Category Archives: Stem Cell Therapy

In building out its cell therapy manufacturing network, Novartis has established four in-house facilities across the US and Europe.

However, the Swiss drugmaker has also used service providers, for example, by partnering with Germanys Fraunhofer IZI to access chimeric antigen receptor (CAR)-T therapies for use in its first European clinical trials and the early years of commercial sales in the region.

Novartis has taken the outsourcing route in Japan, too, completing the technology transfer for CAR-T therapy Kymriah (tisagenlecleucel) to Kobe-based the Foundation for Biomedical Research and Innovation (FBRI) early this year. FBRI subsequently supported clinical supply in Japan.

However, while the Japanese regulator approved Kymriah in March, it is taking longer to get the cell therapy contract manufacturer ready for commercial supply. At a recent R&D day, Novartis said the contract manufacturer will be commercially ready in the second half of next year.

Work to get FBRI commercially ready is part of a broader effort by Novartis to establish the capacity to support its global cell therapy ambitions. Currently, Novartis lacks that capacity.

Novartis CEO Vas Narasimhan said, We are supply constrained on Kymriah. Its not a demand constraint. Our goal is to work hard to relieve that ... constraint. We continue to be optimistic that Kymriah will reach blockbuster potential within the next five years.

The comment follows a period in which Kymriah, Novartis approved CAR-T, has fallen short of sales expectations. Sales over the first nine months of 2019 totalled around $180m (163m).

Novartis struggles to meet demand for Kymriah reflect the novel challenge of making autologous cell therapies, which are produced by taking a patients own cells, processing them and administering them back into the same patient.

Cell therapies are one of a clutch of emerging modalities and therapeutic concepts that are central to Novartis pipeline plans. Novartis recognises that it needs to match its infrastructure to the changing mix of modalities in its pipeline, as Jay Bradner, president of the Novartis Institutes for BioMedical Research, explained.

Bradner said, Its not enough just to do this in drug discovery, as we have learned. This needs to be a coordinated strategy across the entire enterprise.

That coordinated strategy has led Novartis to add in-house and contract capacity to support plans to introduce more CAR-Ts and CRISPR-edited stem cells in the coming years. Bradner said Novartis is building up scale to accelerate all of these programs[going] forward.

The exact demands Novartis places on that capacity are likely to change in the coming years, with Bradner predicting the future of the field will feature a new way of manufacturing CAR-T cells. One big expected change is the arrival of allogeneic CAR-Ts made from donor cells.

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Novartis sets 2020 target for commercial readiness of cell therapy CMO - OutSourcing-Pharma.com

Assessment of the Animal Stem Cell Therapy Market

The latest report on the Animal Stem Cell Therapy Market published by Persistence Market Research (PMR) is a valuable tool for established and upcoming market players to solidify their presence in Animal Stem Cell Therapy Market landscape. Further, by leveraging the real-time and result-driven insights included in the report, readers can formulate effective business strategies to gain an advantage in the competitive nature of the market.

The presented study suggests that the Animal Stem Cell Therapy Market is expected to attain a value of ~US$ XX by the end of assessment period and grow at a CAGR of ~XX% during the forecast period, 2017 2025. The underlying trends, growth opportunities, market drivers, and challenges faced by companies in the Animal Stem Cell Therapy Market are analyzed in detail.

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Vital Information Included in the Report:

The competitive landscape chapter of the report provides valuable insights related to the business prospects of some of the leading companies in the Animal Stem Cell Therapy Market. The product portfolio, pricing structure, revenue growth, and footprint analysis of each market players are included in the report.

Important Queries Related to the Animal Stem Cell Therapy Market Addressed in the Report

The dynamics of the Animal Stem Cell Therapy Market across major geographical regions are included in the report and represented in the study. In addition, to provide a frictionless reading experience the data is depicted using informative graphs and other graphical illustrations.

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Key Participants

The key participants in the animal stem cell therapy market are Magellan Stem Cells, ANIMAL CELL THERAPIES, Abbott Animal Hospital, VETSTEM BIOPHARMA, Veterinary Hospital and Clinic Frisco, CO, etc. The companies are entering into the collaboration and partnership to keep up the pace of the innovations.

The report covers exhaustive analysis on:

Regional analysis for Market includes

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Animal Stem Cell Therapy Market Poised to Expand at a Robust Pace Over 2017 2025 - Downey Magazine

Last year, Arie Belldegrun and David Chang, fresh from selling chimeric antigen receptor (CAR)-T pioneer Kite Pharma to Gilead Sciences, launched Allogene with $300m (271m) and a deal with Pfizer to access off-the-shelf cell therapies. The focus then, as now, was on taking CAR-T therapies mainstream by eliminating factors that could restrict them to niche status, such as the complex, costly production process.

Allogenes founding allogeneic technology moves it some way toward that goal, by enabling therapies to be made from donor cells rather than having to engineer cells taken from the patient themselves.

However, even if the allogeneic approach works as Allogene hopes, it will still require a steady supply of donor cells. When Allogene launched, it set its sights on making up to 100 doses from one donor sample.

The finite nature of donated T cells has spurred interest in induced pluripotent stem cells (iPSC), which could serve as a renewable source of materials for off-the-shelf CAR-T therapies. Notchs work to realize that potential caught the attention of Allogenes CEO, David Chang.

Chang said, We believe [it] to be a scalable and potentially more [good manufacturing practice] amenable manufacturing process. It might be amenable for large-scale manufacturing.

The early stage nature of the iPSC technology Notch is yet to enter the clinic means it is unclear whether the approach can live up to those expectations. An inability to show CAR-T doses are free from undifferentiated iPSCs and failure to generate functioning T cells from iPSCs are two potential stumbling blocks.

Allogene has seen enough potential to bet on Notch, though. The deal will see Allogene pay Notch $10m upfront, take a 25% stake in its new partner and commit to a package of milestones.

Notch will take iPSC AlloCAR T cells through preclinical development before Allogene steps in to test them in humans. Allogene will have global rights to any products resulting from the collaboration.

Other companies are also working to develop oncology cell therapies based on iPSCs. Fate Therapeutics is trialling an engineered natural killer cell therapy created from a clonal master iPSC line, while Bayer-backed startup Century Therapeutics recently raised $250m to take iPSC-derived treatments for blood cancers and solid tumors into the clinic. Takeda is also active in the space.

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Allogene allies with Notch to make CAR-T production more scalable - BioPharma-Reporter.com

A technique called zinc finger nuclease (ZFN) gene editing technology can be used to modify immature red blood cells called precursor cells to boost the production of fetal hemoglobin and help ensure red blood cells maintain a normal shape in people with sickle cell disease (SCD), a study shows.

Sanofi has launched a Phase 1/2 trial (NCT03653247) evaluating the safety, tolerability and efficacy of a BIVV003, a gene editing therapy using the ZFN technology (by Sangamo Therapeutics) in adults with severe SCD. This trial, taking place at four U.S. sites, is currentlyrecruiting eligible patients.

ZFN technology findings will be presented by Samuel Lessard, PhD, a researcher at Sanofi, in the poster, Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients, (abstract No. 974) at the 61st Annual Meeting of the American Society of Hematology (ASH), December 710 in Orlando.

Sickle cell is caused by mutations in the HBB gene, which provides instructions for making part of hemoglobin, a protein responsible for transporting oxygen in the blood.

These mutations change the structure of the protein, resulting in the production of abnormal hemoglobin fibers. These fibers tend to stiffen red blood cells, changing their shape from normal disc-like cells to those with a sickle-like shape.

BIVV003 is an investigational gene edited cell therapy being developed under an agreement between Bioverativ, a Sanofi company, and Sangamo.

It uses Sangamos proprietary ZFN gene editing technology to modify a short sequence of the BCL11Agene in red blood precursor cells acquired from the patients own hematopoietic stem cells (stem cells that give rise to other blood cells) to raise production of fetal hemoglobin, the main form of hemoglobin found in fetuses.

Fetal hemoglobin production is normally switched off in adults and largely disappears at ages 6 months to 1 year, but artificial ways of introducing fetal hemoglobin show increasing promise in treating SCD by preventing red blood cells from taking on a damaging sickle shape.

Sangamoannounced the findings from a proof-of-concept ex-vivo(lab) study aiming to validate the ZFN gene editing technology using immature red blood cell isolated from four healthy donors and one sickle cell patient.

Study findings demonstrated that more than 90% of edited donor cells incorporated genetic modifications in both copies of the BCL11A gene, leading to an increase of 27% to 38% in fetal hemoglobin levels compared to baseline (studys start).

Edited cells from the SCD patient also tended to incorporate these modifications in both gene copies. As a result, patient edited cells produced up to 28% more fetal hemoglobin compared to unedited cells.

Patient red blood cells obtained from already edited precursor cells were also less likely to change to a sickle-like shape, supporting BIVV003 as a potential cell therapy for SCD, the study reports. Further experiments in red blood precursor cells from additional SCD patients are underway.

Sangamo is also enrolling patients with transfusion-dependent beta-thalassemia (TDT) in thePhase 1/2 THALES trial (NCT03432364) to evaluate the safety, tolerability, and efficacy of ST-400, another experimental gene-edited cell therapy that uses the same gene-editing approach as BIVV003.

The companyannounced it will present data from the first three THALESpatients at the ASH meeting.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Gene Editing Tool in Ongoing Sickle Cell Trial of BIVV003 Supported by Early Study - Sickle Cell Anemia News

REHOVOT, Israel and NEW YORK, Nov. 14, 2019 (GLOBE NEWSWIRE) -- Todos Medical Ltd. (TOMDF), a clinical-stage in-vitro diagnostics company focused on the development of blood tests for the early detection of cancer and neurodegenerative disorders, and Amarantus Bioscience Holdings, Inc. a US-based JLABS-alumnus biotechnology holding company developing proprietary orphan neurologic, regenerative medicine and ophthalmic therapies and diagnostics through its subsidiaries, today announced that their joint venture company, Breakthrough Diagnostics, Inc. has completed enrollment of its ongoing clinical trial evaluating the relationship of Alzheimers blood diagnostic Lymphocyte Proliferation Test (LymPro Test) with amyloid PET neuroimaging at Leipzig University in Germany (the LymPro PET 2). Topline results are expected before the end of the first quarter of 2020.

Breakthrough completed a 20-subject clinical study (LymPro PET 1) in 2018 evaluating the correlation between LymPro scores and the diagnosis of Alzheimers disease, as confirmed with amyloid PET neuroimaging and other Alzheimers disease biomarkers. LymPro measures cell cycle dysregulation in peripheral lymphocytes. The top-line data, announced in July 2019, revealed a strong and statistically significant correlation between LymPro scores and amyloid PET neuroimaging cSUVR scores (r = -0.849; p = 0.00000216). Breakthroughs academic collaborators at the Leipzig University then expanded enrollment of that study to include an additional cohort of 20 subjects (LymPro PET 2) to confirm the strong relationship seen from LymPro PET 1. The data from both LymPro 1 and LymPro 2 will be published together in a peer-reviewed journal in 2020.

LymPro is a unique immune system-based Alzheimers blood test, said Dr. Herman Weiss, President & CEO of Todos. LymPro could prove to be a major breakthrough for Alzheimers disease diagnosis by measuring cell cycle dysregulation and amyloid, together, conveniently as part of a blood workup in routine clinical practice. The therapeutic field in Alzheimers has begun to see some renewed hope based upon recent Aducanumab data announced by Biogen that is directly related to the amyloid hypothesis, as well as conditional approval by the National Medical Products Administration in China for the first new Alzheimers drug in over 20 years, called Oligomannate from Shanghai Green Valley Pharmaceuticals, that is based on gut-brain biology of the microbiome and its effects on the immune system. We believe this renewed optimism and broadening of pathophysiological hypotheses relevant to Alzheimers disease being evaluated in the clinic significantly increases the scope for LymPro pharma services collaborations and begins to refine LymPros clinical utility profile for primary care physicians as strategies to correct cell cycle dysregulation emerge.

About Alzheimer's DiseaseAccording to the Alzheimer's Association, it is estimated that over 5.4 million people in the United States suffer from Alzheimer's disease. Over 500,000 patients are diagnosed annually, with nearly one-in-eight older Americans affected by the disease. Alzheimer's disease is the third leading cause of death in the United States. The cost of unpaid care in the United States is estimated at over $210 billion annually.Total payments for care are estimated at over $200 billion annually, including $140 billion in cost to Medicare and Medicaid. Alzheimer's expenditures in the United States are expected to exceed $1.2 trillion by 2050. There is no cure or effective treatment for Alzheimer's disease. Worldwide, about 35.6 million individuals have the disease and, according to the World Health Organization, the number will double every 20 years to 115.4 million people with Alzheimer's by 2050.

About Dr. Arendt's Research at Leipzig UniversityDr. Thomas Arendt is Professor of Neuroscience at Leipzig University where he runs the Paul Flechsig Institute of Brain Research. He has a 30-year record in R&D of therapeutic and diagnostic strategies of neurodegenerative disorders and made several seminal contributions to therapeutic concepts of Alzheimer's disease, including stem cell therapy and modulating tumor suppressor genes. In the early 1980's, he was involved in identifying the degeneration of the cholinergic system in Alzheimer's disease laying the basis for today's only available treatment. He is one of the pioneers of the "cell-cycle theory" of Alzheimer's disease, which he developed towards a diagnostic and therapeutic concept.

Story continues

About Breakthrough Diagnostics, Inc.Breakthrough Diagnostics, Inc. is a joint venture owned by Amarantus Bioscience Holdings, Inc. (AMBS) (80.01%) and Todos Medical Ltd. (19.99%). Breakthrough has been assigned the intellectual property and other rights to the LymPro Test, a diagnostic blood test for Alzheimers disease, as well as rights to other neurological diagnostics testing intellectual property. Todos Medical has provided Amarantus with notice of Todos decision to exercise its exclusive option to acquire the 80.01% of Breakthrough Diagnostics that it currently does not own.

The Lymphocyte Proliferation Test (LymPro Test) determines the ability of peripheral blood lymphocytes (PBLs) and monocytes to withstand an exogenous mitogenic stimulation that induces them to enter the cell cycle. It is believed that certain diseases, most notably Alzheimer's disease, are the result of compromised cellular machinery that leads to aberrant cell cycle re-entry by neurons, which then leads to apoptosis. LymPro is unique in the use of peripheral blood lymphocytes as surrogates for neuronal cell function, suggesting a common relationship between PBLs and neurons in the brain.

About Todos Medical Ltd.Todos Medical Ltd. is an in-vitro diagnostic company engaged in the development of blood tests for the early detection of a variety of cancers, and also has initiated the development of blood tests for neurodegenerative disorders such as Alzheimer's disease through Breakthrough Diagnostics, Inc., its joint venture with Amarantus Bioscience Holdings, Inc. Todos has developed two cancer screening tests based on TBIA (Todos Biochemical Infrared Analyses), a method for cancer screening using peripheral blood analysis. The TBIA screening method is based on the cancers influence on the immune system, which triggers biochemical changes in peripheral blood mononuclear cells and plasma. This proprietary and patented method incorporates biochemistry, physics and signal processing. The companys two cancer screening tests, TM-B1 and TM-B2, have received the CE mark. Breakthrough Diagnostics is developing the LymPro Test, a blood test for diagnosing Alzheimers disease.

For more information, the content of which is not part of this press release, please visithttp://www.todosmedical.com

About Amarantus Bioscience Holdings, Inc.Amarantus Bioscience Holdings (AMBS) is a JLABS alumnus biotechnology company developing treatments and diagnostics for diseases in the areas of neurology, regenerative medicine and orphan diseases through its subsidiaries. The Companys 80.01%-owned subsidiaryBreakthrough Diagnostics, Inc.,currently a joint venture with Todos Medical, Ltd., has licensed intellectual property rights to the Alzheimers blood diagnostic LymPro Test from Leipzig University that was originally developed by Dr. Thomas Arendt, as well as certain rights to multiple sclerosis diagnostic MSPrecise and Parkinsons diagnostic NuroPro. Amarantus entered into a joint venture agreement withTodos Medical, Ltd. to advance diagnostic screening assets and Todos has exercised its exclusive option to acquire Amarantus remaining ownership in Breakthrough in exchange for approximately 50% ownership of Todos. The transaction is expected close before the end of the first quarter of 2020. Amarantus also owns approximately 30% of the common shares of Avant Diagnostics, Inc., a healthcare data-generating technology company that specializes in biomarker assay services that target multiple areas of oncology. Avant provides precision oncology data through its TheraLink assays to assist the biopharmaceutical industry and clinical oncologists in identifying likely responders, initially for breast cancer, to over 70 FDA-approved drug treatments.

AMBS 50%-owned subsidiaryElto Pharma, Inc. has development rights to eltoprazine, a Phase 2b-ready small molecule indicated for Parkinson's disease levodopa-induced dyskinesia, Alzheimers aggression and adult attention deficit hyperactivity disorder, commonly known as ADHD. AMBS acquiredCutanogen Corporationfrom Lonza Group in 2015. Cutanogen is preparing for pivotal studies with Engineered Skin Substitute (ESS) for the treatment of pediatric life-threatening severe burns. ESS is a regenerative medicine-based, autologous full-thickness skin graft technology originally developed by the Shriners Hospital that can be used to treat severe burns, as well as several other catastrophic and cosmetic dermatological indications. AMBS wholly-owned subsidiary,MANF Therapeutics Inc.owns key intellectual property rights and licenses from a number of prominent universities related to the development of the therapeutic protein known as mesencephalic astrocyte-derived neurotrophic factor (MANF). MANF Therapeutics is developing MANF-based products as treatments for ophthalmological disorders such as Wolfram Syndrome, Retinitis Pigmentosa and Glaucoma, as well as neurodegenerative diseases such as Parkinsons disease. MANF was discovered by the Companys Chief Scientific Officer John Commissiong, PhD. Dr. Commissiong discovered MANF from AMBS proprietary discovery engine PhenoGuard, and believes several other neurotrophic factors remain to be discovered. Amarantus has entered into a binding letter of intent to license the therapeutic assets from Elto Pharma, Cutanogen and MANF Therapeutics to Emerald Organic Products.

Forward-looking StatementsCertain statements contained in this press release may constitute forward-looking statements. For example, forward-looking statements are used when discussing our expected clinical development programs and clinical trials. These forward-looking statements are based only on current expectations of management, and are subject to significant risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements, including the risks and uncertainties related to the progress, timing, cost, and results of clinical trials and product development programs; difficulties or delays in obtaining regulatory approval or patent protection for product candidates; competition from other biotechnology companies; and our ability to obtain additional funding required to conduct our research, development and commercialization activities. In addition, the following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; delays or obstacles in launching our clinical trials; changes in legislation; inability to timely develop and introduce new technologies, products and applications; lack of validation of our technology as we progress further and lack of acceptance of our methods by the scientific community; inability to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties that may develop with our process; greater cost of final product than anticipated; loss of market share and pressure on pricing resulting from competition; and laboratory results that do not translate to equally good results in real settings, all of which could cause the actual results or performance to differ materially from those contemplated in such forward-looking statements. Except as otherwise required by law, Todos Medical does not undertake any obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. For a more detailed description of the risks and uncertainties affecting Todos Medical, please refer to its reports filed from time to time with the U.S. Securities and Exchange Commission.

Todos Investor and Corporate Contact:Kim Sutton GolodetzLHA Investor RelationsSenior Vice President (212) 838-3777kgolodetz@lhai.com

Todos Corporate ContactDaniel HirschTodos MedicalInvestor RelationsEmail:Dan.h@todosmedical.comPhone: (347) 699-0029

Amarantus Investor and Media Contact:Gerald CommissiongPresident & CEOOffice: 650-862-5391Email: gerald@amarantus.com

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Todos and Amarantus JV Announces Full Enrollment for Clinical Trial of LymPro Alzheimers Blood Test Relationship with Amyloid PET - Yahoo Finance

CALGARY, Alberta, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Hemostemix Inc. (Hemostemix or the Company) (TSX VENTURE: HEM; OTCQB: HMTXF), a biotechnology company developing and commercializing blood-derived stem cell therapies for unmet medical conditions, is pleased to provide a summary of the presentation entitled Autologous Stem Cell Treatment for CLI Patients with No Revascularization Options: An Update of the Hemostemix ACP-01 Trial With 4.5 Year Followup. Lead investigator Dr. York Hsiang, Professor of Vascular Surgery, University of British Columbia gave this update at the 41st Annual Canadian Society for Vascular Surgery Meeting on September 14, 2019.

Dr. Hsiang reported on the blinded results from the long-term follow-up of the first cohort of patients enrolled at two trial sites, Vancouver Coastal Health Research Institute (VCHRI) located in Vancouver, BC, led by principal investigator, Dr. York N. Hsiang, MB, ChB, MHSc, FRCSC and University Health Network, Peter Monk Cardiac Centre located in Toronto, Ontario, led by principal investigator Dr. Thomas Lindsay, MDCM, MSc, FRCSC, FACS.

Following is a summary of the results and conclusion:

In addition, the Companys Data Safety Monitoring Board (DSMB) recently met to review patient safety data in the ongoing Phase II clinical trial for CLI. The DSMB did not find safety concerns with ACP-01 and recommended continuing to enroll patients in the trial. The clinical trial is ongoing at 13 clinical sites in the US and Canada, with several additional sites in the process of being initiated. To date, 46 of the planned 95 patients have been enrolled and treated in the study.

We are very pleased with these blinded long term follow up results, and the recommendation of the DSMB, which are consistent with the findings reported in our two previous published studies of ACP-01 in CLI patients, said Dr. Alan Jacobs, President and Chief Medical Officer of Hemostemix. Patients with critical limb ischemia face a high rate of amputation when revascularization treatment options are exhausted, so seeing this level of improvement, and outcomes maintained for up to 4.5 years after treatment, is extremely encouraging.

ABOUT HEMOSTEMIX INC.

Hemostemix is a publicly traded clinical-stage biotechnology company that develops and commercializes innovative blood-derived cell therapies for medical conditions not adequately addressed by current treatments. It is one of the first clinical-stage biotech companies to test a stem-cell therapy in an international, multicenter, Phase II clinical trial for patients with critical limb ischemia (CLI), a severe form of peripheral artery disease (PAD) caused by reduced blood flow to the legs. The Phase II trial targets a participants diseased tissue with proprietary cells grown from his or her blood that can support the formation of new blood vessels. The Companys intellectual property portfolio includes over 50 patents issued or pending throughout the world. Hemostemix has a manufacturing contract with Aspire Health Science, LLC (Aspire), for the production of ACP-01 and for research and development purposes at Aspires Orlando, Florida, facility. Building towards commercialization, Hemostemix has also licensed the use, sale and import of ACP-01 for certain indications to Aspire in certain jurisdictions. The Company is continuing research and development of its lead product, ACP-01 with other applications, including cardiovascular, neurological and vascular indications.

For more information, please visit http://www.hemostemix.comor email office@hemostemix.com.

Contact:

Kyle Makofka, CEOSuite 2150, 300 5th Avenue S.W.Calgary, Alberta T2P 3C4Phone: (403) 506-3373E-Mail: kmakofka@hemostemix.com

Neither the TSX Venture Exchange nor its Regulation Service Provider (as that term is defined under the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Forward-Looking Statements

This release may contain forward-looking statements. Forward-looking statements are statements that are not historical facts and are generally, but not always, identified by the words expects, plans, anticipates, believes, intends, estimates, projects, potential, and similar expressions, or that events or conditions will, would, may, could, or should occur. Although Hemostemix believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance and actual results may differ materially from those in forward-looking statements. Forward-looking statements are based on the beliefs, estimates, and opinions of Hemostemix management on the date such statements were made. By their nature forward-looking statements are subject to known and unknown risks, uncertainties, and other factors which may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include, but are not limited to, the Companys stage of development, future clinical trial results, long-term capital requirements and future ability to fund operations, future developments in the Companys markets and the markets in which it expects to compete, risks associated with its strategic alliances and the impact of entering new markets on the Companys operations. Each factor should be considered carefully and readers are cautioned not to place undue reliance on such forward-looking statements. Hemostemix expressly disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events, or otherwise.

Originally posted here:
Hemostemix Announces Positive Results and Conclusions Reported in Phase II CLI Trial Abstract - GlobeNewswire

NEW YORK, Oct. 21, 2019 /PRNewswire/ --

Regenerative Medicine Market Size, Share & Trends Analysis By Product (Primary Cell-based, Stem & Progenitor Cell-based), By Therapeutic Category (Dermatology, Oncology) And Segment Forecasts, 2019 - 2025

Read the full report: https://www.reportlinker.com/p05807250/?utm_source=PRN

The global regenerative medicine market size is expected to reach USD 5.60 billion by 2025, expanding at a CAGR of 11.6% over the forecast period. Regenerative medicines are expected to have a significant impact in healthcare to treat specific indications and chronic conditions. Therefore, high prevalence of cancer, neurodegenerative, orthopedic, and other aging-associated disorders coupled with increasing global geriatric population is driving the market growth. Moreover, rising prevalence of inheritable genetic diseases is anticipated to fuel the demand in the field of biotechnology field.

Market players are engaged in implementing novel protocols for the release of novel therapeutics. For instance, in July 2018, Convelo Therapeutics launched regenerative medicines for the treatment of various neurological diseases, such as multiple sclerosis.Agreements models initiated by the companies coupled with commercialization in emerging countries fuels the growth. For instance, in March 2018, Hitachi Chemical signed an agreement with the Daiichi Sankyo and SanBio Group to conduct clinical manufacturing of regenerative medicines developed by respective companies for Japanese and U.S. markets.

Regenerative medicine is anticipated to witness great attention in healthcare sector due to its wide range of applications and significant advancements tissue engineering, stem cells, gene therapy, drug discovery, and nanotechnology. For example, 3D printing is preferred over scaffold with stem cells to restore structure and functional characteristics of biological specimens.

Dermatology is estimated to hold the largest market share in terms of revenue in 2018, owing to the availability of various products and their application in simple and chronic wound healing. Oncology therapeutic category on the other hand, is projected to expand at the fastest CAGR during the forecast period owing to the presence of strong pipeline of regenerative medicines for cancer treatment.

North America held the largest regenerative medicine market share in terms of revenue in 2018 and is projected to continue its dominance in near future. A significant number of universities and research organizations investigating various stem cell-based approaches for regenerative apposition in U.S. is anticipated to propel the growth.

Further key findings from the report suggest: Therapeutics emerged dominant among product segments in 2018 due to high usage of primary cell-based therapies along with advances in stem cell and progenitor cell therapies Implementation of primary cell-based therapies in dermatological, musculoskeletal, and dental application results in highest share of this segment Stem cell and progenitor cell-based therapies are anticipated to witness rapid growth due to high investments in stem cell research and increasing number of stem cell banks With rise in R&D and clinical trials, key players are offering consulting services leading to lucrative growth of the services segment Asia Pacific is projected to witness the fastest CAGR during the forecast period due to rapid adoption of cell-based approaches in healthcare and emergence of key players Key players operating in the regenerative medicine market including AstraZeneca; F Hoffmann-La Roche Ltd.; Pfizer Inc.; Merck & Co., Inc.; Integra LifeSciences Corporation; and Eli Lilly and Company

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The global regenerative medicine market size is expected to reach USD 5.60 billion by 2025, expanding at a CAGR of 11.6% over the forecast period -...

Vancouver, Canada, October 21, 2019 ISCT, the International Society for Cell and Gene Therapy, the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell and gene therapy sector, today announces it has formed a global consortium of a wide range of leading professional and education societies to combat the rise in the number of unproven commercial cell banking services. Full details of the statement can be foundhere.

The consortium partners include the International Society for Stem Cell Research (ISSCR), Society for Immunotherapy of Cancer (SITC), American Society for Transplantation and Cellular Therapy (ASTCT),American Society of Gene & Cell Therapy (ASGCT), European Society for Blood and Marrow Transplantation (EBMT), Foundation for the Accreditation of Cellular Therapy (FACT), Joint Accreditation Committee ISCT-EBMT (JACIE) and the Forum for Innovative Regenerative Medicine (FIRM).

The consortium has been formed following ISCT issuingpatient advice and concern on unproven T-cell preservation services on August 7, 2019. These services include the banking of T-cells, dental cells and cells for the derivation of induced pluripotent stem cells for potential therapeutic uses.

The joint statement from ISCT and the consortium partners includes an agreement on a number of key points. Commercial cell banking services are not supported by current scientific evidence, as opposed to the range of cell therapies such as CAR-T therapies, that follow established approval processes. Additionally, cell banking services cannot claim to know that the cells they preserve today could ever be appropriate for clinical use, could be used by manufacturers, or meet the requirements of many national and international regulatory agencies. As a result, there is no clear pathway to legitimate clinical use. All parties agree offering these services commercially to patients is thus premature, misleading, and drives false hope.

In addition, the ISCT joint statement makes clear that patients, being misled by these services, are thus prevented from giving a full and valid informed consent. Cell banking companies mislead patients in a number of ways, including using tokens of scientific legitimacy that suggest a stronger scientific basis than currently exists. These tokens include endorsements from individuals or scientific advisory boards that might not fully endorse the specific products, links to scientific articles, and references to ongoing clinical trials.

ISCTs raison detre is to lead the industry in supporting scientifically validated cell and gene therapies. As a result, ISCT will continue to welcome all innovations, including cell banking approaches, that increase the number of patients who can benefit from these therapies, said Bruce Levine,President-Elect, ISCT and one of the inventors of CAR-T therapies.However, ISCT also leads industry action on unproven cell therapies and services in the cell and gene sector. This is why ISCT has forged a consortium throughout the industry against the marketing of speculative cell banking services that do not have appropriate pre-clinical, and clinical evidence and a plausible pathway to the clinical use of banked cells. We collectively believe these banks have the potential to be detrimental to the future development of cell and gene therapies.

About ISCT

Established in 1992, ISCT, the International Society for Cell and Gene Therapy is a global society of clinicians, regulators, researchers, technologists and industry partners with a shared vision to translate cellular therapy into safe and effective therapies to improve patients lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory and Commercialization. Through strong relationships with global regulatory agencies, academic institutions and industry partners, ISCT drives the advancement of research into standard of care.

Comprised of over 1,500 cell therapy experts across five geographic regions and representation from over 50 countries, ISCT members are part of a global community of peers, thought leaders and organizations invested in cell therapy translation. For more information about the society, key initiatives and upcoming meetings, please visit:

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ISCT forms cell and gene therapy sector-wide coalition to combat the rise of unproven commercial cell banking services - PharmiWeb.com

Blood stem cells protect themselves against viruses with structures known as 'interferon-induced transmembrane proteins,' seen here in green. These normally useful proteins are problematic for gene therapy treatments, as they work to keep therapeutic lentiviral vectors from infiltrating cells. Scripps Research scientists found a natural compound that lets down this shield, boosting the success rate of gene delivery.Credit: Image courtesy of the Torbett lab at Scripps ResearchLA JOLLA, CA Gene therapy has broadened the treatment possibilities for those with immune system deficiencies and blood-based conditions, such as sickle cell anemia and leukemia. These diseases, which once would require a bone marrow transplant, can now be successfully treated by modifying patients' own blood stem cells to correct the underlying genetic problem.

But today's standard process for administering gene therapy is expensive and time-consuminga result of the many steps required to deliver the healthy genes into the patients' blood stem cells to correct a genetic problem.

In a discovery that appears in the journalBlood, scientists atScripps Researchbelieve they have found a way to sidestep some of the current difficulties, resulting in a more efficient gene delivery method that would save money and improve treatment outcomes.

"If you can repair blood stem cells with a single gene delivery treatment, rather than multiple treatments over the course of many days, you can reduce the clinical time and expense, which removes some of the limitations of this type of approach," says Bruce Torbett, PhD, associate professor in the Department of Immunology and Microbiology, who led the research.

The new finding centers on caraphenol A, a small molecule closely related to resveratrol, which is a natural compound produced by grapes and other plants and found in red wine. Resveratrol is widely known as an antioxidant and anti-inflammatory agent. Similar to resveratrol, caraphenol A is anti-inflammatory, but in this study, it served a different role.

Torbett and his team became interested in the unique chemical properties of resveratrol and similar types of molecules and wondered if they could enable viral vectors, used in gene therapy to deliver genes, to enter blood stem cells more easily. This would be momentous because stem cellsand in particular, self-renewing hemopoietic stem cellshave many barriers of protection against viruses, making them challenging for gene therapy to infiltrate.

Related Article:Solution to 50-Year-Old Mystery Could Lead to Gene Therapy for Common Blood Disorders

"This is why gene therapy of hemopoietic stem cells has been hit-or-miss," Torbett says. "We saw a way to potentially make the treatment process significantly more efficient."

The gene therapy treatment process currently requires isolating a very small population of hemopoietic stem cells from the blood of patients; these young cells can self-renew and give rise to all other types of blood cells. Therapeutic genes are then delivered to these cells via specially engineered viruses, called "lentiviral vectors," which leverage viruses' natural knack for inserting new genetic information into living cells.

However, hemopoietic stem cells are highly resilient to viral attacks. They protect themselves with structures known as interferon-induced transmembrane (IFITM) proteins, which intercept lentiviral vectors. Because of this, it can take many attemptsand a large quantity of expensive gene therapy vectorsto successfully delivery genes into hemopoietic stem cells, Torbett says.

Torbett and his team found that by adding the resveratrol-like compound, caraphenol A, to human hemopoietic stem cells, along with the lentiviral vector mix, the cells let down their natural defenses and allowed vectors to enter more easily. Once the treated stem cells were placed into mice, they divided and produced blood cells containing the new genetic information.

Another key benefit of the approach is time: If gene delivery treatment of blood stem cells can be accomplished in less time, the cells can be re-administered to the patient sooner. This not only makes treatment more convenient for the patient, but it helps to ensure the stem cells don't lose their self-renewing properties, Torbett says. The longer stem cells exist outside of the body and are manipulated, the more likely it is they will lose their ability to self-generate and ultimately correct disease.

Torbett and his team are continuing to study the underlying reasons for stem cells' inherent resistance to genetic modification, with the goal of further improving treatment efficiency and reducing cost. Because many of the diseases treatable with gene therapy affect children, Torbett says he feels a special urgency to advance this discovery from the lab into the clinic.

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When Added to Gene Therapy, Plant-Based Compound May Enable Faster, More Effective Treatments - Lab Manager Magazine

BEDFORD, Mass., Oct. 21, 2019 (GLOBE NEWSWIRE) -- Homology Medicines Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of preclinical data that support its investigational gene therapy programs for the treatment of metachromatic leukodystrophy (MLD) and phenylketonuria (PKU) at the American Society of Human Genetics (ASHG) 2019 Meeting.

For the first time, Homology presented preclinical data from the murine model and non-human primates that demonstrated that the HMI-202 gene therapy candidate crossed the blood-brain-barrier and the blood-nerve-barrier and increased levels of arylsulfatase a (ARSA) protein to therapeutic levels. In addition, preclinical data on gene therapy candidate HMI-102 showed that a single administration resulted in sustained Phe reduction and increased tyrosine and other important downstream biochemical metabolites in the PKU murine model.

The MLD presentation is part of a growing foundation of HMI-202 data to support a future IND filing, and the PKU preclinical data supported the initiation of our Phase 1/2 trial, which is ongoing and expected to report initial data by the end of this year, said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. Taken together, these presentations demonstrate the potential of our genetic medicines platform, investigational PKU and MLD gene therapies and our continued focus on advancing these treatments to help patients and their families.

Highlights from the posters include:

HMI-202 gene therapy in development for MLD

HMI-102 investigational gene therapy for PKU

This poster received a Reviewers Choice Abstract award during the ASHG Meeting.

A 5-year retrospective study of individuals with PKU treated at two specialized U.S. clinics

For more information, please visit http://www.homologymedicines.com/publications.

About Phenylketonuria (PKU)PKU is a rare, inherited inborn error of metabolism caused by mutations in the PAH gene. The current standard of care is a highly restrictive diet, but it is not always effective, and there are currently no treatments available that address the genetic defect in PKU. If left untreated, PKU can result in progressive and severe neurological impairment. PKU affects approximately 16,500 people in the U.S., and an estimated 350 newborns are diagnosed each year.

About Metachromatic Leukodystrophy (MLD)MLD is a rare lysosomal storage disorder caused by mutations in the ARSA gene. ARSA is responsible for the creation of the arylsulfatase A (ARSA) protein, which is required for the breakdown of cellular components. In MLD, these cellular components accumulate and destroy myelin-producing cells in the peripheral and central nervous system leading to progressive and serious neurological deterioration. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within five to ten years after onset.

About Homology Medicines, Inc.Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of clinical data; our beliefs regarding our manufacturing capabilities; the potential of and related advancement of our novel platform and pipeline; our goal of delivering potential cures to patients; beliefs about preclinical data; our position as a leader in the development of genetic medicines; and the sufficiency of our cash, cash equivalents and short-term investments. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2019 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

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Homology Medicines Presents Data from Investigational PKU and MLD Gene Therapy Programs that Demonstrate Preclinical Proof-of-Concept for Potential...