Celularity Presents Pre-Clinical Data Demonstrating the Potential of Allogeneic Placental-Derived, Cryopreserved NK Cell Therapy (CYNK-001) in…

WARREN, N.J.--(BUSINESS WIRE)--Celularity, Inc. (Celularity ), a clinical-stage cell therapeutics company focused on the development of innovative allogeneic cellular therapies from human placentas, today announced pre-clinical data supporting the advancement of CYNK-001, a placental-derived, cryopreserved natural killer (NK) cell therapy, as a clinical candidate for patients with glioblastoma multiforme (GBM) in a poster presentation at the 2019 Society for Neuro-Oncology (SNO) Annual Meeting in Phoenix, A.Z.

Results from pre-clinical studies demonstrated in vivo anti-GBM activity of CYNK-001. The positive results from these pre-clinical studies highlight the versatility and activity of our investigational, off-the-shelf, placental-derived NK cell therapy program, said Robert Hariri, M.D., Ph.D., Founder, Chairman and CEO at Celularity. There is a clear need for novel therapeutic approaches to the treatment of glioblastoma multiforme. We look forward to advancing our investigational programs and are optimistic about the possibility of bringing another therapeutic option to patients in need.

About CYNK-001CYNK-001 is the only cryopreserved allogeneic, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors. NK cells are a unique class of immune cells, innately capable of targeting cancer cells and interacting with adaptive immunity. When derived from the placenta, these cells offer intrinsic safety and versatility, allowing potential use across a range of organs and tissues. CYNK-001 is currently being investigated as a treatment for acute myeloid leukemia (AML), multiple myeloma (MM), and as a potential treatment option for various solid tumors.

About CelularityCelularity, headquartered in Warren, N.J., is a clinical-stage cell therapeutics company delivering transformative allogeneic cellular therapies, engineered from the postpartum human placenta. Using proprietary technology in combination with its IMPACT platform, Celularity is the only company harnessing the purity and versatility of placental-derived cells to develop and manufacture innovative and highly scalable off-the-shelf treatments for patients with cancer, inflammatory and age-related diseases. To learn more, please visit http://www.celularity.com.

Forward-Looking StatementsThis press release contains forward-looking statements. These forward-looking statements are based on expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. The information contained in this press release is believed to be current as of the date of original issue. Celularity expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.

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Celularity Presents Pre-Clinical Data Demonstrating the Potential of Allogeneic Placental-Derived, Cryopreserved NK Cell Therapy (CYNK-001) in...

Full Alliance Group Announces New EBO2 Sites in Florida and the Caribbean – PRNewswire

BEVERLY HILLS, California, Nov. 21, 2019 /PRNewswire/ -- Full Alliance Group, Inc. (OTCPK: FAGI) is pleased to announce the opening of two new EBO2 treatment offices.

Dr. Yu and his staff arrived in Boca Raton, Florida, and then the Cayman Islands earlier this week with two brand-new portable EBO2 units and are currently training Dr. Joseph Purita and his staff. Dr. Purita is well-known in the stem cell and regenerative medicine sector and is eager to implement our ozone technology within his thriving practice.He has offices in Boca Raton as well as in the Cayman Islands.

Dr. Purita's Pensum Regenerative Clinic in Grand Cayman already specializes in stem cell treatment and anti-aging.The addition of EBO2 technology is expected to work synergistically with the well-established stem cell protocols already in place at the clinic.

"Expanding our footprint to the East and South, as well as the Caribbean, has been a critical part of our strategic plan," said Dr. Brian Volpp, MD, CEO and President of Full Alliance Group, Inc. "We are grateful that Dr. Purita has committed to the Yu Method and that he has made the financial commitment for two of our new units.His reputation within the stem cell community should allow ozone therapy to become more mainstream as he gains more experience with integrating EBO2 into his existing protocols."

Also, we have been advised by our legal team that the third quarter financial filing must be made publicly available to all shareholders. Consequently, the filing has been posted on our website:https://FullAlliance.com

About Full Alliance Group Inc.

Full Alliance Group Inc. (OTCPK: FAGI) is a multi-faceted holding company with various interests in technology, healthcare, and nutraceuticals. Nutra Yu, Inc., a wholly owned subsidiary of Full Alliance Group, develops, markets, and distributes a proprietary line of nutraceutical products. EBO2, Inc., a wholly owned subsidiary of Full Alliance Group, is the provider of ''EBO2'', a modern high volume blood gas exchange unit for the treatment of 5-7 liters of blood with medical ozone. The unit allows extracorporeal blood and oxygenation, ozone exposure and blood filtration.The process filters blood in a unique way by using the integrated diffusing membranes within the filter fibers to trap lipids and proteins which are in excess in the venous blood supply. The EBO2 unit is considered the world's most advanced medical ozone therapy.

For additional information regarding Full Alliance Group, visit, http://www.fullalliance.com.

Paul Brian Volpp, MD, MPH, President / CEO The Full Alliance Group

Forward-Looking StatementsThis shareholder update may contain a number of forward-looking statements. Words and variations of words such as: "expect", "goals", "could", "plans", "believe", "continue", "may", "will", and similar expressions are intended to identify our forward-looking statements, including but not limited to: our expectation for growth, benefits from brand-building, cost savings and margins. These forward-looking statements are subject to a number of risks and uncertainties, many of which are beyond our control, which could cause our actual results to differ materially from those indicated in our forward-looking statements. Such factors include, but are not limited to: continued volatility of, and sharp increase in: costs/pricing actions, increased competition, ability to raise sufficient operating capital, risks from operating internationally, consumer weakness, weakness in economic conditions and tax law changes.

Full Alliance Group Inc. Investor RelationsDave DonlinEmail: Info@TheCervelleGroup.comPhone: (407) 490-6635Web: http://www.StockInvestorDaily.comVisit: http://www.fullalliance.comContact: ir@fullalliance.com

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Full Alliance Group Announces New EBO2 Sites in Florida and the Caribbean - PRNewswire

Allogene and the need for a reliable cell source – Vantage

David Chang saw the end goal early. As chief medical officer of Kite Pharma, before that company was ever in allogeneic cell therapy, he did a deal with UCLA to secure a supply of pluripotent stem cells. Now, as chief executive of Allogene, he has repeated the trick, licensing in Notch Therapeutics stem cell technology this month.

Speaking to Vantage at this weeks Jefferies conference in London Mr Chang said that within a month of Allogenes creation he recognised the need to secure cell supply and of course he had done it before, at Kite. Any allogeneic cell therapy company at some point has to start thinking about renewable cell source, he states.

Since the 2017 approvals of Kymriah and Yescarta the development of autologous cell therapy products has stalled, and many investors see allogeneic, or off-the-shelf, therapy as the breakthrough that will move the needle.

However, the first generation of allogeneic players have hit production bottlenecks, and have struggled with cells derived from healthy donors; these tend to be heterogeneous and of variable quality not what is needed when the goal is to produce a reliable, universal product.

Single cell

Using induced pluripotent stem cells (iPSCs) as the source, however, is likened by another player in this field, Fate Therapeutics, to going by monoclonal antibody principles: starting from a single cell line.

The idea is that virtually any adult cell can, in a petri dish, be regressed to an iPSC phenotype, and this, using clever science, can then be reprogrammed into a defined mature cell that can be infinitely expanded.

What the iPSC is differentiated into depends on each developers focus: Allogene is clearly working on T cells, whereas Fate is initially targeting NK cell therapeutics. Each comes with its own degree of difficulty, and Mr Chang argues that differentiating iPSCs into NK cells is possibly easy.

He says Allogene actively went out to source such a technology, and there were several potential partners, though of course the UCLA work was no longer available. Without going into scientific minutiae, the Notch teams underlying concept is the same as that of UCLA, says Mr Chang.

However, the Notch approach is a serum-free, cell-free system to differentiate iPSCs. The UCLA technology, pioneered by Dr Gay Crooks andalso known as an artificial thymic organoid, uses so-called feeder cells on top of which the desired iPSC-derived cells are differentiated.

All that said, the Notch deal caused a degree of consternation, with some Allogene investors seeing it as demonstrating a lack of confidencein Cellectiss donor-derived approach.

Mr Chang says he was taken aback by such pushback, and stresses that the transition from autologous to allogeneic is not a bet on a single technology but a stepwise process. Nevertheless, the clear implication is that, in time, Cellectis's current healthy donor cell source approach will become obsolete.

Though Mr Chang accepts that the science is not quite there to implement the Notch technology into clinical programmes, he says: The way we see the future, whether its five or 10 years down the line, is in the renewable cell source.

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Autologous Stem Cell and Non Stem Cell Based Therapies Market Shares, Strategies, and Forecasts Analysis – Downey Magazine

In autologous stem cell and non-stem cell based therapies, an individuals cell is cultured and then re-introduced to the donors body. Used for the treatment of various bone marrow diseases, autologous stem cell and non-stem cell based therapies allows patients to have normal bone marrow, which gets destroyed in chemotherapy. The various diseases that can be treated with the help of autologous stem cell and non-stem cell based therapies include: multiple myeloma, aplastic anemia, non-Hodgkins lymphoma, Parkinsons disease, Hodgkins lymphoma, thalassemia, and diabetes. Thus, the demand for this therapy is projected to rise over the coming years.

The report is a thorough analysis of theAutologous Stem Cell and Non-Stem Cell Based Therapies Market. Comprising an in-depth analysis of the various factors boosting and inhibiting the growth of the market, this report is a key to making profitable decisions by investing in the correct segment and sub-segment, which is anticipated to make the most progress in the future.

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Autologous Stem Cell and Non-Stem Cell Based Therapies Market: Trends and Opportunities

One of the key drivers for this market is the rise in the prevalence of cancer and diabetes among people across all age groups. Moreover, the growing geriatric population is another factor, which is likely to create a heightened demand for autologous stem cell and non-stem cell based therapies. Favorable reimbursement policies across several nations are also aiding the growth of this market.

Players in the market are striving to achieve therapies that are not only safe and effective but also affordable and easy to use. Players are also investing in extensive research and development so as to speed up the treatment process of autologous stem cell and non-stem cell based therapies. While currently this treatment is quite expensive, government bodies are expected to take up initiatives and make the therapy affordable in the years to come. This is expected to drive the market in the future.

On the other hand, challenges faced by the global autologous stem cell and non-stem cell based therapies market include risks and complications associated with the therapy, such as diarrhea, hair loss, nausea, severe infections, vomiting, heart complications, and infertility.

Autologous Stem Cell and Non-Stem Cell Based Therapies Market: Geographical Analysis

By geography, North America, trailed by Europe is leading in the autologous stem cell and non-stem cell based therapies market, on account of the minimization of risks associated with the therapy. Also, these therapies are highly in demand owing to their ability to treat a large number of infectious diseases. The fact that autologous stem cell and non-stem cell based therapies do not require an outside donor, makes it more convenient and less infectious. All these factors are boosting the growth of the market in North America.

Asia Pacific is projected to show the most promising growth in the years to come with high demand from China, Vietnam, Malaysia, and India. The demand is expected to be high as autologous stem cell and non-stem cell based therapies help in the effective treatment of cardiovascular diseases. Sophisticated healthcare infrastructure and favorable tax and reimbursement policies are also expected to aid the growth of the Asia Pacific autologous stem cell and non-stem cell based therapies market.

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Autologous Stem Cell and Non-Stem Cell Based Therapies Market: Companies Mentioned

Some of the leading players operating in the autologous stem cell and non-stem cell based therapies market are Fibrocell Science, Inc., Aastrom Biosciences, Dendreon Corporation, NeoStem, Inc., BrainStorm Cell Therapeutics, Regeneus Ltd., and Genzyme Corporation.

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Autologous Stem Cell and Non Stem Cell Based Therapies Market Shares, Strategies, and Forecasts Analysis - Downey Magazine

Human Embryonic Stem Cells (HESC) Market with Future Prospects, Key Player SWOT Analysis and Forecast To 2024 – Chronicles 360

The Global Human Embryonic Stem Cells (HESC) Market Outlook Report is a comprehensive study of the Human Embryonic Stem Cells (HESC) industry and its future prospects.. A comprehensive research report created through extensive primary research (inputs from industry experts, companies, stakeholders) and secondary research, the report aims to present the analysis of Human Embryonic Stem Cells (HESC) Market.

Read Report Details at https://www.proaxivereports.com/51901

List of key players profiled in the report:

ESI BIOThermo FisherBioTimeMilliporeSigmaBD BiosciencesAstellas Institute of Regenerative MedicineAsterias BiotherapeuticsCell Cure NeurosciencesPerkinElmerTakara BioCellular Dynamics InternationalReliance Life SciencesResearch & Diagnostics SystemsSABiosciencesSTEMCELL TechnologiesStemina Biomarker DiscoveryTakara BioTATAA BiocenterUK Stem Cell BankViaCyteVitrolife

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TheHuman Embryonic Stem Cells (HESC)Market Segmentation:

Product Product Type Segmentation SegmentationTotipotent Stem CellsPluripotent Stem CellsUnipotent Stem Cells

Industry SegmentationResearchClinical Trials

The report analyses the Human Embryonic Stem Cells (HESC) Market By Type and By Country for the historical period of 2017-2018 and the forecast period of 2019-2024.

Region Segmentation of Human Embryonic Stem Cells (HESC) Market

North America Country (United States, Canada)

South America

Asia Country (China, Japan, India, Korea)

Europe Country (Germany, UK, France, Italy)

Other Country (Middle East, Africa, GCC)

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The report has covered and analyzed the potential of Human Embryonic Stem Cells (HESC) market and provides statistics and information on market size, shares and growth factors. The report intends to provide cutting-edge market intelligence and help decision makers take sound investment evaluation. Besides, the Human Embryonic Stem Cells (HESC) market report also identifies and analyses the emerging trends along with major drivers, challenges and opportunities. Additionally, the report also highlights market entry strategies for various companies.

Scope of the Human Embryonic Stem Cells (HESC) Market Report

Human Embryonic Stem Cells (HESC) Market (Actual Period: 2017-2018, Forecast Period: 2019-2024)Human Embryonic Stem Cells (HESC) Market Size, Growth, ForecastAnalysis By Type:

Regional Analysis Actual Period: 2017-2018, Forecast Period: 2019-2024Human Embryonic Stem Cells (HESC) Market Size, Growth, ForecastHuman Embryonic Stem Cells (HESC) Market Analysis By Type

Report HighlightsCompetitive Landscape: Company Share AnalysisMarket Dynamics Drivers and Restraints.Market TrendsPorter Five Forces Analysis.SWOT Analysis.Company Analysis

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Human Embryonic Stem Cells (HESC) Market with Future Prospects, Key Player SWOT Analysis and Forecast To 2024 - Chronicles 360

Tokyo’s Heartseed reaps $26M to test its stem cell injections for heart failure – FierceBiotech

Tokyo-based Heartseed has raised 2.8 billion yen, or about $26 million U.S., to help develop its stem cell-based treatment for heart failure.

The company has its eyes on two clinical trials, set to start in the next year: an investigator-initiated study through its research partner Keio University in dilated cardiomyopathy, followed by a phase 1/2 trial in late 2020 for heart failure with reduced left ventricular ejection fraction.

Heartseeds treatment differs in approach from other, similar therapies using induced pluripotent stem cells (iPSCs), where sheets of cells are grafted onto the surface of the heart to improve vascularization and blood flow.

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Instead, the company forms spherical clusters of heart cells from purified, allogeneic iPSCs, which are injected directly into the heart muscle wall. As they grow and electrically sync with other cells within the myocardium, Heartseed expects to see improvements in the strength of contractions.

The companys series B round included new investors SBI Investment, JMDC, Gene Techno Science, Nissay Capital and SMBC Capital as well as Astellas Venture Management, returning from Heartseeds series A raise. The latest proceeds bring its total funding to 3.8 billion yen, or about $35 million.

RELATED: Growing transplantable arteries from stem cells

Heartseed was founded in 2015 through the research of its CEO, Keiichi Fukuda, a professor in the department of cardiology at Keio University, and his teams work in regenerative medicine and cell production, purification and delivery.

We are grateful for the support of our investors, which I believe is a reflection of their expectation and confidence that our lead pipeline HS-001 can be a curative therapy for severe HF, with the mechanism that transplanted ventricular-specific highly-purified cardiomyocytes engraft to patients heart and retain for a long-term, Fukuda said in a statement.

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Tokyo's Heartseed reaps $26M to test its stem cell injections for heart failure - FierceBiotech

Omega 3 Fatty Acid Health Welfare Connected To Stem Cell Regulation Researchers Discover – News Raise

Omega 3 fatty acid health welfare connected to stem cell regulation researchers discover. For years researchers have acknowledged that imperfections in a former cellular antenna known as the primary cilium are connected to obesity and insulin aversion. Presently researchers at the Stanford University School of Medicine have found that the peculiar minuscule cellular attachment is discerning omega 3 fatty acids in the food and that this gesticulation is in a beeline impacting how stem cells in fat tissue splinter and convert into fat cells.

The discovery constitutes an absent connection between two worlds that of dietary science and that of molecular and cellular biology. Dietary studies have long ago discovered that the expending of omega 3 fatty acids crucial fatty acids customary in fish and nuts is linked with lesser of heart disease, stroke, arthritis, and even depression.

Researchers in Jacksons lab was not observing omega 3s when they commenced their research. They were observing the gesturing molecule that fat stem cells were discerning. The molecule could have been anything gesturing trail in cellular biology frequently include esoteric molecules handful of people have listened to. They were aware of the fact that uncommon illnesses including a deficiency in the primary cilium, people are often hungry and cannot put a halt to consuming and thereby become obese and insulin unaffected by. So they were taken aback when the signal flipped out to be omega 3 fatty acids.

Steve Lopez is the Editorial Page Editor for News Raise. He covers Health. He has won more than a dozen national journalism awards for his reporting and column writing at seven newspapers and four news magazines.

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Omega 3 Fatty Acid Health Welfare Connected To Stem Cell Regulation Researchers Discover - News Raise

Junior Research Fellow for Stem Cell-Based Neural Tissue Engineering Project job with VELLORE INSTITUTE OF TECHNOLOGY | 187070 – Times Higher…

Job Description

Junior Research Fellow (JRF)for Translational Research - Stem Cell-based Neural Tissue Engineering Project:

Title of the Project: Human dental pulp stem cells as a multifaceted tool for accelerating neural regenerationDuration: 3 YearsLocation: Vellore Institute of Technology, Vellore

Qualification:

M.Sc/ M. Tech (Biomaterials, Tissue Engineering, Biotechnology, Biology, and Biomedical Sciences) with a minimum of 55% marks.

Skill set required:

Candidate with work experience in biomaterial synthesis, scaffold fabrication and stem cell culture is desirable.

Stipend: Rs.20,000/- per month (consolidated)

Work functions of the JRF: The JRF will be required to do full time research related to this specific project, in particular biomaterial synthesis and characterization, scaffold fabrication, biological assays, dental stem cell culture.

Principal Investigator:

Dr.Murugan RamalingamCentre for Biomaterials, Cellular and Molecular Theranostics (CBCMT)School of Mechanical EngineeringVellore Institute of Technology (VIT),Vellore 632014

Send your resume along with relevant documents pertaining to the details of qualifications, experience and latest passport size photo on or before (30/11/2019) through online http://careers.vit.ac.in.

No TA and DA will be paid for appearing for the interview.

Shortlisted candidates will be called for an interview at a later date which will be intimated by email.

Salary:Not Disclosed by RecruiterIndustry:Education / Teaching / TrainingFunctional Area:Teaching, Education, Training, CounsellingRole:Trainee

Keyskills

stem cellsbiotechnologybiologybiomaterials

Desired Candidate Profile

Please refer to the Job description above

Education-

UG:B.Tech/B.E. - Bio-Chemistry/Bio-Technology, Biomedical, B.Sc - BiologyPG:M.Tech - Bio-Chemistry/Bio-Technology, Biomedical, MS/M.Sc(Science) - Biotechnology, Biology

Company Profile

Vellore Institute of Technology

VIT was established with the aim of providing quality higher education on par with international standards. It persistently seeks and adopts innovative methods to improve the quality of higher education on a consistent basis.The campus has a cosmopolitan atmosphere with students from all corners of the globe. Experienced and learned teachers are strongly encouraged to nurture the students.

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Junior Research Fellow for Stem Cell-Based Neural Tissue Engineering Project job with VELLORE INSTITUTE OF TECHNOLOGY | 187070 - Times Higher...

Europe Stem Cell Assay Market to Witness a Pronounce Growth in Coming Years | Insights, Trends, Growth, Industry Drivers with Cellular Dynamics…

The Europe stem cell assay market is expected to grow US$793 million by 2027. The market growth is expected at the CAGR of +22% in the forecast period.

Stem cells have a remarkable potential to develop into different types of cells and are involved in the internal repair system of the body. Stem cell assays are the techniques for analyzing the living cells on the parameters of shape, size, besides others with the aim to measure the biochemical and cellular functioning of the cells. Such properties make the stem cell assays to be extensively utilized in industrial practices of drug development. The growing biotechnology sector and increasing global burden of diseases like cancer, diabetes, and others are estimated to be the major drivers for the Europe Stem Cell Assay Market growth during the forecast period.

The latest report entitled Europe Stem Cell Assay Market from 2019 to 2027 has been recently added to the repository of report consultants. It encircles the evaluation made on the basis of key competitors who are showing active participation in determining how the market actually works. They have achieved great success in understanding what the expectations of their applications are and what the anticipated trends that may show up in the future.

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Top Key Players Covered in Europe Stem Cell Assay Market:

Cellular Dynamics International (Holding Company Fujifilm), Cell Bio Labs, Inc., Corning Inc., Hemogenix Inc., Promega Corporation, Thermo Fisher, Stem Cell Technologies, Fibrocell Science Inc., Becton Dickinson, Stryker Corporation, Takara Holdings Inc., R&D Systems, Bio-Rad, GE Healthcare and Merck Millipore (holding company is Merck KGaA).

The Europe stem cell assay market by geography is segmented into the UK, France, Germany, Spain, Italy and Rest of Europe. Increasing Adoption of Stem Cell Assay, improvement in the Technology, high expense in Stem Cell Research is the major drivers for the market growth in the Europe region. The rise in the adoption of stem cell assay for drug screening & testing is one of the major reasons driving the industry research

Market Segmentation:

Europe Stem Cell Assay Market by Type:

Europe Stem Cell Assay Market by Application:

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Key highlights of the Europe Stem Cell Assay Market for the forecast years 2019-2026:

It has been accumulated through a couple of research methodologies such as primary and secondary research. The Europe Stem Cell Assay Market data has been observed through industry-specific analysis tools like SWOT and Porters five techniques. This informative data has been gathered through reliable sources such as press releases, websites, interviews, and statistical surveys.

In This Study, The Years Considered To Estimate The Size Of Europe Stem Cell Assay Market Are As Follows:

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CRISPR Therapeutics and Vertex: Promising Gene Therapy Data for Sickle Cell Disease and Beta Thalassemia – BioSpace

CRISPR Therapeutics and Vertex Pharmaceuticals announced positive interim data from the first two patients in the companies ongoing Phase I/II clinical trials of its CRISPR/Cas9 gene-editing therapy CTX001.

One of the patients with transfusion-dependent beta thalassemia (TDT) was treated with CTX001 in the first quarter of 2019. The data reflects nine months of safety and efficacy follow-up.

The second patient has severe sickle cell disease (SCD) and received CTZX001 in mid-2019. The interim data is for four months of safety and efficacy follow-up.

For the treatment, patients have hematopoietic stem and progenitor cells collected from their peripheral blood. The cells are then edited using CRISPR/Cas9. The edited cells, which are called CTX001, are then infused back into the patient as part of a stem cell transplant.

The TDT patient prior to the treatment required 16.5 transfusions per year. After treatment, the patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. There were two serious adverse events, but the principal investigator did not believe they were related to CTX001. One was pneumonia in the presence of neutropenia and veno-occlusive liver disease associated to busulfan conditioning, which is part of a stem cell transplant used to destroy the original bone marrow that has the mutation in it.

Nine months after CTX001 infusion, the patient was transfusion independent. Total hemoglobin levels were 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells.

The SCD patient had seven vaso-occlusive crises (VOCs) annually. Thirty days after CTX001 infusion, the patient reached neutrophil and platelet engraftment. This patient had three severe adverse events, none of which the principal investigator believed were related to CTX001. They were sepsis in the presence of neutropenia, cholelithiasis, and abdominal pain. All resolved. Four months after treatment, the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells.

The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia, said Jeffrey Leiden, chairman, president and chief executive officer of Vertex. While the data are exciting, we are still in the early phase of this clinical program. We look forward to continuing to work with physicians, patients, caregivers and families over the coming months and years to bring forward the best possible therapy for these two serious diseases and to continue to accelerate our gene-editing programs for other serious diseases such as Duchenne muscular dystrophy and myotonic dystrophy type 1.

These data are very strong evidence of the promise of CRISPR treatments that could potentially cure diseases. CRISPR and Vertex are not the first, however. Bluebird Bio received approval in Europe for its gene therapy, Zynteglo, for transfusion-dependent beta-thalassemia. It uses a different approach than Vertex and CRISPR Therapeutics, and it does not work in all patients. A decision by the U.S. Food and Drug Administration (FDA) is expected in 2020. The therapy has a price tag of about $1.75 million spread over a five-year installment plan.

Mitchell Weiss of St. Jude Childrens Research Hospital, told STAT, that after years of sickle cell research neglect, these new approaches represented a perform storm. For decades, we knew about the sickle cell disease mutations, but we didnt know about other genes [involved in the disease] and we didnt have the necessary tools for genetic correction of blood-making stem cells. Now we have a confluence of scientific understanding and technology that can come together to make things happen.

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CRISPR Therapeutics and Vertex: Promising Gene Therapy Data for Sickle Cell Disease and Beta Thalassemia - BioSpace