CAR-T Cell Therapy Market to Witness Great Growth| Autolus, Juno Therapeutics, Kite Pharma, Novartis AG – Markets Gazette 24

Global CAR-T Cell Therapy Market Report contains historic data that spans 2013 to 2019, and then continues to forecast to 2026. That makes this report so invaluable, resources, for the leaders as well as the new entrants in the Industry Research details developments in the Report with Detailed Analysis of Key Companies Capacity, Production, Revenue, Price and Gross Margin. The research is derived through primary and secondary statistics sources and it comprises both qualitative and quantitative detailing. Some of the key players profiled in the study are Autolus., Bellicum Pharmaceuticals, Inc., Bluebird Inc., CELGENE CORPORATION, Cellectis, Celyad, Eureka Therapeutics, Fortress Biotech., Immune Therapeutics, Juno Therapeutics, Kite Pharma, Novartis AG, Sorrento Therapeutics, Inc., TILT Biotherapeutics, Ziopharm Oncology.

Global CAR-T cell therapy market is set to witness a healthy CAGR of 46.35% in the forecast period of 2019- 2026.

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How effectively is the CAR-T Cell Therapy Market Segmented

The report is comprised of information about the market share which each product holds as well as the projected remuneration of the each segment. The research report is also inclusive of details with regards to the consumption (value and growth rate) of each product and the sales price as well.

Geography: North America, South America, Europe, Asia-Pacific, Middle East and Africa

By Target Antigen: CD 19, CD 20, GD2, CD22, CD30, CD33, HER1

By Application: Acute Lymphocytic Leukemia, Chronic Lymphocytic Leukemia

Competitive Landscape:

Mergers & Acquisitions, Agreements & Collaborations, New Product Developments & Launches, Business overview & Product Specification for each player listed in the study. Players profiled are Autolus., Bellicum Pharmaceuticals, Inc., Bluebird Inc., CELGENE CORPORATION, Cellectis, Celyad, Eureka Therapeutics, Fortress Biotech., Immune Therapeutics, Juno Therapeutics, Kite Pharma, Novartis AG, Sorrento Therapeutics, Inc., TILT Biotherapeutics, Ziopharm Oncology.

Read Detailed Index of full Research Study at: https://www.databridgemarketresearch.com/toc/?dbmr=global-car-t-cell-therapy-market

CAR-T Cell Therapy report reveals the nature of demand for the firms product to know if the demand for the product is constant or seasonal. With this report not only an unskilled individual but also a professional can easily extrapolate the entire market within a few seconds. TOC, graphs and tables included in the report helps understand the market size, share, trends, growth drivers and market opportunities and challenges.

Drivers & Restraints

Market Drivers

Rising demand for chemotherapy among consumer is driving the market growth

Increasing number of cancer patient worldwide is also acting as a driver for this market

Increasing awareness among population about the effectiveness of CAR T- cell therapy will also propel the growth of this market

Market Restraints

High cost of the therapy will restrain the growth of this market

Complexity related with manufacturing process will also hamper the growth of this market

Key Highlights from CAR-T Cell Therapy Market Study.

Revenue and Sales Estimation Historical Revenue and sales volume is presented and further data is triangulated with top-down and bottom-up approaches to forecast complete market size and to estimate forecast numbers for key regions covered in the report along with classified and well recognized Types and end-use industry. Additionally macroeconomic factor and regulatory policies are ascertained in CAR-T Cell Therapy industry evolution and predictive analysis.

Manufacturing Analysis the report is currently analyzed concerning various product type and application. The CAR-T Cell Therapy market provides a chapter highlighting manufacturing process analysis validated via primary information collected through Industry experts and Key officials of profiled companies.

Competition Leading players have been studied depending on their company profile, product portfolio, capacity, product/service price, sales, and cost/profit.

Demand & Supply and Effectiveness CAR-T Cell Therapy report additionally provides distribution, Production, Consumption & EXIM** (Export & Import). ** If applicable

According to this CAR-T Cell Therapy report, the market is greatly transforming because of the moves of the key players and brands including developments, product launches, joint ventures, mergers and acquisitions that in turn changes the view of the global face of industry. The CAR-T Cell Therapy market report includes data on patterns and improvements, target business sectors and materials, limits and advancements. It is helpful in determining the discount rates, the actual prices and the price ranges, price elasticity for its products.

Development and Manufacturing Analysis of CAR-T Cell Therapy Market

Capacity and Commercial Production Date

Major Manufacturers Technology Source and Market Position of CAR-T Cell Therapy

Recent Development and Expansion Plans

What are the challenges of the CAR-T Cell Therapy Market?

The report elucidates information about the driving forces influencing the commercialization portfolio of the CAR-T Cell Therapy market and their impact on the revenue graph of this business sphere.

The study is inclusive of the latest trends characterizing the CAR-T Cell Therapy market in consort with the challenges that this industry will present in the future.

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CAR-T Cell Therapy Market to Witness Great Growth| Autolus, Juno Therapeutics, Kite Pharma, Novartis AG - Markets Gazette 24

Targeted Toxin Gene Therapy Of Breast Cancer Stem Cells Using CXCR1 Pr | OTT – Dove Medical Press

Cobra Moradian, Fatemeh Rahbarizadeh

Department of Medical Biotechnology, Faculty of Medical Sciences, Tarbiat Modares University, Tehran, Iran

Correspondence: Fatemeh RahbarizadehDepartment of Medical Biotechnology, Faculty of Medical Sciences, Tarbiat Modares University, Jalal AleAhmad Highway, Tehran 14115-111, IranTel +98 21 82883884Fax +98 21 82884555Email rahbarif@modares.ac.ir

Background: Breast cancer stem cells (BCSCs) are cells with a higher ability to metastasis and resistance to conventional treatments. They have a phenotype of (CD44high/CD24low) and the unlimited ability for proliferation. Development of strategies to target the BCSC population may lead to the establishment of more effective cancer therapies. Pseudomonas exotoxin A (PE) is a potent cytotoxic protein. CXCR1 promoter provides BCSC and HER2 specificity on transcription level. 5UTR of the basic fibroblast growth factor-2 (bFGF 5UTR) provides tumor specificity on translation level. Here, we utilized a mutant form of PE encoding DNA PE38, CXCR1 promoter and bFGF 5UTR to target BCSCs.Methods: The stemness of SK-BR-3, MDA-MB-231 and MCF10A cell lines were evaluated based on the expression of the CD44high/CD24low stem cell signature and the ability to form mammospheres. Then, the cell lines were transfected with constructs encoding luciferase/PE38 under the control of the CMV/CXCR1 promoter with or without bFGF 5UTR. Luciferase protein expression was evaluated using dual-luciferase reporter assay. PE38 transcript expression was measured by real-time PCR, and the cytotoxic effect of PE38 protein expression was determined by MTT assay.Results: The percentage of CD44high/CD24low population did not correlate to mammosphere forming efficiency (MFE). Given that the percentage of CD44 high/CD24 low is not a conclusive BCSC profile, we based our work on the mammosphere assay. However, in comparison with MCF10A, the two tumorigenic cell lines had higher MFE, probably due to their higher BCSC content. Reporter assay and real-time PCR results demonstrated that CXCR1 promoter combined with bFGF 5UTR increased BCSC-specific gene expression. Meanwhile, tightly regulated expression of PE38 using these two gene regulatory elements resulted in high levels of cell death in the two tumorigenic cell lines while having little toxicity toward normal MCF10A.Conclusion: Our data show that PE38, CXCR1 promoter and bFGF 5UTR in combination can be considered as a promising tool for killer gene therapy of breast cancer.

Keywords: breast cancer stem cell, PE38, CXCR1 promoter, bFGF-2, HER2, mammosphere

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Targeted Toxin Gene Therapy Of Breast Cancer Stem Cells Using CXCR1 Pr | OTT - Dove Medical Press

ReNeuron Presents Positive Data at the 27th Annual Congress of the European Society of Gene and Cell Therapy on Lead Cell Line – Yahoo Finance

New data show ReNeuron's lead CTX cell therapy candidate can be re-programmed into a pluripotent state and differentiated into other cell types

These new cell types can be efficiently expanded as potential cell therapy candidates targeting a broad range of diseases

PENCOED, Wales, Oct. 23, 2019 /PRNewswire/ --ReNeuron Group plc (RENE.L), a UK-based global leader in the development of cell-based therapeutics, is pleased to announce that new data relating to its CTX stem cell platform will be presented today at the 27th Annual Congress of the European Society of Gene and Cell Therapy(ESGCT), a leading scientific conference taking place this week in Barcelona, Spain.

Dr. Steve Pells, Principal Investigator at ReNeuron, will present new data showing the phenotypic stability and scalability of a mesenchymal stem cell line derived from the Company's proprietary, conditionally immortalized, human neural stem cell line (CTX) following re-programming to a pluripotent state.

The Company has previously presented data demonstrating that its CTX stem cell line, currently undergoing clinical evaluation for the treatment of stroke disability, can be successfully and rapidly re-programmed to an embryonic stem cell-like state enabling differentiation into any cell type. In essence, this means that the Company is able to take its neural stem cells back to being stem cells that can be made to develop into any other type of stem cell including bone, nerve, muscle and skin.

The new data being presented today show for the first time that these CTX-iPSCs (induced pluripotent stem cells) can indeed be differentiated along different cell lineages to generate, for example, mesenchymal stem cell lines. Further, the mesenchymal stem cell lines generated can be grown at scale by virtue of the Company's conditional immortalization technology, enabling the efficient production of clinical-grade cell therapy candidates.

These results are particularly encouraging as they demonstrate that CTX, a well-characterized, clinical-grade neural stem cell line, could be used to produce new conditionally immortalized allogeneic (i.e. non-donor-specific) cell lines from any of the three primary germ cell layers which form during embryonic development. ReNeuron is currently exploring the potential to develop further new allogeneic cell lines as potential therapeutic agents in diseases of unmet medical need for subsequent licensing to third parties.

Further information about the conference may be found at:

https://www.esgct.eu/congress/barcelona-2019.aspx

"The data we are presenting at the ESGCT Annual Congress represent a significant advance in the use of cell re-programming to generate new allogeneic cell lines as potential therapeutic candidates," commented Dr. Randolph Corteling, Head of Research at ReNeuron. "Importantly, the maintenance of the immortalization technology within these new cell lines may allow for the scaled production of 'off the shelf' allogeneic stem cells, such as haematopoietic stem cells as a potential alternative approach to those cancer immunotherapies currently in development that rely on the use of the patient's own T-cells."

About ReNeuronReNeuron is a global leader in cell-based therapeutics, harnessing its unique stem cell technologies to develop 'off the shelf' stem cell treatments, without the need for immunosuppressive drugs. The Company's lead clinical-stage candidates are in development for the blindness-causing disease, retinitis pigmentosa, and for disability as a result of stroke. ReNeuron is also advancing its proprietary exosome technology platform as a potential delivery system for drugs that would otherwise be unable to reach their site of action. ReNeuron's shares are traded on the London AIM market under the symbol RENE.L. For further information visit http://www.reneuron.com.

ENQUIRIES:

ReNeuron

+44 (0)20 3819 8400

Olav Helleb, Chief Executive Officer

Michael Hunt, Chief Financial Officer

Buchanan (UK)

+44 (0) 20 7466 5000

Mark Court, Tilly Abraham

Argot Partners (US)

Stephanie Marks, Claudia Styslinger

Stifel Nicolaus Europe Limited

+1 212 600 1902

+44 (0) 20 7710 7600

Jonathan Senior, Stewart Wallace, Ben Maddison (NOMAD and Joint Broker)

N+1 Singer

+44 (0) 20 7496 3000

Aubrey Powell, James Moat, Mia Gardner

(Joint Broker)

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ReNeuron Presents Positive Data at the 27th Annual Congress of the European Society of Gene and Cell Therapy on Lead Cell Line - Yahoo Finance

ReNeuron progresses as it gathers more data for developing treatments – Proactive Investors UK

What the company does

Human retinal progenitor cells (hRPC)

Human retinal progenitor cells are cells that differentiate into components of the retina.

Reneuron has developed the ability to scale up the manufacturing of the hRPCs using a patented low-oxygen cell expansion technology.

The hRPC cell therapy candidate is being evaluated in an ongoing phase I/IIa clinical trial in the US in subjects with a blindness-causing inherited retinal disease, retinitis pigmentosa (RP).

CTX Cells

CTX cell therapy candidate is a treatment for patients left disabled by the effects of a stroke.

Reneurons product is a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting with the diseases targeted, without the need for additional immunosuppressive drug treatments.

Data from the Phase II PISCES trial indicated CTX therapy was safe and well-tolerated and produced clinically meaningful and sustained improvement in the level of disability and dependence as well as motor function.

Exosome platform

Exosomes are nanoparticles, released by cells, and contain a number of active proteins and micro RNAs, which are short non-coding RNAs capable of regulating gene expression, that arebelieved to play a key role in cell-to-cell communication.

ExoPr0, Reneurons first CTX-derived exosome therapeutic candidate, has demonstrated potential as both a novel therapeutic candidate as well as a drug delivery vehicle

() has unique stem cell technologies that can be administered off-the-shelf. Its lead candidate has been developed to treat people disabled by a stroke.

Its human retinal progenitor cells (hRPC), meanwhile, have scored some early success.

A Phase I/II assessment of a very small group of sufferers of a blindness-causing disease called retinitis pigmentosa saw a significant improvement in vision after treatment.

Six months after treatment there was a mean improvement of 18.5per treated eye, with a mean improvement of 12 letters per treated eye after nine months, whereasinexorable disease progression is the norm for this disease.

With a total of 22 patients now treated and the study still ongoing, ReNeuron said the efficacy in subsequent patients was seen but at a lower rate and magnitude, with improvement in visual acuity ranging from +5 to +11 letters in the treated eye threemonths after treatment.

We are very encouraged by the data as it matures, it needs to mature further in the patients that we have treated more recently, but the bottom line is all systems go for that programme and we are looking forward to discussing with the regulatory authorities where we go next in terms of the next trial we would like to do.

Its fair to say that the responses we got in the first three patients were so extraordinarily good that it was going to be quite an achievement to try and replicate that in all patients that we treated, but what we are seeing is a nice improvement in all patients that we have treated who have responded to the treatment and the aggregate scores that we are seeing, if you like, for visual accuracy are extremely encouraging.

We have a product here and I think youll find that view is backed up by the investigators working with us on the study.

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ReNeuron progresses as it gathers more data for developing treatments - Proactive Investors UK

Cesca Therapeutics Forms Joint Venture With HealthBanks Biotech (USA) To Provide Immune Cell Banking And Cell Processing Service – Clinical Leader

ImmuneCyte to Begin Operations in Fourth Quarter of 2019

Rancho Cordova, CA /PRNewswire/ - Cesca Therapeutics Inc. (Nasdaq: KOOL), a market leader in automated cell processing and autologous cell therapies for regenerative medicine, and ThermoGenesis, its wholly owned device subsidiary, today announced that the company has entered into a definitive joint venture agreement with HealthBanks Biotech (USA) Inc., one of the world's leading stem cell bank networks, to commercialize its proprietary cell processing platform, CAR-TXpress, for use in immune cell banking as well as for cell-based contract development and manufacturing services (CMO/CDMO). The joint venture will be named ImmuneCyte Life Sciences Inc. ("ImmuneCyte") and is expected to officially launch during the fourth quarter of 2019.

Under terms of the agreement, ImmuneCyte will initially be owned 80% by HealthBanks Biotech and 20% by Cesca. Cesca will contribute to ImmuneCyte exclusive rights to use ThermoGenesis' proprietary cell processing technology for the immune cell banking business and non-exclusive rights for other cell-based contract development and manufacturing services. Cesca will also contribute its clinical development assets to the joint venture, as the company has decided to discontinue these activities in order to focus exclusively on the device business.

Once operational, ImmuneCyte will be among the first immune cell banks in the U.S. to provide clients with the opportunity to bank their own healthy immune cells for future use as a resource for cell-based immunotherapies, such as dendritic cell and chimeric antigen receptor (CAR) T-cell therapies. ImmuneCyte will utilize ThermoGenesis' proprietary CAR-TXpress platform which allows for the isolation of different components from 200 ml of blood in cGMP compliant, closed system. Given that the CAR-TXpress platform can increase cell processing efficiency by up to 16-fold as compared with the traditional, labor-intensive ficoll gradient centrifugation-based cell processing method, ImmuneCyte is expected to offer customers an unparalleled competitive advantage, including an ability to store their own immune cells at a tangibly lower cost.

"The ImmuneCyte joint venture will be paramount to the execution of our strategy to become a preferred cell processing and manufacturing solution provider in the cell and gene therapy field," said Dr. Chris Xu, Chairman and Chief Executive Officer of Cesca Therapeutics. "CAR-T therapeutic research is advancing rapidly. Partnering with HealthBanks Biotech, one of the foremost stem cell bank networks, with an experienced team and an established global infrastructure, will offer customers the ability to preserve younger, healthier and uncontaminated immune cells for potential future use. By applying our proprietary CAR-TXpress technology to immune cell banking and other CDMO cellular manufacturing services, we will allow for the manufacture and production of more effective and less costly immunotherapies."

In 2017, the U.S. Food and Drug Administration (FDA) approved two CAR-T cell therapies, under breakthrough designation, for the treatment of advanced B cell leukemia and lymphomas. Both use autologous (a patient's own) immune T cells to fight cancer and have reported an over 80% response rate in the "no-option" patient group, for those who have failed both chemo- and radiation therapies. This has helped to spur massive global interest for the development of additional CAR-T immunotherapies1. By the end of September 2019, there were over 800 CAR-T cell clinical trials registered on the http://www.clinicaltrials.gov website, targeting a wide variety of blood cancers and solid tumors.

Although highly effective, several recent studies on the eligibility of patients to enroll in CAR-T clinical trials showed that as many as 30-50% of cancer patients may not be eligible to enroll or to get sufficient CAR-T cells manufactured for the therapy. Reasons may include: (1) the function of the immune system declines with age and can be negatively affected by other medical conditions, (2) most standard cancer therapies, such as chemotherapy and radiation, destroy the immune system, and (3) in many cases of advanced cancer, cancer cells will enter circulation, invade and interfere with the body's natural production of immune cells. According to a recently reported JULIE trial, a CAR-T clinical trial in relapsed or refractory diffuse large B-cell lymphoma (DLBCL), one-third of the 238 screened patients failed to be enrolled, and more than half of the 238 failed to receive the intended CAR-T therapy2,3. ImmuneCyte will offer customers the ability to preserve younger, healthier and uncontaminated immune cells, for potential future use in advanced cancer immunotherapy.

About HealthBanks Biotech (USA) Inc.HealthBanks Biotech, headquartered in Irvine, CA, is one of the leading stem cell bank networks in the world and offers services globally through its sister companies located in the United States and other regions and nations. HealthBanks Biotech is accredited by the FDA, AABB, and CAP. The HealthBanks Biotech group was originally founded in 2001 with a vision that stem cells and cell and gene therapies could transform modern medicine. HealthBanks Biotech is a subsidiary of Boyalife Group, Inc. (USA), an affiliate of Boyalife (Hong Kong) Limited, the largest stockholder of Cesca. For more information about HealthBanks Biotech (USA) Inc., please visit: http://www.healthbanks.us.

About ImmuneCyte Life Sciences Inc.ImmuneCyte will provide clients with the opportunity to bank their own immune cells when the cells are "healthy and unaffected" as a future resource for cellular immunotherapies, such as CAR-T. ImmuneCyte utilizes a proprietary CAR-TXpress platform, a GMP compliant close-system capable of automated separating and cryopreserving different components from blood. For more information about ImmuneCyte Life Sciences Inc., please visit: http://www.immunecyte.com.

About Cesca Therapeutics Inc.Cesca Therapeutics develops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. Its device division, ThermoGenesis develops, commercializes and markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology. The Company has developed a semi- automated, functionally closed CAR-TXpress platform to streamline the manufacturing process for the emerging CAR-T immunotherapy market. For more information about Cesca and ThermoGenesis, please visit: http://www.cescatherapeutics.com.

References:1. Facts About Chimeric Antigen Receptor (CAR) T-Cell Therapy, Leukemia and Lymphoma Society (2018). https://www.lls.org

2. Updated Analysis of JULIET Trial: Tisagenlecleucel in Relapsed or Refractory DLBCL (2018).

3. Eligibility Criteria for CAR-T Trials and Survival Rates in Chemorefractory DLBCL. Journal of Clinical Pathways (2018).

SOURCE: Cesca Therapeutics Inc.

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Cesca Therapeutics Forms Joint Venture With HealthBanks Biotech (USA) To Provide Immune Cell Banking And Cell Processing Service - Clinical Leader

CAR-T Cell Therapy Market Poised to Garner Maximum Revenues by 2027 – Health News Office

Global Foamed Plastics Market Report 2019 Market Size, Share, Price, Trend and Forecast is a professional and in-depth study on the current state of the global Foamed Plastics industry.

The report also covers segment data, including: type segment, industry segment, channel segment etc. cover different segment market size, both volume and value. Also cover different industries clients information, which is very important for the manufacturers.

There are 4 key segments covered in this report: competitor segment, product type segment, end use/application segment and geography segment.

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For competitor segment, the report includes global key players of Foamed Plastics as well as some small players.

ArmacellBASFBayerCarpenterChemtura Corporation

Segment by RegionsNorth AmericaEuropeChinaJapanSoutheast AsiaIndia

Segment by TypePolyurethanePolystyrenePVC

Segment by ApplicationPackagingBuilding & ConstructionHousehold ProductsMotor Vehicles

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Important Key questions answered in Foamed Plastics market report:

What will the market growth rate, Overview, and Analysis by Type of Foamed Plastics in 2024?

What are the key factors affecting market dynamics? What are the drivers, challenges, and business risks in Foamed Plastics market?

What is Dynamics, This Overview Includes Analysis of Scope and price analysis of top Manufacturers Profiles?

Who Are Opportunities, Risk and Driving Force of Foamed Plastics market? Knows Upstream Raw Materials Sourcing and Downstream Buyers.

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The content of the study subjects, includes a total of 15 chapters:

Chapter 1, to describe Foamed Plastics product scope, market overview, market opportunities, market driving force and market risks.

Chapter 2, to profile the top manufacturers of Foamed Plastics , with price, sales, revenue and global market share of Foamed Plastics in 2019 and 2015.

Chapter 3, the Foamed Plastics competitive situation, sales, revenue and global market share of top manufacturers are analyzed emphatically by landscape contrast.

Chapter 4, the Foamed Plastics breakdown data are shown at the regional level, to show the sales, revenue and growth by regions, from 2019 to 2025.

Chapter 5, 6, 7, 8 and 9, to break the sales data at the country level, with sales, revenue and market share for key countries in the world, from 2019 to 2025.

Chapter 10 and 11, to segment the sales by type and application, with sales market share and growth rate by type, application, from 2019 to 2025.

Chapter 12, Foamed Plastics market forecast, by regions, type and application, with sales and revenue, from 2019 to 2025.

Chapter 13, 14 and 15, to describe Foamed Plastics sales channel, distributors, customers, research findings and conclusion, appendix and data source.

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CAR-T Cell Therapy Market Poised to Garner Maximum Revenues by 2027 - Health News Office

NIH and Gates Foundation to Invest Combined $200M Towards Sickle Cell and HIV Gene Therapy Research – DocWire News

The National Institutes of Health (NIH) has recently announced plans to invest at least $100 million towards the development of affordable gene therapies for sickle cell disease and HIV. An additional investment of $100 million will be provided by the Bill & Melinda Gates Foundation, with the goal of making gene therapy treatments readily available around the world.

This collaborative investment comes in the wake of President Trump announcing his goal to end the HIV epidemic in the U.S. in the next decade, a statement made during the State of the Union Address earlier this year. Ending the HIV Epidemic: A Plan for Americais designed to use new technology and data to decrease the number of HIV diagnoses made in the U.S. by 75% in the next five years and by 90% by 2030. Sickle cell disease, another condition that gene therapy applies to, has received increased attention under the Trump Administration as well.

Although recent technology has led to FDA-approved gene therapies becoming available for patients, these treatments are not readily accessible to a majority of the world and are extremely expensive. Particularly, low-income countries that are often affected by sickle cell and HIV more than wealthy countries have limited access to these treatments. To bring these treatments to regions where they are most needed, investments like those from the Gates Foundation and the NIH are imperative.

This collaborative effort sets out to advance safe, effective, and durable gene therapies to clinical trials in the U.S. and countries in sub-Saharan Africa, where disease incidence is high, in the next 10 years. The main goal of this work is to bring these transformative treatments to the underserved areas that need them the most.

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

Outside of this collaboration, the organizations will both continue to invest in other research efforts focused on treating sickle cell and HIV.

In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases, said Trevor Mundel, MD, PhD, President of the Bill & Melinda Gates Foundations Global Health Program. While these treatments are exciting, people in low- and middle-income countries do not have access to these breakthroughs. By working with the NIH and scientists across Africa, we aim to ensure these approaches will improve the lives of those most in need and bring the incredible promise of gene-based treatments to the world of public health.

This collaboration will aim to identify potential sickle cell and HIV cures for pre-clinical and clinical evaluation and to define long-term opportunities to work with African partners to advance promising treatments to late-phase clinical trials.

New delivery systems to deliver the therapies to the correct areas of the body are needed to optimize these treatments and target the cells specific to each disease. In treating sickle cell, this entails replacing mutated genes that code for a blood cell protein in hematopoietic stem cells with a healthy copy, restoring normal blood cell production. To treat HIV, the gene therapy would have to target proviral DNA that circulates in a small number of cells years after effective antiviral therapy.

Creating gene therapy treatments for these diseases that can be administered in vivo, or fully in the body, would be a significant improvement for current therapies, which involve the removal, editing, and reinfusion of cells.

We are losing too much of Africas future to sickle cell disease and HIV, said Matshidiso Rebecca Moeti, MBBS, Regional Director for Africa at the World Health Organization. Beating these diseases will take new thinking and long-term commitment. Im very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africas greatest public health challenges.

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NIH and Gates Foundation to Invest Combined $200M Towards Sickle Cell and HIV Gene Therapy Research - DocWire News

Abeona Therapeutics Announces Presentations at the 27th European Society of Gene and Cell Therapy (ESGCT) Congress – Yahoo Finance

NEW YORK and CLEVELAND, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (ABEO), a fully-integrated leader in gene and cell therapy, today announced the presentations of data from the Transpher A Study, the Companys ongoing Phase 1/2 clinical trial evaluating ABO-102 for the treatment of Sanfilippo syndrome type A (MPS IIIA), and research updates from its library of novel AIMTM adeno-associated virus (AAV) capsids at the 27th European Society of Gene and Cell Therapy (ESGCT) Congress, to be held October 22-25, 2019 in Barcelona, Spain.

The data will be presented as follows:

Safety, Tolerability, Biopotency and Neurocognitive Data of ABO-102 in Transpher A, an Open-Label, Multicenter, Single-Dose, Dose-Escalation, Phase 1/2 Clinical Trial in Sanfilippo Syndrome type A (Mucopolysaccharidosis IIIA)Oral Presentation #039Presenter: Maria Jose de Castro, M.D., Hospital Universitario Santiago de CompostelaSession 4c: Metabolic and Genetic DiseasesDate/Time: Friday, October 25, 2019, 9:00 a.m. to 11:00 a.m. CESTLocation: Room 112

Novel AAV Capsids Show Increased Evasion to Neutralizing Antibodies Against Natural SerotypesPoster #P347Session Title: Poster Session IDate/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CESTLocation: Multipurpose Hall

Development of an Improved Novel AAV Capsids for Intramuscular DeliveryPoster #P027Session Title: Poster Session IDate/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CESTLocation: Multipurpose Hall

Novel AAV Capsids for Delivery to the Retina by Intravitreal AdministrationPoster #P009Session Title: Poster Session IDate/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CESTLocation: Multipurpose Hall

Development of a Novel AAV Capsid with Improved PNS Tropism for Treating Pompe Disease by Intravenous AdministrationPoster #P007Session Title: Poster Session IDate/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CESTLocation: Multipurpose Hall

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Companys clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Companys portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM AAV vector platform to address all mutations of cystic fibrosis. Abeona has received twenty regulatory designations from the FDA and EMA for its pipeline candidates. For more information, visit http://www.abeonatherapeutics.com.

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Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Companys clinical trials and its products and product candidates, future regulatory interactions with regulatory authorities, as well as the Companys goals and objectives. We have attempted to identify forward looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Companys Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

Investor Contact:Sofia WarnerSenior Director, Investor RelationsAbeona Therapeutics+1 (646) 813-4710swarner@abeonatherapeutics.com

Media Contact:Scott SantiamoDirector, Corporate CommunicationsAbeona Therapeutics+1 (718) 344-5843ssantiamo@abeonatherapeutics.com

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Abeona Therapeutics Announces Presentations at the 27th European Society of Gene and Cell Therapy (ESGCT) Congress - Yahoo Finance

Sales of Novartis gene therapy approved in May reach $160M in Q3 – MedCity News

The second gene therapy to win approval in the U.S. saw strong sales in the third quarter of this year, the manufacturer said Tuesday.

Basel, Switzerland-based Novartis said in its third-quarter 2019 earnings that Zolgensma (onasemnogene abeparvovec-xioi) had sales of $160 million. The gene therapy was approved in May for treating spinal muscular atrophy in infants, being the second gene therapy to win Food and Drug Administration approval, after Spark Therapeutics Luxturna (voretigene neparvovec), which Novartis commercialized in the European Union and European Economic Area.

The gene therapy was launched with a list price of $2.1 million, the highest list price of any drug in the world. However, pricing watchdogs have said the price is cost-effective, given the greater costs of treatment over a patients lifetime, whereas Zolgensma is meant as a one-time therapy. A competing therapy is Biogens Spinraza (nusinersen), which has a list price of $750,000 for the first year and $375,000 every subsequent year, but must be taken for life, at $125,000 per injection.

Novartis said in its earnings statement that to date, plans are in place that pay for the therapy for around 90 percent of patients covered under commercial plans and about 30 percent of those covered by Medicaid.

Spinal muscular atrophy, or SMA, is a group of genetic diseases that cause progressive muscle weakness, usually starting in infancy, which can eventually lead to respiratory failure. The incidence of the disease is approximately 1-in-10,000 live births, according to the National Organization for Rare Disorders.

Other new therapies from the company had a strong showing as well. Another drug that received FDA approval in May, Piqray (alpelisib), saw sales of $43 million. The drug was approved as the first treatment for patients with a PIK3CA mutation in HR-positive/HER2-negative advanced breast cancer. Kymriah (tisagenlecleucel), which won FDA approval in August 2017 as the first CAR-T cell therapy, had sales of $79 million. The company reported continued strong demand, with ongoing uptake in the U.S. and Europe driving an increase in sales for the therapy, which is used to treat acute lymphoblastic leukemia in children and young adults as well as diffuse large B-cell lymphoma in adults.

Photo: jxfzsy, Getty Images

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Sales of Novartis gene therapy approved in May reach $160M in Q3 - MedCity News