Stem Cell Clinic

Stem Cell Manufacturing in Stem Cell Therapy, Drug Discovery & Development, Stem Cell Banking, and More (2020-2025) – P&T Community

DUBLIN, Feb. 17, 2020 /PRNewswire/ -- The "Stem Cell Manufacturing Market - Growth, Trends, and Forecast (2020 - 2025)" report has been added to ResearchAndMarkets.com's offering.

The Stem Cell Manufacturing market is projected to grow with a CAGR of nearly 3.3% over the forecast period.

The major factors attributing to the growth of the market include the technological advancements in stem cell manufacturing and preservation and growing public awareness about the therapeutic potency of stem cell products.

According to California Institute for Regenerative Medicine, for the millions of people around the world who suffer from incurable diseases and injury, Stem Cell Awareness Day, October 11th is a day to celebrate the scientific advances made to-date. Research and academic institutions and educators are encouraged to participate by hosting public talks and other activities and events in their community. Furthermore, the growing growing public-private investments and funding in stem cell-based research is boosting the market growth. However, the gaining popularity of alternative procedures is the major drawback of market growth.

Stem Cell Banking Segment is the Fastest Growing Segment in the Stem Cell Manufacturing Market.

Stem cell bank is a provision that stores stem cells developed from amniotic fluid for future use. Stem cell samples in private banks are stored specifically for use by the individual person from whom such cells have been collected and the banking costs are paid the person. The sample can later be recovered only by that individual and for the use by such individual or, in many cases, by her or his first-degree blood relatives.

The major factor driving the growth of the segment is the increasing public demand for cord blood stem cell banking and rising awareness regarding the prospective advantages of stem cell preservation. Furthermore, stem cells have been proven to treat approximately 80 diseases and disorders including hematopoietic disorders, immunodeficiency diseases, metabolic disorders, etc. With the continued increase in per capita disposable revenue across developing countries and an expected decrease in product costs associated with stem cell therapies, increasing public awareness and the adoption of stem cell therapies, are the reasons driving the segment growth.

North America Dominates the Market and Expected to do Same in the Forecast Period

North America is expected to dominate the overall market, throughout the forecast period. The market growth is due to the factors such as the presence of key players, high concentration of stem cell research in the region, mounting public-private funding and grants to support the clinical evaluation of stem cells for various applications, robust research infrastructure, and raising public awareness on the therapeutic potency of stem cells. Furthermore, beneficial government initiatives and an increase in the number of research partnerships are some of the drivers expected to increase market growth.

Competitive Landscape

The Stem Cell Manufacturing market is moderately competitive and consists of several major players. Some of the companies which are currently dominating the market are Anterogen, Becton, Dickinson and Company, Cellular Dynamics (Fujifilm Holdings Corporation), Lonza Group, Stemcell Technologies, Medipost, Merck Group, Osiris Therapeutics, Pluristem Therapeutics, Bio-Rad Laboratories.

Key Topics Covered

1 INTRODUCTION1.1 Study Deliverables1.2 Study Assumptions1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS4.1 Market Overview4.2 Market Drivers4.2.1 Technological Advancements in Stem Cell Manufacturing and Preservation4.2.2 Growing Public Awareness About the Therapeutic Potency of Stem Cell Products4.2.3 Growing Public-Private Investments and Funding in Stem Cell-Based Research4.3 Market Restraints4.3.1 High Operational Costs Associated With Stem Cell Manufacturing and Banking4.4 Porter's Five Force Analysis

5 MARKET SEGMENTATION5.1 By Product5.1.1 Culture Media5.1.2 Consumables5.1.3 Instruments5.1.4 Stem Cell Lines5.2 By Application5.2.1 Stem Cell Therapy5.2.2 Drug Discovery and Development5.2.3 Stem Cell Banking5.2.4 Others5.3 By End-User5.3.1 Pharmaceutical and Biotechnology Companies5.3.2 Cell Banks and Tissue Banks5.3.3 Others5.4 Geography5.4.1 North America5.4.2 Europe5.4.3 Asia-Pacific5.4.4 Middle-East and Africa5.4.5 South America

6 COMPETITIVE LANDSCAPE6.1 Company Profiles6.1.1 Anterogen6.1.2 Becton, Dickinson and Company6.1.3 Cellular Dynamics (Fujifilm Holdings Corporation)6.1.4 Lonza Group6.1.5 Stemcell Technologies6.1.6 Medipost6.1.7 Merck Group6.1.8 Osiris Therapeutics6.1.9 Pluristem Therapeutics6.1.10 Bio-Rad Laboratories

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

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Stem Cell Manufacturing in Stem Cell Therapy, Drug Discovery & Development, Stem Cell Banking, and More (2020-2025) - P&T Community

Should California invest another $5.5 Billion into stem cell research? – Long Beach Business Journal – Long Beach News

Over the past 15 years, Californias stem cell program has funded over 1,000 research, training and community engagement projects focused on stem cell technology in the state.

But in October of last year, the agency in charge of administering the states stem cell program awarded its last round of new funds, allocating the last of a total of $3 billion in funding approved by California voters. Now, the author of Proposition 71, which established the program in 2004, is seeking another round of $5.5 billion in funding for stem cell research in a measure vying for a spot on the Nov. 5 ballot.

The scientists and patient advocates in California have proven through the California stem cell initiative funding that they can change the future of medicine and human suffering, the measures author, Robert Bob Klein II told the Business Journal. California funding has filled the gap of the federal governments failure to fund this revolution in medicine.

Kleins interest in stem cell research and regenerative medicine arose from his youngest sons ultimately fatal battle with diabetes. Diagnosed with Type I Diabetes at age 11, Kleins son Jordan required human insulin-producing beta cells, the artificial production of which to the best knowledge of the scientific community at this point requires stem cells.

In 2016, 26-year-old Jordan Klein died of complications related to the disease, two years after scientists first made significant progress on finding a treatment developed with the help of human embryonic stem cells.

Klein blames the federal governments resistance to embracing stem cell research for the lack of adequate treatment options that lead to his sons death. My youngest son died. If they hadnt held it up in D.C., he would be alive, he said. How many children, how many adults are going to die before they create enough stability to advance therapies that mitigate or cure these chronic diseases?

For years, Klein a wealthy real estate developer had tried to affect change on the federal level, before shifting his attention to his home state. I became focused on what California could do, because the federal government was encumbered by religious issues, he explained.

Despite having no experience in the field of scientific research, Klein was able to mount a successful campaign, with 59.1% of California voters approving the creation of a state-funded stem cell program and an agency to govern it. The California Institute of Regenerative Medicine held its first meeting in December 2004 and issued its first round of funding in 2006, after battling several challenges to the proposition in state and federal court.

Since then, CIRMs funding has enabled conferences, translational research projects and clinical trials exploring cures for various types of cancers, diabetes and neurological disorders. While a majority of funding went to the University of Californias main campuses as well as regenerative medicine companies and institutes in major hubs like San Diego and the Bay Area, California State University, Long Beach and Torrance-based Pathways to Stem Cell Science have also received funds from CIRM.

CIRM has created a new industry in California that has been tremendously beneficial for the California economy, Dr. Victoria Fox, president of Pathways, told the Business Journal. Her company, which offers stem cell extraction and education services, has received both direct and indirect funding from CIRM.

Last year, Pathways hosted the SPARK Annual Meeting, an event that highlights the scientific accomplishments of students in the SPARK high school summer training program funded through a CIRM grant. Foxs company also provides educational services through CIRMs Bridges program at CSULB, in which students from state and junior colleges compete for six-month lab positions at UC research labs.

[These programs] are important to us, not only because they generate income to operate, but because they generate a workforce, Fox explained.

She said other companies often approach her in search of stem cell scientists. If voters decided not to extend funding for CIRM and its program in November, Fox said shed be hard-pressed to find an equivalent network for talent acquisition. I dont know where Im going to refer companies to find talent. I really dont know.

Dr. Aaron Levine, an associate professor at the School of Public Policy at Georgia Tech, has focused primarily on the intersection between public policy and bioethics. In this context, Levine has followed and reviewed Californias stem cell program for years, and he agreed that its impact has been transformative.

CIRM stepped in to fill a gap when the National Institutes of Health was restricting its funding in this space, Levine said. The research that CIRM has supported, as well as the training programs, has had quite a big impact on the field.

But Levine also pointed to what he described as missed opportunities, as the program enters its bid for renewal. One example is the new proposals requirement to commit a certain percentage of funds to finding cures for specific diseases, such as Alzheimers and other neurological disorders.

From a science policy perspective, I dont really like that. I would like the funding agency to have the flexibility to fund the best science they see submitted, the most promising science, Levine said. Klein argued that setting aside a certain percentage of funds for research on neurological diseases will ensure that they wont be left behind in favor of less costly research projects.

Levine also noted that the program has yet to resolve one crucial question: Who will pay for patients treatment with costly stem cell therapies once theyre ready to hit the market? Per-patient costs for stem cell therapies can easily reach several hundred thousand dollars and as research advances, more patients are expected to qualify.

Suddenly, thats just such a substantial sum of money that it becomes a fundamental challenge to how we pay for healthcare, how we pay for medicine in the United States, Levine said. Subsidies for California residents, whose taxes helped pay for the research necessary to bring these cures and therapies to market, would be one option, Levine noted.

Despite these concerns, Levine said he supports the measure to extend the program. Even though this is not the perfect measure, I think theres a lot of value in CIRM and it makes sense to continue it, he said. In the end, it will be up to California voters to decide.

It largely will rise and fall on whether theres a motivated campaign for and against it and what people whove never really thought about stem cell research as a state ballot issue are going to think about this particular initiative when it comes in the fall, Levine said.

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Should California invest another $5.5 Billion into stem cell research? - Long Beach Business Journal - Long Beach News

Chinese Scientist Unite Together to Tackle With COVID-19 – Associated Press

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BEIJING, Feb. 18, 2020 /PRNewswire/ -- Reported by Science & Technology Daily:

As to the battle against the COVID-19, Chinese Ministry of Science and Technology (MOST) has been launched the program of Dealing With the Outbreak of COVID-19 Using Science and Technology, a program including four batches of 20 emergency projects until now. Moreover, MOST re-examining two major scientific and technological projects, including the Creation of Important Innovative New Drugs and the Prevention and Treatment of Severe Contagions, and a series of national essential research and development plans. Recently, some important achievements from these projects have already been implemented in the front line of epidemic prevention and control.

When it comes to drug screening, front-line staff from different research teams collaborated on the basis of existing researches, making great efforts to systematically and massively screen the drugs that have been on the market already or already go into clinical trials. As a consequence, they discovered potentially effective anti- coronavirus drugs, such as Chloroquine Phosphate, Remdesivir, and Favipiravir. Furthermore, in recent days, researchers have urgently launched some clinical trials and the results show that the curative effect of drugs on patients is increasingly obvious.

Based on the R&D level and preliminary accumulation in the field of cell therapy, traditional Chinese medicine and plasma therapy in China, the clinical trials of the three therapies organized by the Joint Prevention and Control Group have been gradually carried out, and they are playing significant role in curing critically ill patients.

For the purpose of providing more cutting-edge and effective solutions to the epidemic using stem cell therapy, the leaders of the MOST went to the Innovation Institute of Stem Cell and Regenerative Medicine of the Chinese Academy of Science to confirm their progressing. A new cellular drug called CAStem supposed to cure COVID-19 was created, and it has already made major progress in the experiment about the treatment of the severe acute respiratory distress syndrome (ARDS) previously. The research team has applied for emergency approval from China National Medical Products Administration, and it is cooperating with relevant medical institutions. So far, the CAStem has already been approved by the Ethics Committee, and is ongoing clinical observation and evaluation.

An emergency project titled Clinical Study on the Prevention and Treatment of COVID-19 by Integrated Chinese and Western Medicine was officially launched on February 3 with the support from the MOST. The positive curative effect has been shown in treating COVID-19, said Zhang Boli, the project leader and the headmaster of Tianjin University of Traditional Chinese Medicine. It was reported that a total of 23 patients had been cured and discharged from Hubei Provincial Hospital of Integrated Traditional and Western Medicine and Wuhan Hospital of Traditional Chinese Medicine.

With regard to the field of plasma therapy, China National Biotech Group has completed the collection of plasma from some convalescent patients, as well as the preparation of special immune plasma products and special immune globulin of novel coronavirus on February 13. Through strict blood biosafety testing, virus inactivation, and antiviral activity testing, etc., special immune plasma has been successfully prepared for clinical treatment and has already been put into clinical treatment of severely ill patients.

In terms of field of vaccine research and development, the Chinese Center for Disease Control and Prevention (CDC) had successfully isolated the first COVID-19 virus strain on January 24, which was recognized by the World Health Organization and other international agencies. Several new type of vaccines for COVID-19 have started the animal testing phase, which bring hope to all.

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Chinese Scientist Unite Together to Tackle With COVID-19 - Associated Press

Cell Therapy Market 2020: Growing with Technology Development, New Innovations, Future Projections, In-depth Analysis and Forecast by 2025 – News…

The analysts who wrote the report ranked the global Cell Therapy market by product, application, and region. All sectors were examined in detail, focusing on CAGR, market size, growth potential, market share and other important factors. The segment studies included in the report will help players focus on the lucrative areas of the global Cell Therapy market. Regional analysis will help players strengthen their base in the major regional markets. This shows the opportunities for unexplored growth in local markets and how capital can be used in the forecast period.

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The most commonly used process of cell therapy aims to use healthy cells from a donor (Allogeneic) which is compatible or autogenic that is from the patient itself along with their alteration to increase their therapeutic ability. There are various complex steps involved in the process like genetic screening of cell, cell harvesting and reinfusion into the patients body. All these steps are complex and important and have therapeutic result on the patient. These advanced usage of cell therapy will result in growth of the cell therapy market size during the forecast period.

Cell therapy market trends indicate growth owing to the various regulations being approved by the government in the desire to provide quick relief to the patients. Furthermore, many healthcare industries are working in collaboration with the government to identify the various processes to ways to improve cell therapy. Furthermore, the cell therapy market size is also influenced by the commercialization of stem cells treatments.

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The Stem cell therapy segment dominates the types of cell therapy and is said to have the maximum success rate. It has a special feature that it differentiates into any category of cell, at the same time ensuring the individual identity is intact. Industry experts state that the stem cell would revolutionize regenerative medicine, owing to its extensive use in treatment of fatal disease like neurodegenerative, cardiovascular and cancer. The growth of cell therapy market size is also factored to the increased research and development about the same. However, at the same time the huge cost involved in the various processes involved might be hinder the market growth.

The cell therapy market size is segmented on various categories like Clinical-use, Research and Therapy type and region. On the basis of region, North America is projected to contribute the maximum share to the market owing to increased development.

Key players in the market are JCR Pharmaceuticals Co., Ltd., Kolon TissueGene, Inc.; and Medipost and many more.

Segmentation:

The various segments of cell therapy market size are:

By Use & Type Outlook

By Cell Therapy Type

By Therapeutic Area

By Therapy Type

By Region

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Adroit Market Research provide quantified B2B research on numerous opportunistic markets, and offer customized research reports, consulting services, and syndicate research reports. We assist our clients to strategize business decisions and attain sustainable growth in their respective domain. Additionally, we support them with their revenue planning, marketing strategies, and assist them to make decisions before the competition so that they remain ahead of the curve.

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Cell Therapy Market 2020: Growing with Technology Development, New Innovations, Future Projections, In-depth Analysis and Forecast by 2025 - News...

Lineage Cell Therapeutics to Present New Data From OpRegen and Vision Restoration Programs at the Association for Research in Vision and Ophthalmology…

Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, announced today that updated results from a Phase I/IIa study of its lead product candidate, OpRegen, a retinal pigment epithelium (RPE) cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (dry AMD), have been accepted for presentation at the 2020 Association for Research in Vision and Ophthalmology (ARVO) Meeting, which will be held May 3rd through May 7th, 2020 at the Baltimore Convention Center in Baltimore, MD. The abstract presentation, entitled, "Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results", will be presented as part of the Gene Therapy and Stem cells Session on May 3rd, 2020 from 3:00PM to 4:45PM EDT by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute and University of Cincinnati School of Medicine; Clinical Governance Board, Cincinnati Eye Institute (presentation number 865). The presentation will provide updated data from patient cohorts 1 through 4 of the clinical study and will include data on the first patients dosed with both a new subretinal delivery system as well as with a new Thaw-and-Inject (TAI) formulation of OpRegen.

"We continue to be encouraged by positive data with OpRegen for the treatment of dry AMD," stated Brian M. Culley, CEO of Lineage. "The five patients treated as part of cohort 4, which more closely match our intended patient population, have all demonstrated an increase in the number of letters they can read on an Early Treatment Diabetic Retinopathy Scale (ETDRS), having gained between 10 25 letters. Importantly, the first patient treated using both a new subretinal delivery system and our TAI formulation of OpRegen demonstrated notable improvements in vision, having gained 25 readable letters (or 5 lines) 6 months following administration of OpRegen RPE cells, as assessed by the ETDRS. This represents an improvement in visual acuity from a baseline of 20/250 to 20/100 in the treated eye. These visual acuity measurements are meaningful and can translate into quality of life enhancements to things like reading, driving, or avoiding accidents. With the opening of two leading ophthalmology research centers as clinical sites for our study, we are focused on rapid enrollment so that our clinical update at ARVO can be as mature and informative as possible. Our objective is to combine the best cells, the best production process and the best delivery system, which we believe will position us as the front-runner in the race to address the unmet opportunity in the potential billion-dollar dry AMD market."

In addition, Lineage will present new preclinical results from its Vision Restoration Program, a proprietary program based on the ability to generate 3-dimensional human retinal tissue derived from pluripotent cells. Lineages 3-dimensional retinal tissue technology may address the unmet need of implementing a retinal tissue restoration strategy to address a wide range of severe retinal degenerative conditions including retinitis pigmentosa and advanced forms of AMD. In 2017 and 2019, the Small Business Innovation Research program of the National Institutes of Health awarded Lineage grants of close to $2.3 million to further develop this innovative, next generation vision restoration program.

- The poster presentation, entitled, "Transplantation of organoid-derived human retinal tissue in to the subretinal space of CrxRdy/+ cats)," will be presented as part of the Animal models for visual disease and restoration Session on May 4th, 2020 4:00PM to 5:45PM EDT in Session Number 291 by Igor Nasonkin, Ph.D., Principal Investigator, Director of Research & Development at Lineage (Poster board Number: 2253 - B0162).

- The poster presentation, entitled, " Intraocular biocompatibility of Hystem hydrogel for delivery of pharmaceutical agents and cells," will be presented as part of the Stem cells and organoids: Technical advances Session on May 5th, 2020 between 8:45AM to 10:30AM EDT in Session Number 332 by our collaborator Randolph D. Glickman, Ph.D., Professor of Ophthalmology, UT Health San Antonio (Poster board Number: # A0247).

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About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would," "contemplate," project," "target," "tend to," or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the potential applications in Lineages Vision Restoration Program. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading "Risk Factors" in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200218005395/en/

Contacts

Lineage Cell Therapeutics, Inc. IR Ioana C. Hone(ir@lineagecell.com) (510) 871-4188

Solebury Trout IR Gitanjali Jain Ogawa(Gogawa@troutgroup.com)(646) 378-2949

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Lineage Cell Therapeutics to Present New Data From OpRegen and Vision Restoration Programs at the Association for Research in Vision and Ophthalmology...

bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress – Yahoo Finance

- First conditional approval of ZYNTEGLOTM (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent -thalassemia who do not have 0/0 genotype in Europe achieved in 2019; Germany launch underway

- Announced positive top-line data from pivotal Phase 2 KarMMa study of ide-cel in relapsed and refractory multiple myeloma

- Presented clinical data across studies of LentiGlobin gene therapy for -thalassemia (betibeglogene autotemcel) and LentiGlobin gene therapy for sickle cell disease (SCD) and bb21217 in multiple myeloma at American Society of Hematology (ASH) Annual Meeting

- Ended quarter with $1.24 billion in cash, cash equivalents and marketable securities

bluebird bio, Inc. (NASDAQ: BLUE) today reported financial results and business highlights for the fourth quarter and full year ended December 31, 2019.

"2019 was truly a transformative year for bluebird, with our first commercial product now launched in Europe and exciting progress across our first four clinical programs and pipeline," said Nick Leschly, chief bluebird. "Notably, our data in SCD continues to build, and at the ASH annual meeting in December we presented data that showed a 99% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome (ACS) in HGB-206 Group C patients with history of VOCs and ACS who had at least six months follow-up. In -thalassemia, the consistency with which patients who do not have a 0/0 genotype in our Northstar-2 (HGB-207) study are achieving transfusion independence is very encouraging and were starting to see indications that we may be able to see similar outcomes with many patients with 0/0 genotypes as well in our Northstar-3 (HGB-212 study). These data put us in a strong position as we progress our European launch, currently underway in Germany. At the end of 2019, we also announced positive top-line data from the pivotal KarMMa study of ide-cel. We and our partners at BMS look forward to submitting these data to the FDA in the first half of this year. Amidst all of our progress in 2019, our birds demonstrated time and again their dedication to patients and ability to meet and learn from the many challenges we have faced along the way. I look forward to facing the challenges of 2020 with this amazing flock."

Recent Highlights:

TRANSFUSION-DEPENDENT -THALASSEMIA

SICKLE CELL DISEASE (SCD)

MULTIPLE MYELOMA

COMPANY

Upcoming Anticipated Milestones:

Fourth Quarter and Full Year 2019 Financial Results

LentiGlobin for -thalassemia Safety

Non-serious adverse events (AEs) observed during the HGB-204, HGB-207 and HGB-212 clinical studies that were attributed to LentiGlobin for -thalassemia were hot flush, dyspnoea, abdominal pain, pain in extremities, thrombocytopenia, leukopenia, neutropenia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for -thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

With more than five years of follow-up to date, there have been no new unexpected safety events, no deaths, no graft failure and no cases of vector-mediated replication competent lentivirus or clonal dominance. In addition, there have been no new reports of veno-occlusive liver disease (VOD) as of the data cutoff presented at ASH.

About LentiGlobin for -Thalassemia (betibeglogene autotemcel)

The European Commission granted conditional marketing authorization for LentiGlobin for -thalassemia, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy, for patients 12 years and older with 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.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

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LentiGlobin for -thalassemia adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted LentiGlobin for -thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

bluebird bio has initiated its rolling BLA submission of LentiGlobin for -thalassemia for approval in the U.S. and is engaged with the FDA in discussions regarding the requirements and timing of certain information to be provided in the BLA, including information regarding various release assays for LentiGlobin for -thalassemia. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the second half of 2020.

LentiGlobin for -thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for -thalassemia. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

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 including cerebral adrenoleukodystrophy, sickle cell disease, -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 bluebird bio 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, the execution of the companys commercial launch plans, 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; the risk 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 regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; 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, in the adoption of value-based payment models, or in obtaining sufficient coverage or reimbursement for our products; the risk that our collaborations, including the collaborations with Bristol-Myers Squibb and Forty Seven, 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-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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

For the three months endedDecember 31,

For the year endedDecember 31,

2019

2018

2019

2018

Revenue:

Collaboration revenue

$ 7,159

$ 18,382

$ 36,469

$ 52,353

License and royalty revenue

2,838

861

8,205

2,226

Total revenues

9,997

19,243

44,674

54,579

Operating expenses:

Research and development

161,821

119,722

582,413

448,589

Selling, general and administrative

76,202

53,508

271,362

174,129

Cost of license and royalty revenue

1,073

818

2,978

885

Change in fair value of contingent consideration

1,435

2,156

2,747

2,999

Total operating expenses

240,531

176,204

859,500

626,602

Loss from operations

(230,534)

(156,961)

(814,826)

(572,023)

Interest income, net

6,855

6,209

34,761

14,624

Other (expense) income, net

535

1,916

(10,088)

1,961

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bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress - Yahoo Finance

UCLA researchers discover new compound that promotes lung health – UCLA Newsroom

Sarah C.P. Williams | February 18, 2020

A molecule identified by UCLA researchers helps maintain a healthy balance of cells in airway and lung tissue. If the compound, so far only studied in isolated human and mouse cells, has the same effect in people, it may lead to new drugs to treat or prevent lung cancer.

We think this could help us develop a new therapy that promotes airway health, said Dr. Brigitte Gomperts, a UCLA professor of pediatrics and of pulmonary medicine, a member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and lead author of the study. This could not only inform the treatment of lung cancer, but help prevent its progression in the first place.

Humans respiratory systems are constantly being injured by pollution and germs in the air we breathe and must be replenished with healthy cells. That process is driven by airway basal stem cells, which divide to produce both more stem cells and the mucociliary cells that line the airways and lungs.

There are two types of mucociliary cells: mucus cells, which produce the mucus that trap toxic and infectious particles, and ciliated cells, which have finger-like projections that sweep the mucus away to keep the respiratory system healthy and clear. In healthy lungs, airway basal stem cells stay balanced between producing mucociliary cells and self-renewing to maintain a population of stem cells.

In precancerous cells in the lungs, basal stem cells divide more often than usual, generating a large number of stem cells but too few mucociliary cells. The resulting imbalance of cells in the airway leaves the airways unable to properly clear debris, and it creates a greater risk that the precancerous cells will give rise to a tumor.

In the new study, published today in Cell Reports, Gomperts and her colleagues analyzed airway cells from equal numbers of biopsies of healthy people, people with premalignant lung cancer lesions and people with squamous lung cancer. They discovered that one group of molecules collectively called the Wnt/beta-catenin signaling pathway was present at different levels in the basal stem cells of the patient samples versus the cells from healthy people.

And when the researchers altered the levels of these molecules in healthy airway cells from mice, the balance between stem cells and mucociliary cells shifted, mimicking the imbalance seen in lung pre-cancers.

When you activate the Wnt/beta-catenin signaling pathway, these stem cells just divide and divide, said Gomperts, who is also a member of the UCLA Jonsson Comprehensive Cancer Center.

Finally, the team screened more than 20,000 chemical compounds for their ability to reverse this effect in human cells, lowering levels of Wnt and restoring the balance of stem cells and mature airway cells.

One compound stood out for its ability to limit the proliferation of basal stem cells and restore the balance of the stem cells and mucociliary cells to normal. The compound was also less toxic to airway cells than other, previously discovered, molecules that block Wnt/beta-catenin signaling. The team named the compound Wnt Inhibitor Compound 1, or WIC1.

The identification of this new drug is a nice tool to tease apart the biology of the Wnt/beta-catenin signaling pathway and its effects on lung health, said Cody Aros, the first author of the new paper and a UCLA graduate student. Its also very exciting that it may act in a new way than other existing Wnt/beta-catenin signaling pathway inhibitors and has such low toxicity.

Since WIC1 was identified through a random drug screen, the researchers dont yet know exactly how it works, but theyre planning future studies on its mechanism and safety.

The compound tested by the researchers was used in preclinical tests only and has not been tested in humans or approved by the Food and Drug Administration as safe and effective for use in humans.

The newly identified compound is covered by a patent application filed by the UCLA Technology Development Group on behalf of the Regents of the University of California, with Gomperts and Aros as co-inventors.

Funding for the study was provided in part by the National Institutes of Health, the National Cancer Institute, the Tobacco Related Disease Research Program and the Broad Stem Cell Research Center Training Program, including support from the Rose Hills Foundation Graduate Scholarship.

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UCLA researchers discover new compound that promotes lung health - UCLA Newsroom

Could this patch help mend a broken heart? – Medical Design & Outsourcing

(Image from Trinity College Dublin)

Researchers in Ireland have developed a prototype patch that they say does the same job as crucial aspects of heart tissue.

The patch was designed to withstand the mechanical demands of heart tissue and mimic the electrical signaling properties that allow the heart to pump blood throughout the body. The researchers believe it brings medtech one step closer to a functional design that could mend a broken heart.

Cardiac patches lined with heart cells can be applied surgically to restore heart tissue in patients who have had damaged tissue removed after a heart attack and to repair congenital heart defects in infants and children. Ultimately, though, the goal is to create cell-free patches that can restore the synchronous beating of the heart cells, without impairing the heart muscle movement. The bioengineers report their work in the journal Advanced Functional Materials.

Researchers are continuously looking to develop new treatments which can include stem cell treatments, biomaterial gel injections and assistive devices, said senior author Michael Monaghan, an assistant professor at Trinity College Dublin, in a news release. Ours is one of few studies that looks at a traditional material, and through effective design allows us to mimic the direction-dependent mechanical movement of the heart, which can be sustained repeatably. This was achieved through a novel method called melt electrowriting and through close collaboration with the suppliers located nationally we were able to customize the process to fit our design needs.

This work was performed in the Trinity Centre for Biomedical Engineering, based in the Trinity Biomedical Sciences Institute in collaboration with Spraybase, a subsidiary of Avectas Ltd.

The mechanical demands of heart muscle cannot be met using polyester-based thermoplastic polymers, which are predominantly the approved options for biomedical applications, according to the researchers. However, the functionality of thermoplastic polymers could be leveraged by its structural geometry. They made a patch that could control the expansion of a material in multiple directions and tune this using an engineering design approach.

The patches were manufactured via melt electrowriting, a core technology of Spraybase, which the company says is reproducible, accurate and scalable. The patches were also coated with the polymer polypyrrole to provide electrical conductivity while maintaining cell compatibility. The patch withstood repeated stretching, which is a dominant concern for cardiac biomaterials, and showed good elasticity, to accurately mimic that key property of heart muscle.

Essentially, our material addresses a lot of requirements, Monaghan said. The bulk material is currently approved for medical device use, the design accommodates the movement of the pumping heart, and has been functionalized to accommodate signaling between isolated contractile tissues. This study currently reports the development of our method and design, but we are now looking forward to furthering the next generation of designs and materials with the eventual aim of applying this patch as a therapy for a heart attack.

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Could this patch help mend a broken heart? - Medical Design & Outsourcing