StemCell Therapy

Market Study Report, LLC, adds a thorough analysis of the ' Hemophilia Gene Therapy market', offering a comprehensive report emphasizing every vital aspect of the business vertical. The study has collectively presented refined data characterized by market valuation, SWOT analysis, market participants, regional segmentation, and revenue forecasts, enabling stakeholders to make logical business decisions.

The research report on Hemophilia Gene Therapy market comprises of driving factors and trends that will impact the industry growth during the forecast period. Thorough examination of market remuneration with reference to regional terrain is entailed in the report. It also mentions the challenges this business sphere will face as well as provide information regarding potential growth prospects. Besides, the report also includes COVID-19 case studies to deliver a better picture of this business sphere to all industry partakers.

Request a sample Report of Hemophilia Gene Therapy Market at:https://www.marketstudyreport.com/request-a-sample/2931028?utm_source=algosonline.com&utm_medium=TS

Pivotal pointers from COVID-19 effect assessment:

Overview of regional terrain:

Other important highlights from the Hemophilia Gene Therapy market report:

Ask for Discount on Hemophilia Gene Therapy Market Report at:https://www.marketstudyreport.com/check-for-discount/2931028?utm_source=algosonline.com&utm_medium=TS

Key features of this report are:

Table of Contents:

For More Details On this Report: https://www.marketstudyreport.com/reports/global-hemophilia-gene-therapy-market-report-2020-by-key-players-types-applications-countries-market-size-forecast-to-2026-based-on-2020-covid-19-worldwide-spread

Related Reports:

2. Global Ceiling-Mounted Supply Pendants With Column Market Report 2020 by Key Players, Types, Applications, Countries, Market Size, Forecast to 2026 (Based on 2020 COVID-19 Worldwide Spread)Read More: https://www.marketstudyreport.com/reports/global-ceiling-mounted-supply-pendants-with-column-market-report-2020-by-key-players-types-applications-countries-market-size-forecast-to-2026-based-on-2020-covid-19-worldwide-spread

Read More Reports On: https://www.marketwatch.com/press-release/melamine-market-by-source-product-type-process-and-region-forecast-to-2026-2020-09-21?tesla=y

Contact Us:Corporate Sales,Market Study Report LLCPhone: 1-302-273-0910Toll Free: 1-866-764-2150 Email: [emailprotected]

Original post:
Hemophilia Gene Therapy Market Comprehensive Analysis, Growth Forecast from 2020 to 2025 - AlgosOnline

When James Landon received a mantle cell lymphoma (MCL) diagnosis in 2017, he was told his disease was indolent, meaning it was slow growing and didnt need to be treated right away, especially since he was feeling fine.

But that changed in 2019. Landon, 50, an attorney in Tucson, Arizona, started to feel tired all the time. His physicians discovered that his white blood cell count was sky-high and his spleen was enlarged.

The first-line treatment for MCL has long been high doses of chemotherapy, often followed by a stem cell transplant. But when Landon traveled to The University of Texas MD Anderson CancerCenter in Houston to explore his options, he was offered an alternative: an experimental regimen combining the drugs Imbruvica (ibrutinib) and Rituxan (rituximab). Imbruvica blocks a protein called Bruton tyrosine kinase (BTK), which is a driver of MCL, and Rituxan is an antibody that targets overactive B cells of the immune system that have been implicated in the disease.

Five months after starting the combination four Imbruvica pills every morning and once-weekly infusions of Rituxan Landons positron emission tomography (PET) scan showed no evidence of disease. If he stays clear, hell undergo a short course of chemotherapy and then move to a maintenance therapy of one year of Ibrutinib and two of Rituxan.

Having this option has been fantastic, in my opinion, because the drugs so far have worked well for me, with no toxicity, says Landon, who adds that he has plenty of energy to work full time and play with his 10-year-old son.

BTK inhibitors and immunotherapy are among the newer therapeutic options for patients with MCL that are greatly improving the outlook for long-term survival. In addition to these targeted drugs, Tecartus (brexucabtagene autoleucel) the first cell-based gene therapy for MCL in patients who havent responded to or who have relapsed following other kinds of treatment was approved by the Food and Drug Administration (FDA) in July and is a one-time personalized treatment made from patients own immune cells.

We now have several good nonchemotherapy options for treating MCL, says Dr. Anthony Nguyen, a professor at the University of Nevada, Las Vegas School of Medicine and a medical oncologist at Comprehensive Cancer Centers of Nevada. We may be able to tell patients we can treat them without toxic chemotherapy, which can be reassuring, particularly for older patients.

MCL is a subtype of non-Hodgkin lymphoma (NHL) thats characterized by the overproduction of a protein called cyclin D1. In about 85% of patients, that overproduction is caused by a genetic abnormality called reciprocal chromosomal translocation, which can be detected with diagnostic testing of tumor samples. MCL accounts for about 6% of all NHL diagnoses and is more common in men than in women, according to the Leukemia & Lymphoma Society.

The standard first-line treatment for MCL is high-dose chemotherapy, often with a four-medicine regimen called hyper-CVAD, followed by a stem cell transplant with a patients own cells or with those from a donor. The regimen often puts patients into long-term remissions, but the side effects including nausea, mouth ulcers and kidney damage can be difficult or even dangerous, particularly for patients with other illnesses.

The newer medicines and cell therapy were approved by the FDA to treat patients with MCL who dont respond to chemotherapy and transplants or who relapse. But as physicians gain more experience with these therapies, theres a growing interest in using them earlier in the treatment process to not only improve the chances of long-term remissions, but also to improve the quality of life for patients by sparing them from harsh side effects.

The FDA approved the first BTK inhibitor to treat MCL, Imbruvica, in 2013, based on a study showing an overall response rate (meaning the disease responded to treatment) of 68% and a complete response rate (the disappearance of all signs of cancer) of 21%. The average period that patients lived without their disease progressing was more than a year, and side effects were mild stomach upset and fatigue.

The more recently approved BTK inhibitors Calquence (acalabrutinib) and Brukinsa (zanubrutinib) have improved on those response rates. Patients receiving Brukinsa in a late-stage trial, for example, had an overall response rate of 89% and a complete response rate of 59%.

Another targeted treatment, Venclexta (venetoclax) is also being studied in MCL. Venclexta targets the protein BCL2, which promotes cell survival and is abnormally elevated in MCL helping to drive progression of the disease. In a small trial of Venclexta, 75% of patients with relapsed MCL responded to the drug, 21% of whom had complete responses. Theres even more interest in studying BCL2 inhibition in combination with BTK blockers. In a recent study of Venclexta combined with Imbruvica, the median progression-free survival time was 29 months.

This is an extremely promising combination, says Dr. Abhijeet Kumar, assistant professor in the division of hematology and oncology at the University of Arizona College of Medicine. Kumar is an investigator in an ongoing trial of Venclexta and Imbruvica in MCL.

There is, however, a risk of increased side effects when targeted treatments are combined. Imbruvica can cause bleeding, for example, and both drugs can lower neutrophil (a type of white blood cell) counts. Venclexta is also known to cause tumor lysis syndrome, a rapid release of tumor cells into the bloodstream that can endanger the kidneys and other organs. Still, so far, the combination seems to be well-tolerated, Kumar says.

Another two-drug treatment for MCL that has generated enthusiasm among oncologists treating the disease is dubbed R-squared because it combines Rituxan with Revlimid (lenalidomide), a drug that works by boosting the immune systems T cells and natural killer cells, which work together to attack cancer.

In a study of R-squared in 38 patients with newly diagnosed MCL, the progression-free survival rate after three years was 80% and overall survival reached 90%. The response is durable, says Dr. Bijal Shah, an associate member in the department of malignant hematology at Moffitt Cancer Center and one of the study investigators. During the R-squared trial, patients typically stayed on the combination for three years and then took Revlimid alone as long as the disease remained stable.

Similar benefits have been seen with a combination of Velcade (bortezomib), Revlimid and chemotherapy, a regimen called VR-CAP. Velcade is a targeted drug that works by disrupting the growth of MCL cells and prompting them to die.

In a trial of patients with untreated MCL, adding Velcade to Revlimid and chemotherapy extended progression-free survival by 37%. The addition of Velcade more than doubled the median duration of response to 41 months.

Both R-squared and VR-CAP have moved into the frontline treatment setting, Shah says. With that, were able to see really pronounced clinical benefits. Weve seen very long remissions, he says.

Several other combination strategies also are being investigated for MCL, including some that incorporate the drug Treanda (bendamustine), which works by causing DNA damage to cancer cells. In one study, combining Treanda with Rituxan improved progression-free survival rates over chemotherapy in patients with MCL or indolent NHL. More than 15 studies are now underway combining Treanda with Rituxan and other MCL treatments.

Even though targeted and combination treatments have extended survival times in MCL, most patients eventually relapse. Now theres a new option for those patients: Tecartus, a personalized therapy made from a patients own immune cells. The one-time treatment was approved by the FDA to treat patients who have not responded or have relapsed following other kinds of treatment.

Tecartus is a chimeric antigen receptor (CAR)-T cell therapy similar to Yescarta (axicabtagene ciloleucel), a CAR-T cell therapy approved by the FDA in 2017 to treat some types of large B-cell lymphomas. Like Yescarta, Tecartus targets CD19, a protein thats prevalent in cancerous B cells. Tecartus is made by extracting T cells from the blood of the patient with MCL and genetically modifying those cells to recognize and attack the cancer. In addition, the cells are put through an enrichment process designed to prevent them from wearing down before they are infused back into the patient.

In the clinical trial that led up to the approval, 87% of patients responded to Tecartus and 62% went into remission. Side effects, which included the immune overreaction known as cytokine release syndrome and neurological events, were manageable during the clinical trial, says Dr. Michael Wang, a professor in the department of lymphoma and myeloma at The University of Texas MD Anderson Cancer Center and one of the clinical trial investigators.

Experience with previously approved CAR-T cell treatments led to the widespread use of anti-inflammatory medications such as interleuken-6 inhibitors and steroids to treat cytokine release syndrome, Wang says. We have a variety of supportive measures to manage the side effects, he says.

With the approval of Tecartus, Wang says oncologists can envision a flattening of the survival curve in MCL. Its an option for people who become resistant to targeted therapies and chemotherapy, he says. Its very possible we will be able to put some people into long- term remissions.

Bob Brixner, a 20-year survivor of MCL, has been watching all the new developments with interest. When he received an MCL diagnosis in 2000, he had no choice but to endure chemotherapy followed by a stem cell transplant with his own cells. When he relapsed in 2004, he was prescribed a more intense chemotherapy regimen, followed by a stem cell transplant from an unrelated donor.

Hes grateful the second treatment put him in a long-term remission, but he still remembers the brutal side effects, which included extreme fatigue and a bout with pneumonia. And with the transplants, my immune system didnt come back 100%, says Brixner, 70, a retired Chicago public schoolteacher. Nowadays if I catch a cold, instead of lasting a week, it will last three.

Brixner advises all newly diagnosed patients to ask a lot of questions about their treatment choices and to get a second opinion. I think its really important to be an informed patient, he says, especially since there are so many new choices. Some patients may not have to go through what I did, he says. Im delighted.

Excerpt from:
Advances in the Treatment of Mantle Cell Lymphoma are Greatly Improving the Long-Term Outlook for Patients - Curetoday.com

Axovant Gene Therapies Ltd. (NASDAQ:AXGT) has announced the signing of a strategic partnership with leading Contract Development and Manufacturing Organisation, Viralgen.

Viralgen, which is leveraging AskBios tech platforms, has supported Axovants all manufacturing aspects of the AAV program, including large scale manufacturing, quality control, and fill-finish GMP-certified environment that has been tailored to bring therapies to the market quickly. As per the terms of the agreement, Axovant will get access to manufacturing resources of its AAV-based gene therapy programs with adequate capacity to support the development and eventual commercialization of the therapies. Axovant has is developing the AXO-AAV-GM1 for GM gangliosidosis and AXO-AAV-GM2 for GM2 gangliosidosis.

Gavin Corcoran, the Chief R & D Officer of the company, stated that they are pleased to collaborate with Viralgen, a leading manufacturer of AAV-based gene therapies. He said that the partnership will enable Axovant to produce their novel gene therapies that can improve or stabilize the course of GM1 and GM2 gangliosidoses on a commercial scale. The GM1 and GM2 gangliosidosis are two problematic pediatric diseases that do not have any approved treatment alternatives.

Corcoran said that the partnership with Viralgen offers the company access to a huge facility and experienced team that is focused on delivering tech that will speed up the development of life-saving therapies. He explained that the approach is important as the company continues to advance the AAV programs with data from the current Phase1/2 AXO-AAV-GM1 study expected in Q4 2020. The company is also expecting to file an IND clearance for AXO-AAV-GM2 before the end of this year.

Viralgen CEO Javier Garcia stated that they were delighted to partner with Axovant, and they are looking forward to offering priority access and support to advance AAV gene therapies for the devastating podiatric diseases. He added that the companys scalable and flexible production platform will be vital in complementing the development efforts of Axovant through commercialization from clinical trials.

Visit link:
Axovant Gene Therapies Ltd. (NASDAQ:AXGT) Partners With Viralgen To Scale Production Of Its AAV Gene Therapies - BP Journal

Garner Insights has added a new report titled, Global Gene Therapy Market Professional Report 2026 to its vast repository of research reports. This is a thorough report focused on the current and future prospects of the Global Gene Therapy Market. The report offers data of previous years along with an in-depth analysis from 2020 to 2026 on the basis of revenue (USD Billion). Besides, the report offers a comprehensive analysis about the factors driving and restraining the growth of the market coupled with the impact they have on the demand over the forecast period.

The market is segmented into different sections such as: by product type, by technology type, by application, by end-users, by deployment mode, and by key geography. The report then employs market breakdown and data triangulation procedures to complete the overall market engineering process and arrive at the exact statistics for all segments and sub-segments. The report on the Global Gene Therapy Markethas been curated by analyzing the top players functioning in the market. In order to get an in-depth analysis of the market, the report carried out SWOT analysis, Porters five forces analysis, and Pestel analysis.

Request Sample Report of Global Gene Therapy Market @ (https://garnerinsights.com/Gene-Therapy-Market-Research-Global-StatusForecast-by-Geography-TypeApplication-2016-2026#request-sample)

The report is segmented as follows:

Top Key Players: Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical, Advantagene

By Product Type:Ex vivo, In vivo

By Application:Cancer Diseases, Monogenic Diseases, Infectious Diseases, Cardiovascular Diseases, Others

By Regions:

This report forecasts revenue growth at the global, regional, and local levels and provides an analysis of the most recent industry trends from 2020 to 2026 in each of the segments and sub-segments.In addition, the report highlights the impact of COVID-19 on the Global Gene Therapy Market. Some of the major geographies included in the market are given below:

North America (U.S., Canada)Europe (U.K., Germany, France, Italy)Asia Pacific (China, India, Japan, Singapore, Malaysia)Latin America (Brazil, Mexico)Middle East & Africa

Request for Discount on report @ (https://garnerinsights.com/Gene-Therapy-Market-Research-Global-StatusForecast-by-Geography-TypeApplication-2016-2026#discount)

Key Questions Answered in the Report:

What is the current scenario of the Global Gene Therapy Market? How is the market going to prosper throughout the next 6 years?What are the emerging technologies that are going to profit the market?What is the historical and the current size of the Global Gene Therapy Market?Which segments are the fastest growing and the largest in the market? What is their market potential?What are the driving factors contributing to the market growth during the short, medium, and long term?What are the lucrative opportunities for the key players in the market?Which are the key geographies from the investment perspective?What are the major strategies adopted by the leading players to expand their market shares?Who are the distributors, traders and dealers of Global Gene Therapy market?What are sales, revenue, and price analysis by types and applications of market?

Full Description @ (https://garnerinsights.com/Gene-Therapy-Market-Research-Global-StatusForecast-by-Geography-TypeApplication-2016-2026#description)

See the rest here:
Impact of Covid-19 on Gene Therapy Market Trends by Countries, Type and Application | Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche...

Management to Highlight Progress in Bone Marrow Transplant (BMT) and Chimeric Antigen Receptor T Cell (CAR T) Business Collaborations

Tel Aviv, Israel, Sept. 21, 2020 (GLOBE NEWSWIRE) -- Cellect Biotechnology Ltd. (NASDAQ: APOP”), a developer of innovative technology which enables the functional selection of cells facilitating safer and more efficacious cell and gene therapies, today announced that the leadership will be presenting at the Cell & Gene Meeting on the Mesa, which is being held from October 12th 16th. In addition to providing an overview of the Company, the management team will provide a progress update on the clinical and development programs and an overview of the Company’s business strategy. Management will be virtually meeting cell and gene therapy companies to support collaborations.

This is one of the most prestigious meetings for our industry, and despite it being virtual this year due to the COVID-19 pandemic, it will not dampen our enthusiasm” commented Dr. Yarkoni. We have successfully adjusted our operations, and we continue to make significant progress with our Israel and U.S.-based clinical trials. We are also taking meaningful steps to accelerate nearer-term revenue opportunities as we are collaborating with several partners that are looking to leverage our technology platform to help improve their products, especially in high-growth areas such as CAR T, NK (natural killers) and MSC’s. We are looking forward to sharing the progress we have made and also look forward to meeting with current and potential partners developing cell and gene therapies.”

To schedule a meeting with the Company’s Chief Executive Officer, Dr. Shai Yarkoni or Chief Operating Officer, Amos Ofer, please request a meeting through the meeting portal and/or contact the Company direct at shai@cellect.co or amoso@cellect.co. The presentation will be available on the Company’s website prior to the commencement of the meeting. The Cell & Gene Meeting on the Mesa is the sector’s foremost annual conference bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (APOP) has developed a breakthrough technology, for the functional selection of cells that aims to improve the robustness, safety and efficacy of a variety of cell and gene therapies. The Company’s technology can be used by researchers, clinical community and pharma companies in a wide variety of applications including next generation Car T, NK, MSC and gene therapies.

The Company is also developing its own product that is an improved BMT which is in a current clinical trial for cancer treatment.

Forward Looking Statements

This press release contains forward-looking statements about the Company’s expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as believe”, expect”, intend”, plan”, may”, should”, could”, might”, seek”, target”, will”, project”, forecast”, continue” or anticipate” or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss Cellect’s expectations regarding timing of the commencement of its planned U.S. clinical trial and its plan to reduce operating costs. These forward-looking statements and their implications are based on the current expectations of the management of the Company only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: the Company’s history of losses and needs for additional capital to fund its operations and its inability to obtain additional capital on acceptable terms, or at all; the Company’s ability to continue as a going concern; uncertainties of cash flows and inability to meet working capital needs; the Company’s ability to obtain regulatory approvals; the Company’s ability to obtain favorable pre-clinical and clinical trial results; the Company’s technology may not be validated and its methods may not be accepted by the scientific community; difficulties enrolling patients in the Company’s clinical trials; the ability to timely source adequate supply of FasL; risks resulting from unforeseen side effects; the Company’s ability to establish and maintain strategic partnerships and other corporate collaborations; the scope of protection the Company is able to establish and maintain for intellectual property rights and its ability to operate its business without infringing the intellectual property rights of others; competitive companies, technologies and the Company’s industry; unforeseen scientific difficulties may develop with the Company’s technology; the Company’s ability to retain or attract key employees whose knowledge is essential to the development of its products; and the Company’s ability to pursue any strategic transaction or that any transaction, if pursued, will be completed. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading Risk Factors” in Cellect Biotechnology Ltd.’s Annual Report on Form 20-F for the fiscal year ended December 31, 2019 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC’s website, http://www.sec.gov, and in the Company’s periodic filings with the SEC.

Contact Cellect Biotechnology Ltd. Eyal Leibovitz, Chief Financial Officer http://www.cellect.co +972-9-974-1444

Or

EVC Group LLC Michael Polyviou (732) 933-2754 mpolyviou@evcgroup.com

Read more:
Cellect Biotechnology to Present at the 2020 Cell & Gene Meeting on the Mesa - Stockhouse

BOSTON, Sept. 22, 2020 (GLOBE NEWSWIRE) -- Ziopharm Oncology, Inc. (Ziopharm or the Company) (Nasdaq:ZIOP), today announced the appointment of J. Kevin Buchi to the Companys Board of Directors and as Chair of the Boards Audit Committee. Additionally, Ziopharm announced that, in response to the results from the Companys recent annual meeting of stockholders, Doug Pagn has resigned from the Board.

Mr. Buchi has deep life sciences industry experience, notably 15 years with Cephalon, including serving as its Chief Executive Officer during Cephalons acquisition by Teva Pharmaceuticals in 2011 for $6.8 billion. Subsequently, Mr. Buchi served as Chief Executive Officer of TertraLogic Pharmaceuticals, and more recently as Chief Executive Officer of Biospecifics Technologies, prior to his retirement.

We are delighted to welcome Kevin to our Board of Directors, said Scott Tarriff, Chairman of the Ziopharm Board of Directors. Kevin is an accomplished executive and director, whose extensive industry experience and business and financial acumen will complement the Board. We also wish to thank Doug for his many contributions since joining the Ziopharm Board in 2018, a critical time in the Companys history, while we exited a corporate partnership and established the foundation for our core programs today. Over the past year, we have added four strong directors to our Board, including James Huang in July and Dr. Chris Bowden and Heidi Hagen last year. Looking ahead, we expect our Board will continue to evolve to reflect the needs of our business as we evolve into a commercial-stage company.

Mr. Buchi added, This is an exciting time for Ziopharm, as the Company has established a broad portfolio of innovative clinical programs to treat solid tumors and a talented team to drive company-sponsored trials in all three core programs. I am delighted to begin collaborating with Laurence and his team, as well as the other members of Ziopharms Board of Directors.

Following the acquisition of Cephalon by Teva in 2011, Mr. Buchi served as corporate vice president of global branded products at Teva. Subsequently, he was CEO TetraLogic Pharmaceuticals and Biospecifics Technologies. In addition, Mr. Buchi currently serves as chairman of Dicerna Pharmaceuticals, and as a director of Amneal Pharmaceuticals and Benitec Biopharma Ltd. Mr. Buchi earned a B.A. in Chemistry from Cornell University and a Masters in Management, Accounting and Finance from the Kellogg School of Management at Northwestern University.

The Companys Board consists of eight directors, including seven non-executive directors. The Board will continue to actively review the Board membership to ensure the skills and experience of directors support the progress and future prospects of the business.

About Ziopharm Oncology, Inc.Ziopharm is developing non-viral and cytokine-driven cell and gene therapies that weaponize the bodys immune system to treat the millions of people globally diagnosed with a solid tumor each year. With its multiplatform approach, Ziopharm is at the forefront of immuno-oncology with a goal to treat any type of solid tumor. Ziopharms pipeline is built for commercially scalable, cost effective T-cell receptor T-cell therapies based on its non-viral Sleeping Beauty gene transfer platform, a precisely controlled IL-12 gene therapy, and rapidly manufactured Sleeping Beauty-enabled CD19-specific CAR-T program. The Company has clinical and strategic collaborations with the National Cancer Institute, The University of Texas MD Anderson Cancer Center and Regeneron Pharmaceuticals. For more information, please visit http://www.ziopharm.com.

Forward-Looking Statements DisclaimerThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding the growth of Ziopharm from a development-stage entity to a commercial-stage company, development of its clinical portfolio and research and development programs. Although Ziopharms management team believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Ziopharm, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, changes in our operating plans that may impact our cash expenditures, the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Ziopharms product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. FDA or equivalent foreign regulatory agencies and for which indication; the strength and enforceability of Ziopharms intellectual property rights; competition from other pharmaceutical and biotechnology companies as well as risk factors discussed or identified in the public filings with the Securities and Exchange Commission made by Ziopharm, including those risks and uncertainties listed in Ziopharms Quarterly Report on Form 10-Q filed by Ziopharm with the Securities and Exchange Commission. We are providing this information as of the date of this press release, and Ziopharm does not undertake any obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason.

Investor Relations Contacts:Ziopharm Oncology: Chris Taylor VP, Investor Relations and Corporate CommunicationsT: 617.502.1881E: ctaylor@ziopharm.com

LifeSci Advisors:Mike MoyerManaging DirectorT: 617.308.4306E: mmoyer@lifesciadvisors.com

Media Relations Contact:LifeSci Communications:Patrick BurseyT: 646.876.4932E: pbursey@lifescicomms.com

Read this article:
Ziopharm Oncology Appoints Kevin Buchi to its Board of Directors - BioSpace

University of Bristol researchers have treated a mouse model of glaucoma using a single gene therapy injection

Pixabay/Arek Socha

Writing in Molecular Therapy, scientists described how CRISPR gene editing was used to inactivate a gene in the ciliary body of the eye, decreasing intraocular pressure.

Visiting senior research fellow at Bristol Medical School, Dr Colin Chu, said the team hoped to advance towards clinical trials of the therapy in the near future.

If it's successful it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money," Dr Chu said.

Researchers note that while eye drops are currently used to reduce intraocular pressure in glaucoma patients, the drops may have side effects and there are issues of patient compliance in administering the drops daily.

Gene editing to provide a single but permanent therapeutic alteration is an appealing approach given glaucoma is a chronic disease that requires lifelong intervention, the study authors emphasised.

Original post:
Gene therapy as a potential treatment for glaucoma - AOP

A biotech company spun out of the University of Edinburgh will utilise research to develop new gene therapies to target different types of cancer.

The company, Cellinta Limited, was born out of research from scientists at the University of Edinburgh, and received funding through investments from SV Health Investors and Cancer Research UK.

The funding will be used to develop a pipeline of highly selective gene therapies targeting cancer stem cells in a variety of solid tumour types.

It will draw on research led by Professor Steven Pollard at the University of Edinburgh, which delved into stem cell biology, synthetic biology and cancer genomics.

Cellinta has entered into a collaborative research agreement with the University, and retains the option to license intellectual property developed from research conducted there.

Professor Pollard will join the Cellinta team as scientific founder and a senior adviser, while Dr Soraya Bekkali will take the helm as chief executive officer of the new company.

I am delighted to be leading such an exciting company, said Dr Bekkali.

Cellintas approach offers the opportunity to deliver combinations of therapies selectively to cancer stem cells, bypassing the limitations of traditional treatments which often target single genes or redundant signalling pathways, she added.

Meanwhile, Mike Ross managing partner at London-based SV Health Investors has been appointed chairman of Cellinta.

Cellinta represents a unique opportunity to build a leading company based on exciting and innovative science, with the potential to develop first-in-class therapies, said Ross.

We are delighted with the progress the company has made and extremely pleased to appoint Soraya, who has deep experience in gene therapy across various therapeutic areas, as CEO. We look forward supporting Cellinta in its progress as a pioneer in the field, he added.

Continued here:
University of Edinburgh spinout Cellinta will develop gene therapies for cancer - PharmaTimes

Xtalks Life Science Webinars

TORONTO (PRWEB) September 21, 2020

The International Organization for Standardization (ISO) has recently issued its eagerly awaited guidance on the transportation of cells for therapeutic use (ISO 21973). The ISO document is meant to provide general requirements and points to consider for transportation service providers, clients and senders to ensure cell quality, safety and efficacy during the transportation process. This webinar will discuss the key recommendations and changes that interested parties should review and evaluate when planning their clinical and commercial distribution strategies for cell and gene therapies. Some of the elements to be discussed are documentation, traceability, validation and qualification activities, temporary storage, as well as cleaning and disinfection.

Join Robert Jones, Vice President, BioServices, Cryoport Systems in a live webinar on Thursday, October 1, 2020 at 11am EDT (4pm BST/UK).

For more information or to register for this event, visit Addressing the Recent ISO 21973 Guidance: Considerations for Cell and Gene Therapy Distribution.

ABOUT CRYOPORT, INC.

Cryoport, Inc. is redefining temperature controlled supply chain support for the life sciences industry by providing a broad platform of temperature-controlled supply chain solutions, serving the Biopharma, Reproductive Medicine, and Animal Health markets. Our mission is to support life and health on earth by providing reliable and comprehensive solutions for the life sciences industry through our advanced technologies, global supply chain network and dedicated scientists, technicians and supporting team of professionals. Through our purpose-built, proprietary Cryoport Express Shippers; Cryoportal information technology; validated Global Logistics Centers; smart and sustainable temperature-controlled logistics solutions; and biostorage/biobanking services, Cryoport serves clients in life sciences research, clinical trials, and product commercializations. We support life-saving advanced cell and gene therapies and deliver vaccines, protein producing materials, and IVF materials in over 100 countries around the world. For more information, visit http://www.cryoport.com or follow @cryoport on Twitter at http://www.twitter.com/cryoport for live updates.

ABOUT XTALKS

Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit http://xtalks.comFor information about hosting a webinar visit http://xtalks.com/why-host-a-webinar/

Share article on social media or email:

Continue reading here:
Addressing the Recent ISO 21973 Guidance: Considerations for Cell and Gene Therapy Distribution, Upcoming Webinar Hosted by Xtalks - PR Web

HEIDELBERG, Germany, Sept. 21, 2020 /PRNewswire/ -- GeneWerk GmbH, a cell and gene therapy testing laboratory focused on providing preclinical and clinical trial patient sample analysis services, today announced a majority investment from Ampersand Capital Partners, a private equity firm specializing in growth equity investments in the healthcare sector. Ampersand's growth investment will be used to expand GeneWerk's capabilities to meet rapidly growing demand for cell and gene therapy testing services.

GeneWerk provides cell and gene therapy sponsors with patient testing services in compliance with guidance by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The company is recognized as a leading provider of vector integration site analysis (ISA) services, a method that was developed by GeneWerk's founders and that the FDA and EMA recommend performing after the administration of both integrating and non-integrating cell and gene therapies. The company's test menu also includes vector persistence testing, gene edited off-target analysis, vector copy number (VCN), vector quality control, immune repertoire analysis, and dedicated bioinformatics studies. With a focus on vector safety, characterization, and functionality analysis, the company's 30 employees work in compliance with GCP and in line with GLP standards in a BSL-2 classified state-of-the-art genomics and bioinformatics laboratory in Heidelberg, Germany.

Annette Deichmann, Ph.D., Co-Founder and Co-CEO at GeneWerk commented, "With the benefit of Ampersand as our partner, GeneWerk will strengthen and expand its position in the U.S. and European markets while further investing in our testing capabilities to service cell and gene therapy sponsors and patients." Co-Founder and Co-CEO Manfred Schmidt, Ph.D., then added, "Our partnership with Ampersand solidifies GeneWerk's position in the space and will allow the company to continue to exceed our customers' expectations by facilitating the development of innovative cell and gene therapies. We are very pleased to have Ampersand on board as we take GeneWerk through its next phase of growth." Christof von Kalle, M.D., Co-Founder concluded, "This will greatly further GeneWerk's opportunities to contribute to medical breakthroughs and patient safety."

Marina Pellon-Consunji, Principal at Ampersand said, "GeneWerk is a leading company in its field. Given the exciting developments within the cell and gene therapy market and recent guidance by FDA and EMA, this is an excellent time for an investor with deep experience in cell and gene therapy to partner with the company. We are looking forward to working with the team at GeneWerk to accelerate and continue its success in delivering leading testing services to ensure the safety of patients receiving cutting edge cell and gene therapies."

About GeneWerk GmbHFounded in 2014 by Prof. Dr. Christof von Kalle, Dr. Manfred Schmidt, and Dr. Annette Deichmann with the participation of the German Cancer Research Center (DKFZ) Heidelberg, GeneWerk is a cell and gene therapy testing laboratory focused on providing preclinical and clinical trial patient sample analysis services. The company is recognized as a leading provider of vector integration site analysis (ISA) services, a method that was developed by the company's founders and that the FDA and EMA recommend performing after the administration of both integrating and non-integrating cell and gene therapies. For more information, please visit http://www.genewerk.com.

About Ampersand Capital PartnersFounded in 1988, Ampersand is a middle market private equity firm dedicated to growth-oriented investments in the healthcare sector. With offices in Boston and Amsterdam, Ampersand leverages its unique blend of private equity and operating experience to build value and drive superior long-term performance alongside its portfolio company management teams. Ampersand has helped build numerous market-leading companies across each of the firm's core healthcare sectors. Additional information about Ampersand is available at http://www.ampersandcapital.com.

SOURCE Ampersand Capital Partners

http://www.ampersandventures.com

Go here to see the original:
GeneWerk GmbH Announces Growth Equity Investment from Ampersand Capital Partners - PRNewswire

The first part of the CPhI Annual Report 2020, released ahead of the CPhI Festival of Pharma (taking place online October 5-16) predicts dramatic growth of new mAb production in China. It also forecasts capacity shortages for cell and gene therapies in the US, as well as widespread global adoption of single-use technologies, with only limited continuous bioprocessing.

In the document, CPhI experts from BioPlan Associates (Vicky Qing XIA, senior project manager; Leo Cai Yang, project manager; and Eric Langer, president and managing partner) delve into the rapidly evolving biologics markets. The report highlights implications for contract outsourcing and Chinas continually increasing presence emergence as a hub for both bio innovation and contract services.

The report predicts rapid bio growth in China to continue, forecasting that more than 10 new mAbs likely will be launched each year by 2025. Overall, the authors estimate the total market size will quadruple by 2025, reaching nearly $18b (120b RMB) by that point and rising to more than $28b (190b RMB) by 2030.

As most early-stage biotech in China lack manufacturing facilities, the need for contract manufacturing services is rising quickly, and has been accelerated further by the 2016 MAH reforms, XIA commented.

According to the report, bioprocessing outsourcing in China is currently highly stratified with four tiers and just one domestic company in tier one (WuXi Biologics), with a number of international CDMOs (including BI, Lonza and Merck).

However, by 2025 the report anticipates as many as five more domestic CDMOs may have reached tier one status, with US Food and Drug Administration (FDA) and EU facility approvals. Significantly, pending regulatory changes for contract vaccine production will likely spur further rapid growth amongst the existing tier one CDMOs.

WuXi Biologics in 2019 realized 35.3% of its total revenue of USD 0.57 billion from China, which is ~USD 0.2 billion and would translate to ~35% of the total biopharma outsourcing service market in China1. However, despite sizable growth in revenue, its overall share of the market will now fall, as the mAb market expands rapidly, added XIA.

Langer explained, Because preclinical and clinical pipeline products require flexible manufacturing, SUS lends itself to these scales, but many of these will fail as they progress through the pipeline. This means that while more commercial-scale biologics are going to be made in SUS platforms, or hybrid systems, over the next 2+ years, stainless platforms are, and will remain, critical to bioprocessing as well.

The report also suggested that in the US and European markets will likely ocme under a cell and gene therapy capacity crunch by 2025, seeing CDMOs investing in this area already expanding in an attempt to meet the pipelines impending demand.

However, capacity alone is not the biggest challenge as there is a shortage of both specialized platforms, and trained personnel to operate them. Significantly, for some facilities in these emerging areas the equipment required for expansion and up-scaling may not yet exist, nor are regulatory authorities fully aligned with issues around patient treatments which could slow approvals.

Tara Dougal, head of content at CPhI Festival of Pharma, explained, The cell and gene therapy space is hugely fast-moving and we will run a session on the potential for M&As as both manufacturers and CDMOs try to buy-in expertise rather than build. More generally, our report also points to sustained bio growth in China, almost universal adoption of single use technologies, with continuous processing growing but from a far lower base."

Dougal added that CPhI has engaged Jefferson Institute for Bioprocessing to expand on the topic, exploring current strategies and looking ahead at future approaches.

Langer is scheduled to participate in a panel discussion on M&A activity in the cell and gene sector, taking place at CPhI Festival of Pharma October 6, 2 to 3 pm CET.

The CPhI Festival of Pharma takes place digitally October 5 to 16. For more information about the program or to register, go to https://www.cphi.com/festival-of-pharma/en/home.html.

See the original post:
CPhI: cell and gene therapy capacity shortages on the horizon - OutSourcing-Pharma.com

BOTHELL, Wash., Sept. 21, 2020 /PRNewswire/ -- BioLife Solutions, Inc. (NASDAQ: BLFS)("BioLife" or the "Company"), a leading developer and supplier of a portfolio of class-defining bioproduction tools for cell and gene therapies, today announced it has entered into a definitive agreement to acquire SciSafe, a privately held multi-facility provider of biological materials storage to the cell and gene therapy and pharmaceutical industries. The transaction is expected to close on September 30th.

SciSafe had 2019 unaudited revenue of $6 million and positive EBITDA and is anticipated to be accretive during 2021.

Under the terms of the agreement, BioLife will pay $15 million in cash and $15 million in newly issued shares of BioLife common stock for 100% of the outstanding shares of SciSafe. SciSafe's shareholders are also eligible over the next four years to receive up to 626,000 additional shares of BioLife common stock based on the achievement of annual revenue milestones.

Mike Rice, Chief Executive Officer of BioLife Solutions, commented, "This acquisition enables BioLife to offer even more value to our cell and gene therapy customers through an established business with an excellent reputation, marquee customers and seasoned team. Through SciSafe, we are accelerating profitable growth by expanding into the high growth biostorage segment with a robust quality system, a scalable business model and strong financial performance. We anticipate several vertical integration cost synergies including using SciSafe facilities for cGMP storage of our biopreservation media products, leveraging our CBS facility to manufacture walk-in freezer rooms for SciSafe and deploying our evo Smart Shippers and evoIS cloud app for the thousands of annual inbound and outbound biologic materials shipments managed by SciSafe."

Garrie Richardson, President of SciSafe, remarked, "I'm thrilled to be joining forces with BioLife. With BioLife's stellar reputation in the cell and gene therapy market, key customer relationships, financial resources and commitment to fund our growth, the SciSafe team is poised to deliver significant high-margin incremental revenue to BioLife's growing enterprise."

Benefits of the Transaction

Entry into the fast-growing biostorage segment

Cross-selling opportunities

Potential vertical integration cost synergies

Financial Impact of SciSafe Acquisition

BioLife expects the acquisition of SciSafe to impact the Company's financial performance as follows:

About SciSafeFounded in 2010, SciSafe offers dedicated pharmaceutical and biological specimen storage in its four fully cGMP-compliant state-of-the-art sample management facilities. SciSafe has built flourishing relationships with over 300 of the world's leading and most admired organizations. Clients have repeatedly chosen to store their most valued and irreplaceable biological samples because they trust SciSafe to care for them as if they were their own. SciSafe values and respects its long-term client relationships. With over 60 years combined experience specifically in life sciences, SciSafe personnel fully appreciate the vital requirements of all areas of specimen storage and cold chain management. For more information, please visit http://www.scisafe.com.

About BioLife SolutionsBioLife Solutions is a leading supplier of a portfolio of class-defining cell and gene therapy bioproduction tools and services. Our tools portfolio includes our proprietaryCryoStorfreeze media and HypoThermosolshipping and storage media, ThawSTARfamily of automated, water-free thawing products, evocold chain management system, and Custom Biogenic Systemshigh capacity storage freezers. Services include SciSafe biologic and pharmaceutical materials storage. For more information, please visit http://www.biolifesolutions.com, and follow BioLife on Twitter.

Cautions Regarding Forward Looking Statements Except for historical information contained herein, this press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, statements concerning the expected financial performance of the company following the completion of its acquisition of SciSafe, the expected synergies between the company and SciSafe, the company's ability to realize all or any of the anticipated benefits associated with the acquisition of SciSafe, the company's ability to implement its business strategy and anticipated business and operations, including following the acquisition of SciSafe, the potential utility of and market for the company's and SciSafe's products and services, guidance for financial results for 2020 and 2021, including regarding SciSafe's revenue, and potential revenue growth and market expansion, including with consideration to our acquisition of SciSafe. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements, including among other things, uncertainty regarding unexpected costs, charges or expenses resulting from the company's acquisition of SciSafe or the 2019 acquisitions, charges or expenses resulting from the acquisition of SciSafe; market adoption of the company's products (including the company's recently acquired products) or SciSafe's products; the ability of the SciSafe acquisition to be accretive on the company's financial results; the ability of the company to implement its business strategy; uncertainty regarding third-party market projections; market volatility; competition; litigation; the impact of the COVID-19 pandemic; and those other factors described in our risk factors set forth in our filings with the Securities and Exchange Commission from time to time, including our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. We undertake no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof, other than as may be required by applicable law.

Media & Investor Relations Roderick de Greef Chief Financial Officer (425) 686-6002 [emailprotected]

SOURCE BioLife Solutions, Inc.

http://www.biolifesolutions.com

Visit link:
BioLife Solutions to Acquire SciSafe, a High-Growth Biostorage Service Provider to the Cell and Gene Therapy Industry - PRNewswire

This article was originally published here

Curr Gene Ther. 2020;20(2):127-141. doi: 10.2174/1566523220999200731003206.

ABSTRACT

Suppression of TP53 function is nearly ubiquitous in human cancers, and a significant fraction of cancers have mutations in the TP53 gene itself. Therefore, the wild-type TP53 gene has become an important target gene for transformation research of cancer gene therapy. In 2003, the first anti-tumor gene therapy drug rAd-p53 (recombinant human p53 adenovirus), trade name Gendicine, was approved by the China Food and Drug Administration (CFDA) for treatment of head and neck squamous cell carcinoma (HNSCC) in combination with radiotherapy. The recombinant human TP53 gene is delivered into cancer cells by an adenovirus vector constructed to express the functional p53 protein. Although the only currently approved used of Gendicine is in combination with radiotherapy for treatment of HNSCC, clinical studies have been carried out for more than 20 other applications of Gendicine in treating cancer, including treatment of advanced lung cancer, advanced liver cancer, malignant gynecological tumors, and soft tissue sarcomas. Currently more than 30,000 patients have been treated with Gendicine. This review provides an overview of the clinical applications of Gendicine in China. We summarize a total of 48 studies with 2,561 patients with solid tumors, including 34 controlled clinical studies and 14 open clinical studies, i.e., clinical studies without a control group. There are 11 studies for head and neck cancer, 10 for liver cancer, 6 for malignant gynecological tumors, 4 for non-small cell lung cancer, 4 for soft tissue sarcoma, 4 for malignant effusion, 2 for gastrointestinal tumors, and 7 for other types of cancer. In all the reported clinical studies, the most common side effect was self-limited fever. Intratumoral injection and intra-arterial infusion were the most common routes of administration. Overall, Gendicine combined with chemotherapy, radiotherapy, or other conventional treatment regimens demonstrated significantly higher response rates compared to standard therapies alone. Some of the published studies also showed that Gendicine combination regimens demonstrated longer progression-free survival times than conventional treatments alone. To date, Gendicine has been clinically used in China for treatment of cancers other than HNSCC for more than ten years, mainly for patients with advanced or unresectable malignant tumors. However, the establishment of standard treatment regimens using TP53 gene therapy is still needed in order to advance its use in clinical practice.

PMID:32951572 | DOI:10.2174/1566523220999200731003206

Read more:
Applications of Recombinant Adenovirus-p53 Gene Therapy for Cancers in the Clinic in China - DocWire News

Management to Highlight Progress in Bone Marrow Transplant (BMT) and Chimeric Antigen Receptor T Cell (CAR T) Business Collaborations

Tel Aviv, Israel, Sept. 21, 2020 (GLOBE NEWSWIRE) -- Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of innovative technology which enables the functional selection of cells facilitating safer and more efficacious cell and gene therapies, today announced that the leadership will be presenting at the Cell & Gene Meeting on the Mesa, which is being held from October 12th 16th. In addition to providing an overview of the Company, the management team will provide a progress update on the clinical and development programs and an overview of the Companys business strategy. Management will be virtually meeting cell and gene therapy companies to support collaborations.

This is one of the most prestigious meetings for our industry, and despite it being virtual this year due to the COVID-19 pandemic, it will not dampen our enthusiasm commented Dr. Yarkoni. We have successfully adjusted our operations, and we continue to make significant progress with our Israel and U.S.-based clinical trials. We are also taking meaningful steps to accelerate nearer-term revenue opportunities as we are collaborating with several partners that are looking to leverage our technology platform to help improve their products, especially in high-growth areas such as CAR T, NK (natural killers) and MSCs. We are looking forward to sharing the progress we have made and also look forward to meeting with current and potential partners developing cell and gene therapies.

To schedule a meeting with the Companys Chief Executive Officer, Dr. Shai Yarkoni or Chief Operating Officer, Amos Ofer, please request a meeting through the meeting portal and/or contact the Company direct at shai@cellect.co or amoso@cellect.co. The presentation will be available on the Companys website prior to the commencement of the meeting. The Cell & Gene Meeting on the Mesa is the sectors foremost annual conference bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (APOP) has developed a breakthrough technology, for the functional selection of cells that aims to improve the robustness, safety and efficacy of a variety of cell and gene therapies. The Companys technology can be used by researchers, clinical community and pharma companies in a wide variety of applications including next generation Car T, NK, MSC and gene therapies.

The Company is also developing its own product that is an improved BMT which is in a current clinical trial for cancer treatment.

Forward Looking Statements

This press release contains forward-looking statements about the Companys expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as believe, expect, intend, plan, may, should, could, might, seek, target, will, project, forecast, continue or anticipate or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss Cellects expectations regarding timing of the commencement of its planned U.S. clinical trial and its plan to reduce operating costs. These forward-looking statements and their implications are based on the current expectations of the management of the Company only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: the Companys history of losses and needs for additional capital to fund its operations and its inability to obtain additional capital on acceptable terms, or at all; the Companys ability to continue as a going concern; uncertainties of cash flows and inability to meet working capital needs; the Companys ability to obtain regulatory approvals; the Companys ability to obtain favorable pre-clinical and clinical trial results; the Companys technology may not be validated and its methods may not be accepted by the scientific community; difficulties enrolling patients in the Companys clinical trials; the ability to timely source adequate supply of FasL; risks resulting from unforeseen side effects; the Companys ability to establish and maintain strategic partnerships and other corporate collaborations; the scope of protection the Company is able to establish and maintain for intellectual property rights and its ability to operate its business without infringing the intellectual property rights of others; competitive companies, technologies and the Companys industry; unforeseen scientific difficulties may develop with the Companys technology; the Companys ability to retain or attract key employees whose knowledge is essential to the development of its products; and the Companys ability to pursue any strategic transaction or that any transaction, if pursued, will be completed. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading Risk Factors in Cellect Biotechnology Ltd.s Annual Report on Form 20-F for the fiscal year ended December 31, 2019 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SECs website, http://www.sec.gov, and in the Companys periodic filings with the SEC.

ContactCellect Biotechnology Ltd.Eyal Leibovitz, Chief Financial Officerwww.cellect.co+972-9-974-1444

Or

EVC Group LLCMichael Polyviou(732) 933-2754mpolyviou@evcgroup.com

Read this article:
Cellect Biotechnology to Present at the 2020 Cell & Gene Meeting on the Mesa - GlobeNewswire

Cell and Gene Therapy Market Research Report is a Proficient and In-Depth Study on the Existing State of Cell and Gene Therapy Industry. This Report Focuses on the Major Drivers, Restraints, Opportunities and Threats for Key Players. It also Provides Granular Analysis of Market Share, Segmentation, Revenue Forecasts and Regional Analysis till 2025.

The recent study on Cell and Gene Therapy market offers an all-inclusive analysis of this vertical, with emphasis on the growth driving factors as well as facets such as consumption and production. Constraints and potential threats expected to restrain the expansion along with solutions to overcome the challenges are discussed at length. Moreover, insights of the market share along with estimates reflecting the CAGRs of the listed segments are highlighted in the document.

In hindsight of the COVID-19 pandemic, the report investigates the prevalent business strategies employed by leading organizations and offers tactics for stakeholders to adapt to the industry changes over the forecast period.

Request Sample Copy of this Report @ https://www.aeresearch.net/request-sample/307400

Key pointers from the Cell and Gene Therapy market report TOC:

Additional highlights from the Cell and Gene Therapy market report:

Reasons to buy the report

Request Customization on This Report @ https://www.aeresearch.net/request-for-customization/307400

Read the original post:
Cell and Gene Therapy Market Report 2020 Industry Capacity, Manufactur - News by aeresearch

T-knife GmbH, a next-gen adoptive T-cell company, and Catalent, have signed an agreement to provide technology transfer and CGMP clinical manufacturing of T-knifes T1367 T-cell receptor (TCR) program.

T1367 is an autologous T-cell receptor-based cell therapy derived from T-knifes proprietary humanized T-cell receptor (HuTCR) mouse platform and specifically targets MAGE-A1 positive tumors in cancer patients. The therapy is expected to be manufactured for clinical trials in both the EU and U.S.

Catalent will undertake transfer of T-knifes platform process for T-cell receptor-based cell therapy at its site in Gosselies, Belgium, with the goal of manufacturing clinical batches for European trials in 2021. T-knife will also prepare for the transfer of the TCR manufacturing platform to Catalents Houston, TX, facility for clinical trials in North America in the future.

The product candidates based on our proprietary HuTCR platform require sophisticated, state-of-the-art manufacturing capabilities and deep cell and gene therapy know-how, said Michael Buchholz, Director Manufacturing of T-knife. We are convinced that Catalent is the right partner for T-knife to ensure premier manufacturing of our pipeline programs, covering all stages from clinical trials to market.

Catalent is well-suited to support T-knife with focused technology transfer and process industrialization in both Gosselies and Houston, commented Manja Boerman, Ph.D., President, Cell & Gene Therapy, Catalent. Emerging and innovative treatments like T1367 are moving rapidly to the clinic. Catalent is committed to continual investment and expansion to support our clients as they continue on the journey to commercialization.

Catalents 25,830 sq.-ft. facility in Gosselies, Belgium, provides clinical through commercial-scale cell therapy manufacturing, for both autologous and allogeneic cell therapy treatments. An additional large-scale commercial manufacturing plant is currently under construction at the site and expected to be fully commissioned in 2021. The companys clinical manufacturing site in Houston is under qualification and expected to be fully commissioned in 2020.

Original post:
T-knife, Catalent Sign Tech Transfer And Mfg. Pact For Cell Therapy - Contract Pharma

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, today announced that it has licensed intellectual property relating to its pioneering chlorotoxin chimeric antigen receptor (CLTX-CAR) T cell therapy to Chimeric Therapeutics Limited, an Australian biotechnology company.

The therapy is currently being used in a phase 1 clinical trial at City of Hope to treat glioblastoma (GBM), a type of brain tumor. The first patient in the trial was recently dosed; Behnam Badie, M.D., chief of City of Hopes Division of Neurosurgery and The Heritage Provider Network Professor in Gene Therapy, is leading this innovative, first-of-its-kind trial.

Chimeric has acquired the exclusive worldwide rights to develop and commercialize certain patents relating to City of Hopes CLTX-CAR T cells, as well as to further develop the therapy for other cancers.

City of Hope is excited to enter into this agreement with Chimeric as it supports our innovative research in CAR T cell therapy and our commitment to extend these therapies to more patients, particularly those with GBM and other solid tumors that are difficult to treat, said Christine Brown, Ph.D., The Heritage Provider Network Professor in Immunotherapy and deputy director of City of Hopes T Cell Therapeutics Research Laboratory. Chimeric shares our goal of providing effective CAR T cell therapies to more patients with current unmet medical needs.

Led by Brown and Michael Barish, Ph.D., chair of City of Hopes Department of Developmental and Stem Cell Biology, and Dongrui Wang, Ph.D., a recent graduate of City of Hopes Irell & Manella Graduate School of Biological Sciences, the team developed and tested the first CAR T cell therapy using CLTX, a component of scorpion venom, to direct T cells to target brain tumor cells. The research was published this past March in Science Translational Medicine.

Chimeric is excited to join City of Hope in its quest to find more effective cancer therapies. This is an exceedingly rare opportunity to acquire a promising technology in one of the most exciting areas of immuno-oncology today, said Paul Hopper, executive chairman of Chimeric. Furthermore, the CLTX-CAR T cell therapy has completed years of preclinical research and development, and recently enrolled its first patient in a phase 1 clinical trial for brain cancer.

CARs commonly incorporate a monoclonal antibody sequence in their targeting domain, enabling CAR T cells to recognize antigens and kill tumor cells. In contrast, the CLTX-CAR uses a synthetic 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain.

In this recent study, City of Hope researchers used tumor cells in resection samples from a cohort of patients with GBM to compare CLTX binding with expression of antigens currently under investigation as CAR T cell targets. They found that CLTX bound to a greater proportion of patient tumors, and cells within these tumors.

CLTX binding included the GBM stem-like cells thought to seed tumor recurrence. Consistent with these observations, CLTX-CAR T cells recognized and killed broad populations of GBM cells while ignoring nontumor cells in the brain and other organs. The study team demonstrated that CLTX-directed CAR T cells are highly effective at selectively killing human GBM cells without off-tumor targeting and toxicity in cell-based assays and in animal models.

City of Hope, a recognized leader in CAR T cell therapies for GBM and other cancers, has treated more than 500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has 30 ongoing CAR T cell clinical trials, including CAR T cell trials for HER-2 positive breast cancer that has spread to the brain, and PSCA-positive bone metastatic prostate cancer. It was the first and only cancer center to treat GBM patients with CAR T cells targeting IL13R2, and the first to administer CAR T cell therapy locally in the brain, either by direct injection at the tumor site, through intraventricular infusion into the cerebrospinal fluid, or both. In late 2019, City of Hope opened a first-in-human clinical trial for patients with recurrent GBM, combining IL13R2-CAR T cells with checkpoint inhibitors nivolumab, an anti-PD1 antibody, and ipilimumab, blocking the CTLA-4 protein.

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"City of Hope is delighted to license this technology to Chimeric, said Sangeeta Bardhan Cook, Ph.D., City of Hope director of the Office of Technology Licensing. We are impressed with the ability of their executive team to push and bring therapies to market expeditiously. At City of Hope, our mission is to transform the future of health care. We believe Chimeric has the vision to offer innovative therapies to cancer patients.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nations Best Hospitals in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

View original post here:
City of Hope Enters Licensing Agreement With Chimeric to Develop Its Pioneering Chlorotoxin CAR T Cell Therapy - Business Wire

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (NASDAQ: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, today announced it has named Thomas C. Gallagher, Esq. as the Companys Senior Vice President of Intellectual Property and Licensing. Mr. Gallagher has extensive experience in the area of biotechnology intellectual property (IP) law, business development, and licensing transactions with industry and academic institutions.

Mr. Gallagher will play a critical role in advancing our expanding intellectual property estate by spearheading IP strategy, which is an important element of the Companys overall success and value creation. A seasoned biotech executive, he will provide significant support as we execute on broadening our research and development programs, explore opportunities for partnerships on our existing programs, and opportunistically acquire new technologies to further expand our pipeline, said Rodney Varner, President and Chief Executive Officer of Genprex.

Mr. Gallagher has more than 20 years of experience as an intellectual property attorney. Prior to joining Genprex, he served as Principal at the Fenagh Group, an IP and licensing consultancy providing clients in the healthcare sector guidance on all aspects of patent and trademark portfolio management, intellectual property due diligence, freedom-to-operate analysis and related transactional work. He has also served as Senior Vice President of Intellectual Property and Licensing at Kadmon Corporation, LLC, a biopharmaceutical company based in Manhattan. Prior to joining Kadmon, he served as in-house IP counsel at Neostem Inc. (now Caladrius Biosciences, Inc.), a company focused on stem cell biology. Previously, he held several positions at ImClone Systems Incorporated, most recently as Vice President of Intellectual Property and Licensing. While at ImClone, he was responsible for all aspects of intellectual property and led the IP function in multiple due diligence undertakings by major pharmaceutical companies, which resulted in a $2 billion strategic investment, the highest-valued biotech deal ever at the time, and the eventual sale of the company to Eli Lilly and Company for $6.5 billion.

Mr. Gallagher is experienced in both patent prosecution and litigation, as well as intellectual property issues relating to business development and licensing matters. His patent litigation experience includes European litigation and opposition proceedings. In addition to a law degree, Mr. Gallagher holds a Masters degree in molecular biology. Before becoming an attorney, Mr. Gallagher worked as a molecular biologist in France, Spain and the United States.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, GPX-001 (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). GPX-001 has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. GPX-001 has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for GPX-001 for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone. For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of GPX-001, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including GPX-002, our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our product candidates; the timing and success at all of obtaining FDA approval of GPX-001 and our other potential product candidates including whether we receive fast track or similar regulatory designations; costs associated with developing our product candidates, whether we identify and succeed in acquiring other technologies and whether patents will ever be issued under patent applications that are the subject of our license agreements or otherwise. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

See the original post:
Genprex Names Seasoned IP Executive, Thomas C. Gallagher, as Senior Vice President of Intellectual Property and Licensing - Business Wire

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported that statistical analysis of pooled data from LUMEVOQ trials and natural history studies found a statistically significant and clinically meaningful difference between the visual outcomes in LUMEVOQ-treated patients and untreated patients.

Treated eyes showed progressive and sustained improvement from Month 12 to Month 52, in contrast to the absence of recovery over the same period for untreated eyes. At Month 18, the difference became statistically significant (p=0.01). By Month 48, the difference between the mean visual acuity in treated patients and that in untreated patients was both statistically significant (p<0.01) and clinically meaningful (-0.33 LogMAR, or +16.5 ETDRS letters equivalent, in favor of treated eyes).

Note: All patients had a confirmed G11778A mutation in the ND4 mitochondrial gene and were at least 15 years old. The diagram shows the Locally Estimated Scatterplot Smoothing (LOESS) curves for visual acuity in LUMEVOQ-treated patients and untreated patients. The shaded areas represent the 95% confidence interval for the mean BCVA. Treated eyes refer to all eyes (LUMEVOQ and sham) from the RESCUE, REVERSE and CLIN06 trials (N=76 patients / 152 eyes). Untreated eyes refer to patient-level data from the REALITY study and a matched data set from two prospective and eight retrospective natural history studies1 (N=208 patients / 408 eyes). LOESS curves were estimated using a non-parametric, local regression model that treated each eye as independent of the other. LOESS curves are shown from Month 12 to depict post-treatment progression among treated patients (93% of LUMEVOQ patients had already been treated within 12 months from onset). *Statistically significant difference between mean visual acuity of treated and untreated eyes at M18, M24, M36 and M48, as illustrated by the non-overlapping confidence intervals.

The analysis compared data from the completed Phase III trials RESCUE and REVERSE studies and interim results from the long-term follow-up CLIN06 study to a matched sample created from the REALITY registry study and 10 other natural history studies1. The natural history studies were identified from an extensive review of the scientific literature and selected based on specific inclusion criteria for their patient-level data. In all, the visual outcomes in 76 treated patients could be compared to the visual outcomes of 208 untreated patients.

The extra granularity of this analysis down to individual patient data confirms that the natural history of visual outcomes in ND4-LHON patients is poor and provides the best comparison we have for assessing the therapeutic efficacy of gene therapy, commented Dr. Nancy J. Newman, MD, LeoDelle Jolley Professor of Ophthalmology and Neurology at the Emory University School of Medicine in Atlanta, GA, USA, and a global authority on LHON who recently completed a meta-analysis of the natural history of ND4-LHON.2

Separate analyses of patients enrolled in RESCUE and REVERSE demonstrated similarly favorable results compared to untreated patients. Full findings from the indirect comparison were included in the European Marketing Authorisation Application (MAA) for LUMEVOQ and are being prepared for publication in a peer-reviewed journal.

This indirect comparison represents a significant contribution to our understanding of LUMEVOQs therapeutic effect," said Dr. Jos-Alain Sahel, MD, Director of the Institut de la Vision (Sorbonne-Universit/Inserm/CNRS), Paris, France; Chairman of the Department of Ophthalmology at Centre Hospitalier National dOphtalmologie des XV-XX, Paris, France; Professor and Chairman of the Department of Ophthalmology at University of Pittsburgh School of Medicine and UPMC (University of Pittsburgh Medical Center), USA; and Co-Founder of GenSight Biologics. The use of a large external control group, including analyses of patient-level data, provides more evidence for a significant treatment-related visual improvement.

The findings are a gratifying outcome of our push to overcome a key challenge for assessing LUMEVOQs efficacy, namely the inability of sham eyes to act as a control group, commented Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. We are excited to take this evidence that LUMEVOQ modifies the disease outcome forward into our conversations with national and regional authorities.

The LUMEVOQ MAA was filed in September, and the decision is expected in H2 2021. The Company is also working towards submitting LUMEVOQs Biologics License Application (BLA) to the FDA in H2 2021.

GenSight will host a conference call today, September 21, 2020, at 10am CEST in French, and at 2.00pm CEST (8.00am EST) in English, to discuss these results.

Webcast & Conference call in French (10am CEST)

Dial-in numbers:

United States: +1 212 999 6659France: +33 (0)1 7037 7166United Kingdom: +44 (0)20 3003 2666Password: GenSight FR

Webcast link: https://bit.ly/3mAS0Vy

Webcast & Conference call in English (2.00pm CEST / 8.00am EST)

Dial-in numbers:

United States: +1 212 999 6659France: +33 (0) 1 7037 7166United Kingdom: +44 (0) 20 3003 2666Password: GenSight ENG

Webcast link: https://bit.ly/35RCfnl

A replay of the calls and webcasts will be available by using the above links.

Rationale for the Indirect Comparison

In both the REVERSE and RESCUE studies, as well as in the long-term follow-up study CLIN06, unilaterally injected patients experienced an unexpected visual improvement in their contralateral eye, which mirrored the sustained and clinically relevant gain in eyes treated with LUMEVOQ. This bilateral improvement eliminated the control group that was to consist of sham eyes in the original trial design. An indirect comparison methodology, based on formal statistical methods applied to an external control group, was needed to assess the magnitude of LUMEVOQ efficacy.

Methodology Highlights

The sample of LUMEVOQ-treated patients included all data from the two pivotal studies REVERSE and RESCUE and Year 3 data from the ongoing extension study CLIN06. This approach yielded a treated patient pool consisting of 76 patients (152 eyes), with LUMEVOQ-injected eyes and sham-treated eyes considered equivalent, based on the contralateral effect demonstrated in the studies.

The external control group included data from the REALITY Natural History registry and patient-level data from 10 published articles1 on ND4-LHON, which were identified from a systematic review of the literature. ND4-LHON studies were included in the indirect comparison only if they had individual patient data that would allow indirect comparison with LUMEVOQ-treated patients: confirmed ND4 genotype, at least 15 years of age, at least one BCVA measurement with associated time of vision loss. The final external control group included 208 patients (408 eyes).

The visual acuity curves of treated and untreated patients were defined using a Locally Estimated Scatterplot Smoothing (LOESS), non-parametric, local regression model. They were aligned in terms of time from vision loss for the statistical tests performed at M12, M18, M24, M36 and M48 from vision loss and at the last available observation. The starting time point for the statistical tests (12 months post-vision loss) was defined to enable assessment of LUMEVOQ efficacy, as 93% of LUMEVOQ patients were treated by that time.

1The 10 studies that passed the inclusion criteria were: Hotta 1995, Lam 2014, Nakamura 1993, Newman 1991, Qu 2007, Qu 2009, Romero 2014, Sadun 2004, Yang 2016, and Zhou 2010.

2Newman NJ, Carelli V, Taiel M and Yu-Wai Man P. Visual outcomes in Leber hereditary optic neuropathy patients with the m.11778G>A (MTDN4) mitochondrial DNA mutation. J Neuro-Ophthalmol. In Press. 2020.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQ (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About LUMEVOQ (GS010)

LUMEVOQ (GS010) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,400 to 1,500 new patients who lose their sight every year in the United States and Europe.

About RESCUE and REVERSE

RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint measures the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, will be used for statistical purposes. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.

The secondary endpoints involve the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis was evaluated, including the proportion of patients who maintain vision (< ETDRS 15L loss), the proportion of patients who gain 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, biodissemination and the time course of immune response. Readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials were conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were transferred to a long-term follow-up study that will last for three years.

ClinicalTrials.gov Identifiers:REVERSE: NCT02652780RESCUE: NCT02652767

About CLIN06 (RESCUE and REVERSE Long-term Follow-up)

CLIN06 is the long-term follow-up study of ND4 LHON subjects treated with LUMEVOQ (GS010) gene therapy in the RESCUE or REVERSE Phase III Clinical Trials. The total study period for an individual subject is 3 years, i.e., 5 years post-gene therapy administration. No study treatment is administered during CLIN06.

The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment. The secondary objective is to assess the long-term treatment efficacy of the therapy and the quality of life (QoL) in subjects up to 5 years post-treatment. The first subject was enrolled on January 9, 2018. 61 subjects have enrolled.

ClinicalTrials.gov Identifiers:CLIN06: NCT03406104

About REALITY

REALITY is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on subjects medical charts and the administration of surveys on Health-Related Quality of Life (HRQoL) and direct and indirect costs associated with the disease.

The study aimed to recruit at least 50 subjects (both adult and pediatric) chiefly in the following countries: Spain, Italy, France, United Kingdom and the United States.

The primary objectives for the REALITY study were: to describe the evolution of visual functional and structural changes and other associated symptoms in patients with LHON; understand the impact of LHON-related vision loss on the HRQoL; and understand the economic burden for patients and their families arising from direct and indirect costs associated with the disease. The secondary objective is to describe the relationship between genetic, lifestyle and/or environmental factors and the expression of the LHON phenotype.

The first subject was enrolled on 3 January 2018. Enrollment was completed in early Q2 2020.

ClinicalTrials.gov Identifiers:REALITY LHON Registry: NCT03295071

See original here:
GenSight Biologics Reports New Analysis Demonstrating Statistically Significant and Clinically Meaningful Difference Between Visual Outcomes in...

The Global Urology Implants And Devices Market is projected to grow considerably on account of increasing incidence of urology disorders. Key insights into this development have been provided by Fortune Business Insights in its report, titled Urology Implants and Devices Market Size, Share and Global Trend By Product Type (Endoscopes, Lithotripsy Devices, Urodynamic Systems, Urostomy Bag, Endo Vision Systems & Peripheral Instruments, Urology Implants), By Disease (Kidney Disorders, Urology Cancers, Urinary Tract Disorders, Pelvic Organ Prolapse), By End User (Hospitals, Pharmacies, Ambulatory Surgery Centers (ASC)) and Geography Forecast till 2026.

The report covers:

Request a Sample Copy of the Research Report: https://www.fortunebusinessinsights.com/enquiry/request-sample-pdf/urology-implants-and-devices-market-100434

Leading Players operating in the Urology Implants and Devices Market are:

Key players are involved in mergers and acquisition to strengthen their market position. Owing to increasing competition frequent innovations are taking place in the market. Some of the companies operating the industry are:

Segmentation

By Products

By Disease

By End-Users

By Region

More Trending Topics From Fortune Business [emailprotected]

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Read more here:
Urology Implants and Devices Market Size 2020 Top Manufacturers, Share, Opportunities and Forecast to 2026 - The Daily Chronicle