Kadimastem Finishes Treating Second Group of ALS Patients with AstroRx in Phase 1/2a Trial – ALS News Today

Kadimastem has finished treating its second group of participants in a Phase 1/2a clinical trial testing the safety and preliminary efficacy of AstroRx, aninvestigational stem cell therapy for amyotrophic lateral sclerosis (ALS).

AstroRx is an off-the-shelf cell therapy consisting of fully mature astrocytes star-shaped cells derived from human embryonic stem cells that are injected into the fluid surrounding the spinal cord to support damaged motor neurons.

Astrocytes usually help maintain a healthy environment in the brain, but often malfunction in ALS, contributing to disease progression. AstroRx was designed to compensate for diseased astrocytes and prevent the loss of motor nerve cells. The therapys goal is to potentially slow disease progression, improve quality of life, and extend life expectancy.

The U.S. Food and Drug Administration grantedAstroRx orphan drug status in November 2018 for the treatment of ALS, a designation meant to accelerate the development of AstroRx for this rare condition.

Earlier preclinical studiesshowed that AstroRx delayed disease onset, maintained muscle function, and prolonged survival in mice and rat models of ALS. The treatment also was found to be safe, with no signs of toxicity.

The ongoing, open-label, Phase 1/2a trial (NCT03482050) is testing the safety and preliminary signs of efficacy of AstroRx in people with ALS. Underway at the Hadassah Ein-Kerem Medical Center in Israel, the trial is expected to enroll 21 patients, ages 18-70, with early stage disease. Recruitment is ongoing.

The trial was originally designed to test three doses of AstroRx delivered into the spinal canal: a low dose of 100 million cells, a medium dose of 250 million cells, and a high dose with two consecutive administrations of 250 million cells.

However, after promising early efficacy results from the low-dose group (cohort A), Kadimastem is seeking to amend the trial protocol. It wants the third group of patients (cohort C) to receive two consecutive injections of the low dose, instead of the originally planned medium dose. Pending positive safety and efficacy results from the first three cohorts, a fourth group (cohort D) will receive two injections of the medium dose.

Safety and tolerability are the studys primary outcome measures. Secondary goals include changes in patients ALS Functional Rating Scale revised (ALSFRS-R) scores, respiratory muscle strength, hand grip strength, limb muscle strength, and quality of life.

In cohort A, the low-dose group, the therapy was found to be safe, with no serious side effects or dose-limiting toxicities. Participant had increased ALSFRS-R scores in the three months after treatment, suggesting a gain in functional abilities.

The trial has now treated all five ALS patients in cohort B, the second group, with the medium dose. Participants will be monitored for six months to evaluate the therapys safety and preliminary efficacy. The company expects to report the results for this group in August 2020.

Completing treatment for the additional 5 ALS patients in Cohort B, for a total of 10 patients treated with our product in our clinical trial, serves as an additional demonstration of our ability to develop and produce high quality clinical grade cells and takes us a significant step forward in our path to bringing innovative cure to ALS, Rami Epstein, CEO of Kadimastem, said in a press release.

The expected completion of cohort B 6-months follow-up period will allow us to assess the safety and preliminary efficacy of [250 million]cells, compared to that of the lower dose administered in cohort A, he added.

Kadimastem has recruited the first patient of cohort C, who all will receive two AstroRx injections of 100 million cells, separated by 2-3 months. Results from this group are expected during the first half of 2021.

The results of the next treatment group, Cohort C, in which each patient will be treated with two consecutive injections separated by an interval of 2-3 months, will allow us to assess the possible prolonged efficacy of the repeated dose, compared to the single dose treatment provided in cohorts A and B, said Michel Revel, founder and chief scientific officer of Kadimastem.

The results that will be obtained from the different cohorts, will support us in the process of defining the dose and treatment regimen that will lead to most favorable results for patients over time, Revel said.

Alejandra has a PhD in Genetics from So Paulo State University (UNESP) and is currently working as a scientific writer, editor, and translator. As a writer for BioNews, she is fulfilling her passion for making scientific data easily available and understandable to the general public. Aside from her work with BioNews, she also works as a language editor for non-English speaking authors and is an author of science books for kids.

Total Posts: 6

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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Kadimastem Finishes Treating Second Group of ALS Patients with AstroRx in Phase 1/2a Trial - ALS News Today

Global Stem Cell Therapies Overview Report 2019: The Highs & Lows, Off-The-Shelf Solutions, New Technologies, and Safety, Efficacy & Logistical…

DUBLIN, Jan. 8, 2020 /PRNewswire/ -- The "Highs and Lows of Stem Cell Therapies: Off-The-Shelf Solutions" report has been added to ResearchAndMarkets.com's offering.

The report includes:

Key Topics Covered

Chapter 1 Sources and Characteristics of Stem Cells

Chapter 2 New Technologies Driving Stem Cell Development

Chapter 3 Safety, Efficacy and Logistical Challenges

Chapter 4 Off-the-Shelf Solutions

Chapter 5 Companies and Technologies to Watch

Chapter 6 References

List of TablesTable 1: Stem Cell SourcesTable 2: Comparison of Stem CellsTable 3: Stem Cell Types Versus Cell PropertiesTable 4: Recent Advances in Genome Modulation TechnologiesTable 5: FDA-Approved Cell-Based TherapiesTable 6: Acute and Delayed Adverse Events Associated with Proven and Unproven Stem Cell-Based InterventionsTable 7: Companies Leading the Field in Cell-Based Therapies

List of FiguresFigure 1: Stem Cell Sources Figure 2: Two-Dimensional Versus Three-Dimensional Cell CultureFigure 3: Schematic of Approaches Used for Three-Dimensional Stem Cell Culture Figure 4: Patient Management Strategies to Decrease Side Effects Related with CAR-T Cell Therapy

For more information about this report visit https://www.researchandmarkets.com/r/15o8p9

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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Jasper Therapeutics Announces Expansion of Series A Financing, Bringing Total Corporate Fundraising to More than $50 Million – Business Wire

PALO ALTO, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the expansion of its Series A financing with an additional investment of $14.1 million led by Roche Venture Fund and with participation from other investors, bringing the total company financing to more than $50 million to date. The initial Series A round was led by Abingworth LLP and Qiming Venture Partners USA, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC.

Jasper plans to use the proceeds to advance and expand the study of its lead clinical asset, JSP191. A humanized antibody targeting CD117 on hematopoietic stem cells, JSP191 is designed to replace toxic chemotherapy and radiation therapy as conditioning regimens to prepare patients for curative stem cell and gene therapy. JSP191 is the only antibody of its kind in clinical development as a single conditioning agent for people undergoing curative hematopoietic cell transplantation.

This investigational agent is currently being evaluated in a Phase 1/2 dose-escalation and expansion study as a conditioning agent to enable stem cell engraftment in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that resulted in poor outcome. Initial positive results from this ongoing clinical trial were presented in an oral session at the American Society of Hematology (ASH) Annual Meeting in December 2019. Jasper plans to expand the Phase 1/2 clinical study to include patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) receiving hematopoietic cell transplant. The development of JSP191 is supported by a collaboration with the California Institute for Regenerative Medicine (CIRM).

About Hematopoietic Cell TransplantationBlood-forming, or hematopoietic, stem cells are rare cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow by transplantation and engraftment of healthy stem cells is the only cure for most of these life-threatening conditions. Successful transplantation is currently achieved by subjecting patients to toxic treatment with radiation and/or chemotherapy followed by transplantation of a donor or gene-corrected hematopoietic cell graft. These toxic regimens cause DNA damage and lead to short- and long-term toxicities, including unwanted damage to organs and prolonged hospitalization. As a result, many patients who could benefit from a hematopoietic cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit http://www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant. IND-enabling studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper TherapeuticsJasper Therapeutics is a biotechnology company focused on hematopoietic cell transplant therapies. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at https://jaspertherapeutics.com.

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Jasper Therapeutics Announces Expansion of Series A Financing, Bringing Total Corporate Fundraising to More than $50 Million - Business Wire

Q&A: Cancer Death Rates Are Falling Nationally. Here’s What’s Happening at UVA – University of Virginia

This week, the American Cancer Society released some very welcome news: the cancer death rate in the U.S. dropped by 2.2% from 2016 to 2017, the largest single-year drop ever recorded.

The drop, which the report attributes to plummeting smoking rates as well as new screening and treatment methods, continues a decades-long trend, as cancer death rates have fallen by nearly 30% since 1991 about 2.9 million fewer deaths.

Dr. Thomas Loughran, director of the University of Virginia Cancer Center, said UVA is in step with this national trend.

The UVA Cancer Center is one of 71 National Cancer Institute-designated treatment centers nationwide and ranked among the nations top 50 cancer centers over each of the past four years (No. 26 last year). The center serves approximately 4 million people in Virginia and West Virginia.

We spoke with Loughran about what he is seeing at UVA and beyond, new treatments and research helping to eradicate cancer, and where he sees cancer treatment in five years.

Q. Why have cancer death rates dropped so significantly?

A. As reports of this latest drop have said, a large part of the decline can be attributed to declining rates of lung cancer. The importance of preventing cancer particularly behavioral interventions like stopping smoking has become more prominent, and there have been remarkable declines in smoking across the United States.

This is a very important focus for us at UVA. We serve a large geographical area 90 contiguous counties in Virginia and West Virginia, including rural Appalachia. Southwest Virginia in Appalachia still has high smoking rates, and as a result, high rates of lung cancer. Education, screening and tobacco cessation programs are critically important, especially in those areas.

Q. What advances in treatment have contributed to falling cancer death rates, nationally and at UVA?

A. Screening technology, especially for the more common cancers like lung, colorectal, prostate and breast cancer, has improved. The latest report probably doesnt fully reflect recent implementation of lung cancer screening using a low-dose CT scan, recommended for high risk individuals and especially those with a history of heavy smoking. That has only been around a few years, and its impact will likely show up in future reports.

The second big factor is the development of immunotherapy [cancer treatments that utilize and help the patients immune system]. UVA has invested quite a lot of institutional resources in becoming a state-of-the-art immunotherapy center, and I am proud to say we are a leader in the field.

We have created a Cancer Therapeutics Program to support the development of new therapies. Dr. Craig Slingluff, who leads that program, is a surgical oncologist internationally famous for immunotherapy treatments for melanoma. To strengthen this program, we have recruited a cadre of leading physician scientists from across the country. Dr. Karen Ballen came here to lead our stem cell and bone marrow transplant program. Dr. Lawrence Lum, the scientific director of the transplant program, has developed a novel therapy using antibodies that bind to both T-cells [patient cells that can kill cancer cells] and tumor cells, forming a bridge between the two that helps the T-cells kill the cancer cells. Dr. Trey Lee is a leader in CAR-T cell therapy.

I could keep going; there are so many great people working on this. We also have a new Good Manufacturing Practice lab, supported by a grant from the commonwealth, that will help us grow and modify T-cells as needed and give them to patients under sterile conditions. That just opened and we are very excited about that program.

Q. What other areas of research have shown great promise?

A. Some of our work in nanotechnology is really unique and exciting. [Biomedical engineering professor] Mark Kester directs UVAs nanoSTAR Institute, which is working on delivering cancer therapies by nanotechnology basically, engineering at a very small scale. For example, nanoliposomes a sort of delivery system for cancer therapy are actually smaller than individual cells and can therefore penetrate cancer cells and release treatment from inside those cells.

We are very excited about early phase trials testing this technology on solid tumors, and we also hope to use it to treat patients with acute leukemia over the next few years.

Q. Looking ahead, where do you see the next big gains coming from?

A. Immunotherapy has revolutionized cancer treatment, but why some patients respond well and some dont remains puzzling. I hope that we can begin to discover why some patients are reacting to these newer treatments differently than others. Once we figure out why some patients respond to immunotherapy, we can begin to make improvements that could benefit a larger percentage of patients with these deadly cancers.

CAR T Cell therapy one method of immunotherapy is very effective against leukemia, lymphoma and cancers of the blood, but not yet against solid tumors. Over the next five years, I hope we can determine how to deliver these T-cells to solid tumors such as those found in lung, colorectal and other common cancers again to make this advance more widely applicable to a larger number of patients.

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Q&A: Cancer Death Rates Are Falling Nationally. Here's What's Happening at UVA - University of Virginia

SVC112 molecule reduces effects of cancer stem cells with little toxicity – Drug Target Review

A study has shown that the SVC112 molecule can prevent cancer stem cells from producing more tumour cells in cell lines and mice, with no impact on healthy cells.

Researchers have found that SVC112, a molecule based on the chemical bouvardin, can act specifically against head and neck cancer stem cells (CSCs).

Bouvardin can be extracted from the Bouvardia ternifolia bush, native to Central America. Research from the University of Colorado, US has previously shown that bouvardin can slow a cancers ability to make proteins which allow it to grow and metastasise.

As CSCs manufacture cells that become the bulk of cancer tissue, they are a viable drug target. However, they often resist treatments like radiation and chemotherapy.

when the population of stem cells was reduced to under one percent of the tumour cells, the tumour began to shrink

The current study, conducted by scientists at SuviCa, synthesised SVC112 in order to control tumours more effectively than current FDA-approved protein synthesis inhibitors and with less toxicity.

The groups work showed that SVC112 acts specifically against proteins like Myc and Sox2 which are required by CSCs, while leaving healthy cells unharmed. They demonstrated this by comparing the effects of the drug in matched pairs of cancer cells and healthy cells grown from samples donated by five head and neck cancer patients.

Many groups have linked the production of transcription factors to the survival and growth of CSCs, but inhibitors have just been too toxic they come with too many side effects. Definitely our studies suggest that this drug could be an advantage over existing drugs. It inhibits protein synthesis in a way that no other drug does and thats why were excited, says lead researcher Dr Tin Tin Su.

For further comparison, the researchers conducted the same experiment with the FDA-approved protein synthesis inhibitor omacetaxine mepesuccinate (also called homoharringtonin, or HHT).

Having cancer cells along with matched non-cancer cells from the same patient is pretty unique. When we tested these matched pairs with SVC112 and with HHT, what we saw is the approved drug eliminated both cancer and normal cells, whereas SVC112 had selectivity it affected cancer cells but not healthy cells so theoretically the effects on the normal tissue will be less, Su says.

Next, the team used SVC112 to treat head and neck tumours in mouse models, grown from samples of human tumours. Earlier work showed that SVC112 sensitised previously radiation-resistant CSCs to radiation treatment, so the group tested the molecule and radiation alone and in combination.

They found that when the population of stem cells was reduced to under one percent of the tumour cells, the tumour began to shrink. They concluded that the CSCs act as controllers for the growth of tumours, so impairing this group of stem cells will slow down or stop cancer.

The researchers hope that their pre-clinical results will lay the groundwork for human clinical trials of SVC112 in head and neck cancer patients.

The findings were published in Cancer Research.

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SVC112 molecule reduces effects of cancer stem cells with little toxicity - Drug Target Review

Helius Medical Technologies Awarded Pioneer Technology Development AwardBrain Mapping Foundation and Society for Brain Mapping Therapeutics name the…

NEWTOWN, Pa., Jan. 10, 2020 (GLOBE NEWSWIRE) -- Helius Medical Technologies, Inc. (NASDAQ:HSDT) (TSX:HSM) (Helius or the Company), a leading neurotech company focused on neurological wellness, today announced that it is the recipient of the Pioneer Technology Development Award for its development of the PoNS device. This prestigious award, presented by the Brain Mapping Foundation (BMF) and Society for Brain Mapping and Therapeutics (SBMT), is given to trailblazing technology companies and their CEOs/Presidents who have facilitated the development of pioneering technologies through interdisciplinary approaches that have impacted diagnostics, treatment, and healthcare delivery in unprecedented ways.

Annually the BMF, SBMT award committee identifies many cutting-edge technologies but we only award those who are truly visionary, pioneering and lifesaving inventions. This year we have identified two pioneering technologies, one developed by Helius Medical Technologies, a neuromodulation device, and another, developed by Monteris Medical, a brain tumor ablation device, says Babak Kateb, M.D., Founding Chairman of the Board of Directors, CEO and Scientific Director, Society for Brain Mapping & Therapeutics (SBMT). Retinal, peripheral and or direct neuromodulation are the future for treatment of neurological disorders. SBMTs role is to make sure game-changing diagnostics and therapeutics such as PoNS are identified and provided to patients with limited treatment options in the US and around the world.

The PoNS device delivers mild electrical stimulation to the nerves of the tongue that connect with the nerves in the brain. When this stimulation is combined with physical activity, changes may occur in the neural network, which may result in improvements of balance and gait. The device is intended for use as a short-term treatment (14 weeks) of chronic balance deficit due to mild-to-moderate traumatic brain injury (mmTBI) and is to be used in conjunction with physical therapy.

I am honored to receive this award on behalf of the gifted scientists who developed the PoNS device and for all the stakeholders that have made its development intended for the treatment of neurological symptoms of disease or trauma possible, said Philippe Deschamps, Chief Executive Officer of Helius.

About Helius Medical Technologies, Inc.

Helius Medical Technologies is a neurotech company focused on neurological wellness. The Companys purpose is to develop, license and acquire unique and non-invasive platform technologies that amplify the brains ability to heal itself. The Companys first commercial product is the Portable Neuromodulation Stimulator (PoNS). For more information, visit http://www.heliusmedical.com.

About the Society for Brain Mapping and Therapeutics

The Society for Brain Mapping and Therapeutics (SBMT) is a non-profit society organized for the purpose of encouraging basic and clinical scientists who are interested in areas of Brain Mapping, engineering, stem cell, nanotechnology, imaging and medical device to improve the diagnosis, treatment and rehabilitation of patients afflicted with neurological disorders.

This society promotes the public welfare and improves patient care through the translation of new technologies/therapies into lifesaving diagnostic and therapeutic procedures. The society is committed to excellence in education, and scientific discovery. The society achieves its mission through multi-disciplinary collaborations with government agencies, patient advocacy groups, educational institutes and industry as well as a philanthropic organization.

The Society for Brain Mapping and Therapeutics (SBMT) was founded in 2004 to break boundaries in healthcare. The society promotes policies that support rapid, safe, and cost-effective translation of new technology into medicine.

Translational research applies cutting edge basic science and advanced technologies to clinical neurosciences. The Society examines emerging disciplines such as nanotechnology, image-guided therapy, stem cell therapy, neuromodulation, multi-modality imaging, biophotonics, and biomaterial and tissue engineering for their application to the diagnosis, treatment, and rehabilitation from neurological diseases. The Society seeks to apply these technologies to clinical problems such as brain tumors, stroke, epilepsy, neurodegenerative diseases (Parkinson, Alzheimers, multiple sclerosis and ALS), traumatic brain and spinal cord injuries, autism, post-traumatic stress disorder and other psychiatric illnesses.

About the PoNS Device and PoNS Treatment

The Portable Neuromodulation Stimulator (PoNS) is an authorized class II, non-implantable, medical device in Canada intended for use as a short term treatment (14 weeks) of chronic balance deficit due to mild-to-moderate traumatic brain injury (mmTBI) and is to be used in conjunction with physical therapy. The PoNS is an investigational medical device in the United States, the European Union (EU), and Australia (AUS), and it is currently under review for clearance by the AUS Therapeutic Goods Administration. PoNS Treatment is not commercially available in the United States, the European Union or Australia.

Investor Relations Contact:

Westwicke Partners on behalf of Helius Medical Technologies, Inc.Mike Piccinino, CFA443-213-0500investorrelations@heliusmedical.com

Cautionary Disclaimer Statement:

Certain statements in this news release are not based on historical facts and constitute forward-looking statements or forward-looking information within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and Canadian securities laws. All statements other than statements of historical fact included in this news release are forward-looking statements that involve risks and uncertainties. Forward-looking statements are often identified by terms such as believe, continue, look forward, will and similar expressions. Such forward-looking statements include, among others, statements regarding the Companys future clinical and regulatory development plans for the PoNS device and the potential regulatory clearance of the PoNS device.

There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those expressed or implied by such statements. Important factors that could cause actual results to differ materially from the Companys expectations include the uncertainties associated with the marketing, sale and regulation of medical devices in Canada, the clinical development, regulatory submission and approval process in the United States, the European Union and Australia, as well as the Companys capital requirements needed to achieve its business objectives and its ability to raise the needed capital, as well as other risks detailed from time to time in the filings made by the Company with securities regulators, and including the risks and uncertainties about the Companys business described in the Risk Factors sections of the Companys Annual Report on Form 10-K for the year ended December 31, 2018, its Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 and its other filings with the United States Securities and Exchange Commission and the Canadian securities regulators, which can be obtained from either at http://www.sec.gov or http://www.sedar.com.

The reader is cautioned not to place undue reliance on any forward-looking statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company assumes no obligation to update any forward-looking statement or to update the reasons why actual results could differ from such statements except to the extent required by law.

The Toronto Stock Exchange has not reviewed and does not accept responsibility for the adequacy or accuracy of the content of this news release.

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ANP Technologies, in Partnership with Fulgent Pharma, Teams with Moffitt Cancer Center to Develop a New Class of Leukemia Therapies – Business Wire

NEWARK, Del.--(BUSINESS WIRE)--ANP Technologies Inc. (ANP) and Fulgent Pharma LLC through their partner Moffitt Cancer Center have successfully licensed the rights to develop a novel targeted therapy in the area of leukemia to Celgene (CELG), now Bristol Myers Squibb (BMY), a landmark deal that leverages ANPs nanotherapeutic platform technology. The partners will work together to develop a new cancer therapy for Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML). The potential new therapy will target a novel pathway receptor.

The Moffitt research team recently discovered that a specific pathway receptor is up-regulated in MDS and AML malignant cells, and in particular the malignant stem cells, thus offering a potentially favorable disease-specific target for therapies. By utilizing a ligand specific for this pathway receptor along with a covalently linked nanoparticle developed by ANP and licensed to Fulgent Pharma, the team was able to show potential for treating this type of leukemia at the stem cell level.

Moffitt takes a team approach when it comes to cancer care and research. Our immunology and hematology teams worked together on this novel therapy. We are taking it to the next level, partnering with ANP/Fulgent Pharma to help accelerate translating this discovery from the laboratory to patients in need, said Jarett Rieger, Sr. Director, Innovation & Industry Alliances of Moffitt.

With our proprietary nano-delivery and nanotherapeutic technology platform, ANP has successfully developed multiple therapies including nanoencapsulated pactlitaxel, which is currently in clinical and licensed to Fulgent Pharma, as well as a nanoencapsulated antibody cocktail of drugs for the treatment of Ebola infection, which was funded for nonhuman primate testing by the US Department of Defense, says Dr. Ray Yin, President and CEO of ANP. The Moffitt collaboration expands our nanotechnology platform and spectrum of drug development, enabling ANP and Fulgent Pharma to develop new targeted therapies to benefit cancer patients.

About Moffitt Cancer Center

Moffitt is dedicated to one lifesaving mission: to contribute to the prevention and cure of cancer. The Tampa-based facility is one of only 51 National Cancer Institute-designated Comprehensive Cancer Centers, a distinction that recognizes Moffitts scientific excellence, multidisciplinary research, and robust training and education. Moffitt is a Top 10 cancer hospital and has been nationally ranked by U.S. News & World Report since 1999. Moffitts expert nursing staff is recognized by the American Nurses Credentialing Center with Magnet status, its highest distinction. With more than 6,500 team members, Moffitt has an economic impact in the state of $2.4 billion. For more information, call 1-888-MOFFITT (1-888-663-3488), visit MOFFITT.org, and follow the momentum on Facebook, Twitter, Instagram and YouTube.

About ANP Technologies, Inc.

ANP Technologies, Inc. is a world leader in developing innovative nano-therapeutics. In addition to the novel targeted therapy, ANP has also developed nanoencapsulated chemotherapeutics, antibody therapies, immune-oncology and mRNA-based vaccines. Visit ANPTINC.com for more information.

About Fulgent Pharma

Fulgent Pharma is a clinical-stage specialty pharmaceutical company developing oncology therapies that leverage a proprietary nano-drug delivery technology. Fulgent Pharmas pipeline features three unique drug platforms: nanoencapsulated chemotherapy drugs being developed via the 505(b)(2) pathway, novel targeted therapies, and small molecule based immuno-oncology drugs. The Companys lead asset, FID-007, is a nanoencapsulated paclitaxel with improved drug solubility and efficacy, as well as decreased toxicity, and is currently tested in clinical trials. Fulgent Pharma was founded in 2015 and is headquartered in Temple City, California. Fulgent Pharma was spun off from Fulgent Genetics, Inc., (NASDAQ:FLGT) a comprehensive genetic testing company, in 2016.

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ANP Technologies, in Partnership with Fulgent Pharma, Teams with Moffitt Cancer Center to Develop a New Class of Leukemia Therapies - Business Wire

Orgenesis adds UC Davis to its point-of-care network for developing cell, gene therapy products – Proactive Investors USA & Canada

The first project with University of California, Davis is on developing and commercializing a lentiviral manufacturing system

Inc (), a developer of advanced cell therapies, said Friday that it has added the University of California, Davis (UC Davis) to its point-of-care network.

In a statement, the Germantown, Maryland-based company, said that under the terms of the agreement, UC Davis Health will utilize Orgenesis point-of-care platform to develop, commercialize and supply cell and gene products and therapies.

The point-of-care platform is designedto enable hospitals to implement Orgenesis proprietary automated, closed systems and know-how to collect, process and supply cells within the patient care setting for various treatments.

The first collaboration under the deal involves scaling up and integrating UC Davis lentiviral vector process as part of the Orgenesis point-of-care network platform for the development of cell and gene therapies for treating patients, said the company.

According to the company, the UC Davis GMP facility has developed a small-intermediate scale, high-quality vector process that has been successfully utilized to manufacture lentiviral vectors in several clinical trials, including the manufacturing of CAR T cell therapies. Its point-of-care network, which combines processing and therapeutic technologies, is designed to allow for the efficient production of high quality, affordable cell, and gene-based products.

On successfully completing the collaboration, Orgenesis and UC Davis plan to pursue the commercialization of the technology and expand the processing and supply of their products under development at the UC Davis site. Lentivirus is a family of viruses that insert their DNA into the host cells' genome. Lentiviral vectors are increasingly utilized in cell and gene therapy as a method for inserting, modifying, or deleting specific genes within cells.

We are delighted to add UC Davis to our point-of-care network, which will allow us to collaborate with the university to develop and supply therapeutics within the point-of-care setting in general and specifically in our need for virus supply, said Orgenesis CEO Vered Caplan.

Orgenesis' boss said she looked forward to leveraging the companys point-of-care platform to assist UC Davis in expanding its ability to address the worldwide shortages of lentiviral vectors.

Their new system is designed to address the global need for a more efficient, large scale vector manufacturing processes in an efficient manner. We believe this partnership further validates the significant value proposition of our point-of-care, said Caplan.

For the fiscal third quarterended September 30, Orgenesis generated meaningful revenue, over $1 million, through its rapidly advancing point-of-care cellular therapy platform.

We, at UC Davis Health, in the Stem Cell Program and in the GMP Facility, are committed to bringing these novel cell and gene therapy based treatments to patients in need and making them affordable, said Adjunct Professor Gerhard Bauer, director of the GMP Facility, at UC Davis.

We look forward to leveraging Orgenesis expertise to accelerate the development and commercialization of our lentiviral vector manufacturing system, which addresses a significant unmet need in the market for an efficient and scalable manufacturing process, said Professor Jan A Nolta, director of the Stem Cell Program and the Gene Therapy Center at UC Davis Health.

Orgenesis Inc, a leading cell and gene therapy enabling company provides centralized contract development and manufacturing organization (CDMO) services, as well as localized point-of-care development and processing centers through its subsidiary Orgenesis Maryland Inc.

Contact the author Uttara Choudhury at[emailprotected]

Follow her onTwitter:@UttaraProactive

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Orgenesis adds UC Davis to its point-of-care network for developing cell, gene therapy products - Proactive Investors USA & Canada

Barry reveals the truth about Fiona on Fair City – RTE.ie

This week on Fair City, Orla rejects contact with Olivia and lies to get out of minding her. Wayne is worried about Orla, and comforts her as she mourns after Sarah.

Later, Sash covers her stress when Laura is unable to help her out. Orla comes to Sash's aid and admits her true feelings to Sash. She is stunned by Sash's kind gesture.

Orla and Wayne later delight in the news that the stem cell treatment was successful. Orla later bonds with Olivia but she isn't as confident in the idea of a modern family as Wayne is.

Damien is excited by Bosco's talk of making a film noir and Mairead inadvertently belittles Damien's movie ambitions. Ray is left red in the face when his audition for the film goes badly.

Mairead later reads too much into a friendly text from Damien. Things take a turn when she accidentally injures Damien during filming and she is uneasy when the cast tease them. Mairead later reveals to Jane her true motives for being in the film, but Damien is slow to pick up on her hints about getting back together.

Jane rewrites some of the film and Mairead isn't pleased, accusing her of stealing the lead role. Jane rehearses her murder scene.

Meanwhile, Hayley assures Anto she won't be trading fathers. She is shocked when she finds out Ger doesn't know who her father is. Hayley has an idea to find out about her past, but her birth cert turns out to be a dead end.

Hayley looks for clues in old photographs and with Anto's help finds a lead. Hayley asks for Carol's help identifying Ger's friend from the photo. Carol hides her alarm when she recognises the men in the picture, later telling Nora they were Trigger's cronies.

Sarah's mother Rebecca arrives to confront Barry. When Bob asks Rebecca to hear Barry out about the foundation, Rebecca accuses Bob of taking his side. Barry feels more isolated as Paul underestimates his pain.

Later, Barry is nervous before his meeting with Rebecca, and it takes a turn for the worse. Rebecca tells Bob she wishes she never came back.

Fiona has her own agenda when she encourages Paul to be more sympathetic to Barry and she threatens Barry with blackmail. Barry tells Paul about laundering money through Vino's.

Paul kicks Barry out after he reveals the tax audit was a con. Paul lets nothing on with Fiona and tells Barry to keep schtum.

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Barry reveals the truth about Fiona on Fair City - RTE.ie

What will be the biggest scientific breakthrough of 2020? – Stuff.co.nz

Cheap, re-purposed cancer drugs, negative carbon-emissions technology, calculating how fast the universe is expandingand huge leaps forward in quantum computing.

Will one of these be the biggest scientific breakthrough of 2020?

We asked a handful of New Zealand's top scientists what "Eureka!" moments might be on the cards next year but even with their formidable combined brain power and expertise, some found it hard to answer and hinted it was difficult to sheet home specific advances to any one year.

And, as one scientist says, 2020's most ground-breaking discovery may come as a total surprise, made accidentally by a student in a lab late one night.

READ MORE:*Scientists are baffled: What's up with the universe?*Medicine already in use may help cancer treatments*Roger Hanson: How you figure out the age of the universe*Doing my part not only to be carbon neutral but carbon negative

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2020's most ground-breaking discovery may come as a total surprise, made accidentally by a student in a lab late one night.

Wellington's Gillies McIndoe Research Institute is carrying out cutting-edge research into ways of treating cancer without radiotherapy, chemotherapy or surgery.

The institute's founder and executive director, Dr Swee Tan, believes significant steps will be made in 2020 towards "re-purposing" existing drugs for cancer treatment.

Tan, a plastic surgeon and medical researcher who has received international recognition for dealing with life-threatening and disfiguring conditions, saiddrugs licensed and marketed for a particular treatment often had other benefits.

"They can be re-purposed for another condition, for the treatment of cancer. This is usually with drugs that have been around for some time they are off-patent, so they become generic, which means they cost next to nothing.

"Another advantage is their safety profile is well understood."

Wellington's Gillies McIndoe Research Institute's founder and executive director, Dr Swee Tan, believes significant steps will be made in 2020 towards "re-purposing" existing drugs for cancer treatment.

Globally, 18 million new cancer cases are diagnosed each year, resulting in nearly 10 million deaths.

"In New Zealand alone, new cancer cases are about 25,000 a year, and on top of that there are 11,000 non-melanoma skin cancers.

"The incidence of cancer is predicted to increase by 50 per cent by 2035, which is just around the corner. We are completely unprepared for that."

The increased incidence of cancer largely bowel, breast, prostate and lung cancers, and melanoma is mostly because people are living longer and due to lifestyle and diet, Tan says.

"Part of the problem now is the expense of treatment. In New Zealand, we spend $1 billion a year to treat cancer, and that is just the fiscal cost, that doesn't count emotional or personal.

"The cost is escalating because of the novel cancer drugsand, at some point, we are not going to be able to afford treatment for cancer.

"This creates disparity in access to treatment because some of these drugs are not funded and, if you have the means, you can buy it, but I don't think that is a good way to run a society. I think a society should allow people to access healthcare, regardless of personal circumstance."

Royal Society Te Aprangi/VIMEO

New Zealand is as vulnerable as all countries to the global growth in antimicrobial resistance that is making some diseases untreatable. Dr Siouxsie Wiles, a microbiologist from the University of Auckland, a Royal Society Te Aprangi Councillor and an expert adviser on the report on antimicrobial resistance produced by Royal Society Te Aprangi explains why it is such a big issue for us.

The institute has been undertaking a clinical trial based on drug re-purposing to control cancer stem cells, the proposed origin of cancer.

"It consists of a combination of low-cost, off-patent, oral medications to control cancer stem cells. We believe this would be more effective than using a single drug.

"For the cost of the drug itself, it costs about $4000 a year a patient, compared with the average cancer treatment, which is about $50,000 per patient.

"You have to prove it is effective first. The big issue with drug re-purposing is 'big pharma' is not interested because there is no money in it. The only way that we can realise the potential is for philanthropy and government to support initiatives like this."

Propranolol, a beta-blocker, had been added to the treatment for melanoma and angiosarcoma.

During 2020, GMRI would also be working on treatments for disfiguring keloid scars.

"They can cause quite significant issues with quality of life. They affect about 2 per cent of the general population, but in dark-skinned races, especially from the African continent, incidence can be up to 16 per cent.

"Treatment is really quite unsatisfactory, hence the reason why we are researching a solution. If you do surgery to remove it, almost every single one returns. Sometimes surgery is followed by radiotherapy to prevent recurrence. Topical chemotherapy is also used.

"What we have found is stem cells as the underlying problem. We are doing further work, which may allow us to develop a simpler, more effective, low-cost treatment taken by mouth, or by applying to the keloid lesion directly."

University of Auckland molecular biologist Dr Hilary Sheppard, a specialist in developmental and stem-cell biology, thinks there will be more emphasis next year on the gene-editing of adult cells.

University of Auckland microbiologist,associate professor Siouxsie Wiles, a specialist in infectious diseases and antimicrobial resistance, says there are "desperately" needed breakthroughs in her field next year and beyond, including:

- Rapid "bedside" diagnostic tests that are cheap and can tell the medical practitioner if the patient has a bacterial or viral infection "a bonus if it can tell, if bacterial, what antibiotics would kill the bacterium responsible".

- Effective vaccines for tuberculosis, gonorrhoea, Staphylococcus aureus, Group A and B Streptococci, giardia "I could go on and on".

- Drugs that can kill carbapenemase-producing Enterobacteriaceae "a very scary group of organisms that are becoming untreatable".

"The other breakthroughs we need aren't scientific, they are political," she says.

"[We need] a global agreement on tackling antimicrobial resistance, which would include incentives to bring the pharmaceutical industry back in to antimicrobial development.

"Failing that, nationalisation of pharmaceutical companies so that development of drugs isn't a for-profit initiative."

Phil Doyle/Stuff

University of Auckland microbiologist, associate professor Siouxsie Wiles, says a global agreement on tackling antimicrobial resistance is desperately needed.

University of Auckland molecular biologist Dr Hilary Sheppard, a specialist in developmental and stem-cell biology, thinks there will be more emphasis next year on the gene-editing of adult cells.

"We have seen some major breakthroughs this year, such as the versatile tool which allows for gene-editing with increased precision over existing tools so the technique is becoming more reliable and safer. Hopefully, next year, we will see these newer techniques being tested in clinically relevant cells.

"As part of that, I hope we will see a community-wide discussion about the ethics of gene-editing with a particular focus on adult cells. Personally, I do not think we should be editing germline cells or embryos at least not for the next five years, while the ethical issues are debated.

"Part of gene-editing is knowing what DNA sequence needs to be edited, so I think I hope this could be the year where personalised medicine and individual genotyping takes off.

"Our research focuses on patients with a fragile skin condition called epidermolysis bullosa (EB). We are pushing to get patients with EB genotyped so that we can perform gene-editing on their skin cells we can't do anything useful without this information.

"Currently, we are paying for the genotyping out of our research budgets. Of course, genotyping raises its own ethical and societal issues, so I hope we will see more discussion about this."

In 2020, results should start rolling in from clinical trials using edited T-cells against melanoma and edited bone-marrow cells to treat patients with sickle-cell anaemia, Sheppard says.

"These are very exciting times. I'm sure we'll see more clinical trials targeting previously untargeted disorders soon perhaps for conditions like Duchenne muscular dystrophy and cystic fibrosis."

RNZ

In this podcast, The Detail's Sharon Brettkelly talks to Auckland University physics professor Shaun Hendy about his no-flying mission for the whole of last year.

While Tan, Sheppard and Wiles are looking for discoveries on the tiniest of scales, other scientists are grappling with the biggest question in the universe.

Theoretical cosmologist and University of Auckland professor of physics Richard Easther is among those hoping for a resolution of what has become known as "Hubble tension" a growing disagreement in calculations of how fast the universe is expanding, which has repercussions on its likely age.

The Hubble Constant the number that tells us how fast the universe was expanding has always been hard to measure, he says.

"There was a period of time when a whole different bunch of approaches to measuring it had converged on a single value, which is fascinating. But just over the last couple of years, it seems like there are now two different sets of numbers you get and they've pulled apart a little bit.

"You know, there's this joke that science isn't so much about people saying 'Eureka!' but about someone looking at something and going, 'well, you know, that's funny'.

"This is increasingly resembling one of those moments.

"The numbers are clustering around two values one that would put it in the early to mid-70s [kilometres per second per megaparsec], and one that would put it in the mid- to late 60s, and the uncertainty in the measurements is such that they don't really overlap with each other.

"As the individual measurements get more accurate, the sharpness of the disagreement is growing."

University of Auckland physicist, professor Shaun Hendy, is expecting leaps ahead in clean energy in 2020.

Does it matter? Of course, Easther says.

"There are different physical assumptions that go into the different measurementsandso, if there is a real discrepancy, it would tell us there's something about the expansion of the universe that we don't understand.

"It's hinting at that. The implication seems to be that the story is one step more complicated than current models of the expanding universe might recognise.

"One thought is, that in one set of numbers, there's something that got missed and kind of got away. The other possibility is, there's something kind of physical, that isn't included in our current thinking of the expanding universe.

"The idea that there is something interesting going on is something that cosmologists over the course of the last year have grown substantially more willing to entertain."

Chris Skelton/STUFF

Nicola Gaston is an Associate Professor in the Department of Physics at the University of Auckland and Co-Director of the MacDiarmid Institute for Advanced Materials and Nanotechnology.

At the University of Otago, associate professor Mikkel Andersen, a physicist in the university's Dodd-Walls Centre for Photonic and Quantum Technologies, has been making astounding international discoveries and controlling the movement of individual atoms in a world-first laboratory experiment.

Such control opens up possibilities for a "second quantum technology revolution" and quantum supremacy, something Andersen says will creep closer in 2020.

The first quantum revolution was made possible by the discovery of quantum mechanics in the 1920s, leading to the development of transistors and lasers, the building blocks of all computers.

In the second, he says quantum computers of fewer than 100 atoms will ultimately be able to out-compete "the world's combined conventional computing power".

"Reaching quantum supremacy means that a quantum computer will be able to do calculations that cannot be done on the world's conventional computers. I do not know if it will happen next year, but it will happen eventually.

"In recent years, Google, IBM, Microsoft and a lot of others have invested enormously in development of quantum computers. Quantum supremacy is likely still some years away, but it is one of those things that would clear all the headlines if it happened in 2020."

University of Auckland physicist,professor Shaun Hendy, agrees.

"Google declared quantum supremacy last month they demonstrated that a quantum computer could beat a conventional computer, albeit at a very niche task.

"We'll see more of this next year, as quantum computers start to stretch their legs just don't expect to see one on your phone any time soon."

Ross Giblin

Victoria University of Wellington's professor James Renwick hopes to see breakthroughs in climate change science which reduce greenhouse gas emissions.

Hendy is also expecting leaps ahead in clean energy in 2020.

"We will continue to see the cost of solar and battery technologies fall, to the extent that they will start to disrupt other energy systems. We have seen this already in Australia, where it has become a defining political issue.

"It will play out differently in New Zealand, because our grid is already more than 80 per cent renewable, while many of our industrial energy systems are not. Expect to see some of our big industrial corporates Fonterra, NZ Steel etc moving to greener industrial processes."

Victoria University of Wellington's Professor James Renwick, head of the school of geography, environment and earth sciences, hopes to see breakthroughs in climate change science which reduce greenhouse gas emissions.

He points to work being done at the Cawthron Institute in Nelson into the benefits of using the seaweed Asparagopsis armata as cattle feed. Chemicals in the red seaweed reduce microbes in the stomachs of cattle that make them burp when eating grass.

Renwick is also excited about the use of artificial intelligence (AI) and machine learning to help with severe weather prediction.

"Weather forecasters are totally inundated with information these days, volumes of radar data and satellite data coming through every 10 minutes. So, AI can help in making sense of all that, and what is the most important in determining where, for example, a severe storm will happen."

University of Otago associate professor Mikkel Andersen believes a "second quantum technology revolution" and quantum supremacy will creep closer in 2020.

Auckland University of Technology senior lecturer Dr Mahsa Mohaghegh also foresees huge steps forward in AI and its applications next year and beyond.

"In the medical sector, AI is being used to speed up symptom recognition and diagnosis. Early warning signs can be easily detected, allowing fast reaction.

"Environment and climate monitoring using AI can assist with weather-cycle predictions, frost warnings, and harvest alerts. Automated irrigation is possible using moisture and temperature sensors."

There are also uses in New Zealand's burgeoning space industry, in traffic management and in the "smart home" of the future, she says.

"New Zealand is a leader and frontrunner in the development of AI and related fields. The next 10 years of technology development are set to be exciting."

David White

Auckland University of Technology senior lecturer Dr Mahsa Mohaghegh foresees huge steps forward in AI and its applications next year.

University of Auckland physicist, associate professor Nicola Gaston, co-director of the Victoria University of Wellington-hosted MacDiarmid Institute for Advanced Materials and Nanotechnology, told Stuff scientific discovery did not happen "one year at a time".

"Discoveries that impact on our lives next year will be built on work that has been going on for decades. The biggest discoveries of next year will be the ones that impact on our lives in a decade or two.

"But there is no competition between this fundamental scientific work of discovery and the development of technologies. The two go in tandemand, perhaps in 2020, we can try to appreciate that."

That may be finding a way of moving to negative emissions technologies, or changing the chemistry of materials so they are recyclable and avoid environmental pollution.

"The most important breakthrough of 2020 will be one that none of us sees.

"It'll be a dedicated student or post-doc in a lab somewhere, or up late at night on a computer, who solves the last remaining piece of one of the puzzles that underpin so much of what we hope technology can do for us in the future."

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What will be the biggest scientific breakthrough of 2020? - Stuff.co.nz