BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference – GlobeNewswire

NEW YORK, Oct. 25, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced that it will be presenting at the Dawson James Securities 5th Annual Small Cap Growth Conference, being held on October 28-29, 2019 at the Wyndham Grand Hotel in Jupiter, Florida.

Preetam Shah, PhD, MBA, Chief Financial Officer is scheduled to present on Tuesday, October 29th at 3:40 p.m. Eastern Time, in Track 2 - Preserve Ballroom B, with one-on-one meetings to be held throughout the conference.

Chaim Lebovits, President and CEO of BrainStorm said, We are pleased to have the opportunity to have Dr. Shah present at the Dawson James Small Cap Growth Conference. Dr. Shah, joined BrainStorm in September 2019, and we look forward to having him present the Companys growth strategy and future to a wide audience of accreditied investors.

About NurOwn NurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

AboutBrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PR Phone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference - GlobeNewswire

Stem Cell Therapy Market Will Achieve 10.2% CAGR to Cross $15bn by 2025: Global Market Insights, Inc. – State-Journal.com

Global Stem Cell Therapy Market value will achieve over 10% CAGR to surpass USD 15 billion by 2025; according to a new research report by Global Market Insights, Inc. Rapid advancements in stem cell therapies in developed region such as North America and Europe will boost industry growth. Key industry players are indulged in introducing novel stem cell therapies that address limitations of existing therapies. Significant efficiency possessed by stem cell therapy has increased its preference in treatment of cancer that should positively impact the industry growth. However, high cost associated with stem cell therapies may hinder stem cell therapy industry growth to certain extent.

Increasing prevalence of chronic disease such as cancer, cardiovascular diseases will surge the demand for stem cell therapy business. Stem cell therapies have unique properties, such as immunosuppression, secretion of bioactive factors that fosters the development of tumor targeting technologies. Introduction of innovations in the regenerative medicine will help in conquering challenges in combating numerous diseases that should foster the industry growth.

Allogenic segment of stem cell therapy market accounted for over 39.5% revenue in 2018. Significant growth is attributed to the advantages associated with allogenic stem cell therapies. Allogenic stem cell involves transfer of stem cell from donor to patients. It overrules the limitations of autologous stem cell therapy such as difficulty in obtaining healthy and sufficient amount of stem cells from patients of diabetes, rheumatoid arthritis and other chronic diseases. Therefore, people have started relying more on the allogenic regenerative therapies that should augment the segmental growth.

Cardiovascular segment of stem cell therapy market is anticipated to witness around 9.5% CAGR throughout the forecast time frame. Increasing incidence of cardiovascular disease and growing healthcare concerns will surge the demand for stem cells employed in cardiovascular diseases. According to WHO, every year 17.9 million people die of cardiovascular disease. Stem cell therapies possess enormous potential to replace the conventional cardiovascular treatments that should increase its adoption over the forecast period.

Stem Cell Therapy Market Statistics, By Application

1.2. Oncology 1.2.1. Market size, by region, 2014-2025 (USD Million)

1.3. Orthopedic 1.3.1. Market size, by region, 2014-2025 (USD Million)

1.4. Cardiovascular 1.4.1. Market size, by region, 2014-2025 (USD Million)

1.5. Neurology 1.5.1. Market size, by region, 2014-2025 (USD Million)

1.6. Others 1.6.1. Market size, by region, 2014-2025 (USD Million)

Browse key industry insights spread across 130 pages with 91 market data tables & 8 figures & charts from the report, Stem Cell Therapy Market Forecast 2019 - 2025 in detail along with the table of contents:

Clinics segment of stem cell therapy industry was valued at USD 2.5 billion in 2018. Considerable revenue size is attributed to superior treatment provided by clinics to cure life threating disease. Clinics are equipped with advance equipment and skilled workforce that ultimately enhance treatment outcomes. Moreover, clinics are believed to provide sophisticated stem cell therapies that will stimulate the segmental growth.

North America stem cell therapy market held over 9.5% CAGR in 2018 owing to favorable regulatory scenario and demographic trends. Improving regulatory scenario for stem cell therapies in the U.S. and Canada should positively impact the market growth. For instance, FDA has framed pre-marketing authorization rules for the commercialization of stem cell therapies. This helps to minimize adverse events caused due to defective regenerative therapies. Moreover, several companies focus on R&D activities to develop innovative stem cell therapies that should surge the regional growth.

Notable industry players operational in industry are Astellas Pharma, Cellectis, Celyad, DiscGenics, Gamida Cell, Capricor Therapeutics, Novadip Biosciences, Cellular Dynamics, CESCA Therapeutics, OxStem, ReNeuron Group, Mesoblast, and Takeda Pharmaceuticals. Industry players are adopting strategic initiatives such as collaborations product launches, geographic expansions, mergers and acquisitions in order to sustain industry competition and acquire prominent market. For instance, in 2019 Gamida cell formed agreement with Lonza to commercialize omidubicel after its FDA approval. Omiducel is potential life-saving stem cell treatment for treating hematologic malignancies. Thus, such collaborations will boost the companys growth considerably.

Bioreactors Market Growth Report 2025: Biopharmaceutical products are individualized products with highly specific manufacturing requirements. Advanced biopharmaceutical manufacturing technologies have enabled development of effective drug delivery systems and drug device combination products. Some of the key industry players operating in the market include Eppendorf, GE Healthcare, Merck Millipore, Sartorius, and Thermo Fisher Scientific.

About Global Market Insights

Global Market Insights, Inc., headquartered in Delaware, U.S., is a global market research and consulting service provider; offering syndicated and custom research reports along with growth consulting services. Our business intelligence and industry research reports offer clients with penetrative insights and actionable market data specially designed and presented to aid strategic decision making. These exhaustive reports are designed via a proprietary research methodology and are available for key industries such as chemicals, advanced materials, technology, renewable energy and biotechnology.

Arun Hegde Corporate Sales, USA Global Market Insights, Inc. Phone:1-302-846-7766 Toll Free:1-888-689-0688 Email: sales@gminsights.com

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United Therapeutics Receives Permit For Cell Therapy Facility Build-Out At Mayo – Pharmaceutical Online

The build-out is estimated at $9.5M.

United Therapeutics received a building permit Tuesday for a $9.5M build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4M building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

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United Therapeutics Receives Permit For Cell Therapy Facility Build-Out At Mayo - Pharmaceutical Online

American Academy of Stem Cell Physicians Announced Today That Their Safety Panel Session is Open and Free to the Public – PRNewswire

MIAMI, Oct. 24, 2019 /PRNewswire/ -- The AASCP has recently created guidelines thatare current safety recommendations given to physicians who are using biologics in their medical practice. A highly anticipated and sought after Safety StandardsPanel session, hosted by AASCP on Nov. 2, 2019, will be moderated by The Alliance for Cell Therapy Now,with President Ms. Janet Marchbrody.The sessions normally are closed to the public but this particular SafetyStandard Panel discussion will be open to the public, covering the growing safety concerns of the industry.

Alliance for Cell Therapy Now is a coalition of organizations representing patients, health care providers and the academic and scientific community, who are working together to advance safe and effective regenerative cell therapies. The mission is to advance the development, manufacturing and delivery of safe and effective regenerative cell therapies through policy development, consensus and advocacy. Alliance for Cell Therapy Now is bringing together experts and stakeholders to gain consensus on and advocate for policies that will advance the science and the field, including those focused on promoting clinical research, assuring the adoption of consensus standards to promote safety and quality, building capacity and expertise within the workforce, and establishing a national outcomes database to advance the science, promote improvements in quality and safety, and inform regulatory, paymentand patient decision-making.

Alliance for Cell Therapy Now is guided by an Advisory Board comprised of leaders in the scientific, academicand patient communities; Ms.Janet M. MarchibrodaPresident, Alliance for Cell Therapy Now Fellow, Bipartisan Policy Center Senior Vice President, Health Policy, Bockorny Group, has agreed to join theAASCP as a moderator for their SafetyPanelat The Hyatt Regency in Miami. This particular coveted safetypanel session will be open to the public and broadcast live on YouTube at 3:00 p.m. on Nov. 2, 2019.

According to AASCP, if you are using biologics in your practice, whether you are using SVF, PRP, bone marrow, UCB, amniotic products,exosomes,xenografts, or peptides, there are key considerations to take into account to achieve the best safety for your patients. The AASCP also recommends communication with the Chief Scientific Officer from the laboratory you work with.AASCP advises that just talking to a sales agent is not sufficient enough when determining the quality of products for your patients. Sales agents typically do not have a medical or scientific background.

The spokesman for the AASCP, Dr. AJFarshchian,said earlier: "The American Academy of Stem Cell Physicians is a group of physicians, scientists and researchers who collectively represent the most authoritativenon-federal group advocating for guidelines and education on stem cell therapy and regenerative medicine. AASCP members are experts within all fields of stem cell therapy from: SVF, BM, UCB, Exosomes, Peptides, Xenografts, Allografts and Amniotic Fluids and are considered the most experienced leaders for proper advocacy in the field. The AASCP is involved directly with other authorities within the field and seeks only to bring knowledge and awareness for the ever growing regenerative medicine industry.My hope is that the SafetyPanel discussion on Nov.2, 2019, is to help get rid of the bad actors that are damaging the field for everyone."

AASCP is hosting their medical conference in Miami on Nov. 1-3 , 2019. Sessions are normally closed to the public and, therefore, require registration. The conference is taking place at the downtown MiamiHyatt Regency, located at 400 SE 2nd Ave, Miami, FL 33131.Becauseof limited seating, we encourage everyone to please RSVP ataascp.net andto register.

The American Academy of Stem Cell Physicians (AASCP) is an organization created to advance research and the development of therapeutics in regenerative medicine, including diagnosis, treatmentand prevention of disease related to or occurring within the human body. Secondarily, the AASCP aims to serve as an educational resource for physicians, scientistsand the public in diseases that can be caused by physiological dysfunction that areameliorableto medical treatment.

For further information, please contact Marie Barbaat AASCP 305-891-4686 and you can also visit us at http://www.aascp.net.

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dr-farshchian-teaching-at-aaoscp.jpeg Dr. Farshchian teaching at AAOSCP workshop Dr. Alimorad Farshchian speaking at AASCP

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American Academy of Stem Cell Physicians Announced Today That Their Safety Panel Session is Open and Free to the Public - PRNewswire

Global Stem Cell and Regenerative Therapy Market – Yahoo Finance

Report Scope: The scope of this report is broad and covers various type of product available in the stem cell and regenerative medicines market and potential application sectors across various industries.

New York, Oct. 24, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Stem Cell and Regenerative Therapy Market" - https://www.reportlinker.com/p05791357/?utm_source=GNW The current report offers a detailed analysis of the stem cell and regenerative medicines market.

The report highlights the current and future market potential of stem cell and regenerative medicines and provides a detailed analysis of the competitive environment, recent development, merger and acquisition, drivers, restraints, and technology background in the market. The report also covers market projections through 2024.

The report details market shares of stem cell and regenerative medicines based on products, application, and geography.Based on product the market is segmented into therapeutic products, cell banking, tools and reagents.

The therapeutics products segments include cell therapy, tissue engineering and gene therapy. By application, the market is segmented into oncology, cardiovascular disorders, dermatology, orthopedic applications, central nervous system disorders, diabetes, others

The market is segmented by geography into the following regions: North America, Europe, Asia-Pacific, South America, and the Middle East and Africa. The report presents detailed analyses of major countries such as the U.S., Canada, Mexico, Germany, the U.K. France, Japan, China and India. For market estimates, data is provided for 2018 as the base year, with forecasts for 2019 through 2024. Estimated values are based on product manufacturers total revenues. Projected and forecasted revenue values are in constant U.S. dollars, unadjusted for inflation.

Report Includes: - 28 data tables - An overview of global markets for stem cell and regenerative medicines - Analyses of global market trends, with data from 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 - Details of historic background and description of embryonic and adult stem cells - Information on stem cell banking and stem cell research - A look at the growing research & development activities in regenerative medicine - Coverage of ethical issues in stem cell research & regulatory constraints on biopharmaceuticals - Comprehensive company profiles of key players in the market, including Aldagen Inc., Caladrius Biosciences Inc., Daiichi Sankyo Co. Ltd., Gamida Cell Ltd. and Novartis AG

Summary The global market for stem cell and regenerative medicines was valued at REDACTED billion in 2018.The market is expected to grow at a compound annual growth rate (CAGR) of REDACTED to reach approximately REDACTED billion by 2024.

Growth of the global market is attributed to the factors such as growingprevalence of cancer, technological advancement in product, growing adoption of novel therapeuticssuch as cell therapy, gene therapy in treatment of chronic diseases and increasing investment fromprivate players in cell-based therapies.

In the global market, North America held the highest market share in 2018.The Asia-Pacific region is anticipated to grow at the highest CAGR during the forecast period.

The growing government funding for regenerative medicines in research institutes along with the growing number of clinical trials based on cell-based therapy and investment in R&D activities is expected to supplement the growth of the stem cell and regenerative market in Asia-Pacific region during the forecast period.

Reasons for Doing This Study Global stem cell and regenerative medicines market comprises of various products for novel therapeutics that are adopted across various applications.New advancement and product launches have influenced the stem cell and regenerative medicines market and it is expected to grow in the near future.

The biopharmaceutical companies are investing significantly in cell-based therapeutics.The government organizations are funding research and development activities related to stem cell research.

These factors are impacting the stem cell and regenerative medicines market positively and augmenting the demand of stem cell and regenerative therapy among different application segments.The market is impacted through adoption of stem cell therapy.

The key players in the market are investing in development of innovative products. The stem cell therapy market is likely to grow during the forecast period owing to growing investment from private companies, increasing in regulatory approval of stem cell-based therapeutics for treatment of chronic diseases and growth in commercial applications of regenerative medicine.

Products based on stem cells do not yet form an established market, but unlike some other potential applications of bioscience, stem cell technology has already produced many significant products in important therapeutic areas. The potential scope of the stem cell market is now becoming clear, and it is appropriate to review the technology, see its current practical applications, evaluate the participating companies and look to its future.

The report provides the reader with a background on stem cell and regenerative therapy, analyzes the current factors influencing the market, provides decision-makers the tools that inform decisions about expansion and penetration in this market.Read the full report: https://www.reportlinker.com/p05791357/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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GeoVax Vaccine To Be Used in a Phase 1 Trial of Genetically Modified Autologous Cell Therapy for HIV – GlobeNewswire

IND Application Submitted to FDA for Phase 1 Trial

ATLANTA, GA, Oct. 24, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE GeoVax Labs, Inc. (OTCQB: GOVX) announced today its participation in a planned clinical trial led by researchers at American Gene Technologies (AGT) (www.americangene.com), to develop a therapy aimed at eliminating HIV from infected people.

On October 18, 2019, AGT announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for AGTs lead HIV program, AGT103-T, a lentiviral vector-based gene therapy. Upon clearance by the FDA, this IND will allow AGT to initiate a Phase 1 clinical trial that will investigate the safety of AGT103-T in humans, measure key biomarkers, and explore surrogate markers of efficacy. AGT expects to begin recruiting patients for the Phase 1 study in January.

Pursuant to its collaboration agreement with AGT, GeoVax will provide its novel MVA-VLP HIV vaccine (MVA62B) for evaluation in an arm of the clinical trial in combination with AGT103-T. T cells obtained from vaccinated individuals will be reprogrammed by AGTs lentivirus vector and infused back into the study participants as a therapeutic cell product. MVA62B is the boosting component for GeoVaxs preventive HIV vaccine (GOVX-B11) which has successfully completed a Phase 2a clinical trial and is awaiting funding in support of a Phase 2b pivotal trial.

Farshad Guirakhoo, PhD, GeoVaxs Chief Scientific Officer, commented, In a previous therapeutic Phase 1 clinical trial conducted by GeoVax, we demonstrated that our vaccine can potently stimulate production of both CD8+ and CD4+ T cells in HIV-positive individuals the intended use of the MVA62B vaccine in the proposed AGT therapeutic study. Our vaccine also has a well-documented safety profile in >500 humans. We are hopeful that the combination of technologies in these studies will yield a promising regimen that eliminates the HIV reservoir from people infected with the disease in the absence of antiretroviral drugs.

David Dodd, GeoVaxs President and CEO, stated, Finding a cure for HIV/AIDS remains an elusive but critical goal, and GeoVax is pleased to be a contributor to this important effort. While current antiretroviral therapy is effective at suppressing the viral load of HIV-infected individuals and preventing progression to AIDS, the long-term use of ART can lead to loss of drug effectiveness and can come with severe side effects. Additionally, the financial burden of drug and other medical costs to both the individual and to society at large is staggering, with average lifetime medical costs of treating an HIV-positive individual in the U.S. estimated to exceed $350,000.

About GeoVax

GeoVax Labs, Inc. is a clinical-stage biotechnology company developing human vaccines against infectious diseases and cancer using a novel patented Modified Vaccinia Ankara-Virus Like Particle (MVA-VLP) based vaccine platform. On this platform, MVA, a large virus capable of carrying several vaccine antigens, expresses proteins that assemble into VLP immunogens within (in vivo) the person receiving the vaccine. The production of VLPs in the person being vaccinated mimics virus production in a natural infection, stimulating both the humoral and cellular arms of the immune system to recognize, prevent, and control the target infection. The MVA-VLP derived vaccines elicit durable immune responses in the host similar to a live-attenuated virus, while providing the safety characteristics of a replication-defective vector.

GeoVaxs current development programs are focused on preventive vaccines against HIV, Zika Virus, hemorrhagic fever viruses (Ebola, Sudan, Marburg, and Lassa), and malaria, as well as therapeutic vaccines against chronic Hepatitis B infections and multiple cancers. The Company has designed the leading preventative HIV vaccine candidate to fight against the subtype of HIV prevalent in the commercial markets of the Americas, Western Europe, Japan, and Australia; this program is currently undergoing human clinical trials managed by the HIV Vaccine Trials Network (HVTN) with the support of the National Institutes of Health (NIH). For more information, visitwww.geovax.com.

Forward-Looking Statements

Certain statements in this document are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These statements are based on management's current expectations and are subject to uncertainty and changes in circumstances. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax can develop and manufacture its vaccines with the desired characteristics in a timely manner, GeoVax's vaccines will be safe for human use, GeoVax's vaccines will effectively prevent targeted infections in humans, GeoVaxs vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete vaccine development, there is development of competitive products that may be more effective or easier to use than GeoVax's products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control. GeoVax assumes no obligation to update these forward-looking statements and does not intend to do so. More information about these factors is contained in GeoVax's filings with the Securities and Exchange Commission including those set forth at "Risk Factors" in GeoVax's Form 10-K.

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Could the Next Big Cell Therapy Technology Come Out of the Department of Defense? – BioBuzz

(U.S. Army photo by Sgt. 1st Class Michel Sauret)(Photo Credit: Sgt. 1st Class Michel Sauret) VIEW ORIGINAL

October 22, 2019

Many of the regions successful life science startups are built on technologies that were licensed from The National Institutes of Health (NIH), The National Cancer Institute (NCI), other government biomedical research institutions and Biohealth Capital Region (BHCR) research universities like Johns Hopkins and the University of Maryland.

An often overshadowed regional source of promising life science/medical device technology is the Department of Defense (DoD). Several local companies like Emergent Biosolutions, Adaptive Phage Therapeutics and ARMR Systems, among others, have leveraged DoD tech and other defense-related agency resources to launch and build their successful and growing organizations.

In fact, Frederick-based Theradaptive was launched with a DoD grant to develop their regenerative technologies aimed at regenerating tissues in a controlled way to keep soldiers from losing their limbs. Now they are applying their platform technology to address a range of issues such as localized cancer treatments, next-generation vascular stents, tissue repair patches for cardiac damage and more.

DoD research facilities are abundant within the BHCR, including those at Fort Detrick, within The Medical Research and Materiel Command and Walter Reed Army Institute of Research (WRAIR). The close proximity to these research labs is a unique asset for the region that is not as prevalent elsewhere, even in Boston or San Francisco.

Another reason to consider evaluating potential DoD technologies and resources is the FDA and DoDs program to expedite medical product availability for American military personnel emergency care. Under this new program, the FDA will give the highest level of attention to, and expedite its review of, priority DoD medical products in a manner similar to products under the breakthrough designation program.

A new initiative called DefTech has recently launched to help uncover DoD-developed technologies with commercial potential and get them into the market. DefTech is a resource that should not be overlooked by biohealth entrepreneurs and startups.

DefTech, or the Maryland Defense Technology Commercialization Center, is a Maryland Department of Commerce program launched in 2018 to better connect entrepreneurs and emerging life science companies with available DoD tech transfer and resource opportunities.

Funded initially by the DoDs Office of Economic Adjustment via a pilot program, and currently funded by the U.S. Department of Commerce Economic Development Administration, DefTech has developed a strong network of 360 clients and partners since its launch just less than two years ago.

According to Executive Director Dr. Gary Evans, DefTechs mission has three fundamental focus areas:

The process is highly complex and weve built and continue to refine our tech transfer process model. Ultimately, we serve as a matchmaker between tech transfer teams and business, helping to guide a conversation so that theres clarity about the level of interest, process and legalities, added Evans, who leverages his scientific knowledge, serial entrepreneurial background (he founded a number of successful companies and a venture capital studio) and licensing experience to help connect entrepreneurs and early-stage businesses with the right DoD research lab opportunities.

DefTechs story starts with the Maryland Department of Commerce recognizing the untapped potential of DoD research lab patents and mapping out a broad economic development strategy to create stronger awareness among businesses and entrepreneurs about DoD intellectual property licensing opportunities. Whats more, it was evident that there was minimal awareness about the remarkable DoD lab facilities, equipment and expertise that these same businesses and entrepreneurs could access.

In February 2018, the pilot program began with DoD Office of Economic Adjustment funding provided to the Maryland Department of Commerce, and non-federal matching funds from Harford County, Maryland, in the form of office space adjacent to Aberdeen Proving Ground, coupled with a range of operational support.

DefTech quickly established relationships with three research labs at Aberdeenunder the Combat Capabilities Development Command: The Chemical Biological Center (CBC), the Army Research Lab (ARL) and the C5 ISR lab. DefTechs successful engagement with these labs at Aberdeen Proving Ground created positive momentum for the program, enabling its growth and winning more support from its partners to scale the operation. In January 2019, the program received additional funding from the Economic Development Administration that allowed DefTech to expand the initiative to include Fort Meade and Fort Detrick, including research labs at the National Security Agency (NSA) and The Medical Research and Materiel Command, respectively.

In a short period of time, DefTech has built a significant network that now includes five research labs and 14 project partners, including Howard County and Frederick County, which have also provided DefTech with off-base meeting space that helps facilitate connections between tech transfer teams and the private sector.

All of our partners have been very supportive, including Frederick County Economic Development, The City of Frederick and particularly FITCI, which has been a really great partner. Theyve given us many opportunities to connect with people and tell them about what we do. A number of our current clients have come through FITCI. I cant say enough good things about them and all of our partners that have supported us along the way, stated Evans.

Today, DefTech has an agile process model that empowers tech transfer offices and the private sector to overcome common impediments to commercializing federal lab intellectual property. These common obstacles, according to Evans, include difficulties getting on base, a general lack of familiarity with the licensing process and getting overwhelmed by its complexities, which can cause some companies to walk away. Evans also cites a lack of understanding about what resources are actually available as a major obstacle to tech transfer. To assist with this, DefTech maintains a database of available patents from Maryland DOD labs, which contains nearly 1,800 entries.

Often, potential licensees dont know where to look and the DefTech team efficiently searches through a morass of patents to find potentially strong fits. To date, DefTech has found 131 patents that meet the needs and interests of their clients.

Our goal is to facilitate the process and make it as workable as possible for all parties, he added.

The commercialization potential of federal DoD lab technology is significant yet often overlooked for a variety of reasons. Federal lab patents are not necessarily blockbuster technologies, but there are an enormous number of strong potential products that can be picked up and leveraged to create a successful business, according to Evans.

Theres a phenomenal pool of assets out there that are waiting for the right entrepreneurial innovator to bring their full potential to fruition, stated Evans. Weve had some success stories early on and right now we have seven companies in various stages of the process with serious intent to reach agreement with a federal lab. These include some licensing opportunities as well lab resource partnerships. We have 35 clients overall and were seeing an ever increasing number of people with an interest in what were doing, shared Evans.

Whether its generating stronger awareness of federal lab opportunities and resources via educational programming, matching businesses with the right patents, facilitating dialog between tech transfer teams and entrepreneurs or working one-on-one with clients as they navigate a complex tech transfer process, DefTech has the experience and partner network to bring more federal lab technologies to market.

Tech transfer is a big driver of BHCR innovation and growth but its full potential is yet to be realized. Programs like DefTech and others can play a critical role in spurring the BHCR on to becoming the top biohealth cluster in the U.S.

To learn more about DefTech and the federal lab tech transfer process, visit us at Booth #14 at TEDCOs Entrepreneur Expo on October 29th or attend one of their upcoming educational seminars:

Click here to learn more and register.

Over the past 8 years, Chris has grown BioBuzz into a respected brand that is recognized for its community building, networking events and news stories about the local biotech industry. In addition, he runs a Recruiting and Marketing Agency that helps companies attract top talent through a blended model that combines employer branding and marketing services together with a high powered recruiting solution.

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Could the Next Big Cell Therapy Technology Come Out of the Department of Defense? - BioBuzz

Intellia Therapeutics Presents In Vivo and Ex Vivo Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) | DNA RNA…

DetailsCategory: DNA RNA and CellsPublished on Friday, 25 October 2019 09:51Hits: 64

CAMBRIDGE, MA, USA I October 24, 2019 IIntellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology is presenting one oral presentation and four poster presentations at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) meeting taking place October 22-25, 2019, in Barcelona, Spain.

We are excited to share progress across Intellias in vivo and ex vivo programs at this important scientific venue, said Laura Sepp-Lorenzino, Ph.D., chief scientific officer, Intellia Therapeutics. Our data shows the complexity of the edits we are able to make with CRISPR/Cas9, while achieving important therapeutically relevant results. We are building on the success of our modular platform now having demonstrated consecutive targeted knockout and insertion genome edits in preclinical studies. Additionally, we presented data from our engineered cell therapy program, which continues to demonstrate the use of CRISPR/Cas9 for combined knockout and targeted integration in human T cells.

Intellia Demonstrates Consecutive In Vivo Genome Editing in Alpha-1 Antitrypsin Deficiency Mouse Model

Intellias oral presentation highlights its alpha-1 antitrypsin deficiency (AATD) study showing that consecutive dosing of two distinct lipid nanoparticle (LNP) formulations, in adultmice, achieves two targeted genome editing events, resulting in knocking out the faulty gene and restoring therapeutic levels of normal alpha-1 antitrypsin protein (hAAT). Intellias approach for AATD uses a modular hybrid delivery system combining a non-viral LNP which encapsulates CRISPR/Cas9 with an adeno-associated virus (AAV) carrying donor DNA template. Compared to traditional viral-based delivery of gene editing components, Intellias LNP delivery system can overcome the inherent limitations of immunogenicity to facilitate multiple in vivo gene editing events.

In a mouse model harboring the human PiZ allele, the most severe genetic defect in AATD patients, Intellia first reduced expression of the defective protein using gene knockout. Three weeks following the PiZ allele knockout, Intellia inserted the normal human alpha-1 antitrypsin gene, resulting in stable (throughout 12 weeks of observation), therapeutically relevant circulating protein levels. In the study, a sustained reduction of the circulating PiZ protein levels of >98% was observed for over 15 weeks. This is the first in vivo demonstration of a non-viral delivery platform, enabling a consecutive dosing approach for achieving multiple genome edits in the same tissue of the same animal. Intellias oral presentation, titled In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression, will be given by Anthony Forget, Ph.D., on October 25, 2019. This presentationwill be available on Intellias website at http://www.intelliatx.com.

Intellias Poster Presentations

WT1-Specific TCR Engineered Cell Therapy Studies

Intellia presented new in vitro data showing that CRISPR/Cas9-mediated genome editing for in locus insertion, combined with endogenous T Cell Receptor (TCR) knockout, leads to significant reduction in mispairing of endogenous and transferred TCR chains. This approach is expected to generate transgenic-TCR (tg-TCR) T cell therapies for hematological cancers and solid tumors. Results demonstrate a highly efficient reduction of >98% in endogenous TCR and chains while reaching >70% insertion rates of tg-TCRs without further purification. The poster titled Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In Locus Insertion Combined with Endogenous TCR Knockout, was presented on October 24, 2019, by Birgit Schultes, Ph.D.

Researchers also presented in vitro data showing that a library of WT1-specific TCRs were generated, several of which Intellia is currently evaluating as part of its lead engineered cell therapy program targeting Acute Myeloid Leukemia (AML). This presentation, Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia, was presented on October 23, 2019 by Intellias collaborator, Erica Carnevale, Ph.D., IRCCS Ospedale San Raffaele.

Primary Hyperoxaluria Study

Intellia showed the continued progression of its modular platform capability using CRISPR/Cas9 to knockout either hydroxyacid oxidase 1 (Hao1) or lactate dehydrogenase A (Ldha), leading to a dose-dependent and persistent reduction of urinary oxalate levels in a Primary Hyperoxaluria Type 1 (PH1) mouse model. Data shows Ldha gene disruption also decreased LDH enzyme activity in the liver and did not impair the disposition of lactate in either wild type or renally-impaired mice. These results highlight the potential of editing genes in the glyoxylate detoxification pathway using a non-viral delivery approach as a one-time treatment option for PH1. These data were presented as a poster, titled CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria, by Sean Burns, M.D., on October 24, 2019.

Off-Target Screening Platform

Intellia demonstrated its approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Results from targeted off-target sequencing in edited cells showed that biochemical off-target discovery approaches were the most sensitive and accurate. These data were presented as a poster on October 23, 2019, titled In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics, by Dan OConnell, Ph.D.

About Intellia Therapeutics

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

SOURCE: Intellia Therapeutics

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Intellia Therapeutics Presents In Vivo and Ex Vivo Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) | DNA RNA...

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

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

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

Cobra Moradian, Fatemeh Rahbarizadeh

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

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

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

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

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

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