Category Archives: Cell Therapy

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced one oral presentation and four poster presentations were accepted for the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 22-25, 2019, in Barcelona, Spain.

Intellias data includes important updates about the companys programs and platform development activities:

Oral Presentation:

In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression

Intellia will present data on its alpha-1 antitrypsin deficiency (AATD) program, which uses a modular hybrid delivery system combining lipid nanoparticle (LNP) encapsulated CRISPR/Cas9 with an adeno-associated virus (AAV) donor DNA template. Intellias gene knockout approach eliminates the production of the faulty PiZ variant of the protein, while targeted insertion of a wild-type gene copy facilitates production of a functional circulating protein. This builds on Intellias similar approach for targeted gene insertion of Factor 9, which achieved increased levels of circulating human Factor IX protein through two months in non-human primates and sustained through 12 months in mice.

Presenter: Anthony Forget, Ph.D.Abstract number: OR48Session 5b: New delivery systems and technologiesPresentation date/time: Friday, October 25, 2019, 11:30 a.m. 1:30 p.m. CETLocation: Room 113-115

Poster Presentations:

In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics

This poster presentation will highlight Intellias approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Daniel OConnell, Ph.D.Poster ID Number: P655Date: Wednesday, October 23, 2019

Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia

This poster presentation focuses on Intellias ongoing research collaboration with IRCCS Ospedale San Raffaele to develop CRISPR/Cas9-edited T cell therapies to address intractable cancers, such as acute myeloid leukemia (AML). Researchers have successfully established a protocol enabling consistent and efficient tumor-specific TCR isolation and characterization from healthy donors. Based on these results, Intellia has selected multiple lead TCRs, which are undergoing development candidate evaluation.

Presenter: Erica Carnevale, Ph.D., Ospedale San RaffaelePoster ID Number: P111Date: Wednesday, October 23, 2019

Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout

This poster presentation focuses on the companys T cell engineering technology, which is being applied in its Wilms Tumor 1 (WT1) lead ex vivo program. Intellia has identified an efficient CRISPR/Cas9-mediated process that inserts tumor-specific TCRs with high yield into the TRAC locus. Simultaneous knockout of the TRBC1 and TRBC2 loci substantially eliminates production of the endogenous T cell receptors.

Presenter: Birgit Schultes, Ph.D.Poster ID Number: P162Date: Thursday, October 24, 2019

CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria

This poster presentation will demonstrate the effects of independent CRISPR/Cas9-mediated knockout of each of two target genes involved in oxalate formation, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria type 1 (PH1).

Presenter: Sean Burns, M.D.Poster ID Number: P552Date: Thursday, October 24, 2019

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.comand follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms; develop our proprietary LNP/AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half of 2021.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position, including through our arbitration proceedings against Caribou; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations withNovartisor Regeneron or its otherex vivocollaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, andIntellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Announces Presentations at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) - BioSpace

Results of an ex vivo study evaluating the phenotypic and growth characteristics of T cells collected by leukapheresis from cohorts of patients with newly diagnosed or relapsed/refractory multiple myeloma support use of chimeric antigen receptor (CAR)-T therapy earlier in course of the disease. The hypothesis-generating findings from this study were published in Blood Advances.

While CAR-T therapy targeted against the B-cellmaturation antigen (BCMA) has been associated with promising results inpatients with multiple myeloma, nearly all of the patients responding to thisapproach eventually develop progressive disease. Hence, strategies to optimizepatient selection for CAR-T therapy in the setting of multiple myeloma arebeing actively pursued.

Theratio of CD4 to CD8 T cells and/or the frequency of the CD81 CD45RO2 CD271 T-cell memory phenotype were usedin this study as surrogates for the clinical effectiveness of CAR-T therapysince previous studies ofCAR-T therapy in patients with chronic lymphocyticleukemia and multiple myeloma showed that of all baseline patient- anddisease-related characteristics considered, clinical response to CAR-T therapywas associated only with this T-cell ratio and/or the frequency of this subsetof memory T cells in the premanufacturing leukapheresis product.

Twocohorts of patients where compared in this study: 38 patients with newly diagnosedmultiple myeloma who had participated in clinical trials of induction therapy andon whom leukapheresis was performed before consolidation therapy and autologousstem cell transplantation (ASCT); and 25 patients with relapsed/refractorymultiple myeloma enrolled in a phase 1 clinical trial of anti-BCMA CAR-Ttherapy and on whom leukapheresis was performed during a washout period shortlyfollowing study enrollment.

Inboth patient cohorts, leukapheresis samples were exposed ex vivo to anti-CD3and anti-CD28 monoclonal antibodies covalently linked to magnetic beads toprovide stimulatory/costimulatory signals for T-cell proliferation and theexpansion of functional T cells.

The 2 patient cohorts were similar with respect to median age (ie, 55 years; 58 years [relapsed/refractory]), although the time from multiple myeloma diagnosis was 222 days for those treated with induction therapy and 4.6 years for those with relapsed/refractory disease.

Inaddition, differences in the median number of prior lines of therapy (1 vs 7),and bone marrow cellularity occupied by myeloma plasma cells (13% vs 65%) wereobserved when the former and latter cohorts were compared at the time thatleukapheresis was performed.

Akey finding from this study was a significantly higher frequency of T cellswith the CD81 CD45RO2CD271 T-cell memory phenotype(43.9% vs 29.0%; P =.001), as well asa significantly higher median CD4/CD8 ratio (2.6 vs 0.87; P <.0001) in the postinduction versus the relapsed/refractorypatient cohort.

Inaddition, the CD4/CD8 ratio was also significantly higher in the postinductioncohort compared with responders to anti-BCMA CAR-T therapy from the relapsed/refractorycohort (2.6 vs 1.3; P= .0009); however,while higher in the postinduction cohort, the difference in the frequency of Tcells with the CD81 CD45RO2CD271 T-cell memory phenotypewas not statistically significant when these 2 groups were compared.

Regardingcapacity for ex vivo proliferation during manufacturing, significantly highernumbers of population doubling by day 9 (PD9) were observed for thepostinduction cohort compared with either the overall relapsed/refractorycohort or the group of responders within the relapsed/refractory cohort.

Ourresults suggest that CAR T cells manufactured from leukapheresis samplesobtained after response to induction therapy would be, on average, moreclinically effective than those obtained from heavily relapsed/refractorymultiple myeloma patients, the study authors concluded.

Reference

Garfall AL, Dancy EK, Cohen AD, et al. T-cell phenotypes associated with effective CAR T-cell therapy in postinduction vs relapsed multiple myeloma. Blood Adv. 2019;3:2812-2815.

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CAR-T Therapy May Be More Effective When Administered Earlier in the Multiple Myeloma Treatment Continuum - Cancer Therapy Advisor

CAMBRIDGE, Mass. Sanofi is accelerating nascent efforts in gene therapy, aiming to use its expertise in vaccines to catch up in a competitive field that's well ahead of the French pharma.

The company has prioritized gene therapy programs amid a broader effort to boost internal R&D speed and impact, said John Reed, Sanofi's head of research and development, in a Wednesday interview at Sanofi's Cambridge office.

"When I joined, I saw that we were dabbling in gene therapy and decided that we need to get more serious about gene therapy if we are going to continue to be impactful in that space," said Reed, who came over to Sanofi from Roche last July.

In particular, the company is retrofitting one of its vaccine facilities near Lyon, France, to produce GMP-grade adeno-associated viral vectors, or AAVs. Reed said he expects the plant to be operational in about a year.

The new R&D chief is steering the company away from areas for which it's historically been known, including, most notably, cardiovascular disease and diabetes. Sanofi is largely exiting cardiovascular R&D and is cutting spending in half on diabetes R&D, Reed said.

While vaccines make up a comparatively smaller portion of Sanofi's revenues, Reed noted the company's decades-long expertise in producing inactivated viruses could translate well to gene therapy. Reed was recently in Lyon to discuss the budget and headcount requirements for the change, he said.

"We have an opportunity to really leverage those competencies around vaccines for the gene therapy area," Reed said. "We are looking at how we can use that as a competitive advantage to be players in that space."

Several of Sanofi's pharma peers have bet heavily on gene therapy, investing in manufacturing and snapping up biotech leaders through multi-billion dollar acquisitions, such as by Novartis for AveXis and Roche for Spark Therapeutics.

Smaller companies like BioMarin Pharmaceutical, meanwhile, hold sizable leads in therapeutic areas that Sanofi hopes to play a larger role in, like hemophilia.

Reed acknowledged an acquisition "could be an accelerator" in establishing Sanofi's presence in cell and gene therapy.

"We flirt with those things all the time," he said, when asked about his openness to a deal like those for AveXis and Spark. "It's a bit challenging to point your finger at any one gene therapy company and say that solves all our problems."

"It's been really tough to pull the trigger on something like that," he added. "In the interim, we've been establishing the capabilities more internally."

How much it would be willing to pay, or afford, is another question. Under former CEO Olivier Brandicourt, the company last year targeted roughly 20 billion euros in acquisitions, a budget largely consumed by deals for Bioverativ and Ablynx in the blood disease space.

The company's first AAV-delivered gene therapy recently entered the clinic for a form of a rare eye disease called Leber congenital amaurosis, Reed added.

Two gene-edited cell therapies are in Phase 1/2 testing via a collaboration with Sangamo Therapeutics. Other programs remain preclinical as the group works on establishing GMP manufacturing capabilities.

All of this is taking place against a backdrop of change for Sanofi research and development teams.

Reed is working to narrow the company's focus to advance only first- or potentially best-in-class therapies, a bar that led Sanofi to cut several dozen programs from its pipeline earlier this year.

Reed has also restructured employee's incentives, taking away bonuses for starting projects and replacing them with an emphasis on starting first-in-human studies, a milestone Sanofi usually reaches slower than industry leaders.

"I don't want to reward people for starting projects, I want to reward them for finishing projects," he said. "We have too many projects."

Part of that's involved reducing bureaucracy and streamlining decision-making, moving from 33 committees that interact with R&D teams to three. Reed's given decision-making authority to team leaders for each molecule, calling them CEOs of their drug candidate.

Even before Reed came on board, productivity had begun to improve from a nadir in 2014, when Sanofi's entire organization produced only two clinical candidates that year. Now, Sanofi is delivering about six per year and, with the 2018 acquisitions of Bioverativ and Ablynx, should reach eight or nine per year.

Still, of the last 10 drugs Sanofi has won approvals for, only one was an internal project, Reed said. For the company's next 10 assets, Reed expects six or seven to have been internally developed.

As Reed re-focuses, Sanofi has exited or restructured partnerships this year with Regeneron, Alnylam Pharmaceuticals and Lexicon Pharmaceuticals.

Paring down the pipeline and restructuring deals also speaks to Sanofi's R&D budget, which the company expects to keep flat for the next few years. The pharma spends about half what companies with larger revenues like Pfizer, Novartis and Roche do.

Reed says the ultimate goal is to bring about 12 programs into clinical development each year, and growing internal R&D to the point where it's responsible for the majority of those candidates progressing.

"With the resources we have, that would be industry competitive," he added.

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Sanofi investing in gene therapy as R&D focus turns toward rare disease - BioPharma Dive

LOUISVILLE, Ky. Cancer patients in Louisville and throughout the region soon will have access to some of the most advanced immunotherapy treatments available.

Louisville resident Thomas E. Dunbar has pledged $1 million to the University of Louisville to create a specialized center to provide chimeric antigen receptor positive T (CAR T) cell therapies to patients at the U of L James Graham Brown Cancer Center and other centers in the Midwest.

The new program will be named the Dunbar CAR T-Cell Program.

This gift will allow both kids and adults to be treated right here in Kentucky with the most innovative cell-based immunotherapy being developed, said Jason Chesney, M.D., Ph.D., director of the U of L Brown Cancer Center.

In CAR T-cell therapies, immune cells are extracted from the patients own blood and then are genetically modified to fight cancer. The modified cells are infused back into the patient where they fight the cancer and create long-term immunity to its recurrence.

In addition to dramatic treatment results, CAR T-cell immunotherapy leads to fewer toxic side effects than traditional chemotherapy.

Patients who have been treated with all the conventional therapies who then underwent treatment in clinical trials with CAR T cells had dramatic response rates. Eighty-three percent of kids in the original trial who had lethal, terminal B-cell acute lymphoblastic leukemia responded to this therapy, Chesney said.

The Dunbar CAR T-Cell Program will include laboratories for manufacturing the CAR T cells and will administer both FDA-approved and clinical-trial therapies to adult and pediatric cancer patients.

The goal is for the facilities to be fully functional and receiving patients by Sept. 30, 2020.

Tom Dunbars son, Evan, lost his battle to cancer with neuroblastoma in 2001 at the age of 6. In 2009, Wally Dunbar, Tom Dunbars father, lost his battle with melanoma.

Donor Tom Dunbar with his son, Evan

U of L Brown Cancer Center

This year, Toms physician wife, Stephanie Altobellis, M.D., helped identify his own cancer.

Kentucky is at ground zero, with the nations highest rates of cancer diagnosis and death, Tom Dunbar said. Its completely unacceptable. We have to lead the charge right here where the need is the greatest and we can do the most good. We need treatments that are not toxic. Watching our loved ones miserable with pain, often just from the treatments, and yet still die in front of us simply cant be the best that we can do.

To learn more about how CAR T-cell treatment works visit: uoflbrowncancercenter.org

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Dunbar CAR T-Cell Program brings advanced immunotherapy to cancer patients - WHAS11.com

The agreement allows the company to integrate Accellixs proprietary sampling and analysis technology into its Point-of-Care platform

Inc (), a cell and gene therapy company, has signed a co-development agreement with cell analytics company Accellix Inc.

The agreement allows Orgenesis to integrate Accellixs proprietary sampling and analysis technology its optic system, cartridges, reagents and software into the companys Point-of-Care (POCare) cellular therapy platform.

POCare works by collecting, processing and supplying cells for various therapeutic treatments. The idea is to shrink the cost of cell and gene therapy through automation. By integrating Accellixs technology, Orgenesis can greatly improve its quality control.

By partnering with Accellix and integrating its advanced optic technologies, cartridges and software, we believe that this will advance Orgenesis processing capabilities for different types of cells for our POCare services and platform, Orgenesis CEO Vered Caplan said.

We believe the Orgenesis POCare platform can deliver a transformative option to the cell and gene therapy market that will lower costs in order to potentially bring our autologous therapies to patients in a cost effective, high quality and scalable manner.

Orgenesis is a biotechnology company based in Germantown, Maryland. In addition to POCare, it also owns a subsidiary, Masthercell Global, which operates a Contract Development and Manufacturing Organization platform, which provides manufacturing and development services for biopharmaceutical companies.

Contact Andrew Kessel at [emailprotected]

Follow him on Twitter @andrew_kessel

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Orgenesis inks co-development agreement with Accellix to improve quality control in its cellular therapy system - Proactive Investors USA & Canada

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- LogicBio Therapeutics Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients, today announced upcoming presentations at the European Society of Gene and Cell Therapy (ESGCT) 27th Annual Congress, held in Barcelona, Spain, October 22-25, 2019.

We are thrilled to be presenting positive data on our Next Generation Capsid Development Program on the anniversary of our collaboration with Childrens Medical Research Institute of Australia, a leader in gene therapy, childhood cancer, embryology and neurological diseases. The goal of the collaboration is to develop novel, synthetic adeno-associated virus (AAV) capsids which are highly tropic for human tissues and optimized for manufacturing. These data give us further confidence that we can improve the performance of current AAV vectors, expanding our pipeline and strengthening our GeneRide platform, said Fred Chereau, CEO of LogicBio. Further, we are pleased to present additional preclinical data further supporting the durability of expression, compared to canonical gene therapy, in one of our GeneRide platform programs and to have been invited to speak on AAV manufacturing.

Panel PresentationTitle: AAV manufacturing: critical parameters influencing vector quality attributesPresenter: Matthias Hebben, Ph.D., VP, Technology Development, LogicBio Therapeutics (INV36)Session: 1d ATMP manufacturingSession date/time: October 23, 2019, 8:30-10:30 a.m. CEST

Poster PresentationsTitle: AAV development program: towards next generation of livertropic AAV variants (P025)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Title: Durability of factor IX expression in mice treated neonatally with a nuclease-free, promoterless, AAV-based gene therapy, GeneRide (P423)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Additional information on the meeting can be found on the ESGCT website: https://www.esgct.eu/home.aspx

About LogicBio TherapeuticsLogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.

For more information, please visit http://www.logicbio.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on April 1, 2019 with the SEC, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts

Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Stephanie SimonTen Bridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics to Present New Data on Next Generation Capsid Development Program and GeneRide Platform Program at the European Society of Gene...

Cell Therapy Market report provides the market potential for each geographical region based on the growth rate, macroeconomic parameters, consumer buying patterns, and market demand and supply scenarios. This analysis gives an examination of various segments that are relied upon to witness the quickest development amid the estimate forecast frame. This report categorizes the market based on manufacturers, regions, type and application. This study also analyzes the market status, market share, growth rate, future trends, market drivers, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

Cell Therapy Market Figures is now released by Data Bridge Market Research. Cell Therapy Market report presents a complete assessment of covering future trend, Latest Trend, current growth factors, facts, and industry validated market data forecast till 2026. The report provides an in-depth analysis of the latest trends, business scenario, size and share of Major Competitors such as CELGENE CORPORATION, Bone Therapeutics, Cell Therapies, Celyad, Regen BioPharma, Cellular Therapeutics Ltd, TxCell, and others

Global cell therapy market is expected to rise from its initial estimated value of USD 5.52 billion in 2018 to an estimated value of USD 8.70 billion by 2026, registering a CAGR of 5.85% in the forecast period of 2019-2026. This rise in market value can be attributed to the increasing investments and funding from government and private institutions.

Competitive Landscape:

Global cell therapy market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of cell therapy market for global, Europe, North America, Asia Pacific, South America and Middle East & Africa.

Market Drivers and Restraints:

Few of the major competitors currently working in the cell therapy market are Vericel, Kolon TissueGene Inc., JCR Pharmaceuticals Co. Ltd., MEDIPOST, Osiris, Stemedica Cell Technologies Inc., NuVasive Inc., Fibrocell Science Inc., Cellectis, BioNTech IMFS, pluristem, Grupo Praxis, Genzyme Corporation, Advanced Tissue, Cells for Cells, PHARMICELL Co. Ltd, ANTEROGEN.CO.LTD., Novartis AG, GlaxoSmithKline plc,and others

Segmentation:

Get Detailed TOC with Tables and Figures at https://www.databridgemarketresearch.com/toc/?dbmr=global-cell-therapy-market

Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analysed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report.

Potential Held by the Report:

About Data Bridge Market Research

An absolute way to forecast what future holds is to comprehend the trend today!

Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

Data Bridge is an aftermath of sheer wisdom and experience which was formulated and framed in the year 2015 in Pune. We ponder into the heterogeneous markets in accord with our clients needs and scoop out the best possible solutions and detailed information about the market trends. Data Bridge delve into the markets across Asia, North America, South America, Africa to name few.

Data Bridge adepts in creating satisfied clients who reckon upon our services and rely on our hard work with certitude. We are content with our glorious 99.9 % client satisfying rate.

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Cell Therapy Market Size Overview by Rising Demands, Trends and Huge Bussiness Opportunities 2019 to 2026 - Herald Correspondent

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When the scene unfoldsYoung girls thirteen years oldExpose themselves to any Tom, Dick, and HankGot mo' stretch marks than these hoesHollin they got rankSee Sega ain't in this new world order Dem experimenting in Atlanta, GeorgiaUnited Nations, overseasthey trained assassins to do search and seizeAin't knocking or askingDem coming for niggas like mePo' white trash, like theyTricks like her back in slaveryConcentration camps lace with gas pipes linesInferno's outdoors like they had backWhen Adolf Hitler was living in 1945Listen to me now, believe meLater on in the future look it upWhere they say it? Aint no more ConstitutionIn the event of a race warPlaces like operation heartbreak hotelMoments tear until air tight vents seat off despairDem say expect no mercyFoot you should be my least worries got to deal withWhere my W-2's, 1099'sUnmarked black helicopters swoop downAnd try to put missiles in mines

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Link:
Goodie Mob - Cell Therapy (Official Video)

In recent years, stem cells have often been portrayed by the news & media as a new wonder therapy that could regenerate virtually any damaged organs and tissues in the human body. But are stem cells really a new discovery and what is their impact in medicine?

Contrary to popular belief, its been understood for millennia that different organs have regenerative potential. The Greek poet, Hesiod, recognised this ability in the liver over 2800 years ago when he wrote about the legend of Prometheus. Such observations prove true in our modern era when the liver can regenerate even after 70% of it is has been surgically removed. This process is now attributed to the presence of stem cells in this organ. However, it wasnt until 1868 that the term stem cell was first used when the German scientist, Ernst Haechel, was studying evolution in multiple species including crustaceans. In his native language, he called it the stammzelle (stem cell in English) and he recognised the unique capability of these cells: namely, to replicate and make more stem cells whilst also being able to produce differentiated cell types, such as heart, liver and blood cells.

Such stem cells found in organs of the adult body are known as adult stem cells. Perhaps the most well studied of the adult stem cells are blood (or haematopoietic) stem cells. In the 1960s it was recognised that a blood transfusion from a healthy donor could restore the wellbeing of someone exposed to harmful levels of radiation. Many of these findings were precipitated following the horrific radiation injuries in civilian populations after atomic bombs were dropped on in Hiroshima and Nagasaki. Now blood transfusion is commonly-used to help the recovery of patients undergoing treatment for cancer. This is because the chemo- and radio-therapy used damages their own blood stem cells, which are replaced by an infusion of healthy stem cells from a suitable donor. Indeed, pioneering work in the field of blood transfusion led to Peter Medwar being awarded the Nobel Prize in Medicine in 1960. Other adult stem cells being investigated as therapies include those in skin (for treatment of burns), cornea (for repairing damage to the eye) and brain (for injuries such as stroke).

In parallel to the discoveries being made in adult stem cell research, work was starting with a different class of stem cells, known as the embryonic stem cells. During the 1960s and 70s, scientists found that testicular cancers formed bizarre tumour masses that didnt just contain testicular material but also had hair, bone, neurons and so on. The cause of these strange tumours was identified as embryonal carcinoma cells, which are stem cells that have become mutated. A few years later in 1981, better behaved embryonic stem cells were isolated from mouse embryos. These remarkable cells could be grown in the lab for long periods of time and yet be coaxed into become virtually every cell type in the mouse (about 200 different cell types). Indeed, when implanted into early stage mouse embryos, these embryonic stem cells can contribute to every tissue of the adult mouse. The embryonic stem cells could also be genetically modified and used to make new strains of mice with specifically-engineered changes in their genome. This finding revolutionised our understanding of genetics and disease, which ultimately led to the Novel Prize in Medicine being jointly awarded to Mario Capecchi, Martin Evans and Oliver Smithies in 2007.

It took another 17 years before human embryonic stem cells (hESC) were isolated in 1998, largely because different conditions were needed to grow these cells relative to their mouse counterparts and the ethics of working with human embryos. Close regulation by government groups such as the Human Fertilisation & Embryology Authority (HFEA) make sure all work is carried out in an ethical manner. Thus, hESCs can only be produced from spare embryos donated by consenting couples undergoing in vitro fertilisation (IVF, or test-tube baby) treatment. The hESCs are often considered as master or pluripotent stem cells because they make many if not all of the cell types in the human body. Unlike in the mouse, the intention with hESCs is not to make new humans but rather to use their power to make replacement cells for sick patients. This is slow and painstaking work but the first clinical trials have begun to repair macular degeneration (eye disease) by the US company Advance Cell Technologies (ACT) with initial results expected from just a few patients in 2014.

A major breakthrough came with cloning of Dolly the Sheep in 1997 by Keith Campbell, Bill Ritchie and Ian Wilmut at the Roslin Institute in Scotland. This work disproved the central dogma that stated development was unidirectional i.e. the embryo becomes the fetus which becomes the adult. In these cloning experiments, the nucleus of a fertilised sheep egg was replaced with the nucleus from an udder cell. This reconstructed nuclear transfer embryo was transplanted into a surrogate sheep mother, which gave birth to Dolly. Since this landmark discovery, many scientists worked tirelessly to see if different somatic cells (e.g. skin cells, blood cells etc) could be converted into stem cells in the lab but without the need for nuclear transfer and fertilised eggs, which is ethically-sensitive in many countries.

The major breakthrough came in 2006 by Shinya Yamanaka in Japan. To the astonishment of the worlds scientific community, his group showed skin cells from a mouse could be reprogrammed into stem cells by adding just 4 genetic factors (Oct4, Sox2, Klf4 and cMyc), now known as the Yamanaka Factors. In 2007, Yamanaka went on to show this process also worked in human cells in a process called induced pluripotency giving rise to human induced pluripotent stem cells (hiPSC). Already, hiPSC have been used to produce retinal cells, which are being transplanted into patients with eye disease in clinical trials in Japan.

It is noteworthy that transplantation is not the only use for hiPSCs and hESCs. For example, new drugs are currently tested in animals to determine whether unwanted side effects occur in organs such as the heart, liver and brain. These cell types can be made from hESC and hiPSC, so there is growing interest by the pharmaceutical industry in reducing or replacing animals with drug testing platforms that use human stem cells instead. Furthermore, because hiPSC can be made from patients who harbour genetic disorders, an exploding area of research is to recreate these disorders in the lab in what is becoming known as disease in a dish technology. This gives new opportunities to better understand these disorders and develop novel drugs or genetic therapies. It is for these reasons that the Nobel Prize for Medicine in 2012 was awarded jointly to John Gurdon in the UK for his early work on reprogramming and to Shinya Yamanaka for his work on hiPSC.

Unequivocally, the long history of stem cells shows their potential in biomedicine and there is every indication that this utility will expand in the future. However, progressing stem cells from bench to bedside takes decades of hard, slow work, which is not so exciting for the media to present. Nevertheless, the coming years should prove to be an exciting time for stem cell research and medicine.

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What is Cell Therapy | Cell Therapy Explained

BioTime is a leader in pluripotent cell asset development and lineage derivation protocols. Pluripotent cells, which are widely published as capable of being induced to become any cell type of the human body, have potential applications in many areas of medicine with large unmet patient needs, including certain age-related degenerative diseases and degenerative conditions for which there presently are no cures.

Unlike pharmaceuticals that require a molecular target, cellular therapies are often aimed at regenerating or replacing affected cells and tissues and or improving bodily functions such as immune surveillance, and therefore, may have broader or more suitable applicability than many pharmaceutical products. Small molecules and biologic therapies that require systemic delivery into the body often have unexpected results, or side effects, that can limit their usefulness. Cell replacement is locally administered, so systemic side effects are usually not a primary concern in therapeutic development. The risk profile more closely resembles that of transplant medicine, focused more on whether the transplanted cells are rejected by the body and whether the cells function as expected.

We currently are using our pluripotent stem cells as biological starting material from which we derive three separate and specific cell types, each of which are product candidates currently in clinical testing (see Pipeline).

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Cell Therapy - BioTime, Inc.