A Therapeutic Counterblow to Traumatic Brain Injury – Technology Networks

A blow to the head or powerful shock wave on the battlefield can cause immediate, significant damage to a person's skull and the tissue beneath it. But the trauma does not stop there. The impact sets off a chemical reaction in the brain that ravages neurons and the networks that supply them with nutrients and oxygen.

It is the secondary effects of traumatic brain injury (TBI), which can lead to long-term cognitive, psychological and motor system damage, that piqued the interest of a team of NJIT biomedical engineers. To counter them, they are developing a therapy, to be injected at the site of the injury, which shows early indications it can protect neurons and stimulate the regrowth of blood vessels in the damaged tissue.

The challenge, researchers say, is that brain cells don't regenerate as well as other tissues, such as bone, which may be an evolutionary strategy for preserving the synaptic connections that retain memories. To date, there is no effective treatment for restoring damaged neurons. The body's protective mechanisms also make it difficult to penetrate the blood-brain barrier, which hampers the delivery of medications.

"Nerve cells respond to trauma by producing excessive amounts of glutamate, a neurotransmitter that under normal conditions facilitates learning and memory, but at toxic levels overexcites cells, causing them to break down. Traumatic brain injury can also result in the activation and recruitment of immune cells, which cause inflammation that can lead to short- and long-term neural deficits by damaging the structure around cells and creating a chronic inflammatory environment," says Biplab Sarkar, a post-doctoral fellow in biomedical engineering and member of the team that presented this work at a recent American Chemical Society conference.

The team's treatment consists of a lab-created mimic of ependymin, a protein shown to protect neurons after injury, attached to a delivery platform -- a strand of short proteins called peptides, contained in a hydrogel -- that was developed by Vivek Kumar, director of NJIT's Biomaterial Drug Development, Discovery and Delivery Laboratory. After injection, the peptides in the hydrogel reassemble at the localized injury site into a nanofibrous scaffold that mimics extracellular matrix, the supporting structure for cells. These soft materials possess mechanical properties similar to brain tissue, which improves their biocompatibility. They promote rapid infiltration by a variety of stem cells which act as precursors for regeneration and may also provide a biomimetic niche to protect them.

Now in preclinical animal trials, rats injected with the hydrogel retained twice as many functioning neurons at the injury site as compared to the control group. They also formed new blood cells in the region.

"The idea is to intervene at the right time and place to minimize or reverse damage. We do this by generating new blood vessels in the area to restore oxygen exchange, which is reduced in patients with a TBI, and by creating an environment in which neurons that have been damaged in the injury are supported and can thrive," Kumar says. "While the exact mechanism of action for these materials is currently under study, their efficacy is becoming apparent. Our results need to be expanded, however, into a better understanding of these mechanisms at the cellular level, as well as their long-term efficacy and the resulting behavioral improvements."

Collaborators James Haorah, an associate professor of biomedical engineering, and his graduate student Xiaotang Ma at NJIT's Center for Injury Biomechanics, Materials and Medicine have shown how a number of TBI-related chemical effects can disrupt and destroy integral brain vasculature in the blood-brain barrier, the brain's protective border, promoting chronic inflammation that can lead to symptoms such as post-traumatic stress disorder and anxiety, among others. Their current work provides insights into the potential neuroprotective and regenerative response guided by the Kumar lab's materials, while future studies will attempt to analyze other mediators of inflammation and blood flow in the brain.

Kumar's delivery mechanism is a customizable, Lego-like strand made of short proteins called peptides, which are composed of amino acids, with a biological agent attached at one end that can survive in the body for weeks and even months, where other biomaterials degrade quickly. Its self-assembling bonds are designed to be stronger than the body's dispersive forces; it forms stable fibers, with no signs of inducing inflammation, that rapidly incorporate into specific tissues and collagen, recruiting native cells to infiltrate. The hydrogel, which is also composed of amino acids, is engineered to trigger different biological responses depending on the payload attached. These platforms can deliver drugs and other small cargo over day-, week- or month-long periods. Kumar's lab has recently published research on applications ranging from therapies to prompt or prevent the creation of new blood vessel networks, to reduce inflammation and to combat microbes.

"The ultimate hope is that that localized delivery of regenerative materials may provide significant benefits for a number of pathologies," he notes.

For example, the team recently developed a class of materials that may be useful against infection. These novel anti-microbial peptides are capable of disrupting dense bacterial colonies and have shown promise against a number of yeasts. Additionally, they promote human cell proliferation and are currently being studied for wound healing. That work was published this summer in the journal ACS Biomaterials Science and Engineering.

Kumar and his lab have created another hydrogel designed to recruit autologous (a person's own) dental pulp stem cells directly to the disinfected cavity after root canal therapy. The tooth would be regenerated in part by prompting growth of the necessary blood vessels to support the new tissue. Yet another peptide-based therapy, armed with antiangiogenic capabilities, targets diabetic retinopathy, an ocular disease affecting more than 90 million people worldwide. People with the disease form immature blood vessels in the retina, obstructing their vision. The hydrogel can be injected directly into the vitreous gel of the eye, where the peptide interacts with the endothelial cells in the aberrant blood vessels, causing them to die.

"Conventional biomaterials used in tissue regeneration suffer from a variety of problems with delivery, retention and biocompatibility, which can lead to rejection by the host," Kumar says. "We're trying to address these issues with a technology designed to be universal in its application, delivering materials that persist in the tissue and promote their biologic effects for long periods of time."

Reference: Sarkar et al. 2019.Membrane-Disrupting Nanofibrous Peptide Hydrogels. ACS Publications. DOI: https://doi.org/10.1021/acsbiomaterials.9b00967.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Cancer cell therapy success grows, but hospitals struggle with cost – The San Diego Union-Tribune

A lifesaving new cancer therapy using a patients own immune cells continues to show success, but hospitals are struggling with its immense and under-reimbursed costs.

Doctors with these programs at UC San Diego and City of Hope in Duarte gave that cautionary message Thursday at the annual Cell and Gene Meeting on the Mesa in Carlsbad.

The therapy uses immune cells called T cells, which are genetically engineered to fight the patients cancer. Two therapies are approved for commercial use, and several others are in clinical testing.

While these CAR T cell therapies have risks and often fail, they save many extremely ill patients whose disease has grown resistant to multiple previous rounds of treatment, said Dr. John Zaia, director of its Center for Gene Therapy.

But as these therapies become more accepted, hospitals are now faced with a new drain on revenues that needs a long-term solution, Zaia said.

Its a real problem, Zaia said. The institution loses money on the commercial products that we use.

Medicare reimbursement doesnt cover the hospitals expense for the treatment, he said. In addition, patients are very sick and need to be cared for before and after therapy.

It costs up to $1.5 million to manage a CAR T cell patient, he said, citing a study from Oregon Health & Science University.

The success rate with CAR T cells is higher than with other therapies for difficult cancers, Zaia said.

So youre probably getting more value for your dollar if you use CARs, but you still cant afford it. Its got to be solved, because its not a sustainable model.

UC San Diego has similar financial issues with the therapy, said Dr. Dimitrios Tzachanis, an associate clinical professor of medicine at UCSD Blood and Marrow Transplant Program.

It requires strong commitment at the very top of academic centers to say were losing money but we want to be part of this, Tzachanis said. But I dont know how long it will be sustainable.

UCSD administrators are examining CAR T cell costs every month, Tzachanis said.

City of Hope has also seen an unexpected increase in cancer patients who hang on despite being extremely ill, Zaia said. These patients have heard of successes with the therapy and want to give it a try.

Youre talking with patients who are extremely sick about hospice care and end-of-life care, he said. That conversation was curtailed in many cases ... That changes the whole dynamic of patient load into a hospital. Our census in the hospital has been a major problem in the last two years.

City of Hope has been using donor dollars to support the program, Zaia said.

Patients who expect a sure cure are likely to be disappointed, Tzachanis said.

The so-called cure rate is probably in the 40 percent range at this point, he said. So most patients wont respond to the CAR T cells.

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Cancer cell therapy success grows, but hospitals struggle with cost - The San Diego Union-Tribune

CAR T-cell Therapy: Reprogramming the Immune System To Treat Cancer – Technology Networks

At the Malaghan Institute for Biomedical Reserach, researchers are developing third generation CAR-T immunotherapy treatments which aim to be more effective with less side effects than previous therapies.

Using patients own modified T-cells, CAR-T therapy has the ability to vanquish cancer cells, vastly improving outcomes of once life threatening diseases.

This year, 2019, Rob hopes to begin clinical trials for CAR-T therapy in New Zealand through the collaboration of scientists from the Malaghan Institute and international researchers. He is optimistic that this next generation treatment will present a new paradigm for treatment of lymphomas and possibly for other cancers in the future.

Dr Robert Weinkove is Clinical Director of the Malaghan Institute, a biomedical research institute in Wellington, New Zealand. After studying medicine at the University of Cambridge and Kings College London, Rob trained in Hematology in London and Germany before moving to New Zealand in 2008 and gaining a doctorate with the University of Otago.

Rob is a key member of the Cancer Immunotherapy Programme to develop and manufacture chimeric antigen receptor (CAR) T-cells for treatment of lymphoma and other blood cancers. He also holds a joint role as a Hematologist at Wellington Blood & Cancer Centre.

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CAR T-cell Therapy: Reprogramming the Immune System To Treat Cancer - Technology Networks

Global Personalized Cell Therapy Market Analysis Report 2019 || What are Things You Should Keep in Mind to Grow Your Business? – Sound On Sound Fest

New York City, NY: October 07, 2019 Published via (Wired Release) The global Personalized Cell Therapy market is carefully researched in the report while largely focusing on top key players and their business strategies, geographical development, market segments, competitive landscape, manufacturing, and pricing & cost structures. Each section of the research study is specially prepared to explore key aspects of the global Personalized Cell Therapy market. For instance, the market dynamics section digs deep into the drivers, trends, restraints, and opportunities of the global Personalized Cell Therapy Market. With qualitative and quantitative analysis, we help you with thorough and comprehensive research on the global Personalized Cell Therapy market. We have also focused on SWOT, PESTLE, and Porters Five Forces analyses of the global Personalized Cell Therapy market.

Leading players of the global Personalized Cell Therapy market are analyzed taking into account their market share, latest developments, new product launches, partnerships, mergers/acquisitions, and markets served. We also provide a comprehensive analysis of their product portfolios to explore the products and applications they concentrate on when operating in the global Personalized Cell Therapy market. Furthermore, the report offers two separate market forecasts production side and consumption side of the global Personalized Cell Therapy market. It also provides useful recommendations for new as well as established players of the global Personalized Cell Therapy market.

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Cytori Therapeutics Inc, Bellicum Pharmaceuticals Inc, Saneron CCEL Therapeutics Inc, MolMed S.p.A., Vericel Corporation, Oxford Nanopore Technologies, Cell Medica, MediGene AG, TxCell

Personalized Cell Therapy Market Segmentation:

Global personalized cell therapy market segmentation, by technique:

Platelet TransfusionsBone Marrow TransplantationPacked Red Cell TransfusionsOrgan TransplantationGlobal personalized cell therapy market segmentation, by therapeutic area:

Cardiovascular DiseasesNeurological DisordersInflammatory DiseasesDiabetesCancer

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Overview of the Personalized Cell Therapy market share, supply chain analysis

Competitive landscape of key players in Personalized Cell Therapy market

Forecast for global Personalized Cell Therapy market up to 2028

Personalized Cell Therapy Market Overview and success factors

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In conclusion, the global Personalized Cell Therapy market report provides a systematic and descriptive analysis of the Personalized Cell Therapy market, supported by historical and current information of key players and vendors, and all the above-mentioned factors and potential developments in future to aid in gaining crucial insights regarding volume, revenue, and others, which could aid clients in business-related needs.

* Inclusion of Import/export data, production volume are subjected to the scope of the market study

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Cell therapy safe for liver patients, trial shows – Mirage News

Liver disease patients could one day benefit from a new cell therapy that has just completed its first clinical trial.

Researchers who tested the potential treatment in patients with liver cirrhosis where long term damage produces scarring found the therapy had no significant adverse effects.

Now the team, based at the Universitys MRC Centre for Regenerative Medicine, is to gauge the effectiveness of the treatment which is based on white blood cells called macrophages, that are key to normal liver repair.

The next stage of the trial will measure whether the therapy helps the liver to reduce scarring and stimulate regeneration. The results should be known within the next two years.

At present the only successful treatment for end-stage liver cirrhosis which claims around 14,000 lives in the UK each year (British Liver Trust) is an organ transplant. The safety trial is a vital step forward in finding an alternative therapy.

During the trial scientists took cells from the blood of nine patients with the disease and turned them into macrophages, in the Scottish National Blood Transfusion Services (SNBTS) cell therapy facility.

The new cells were then re-injected into the patient with the hope of repairing the damaged organ from within.

Causes of liver cirrhosis include infections such as hepatitis C, obesity, alcohol excess and some genetic and immune conditions.

Liver cirrhosis is a major healthcare issue in the UK and is one of the top five killers. The results from this first safety trial are encouraging and we can now progress to testing how effective it is in a larger group of people. If this was found to be effective it would offer a new way to tackle this important condition.

The research which was published in the journal Nature Medicine, received funding from the Medical Research Council and was conducted in partnership with the SNBTS and the Cell and Gene Therapy Catapult.

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Cell therapy safe for liver patients, trial shows - Mirage News

New Automation Solution to Accelerate Cell and Gene Therapy Manufacturing Announced – Labmate Online

Terumo BCTs latest cell therapy technology, the Finia Fill and Finish System, is a first-of-its-kind device developed to help bring reproducibility and control to cell therapy manufacturing to get therapies to more patients who need them.

Finia is a fully automated, modular, functionally closed system that creates the final formulation of cell and gene therapies and divides them into user-defined doses for patients. The technology automates a process that is currently manual and labour- intensive with the added risk of error.

Terumo BCT technologies have been automating the collection and processing of blood and cells for decades, said Antoinette Gawin, President and Chief Executive Officer, Terumo BCT. Now, were translating this knowledge to support technology breakthroughs in cell and gene therapies. Finia helps cell therapy developers and researchers simplify processes while increasing accuracy, consistency, reliability and reproducibility - key to securing regulatory approval.

The production and delivery of cell-based therapies is a complex and logistically challenging process, said Delara Motlagh, Vice President, Cell Therapy Technologies, Terumo BCT. Developers and manufacturers appreciate support in determining when to automate, which processes to automate and how to integrate automation into their existing process.

Finia combines, mixes and divides the final product into predetermined volumes, all under controlled, refrigerated temperature. It automatically removes air before it seals the final product bags, simplifying the downstream process. Finia also works with Terumo BCT's Cell Processing Application software in the customers' network to facilitate compliance to current good manufacturing practices (cGMP), including electronic recordkeeping, control of process workflow, user credentialing and permission, and guidance of operators through the process with an intuitive user interface module (UIM).

With Finia, we are expanding our portfolio of automation technologies aimed at this growing industry, said Motlagh. We are bringing multiphase solutions for cell and gene therapies helping to scale manufacturing and drive toward commercialisation to reach larger patient numbers.

More information online: https://ilmt.co/PL/6VaE

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New Automation Solution to Accelerate Cell and Gene Therapy Manufacturing Announced - Labmate Online

Arcellx Raises $85 Million in a Series B Financing to Advance its Intelligent Cell Therapy Platform – GlobeNewswire

GAITHERSBURG, Md., Oct. 03, 2019 (GLOBE NEWSWIRE) -- Arcellx, a privately-held biopharmaceutical company, today announced that it has raised $85 million in an oversubscribed Series B financing. Proceeds will be used to advance the Companys ARC-T + sparX programs, including clinical development of a bivalent BCMA-targeted cell therapy in multiple myeloma, and a CD123-targeted therapy in acute myeloid leukemia. The Series B will also fund earlier stage ARC-T + sparX programs for patients with solid tumors and diseases outside oncology.

Participants in the Series B include both existing and new investors to Arcellx. New investors Aju IB and Quan Capital co-led the round, followed by Mirae Asset Venture Investment, Mirae Asset Capital, LG Technology Ventures, JVC Investment Partners, and certain funds managed by Clough Capital Partners, L.P. Existing investors Novo Holdings, S.R. One Limited, NEA and Takeda Ventures also participated in the financing.

Concurrent with the financing, Hugo Beekman, Partner at Aju IB, and Lewis (Rusty) Williams, M.D., Ph.D., Venture Partner at Quan Capital, have joined the Arcellx board of directors.

The financial and strategic support from our investors allows Arcellx to accelerate development of a robust pipeline of ARC-T + sparX programs for patients in need, commented David Hilbert, Ph.D., President and Chief Executive Officer of Arcellx. As impressive as conventional CART therapies have been, their safety and efficacy profiles are challenged by severe toxicities, high rates of relapse, and challenging target selection in the solid tumor setting. The ARC-T + sparX platform addresses these concerns by placing ARC-T cells under the control of one or more sparX proteins that uniquely determine how the ARC-T cells recognize tumor, and the speed with which ARC-T cells kill tumor. In the coming months we will begin clinical testing of our lead BCMA-targeted therapy in multiple myeloma.

Rusty Williams, M.D., Ph.D., commented, Arcellx hasreached a positive inflectionin its novel platform and pipeline with the potential to improve efficacy and safety. We are excited to support the company as it advances new cell therapies with the potential to deliver better outcomes for patients.

Hugo Beekman, Partner at Aju IB, commented, Arcellx has invented a differentiated cell therapy platform with ARC-T + sparX that allows simultaneous and sequential targeting of multiple tumor antigens. The ability of sparX proteins to reprogram the specificity of ARC-T cells has the potential to address the high incidence of tumor relapse, as well as the inherent diversity of tumor antigens expressed within solid tumors. The features of this platform, along with scalable and efficient manufacturing processes, are intended to facilitate the Companys development of new therapies in oncology, and more broadly, in autoimmune disease and the transplant setting.

About ARC-T + sparX Technology

Arcellx has pioneered a proprietary sparX + ARC-T platform in which a Soluble Protein Antigen-Receptor X-linker (sparX) simultaneously binds one or more tumor antigens and engages a universal receptor expressed on the Antigen-Receptor Complex T cells (ARC-T). The formation of a sparX + ARC-T + tumor cell complex results in tumor killing. This therapeutic platform is designed to enhance safety and efficacy while accelerating development by broadening patient accessibility and increasing efficiency of manufacturing relative to existing cell therapies.

About Arcellx, Inc.

Arcellx is a privately held biopharmaceutical company located in Montgomery County, Maryland. The Arcellx team is devoted to providing patients with superior immune cell therapies through scientific innovation, accelerated development, and responsible patient care. Although our initial clinical focus is cancer therapy, we are committed to extending our Antigen-Receptor Complex T cell (ARC-T) therapies across a broad spectrum of human disease.

Contact:Solebury TroutZara Lockshin (media)Tel: +1 646-378-2960Email: zlockshin@troutgroup.com

Alan Lada (investors)Tel: +1 646-378-2927Email: alada@troutgroup.com

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Arcellx Raises $85 Million in a Series B Financing to Advance its Intelligent Cell Therapy Platform - GlobeNewswire

Debating the Use of MRD Testing for Treatment Decision Making – AJMC.com Managed Markets Network

Although minimal residual disease (MRD) is increasingly being used to predict treatment outcomes and as a surrogate marker of progression-free survival, there remains controversy over whether it is ready to be used in treatment decision making.

There are some scenarios when having MRD information would make management decisions easier, according to an abstract from Jeffrey L. Wolf, MD, of the University of California, San Francisco.1 One scenario is when clinicians are deciding whether or not to stop maintenance therapy. There are likely many situations in which patients are being overtreated because patients stay on prolonged maintenance therapy until progression. These therapies not only are costly, but they also have side effects.

The knowledge that a patient has achieved MRD negativity year after year would certainly help with the decision to stop therapy and not necessarily start another agent, Wolf wrote.

In another scenario, rising MRD could indicate to clinicians that the current therapy was ineffective and should be stopped and that the patient should perhaps even switch to another therapy.

In both scenarios, Wolf noted that there would be no issue to use MRD in this way if only MRD was a peripheral blood test. However, MRD testing is currently a bone marrow test, which is why it is not used more. He argued that a bone marrow procedure should not stand in the way of MRD testing being used more.

Wolf added that MRD would be helpful for clinicians deciding whether to proceed with renal allograftingpatients with persistent MRD negativity would do well to have the allograft, he wroteor whether or not to transplantnegative MRD after induction could mean a patient does just as well going to maintenance therapy rather than being subjected to transplant first.

I conclude that the time has come to begin to use MRD, along with other information, to make clinical decisions, Wolf wrote.

However, in another abstract from the meeting, Thomas Martin, MD, of the University of California, San Francisco, argues that MRD testing has not realized its true potential yet in multiple myeloma.2

Martin agreed that blood-based testing would allow for greater use of MRD testing, but it is currently not ready for primetime, and, for now, MRD assessments should only be used to evaluate prognosis.

Some remaining important questions include how many cells are needed from the bone marrow biopsy to be an adequate sample. In addition, Martin noted that cross-study comparisons remain difficult as studies report MRD-negative results in different ways. For instance, some chimeric antigen receptor T-cell therapy studies label patients as MRD negative even when they have detectable m-proteins; however, this does not align with MRD negativity as defined by the International Myeloma Working Group.

MRD results provide prognostic information only and future clinical trials with adaptive designs will define how MRD can guide treatment decisions, Martin concluded.

References

1. Wolf JL. Debate: can MRD be used for treatment decisions? Yes!!! Clin Lymphoma Myeloma Leuk. 2019;19(suppl 1):S44-S45.

2. Martin T. Multiple myeloma: minimal residual disease testingnot ready for primetime. Clin Lymphoma Myeloma Leuk. 2019;19(suppl 1):S46-S48.

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Genprex’s Pioneering Use of Non-Viral Delivery for Gene Therapy is Gaining Industry Support – Business Wire

AUSTIN, Texas & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genprex, Inc. (NASDAQ: GNPX), a clinical-stage company and leader in gene therapy using non-viral vector transfection delivery, wishes to draw attention to additional research in the field validating non-viral vector delivery as the next evolution in gene therapy.

A recently published paper by researchers in Australia, Spain and Austria supports the belief that non-viral delivery could be safer for patients than viral vectors and could speed up the production time while reducing the costs of production. The paper titled, Encapsulation, Visualization and Expression of Genes with Biomimetically Mineralized Zeolitic Imidazolate Framework-8 (ZIF-8) published in the September 4, 2019 issue of the scientific journal Small, presents data from a biomolecule-metal-organic framework (nano MOF) in zeolitic imidazolate framework-8 (ZIF-8) and found it to be a viable vehicle for intracellular transfection and gene delivery. Genprex was not involved in the study, which used a different nanotechnology.

One of the biggest differentiators between Genprex and other gene therapy companies developing technologies to treat cancer and other serious diseases is our proprietary non-viral nanoparticle delivery system, which has already been used to safely treat more than 50 patients to date, said Genprexs Chairman and Chief Executive Officer, Rodney Varner. Most gene therapy research has been focused on using viral delivery systems to deliver genes to cancer cells, and today most approved gene therapies for non-blood cell therapies use a viral vector to deliver the gene to the patient. Our proprietary non-viral delivery system enables us to potentially treat patients with a system that may be safer, with lower production costs and better scalability.

Based on the shortcomings that viral vectors have historically had, including severe adverse reactions, high production costs, difficulty in scaling and high immunogenicity responses, Genprexs founders partnered with the National Institutes of Health (NIH) during the companys inception to develop its proprietary non-viral delivery system.

Specifically, Genprexs platform technologies are designed to administer cancer fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. The nanovesicles are non-immunogenic, allowing repetitive therapeutic dosing. Genprexs nanovesicles are also clinically proven to effectively deliver molecular kinase inhibitors effectively.

A Phase I human clinical trial showed that Genprexs lead drug candidate, Oncoprex immunogene therapy, which is delivered through its nanovesicle non-viral delivery system, selectively and preferentially targeted primary and metastatic tumor cells, resulting in clinically significant anticancer activity. Genprexs clinical trials have also demonstrated that its delivery system is well tolerated in humans and can safely deliver high therapeutic doses.

About Genprex, Inc.

Genprex, Inc. is a clinical stage gene therapy company developing potentially life-changing technologies for cancer patients, based upon a unique proprietary technology platform, including Genprexs initial product candidate, Oncoprex immunogene therapy for non-small cell lung cancer (NSCLC). Genprexs platform technologies are designed to administer cancer fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. Oncoprex has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for apoptosis, or programmed cell death, in cancer cells, and modulates the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. 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 effects of Oncoprex on cancer and the safety, production cost and scalability of Oncoprex and its non-viral delivery system. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of Oncoprexs effect on cancer, the safety, cost and scalability of Oncoprex and its delivery system, as well as the timing and success of our clinical trials and planned clinical trials, Oncoprex and our other potential product candidates. These and other risks and uncertainties associated with Genprex and its lead product candidate Oncoprex 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.

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Genprex's Pioneering Use of Non-Viral Delivery for Gene Therapy is Gaining Industry Support - Business Wire

New NIH viral vector flips the script on sickle cell disease gene therapy – Endpoints News

Researchers at the NIH have rolled out a new vehicle for sickle cell gene therapy with higher speeds and better horsepower, potentially allowing vastly more efficient gene transfer and a much larger carrying capacity. The best part? Unlike current sickle cell gene therapy models, the NIH one doesnt have to drive in reverse.

In mice and monkeys, the new vehicle was up to 10 times more efficient and had a carrying capacity the amount of DNA it can haul of up to 6 times that of the conventional vectors currently deployed in gene therapy trials across the country. Most notably, the new vector can read the therapeutic gene sequence forward rather than reading them backward a counter-intuitive trick researchers had used to overcome long-running barriers to gene therapy but which sacrificed efficiency. The results were published open access inNature Communications.

Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease, said study senior author John Tisdale, chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI). Its the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high-efficiency vectors for treating this devastating disorder.

Gene therapy trials for SCD have launched the past few years, bringing a handful of well-covered cases of patients responding strongly to the treatment, even as more data shows current techniques are no cure-all. One of the bigger longstanding questions, though, is how to best deliver the genetic fix.

The simple genetic underpinnings of the disease have been well-understood since the 1950s one A-T substitution in the -globin gene and researchers have accordingly targeted it since the first gene therapy research in the 1980s. But the particular problems of building a proper vector for the hemoglobin gene, in addition to the myriad other obstacles to gene therapy broadly, have impeded progress.

Click on the image to see the full-sized version

The lentiviral vector bluebird bio has used to bring its sickle cell gene therapy to trial is a workaround to an early problem unique to sickle cell therapy. RNA splicing a natural process critical to preparing the vector will remove introns that are key to expressing the genes to produce hemoglobin. Developers have been able to get around this by using a vector that reads the DNA backwards, last gene to first. Most gene therapy techniques read as you would a sentence, first word to last.

The researchers also noted their vectors were cheaper to produce.

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New NIH viral vector flips the script on sickle cell disease gene therapy - Endpoints News