Archive for the ‘Gene Therapy Research’ Category

Amgen exiting neuroscience R&D

Amgen Inc. (AMGN) announced during the 3Q-2019 earnings call conference on 10/29/2019, its exit from the neurosciences research and early development programs. The company's head of research and development, David Reese stated:

Upon careful evaluation of our pipeline and the challenges inherent in developing drugs for major neurologic diseases, we've made the decision to end our neuroscience research and early development programs with the exception of programs centered on neuro inflammation that will be pursued by our inflammation TA."

The company saw success with aimovig for migraine patients, but its study of a late-stage candidate in Alzheimer's disease in partnership with Novartis (NVS) was halted a few months back. In response to a caller's question about the neuroscience decision, David said that the company was looking at alternative models to maintain a hand in the neuroscience segment. It could potentially be with venture capital or maybe academic institutions. The company's CEO, Bob Bradway added that Amgen would capitalize on the insights from its work with deCODE in human genome sequencing. Amgen's R&D cost-cutting could be an indirect effect of its product price restructuring strategy following the results of such exercise with repatha, and aimovig, which are now available at "relatively affordable co-pay levels." According to unconfirmed reports, there will be 180 layoffs across the company, with the highest impact being at Cambridge, MA location.

In a major setback to Novartis, the U.S. FDA has suspended enrollment of new study participants in the company's phase 1 STRONG trial of zolgensma (anasemnogene abeparvovec-xioi) (AVX-101) in patients with Type 2 spinal muscular atrophy (SMA). The company has clarified that this was a partial clinical hold on the intrathecal (injection into the spinal canal) administration of the gene therapy. The hold was the result of the FDA receiving information from Novartis' subsidiary AveXis, about a preclinical animal study that showed "dorsal root ganglia (DRG) mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss." AveXis is already under investigation for data manipulation related to the company's submission for approval of zolgensma. Novartis blamed two "senior," "founder" executives at AveXis for the scandal, who have since been terminated.

Novartis further clarified that the partial clinical hold does not affect the approved use of zolgensma and IV administration.

Zolgensma competitors: risdiplam from the collaboration of Roche (OTCQX:RHHBY) and PTC Therapeutics, Inc. (PTCT), and spinraza from the collaboration of Biogen (BIIB) and Ionis Pharmaceuticals, Inc. (IONS) stand to benefit. All the four companies' stocks gained, while Regenxbio Inc. (RGNX) shares lost over 10% as the company receives milestones and royalties from zolgensma licensor AveXis.

This company's stock price touched the 52 weeks low and high: $0.9 and $3.6 respectively, in the past one week (10/25/2019 to 10/30/2019), and a gain of over 57% year-to-date. IVERIC bio, Inc. (ISEE) is a clinical-stage company developing treatments for patients with orphan, inherited retinal diseases with significant unmet medical needs. Iveric announced on 10/28/2019, initial top-line data from the randomized, controlled, phase 2b clinical trial of Zimura (avacincaptad pegol) in patients with geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). The pre-specified primary endpoint of reduction in the mean rate of GA growth over 12 months was met. Data showed statistical significance, with the reduction in the mean rate of GA growth being 27.38% (p-value = 0.0072) for the zimura 2 mg group, and 27.81% (p-value = 0.0051) for the zimura 4 mg group, as compared to the respective corresponding sham control groups. Zimura was generally well tolerated, with no zimura treatment-related inflammation or discontinuations from the trial. There also were no ocular serious adverse events (SAEs) or cases of endophthalmitis reported in the study eye. The most frequently reported ocular adverse events (AEs) were related to the injection procedure.

Zimura is a complement factor C5 inhibitor. It binds to C5, inhibiting its cleavage into terminal C5a and C5b, thereby decreasing the activation of inflammasomes and the formation of membrane attack complex (MAC). The potential of this mechanism to prevent or reduce the degeneration of retinal pigment epithelial (RPE) cells provides the basis for the zimura monotherapy in GA secondary to dry AMD, and in stargardt disease (STGD1). The company's therapeutics pipeline includes a HtrA1 inhibitor candidate in preclinical stage for GA secondary to dry AMD.

The company also has a gene therapy focused pipeline of product candidates in the preclinical stage.

(Pipeline images source: company website)

The company seems to be looking for partnerships in zimura monotherapy for both GA secondary to dry AMD and STGD1.

AbbVie (ABBV) announced positive top-line data from the SELECT-PsA 2 phase 3 study of RINVOQ (upadacitinib) in adult patients with active psoriatic arthritis who have responded inadequately to one or more biologic disease-modifying anti-rheumatic drugs (bDMARDs). The study results showed that both doses of RINVOQ: 15 mg and 30 mg, once daily, met the primary endpoint at week 12 versus placebo, with 57% on the 15 mg arm and 64% on the 30 mg arm of RINVOQ compared to 24% in the placebo arm achieving ACR20. At week 12, patients on RINVOQ treatment arms also had greater improvements in physical function, as measured by the Health Assessment Questionnaire Disability Index (HAQ-DI). At week 16, 52% of patients on 15 mg and 57% of patients on 30 mg of RINVOQ achieved a Psoriasis Area Severity Index (PASI) 75 response compared to 16% on placebo. At week 24, 25% of patients on 15 mg and 29% of patients on 30 mg RINVOQ achieved minimal disease activity (MDA) compared to 3% of the placebo group.

SELECT-PsA 2 Efficacy Results

RINVOQ 15 mg

(n=211)

RINVOQ 30 mg

(n=218)

Placebo

(n=212)

p-value

ACR20a at week 12

57%

64%

24%

p<0.0001

ACR50a at week 12

32%

38%

5%

p<0.0001

ACR70a at week 12

9%

17%

0.5%

p<0.0001

HAQ-DIb at week 12

-0.30

-0.41

-0.10

PASI 75c at week 16

52%

57%

16%

p<0.0001

MDAd at week 24

25%

29%

3%

p<0.0001

(Table source: company PR linked above)

There were no new safety signals, putting the safety profile of RINVOQ consistent with that of previous studies across indications. Discovered and developed by AbbVie, RINVOQ is a selective and reversible JAK inhibitor being studied as a once-daily therapy in psoriatic arthritis and multiple immune-mediated diseases. Psoriatic arthritis is a heterogeneous systemic inflammatory disease affecting more than 50 million people worldwide.

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Amgen Exits Neuroscience Research: The Good, Bad, And Ugly Of Biopharma - Seeking Alpha

The BLOOD CANCER DRUGS market research report added by Report Ocean, is an in-depth analysis of the latest trends, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The research study provides market introduction, BLOOD CANCER DRUGS market definition, regional market scope, sales and revenue by region, manufacturing cost analysis, Industrial Chain, market effect factors analysis, BLOOD CANCER DRUGS market size forecast, 100+ market data, Tables, Pie Chart, Graphs and Figures, and many more for business intelligence.

The global blood cancer drugs market is anticipated to reach USD 55.6 billion by 2025.

UPTO 30% OFF ON SINGLE USER PDF: https://www.reportocean.com/industry-verticals/sample-request.php?report_id=5256

In the BLOOD CANCER DRUGS Market, some of the major companies are:

Johnson & Johnson Inc., Amgen Inc., Bayer AG., Pfizer, Inc., AbbVie Inc., Roche Holding AG., Celgene Corporation, AstraZeneca, Novartis AG, GlaxoSmithKline PLC, Merck & Co., Inc., and Eli Lily & Co. among others.

The report consists of various chapters and company profiling is a major among them. Company profiling garners business intelligence and track key elements of a business, such as:

BLOOD CANCER DRUGS Market: Insights

The global blood cancer drugs market is anticipated to reach USD 55.6 billion by 2025. The demand for blood cancer drug is primarily driven by growing death incidences by blood cancer, and continuous innovation for developing novel treatments with the help of several ongoing clinical trials. Moreover, increasing research and development of biological and targeted therapies as treatment will spur the blood cancer drugs market during the upcoming period. However, the high price of drugs and the stringent government policies will limit the growth of blood cancer drugs market during the forecast period.

Most of the blood cancers start in the bone marrow, where blood is produced. In blood cancer the growth of normal blood cells is dislodged by the uncontrollable growth of abnormal blood cells. These cancerous cells prevent the blood from performing many of its functions. Hence, the existing treatments of blood cancer are being the foundation for developing the new drugs. The steady flow of the blood cancer drugs has created opportunity for research and development in the existing market. For instance, Amgen Inc. received approval for BLINCYTO in July 2017, which is used in treating B-cell precursor Acute Lymphoblastic leukemia. Similarly, European blood cancer drugs market witnessed the approvals of Gazyvaro, by Roche AG that is used in treating advanced follicular lymphoma. Also, novel technologies like CAR-T are likely to be launched this year.

The global blood cancer drugs market is segmented into blood cancer type, drugs and treatment approaches. On the basis of blood cancer type, the global blood cancer drugs market is segmented into leukemia, lymphoma and myeloma. The lymphoma segment is expected to drive the majority market of blood cancer drugs followed by leukemia. The global market of this segment is primarily driven by the increasing prevalence of lymphoma, and presence of effective treatments in the market. On the basis of drugs, the global blood cancer drugs market is further categorized into Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others. This continuous innovation for treating various sub-types of blood cancers has led to the development of novel types of treatments. For instance, the combination of Revlimid and Velcade has emerged as the preferential drugs in trials for treating multiple myeloma.

On the basis of treatment approaches the global blood cancer drugs market is further segmented into Chemotherapeutic, mAbs/Targeted Therapies, and Immunotherapeutic. Due to availability of variety of chemotherapeutic agent in the market chemotherapeutic drugs are expected to hold the higher share in blood cancer drugs market. Moreover, their effectiveness for the treatment of blood cancer and increasing number of cancer patients globally has garnered more demand for chemotherapeutic drugs throughout the world.

Geographically, the global blood cancer drugs market is segmented into North America, Europe, Asia Pacific, and the rest of the world. North America dominates the blood cancer drugs market which is followed by Europe and Asia Pacific. Favorable reimbursement policies, surge in R&D investments of various companies, as well as the increase in the number of blood cancer treatments are some of the major factors responsible for the growth of North Americas blood cancer drugs market. Moreover, Asia-Pacific region has been identified as the lucrative market for the for blood cancer drugs due to increasing awareness of the use of these drugs, increased healthcare expenditure, and rising per capita disposable income. These are some of the major factors which are influencing the growth of the blood cancer drugs in Asia-pacific region.

The leading companies operating in this industry include Johnson & Johnson Inc., Amgen Inc., Bayer AG., Pfizer, Inc., AbbVie Inc., Roche Holding AG., Celgene Corporation, AstraZeneca, Novartis AG, GlaxoSmithKline PLC, Merck & Co., Inc., and Eli Lily & Co. among others.

Key Findings from the study suggest blood cancer drugs in the market are much innovative and manufacturers are progressively concentrating on innovation of combination drugs. Companies are in a stage of development of new drugs in order to provide novel treatments for blood cancer. The immunotherapy segment is anticipated to grow at a high growth rate over the forecast period. The growth of this segment is primarily driven by increased awareness for its use as an alternative and effective treatment for blood cancer. North America is presumed to dominate the global blood cancer drugs market over the forecast period. Asia Pacific region which shows signs of high growth potential owing to the booming economies of India, and China.

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The Global BLOOD CANCER DRUGS Market is segmented into various sub-groups to understand the market scenario in detail, the market segmentation are as follows:

[By Blood Cancer Type (Leukemia (Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia), Lymphoma (Hodgkin Lymphoma, Non-Hodgkin Lymphoma (B-Cell Lymphoma, T-Cell Lymphoma)), and Myeloma; By Drugs (Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others); By Treatment Approaches (Chemotherapeutic, mAbs/Targeted Therapies, and Immunotherapeutic); By Region]

Furthermore, the years considered for the study are as follows:

Historical year 2013-2017

Base year 2018

Forecast period** 2019 to 2025 [** unless otherwise stated]

Regional split of the Global BLOOD CANCER DRUGS Market research report is as follows:

The market research study offers in-depth regional analysis along with the current market scenarios. The major regions analyzed in the study are:

Key highlights and important features of the Report:

Overview and highlights of product and application segments of the global BLOOD CANCER DRUGS Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Explore about Sales data of key players of the global BLOOD CANCER DRUGS Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the BLOOD CANCER DRUGS Market.

Explore about gross margin, sales, revenue, production, market share, CAGR, and market size by region.

Describe BLOOD CANCER DRUGS Market Findings and Conclusion, Appendix, methodology and data source;

Research Methodology:

The market research was done by adopting various tools under the category of primary and secondary research. For primary research, experts and major sources of information have been interviewed from suppliers side and industries, to obtain and verify the data related to the study of the Global BLOOD CANCER DRUGS Market. In secondary research methodology, various secondary sources were referred to collect and identify extensive piece of information, such as paid databases, directories and annual reports and databases for commercial study of the Global BLOOD CANCER DRUGS Market. Moreover, other secondary sources include studying technical papers, news releases, government websites, product literatures, white papers, and other literatures to research the market in detail.

Browse Premium Research Report with Tables and Figures at @ https://www.reportocean.com/industry-verticals/details?report_id=5256

There are 15 Chapters to display the Global BLOOD CANCER DRUGS Market:

Chapter 1, to describe Definition, Specifications and Classification of Global BLOOD CANCER DRUGS, Applications of, Market Segment by Regions;Chapter 2, to analyze the Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;Chapter 3, to display the Technical Data and Manufacturing Plants Analysis of , Capacity and Commercial Production Date, Manufacturing Plants Distribution, Export & Import, R&D Status and Technology Source, Raw Materials Sources Analysis;Chapter 4, to show the Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);Chapter 5 and 6, to show the Regional Market Analysis that includes United States, EU, Japan, China, India & Southeast Asia, Segment Market Analysis (by Type);Chapter 7 and 8, to explore the Market Analysis by Application Major Manufacturers Analysis;Chapter 9, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type, Market Trend by Application;Chapter 10, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;Chapter 11, to analyze the Consumers Analysis of Global BLOOD CANCER DRUGS by region, type and application;Chapter 12, to describe BLOOD CANCER DRUGS Research Findings and Conclusion, Appendix, methodology and data source;Chapter 13, 14 and 15, to describe BLOOD CANCER DRUGS sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

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Cancer Gene Therapy Market Research, Insights, Revenue and Forecast by 2023 - Health News Office

CAMBRIDGE, Mass., Oct. 31, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), reported operational highlights and financial results for the third quarter ended September 30, 2019.

In 2019, we continued to leverage the breadth of our genome editing platform to advance our in vivo and engineered cell therapy programs. We have demonstrated that we can knock out a disease-causing gene as well as introduce a functional gene to restore normal protein production. Now, we have achieved consecutive editing in vivo by combining both these edit types, further highlighting the versatility of our modular platform, said Intellia President and Chief Executive Officer, John Leonard, M.D. Our full-spectrum strategy and platform capabilities are enabling Intellias development of a robust pipeline to address a range of severe diseases. We look forward to the planned nomination of our first engineered cell therapy development candidate for acute myeloid leukemia by year-end and the submission of our first IND application for NTLA-2001 for the treatment of transthyretin amyloidosis in mid-2020.

Third Quarter 2019 and More Recent Operational Highlights

Upcoming Milestones

The Company has set forth the following for pipeline progression:

Upcoming Events

The Company will participate in the following investor events:

Third Quarter 2019 Financial Results

Financial Guidance

Intellia expects that its cash, cash equivalents and marketable securities as of September 30, 2019, as well as technology access and funding from Novartis and Regeneron, will enable Intellia to fund its anticipated operating expenses and capital expenditure requirements into the second half of 2021. This expectation excludes any potential milestone payments or extension fees that could be earned and distributed under the collaboration agreements withNovartisand Regeneron or any strategic use of capital not currently in the Companys base-case planning assumptions.

Conference Call to Discuss Third Quarter 2019 Earnings

The Company will discuss these results on a conference call today, October 31, 2019, at 8 a.m. ET.

To join the call:

A replay of the call will be available through the Events and Presentations page of the Investor Relations section on Intellias website, beginning on October 31, 2019 at 12 p.m. ET.

About Intellia Therapeutics

Intellia Therapeutics is 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 about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements of Intellia 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 investigational new drug (IND) application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020; its plans to nominate a first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (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 other in vivo and ex vivo programs; 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, 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 other in vivo orex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the ability to continue its growth and realize the anticipated contribution of the members of its board of directors and executives to its operations and progress; the impact of its collaborations on its development programs, including but not limited to its collaborations with Regeneron Pharmaceuticals, Inc. and Novartis Institutes for BioMedical Research; statements regarding the timing of regulatory filings regarding its development programs; its use of capital, including ATM receivables, expenses, future accumulated deficit and other financial results during the third quarter of 2019; 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 or clinical studies will not be predictive of future results in connection with future studies; the risk that Novartis will not continue to pursue programs it has selected through its collaboration with Intellia; and the risk that Intellias collaborations withNovartisor Regeneron or its otherex vivo collaborations 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:

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

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

Continue reading here:
Intellia Therapeutics Announces Third Quarter 2019 Financial Results - GlobeNewswire

We will be contrasting the differences between Proteostasis Therapeutics Inc. (NASDAQ:PTI) and Abeona Therapeutics Inc. (NASDAQ:ABEO) as far as dividends, analyst recommendations, profitability, risk, institutional ownership, earnings and valuation are concerned. The two businesses are rivals in the Biotechnology industry.

Earnings & Valuation

Table 1 shows the gross revenue, earnings per share and valuation for Proteostasis Therapeutics Inc. and Abeona Therapeutics Inc.

Profitability

Table 2 provides us Proteostasis Therapeutics Inc. and Abeona Therapeutics Inc.s return on equity, return on assets and net margins.

Volatility and Risk

Proteostasis Therapeutics Inc.s volatility measures that its 171.00% less volatile than Standard and Poors 500 due to its -0.71 beta. From a competition point of view, Abeona Therapeutics Inc. has a 1.84 beta which is 84.00% more volatile compared to Standard and Poors 500.

Liquidity

The Current Ratio and Quick Ratio of Proteostasis Therapeutics Inc. are 11.3 and 11.3 respectively. Its competitor Abeona Therapeutics Inc.s Current Ratio is 3.3 and its Quick Ratio is 3.3. Proteostasis Therapeutics Inc. can pay off short and long-term obligations better than Abeona Therapeutics Inc.

Analyst Recommendations

The next table highlights the given recommendations and ratings for Proteostasis Therapeutics Inc. and Abeona Therapeutics Inc.

Abeona Therapeutics Inc. on the other hand boasts of a $12.33 consensus target price and a 387.35% potential upside.

Institutional & Insider Ownership

Roughly 60.5% of Proteostasis Therapeutics Inc. shares are owned by institutional investors while 64.4% of Abeona Therapeutics Inc. are owned by institutional investors. Insiders owned roughly 0.3% of Proteostasis Therapeutics Inc.s shares. Competitively, 0.3% are Abeona Therapeutics Inc.s share owned by insiders.

Performance

Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Proteostasis Therapeutics Inc.s stock price has bigger decline than Abeona Therapeutics Inc.

Summary

Abeona Therapeutics Inc. beats on 6 of the 10 factors Proteostasis Therapeutics Inc.

Proteostasis Therapeutics, Inc., a biopharmaceutical company, engages in the discovery and development of novel therapeutics that treat diseases caused by dysfunctional protein processing, such as cystic fibrosis. Its lead product candidate is PTI-428, an orally bioavailable cystic fibrosis transmembrane conductance regulator modulator belonging to the amplifier class that is in Phase-I studies. The company is also developing PTI-801, a corrector molecule; PTI-808, a potentiator molecule; and unfolded protein response (UPR) modulators that are in preclinical development. It has collaboration with Astellas Pharma, Inc. to research and identify therapies targeting the Unfolded Protein Response (UPR) pathway. The company was formerly known as Proteoguard, Inc. and changed its name to Proteostasis Therapeutics, Inc. in September 2007. Proteostasis Therapeutics, Inc. was founded in 2006 and is headquartered in Cambridge, Massachusetts.

Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. The companys lead programs are ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B; and ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A. It is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); EB-201 for for epidermolysis bullosa (EB); ABO-201 gene therapy for juvenile Batten disease; ABO-202 gene therapy for treatment of infantile Batten disease; ABO-301, an AAV-based gene therapy for Fanconi anemia disorder; and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. In addition, the company is developing plasma-based protein therapy pipeline, including SDF Alpha, an alpha-1 protease inhibitor for inherited COPD using its proprietary salt diafiltration ethanol-free process. Further, it is involved in marketing MuGard, a mucoadhesive oral wound rinse for the management of mucositis, stomatitis, aphthous ulcers, and traumatic ulcers. Abeona Therapeutics Inc. has collaborations with EB Research Partnership and Epidermolysis Bullosa Medical Research Foundation that focus on gene therapy treatments for EB; and Brammer Bio for commercial translation of ABO-102. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.

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Reviewing Proteostasis Therapeutics Inc. (PTI)'s and Abeona Therapeutics Inc. (NASDAQ:ABEO)'s results - MS Wkly

The BLOOD CANCER DRUGS market research report added by Report Ocean, is an in-depth analysis of the latest trends, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The research study provides market introduction, BLOOD CANCER DRUGS market definition, regional market scope, sales and revenue by region, manufacturing cost analysis, Industrial Chain, market effect factors analysis, BLOOD CANCER DRUGS market size forecast, 100+ market data, Tables, Pie Chart, Graphs and Figures, and many more for business intelligence.

The global blood cancer drugs market is anticipated to reach USD 55.6 billion by 2025.

UPTO 30% OFF ON SINGLE USER PDF: https://www.reportocean.com/industry-verticals/sample-request.php?report_id=5256

In the BLOOD CANCER DRUGS Market, some of the major companies are:

Johnson & Johnson Inc., Amgen Inc., Bayer AG., Pfizer, Inc., AbbVie Inc., Roche Holding AG., Celgene Corporation, AstraZeneca, Novartis AG, GlaxoSmithKline PLC, Merck & Co., Inc., and Eli Lily & Co. among others.

The report consists of various chapters and company profiling is a major among them. Company profiling garners business intelligence and track key elements of a business, such as:

BLOOD CANCER DRUGS Market: Insights

The global blood cancer drugs market is anticipated to reach USD 55.6 billion by 2025. The demand for blood cancer drug is primarily driven by growing death incidences by blood cancer, and continuous innovation for developing novel treatments with the help of several ongoing clinical trials. Moreover, increasing research and development of biological and targeted therapies as treatment will spur the blood cancer drugs market during the upcoming period. However, the high price of drugs and the stringent government policies will limit the growth of blood cancer drugs market during the forecast period.

Most of the blood cancers start in the bone marrow, where blood is produced. In blood cancer the growth of normal blood cells is dislodged by the uncontrollable growth of abnormal blood cells. These cancerous cells prevent the blood from performing many of its functions. Hence, the existing treatments of blood cancer are being the foundation for developing the new drugs. The steady flow of the blood cancer drugs has created opportunity for research and development in the existing market. For instance, Amgen Inc. received approval for BLINCYTO in July 2017, which is used in treating B-cell precursor Acute Lymphoblastic leukemia. Similarly, European blood cancer drugs market witnessed the approvals of Gazyvaro, by Roche AG that is used in treating advanced follicular lymphoma. Also, novel technologies like CAR-T are likely to be launched this year.

The global blood cancer drugs market is segmented into blood cancer type, drugs and treatment approaches. On the basis of blood cancer type, the global blood cancer drugs market is segmented into leukemia, lymphoma and myeloma. The lymphoma segment is expected to drive the majority market of blood cancer drugs followed by leukemia. The global market of this segment is primarily driven by the increasing prevalence of lymphoma, and presence of effective treatments in the market. On the basis of drugs, the global blood cancer drugs market is further categorized into Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others. This continuous innovation for treating various sub-types of blood cancers has led to the development of novel types of treatments. For instance, the combination of Revlimid and Velcade has emerged as the preferential drugs in trials for treating multiple myeloma.

On the basis of treatment approaches the global blood cancer drugs market is further segmented into Chemotherapeutic, mAbs/Targeted Therapies, and Immunotherapeutic. Due to availability of variety of chemotherapeutic agent in the market chemotherapeutic drugs are expected to hold the higher share in blood cancer drugs market. Moreover, their effectiveness for the treatment of blood cancer and increasing number of cancer patients globally has garnered more demand for chemotherapeutic drugs throughout the world.

Geographically, the global blood cancer drugs market is segmented into North America, Europe, Asia Pacific, and the rest of the world. North America dominates the blood cancer drugs market which is followed by Europe and Asia Pacific. Favorable reimbursement policies, surge in R&D investments of various companies, as well as the increase in the number of blood cancer treatments are some of the major factors responsible for the growth of North Americas blood cancer drugs market. Moreover, Asia-Pacific region has been identified as the lucrative market for the for blood cancer drugs due to increasing awareness of the use of these drugs, increased healthcare expenditure, and rising per capita disposable income. These are some of the major factors which are influencing the growth of the blood cancer drugs in Asia-pacific region.

The leading companies operating in this industry include Johnson & Johnson Inc., Amgen Inc., Bayer AG., Pfizer, Inc., AbbVie Inc., Roche Holding AG., Celgene Corporation, AstraZeneca, Novartis AG, GlaxoSmithKline PLC, Merck & Co., Inc., and Eli Lily & Co. among others.

Key Findings from the study suggest blood cancer drugs in the market are much innovative and manufacturers are progressively concentrating on innovation of combination drugs. Companies are in a stage of development of new drugs in order to provide novel treatments for blood cancer. The immunotherapy segment is anticipated to grow at a high growth rate over the forecast period. The growth of this segment is primarily driven by increased awareness for its use as an alternative and effective treatment for blood cancer. North America is presumed to dominate the global blood cancer drugs market over the forecast period. Asia Pacific region which shows signs of high growth potential owing to the booming economies of India, and China.

Get a Sample Report for more Expert and Official insights: @https://www.reportocean.com/industry-verticals/sample-request.php?report_id=5256

The Global BLOOD CANCER DRUGS Market is segmented into various sub-groups to understand the market scenario in detail, the market segmentation are as follows:

[By Blood Cancer Type (Leukemia (Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia), Lymphoma (Hodgkin Lymphoma, Non-Hodgkin Lymphoma (B-Cell Lymphoma, T-Cell Lymphoma)), and Myeloma; By Drugs (Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others); By Treatment Approaches (Chemotherapeutic, mAbs/Targeted Therapies, and Immunotherapeutic); By Region]

Furthermore, the years considered for the study are as follows:

Historical year 2013-2017

Base year 2018

Forecast period** 2019 to 2025 [** unless otherwise stated]

Regional split of the Global BLOOD CANCER DRUGS Market research report is as follows:

The market research study offers in-depth regional analysis along with the current market scenarios. The major regions analyzed in the study are:

Key highlights and important features of the Report:

Overview and highlights of product and application segments of the global BLOOD CANCER DRUGS Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Explore about Sales data of key players of the global BLOOD CANCER DRUGS Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the BLOOD CANCER DRUGS Market.

Explore about gross margin, sales, revenue, production, market share, CAGR, and market size by region.

Describe BLOOD CANCER DRUGS Market Findings and Conclusion, Appendix, methodology and data source;

Research Methodology:

The market research was done by adopting various tools under the category of primary and secondary research. For primary research, experts and major sources of information have been interviewed from suppliers side and industries, to obtain and verify the data related to the study of the Global BLOOD CANCER DRUGS Market. In secondary research methodology, various secondary sources were referred to collect and identify extensive piece of information, such as paid databases, directories and annual reports and databases for commercial study of the Global BLOOD CANCER DRUGS Market. Moreover, other secondary sources include studying technical papers, news releases, government websites, product literatures, white papers, and other literatures to research the market in detail.

Browse Premium Research Report with Tables and Figures at @ https://www.reportocean.com/industry-verticals/details?report_id=5256

There are 15 Chapters to display the Global BLOOD CANCER DRUGS Market:

Chapter 1, to describe Definition, Specifications and Classification of Global BLOOD CANCER DRUGS, Applications of, Market Segment by Regions;Chapter 2, to analyze the Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;Chapter 3, to display the Technical Data and Manufacturing Plants Analysis of , Capacity and Commercial Production Date, Manufacturing Plants Distribution, Export & Import, R&D Status and Technology Source, Raw Materials Sources Analysis;Chapter 4, to show the Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);Chapter 5 and 6, to show the Regional Market Analysis that includes United States, EU, Japan, China, India & Southeast Asia, Segment Market Analysis (by Type);Chapter 7 and 8, to explore the Market Analysis by Application Major Manufacturers Analysis;Chapter 9, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type, Market Trend by Application;Chapter 10, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;Chapter 11, to analyze the Consumers Analysis of Global BLOOD CANCER DRUGS by region, type and application;Chapter 12, to describe BLOOD CANCER DRUGS Research Findings and Conclusion, Appendix, methodology and data source;Chapter 13, 14 and 15, to describe BLOOD CANCER DRUGS sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

About Report Ocean:

We are the best market research reports provider in the industry. Report Ocean believe in providing the quality reports to clients to meet the top line and bottom line goals which will boost your market share in todays competitive environment. Report Ocean is one-stop solution for individuals, organizations, and industries that are looking for innovative market research reports.

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In Vitro Fertilization (IVF) Services Market to Witness Exponential Growth by 2023 - Health News Office

The BLOOD CANCER DRUGS market research report added by Report Ocean, is an in-depth analysis of the latest trends, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The research study provides market introduction, BLOOD CANCER DRUGS market definition, regional market scope, sales and revenue by region, manufacturing cost analysis, Industrial Chain, market effect factors analysis, BLOOD CANCER DRUGS market size forecast, 100+ market data, Tables, Pie Chart, Graphs and Figures, and many more for business intelligence.

The global blood cancer drugs market is anticipated to reach USD 55.6 billion by 2025.

UPTO 30% OFF ON SINGLE USER PDF: https://www.reportocean.com/industry-verticals/sample-request.php?report_id=5256

In the BLOOD CANCER DRUGS Market, some of the major companies are:

Johnson & Johnson Inc., Amgen Inc., Bayer AG., Pfizer, Inc., AbbVie Inc., Roche Holding AG., Celgene Corporation, AstraZeneca, Novartis AG, GlaxoSmithKline PLC, Merck & Co., Inc., and Eli Lily & Co. among others.

The report consists of various chapters and company profiling is a major among them. Company profiling garners business intelligence and track key elements of a business, such as:

BLOOD CANCER DRUGS Market: Insights

The global blood cancer drugs market is anticipated to reach USD 55.6 billion by 2025. The demand for blood cancer drug is primarily driven by growing death incidences by blood cancer, and continuous innovation for developing novel treatments with the help of several ongoing clinical trials. Moreover, increasing research and development of biological and targeted therapies as treatment will spur the blood cancer drugs market during the upcoming period. However, the high price of drugs and the stringent government policies will limit the growth of blood cancer drugs market during the forecast period.

Most of the blood cancers start in the bone marrow, where blood is produced. In blood cancer the growth of normal blood cells is dislodged by the uncontrollable growth of abnormal blood cells. These cancerous cells prevent the blood from performing many of its functions. Hence, the existing treatments of blood cancer are being the foundation for developing the new drugs. The steady flow of the blood cancer drugs has created opportunity for research and development in the existing market. For instance, Amgen Inc. received approval for BLINCYTO in July 2017, which is used in treating B-cell precursor Acute Lymphoblastic leukemia. Similarly, European blood cancer drugs market witnessed the approvals of Gazyvaro, by Roche AG that is used in treating advanced follicular lymphoma. Also, novel technologies like CAR-T are likely to be launched this year.

The global blood cancer drugs market is segmented into blood cancer type, drugs and treatment approaches. On the basis of blood cancer type, the global blood cancer drugs market is segmented into leukemia, lymphoma and myeloma. The lymphoma segment is expected to drive the majority market of blood cancer drugs followed by leukemia. The global market of this segment is primarily driven by the increasing prevalence of lymphoma, and presence of effective treatments in the market. On the basis of drugs, the global blood cancer drugs market is further categorized into Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others. This continuous innovation for treating various sub-types of blood cancers has led to the development of novel types of treatments. For instance, the combination of Revlimid and Velcade has emerged as the preferential drugs in trials for treating multiple myeloma.

On the basis of treatment approaches the global blood cancer drugs market is further segmented into Chemotherapeutic, mAbs/Targeted Therapies, and Immunotherapeutic. Due to availability of variety of chemotherapeutic agent in the market chemotherapeutic drugs are expected to hold the higher share in blood cancer drugs market. Moreover, their effectiveness for the treatment of blood cancer and increasing number of cancer patients globally has garnered more demand for chemotherapeutic drugs throughout the world.

Geographically, the global blood cancer drugs market is segmented into North America, Europe, Asia Pacific, and the rest of the world. North America dominates the blood cancer drugs market which is followed by Europe and Asia Pacific. Favorable reimbursement policies, surge in R&D investments of various companies, as well as the increase in the number of blood cancer treatments are some of the major factors responsible for the growth of North Americas blood cancer drugs market. Moreover, Asia-Pacific region has been identified as the lucrative market for the for blood cancer drugs due to increasing awareness of the use of these drugs, increased healthcare expenditure, and rising per capita disposable income. These are some of the major factors which are influencing the growth of the blood cancer drugs in Asia-pacific region.

The leading companies operating in this industry include Johnson & Johnson Inc., Amgen Inc., Bayer AG., Pfizer, Inc., AbbVie Inc., Roche Holding AG., Celgene Corporation, AstraZeneca, Novartis AG, GlaxoSmithKline PLC, Merck & Co., Inc., and Eli Lily & Co. among others.

Key Findings from the study suggest blood cancer drugs in the market are much innovative and manufacturers are progressively concentrating on innovation of combination drugs. Companies are in a stage of development of new drugs in order to provide novel treatments for blood cancer. The immunotherapy segment is anticipated to grow at a high growth rate over the forecast period. The growth of this segment is primarily driven by increased awareness for its use as an alternative and effective treatment for blood cancer. North America is presumed to dominate the global blood cancer drugs market over the forecast period. Asia Pacific region which shows signs of high growth potential owing to the booming economies of India, and China.

Get a Sample Report for more Expert and Official insights: @https://www.reportocean.com/industry-verticals/sample-request.php?report_id=5256

The Global BLOOD CANCER DRUGS Market is segmented into various sub-groups to understand the market scenario in detail, the market segmentation are as follows:

[By Blood Cancer Type (Leukemia (Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia), Lymphoma (Hodgkin Lymphoma, Non-Hodgkin Lymphoma (B-Cell Lymphoma, T-Cell Lymphoma)), and Myeloma; By Drugs (Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others); By Treatment Approaches (Chemotherapeutic, mAbs/Targeted Therapies, and Immunotherapeutic); By Region]

Furthermore, the years considered for the study are as follows:

Historical year 2013-2017

Base year 2018

Forecast period** 2019 to 2025 [** unless otherwise stated]

Regional split of the Global BLOOD CANCER DRUGS Market research report is as follows:

The market research study offers in-depth regional analysis along with the current market scenarios. The major regions analyzed in the study are:

Key highlights and important features of the Report:

Overview and highlights of product and application segments of the global BLOOD CANCER DRUGS Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Explore about Sales data of key players of the global BLOOD CANCER DRUGS Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the BLOOD CANCER DRUGS Market.

Explore about gross margin, sales, revenue, production, market share, CAGR, and market size by region.

Describe BLOOD CANCER DRUGS Market Findings and Conclusion, Appendix, methodology and data source;

Research Methodology:

The market research was done by adopting various tools under the category of primary and secondary research. For primary research, experts and major sources of information have been interviewed from suppliers side and industries, to obtain and verify the data related to the study of the Global BLOOD CANCER DRUGS Market. In secondary research methodology, various secondary sources were referred to collect and identify extensive piece of information, such as paid databases, directories and annual reports and databases for commercial study of the Global BLOOD CANCER DRUGS Market. Moreover, other secondary sources include studying technical papers, news releases, government websites, product literatures, white papers, and other literatures to research the market in detail.

Browse Premium Research Report with Tables and Figures at @ https://www.reportocean.com/industry-verticals/details?report_id=5256

There are 15 Chapters to display the Global BLOOD CANCER DRUGS Market:

Chapter 1, to describe Definition, Specifications and Classification of Global BLOOD CANCER DRUGS, Applications of, Market Segment by Regions;Chapter 2, to analyze the Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;Chapter 3, to display the Technical Data and Manufacturing Plants Analysis of , Capacity and Commercial Production Date, Manufacturing Plants Distribution, Export & Import, R&D Status and Technology Source, Raw Materials Sources Analysis;Chapter 4, to show the Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);Chapter 5 and 6, to show the Regional Market Analysis that includes United States, EU, Japan, China, India & Southeast Asia, Segment Market Analysis (by Type);Chapter 7 and 8, to explore the Market Analysis by Application Major Manufacturers Analysis;Chapter 9, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type, Market Trend by Application;Chapter 10, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;Chapter 11, to analyze the Consumers Analysis of Global BLOOD CANCER DRUGS by region, type and application;Chapter 12, to describe BLOOD CANCER DRUGS Research Findings and Conclusion, Appendix, methodology and data source;Chapter 13, 14 and 15, to describe BLOOD CANCER DRUGS sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

About Report Ocean:

We are the best market research reports provider in the industry. Report Ocean believe in providing the quality reports to clients to meet the top line and bottom line goals which will boost your market share in todays competitive environment. Report Ocean is one-stop solution for individuals, organizations, and industries that are looking for innovative market research reports.

Get in Touch with Us:

Name: Varda

Email: [emailprotected]

Address: Classic Tower, Rajnagar Extension, Ghaziabad, 201017 India

Tel: +1 888 212 3539 (US TOLL FREE)

Website: https://www.reportocean.com/

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Designer and Luxury Footwear Market to Witness Robust Expansion throughout the Forecast 2018-2023 - Health News Office

Analysts predict that Axovant Gene Therapies Ltd (NASDAQ:AXGT) will announce earnings per share (EPS) of ($1.15) for the current quarter, according to Zacks. Zero analysts have made estimates for Axovant Gene Therapies earnings. The lowest EPS estimate is ($1.39) and the highest is ($0.76). Axovant Gene Therapies posted earnings per share of ($2.24) during the same quarter last year, which indicates a positive year-over-year growth rate of 48.7%. The company is scheduled to report its next quarterly earnings results on Wednesday, November 6th.

According to Zacks, analysts expect that Axovant Gene Therapies will report full-year earnings of ($4.25) per share for the current fiscal year, with EPS estimates ranging from ($5.59) to ($3.47). For the next fiscal year, analysts expect that the company will report earnings of ($3.32) per share, with EPS estimates ranging from ($4.35) to ($2.89). Zacks earnings per share calculations are an average based on a survey of sell-side analysts that cover Axovant Gene Therapies.

Axovant Gene Therapies (NASDAQ:AXGT) last issued its quarterly earnings data on Friday, August 9th. The company reported ($1.23) EPS for the quarter, topping the consensus estimate of ($1.34) by $0.11.

Several large investors have recently added to or reduced their stakes in AXGT. BlackRock Inc. acquired a new position in shares of Axovant Gene Therapies in the second quarter valued at approximately $1,482,000. Marshall Wace LLP acquired a new position in Axovant Gene Therapies during the first quarter worth $272,000. Jane Street Group LLC grew its holdings in Axovant Gene Therapies by 28.8% during the second quarter. Jane Street Group LLC now owns 46,455 shares of the companys stock worth $289,000 after acquiring an additional 10,375 shares during the period. Finally, Tower Research Capital LLC TRC grew its holdings in Axovant Gene Therapies by 955.3% during the second quarter. Tower Research Capital LLC TRC now owns 4,221 shares of the companys stock worth $27,000 after acquiring an additional 3,821 shares during the period. 13.76% of the stock is currently owned by institutional investors and hedge funds.

Axovant Gene Therapies stock traded down $0.01 during mid-day trading on Thursday, reaching $6.29. 38,700 shares of the companys stock were exchanged, compared to its average volume of 118,874. The company has a fifty day moving average of $6.55 and a 200-day moving average of $5.80. The company has a quick ratio of 1.70, a current ratio of 1.70 and a debt-to-equity ratio of 0.60. The company has a market cap of $143.40 million, a P/E ratio of -0.78 and a beta of 1.25. Axovant Gene Therapies has a twelve month low of $3.81 and a twelve month high of $19.60.

About Axovant Gene Therapies

Axovant Gene Therapies Ltd., a clinical-stage gene therapy company, focuses on developing a pipeline of product candidates for debilitating neurological and neuromuscular diseases. The company's current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis, Parkinson's disease, oculopharyngeal muscular dystrophy, amyotrophic lateral sclerosis, and frontotemporal dementia.

Recommended Story: What are earnings reports?

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Axovant Gene Therapies Ltd (NASDAQ:AXGT) Expected to Post Earnings of -$1.15 Per Share - Mitchell Messenger

The BLOOD CANCER DRUGS market research report added by Report Ocean, is an in-depth analysis of the latest trends, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The research study provides market introduction, BLOOD CANCER DRUGS market definition, regional market scope, sales and revenue by region, manufacturing cost analysis, Industrial Chain, market effect factors analysis, BLOOD CANCER DRUGS market size forecast, 100+ market data, Tables, Pie Chart, Graphs and Figures, and many more for business intelligence.

The global blood cancer drugs market is anticipated to reach USD 55.6 billion by 2025.

UPTO 30% OFF ON SINGLE USER PDF: https://www.reportocean.com/industry-verticals/sample-request.php?report_id=5256

In the BLOOD CANCER DRUGS Market, some of the major companies are:

Johnson & Johnson Inc., Amgen Inc., Bayer AG., Pfizer, Inc., AbbVie Inc., Roche Holding AG., Celgene Corporation, AstraZeneca, Novartis AG, GlaxoSmithKline PLC, Merck & Co., Inc., and Eli Lily & Co. among others.

The report consists of various chapters and company profiling is a major among them. Company profiling garners business intelligence and track key elements of a business, such as:

BLOOD CANCER DRUGS Market: Insights

The global blood cancer drugs market is anticipated to reach USD 55.6 billion by 2025. The demand for blood cancer drug is primarily driven by growing death incidences by blood cancer, and continuous innovation for developing novel treatments with the help of several ongoing clinical trials. Moreover, increasing research and development of biological and targeted therapies as treatment will spur the blood cancer drugs market during the upcoming period. However, the high price of drugs and the stringent government policies will limit the growth of blood cancer drugs market during the forecast period.

Most of the blood cancers start in the bone marrow, where blood is produced. In blood cancer the growth of normal blood cells is dislodged by the uncontrollable growth of abnormal blood cells. These cancerous cells prevent the blood from performing many of its functions. Hence, the existing treatments of blood cancer are being the foundation for developing the new drugs. The steady flow of the blood cancer drugs has created opportunity for research and development in the existing market. For instance, Amgen Inc. received approval for BLINCYTO in July 2017, which is used in treating B-cell precursor Acute Lymphoblastic leukemia. Similarly, European blood cancer drugs market witnessed the approvals of Gazyvaro, by Roche AG that is used in treating advanced follicular lymphoma. Also, novel technologies like CAR-T are likely to be launched this year.

The global blood cancer drugs market is segmented into blood cancer type, drugs and treatment approaches. On the basis of blood cancer type, the global blood cancer drugs market is segmented into leukemia, lymphoma and myeloma. The lymphoma segment is expected to drive the majority market of blood cancer drugs followed by leukemia. The global market of this segment is primarily driven by the increasing prevalence of lymphoma, and presence of effective treatments in the market. On the basis of drugs, the global blood cancer drugs market is further categorized into Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others. This continuous innovation for treating various sub-types of blood cancers has led to the development of novel types of treatments. For instance, the combination of Revlimid and Velcade has emerged as the preferential drugs in trials for treating multiple myeloma.

On the basis of treatment approaches the global blood cancer drugs market is further segmented into Chemotherapeutic, mAbs/Targeted Therapies, and Immunotherapeutic. Due to availability of variety of chemotherapeutic agent in the market chemotherapeutic drugs are expected to hold the higher share in blood cancer drugs market. Moreover, their effectiveness for the treatment of blood cancer and increasing number of cancer patients globally has garnered more demand for chemotherapeutic drugs throughout the world.

Geographically, the global blood cancer drugs market is segmented into North America, Europe, Asia Pacific, and the rest of the world. North America dominates the blood cancer drugs market which is followed by Europe and Asia Pacific. Favorable reimbursement policies, surge in R&D investments of various companies, as well as the increase in the number of blood cancer treatments are some of the major factors responsible for the growth of North Americas blood cancer drugs market. Moreover, Asia-Pacific region has been identified as the lucrative market for the for blood cancer drugs due to increasing awareness of the use of these drugs, increased healthcare expenditure, and rising per capita disposable income. These are some of the major factors which are influencing the growth of the blood cancer drugs in Asia-pacific region.

The leading companies operating in this industry include Johnson & Johnson Inc., Amgen Inc., Bayer AG., Pfizer, Inc., AbbVie Inc., Roche Holding AG., Celgene Corporation, AstraZeneca, Novartis AG, GlaxoSmithKline PLC, Merck & Co., Inc., and Eli Lily & Co. among others.

Key Findings from the study suggest blood cancer drugs in the market are much innovative and manufacturers are progressively concentrating on innovation of combination drugs. Companies are in a stage of development of new drugs in order to provide novel treatments for blood cancer. The immunotherapy segment is anticipated to grow at a high growth rate over the forecast period. The growth of this segment is primarily driven by increased awareness for its use as an alternative and effective treatment for blood cancer. North America is presumed to dominate the global blood cancer drugs market over the forecast period. Asia Pacific region which shows signs of high growth potential owing to the booming economies of India, and China.

Get a Sample Report for more Expert and Official insights: @https://www.reportocean.com/industry-verticals/sample-request.php?report_id=5256

The Global BLOOD CANCER DRUGS Market is segmented into various sub-groups to understand the market scenario in detail, the market segmentation are as follows:

[By Blood Cancer Type (Leukemia (Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Lymphocytic Leukemia), Lymphoma (Hodgkin Lymphoma, Non-Hodgkin Lymphoma (B-Cell Lymphoma, T-Cell Lymphoma)), and Myeloma; By Drugs (Rituaxan/Mabthera (Rituximab), Gleevac/Glivec (Imatinib), Revlimid (Lenalidomide), Velcade (Bortezomib), Tasigna (Nilotinib), Pomalyst (Pomalidomide), Vidaza (Azacitidine), Kyprolis (Carfilzomib), Adcetris (Brentuximab Vedotin), and Others); By Treatment Approaches (Chemotherapeutic, mAbs/Targeted Therapies, and Immunotherapeutic); By Region]

Furthermore, the years considered for the study are as follows:

Historical year 2013-2017

Base year 2018

Forecast period** 2019 to 2025 [** unless otherwise stated]

Regional split of the Global BLOOD CANCER DRUGS Market research report is as follows:

The market research study offers in-depth regional analysis along with the current market scenarios. The major regions analyzed in the study are:

Key highlights and important features of the Report:

Overview and highlights of product and application segments of the global BLOOD CANCER DRUGS Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Explore about Sales data of key players of the global BLOOD CANCER DRUGS Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the BLOOD CANCER DRUGS Market.

Explore about gross margin, sales, revenue, production, market share, CAGR, and market size by region.

Describe BLOOD CANCER DRUGS Market Findings and Conclusion, Appendix, methodology and data source;

Research Methodology:

The market research was done by adopting various tools under the category of primary and secondary research. For primary research, experts and major sources of information have been interviewed from suppliers side and industries, to obtain and verify the data related to the study of the Global BLOOD CANCER DRUGS Market. In secondary research methodology, various secondary sources were referred to collect and identify extensive piece of information, such as paid databases, directories and annual reports and databases for commercial study of the Global BLOOD CANCER DRUGS Market. Moreover, other secondary sources include studying technical papers, news releases, government websites, product literatures, white papers, and other literatures to research the market in detail.

Browse Premium Research Report with Tables and Figures at @ https://www.reportocean.com/industry-verticals/details?report_id=5256

There are 15 Chapters to display the Global BLOOD CANCER DRUGS Market:

Chapter 1, to describe Definition, Specifications and Classification of Global BLOOD CANCER DRUGS, Applications of, Market Segment by Regions;Chapter 2, to analyze the Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;Chapter 3, to display the Technical Data and Manufacturing Plants Analysis of , Capacity and Commercial Production Date, Manufacturing Plants Distribution, Export & Import, R&D Status and Technology Source, Raw Materials Sources Analysis;Chapter 4, to show the Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);Chapter 5 and 6, to show the Regional Market Analysis that includes United States, EU, Japan, China, India & Southeast Asia, Segment Market Analysis (by Type);Chapter 7 and 8, to explore the Market Analysis by Application Major Manufacturers Analysis;Chapter 9, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type, Market Trend by Application;Chapter 10, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;Chapter 11, to analyze the Consumers Analysis of Global BLOOD CANCER DRUGS by region, type and application;Chapter 12, to describe BLOOD CANCER DRUGS Research Findings and Conclusion, Appendix, methodology and data source;Chapter 13, 14 and 15, to describe BLOOD CANCER DRUGS sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

About Report Ocean:

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Water Heater Market Projected to Garner Significant Revenues by 2025 - Health News Office

CAMBRIDGE, Mass., Oct. 24, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report third quarter 2019 financial results after the Nasdaq Global Market closes on Thursday, November 7, 2019. Subsequently, at 4:30 p.m. E.T., the Company will host a conference call to discuss its third quarter 2019 financial results and to provide a corporate update.

The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 8998299. Please specify to the operator that you would like to join the "Sarepta Third Quarter 2019 Earnings Call." The conference call will be webcast live under the investor relations section of Sarepta's website at http://www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics to Announce Third Quarter 2019 Financial Results and Recent Corporate Developments on November 7, 2019 - BioSpace

Image: Breast cancer cell. Image credit:Anne Weston, Francis Crick Institute. Licence: CC-BY-NC-4.0.

Scientists in ourDivision for Breast Cancer Researchhave been involved in some of the most famous discoveries in the history of breast cancer research.

They discovered the BRCA2 gene, for example, and over the past 20 years have been pioneers in establishing the concept of synthetic lethality in cancer research and treatment. Their work underpinned and stimulated the development of olapariband other PARP inhibitors now standard treatments for genetic breast and platinum-sensitive ovarian cancers.

In the past five years, results of a clinical trial co-led by Professor Andrew Tutt, Head of our Division of Breast Cancer, have also established carboplatin a drug discovered at the ICR as a better treatment forBRCA1 andBRCA2mutated forms of advanced breast cancer than the usual breast cancer standard.

Many of our successes in breast cancer research have come from The Breast Cancer Now Toby Robins Research Centre at the ICR.

The integrated teams at the ICR, together with colleagues at our partner hospital The Royal Marsden NHS Foundation Trustand through Breast Cancer Nowprogramme integration with Kings College Londonand Guys Cancer Centreat Guys and St Thomas NHS Foundation Trust, have committed to define what they term the evolving unmet need for breast cancer research by 2025 with funding from Breast Cancer Now underpinning these ambitious plans.

The Division of Breast Cancer Research, which incorporates theBreast Cancer Now Research Centre, contains over 100 scientists and clinicians working in 12 teams.

Find out more

Researchers at the Centre lead important programmes to understand the genetic and environmental causes of breast cancer, and how a tumours molecular biology and surrounding microenvironment affect its behaviour and response to treatment.

They are also working on identifying new therapeutic approaches to breast cancer, and understanding the mechanisms tumours use in becoming resistant to treatment.

In a recent review of the Centres funding by Breast Cancer Now, Professor Tutt and colleagues across the centre set out a raft of successes over the past five years including 398 research manuscripts, two new drug discovery programmes, the development of a new drug candidate, and 13 breast cancer clinical trials.

The Centre is clear in its aims to keep the fundamental science taking place in its laboratories relevant to real patient problems. This includes close collaboration with hospital colleagues, a focus on rarer but biologically distinct forms of breast cancers, the study of tumour evolution during treatment in clinical trials, and extending the impact of findings to broader patient groups.

First, a thank you to Professor Mitch Dowsett, Head of the joint Academic Department of Biochemistry at The Royal Marsden and the ICR. Professor Dowsetts work on the role of hormones in breast cancer has led to the development of both the most effective endocrine therapy treatments used for patients today and the biomarkers that identify those who may benefit most from them helping patients to avoid lengthy treatments and chemotherapy. Professor Dowsett's work on endocrine therapy resistance has been led with Dr Lesley-Ann Martin.

This work will sadly not be continuing in the next five-year round of funding from Breast Cancer Now as Professor Dowsett scales down his work. We are hugely proud of the team's myriad achievements to date, and the centre will continue research into hormones and breast cancer with Professor Stephen Johnston at The Royal Marsden and new appointments to ICR Faculty including Professor Cathrin Brisken.

The approval of the innovative breast cancer drug palbociclibis another example ofthe Centres success in achieving meaningful improvements for patients in the past five years.

Palbociclib is known as a first-in-class drug, because its the first drug to work in the way that it does blocking two proteins, called CDK4 and CDK6 that help cancer cells divide and spread.

Professor Nicholas Turner, Professor in Molecular Oncologyat the ICR and Consultant Oncologist at The Royal Marsden led a key clinical trial of palbociclib, showing that it could slow the progression of advanced breast cancer in conjunction with standard treatments, and substantially extend the lives of patients.

This innovation is already changing lives more than 90,000 patients have been prescribed the drug.

Professor Turner, along with Professor Spiros Linardopoulos, Head of Drug Discovery at the Centre and other colleagues, continue to work on how resistance develops and how it may be targeted.

ICR patient advocate Christine was initially diagnosed with breast cancer in 2012 but, nearly five years after surgery and chemotherapy left her seemingly cancer free, she found the disease had returned, and spread to her brain and bones.

Following radiotherapy, she needed a targeted treatment to keep her cancer at bay. Fortunately, palbociclib had been approved for use on the NHS just months before, thanks to clinical trials that showed how palbociclib could benefit patients.

Christine is doing well on palbociclib: Im on the 21st cycle and my cancer is currently stable, she says. Im able to work part-time, and I can keep up my cycling, which would have been more difficult on conventional therapy.

Professor Linardopoulos leads the Centres work to search for gene targets for which new anticancer drugs can be developed.

A recent success for his team helped to progress work on a new drug candidate effective against fast-dividing cells, including triple-negative breast cancers. The target for the drug is a protein known to play a key part in controlling cell division monopolar spindle 1 (MSP1). The first clinical trial of the new treatment is under way at The Royal Marsden and will shortly expand to other NHS Hospital partners like Guys Hospital.

Professor Spiros Linardopoulos said: This exciting cancer treatment uses cancer's rapid growth against it, by forcing cells through cell division so quickly that they accumulate fatal errors.

With the drug now entering clinical trials, we can see how our translational approach at the ICR to get drugs from the lab to the clinic really has an impact for patients.

Professor Tutt explains that: together with other Team Leaders in the Breast Cancer Now centre at the ICR, there is a focus on driving progress to address niche, harder problems which are biologically distinctive but often not an initial focus for industry.

These are some examples of programmes of work currently taking place across the centre:

Unpicking the functional genetics of breast cancer risk

Dr Olivia Fletcher, Team Leader in Functional Genetic Epidemiologyleads a team of genetic epidemiologists and molecular biologists working in partnership with the Breast Cancer Now Generations Study, the British Breast Cancer Study and other population-based studies.

Population based studies allow us important insights into who genetic variants are associated with breast cancer risk specifically, variants that map to non-coding DNA, and which are as a result difficult to identify. Olivias team are understanding how some of these variants act to drive risks in the hope of identify groups for early detection or prevention strategies.

Targeting neighbouring healthy cells

Professor Clare Isacke, Team Leader in Molecular Cell Biology, is leading work to identify the processes by which tumour cells recruit and activate non-cancerous cells during metastasis, the process of cancers growth and spread from its original site.

The team is identifying strategies to effectively target these neighbouring heathy cells, due to the important part they play in modulating the response of tumour cells to treatment and as a result, tumour progression and resistance.

Mobilising the bodys immune system

Mobilising the bodys immune system after cell death is one major area of focus and innovation for the centre. Professor Pascal Meier, Team Leader in Cell Death and Immunity, is leading a team to explore the complex relationship between cell death, immunity and tumour growth and survival.

In particular, the team is focussing on the role of cell death and inflammation in adaptation to tissue stress, treatment resistance and tumour surveillance.

The team hope that new combination treatments they are trialling will allow doctors to manipulate the types of cell death which occur after treatment with current standard-of-care drugs and emerging targeted therapies, to more effectively mobilise the bodys natural immune response and enable better treatment outcomes.

Exploiting vulnerabilities in different subsets of breast cancer

Biomarkers are biological products like proteins or genes that can be measured in patient samples to detect disease. Biomarker development plays an important role in allowing researchers the ability to identify different tumour subsets with great accuracy, and in turn with a greater understanding of these subtypes for the biomarkers to help predict disease outcome.

Dr Rachael Natrajan, Team Leader in Functional Genomics, is researching the ways in which different subtypes of breast cancer can be characterised through genetic analysis of the tumour, to understand how different subgroups of a cancer and subpopulations of cells or clones become treatment resistant and can cooperate in doing so to drive relapse.

Other teams are leading work to understand the different dependencies and potential new drug targets within different subpopulations of a tumour.

Professor Chris Lord, Deputy Head of the Division of Breast Cancer Research, approaches this as an expert in the field of synthetic lethality. The term refers to when scientists exploit the fatal effects of disruption of the activity of two genes even though either disruption alone would not be fatal for the cells. Combining the effect of mutations in the tumour and use of targeted drugs can allow potent and tumour specific therapeutic effects. His team use tumour cell models and CRISPR-CAS9 gene perturbation techniques to help build their understanding of models of resistance in tumour cells.

Professor Tutt and Professor Lords laboratories are also working together to understand and target DNA repair deficient and genetically unstable breast cancers. Professor Tutts group have a special focus on basal-like Triple Negative breast cancers and create tumour organoid models, using tumours from patients treated with drugs that target DNA damage response and DNA replication stress.

To date, breast cancer research at the ICR has had a huge impact in helping patients assess their risks of developing breast cancer, choose the right treatment options and live longer.

Find out more

Ultimately, the end-goal for everything taking place in our Division of Breast Cancer Research is to improve the outcome and quality of life of women with or at risk of breast cancer.

Professors Tutt and Turner, as practicing oncologists and trial leaders, work with many others in the Centre to translate findings through to clinical trials using an integrated and patient-focussed strategic approach.

The centre also works closely with the wider national Breast Cancer Now network of research centres, and the international breast cancer research community. In partnership with The Royal Marsden, the Centre acts as a beacon for discovering therapy approaches and developing proof of concept early phase and international practice changing late phase trials.

October is Breast Cancer Awareness Month a good time to reflect on the Centres work ahead, as 2020 draws close and the next five-year phase for the Centres strategy begin in earnest. We know the work is in good hands.

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How ICR researchers are leading the way in breast cancer research - The Institute of Cancer Research

The Viral Vector Manufacturing Market research report 2019 has been estimated considering the application and regional segments, market share, and size, while the forecast for each product type and application segment has been provided for the global and regional markets. Viral Vector Manufacturing report offers detailed profiles of the key players to bring out a clear view of the competitive landscape of the Viral Vector Manufacturing outlook. It also comprehends market new product analysis, financial overview, strategies and marketing trends.

In Viral Vector Manufacturing Market Report, Following Companies Are Covered:

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Market Overview:

Scope of the Report:

The Research Document Will Answer Following Questions Such as:

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Key Market Trends:

The Cancer Sub-segment is Expected to Grow Faster in the Disease Segment

In the field of oncology, viral vector-based gene therapy has demonstrated steady progress. A multitude of viral vectors has been engineered for both therapeutic and preventive applications, in cancers. A critical development in viral vector-based cancer therapy has been the application of engineered and naturally occurring oncolytic viral vectors. These vectors are programmed to specifically replicate inside the cancer cells and induce toxic effects, which ultimately results in apoptosis. The attractive features of viral vectors relate to their capability to provide high levels of transgene expression, in a broad range of host cells.

The high demand for effective therapeutics for the management of cancers, the presence of fast track approval process, and the prospects of novel drugs to turn into blockbuster products are primary reasons responsible for the significant R&D investments in the field of viral vector-based cancer therapeutics, which, in turn, is driving the markets growth.

North America Dominates the Market and is Expected to Follow the Same Trend in the Future As Well

North America currently dominates the market for viral vector manufacturing and is expected to continue its stronghold for a few more years. The United States holds the largest market share in the North American region owing to factors, such as the high adoption rate of new therapies and high awareness of the general population. The increasing prevalence of genetic and chronic disorders, such as cancer, an aging population, growing demand for targeted and personalized medicine, and favorable government initiatives are the factors responsible for the significant market size in the United States.

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Detailed TOC of Viral Vector Manufacturing Market Report 2019-2024:

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

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS4.1 Market Overview4.2 Market Drivers4.2.1 Rising Prevalence of Genetic Disorders, Cancer, and Infectious Diseases4.2.2 Increasing Number of Clinical Studies And Availability of Funding For Gene Therapy Development4.2.3 Potential Applications in Novel Drug Delivery Approaches4.3 Market Restraints4.3.1 High Cost of Gene Therapies4.3.2 Challenges in Viral Vector-manufacturing Capacity4.4 Porters Five Forces Analysis4.4.1 Threat of New Entrants4.4.2 Bargaining Power of Buyers/Consumers4.4.3 Bargaining Power of Suppliers4.4.4 Threat of Substitute Products4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION5.1 By Type5.1.1 Adenoviral Vectors5.1.2 Adeno-associated Viral Vectors5.1.3 Lentiviral Vectors5.1.4 Retroviral Vectors5.1.5 Other Types5.2 By Disease5.2.1 Cancer5.2.2 Genetic Disorders5.2.3 Infectious Diseases5.2.4 Other Diseases5.3 By Application5.3.1 Gene therapy5.3.2 Vaccinology5.4 Geography5.4.1 North America5.4.1.1 US5.4.1.2 Canada5.4.1.3 Mexico5.4.2 Europe5.4.2.1 Germany5.4.2.2 UK5.4.2.3 France5.4.2.4 Italy5.4.2.5 Spain5.4.2.6 Rest of Europe5.4.3 Asia-Pacific5.4.3.1 China5.4.3.2 Japan5.4.3.3 India5.4.3.4 Australia5.4.3.5 South Korea5.4.3.6 Rest of Asia-Pacific5.4.4 Middle East & Africa5.4.4.1 GCC5.4.4.2 South Africa5.4.4.3 Rest of Middle East & Africa5.4.5 South America5.4.5.1 Brazil5.4.5.2 Argentina5.4.5.3 Rest of South America

6 COMPETITIVE LANDSCAPE6.1 Company Profiles6.1.1 Cobra Biologics6.1.2 Finvector Oy6.1.3 Fujifilm Diosynth Biotechnologies U.S.A., Inc.6.1.4 Kaneka Corporation (Eurogentec)6.1.5 Merck KGaA6.1.6 Novasep Inc.6.1.7 Oxford BioMedica Plc.6.1.8 Sanofi SA6.1.9 Spark Therapeutics Inc.6.1.10 Thermo Fisher Scientific Inc.

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

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Viral Vector Manufacturing Market Report Extensive Analysis 2019 | Specified by Production, Technology, Competition, and Revenue Forecast till 2024 -...

Although Lo Vauclare is just 3 years old, his kisses are known around the world.

A Franco-American living in Paris with his parents and little sister, Lo is the boy behind Bisous for Leo, a nonprofit organization named with the French word for kisses.

He loves swimming in the pool, listening to music and laughing. His big eyes light up behind a pair of large, round spectacles every he time he smiles which is often.

But he has trouble eating. He can no longer hold himself upright without support.

When Lo was born, he developed like any other child crawling, cruising and then finally taking his first steps. But his mother, Deborah Vauclare, said he never got past that. He would fall every few paces.

At about 15 months, Los parents took him to the doctor, who assured them that some children take a few more months to learn how to walk.

But when the toddler continued to falter, his parents worries grew. Extensive testing and therapy followed. Then they pursued genetic testing.

The results showed their son had Infantile Neuroaxonal Dystrophy or INAD, an extremely rare neurodegenerative disease that robs children of their fledgling abilities. It can weaken their muscles, taking away their ability to walk, hold up their own heads or control their eye movements, according to the INADcure Foundation.

Vauclare called the disease a mixture of Alzheimers and Parkinsons that impacts children.

Antoine, Deborah and Lo Vauclare are the family behind Bisous for Lo, a nonprofit organization that raises funds for research on INAD, a neurodegenerative disease that affects Lo.

(Courtesy of Deborah Vauclare)

The Vauclares had just received the devastating news last June when their close family friend, Emily Rogath Steckler, visited Paris with her daughter, Chloe.

The Stecklers had scarcely arrived at the Vauclares apartment when the two little friends perched together on the windowsill.

Chloe planted a kiss on Lo.

And from there, the nonprofit organization was born.

I literally couldnt get it out of my head, said Steckler, who co-founded Bisous for Lo with Vauclare. I just knew everybody needed to be embraced and to have the kiss.

Bisous for Lo has since raised more than $200,000 for gene therapy research at Washington University in St. Louis, Vauclare said.

The organization funnels funds through the INADcure Foundation to researchers addressing INAD and similar diseases. The organizations Facebook page collects posts tagged with kisses and stories from other children with INAD worldwide.

Recently, the group collaborated nationwide with Martin Lawrence Galleries, which is owned by Stecklers parents, David and Leslee Rogath. A portion of sales from reprints of artist Robert Deybers piece, Seal with a Kiss, will benefit Bisous for Lo.

Deyber is known for his artistic plays on literal phrases. His works include Love Sick, depicting a polar bear spewing hearts out of its mouth, and The Right to Arm Bears showing a polar bear standing beside a cannon.

Seal with a Kiss, a painting of a proud pinniped with a big red kiss on its cheek preening atop a rock, was the perfect fit for Bisous for Lo.

I think it may have popped up in a conversation or something. ... It will hit me, and Ill be like, Oh my God, seal! Deyber said with a laugh. Its gotten to be where its taken over my life at this point.

Funds from the lithograph sales of artist Robert Deybers piece Seal with a Kiss will be donated to the nonprofti organization Bisous for Lo.

(Courtesy of the Martin Lawrence Galleries)

Funds from the lithograph sales will support research on gene therapy and treatment taking place in the United States and Europe, Vauclare said.

The challenge, Vauclare added, is to convince researchers studying Alzheimers and Parkinsons to pay attention to INAD a disease that Vauclare said impacts only about 150 children worldwide.

Vauclare recently resigned from her job at an architectural firm so she can spend more time working on the nonprofit and supporting her son.

The second my kids go to bed at night, Im on the computer basically until my eyes shut, every single night, she said.

Lo, now 3, is participating in a clinical trial that will last until June. Despite trips between the United States and Paris for treatments, Vauclare said her son remains cheerful.

I definitely think as the disease progresses, we see a little bit less, she said. But even though we see less of it, its very pleasant in the morning when he wakes up and gives you a big smile. It takes him longer to connect with you.

With every social media post and painting sold, the Vauclare family hopes to kiss the disease goodbye.

Donations can be made at BisousForLeo.org and lithographs may be purchased at martinlawrence.com/pages/every-kiss-counts.

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Sealed with a kiss: how one little boy in Paris is stealing hearts in Costa Mesa - Los Angeles Times

The National Institutes of Health (NIH) announced a partnership with the Bill and Melinda Gates Foundation on Wednesday to fund the development of targeted cures for HIV and sickle cell disease with a view to helping people in developing countries using gene therapy. With most of the populations affected by each disease residing in sub-Saharan Africa, treatments are being sought with regional conditions in mind.

The NIH and the Gates Foundation are investing $100 million in the initiative to develop low-cost gene therapies. The announcement follows President Donald Trump's pledge in his 2019 State of the Union address that the United States would eradicate HIV within the next decade. The Trump administration has also tried to draw more attention to sickle cell disease (SCD) in the past few years, according to a press release from NIH.

Sickle cell disease is a blood disorder that can cause anything from mild pain to heart failure. Human immunodeficiency virus (HIV) is a communicable disease that, if left untreated, wipes out the immune system. People with SCD inherit the disease from their parents, whereas HIV is acquired through blood contamination with certain bodily fluids of an infected person. While the mechanisms of transmission are different, both diseases are carried in the genome of infected individuals. Globally, both diseases also disproportionately impact individuals in lower-income communitiesand scientists believe that both could be combatted with gene-based treatments.

The past few years have seen unprecedented strides toward cures for these two diseases using gene therapy, which the NIH defines as experimental technique wherein doctors insert genes into a patient's cells so their body can more effectively resist a disease. It can include inserting a healthy variant of a gene to replace the unhealthy copy that causes a disease, or placing an entirely new gene in the body to fight the disease.

"Dramatic advances in genetics over the last decade have made effective gene-based treatments a reality... Yet these breakthroughs are largely inaccessible to most of the world by virtue of the complexity and cost of treatment requirements, which currently limit their administration to hospitals in wealthy countries," the press release states. The new initiative will focus on developing treatments that can be delivered in "low-resource settings."

Speaking on the initiative's viability, Dr. Ronald Mitsuyasu, a professor of medicine in hematology-oncology at the University of California, Los Angeles with more than 25 years of experience in HIV clinical trials research, told Newsweek that this sort of solution has been attempted in the past, but gene therapy hasn't yet proved successful in treating HIV.

"There have been several attempts to use gene therapy for HIV by either incorporating genes that suppress HIV genes, producing decoys for various viruses required processes needed for viral replication, or substituting inactive genes for functional genes of HIV," he said.

But those living in developing countries have not had as many chances to benefit from these solutions as those living in places like the U.S., according to the press release.

"SCD and HIV are major burdens on health in low-resource communities around the world," the press release read. "Approximately 95% of the 38 million people living with HIV globally are in the developing world, with 67% in sub-Saharan Africa, half of whom are living untreated. Fifteen million babies will be born with SCD globally over the next 30 years, with about 75% of those births occurring in sub-Saharan Africa."

Further, the prediction indicates that three-quarters of those infants will be born into low-income countries and communities. Between 50 and 90 percent of babies born with the disease in sub-Saharan African countries will die before the age of five, according to the release.

So, the NIH and the Gates Foundation's initiative aims to identify potential cures for both diseases as well as partner with groups in Africa to identify candidates on whom these new cures can be tested.

We are losing too much of Africa's future to sickle cell disease and HIV. Beating these diseases will take new thinking and long-term commitment. I'm very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africa's greatest public health challenges." Matshidiso Rebecca Moeti, M.B.B.S., the World Health Organization's regional director for Africa said of the initiative.

Mitsuyasu said he agreed that continued investigation into gene-based cures would eventually yield worthwhile results. "I personally believe that it should be possible to ultimately develop a gene therapy approach to overcome ... HIV," Mitsuyasu said. "Continued scientific developments in the field of gene therapy will eventually allow for the conquest of most genetic and viral gene integrated diseases."

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Gates Foundation, NIH Bet on Gene Therapy To Bring Cheap HIV and Sickle Cell Cures to Sub-Saharan Africa - Newsweek

NEW YORK and BASEL, Switzerland, Oct. 23, 2019 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (AXGT), a clinical-stage company developing innovative gene therapies, today announced preliminary data from an expanded access study administering investigational AXO-AAV-GM2 gene therapy in two patients with infantile Tay-Sachs disease (TSD) at the 27th Annual Congress of the European Society of Gene and Cell Therapy. Infantile TSD, a rapidly progressive and fatal pediatric neurodegenerative genetic disorder, has a median life expectancy of approximately 3-4 years. This data indicates the potential to modify the rate of disease progression in children with infantile TSD.

Todays exciting clinical results from the AXO-AAV-GM2 program are the first reported evidence for potential disease modification in Tay-Sachs disease, and suggest an opportunity for gene replacement therapy to improve outcomes for children with this devastating condition, said Dr. Gavin Corcoran, chief R&D officer at Axovant. Myelination is an important component of healthy brain development in infants and is often abnormal in children with Tay-Sachs disease. We were encouraged to see MRI evidence of preserved brain architecture and improved myelination in the early symptomatic child treated at 10 months of age, coupled with stability of neuromuscular function as measured on the CHOP INTEND scale. We look forward to Dr. Terry Flottes presentation of this data at the ESGCT conference where he will describe these two clinical cases in detail.

Key findings from this first-in-human study in patients treated with AXO-AAV-GM2, an investigational gene therapy designed to restore -Hexosaminidase A enzyme activity in the central nervous system, include:

*CHOP INTEND is a 16-item scale of motor function that has been validated in infants with neuromuscular disorders. Items of motor function are graded from 0 to 4 for each item, where zero equals no response, and 4 equals a complete response. Change from baseline in total score of 4 points or a total score sustained > 40 points has been associated with a clinically meaningful benefit.

Dr. Terence Flotte, Professor of Pediatrics and Dean at the School of Medicine, University of Massachusetts Medical School, said, Bilateral intrathalamic and intrathecal delivery of rAAV gene therapy may surmount the obstacle of providing widespread distribution of therapeutic enzyme throughout the brain and CNS. This innovative delivery could overcome one of the primary challenges for developing treatments for Tay-Sachs, Sandhoff and many other severe pediatric genetic disorders, providing much needed hope for these families.

Dr. Flotte will present this data on AXO-AAV-GM2 in the First-in-Human gene therapy session on October 23, 2019 at 5:30 PM Central European Time (CET).

About AXO-AAV-GM2

AXO-AAV-GM2 is an investigational gene therapy for Tay-Sachs and Sandhoff disease, which rare and fatal pediatric neurodegenerative genetic disorders within the GM2 gangliosidosis family, caused by defects in theHEXA(leading to Tay-Sachs disease) orHEXB(leading to Sandhoff disease) genes that encode the two subunits of the -hexosaminidase A (HexA) enzyme. Both forms of GM2 gangliosidosis are caused by overwhelming storage of GM2 ganglioside within neurons throughout the central nervous system), which is normally degraded in the lysosome by the isozyme HexA. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA levels by introducing a functional copy of theHEXAandHEXBgenes via delivery of two co-administered AAVrh8 vectors.

In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

About Axovant Gene Therapies

Axovant Gene Therapies, part of the Roivant family of companies, is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinsons disease.

Story continues

Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visitwww.axovant.com.

About Roivant

Roivantaims to improve health by rapidly delivering innovative medicines and technologies to patients.Roivantdoes this by buildingVants nimble, entrepreneurial biotech and healthcare companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. Roivant today is comprised of a central technology-enabled platform and 20 Vants with over 45 investigational medicines in clinical and preclinical development and multiple healthcare technologies. For more information, please visitwww.roivant.com.

About the University of Massachusetts Medical School

The mission of the University of Massachusetts Medical School is to advance the health and well-being of the people of the commonwealth and the world through pioneering education, research, public service and health care delivery.

Research into potential therapies for lysosomal storage diseases such as Tay-Sachs, Sandhoff disease and GM1 gangliosidosis at UMass Medical School and Auburn University has led to significant advances in the field. Miguel Sena-Esteves, PhD, associate professor of neurology at UMass Medical School; Heather Gray-Edwards, PhD, DVM, formerly of Auburn and currently assistant professor of radiology at UMass Medical School; and Douglas Martin, PhD, professor of anatomy, physiology and pharmacology in the College of Veterinary Medicine and the Scott-Ritchey Research Center at Auburn University, have worked collaboratively for more than a decade on animal models and therapeutic approaches for these and similar disorders. For more information, visit http://www.umassmed.edu.

Forward Looking Statements and Information

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, believe, estimate, and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the initiation, timing, progress, and reporting of results of its preclinical programs, clinical trials, and research and development programs; cash to be used in operating activities; its ability to advance its gene therapy product candidates into and successfully initiate, enroll, and complete clinical trials; the potential clinical utility of its product candidates; its ability to continue to develop its gene therapy platforms; its ability to develop and manufacture its products and successfully transition manufacturing processes; its ability to perform under existing collaborations with, among others, Oxford Biomedica, and theUniversity of Massachusetts Medical School, and to add new programs to its pipeline; its ability to enter into new partnerships or collaborations; its ability to retain and successfully integrate its leadership and personnel; and the timing or likelihood of its regulatory filings and approvals are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovants management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected.Such risks and uncertainties include, among others, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the expectations for regulatory submissions and approvals; the continued development of its small molecule and gene therapy product candidates and platforms; Axovants scientific approach and general development progress; and the availability or commercial potential of Axovants product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovants most recent Quarterly Report on Form 10-Q filed with theSecurities and Exchange CommissiononAugust 9, 2019, as updated by its subsequent filings with theSecurities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made.Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media and Investors

Parag MeswaniAxovant Gene Therapies (212) 547-2523investors@axovant.commedia@axovant.com

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Axovant Presents First Evidence of Clinical Stabilization in Tay-Sachs Disease at the European Society of Gene and Cell Therapy 27th Annual Congress -...

Here we go again.

This week, investors may have seen that a new gene editing technique will soon be available to scientists attempting to engineer DNA for human health applications. Dubbed "prime editing," some have called it a game-changer due to its inherent advantages over more commonly used CRISPR techniques. It has the potential to correct up to 89% of disease-causing mutations and is engineered to make more precise edits than other gene editing techniques. And it all belongs to a new start-up: Prime Medicine.

But despite all of the media hype, prime editing has one enormous problem that may make it impossible to commercialize: The molecular components are way too big to be delivered into cells using current methods. It's as if an automaker designed a car with an amazing interior, but forgot to add wheels.

The "big" obstacle facing prime editing doesn't necessarily mean CRISPR Therapeutics (NASDAQ:CRSP), Editas Medicine (NASDAQ:EDIT), and Intellia Therapeutics (NASDAQ:NTLA) will have smooth sailing ahead, but it does highlight an important facet of gene editing technologies that investors can't overlook: delivery.

Cutting both strands of DNA is bad. Image source: Getty Images.

Healthcare investors are probably familiar with the basics of how CRISPR gene editing works by now, but a little refresher can't hurt.

The payload comprises a guide RNA and a cutting enzyme such as Cas9. The guide RNA helps the payload locate the target sequence of DNA that will be edited. Once located, the payload binds to the target sequence of DNA, and the cutting enzyme makes a double-stranded break (meaning it cuts through both strands of DNA) to remove or insert DNA.

But there are problems. The guide RNA and cutting enzyme aren't always 100% accurate, and can sometimes edit the genome relatively far from the target site. That can introduce changes to the genome, which can be harmless -- or potentially harmful.

An even larger problem is that traditional CRISPR gene editing techniques make double-stranded DNA breaks that require one of two natural DNA repair mechanisms to patch up. Unfortunately, there can be significant consequences to this, such as allowing a dormant cancer cell to proliferate and reduced editing efficiency. This problem isn't alleviated by using different cutting enzymes, such as switching from Cas9 to Cas12.

While CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics have gone all-in on CRISPR-Cas systems, many scientists aren't confident that the technique will deliver on its promise in a clinical setting. But the learning experiences in the lab have spurred a search for new gene editing techniques that don't make as many off-target edits and don't make double-stranded breaks. Prime editing is the latest tool.

Cutting one strand of DNA is better. Image source: Getty Images.

Prime editing makes some notable modifications to the traditional CRISPR-Cas setup. The cutting enzyme, Cas9, has been modified to only cut one strand of DNA. The guide RNA deployed also contains the instructions for the desired edit. And a second enzyme, called reverse transcriptase, has been added to the payload to write the desired edits into the exposed strand of target DNA.

The changes allow prime editing to change any DNA base -- A, T, C, or G -- into any other. Prime editing has also been used to insert up to 44 base letters and delete up to 80 base letters from target DNA in limited studies. It may not seem like much considering the human genome contains over 3 billion base pairs, but the characteristics described above could allow the new technique to correct up to 89% of disease-causing mutations in humans.

The versatility is pretty amazing, but the problem is that prime editing payloads -- comprising two enzymes and a longer guide RNA -- are too large to be delivered with today's delivery vehicles, such as adeno-associated viruses (AAV); after all, enzymes are big. The field of gene therapy only recently overcame the hurdle of delivery, and gene editing is still trying to figure it out.

CRISPR Therapeutics has (perhaps wisely) kicked that can down the road by focusing initial efforts on ex vivo editing, or editing cells in a controlled environment outside of the human body, where delivery is somewhat less complicated. Editas Medicine has taken up the mantle of being the first to try in vivo editing, or editing cells directly in the human body. It's using AAV delivery vehicles initially.

Meanwhile, Intellia Therapeutics has fallen out of favor with investors because its slow-and-steady approach won't let it enter clinical trials until 2020 or 2021. But investors may be missing the point. Rather than plowing ahead with AAV delivery, the company is working to develop new and novel delivery systems based on lipid nanoparticle (LNP) tech licensed from Novartis. It could end up being the main differentiator in the CRISPR gene editing space, and the move is entirely aimed at solving the tricky problem of delivery.

There's also Precision BioSciences (NASDAQ:DTIL), which has a proprietary gene editing system called ARCUS. The tech is completely shielded from CRISPR patent disputes, doesn't make double-stranded DNA cuts, is ahead of CRISPR Therapeutics in an important clinical area, and can diversify its revenue by forging into agriculture or industrial biotech. It relies on AAV delivery vehicles.

Simply put, at a time when gene editing techniques are attempting to figure out the best solution to overcome delivery obstacles, prime editing went all-in on optimizing other characteristics of the tool, while betting the delivery problem will solve itself. That's a risky bet.

Image source: Getty Images.

Luckily for scientists and investors, the swift rise of CRISPR gene editing in research settings has led to the development of robust testing tools. That should shorten the learning curve for researchers wanting to know whether or not prime editing is going to live up to the hype -- or overcome its big delivery problem.

Nonetheless, the media hype highlights that individual investors need to be more selective in their approach to gene editing stocks. Tools that make double-stranded breaks (read: CRISPR) are likely to have a high rate of failure, but ex vivo applications in engineering immune cells could yield success. That would bode well for CRISPR Therapeutics and Precision BioSciences.

The delivery problem will make in vivo editing much more difficult, which could be a problem for Editas Medicine's lead drug candidates and future areas of focus of CRISPR Therapeutics. Intellia Therapeutics is hopeful its LNP approach could solve the problem, but it's too soon to know.

That said, there's one gene editing technique that could strike a balance between traditional CRISPR-Cas systems and prime editing: base editing. It was developed by the same lab that pioneered prime editing and belongs to a start-up called Beam Therapeutics, and Editas Medicine may already have one foot in the doorto licensing the tech. Then again, the gene editing space is very young and rapidly evolving, so investors cannot dismiss the inherent risks of owning gene editing stocks.

More:
There's Yet Another Gene Editing Technique, but It Has 1 Glaring Problem - Motley Fool

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Newswise October 25, 2019 Major burns lead to changes in the gastrointestinal tract bacteria, and these alterations of the gut microbiome are influenced by resuscitation with intravenous (IV) fluids, according to animal studies reported inSHOCK: Injury, Inflammation, and Sepsis: Laboratory and Clinical Approaches,Official Journal of theShockSociety. The journal is published in the Lippincott portfolio byWolters Kluwer.

Potentially disease-causing gut bacteria are increased after a major burn, while large volumes of IV fluids promote recovery of beneficial bacteria, suggest the experiments by David Burmeister,PhD, of the US Army Institute of Surgical Research and colleagues. They write, "This study is the first to show that the gut microbiome is altered following a large burn injury in pigs and that the gut microbiome may be influenced by the resuscitation strategy used."

Gut Microbiome Changes May Affect Outcomes After Major Burns

Burnsdon'tjust cause skin damage they result in physiological stress and inflammation that may lead to infections and other serious complications. While a growing body of research has shown that the gut microbiome has important effectsforhealth and disease, little is known about how burns may alter the microbiome.

The researchers examined changes in the gut bacteria after burns and how those alterations are affected by fluid resuscitation. Major burns, covering 40 percent of body surface area, were induced in anesthetized swine. Because their skin and gastrointestinal tract are similar to those of humans, pigs are an important model for studying burns and their treatment.

The animals then received low- or high-volume fluid resuscitationor no IV fluids. Effects on gut microbiome diversity the proportions of different types of bacteria were analyzed by isolating and analyzing bacterial DNA.

The results showed significant changes in the gut microbiome after burns, with disruption of the normal balance between different groups of bacteria. Findings included a "hyperacute spike" in one major group called Proteobacteria, which includes many potentially pathogenic (disease-causing) organisms.

In contrast, high volumes of resuscitation fluids seemed to promote recovery of potentially beneficial microbes, such asBacteroidesbacteria. SinceBacteroidesplays a role in converting sugar into energy, this shift might help in coping with the increased metabolic demands on the body after burns. Fluid resuscitation also prevented increases in certain opportunistic bacteria, which might protect against the risk of sepsis and other serious infections.

Resuscitation fluids also seemed to affect some functional proteins in the gut for example, heat shock proteins that promote resistance to stress-induced cell damage. That provides a clue to understanding how fluid resuscitation helps the body recover from inflammation and other metabolic changes after burns.

Despite increasing evidence on the role of the gut microbiome, there has been little information on how bacterial diversity in the gut is affected by burns and their treatment. While early, aggressive fluid resuscitation is a key part treatment for burns,debatecontinues over the resuscitation strategies used.

Dr. Burmeister and colleagues call for further studies to clarify shifts in microbiome diversity after burns, and how these changes affect the outcomes of burns and responses to fluid resuscitation. Future research might lead to new treatments targeting the microbiome for example, using fecal transplant or antibiotics to promote recovery after burns. The researchers add, "Future clinical trials of resuscitation strategies should include gut microbiome analysis."

Click here to read "The Effect ofBurnResuscitationVolumes on theGutMicrobiomein aSwineModel"

DOI:10.1097/SHK.0000000000001462

###

AboutSHOCK

SHOCK:Injury, Inflammation, and Sepsis: Laboratory and Clinical Approachesincludes studies of novel therapeutic approaches, such as immunomodulation, gene therapy, nutrition, and others. The mission of the Journal is to foster and promote multidisciplinary studies, both experimental and clinical in nature, that critically examine the etiology, mechanisms and novel therapeutics of shock-related pathophysiological conditions.SHOCK is the Official Journal of theShock Society, the European Shock Society, the Indonesian Shock Society, the International Federation of Shock Societies, and the Official and International Journal of the Japan Shock Society

About Wolters Kluwer

Wolters Kluwer is a global leader in professional information, software solutions, and services for the health, tax & accounting, finance, risk & compliance, and legal sectors. We help our customers make critical decisions every day by providing expert solutions that combine deep domain knowledge with specialized technology and services.

Wolters Kluwer reported 2018 annual revenues of 4.3 billion. The group serves customers in over 180 countries, maintains operations in over 40 countries, and employs approximately 18,600 people worldwide. The company is headquartered in AlphenaandenRijn, the Netherlands.

Wolters Kluwer provides trusted clinical technology and evidence-based solutions that engage clinicians, patients, researchers and students with advanced clinical decision support, learning and research and clinical intelligence. For more information about our solutions, visithttp://healthclarity.wolterskluwer.comand follow us onLinkedInand Twitter@WKHealth.

For more information, visitwww.wolterskluwer.com, follow us onTwitter,Facebook,LinkedIn, andYouTube.

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Gut Microbiome Is Altered by Burns, Affected by Fluid Resuscitation: New Evidence in SHOCK - Newswise

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

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Who Are Opportunities, Risk and Driving Force of Nebulizer 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:

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

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

Chapter 4, the Nebulizer 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, Nebulizer market forecast, by regions, type and application, with sales and revenue, from 2019 to 2025.

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

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CNS Gene Therapy Market Projected to Discern Stable Expansion During 2018 2028 - Health News Office

The given report is an excellent research study specially compiled to provide latest insights into critical aspects of the Global Global Gene Therapy for CNS Disorders Market Market by INNOVATE INSIGHTS.

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Key players operating world wide: Gilead (Kite Pharma), Amgen (BioVex), Novartis, Roche (Spark Therapeutics), Bluebird Bio

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Segmentation by Product Type: Ex Vivo, In VivoMarket Growth by Applications: Hospitals, Clinics, Others

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Global Gene Therapy for CNS Disorders Market 2019 - Gilead (Kite Pharma), Amgen (BioVex), Novartis, Roche (Spark Therapeutics), Bluebird Bio -...

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

Make An EnquiryAbout This Report @ https://www.researchmoz.us/enquiry.php?type=E&repid=2527036&source=atm

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

3A Health CareDeVilbiss HealthcarePHILIPSRossmax International Ltd.CareFusionOmronPARIGFAllied Healthcare Products

Segment by RegionsNorth AmericaEuropeChinaJapanSoutheast AsiaIndia

Segment by TypePneumatic NebulizersUltrasonic NebulizersMesh NebulizersOther

Segment by ApplicationCOPDCystic fibrosisAsthmaOther

Request Sample Report @ https://www.researchmoz.us/enquiry.php?type=S&repid=2527036&source=atm

Important Key questions answered in Nebulizer market report:

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

What are the key factors affecting market dynamics? What are the drivers, challenges, and business risks in Nebulizer 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 Nebulizer market? Knows Upstream Raw Materials Sourcing and Downstream Buyers.

Who are the key manufacturers in space? Business Overview by Type, Applications, Gross Margin, and Market Share

What are the opportunities and threats faced by manufacturers in the global market?

You can Buy This Report from Here @ https://www.researchmoz.com/checkout?rep_id=2527036&licType=S&source=atm

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

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

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

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

Chapter 4, the Nebulizer 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, Nebulizer market forecast, by regions, type and application, with sales and revenue, from 2019 to 2025.

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

Original post:
Mannequin-based Simulation Market Insights on Emerging Scope 2026 - Health News Office

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

Make An EnquiryAbout This Report @ https://www.researchmoz.us/enquiry.php?type=E&repid=2527036&source=atm

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

3A Health CareDeVilbiss HealthcarePHILIPSRossmax International Ltd.CareFusionOmronPARIGFAllied Healthcare Products

Segment by RegionsNorth AmericaEuropeChinaJapanSoutheast AsiaIndia

Segment by TypePneumatic NebulizersUltrasonic NebulizersMesh NebulizersOther

Segment by ApplicationCOPDCystic fibrosisAsthmaOther

Request Sample Report @ https://www.researchmoz.us/enquiry.php?type=S&repid=2527036&source=atm

Important Key questions answered in Nebulizer market report:

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

What are the key factors affecting market dynamics? What are the drivers, challenges, and business risks in Nebulizer 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 Nebulizer market? Knows Upstream Raw Materials Sourcing and Downstream Buyers.

Who are the key manufacturers in space? Business Overview by Type, Applications, Gross Margin, and Market Share

What are the opportunities and threats faced by manufacturers in the global market?

You can Buy This Report from Here @ https://www.researchmoz.com/checkout?rep_id=2527036&licType=S&source=atm

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

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

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

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

Chapter 4, the Nebulizer 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, Nebulizer market forecast, by regions, type and application, with sales and revenue, from 2019 to 2025.

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

More:
Serving numerous end-users, Cladding Panels market anticipated to continue to rise between 2019 2027 - Health News Office

An unexpected early morning phone call from the hospital is never good news. When Joy Johnson answered, her first thought was that Sharon Birzer, her partner of 15 years, was dead. Her fears were amplified by the voice on the other end refusing to confirm or deny it. Just come in and talk to one of the doctors, she remembers the voice saying.

Johnson knew this was a real possibility. A few weeks earlier, she and Birzer sat in the exam room of a lymphoma specialist at Stanford University. Birzers cancer had grown, and fast first during one type of chemotherapy, then through a second. Out of standard options, Birzers local oncologist had referred her for a novel treatment called chimeric antigen receptor T-cell therapy or CAR-T. Birzer and Johnson knew the treatment was risky. They were warned there was a chance of death. There was also a chance of serious complications such as multi-organ failure and neurological impairment. But it was like warning a drowning person that her lifeboat could have problems. Without treatment, the chance of Birzers death was all but certain. She signed the consent form.

Johnson hung up the phone that early morning and sped to the hospital. She met with a doctor and two chaplains in a windowless room in the cancer ward, where happy photos of cancer alumni smiled down from the walls. This is getting worse and worse, Johnson thought. As she remembers it, the doctor went through the timeline of what happened for 10 minutes, explaining how Birzer became sicker and sicker, before Johnson interrupted with the thought splitting her world in two: I need you to tell me whether shes alive or dead.

Birzer wasnt dead. But she was far from okay. The ordeal began with Birzer speaking gibberish. Then came seizures so severe there was concern she wouldnt be able to breathe on her own. When it took a few different medications to stop Birzer from seizing, her doctors sedated her, put a breathing tube down her throat, and connected her to a ventilator. Now, she was unconscious and in the intensive care unit (ICU).

Birzer was one of the early patients to receive CAR-T, a radical new therapy to treat cancer. It involved removing Birzers own blood, filtering for immune cells called T-cells, and genetically engineering those cells to recognise and attack her lymphoma. CAR-T made history in 2017 as the first FDA-approved gene therapy to treat any disease. After three to six months of follow-up, the trials that led to approval showed response rates of 80% and above in aggressive leukemias and lymphomas that had resisted chemotherapy. Patients on the brink of death were coming back to life.

Also read:Rwanda on Track to Become the First Country to Eliminate Cervical Cancer

This is something I often dream of seeing but rarely do. As a doctor who treats cancer, I think a lot about how to frame new treatments to my patients. I never want to give false hope. But the uncertainty inherent to my field also cautions me against closing the door on optimism prematurely. We take it as a point of pride that no field of medicine evolves as rapidly as cancer the FDA approves dozens of new treatments a year.

One of my biggest challenges is staying up to date on every development and teasing apart what should and shouldnt change my practice. I am often a mediator for my patients, tempering theoretical promises with everyday realism. To accept a research finding into medical practice, I prefer slow steps showing me proof of concept, safety, and efficacy.

CAR-T, nearly three decades in the making, systemically cleared these hurdles. Not only did the product work, its approach was also unique among cancer treatments. Unlike our usual advances, this wasnt a matter of prescribing an old drug for a new disease or remixing known medications. CAR-T isnt even a drug. This is a one-time infusion giving a person a better version of her own immune system. When the FDA approved its use, it wasnt a question of whether my hospital would be involved, but how we could stay ahead. We werent alone.

Today, two FDA-approved CAR-T products called Kymriah and Yescarta are available in more than 100 hospitals collectively across the US. Hundreds of clinical trials are tinkering with dosages, patient populations, and types of cancer. Some medical centres are manufacturing the cells on-site.

The FDA approved CAR-T with a drug safety program called a Risk Evaluation and Mitigation Strategy (REMS). As I cared for these patients, I quickly realised the FDAs concerns. Of the 10 or so patients Ive treated, more than half developed strange neurological side effects ranging from headaches to difficulty speaking to seizures to falling unconscious. We scrambled to learn how to manage the side effects in real time.

Johnson and Birzer, who I didnt treat personally but spoke to at length for this essay, understood this better than most. Both had worked in quality control for a blood bank and were medically savvier than the average patient. They accepted a medical system with a learning curve. They were fine with hearing I dont know. Signing up for a trailblazing treatment meant going along for the ride. Twists and bumps were par for the course.

Cancer, by definition, means something has gone very wrong within a cell has malfunctioned and multiplied. The philosophy for fighting cancer has been, for the most part, creating and bringing in treatments from outside the body. Thats how we got to the most common modern approaches: Chemotherapy (administering drugs to kill cancer),radiation(using high energy beams to kill cancer), and surgery (cutting cancer out with a scalpel and other tools). Next came the genetics revolution, with a focus on creating drugs that target a precise genetic mutation separating a cancer cell from a normal one. But cancers are genetically complex, with legions of mutations and the talent to develop new ones. Its rare to have that one magic bullet.

Over the last decade or so, our approach shifted. Instead of fighting cancer from the outside, we are increasingly turning in. The human body is already marvellously equipped to recognise and attack invaders, from the common cold to food poisoning, even if the invaders are ones the body has never seen before. Cancer doesnt belong either.

But since cancer cells come from normal ones, theyve developed clever camouflages to trick and evade the immune system. The 2018 Nobel Prize in Physiology or Medicine was jointly awarded to two researchers for their work in immunotherapy, a class of medications devoted to wiping out the camouflages and restoring the immune systems upper hand. As I once watched a fellow oncologist describe it to a patient: Im not treating you. You are treating you.

What if we could go one step further? What if we could genetically engineer a patients own immune cells to spot and fight cancer, as a sort of best hits of genetic therapy and immunotherapy?

Enter CAR-T. The technology uses T-cells, which are like the bouncers of the immune system. T-cells survey the body and make sure everything belongs. CAR-T involves removing a persons T-cells from her blood and using a disarmed virus to deliver new genetic material to the cells. The new genes given to the T-cells help them make two types of proteins. The first giving the technology its name is a CAR, which sits on the T-cells surface and binds to a protein on the tumour cells surface, like a lock and key.

The second serves as the T-cells caffeine jolt, rousing it to activate. Once the genetic engineering part is done, the T-cells are prodded to multiply by being placed on a rocking device that feeds them nutrients while filtering their wastes. When the cells reach a high enough number a typical dose ranges from hundreds of thousands to hundreds of millions they are formidable enough to go back into the patient. Once inside, the cancer provokes the new cells to replicate even more. After one week, a typical expansion means multiplying by about another 1,000-fold.

Practically, it looks like this: A person comes in for an appointment. She has a catheter placed in a vein, perhaps in her arm or her chest, that connects to a large, whirring machine which pulls in her blood and separates it into its components. The medical team set the T-cells aside to freeze while the rest of the blood circulates back into the patient in a closed loop. Then, the hospital ships the cells frozen to the relevant pharmaceutical companys headquarters or transports them to a lab on-site, where thawing and manufacturing takes from a few days to a few weeks.

When the cells are ready, the patient undergoes about three days of chemotherapy to kill both cancer and normal cells, making room for the millions of new cells and eradicating normal immune players that could jeopardise their existence. She then gets a day or two to rest. When the new cells are infused back into her blood, we call that Day 0.

I remember the first time I watched a patient get his Day 0 infusion. It felt anti-climactic. The entire process took about 15 minutes. The CAR-T cells are invisible to the naked eye, housed in a small plastic bag containing clear liquid.

Thats it? my patient asked when the nurse said it was over. The infusion part is easy. The hard part is everything that comes next.

Once the cells are in, they cant turn off. That this may cause collateral damage was evident from the start. In 2009 working in parallel with other researchers at Memorial Sloan Kettering Cancer Centre in New York and the National Cancer Institute in Maryland oncologists at the University of Pennsylvania opened a clinical trial for CAR-T in human leukaemia patients. (Carl June, who led the CAR-T development, did not respond to Undarks interview request.)

Also read:Explainer: What Is Soft Tissue Cancer?

Of the first three patients who got CAR-T infusions, two achieved complete remission but nearly died in the process. The first was a retired corrections officer named Bill Ludwig, who developed extremely high fevers and went into multi-organ failure requiring time in the ICU. At the time, the medical teams had no idea why it was happening or how to stop it. But time passed. Ludwig got better. Then came the truly incredible part: His cancer was gone.

With only philanthropic support, the trial ran out of funding. Of the eligible patients they intended to treat, the Penn doctors only treated three. So they published the results of one patient in the New England Journal of Medicine and presented the outcomes of all three patients, including Ludwig, at a cancer conference anyway. From there, the money poured in. Based on the results, the Swiss pharmaceutical company Novartis licensed the rights of the therapy.

The next year, six-year-old Emily Whitehead was on the brink of death when she became the first child to receive CAR-T. She also became extremely ill in the ICU, and her cancer was also eventually cured. Her media savvy parents helped bring her story public, making her the poster child for CAR-T. In 2014, the FDA granted CAR-T a breakthrough therapy designation to expedite the development of extremely promising therapies. By 2017, a larger trial gave the treatment to 75 children and young adults with a type of leukaemia B-cell acute lymphoblastic leukaemia that failed to respond to chemotherapy. Eighty-one percent had no sign of cancer after three months.

In August 2017, the FDA approved a CAR-T treatment as the first gene therapy in the US. The decision was unanimous. The Oncologic Drugs Advisory Committee, a branch of the FDA that reviews new cancer products, voted 10 to zero in favour of Kymriah. Committee members called the responses remarkable and potentially paradigm changing. When the announcement broke, a crowd formed in the medical education centre of Penn Medicine, made up of ecstatic faculty and staff. There were banners and T-shirts. A remarkable thing happened was the tagline, above a cartoon image of a heroic T-cell.

Two months later, in October 2017, the FDA approved a second CAR-T formulation called Yescarta from Kite Pharma, a subsidiary of Gilead Sciences, to treat an aggressive blood cancer in adults called diffuse large B-cell lymphoma, the trial of which had shown a 54 percent complete response rate, meaning all signs of cancer had disappeared. In May 2018, Kymriah was approved to treat adults with non-Hodgkin lymphoma.

That year, the American Society of Clinical Oncology named CAR-T the Advance of the Year, beating out immunotherapy, which had won two years in a row. When I attended the last American Society of Hematology meeting in December 2018, CAR-T stole the show. Trying to get into CAR-T talks felt like trying to get a photo with a celebrity. Running five minutes late to one session meant facing closed doors. Others were standing room only.

With every slide, it became difficult to see over a sea of smartphones snapping photos. At one session I found a seat next to the oncologist from my hospital who treated Birzer. Look, she nudged me. Do you see all these non-member badges? I turned. Members were doctors like us who treated blood cancers. I couldnt imagine who else would want to be here. Who are they? I asked. Investors, she said. It felt obvious the moment she said it.

For patients, the dreaded c word is cancer. For oncologists, its cure. When patients ask, Ive noticed how we gently steer the conversation toward safer lingo. We talk about keeping the cancer in check. Cure is a dangerous word, used only when so much time has passed from her cancer diagnosis we can be reasonably certain its gone. But that line is arbitrary. We celebrate therapies that add weeks or months because the diseases are pugnacious, the biology diverse, and the threat of relapse looming. Oncologists are a tempered group, or so Ive learned, finding inspiration in slow, incremental change.

This was completely different. These were patients who would have otherwise died, and the trials were boasting that 54 to 81 percent were cancer-free upon initial follow-up. PET scans showed tumours that had speckled an entire body melt away. Bone marrow biopsies were clear, with even the most sensitive testing unable to detect disease.

The dreaded word was being tossed around could this be the cure weve always wanted?

When a new drug gets FDA approval, it makes its way into clinical practice, swiftly and often with little fanfare. Under the drug safety program REMS, hospitals offering CAR-T were obligated to undergo special training to monitor and manage side effects. As hospitals worked to create CAR-T programs, oncologists like me made the all too familiar transition from first-time user to expert.

It was May 2018 when I rotated through my hospitals unit and cared for my first patients on CAR-T. As I covered 24-hour shifts, I quickly learned that whether I would sleep that night depended on how many CAR-T patients I was covering. With each treatment, it felt like we were pouring gasoline on the fire of patients immune systems. Some developed high fevers and their blood pressures plummeted, mimicking a serious infection. But there was no infection to be found. When resuscitating with fluids couldnt maintain my patients blood pressures, I sent them to the ICU where they required intensive support to supply blood to their critical organs.

We now have a name for this effect cytokine release syndrome that occurs in more than half of patients who receive CAR-T, starting with Ludwig and Whitehead. The syndrome is the collateral damage of an immune system on the highest possible alert. This was first seen with other types of immunotherapy, but CAR-T took its severity to a new level. Usually starting the week after CAR-T, cytokine release syndrome can range from simple fevers to multi-organ failure affecting the liver, kidneys, heart, and more. The activated T-cells make and recruit other immune players called cytokines to join in the fight. Cytokines then recruit more immune cells. Unlike in the early trials at Penn, we now have two medicines to dampen the effect. Steroids calm the immune system in general, while a medication called tocilizumab, used to treat autoimmune disorders such as rheumatoid arthritis, blocks cytokines specifically.

Also read:The DNA Detectives Hunting the Causes of Cancer

Fortuity was behind the idea of tocilizumab: When Emily Whitehead, the first child to receive CAR-T, developed cytokine release syndrome, her medical team noted that her blood contained high levels of a cytokine called interleukin 6. Carl June thought of his own daughter, who had juvenile rheumatoid arthritis and was on a new FDA-approved medication that suppressed the same cytokine. The team tried the drug, tocilizumab, in Whitehead. It worked.

Still, we were cautious in our early treatments. The symptoms of cytokine release syndrome mimic the symptoms of severe infection. If this were infection, medicines that dampen a patients immune system would be the opposite of what youd want to give. There was another concern: Would these medications dampen the anti-cancer activity too? We didnt know. Whenever a CAR-T patient spiked a fever, I struggled with the question is it cytokine release syndrome, or is it infection? I often played it safe and covered all bases, starting antibiotics and steroids at the same time. It was counterintuitive, like pressing both heat and ice on a strain, or treating a patient simultaneously with fluids and diuretics.

The second side effect was even scarier: Patients stopped talking. Some, like Sharon Birzer spoke gibberish or had violent seizures.Some couldnt interact at all, unable to follow simple commands like squeeze my fingers. How? Why? At hospitals across the nation, perfectly cognitively intact people who had signed up to treat their cancer were unable to ask what was happening.

Our nurses learned to ask a standardised list of questions to catch the effect, which we called neurotoxicity: Where are we? Who is the president? What is 100 minus 10? When the patients scored too low on these quizzes, they called me to the bedside.

In turn, I relied heavily on alaminated booklet, made by other doctors who were using CAR-T, which we tacked to a bulletin board in our doctors workroom. It contained a short chart noting how to score severity and what to do next. I flipped through the brightly colour-coded pages telling me when to order a head CT-scan to look for brain swelling and when to place scalp electrodes looking for seizures. Meanwhile, we formed new channels of communication. As I routinely called a handful of CAR-T specialists at my hospital in the middle of the night, national consortiums formed where specialists around the country shared their experiences. As we tweaked the instructions, we scribbled updates to the booklet in pen.

I wanted to know whether my experience was representative. I came across an abstract and conference talk that explored what happened to 277 patients who received CAR-T in the real world, so I emailed the lead author, Loretta Nastoupil, director of the Department of Lymphoma and Myeloma at the University of Texas MD Anderson Cancer Center in Houston. Fortuitously, she was planning a trip to my university to give a talk that month. We met at a caf and I asked what her research found. Compared to the earlier trials, the patients were much sicker, she said. Of the 277 patients, more than 40 percent wouldnt have been eligible for the very trials that got CAR-T approved. Was her team calling other centres for advice? They were calling us, she said.

Patients included in clinical trials are carefully selected. They tend not to have other major medical problems, as we want them to survive whatever rigorous new therapy we put them through. Nastoupil admits some of it is arbitrary. Many criteria in the CAR-T trials were based on criteria that had been used in chemotherapy trials. These become standard languages that apply to all studies, she said, listing benchmarks like a patients age, kidney function, and platelet count. But we have no idea whether criteria for chemotherapy would apply to cellular therapy.

Now, with a blanket FDA approval comes clinical judgement. Patients want a chance. Oncologists want to give their patients a chance. Young, old, prior cancer, heart disease, or liver disease without strict trial criteria, anyone is fair game.

When I was making rounds at my hospital, I never wandered too far from these patients rooms, medically prepared for them to crash at any moment. At the same time, early side effects made me optimistic. A bizarre truism in cancer is that side effects may bode well. They could mean the treatment is working. Cancer is usually a waiting game, requiring months to learn an answer. Patients and doctors alike seek clues, but the only real way to know is waiting: Will the next PET scan show anything? What are the biopsy results?

CAR-T was fundamentally different from other cancer treatments in that it worked fast. Birzers first clue came just a few hours after her infusion. She developed pain in her lower back. She described it as feeling like she had menstrual cramps. A heavy burden of lymphoma lay in her uterus. Could the pain mean that the CAR-T cells had migrated to the right spot and started to work? Her medical team didnt know, but the lead doctors instinct was that it was a good sign.

Two days later, her temperature shot up to 102. Her blood pressure dropped. The medical team diagnosed cytokine release syndrome, as though right on schedule, and gave her tocilizumab.

Every day, the nurses would ask her questions and have her write simple sentences on a slip of paper to monitor for neurotoxicity. By the fifth day, her answers changed. She started saying things that were crazy, Johnson explained.

One of Birzers sentences was guinea pigs eat greens like hay and pizza. Birzer and Johnson owned two guinea pigs, so their diet would be something Birzer normally knew well. So Johnson tried to reason with her: They dont eat pizza. And Birzer replied, They do eat pizza, but only gluten-free.

Johnson remembers being struck by the certainty in her partners delirium. Not only was Birzer confused, she was confident she was not. She was doubling down on everything, Johnson described. She was absolutely sure she was right.

Johnson vividly remembers the evening before the frightening early-morning phone call that brought her rushing back to the hospital. Birzer had said there was no point in Johnson staying overnight; she would only watch her be in pain. So Johnson went home. After she did, the doctor came by multiple times to evaluate Birzer. She was deteriorating and fast. Her speech became more and more garbled. Soon she couldnt name simple objects and didnt know where she was. At 3 a.m., the doctor ordered a head CT to make sure Birzer wasnt bleeding into her brain.

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Fortunately, she wasnt. But by 7 a.m. Birzer stopped speaking altogether. Then she seized. Birzers nurse was about to step out of the room when she noticed Birzers arms and legs shaking. Her eyes stared vacantly and she wet the bed. The nurse called a code blue, and a team of more doctors and nurses ran over. Birzer was loaded with high-dose anti-seizure medications through her IV. But she continued to seize. As nurses infused more medications into her IV, a doctor placed a breathing tube down her throat.

Birzers saga poses the big question: Why does CAR-T cause seizures and other neurological problems? No one seemed to know. My search of the published scientific literature was thin, but one name kept cropping up. So I called her. Juliane Gust, a paediatric neurologist and scientist at Seattle Childrens Hospital, told me her investigations of how CAR-T affects the brain were motivated by her own experiences. When the early CAR-T trials opened at her hospital in 2014, she and her colleagues began getting calls from oncologists about brain toxicities they knew nothing about. Where are the papers? she remembered thinking. There was nothing.

Typically, the brain is protected by a collection of cells aptly named the blood-brain-barrier. But with severe CAR-T neurotoxicity, research suggests, this defence breaks down. Gust explained that spinal taps on these patients show high levels of cytokines floating in the fluid surrounding the spine and brain. Some CAR-T cells circulate in the fluid too, she said, but these numbers do not correlate with sicker patients. CAR-T cells are even seen in the spinal fluid of patients without any symptoms.

What does this mean? Gust interprets it as a patients symptoms having more to do with cytokines than the CAR-T cells. Cytokine release syndrome is the number one risk factor for developing neurotoxicity over the next few days, she said. The mainstay for neurotoxicity is starting steroids as soon as possible. In the beginning we didnt manage as aggressively. We were worried about impairing the function of the CAR-T, she added. Now we give steroids right away.

But the steroids dont always work. Several doses of steroids didnt prevent Birzer from seizing. The morning after Johnsons alarming phone call, after the meeting at the hospital when she learned what had happened, a chaplain walked her from the conference room to the ICU. The first day, Johnson sat by her partners bedside while Birzer remained unconscious. By the next evening, she woke up enough to breathe on her own. The doctors removed her breathing tube, and Birzer looked around. She had no idea who she was or where she was.

Birzer was like a newborn baby, confused and sometimes frightened by her surroundings. She frequently looked like she was about to say something, but she couldnt find the words despite the nurses and Johnsons encouragement. One day she spoke a few words. Eventually she learned her name. A few days later she recognised Johnson. Her life was coming back to her, though she was still suspicious of her reality. She accused the nurses of tricking her, for instance, when they told her Donald Trump was president.

She took cues from the adults around her on whether her actions were appropriate. The best example of this was her I love you phase. One day, she said it to Johnson in the hospital. A few nurses overheard it and commented on how sweet it was. Birzer was pleased with the reaction. So she turned to the nurse: I love you! And the person emptying the trash: I love you! Months later, she was having lunch with a friend who asked, Do you remember when you told me you loved me? Birzer said, Well, I stand by that one.

When she got home, she needed a walker to help with her shakiness on her feet. When recounting her everyday interactions, she would swap in the wrong people, substituting a friend for someone else. She saw bugs that didnt exist. She couldnt hold a spoon or a cup steady. Johnson would try to slow her down, but Birzer was adamant she could eat and drink without help. Then peas would fly in my face, Johnson said.

Patients who experience neurotoxicity fall into one of three categories. The majority are impaired but then return to normal without long-term damage. A devastating handful, less than 1 percent, develop severe brain swelling and die. The rest fall into a minority that have lingering problems even months out. These are usually struggles to think up the right word, trouble concentrating, and weakness, often requiring long courses of rehabilitation and extra help at home.

As Birzer told me about her months of rehab, I thought how she did seem to fall somewhere in the middle among the patients Ive treated. On one end of the spectrum was the rancher who remained profoundly weak a year after his infusion. Before CAR-T, he walked across his ranch without issue; six months later, he needed a walker. Even with it, he fell on a near weekly basis. On the other end was the retired teacher who couldnt speak for a week she would look around her ICU room and move her mouth as though trying her hardest and then woke up as though nothing happened. She left the hospital and instantly resumed her life, which included a recent trip across the country. In hindsight, I remember how we worried more about giving the therapy to the teacher than the rancher, as she seemed frailer. Outcomes like theirs leave me with a familiar humility I keep learning in new ways as a doctor: We often cant predict how a patient will do. Our instincts can be just plain wrong.

I asked Gust if we have data to predict who will land in which group. While we can point to some risk factors higher burdens of cancer, baseline cognitive problems before therapy the individual patient tells you nothing, she confirmed.

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So we wait.

Doctors like me who specialise in cancer regularly field heart-wrenching questions from patients. They have read about CAR-T in the news, and now they want to know: What about me? What about my cancer?

So, who gets CAR-T? That leads to the tougher question who doesnt? That depends on the type of cancer and whether their insurance can pay.

CAR-T is approved to treat certain leukaemias and lymphomas that come from the blood and bone marrow. Since the initial approval, researchers have also set up new CAR-T trials for all sorts of solid tumours from lung cancer to kidney cancer to sarcoma. But progress has been slow. While some promising findings are coming from the lab and in small numbers of patients on early phase trials, nothing is yet approved in humans. The remarkable responses occurring in blood cancers just werent happening in solid tumours.

Cancer is one word, but its not one disease. Its easier to prove why something works when it works than show why it doesnt work when it doesnt work, said Saar Gill, a hematologist and scientist at the University of Pennsylvania who co-founded a company called Carisma Therapeutics using CAR-T technology against solid tumours. That was his short answer, at least. The longer answer to why CAR-T hasnt worked in solid cancers involves what Gill believes are two main barriers. First, its a trafficking problem. Leukaemia cells tend to be easier targets; they bob through the bloodstream like buoys in an ocean. Solid tumours are more like trash islands. The cancer cells stick together and grow an assortment of supporting structures to hold the mound together. The first problem for CAR-T is that the T-cells may not be able to penetrate the islands. Then, even if the T-cells make it in, theyre faced with a hostile environment and will likely die before they can work.

At Carisma, Gill and his colleagues look to get around these obstacles though a different immune cell called the macrophage. T-cells are not the only players of the immune system, after all. Macrophages are gluttonous cells that recognise invaders and engulf them for destruction. But studies have shown they cluster in solid tumours in a way T-cells dont. Gill hopes genetically engineered macrophages can be the stowaways that sneak into solid tumour and attack from the inside out.

Another big challenge, even for leukaemias and lymphomas, is resistance, where the cancers learn to survive the CAR-T infusion. While many patients in the trials achieved remission after a month, we now have two years worth of data and the outlook isnt as rosy. For lymphoma, that number is closer to 40 percent. Patients celebrating cures initially are relapsing later. Why?

The CAR-T cells we use target a specific protein on cancer cells. But if the cancer no longer expresses that protein, that can be a big problem, and were finding thats exactly whats happening. Through blood testing, we see that many patients who relapse lose the target.

Researchers are trying to regain the upper hand by designing CAR-Ts to target more than one receptor. Its an old idea in a new frame: An arms race between our medicines and the illnesses that can evolve to evade them. Too much medical precision in these cases is actually not what we want, as it makes it easier for cancer to pinpoint whats after it and develop an escape route. So, the reasoning goes, target multiple pieces at once. Confuse the cancer.

Then theres the other dreaded c word: Cost. Novartis Kymriah runs up to $475,000 while Kite Pharmas Yescarta is $373,000. That covers manufacturing and infusion. Not included is the minimum one-week hospital stay or any complications.

They are daunting numbers. Some limitations on health care we accept maybe the patients are too sick; maybe they have the wrong disease. The wrong cost is not one we as a society look kindly upon. And drug companies shy away from that kind of attention.

Cost origins in medicine are notoriously murky. Novartis, confident in its technology, made an offer to offset the scrutiny in CAR-T. If the treatment didnt work after one month, the company said it wouldnt send a bill.

Not everyone agrees that cost is an issue. Gill, for example, believes the concern is over-hyped. Its not a major issue, he told me over the phone. Look, of course [with] health care in this country, if you dont have insurance, then youre screwed. That is no different when it comes to CAR-T as it is for anything else, he said. The cost conversation must also put CAR-T in context. Gill went on to list what these patients would be doing otherwise months of chemotherapy, bone marrow transplants, hospital stays for cancer-associated complications and the associated loss of income as patients and caregivers miss work. These could add up to far more than a one-time CAR-T infusion. A bone marrow transplant, for example, can cost from $100,000 to more than $300,000. The cancer-fighting drug blinatumomab, also used to treat relapsed leukaemia, costs $178,000 a year. Any discussion of cost is completely irresponsible without weighing the other side of the equation, Gill said.

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How the system will get on board is another question. Logistics will be an issue, Gill conceded. The first national Medicare policy for covering CAR-T was announced in August 2019, two years after the first product was approved. The Centres for Medicare and Medicaid Services has offered to reimburse a set rate for CAR T-cell infusion, and while this figure was recently raised, it remains less than the total cost. Despite the expansion of medical uses, at some centres referrals for CAR-T are dropping as hospitals worry its a net loss. And while most commercial insurers are covering CAR-T therapies, companies less accustomed to handling complex therapies can postpone approval. Ironically, the patients considering CAR-T are the ones for whom the window for treatment is narrowest. A delay of even a few weeks can mean the difference between a cure and hospice.

This, of course, poses a big problem. A breakthrough technology is only as good as its access. A major selling point of CAR-T besides the efficacy is its ease. Its a one-and-done treatment. Engineered T-cells are intended to live indefinitely, constantly on the alert if cancer tries to come back. Compare that to chemotherapy or immunotherapy, which is months of infusions or a pill taken indefinitely. CAR-T is more akin to surgery: Cut it out, pay the entire cost upfront, and youre done.

Birzer was lucky in this respect. I asked her and Johnson if cost had factored into their decision to try CAR-T. They looked at each other. It wasnt an issue, said Johnson. They remembered getting a statement in the mail for a large sum when they got home. But Birzer had good insurance. She didnt pay a cent.

One year after Birzers infusion, I met her and Johnson at a coffee shop near their home in San Francisco. They had saved a table. Johnson had a newspaper open. Birzer already had her coffee, and I noticed her hand trembling as she brought it to her mouth. She described how she still struggles to find exactly the right words. She sometimes flings peas. But shes mostly back to normal, living her everyday life. She has even returned to her passion, performing stand-up comedy, though she admitted that at least for general audiences: My jokes about cancer didnt kill.

People handed a devastating diagnosis dont spend most of their time dying. They are living, but with a heightened awareness for a timeline the rest of us take for granted. They sip coffee, enjoy their hobbies, and read the news while also getting their affairs in order and staying on the lookout, constantly, for the next treatment that could save them.

Hoping for a miracle while preparing to die are mutually compatible ideas. Many of my patients have become accustomed to living somewhere in that limbo. It is humbling to witness. They hold out hope for a plan A, however unlikely it may be, while also adjusting to the reality of a plan B. They live their lives; and they live in uncertainty.

I see patients in various stages of this limbo. In clinic, I met a man with multiple myeloma six months after a CAR-T trial that supposedly cured him. He came in with a big smile but then quietly began praying when it was time to view PET results. He asked how the other patients on the trial were doing, and I shared the stats. While percentages dont say anything about an individual experience, theyre also all patients have to go on. When someone on the same treatment dies, its shattering for everyone. Was one person the exception, or a harbinger anothers fate? Who is the outlier?

I look at these patients and think a sober truth: Before CAR-T, all would likely die within six months. Now, imagine taking 40 percent and curing them. Sure, a naysayer might point out, its only 40 percent. Whats the hype if most still succumb to their cancer? But there was nothing close to that before CAR-T. I agree with how Gill described it: I think CAR-T cells are like chemotherapy in the 1950s. Theyre not better than chemotherapy theyre just different. For an adversary as tough as cancer, well take any tool we can get.

There remain many questions. Can we use CAR-T earlier in a cancers course? Lessen the side effects? Overcome resistance? Streamline manufacturing and reimbursement? Will it work in other cancers? Patients will sign up to answer.

For now, Birzer seems to be in the lucky 40%. Her one-year PET scan showed no cancer. I thought of our last coffee meeting, where I had asked if she ever worried she wouldnt return to normal. She didnt even pause. If youre not dead, she said, youre winning.

This article was originally published on Undark. Read the original article.

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Behind the Scenes of a Radical New Cancer Cure - The Wire

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

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

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Leading Manufacturers and their Strategies to see Distinctly Growth in Antidiuretic Drugs Market by 2025 - Health News Office

A gene therapy for a rare blood disorder has shown what the manufacturer calls the first evidence of long-term improvement associated with the disease.

New York-based Rocket Pharmaceuticals said Thursday that it had presented long-term follow-up data from the Phase I/II study of RP-L102, its gene therapy for Fanconi anemia, at the annual congress of the European Society of Cell and Gene Therapy in Barcelona, Spain. The company said it represented the first evidence of long-term improvement and stabilization in blood counts and durable mosaicism among patients who received the therapy without the use of the conditioning regimens normally used for allogeneic stem cell transplants, which the company calls Process A.

Shares of Rocket were up slightly on the Nasdaq following the news. RP-L102 is a lentiviral vector-based gene therapy. Most other gene therapies in development, and both of the currently marketed ones Spark Therapeutics Luxturna (voretigene neparvovec-rzyl) and Novartis Zolgensma (onasemnogene abeparvovec-xioi) are adeno-associated viral vector-based.

According to the data, representing four of nine patients, there were improved blood counts and long-term bone marrow mitomycin C (MMC) resistance, thereby indicating durable phenotypic correction. The data met or exceeded a 10 percent threshold that the company said the Food and Drug Administration and European Medicines Agency had agreed to for its upcoming Phase II registration study, for which it plans to start enrolling patients by the end of the year.

FA is a rare, genetic bone marrow failure disorder, half of whose patients are diagnosed before the age of 10, while about 10 percent of patients are diagnosed as adults, according to the National Organization for Rare Disorders. It is often associated with progressive deficiency of production of red and white blood cells and platelets in the bone marrow and can eventually lead to certain solid and liquid tumor cancers. It occurs in 1-in-136,000 births and is more common among Ashkenazi Jews, Spanish Roma and black South Africans.

These results indicate the feasibility of engraftment in FA patients using autologous, gene corrected [hematopoietic stem cells] in the absence of any conditioning regimen, said Dr. Juan Bueren, scientific director of the FA gene therapy program at Spains Center for Energy, Environmental and Technological Research, in a statement. This indicates the potential of this therapeutic approach as a definitive hematologic treatment, while avoiding the burdensome side effects associated with allogeneic transplant, including the risk of post-transplant mortality and a substantially higher risk of head and neck cancer.

Photo: virusowy, Getty Images

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Rocket's gene therapy shows long-term efficacy in rare blood disorder - MedCity News

Assessing the challenges

As part of the CRA analysis, the team reached out to a range of industry stakeholders including experts in drug development and commercialisation, academic researchers, investors and payers for insights about emerging annuity pricing models and the challenges they can present.

Research also included a global review of launch dates and regulatory information related to several cell and gene therapies.

Input from industry insiders and experts identified a range of concerns associated with annuity payment models, including a potentially significant impact on cost of capital and company valuations, costs and burden of long-term patient monitoring, the need to make reimbursement decisions based on very limited long-term clinical data, and the need to consider entirely new business models and forecasting strategies based on extended payment schedules.

Despite the challenges, some cell and gene therapy companies have already launched products based on annuity pricing models. GlaxoSmithKline (GSK) launched its gene therapy STRIMVELIS, a one dose treatment for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), in Europe in 2016 with an outcomes-based annuity pricing model.

Under the terms of this model, payments to GSK by payers are spread out over a pre-determined timeline for each treated patient. The model confirms that GSK must return a portion of the reimbursement to the Italian Medicines Agency if the drug does not demonstrate a sufficient level of efficacy based on pre-determined outcomes measures.

In their assessment, based on the mutually determined outcomes measures, GSK projected that aboutone in six treatments on average might need to be partially refunded.

The financial impact of annuity payment models

Annuity models require manufacturers to consider several factors that can increase their own operational costs as well as the cost of capital. With annuity models, the risk that a payer might not be a viable long-term business entity or may contest a payment schedule could mean a potentially devastating disruption in revenue fora manufacturer.

While the use of insurer-backed annuity payments as collateral for secured loans can help mitigate this risk, in considering this option manufacturers need to carefully consider multiple factors, including trends in interest rates and the duration of the annuity contract, the cost of capital based on a lenders fees, operational repayment milestones and the expected costs of goods.

Both manufacturers and payers must also complete risk assessments based on available data, which will often be more limited than data used in traditional outcomes and risk assessments.

Banks and other lenders will also likely complete their own due diligence in evaluating many factors, including levels of payer interest, patient outcomes measures and the terms of annuity contracts to determine their own acceptable level of risk, often with limited relevant experience. In many cases, lenders will also require drug developers to provide audited cash flow statements.

Optimal strategies for mitigating risk

As progress in the development of cell and gene therapies continues to advance, payers andhealth systems can anticipate the introduction of many new high-priced drugs in the near future.

While annuity pricing models are already a widely considered option to address the unique reimbursement issues associated with cell and gene therapies, developing and implementing these plans successfully will require careful analysis and successful efforts in building consensus among all stakeholders.

The risks may be especially acute among biotechnology companies with only one drug on the market. Manufacturers will need to bring together the full range of resources and expertise necessary to assess the impact these models will have on capacity, cash flow and long-term business planning.

To achieve this goal, drug developers will need to reach out to a broad set of stakeholders for guidance and expertise.

They may need to work closely with finance providers such as banks and other lenders and seek collaborations with consultants in market access early in a development programme to begin what is likely to be a long-term initiative in planning and programme execution.

It is likely that there will not be one simple solution. Effective approaches in reimbursement of cell and gene therapies may need to include elements from several potential models including, but not limited to, annuity-based models.

But the progress in research means that development of innovative pricing models that optimise benefits and balance risks for manufacturers, payers and health systems will become increasingly time sensitive as more paradigm-changing cell and gene therapies advance toward commercialisation.

The views expressed herein are the authors and not those of Charles River Associates (CRA) or any of the organisations with which the author is affiliated.

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Assessing the risks in annuity pricing models - PMLiVE

The National Institutes of Health and the Bill and Melinda Gates Foundation will together invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV with an attribute even rarer in the world of genetic medicine than efficacy, the groups announced on Wednesday: The cures, they vowed, will be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.

The effort reflects growing concerns that scientific advances in genetic medicine, both traditional gene therapies and genome-editing approaches such as CRISPR, are and will continue to be prohibitively expensive and therefore beyond the reach of the vast majority of patients. Spark Therapeutics Luxturna, a gene therapy for a rare form of blindness, costs $425,000 per eye, for instance, and genetically engineered T cells (CAR-Ts) to treat some blood cancers cost about the same.

With CRISPR-based treatments already being tested in clinical trials for sickle cell disease, the blood disorder beta thalassemia, and another form of blindness, and with additional CRISPR treatments in development, scientists, ethicists, and health policy experts have grown increasingly concerned that the divide between haves and have-nots will grow ever-wider.

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Gene-based treatments are largely inaccessible to most of the world by virtue of the complexity and cost of treatment requirements, which currently limit their administration to hospitals in wealthy countries, the NIH said in a statement. To help right that, its collaboration with the Gates Foundation aims to develop curative therapies that can be delivered safely, effectively and affordably in low-resource settings.

Scientists whose research focuses on gene-based cures welcomed the infusion of funding and the recognition that genetic cures are on track to be unaffordable to the majority of patients. But they noted one irony. The most effective sickle cell drug, hydroxyurea, has hardly even been studied in sub-Saharan Africa, let alone made widely available. Yet a 2019 study found that giving children the drug cut their death rate by two-thirds and halved the pain crises that are common in sickle cell disease, caused by misshapen red blood cells that cannot flow through blood vessels.

The NIH-Gates collaboration is tremendously exciting and has the potential to have a great impact on sickle cell disease in sub-Saharan Africa, said Dr. Vijay Sankaran of the Dana-Farber/Boston Childrens Cancer and Blood Disorders Center, who has done pioneering research on genetic cures for the disease. But my hesitation is that even the inexpensive therapies we have today, such as hydroxyurea, are largely unavailable there. The question is, how do we best approach this disease, with therapies that are working today or with genetic therapies that might work?

The same concerns surround HIV. Very inexpensive less than $100 per year in the U.S. antiretroviral drugs can keep the virus in check, but only 67% of HIV-positive adults and 62% of HIV-positive in children in east and southern Africa are estimated to be on antiretroviral treatment.

The new collaboration aims to move gene-based cures into clinical trials in the U.S. and countries in sub-Saharan Africa within the next seven to 10 years, and to eventually make such treatments available in areas hardest hit by sickle cell disease and HIV/AIDS. The idea is to focus on access, scalability, and affordability to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries, NIH Director Francis Collins said in a statement. We aim to go big or go home. But the challenge is enormous, he told reporters on Wednesday: Im not going to lie. This is a bold goal.

An estimated 95% of the 38 million people with HIV live in the developing world, with 67% in sub-Saharan Africa. Up to 90% of children with sickle cell disease in low-income countries die before they are 5 years old. In the U.S., the life expectancy for people with sickle cell disease is in the low 40s.

The NIH and the Gates Foundation will fund research to identify potential gene-based cures for sickle cell and HIV, and also work with groups in Africa to test those cures in clinical trials.

The science of genetic cures for both diseases is within reach, experts say. CRISPR Therapeutics and Vertex (VRTX) are already running a clinical trial for sickle cell disease, using the CRISPR genome editor to do an end-run around the disease-causing mutation in the hemoglobin gene: The therapy releases the brake on red blood cells production of fetal hemoglobin, whose production shuts off in infancy but which does not have the sickling damage of adult hemoglobin.

Developing effective, safe genetic cures for sickle cell and HIV would be only a first step, however. As currently conceived, such therapies require advanced medical facilities to draw blood from patients, alter their cells genomes in a lab, give the patients chemotherapy to kill diseased blood-making cells, and then perform whats essentially a bone marrow transplant, followed by monitoring patients in a hospital for days to prevent infection and provide intensive medical support, said Dr. Dan Bauer, a sickle cell expert at Boston Childrens.

He called the NIH-Gates effort terrific, but cautioned that delivering advanced gene therapies requires tremendous effort, extended hospitalization, and large supplies of blood products. All of those requirements mean that even if a CRISPR-based cure for sickle cell disease or HIV were provided at cost, there will still be barriers to access.

Recognizing that, Collins said, a genetic cure would have to be given directly into patients (in vivo), presumably through an infusion, rather than by treating blood or other cells removed from patients and genetically transformed in a lab (ex vivo). That could avoid the resources needed for and the complications that can occur with ex vivo therapies, said Sankaran, who has discussed the approach with Gates officials.

This story has been updated with additional comments.

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NIH, Gates Foundation aim to bring genetic cures to the poor - STAT