (anti-PDL1) for the treatment of MCC. Results from the academic phase 1/2 single-arm study are expected to inform next-generation T cell approaches including TCR engineering and checkpoint inhibition. The study will enroll approximately 16 patients. Development of this program is led by Fred Hutchinson Cancer Research Center. bluebird bio retains the exclusive option to license this program.

NOVO NORDISK COLLABORATION In October 2019, bluebird bio and Novo Nordisk announced a research collaboration to jointly develop next-generation in vivo genome editing treatments for genetic diseases, including hemophilia. During the three-year research collaboration, bluebird and Novo Nordisk will focus on identifying a development gene therapy candidate with the ambition of offering people with hemophilia A a lifetime free of factor replacement therapy.

MANAGEMENT UPDATE In October 2019, bluebird bio announced that Jeffrey T. Walsh, chief strategy officer, has decided to transition from his current role effective January 6, 2020. Jeff has not only built a strong foundation for bluebirds overall growth strategy but also leaves an experienced and passionate team. Both Chip Baird, chief financial officer, and Joanne Smith-Farrell, chief business officer, will assume broader corporate development and strategic responsibilities as bluebird continues to deliver on its mission for patients.

NEW BOARD APPOINTMENT In September 2019, bluebird bio announced the appointment of William R. Sellers, M.D. to its Board of Directors.

Upcoming Anticipated Milestones:

TDT

Initiation of a rolling Biologics Licensing Application submission to the U.S. FDA for ZYNTEGLO in patients with TDT and non-0/0 genotypes by the end of 2019

Presentation of ZYNTEGLO clinical data from the Northstar-2 (HGB-207) clinical study in patients with TDT and non-0/0 genotypes by the end of 2019

Presentation of ZYNTEGLO clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and a 0/0 genotype or an IVS-I-110 mutation by the end of 2019

SCD

Phase 3 HGB-210 study of LentiGlobin in patients with SCD open and enrolling by the end of 2019

Presentation of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD by the end of 2019

Multiple Myeloma

Ide-cel clinical data update from the registration-enabling KarMMa study in patients with relapsed/refractory multiple myeloma by the end of 2019

Presentation of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma by the end of 2019

Third Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2019 and December 31, 2018 were $1.41 billion and $1.89 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating activities and cash used to purchase property, plant and equipment, including those purchases related to the companys buildout of its manufacturing facility in Durham, North Carolina.

Revenues: Collaboration and license and royalty revenues were $8.9 million for the three months ended September 30, 2019 compared to $11.5 million for the three months ended September 30, 2018. Collaboration and license and royalty revenues were $34.7 million for the nine months ended September 30, 2019 compared to $35.3 million for the nine months ended September 30, 2018. The decrease in both periods was primarily attributable to a decrease in collaboration revenue under our arrangement with Celgene, partially offset by an increase in license and royalty revenue and collaboration revenue under our arrangement with Regeneron.

R&D Expenses: Research and development expenses were $151.4 million for the three months ended September 30, 2019 compared to $116.7 million for the three months ended September 30, 2018. Research and development expenses were $420.6 million for the nine months ended September 30, 2019 compared to $328.9 million for the nine months ended September 30, 2018. The increase in both periods was primarily driven by costs incurred to advance and expand the companys pipeline.

G&A Expenses: General and administrative expenses were $66.3 million for the three months ended September 30, 2019 compared to $44.5 million for the three months ended September 30, 2018. General and administrative expenses were $195.2 million for the nine months ended September 30, 2019 compared to $120.6 million for the nine months ended September 30, 2018. The increase in both periods was largely attributable to costs incurred to support the companys ongoing operations and growth of its pipeline as well as commercial-readiness activities.

Net Loss: Net loss was $206.0 million for the three months ended September 30, 2019 compared to $145.5 million for the three months ended September 30, 2018. Net loss was $566.3 million for the nine months ended September 30, 2019 compared to $406.6 million for the nine months ended September 30, 2018.

About bluebird bio, Inc. bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene 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. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent -thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

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 companys financial condition, results of operations, as well as statements regarding the plans for regulatory submissions and commercialization for ZYNTEGLO and the companys product candidates, including anticipated regulatory milestones, planned commercial launches, planned clinical studies, as well as the companys intentions regarding the timing for providing further updates on the development and commercialization of ZYNTEGLO and the companys product candidates. Any forward-looking statements are based on managements 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 will not continue or be repeated in our ongoing or future clinical trials, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, risks that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, the risk that we will encounter challenges in the commercial launch of ZYNTEGLO in the European Union, including in managing our complex supply chain for the delivery of drug product or in the adoption of value-based payment models or in obtaining sufficient coverage or reimbursement for our products if approved, the risk that our collaborations, including the collaboration with Celgene, will not continue or will not be successful, 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.

bluebird bio, Inc. Consolidated Statements of Operations (in thousands, except per share data) (unaudited)

For the three months ended September 30,

For the nine months ended September 30,

2019

2018

2019

2018

Revenue:

Collaboration revenue

$

6,575

$

10,926

$

29,310

$

33,971

License and royalty revenue

2,335

602

Excerpt from:
bluebird bio Reports Third Quarter 2019 Financial Results and Highlights Operational Progress - Yahoo Finance

BioLineRx to Present Two Posters at the Society for Immunotherapy of Cancer (SITC) 2019 – PRNewswire

By Stem Cell Medical Center

TEL AVIV, Israel, Oct. 31, 2019 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, announced today that it will deliver the following poster presentations at the Society for Immunotherapy of Cancer(SITC) 34th Annual Meeting to take place November 6-10, 2019 at the Gaylord National Hotel & Convention Center in Baltimore, Maryland:

About BL-8040

BL-8040 is a short synthetic peptide that functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor over-expressed in many human cancers, where it has been shown to be correlated with poor prognosis, and plays a key role in tumor growth, invasion, angiogenesis, metastasis and therapeutic resistance. CXCR4 is also directly involved in the homing and retention of hematopoietic stem cells (HSCs) and various hematological malignant cells in the bone marrow.

In a number of clinical and pre-clinical studies, BL-8040 has shown a critical role in immune cell trafficking, tumor infiltration by immune effector T cells and reduction in immunosuppressive cells within the tumor niche, turning "cold" tumors, such as pancreatic cancer, into "hot" tumors (i.e., sensitizing them to immune check point inhibitors). BL-8040-mediated inhibition of the CXCR4-CXCL12 (SDF-1) axis has also shown robust mobilization of HSCs for transplantation in hematological malignancies.

BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About BioLineRx

BioLineRx is a clinical-stage biopharmaceutical company focused on multiple oncology indications. The Company'slead program, BL-8040, is a cancer therapy platform currently being evaluated in a Phase 2a study in pancreatic cancer in combination with KEYTRUDA and chemotherapy under a collaboration agreement with MSD. BL-8040 is also being evaluated in a Phase 2b study in consolidation AML and a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation. In addition, the Company has an ongoing collaboration agreement with Genentech, a member of the Roche Group, evaluating BL-8040 in combination with Genentech's atezolizumab in two Phase 1b/2 solid tumor studies.

BioLineRx is developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being evaluated in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated on Facebook,Twitter, and LinkedIn.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Some of these risks are: changes in relationships with collaborators; the impact of competitive products and technological changes; risks relating to the development of new products; and the ability to implement technological improvements. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 28, 2019. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564tim@lifesciadvisors.com

or

Tsipi HaitovskyPublic Relations+972-52-598-9892tsipihai5@gmail.com

SOURCE BioLineRx Ltd.

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BioLineRx to Present Two Posters at the Society for Immunotherapy of Cancer (SITC) 2019 - PRNewswire

A winding romp through advances in cell biology pushes readers to ponder the boundaries of life – Science Magazine

By Embryonic Stem Cells

Philip BallUniversity of Chicago Press2019384 pp.Purchase this item now

A small bundle of human nerve cells are being cultured in a petri dish. The cells divide. They differentiate into cell types found in the brain. The cell network grows dense and develops brain-like structureslayers and folds. The cells begin to signal. The brain cell cluster has been derived from skin cells harvested from science writer Philip Balls shoulder.

The scientists who created Balls skin-turned-brain organoid study brain development and want to understand the basis of neurodegeneration. But what exactly goes on inside these cell aggregates, and could we reach a point at which they are more brain than brain-like?

Biologists can also build embryo-like structures (embryoids) from human stem cells, which can be used to study early prenatal development. However, synthetic embryos can develop certain featuressuch as the primitive streak, a structure that establishes bilateral symmetry in an organismthat mark, for some, the transition from embryo to individual human being.

Balls experience grappling with how to think about these living structures, as documented in his new book, How to Grow a Human, is part of a larger question with which humanity has wrestled for centuries: What is life, and how might our understanding of it change with our ever-increasing capability to manipulate it?

The book offers a provocative, meandering take on the progression of groundbreaking biotechnological capabilities. For example, in chapter 3, Ball explores the dawn of tissue culture at the turn of the 20th century and the motivations of the scientists who conducted the research. Ross Harrison sought to settle a debate between Camillo Golgi and Santiago Ramny Cajal over the makeup of nervous systems; the former argued that nervous systems were one uninterrupted structure, whereas the latter believed there to be distinct nerve cells. Along the way to showing that nerve fibers lengthen through nerve cell proliferation, confirming Ramny Cajals hypothesis, Harrison was the first to develop a technique to keep tissues alive with active cell growth in vitro, sustaining amphibian embryonic tissue in jars.

Alexis Carrel, on the other hand, was a white supremacist striving to preserve a superior stock of humankind. Carrel and his team iterated on and applied Harrisons method to many different tissues, including those of birds, embryonic chickens, and, of course, humans. Here, Ball also works in how science fiction writing was influenced by early advances in cell biology, describing Julian Huxleys The Tissue-Culture King, which centers on a biologist who redesigns members of a remote tribe and builds living objects of worship from the flesh of the tribes king. Although interesting, asides such as this disrupt the narratives continuity.

Balls writing is most absorbing when he reflects on boundary-pushing research, such as advances toward converting human skin cells to eggs or sperm or the promise of approaches for fabricating human organs to help people who need transplants. In chapter 5, for example, he describes experiments in which rat cells formed pancreases in mice, and others in which human cells survived in pig and cattle embryos, and then considers how governments and the public might approach the prospect of harvesting human organs grown in other animals.

Discussing how and where we have drawn ethical and legal lines for procedures such as in vitro fertilization and preimplantation genetic diagnosis (PGD) of embryos, Ball contemplates what historical precedent may mean for the governance of emerging biotechnological capabilities. Unlike in the United Kingdom, where PGD is permitted only to avoid implanting an embryo with a serious heritable disease, the United States does not regulate PGD-enabled embryo selection at the federal level, meaning PGD can be used to select for offspring of a particular gender or to rule out embryos that have an elevated risk of intellectual disability. (As Ball points out, it may be possible to adapt this testing to screen for embryos that are predicted to have exceptional cognitive ability.)

At the center of an adjacent debate are germline genome-editing technologies. As exemplified by the so-called CRISPR-baby controversy and expounded upon by Ball, access to, and affordability of, new biotechnologies may serve some segments of society while underserving others. Ball appeals to the democratic process to determine the balance between personal liberty and state-dictated equity, acknowledging that everyone has a stake in and therefore the right to be heard on this important issue.

Because of the immense power of emerging biotechnologies, those of us who are intimately involved with these advances must make a concerted effort to equip both policy-makers and the public with the knowledge and tools needed to navigate this evolving landscape. Ambitious and expansive, How to Grow a Human could be one piece of this effortBalls look at the state of human-facing cutting-edge bioscience is a thought-provoking read

The reviewer co-leads the Congressional Science Policy Initiative at the Federation of American Scientists, Washington, DC 20036, USA

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A winding romp through advances in cell biology pushes readers to ponder the boundaries of life - Science Magazine

Stem Cell Research and Hair Transplants How Far Have We Come? – Science Times

By Stem Cell Clinic

(Photo : Stem cell research and hair transplants how far have we come?)

We all know that hair loss is a major problem, particularly for men around the world. While men who have enough hair left can have hair transplant procedures such as the FUT, FUE or DHI, done, this is not always possible.

It is always a smart move to consult with a hair loss specialist regardless of your situation because they may still be able to suggest something if you still have some hair. Only the specialist can tell assess what your situation is and what options are available. You can, for example, always contact a specialist at the Vera Clinic in Turkey to get evaluated.

In fact, men who are already very bald and have lost most of the hair found in the donor areas such as the back of the head may be out of luck when it comes to being able to have transplant surgery. This is a reason that scientists have been conducting experiments using stem cells. Some types of stem cells can be used to regenerate other types of cells and tissues in the body, so it is only natural that the idea came about to use stem cells to grow new hair follicles.

Stem cell research

There is often a lot of controversy regarding stem cell research because many people, including politicians, think that only human fetuses have stem cells and thus they argue that it is an unethical area of research. However, everybody has stem cells, not just fetuses, and it is a person's own stem cells that hold the potential to make new tissues. This is also partly why PRP therapy has been so successful, because the plasma actually contains many stem cells which trigger growth and repair of tissues.

Researchers have been able to grow some human hair follicles in the laboratory using stem cells. These same hairs were then transplanted into a mouse. Human testing cannot begin until animal testing is completed and many countries have rigorous processes in place when it comes to scientific investigations in humans.

This means that we can expect it to be some time yet before any human trials can take place using stem cells and hair transplants from follicles that are grown in the lab. The other problem which the scientists have noted is that it is more complicated than it seems since even though hair was transplanted into the mice, the outcome was not good and the hair was found to grow at odd angles. This could have been because it was a transplant between different species, but the reality is that hair transplantation is not as simple as it seem.

Stem cells may, however, be the last chance for people who have lost most of their hair. Scientists think that autologous hair transplants based on growing hair from a person's own stem cells may be something that becomes a reality in the future. At the moment, more studies need to be undertaken and the best advice is to seek treatment for hair loss before it reaches a point where nothing can be done.

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Stem Cell Research and Hair Transplants How Far Have We Come? - Science Times

New NCCN Guidelines Debut to Manage Complications and Improve Readiness for Stem Cell Transplant Recipients – Yahoo Finance

By Stem Cell Clinic

National Comprehensive Cancer Network expands resources to meet growing utilization of cell-based cancer treatments

PLYMOUTH MEETING, Pa., Oct. 30, 2019 /PRNewswire/ --Today, the National Comprehensive Cancer Network (NCCN ) published new NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines ) for Hematopoietic Cell Transplantation (HCT), also known as stem cell transplant or historically as bone marrow transplant. This new resource provides step-by-step information on best practices in evaluating patients for hematopoietic cell transplantation and managing complications afterwards. This type of specialized treatment is increasingly common, occurring approximately 22,000 times a year in the United States in people with various malignancies, most commonly for blood-related cancers.1

NCCN Logo (C)NCCN(R) 2018. All rights reserved.

"Establishing NCCN Guidelines for Hematopoietic Cell Transplantation is a key accomplishment in the management of blood cancers," said Ayman A. Saad, MD, Professor of Clinical Medicine, The Ohio State University Comprehensive Cancer Center James Cancer Hospital and Solove Research Institute, Chair of the NCCN Guidelines Panel for HCT. "The current version of the guidelines addresses both pre-transplant evaluation and the management of a common complication: graft versus host disease (GVHD). Given the diversity of practice and expertise, we believe these guidelines will provide a pivotal tool for learning about the continuously updated therapy landscape in HCT. We hope this will help streamline clinical practices and educate new generations of physicians-in-training."

The guidelines provide recommendations on how to evaluate a potential transplant recipient to determine if the patient is an appropriate candidate for the procedure, and how to best manage different manifestations of post-transplant GVHD. They reflect the latest evidence and consensus from foremost experts across the 28 leading academic cancer centers that comprise NCCN, including hematologists/oncologists, transplant-specific practitioners, and infectious disease specialists.

"These guidelines provide an algorithmic pathway for a systematic approach to allogeneic (donor) transplantation across several different cancer types including leukemia, lymphoma, and multiple myeloma," explained Marcos de Lima, MD, Professor of Medicine, Case Comprehensive Cancer Center and University Hospitals of Cleveland , Vice-Chair of the NCCN Guidelines Panel for HCT. "Thankfully, the number of blood and bone marrow donors has increased substantially in just the past decade. When you combine the National Marrow Donor Program registry adult donors with cord blood donors and relatives (matched and mismatched), we are now able to perform this potentially cancer-curing procedure on significantly more patients than we could in the past. That's why it's so important to set standards for preventing and treating common adverse events and infections."

"Early referral for consideration of HCT can be life-saving, so we strongly encourage all oncologists to take a look at these guidelines and refer any possible candidates to transplant centers for evaluation," said Alison W. Loren, MD, MSCE, Director, Blood & Marrow Transplant, Cell Therapy & Transplant Program, Abramson Cancer Center of the University of Pennsylvania , Member of the NCCN Guidelines Panel for HCT. "We also urge oncologists who may be caring for patients after HCT to familiarize themselves with the varied manifestations of GVHDa very common and significant post-transplant complicationand to consult with transplant providers to optimize their ongoing care. The guidelines explain how to diagnose and treat this condition in order to achieve the best possible outcomes."

Story continues

The NCCN Guidelines for Hematopoietic Cell Transplantation are available free-of-charge for non-commercial use at NCCN.org and via the recently improved Virtual Library of NCCN Guidelines App for smartphone and tablet. NCCN will continue expanding blood cancer resources through continuous updates to the HCT guidelines, along with upcoming new NCCN Guidelines for Histiocytosis, Myeloid/Lymphoid Neoplasms, Pediatric B-Cell Lymphomas, and Pediatric Hodgkin Lymphoma.

About the National Comprehensive Cancer NetworkThe National Comprehensive Cancer Network (NCCN) is a not-for-profit alliance of 28 leading cancer centers devoted to patient care, research, and education. NCCN is dedicated to improving and facilitating quality, effective, efficient, and accessible cancer care so patients can live better lives. Through the leadership and expertise of clinical professionals at NCCN Member Institutions, NCCN develops resources that present valuable information to the numerous stakeholders in the health care delivery system. By defining and advancing high-quality cancer care, NCCN promotes the importance of continuous quality improvement and recognizes the significance of creating clinical practice guidelines appropriate for use by patients, clinicians, and other health care decision-makers around the world.

The NCCN Member Institutions are: Abramson Cancer Center at the University of Pennsylvania, Philadelphia, PA; Fred & Pamela Buffett Cancer Center, Omaha, NE; Case Comprehensive Cancer Center/University Hospitals Seidman Cancer Center and Cleveland Clinic Taussig Cancer Institute, Cleveland, OH; City of Hope National Medical Center, Duarte, CA; Dana-Farber/Brigham and Women's Cancer Center | Massachusetts General Hospital Cancer Center, Boston, MA; Duke Cancer Institute, Durham, NC; Fox Chase Cancer Center, Philadelphia, PA; Huntsman Cancer Institute at the University of Utah, Salt Lake City, UT; Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance, Seattle, WA; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD; Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL; Mayo Clinic Cancer Center, Phoenix/Scottsdale, AZ, Jacksonville, FL, and Rochester, MN; Memorial Sloan Kettering Cancer Center, New York, NY; Moffitt Cancer Center, Tampa, FL; The Ohio State University Comprehensive Cancer Center - James Cancer Hospital and Solove Research Institute, Columbus, OH; O'Neal Comprehensive Cancer Center at UAB, Birmingham, AL; Roswell Park Comprehensive Cancer Center, Buffalo, NY; Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine, St. Louis, MO; St. Jude Children's Research Hospital/The University of Tennessee Health Science Center, Memphis, TN; Stanford Cancer Institute, Stanford, CA; UC San Diego Moores Cancer Center, La Jolla, CA; UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, CA; University of Colorado Cancer Center, Aurora, CO; University of Michigan Rogel Cancer Center, Ann Arbor, MI; The University of Texas MD Anderson Cancer Center, Houston, TX; University of Wisconsin Carbone Cancer Center, Madison, WI; Vanderbilt-Ingram Cancer Center, Nashville, TN; and Yale Cancer Center/Smilow Cancer Hospital, New Haven, CT.

Clinicians, visit NCCN.org. Patients and caregivers, visit NCCN.org/patients. Media, visit NCCN.org/news. Follow NCCN on Twitter @NCCN, Facebook @NCCNorg, and Instagram @NCCNorg.

1 D'Souza A, Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides. 2018. Available at: https://www.cibmtr.org/ReferenceCenter/SlidesReports/SummarySlides/pages/index.aspx.

Media Contact: Rachel Darwin 267-622-6624 darwin@nccn.org

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New NCCN Guidelines Debut to Manage Complications and Improve Readiness for Stem Cell Transplant Recipients - Yahoo Finance

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