Category Archives: Cell Therapy

The global autologous cell therapy market is poised to grow by USD 1.97 billion during 2020-2024, progressing at a CAGR of almost 22% during the forecast period. Request free sample pages

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Technavio has published a latest market research report titled Global Autologous Cell Therapy Market 2020-2024 (Graphic: Business Wire)

Read the 120-page report with TOC on "Autologous Cell Therapy Market Analysis Report by Therapy (Autologous stem cell therapy and Autologous cellular immunotherapies), Application (Oncology, Musculoskeletal disorders, and Dermatology), Geography (North America, APAC, Europe, South America, and MEA), and the Segment Forecasts, 2020-2024".

https://www.technavio.com/report/autologous-cell-therapy-market-industry-analysis

The market is driven by the increasing demand for effective drugs for cardiac and degenerative disorders. In addition, the limitations in traditional organ transplantations are fueling the demand for stem cell therapies. All these factors are anticipated to boost the growth of the autologous cell therapy market.

The demand for effective drugs for cardiac and degenerative disorders has been increasing across the world. In addition, the discovery of possible cardiac autologous cells has enabled vendors to develop novel drugs for the treatment of various cardiac diseases. For instance, Mesoblast is developing MPC-150-IM. It is a Phase III candidate for the treatment of advanced and end-stage chronic heart failure. Similarly, Shire has been developing autologous stem cell therapies for chronic myocardial ischemia. These products are expected to be launched during the forecast period and will have a positive impact on the growth of the global autologous cell therapy market.

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Major Five Autologous Cell Therapy Market Companies:

Bayer AG

Bayer AG operates its business through segments such as Pharmaceuticals, Crop Science, Consumer Health, and Animal Health. The company offers induced pluripotent stem cells. They are developed by reprogramming mature body cells to behave like embryonic stem cells that are injected to restore diseased tissue in patients.

Brainstorm Cell Therapeutics Inc.

Brainstorm Cell Therapeutics Inc. operates its business through an unified business segment. NurOwn is the key offering of the company. It is a cell therapy platform, which develops mesenchymal stem cells for the treatment of human diseases such as immune and inflammatory diseases.

Daiichi Sankyo Co. Ltd.

Daiichi Sankyo Co. Ltd. operates its business through segments such as Innovative Pharmaceuticals, Generic, Vaccine, and OTC Related. Heartcel is the key offering of the company. It is an immune-modulatory progenitor cell therapeutic agent, which is used for ischemic heart failure.

FUJIFILM Holdings Corp.

FUJIFILM Holdings Corp. operates its business through segments such as Imaging solutions, Healthcare and material solutions, and Document solutions. The company uses induced pluripotent stem cells to derive differentiated cells, which are used in researching various diseases and conditions.

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Holostem Terapie Avanzate Srl

Holostem Terapie Avanzate Srl operates its business through an unified business segment. Holoclar is the key offering of the company. It is an advanced therapy medicinal product containing stem cells indicated to repair the cornea after injury.

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Autologous Cell Therapy Market Therapy Outlook (Revenue, USD Billion, 2020-2024)

Autologous Cell Therapy Market Application Outlook (Revenue, USD Billion, 2020-2024)

Autologous Cell Therapy Market Regional Outlook (Revenue, USD Billion, 2020-2024)

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Related Reports on Healthcare Include:

Global Cancer Stem Cell Therapeutics Market Global cancer stem cell therapy market by type (allogeneic stem cell transplant and autologous stem cell transplant) and geography (Asia, Europe, North America, and ROW).

Global Mantle Cell Lymphoma Therapeutics Market Global mantle cell lymphoma therapeutics market by product (combination therapy and monotherapy) and geography (Asia, Europe, North America, and ROW).

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Global Autologous Cell Therapy Market 2020-2024 | Evolving Opportunities with Bayer AG and Brainstorm Cell Therapeutics Inc. | Technavio - Yahoo...

GAITHERSBURG, Md. and SOUTH SAN FRANCISCO, Calif., March 24, 2020 /PRNewswire/ --MaxCyte, Inc., a global cell-based therapies and life sciences company, and Allogene Therapeutics, Inc.(Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, today announced a clinical and commercial license agreement. Under the terms of the agreement, Allogene gains rights to use MaxCyte's Flow Electroporation technology and ExPERT platform to develop and advance its AlloCAR Tcandidates through to commercialization.In return, MaxCyte will receive undisclosed development, approval and commercial milestones in addition to other licensing fees. The first two Allogene investigational therapies intended to utilize this validated gene editing and advanced proprietary cell manufacturing technology are directed at CD19 and BCMA targets.

MaxCyte's ExPERT instrument family represents the next generation of leading, clinically validated, electroporation technology for complex and scalable cellular engineering. By delivering high transfection efficiency with enhanced functionality, the ExPERT platform delivers the high-end performance essential to enable the next wave of biological and cellular therapeutics. Allogene intends to deploy the MaxCyte technology to effect the gene editing steps during the production process. The closed system and high efficiency make it an ideal addition for GMP operations.

"MaxCyte's ExPERT platform has become the industry standard in electroporation technology and allows us to increase efficiency and improve yield, which is a critical component to the value proposition of our AlloCAR T therapies," said Alison Moore, Ph.D., Chief Technical Officer of Allogene.

Doug Doerfler, President & CEO of MaxCyte, said: "We're honored to partner with Allogene to help unlock the full potential of its next-generation allogeneic CAR T therapies through utilization of our Flow Electroporation technology and ExPERT platform."

About MaxCyte

MaxCyte is a clinical-stage global cell-based therapies and life sciences company applying its proprietary cell engineering platform to deliver the advances of cell-based medicine to patients with high unmet medical needs. Through its life sciences business, MaxCyte biopharmaceutical partners leverage the Company's Flow Electroporation Technology to advance the development of innovative, transformative medicines, particularly in cell therapy. MaxCyte has placed its technology worldwide, including with all of the top ten global biopharmaceutical companies. The Company now has more than 100 partnered programme licenses in cell therapy with more than 70 licensed for clinical use. The Company has now entered into nine clinical/commercial license partnerships with leading cell therapy developers and the potential pre-commercial milestones from these relationships now significantly exceeds $650 million. With its robust delivery technology platform, MaxCyte helps its partners to unlock the full potential of their therapeutic products. MaxCyte is also developing novel CARMA therapies, with its first drug candidate in a Phase I clinical trial. CARMA is MaxCyte's mRNA-based proprietary therapeutic platform for autologous cell therapy for the treatment of solid cancers. MaxCyte has established CARMACell Therapiesas a wholly owned subsidiary to facilitate independent investment and new partnerships to advance the CARMA platform. For more information, visit http://www.maxcyte.com.

AboutAllogene Therapeutics

Allogene Therapeutics, with headquarters in South San Francisco, is a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T) therapies for cancer. Led by a world-class management team with significant experience in cell therapy, Allogene is developing a pipeline of "off-the-shelf" CAR T cell therapy candidates with the goal of delivering readily available cell therapy on-demand, more reliably, and at greater scale to more patients. For more information, please visit http://www.allogene.com, and follow @AllogeneTx on Twitter and LinkedIn.

Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the ability of the MaxCyte technology and platform to increase efficiency and improve yield of AlloCAR T therapies, the ability to manufacture AlloCAR T therapies, the ability to progress AlloCAR T therapies through clinical trials and obtain regulatory approval, and the potential benefits of AlloCAR T therapy. Various factors may cause differences between Allogene's expectations and actual results as discussed in greater detail in Allogene's filings with the Securities and Exchange Commission (SEC), including without limitation in its Form 10-K for the year ended December 31, 2019. Any forward-looking statements that are made in this press release speak only as of the date of this press release. Allogene assumes no obligation to update the forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

This announcement contains inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 (MAR).

AlloCAR Tis a trademark of Allogene Therapeutics, Inc.

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MaxCyte and Allogene Therapeutics Sign Clinical and Commercial License Agreement to Enable the Advancement of Allogeneic CAR T (AlloCAR T(TM))...

Chimeric antigen receptor (CAR) T cell therapy has been a game-changer for blood cancers but has faced challenges in targeting solid tumors. Now researchers from thePerelman School of Medicine at the University of Pennsylvaniamay have an alternative to T cell therapy that can overcome those challenges. Their research shows genetically engineering macrophages an immune cell that eats invaders in the body could be the key to unlocking cellular therapies that effectively target solid tumors. The team was able to show these CAR macrophages can kill tumors both in human samples in the lab and in mouse models. They published their proof-of-concept paper inNature Biotechnologytoday.The approach in this study is closely related to CAR T cell therapy, in which patient immune cells are engineered to fight cancer, but it has some key differences. Most importantly, it centers around macrophages, which eat invading cells rather than targeting them for destruction the way T cells do; while T cells are more like a game of Space Invaders, macrophages are like Pac-Man.

Macrophages also have another key difference from T cells in that they are the bodys first responders to viral infections. This has historically presented challenges in trying to engineer them to attack cancer, since macrophages are resistant to infection by the standard viral vectors used in gene and cell therapy.

We have known how to engineer T cells to do this for years, but the fact that macrophages are innately resistant to the viral vectors that we use in our CAR T cells presented a unique challenge, which we show here we were able to overcome, said the studys senior authorSaar Gill, MD, PhD, an assistant professor of Hematology-Oncology and a member of PennsAbramson Cancer Center.

In fact, this anti-viral property carried another unexpected benefit. Macrophages are generally among the first cells to be drawn in by cancer, and they are exploited to help tumors instead of eating them. However, the research team showed that when the viral vector is inserted, not only do these engineered macrophages express the CAR, they also transform into highly inflammatory cells. This transformation allows macrophages to resist being co-opted by tumors.

Researchers say CAR macrophages may also be able to stimulate the rest of the immune system as they attack, potentially opening the door to a greater immune response.

Finding ways to draw the rest of the bodys powerful immune system into the fight would mean an even greater impact than what a cellular therapy can do on its own, so our future research will include efforts to better understand this possibility and how we might be able to exploit it to kill cancer, said the studys first authorMichael Klichinsky, PhD, who was a graduate student at Penn while he completed the work. This paper represents the culmination of his graduate career in Gills lab. Klichinsky and Gill went on to co-found a company, Carisma Therapeutics, based on the CAR Macrophage approach.

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

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CAR Macrophages An Alternative to CAR T-cell Therapy? - Technology Networks

GOLDEN, CO / ACCESSWIRE / March 18, 2020 / Vitro Diagnostics, Inc. (OTC PINK:VODG), dba Vitro Biopharma reports on its therapy for the Coronavirus (COVID-19) pandemic. Recent umbilical cord stem cell therapies in China to fight the Coronavirus are producing encouraging safety and efficacy results.

https://www.scmp.com/news/china/society/article/3053080/coronavirus-critically-ill-chinese-patient-saved-stem-cell

http://www.aginganddisease.org/article/0000/2152-5250/ad-0-0-216.shtml

Dr. Jim Musick, Ph.D., CEO said, "As the world struggles to deal with the COVID-19 pandemic, it is important to consider stem cell therapy to reduce death rates. This is supported by clinical studies and clinical trials are underway to substantiate safety and efficacy. Vitro Biopharma is ideally suited to provide high quality stem cells to US patients, produced under strict quality control in a cGMP, ISO9001 & ISO13485 Certified environment. Our umbilical cord AlloRx Stem Cells and stem cell growth media MSC-Gro have been used in the treatment of hundreds of patients without adverse events and show efficacy in treatment of COPD, osteoarthritis, multiple sclerosis and Alzheimer's disease.

Out of years of research, we developed our patent-pending and proprietary line of umbilical cord derived stem cells AlloRx Stem Cells now being used in offshore regenerative medicine clinical trials. Our stem cells are used in regenerative medicine clinical trials with our partner in the Cayman Islands http://www.DVCStem.com. We have a recently approved clinical trial using our AlloRx Stem Cells to treat musculoskeletal conditions at The Medical Pavilion of the Bahamas http://www.tmp-bahamas.com in Nassau.

Vitro Biopharma has a patent-pending, proprietary and scalable manufacturing platform to provide stem cell therapies to critically ill Coronavirus patients. Our stem cells have been shown to be safe in Phase I protocols as we also gain evidence of efficacy.

We are in contact with federal, state and local governments to inform them of our AlloRx Stem Cell therapy option for Coronavirus infections.. The pandemic deserves consideration of all therapeutic options and there is evidence that stem cell therapy reduces the death rate from Coronavirus COVID-19 infections. We believe that critically ill patients should have the right to try stem cell therapy in the United States."

CONTACT:

Dr. James MusickChief Executive OfficerVitro Biopharma(303) 550-2778E-mail: jim@vitrobiopharma.com

Forward-Looking Statements

Statements herein regarding financial performance have not yet been reported to the SEC nor reviewed by the Company's auditors. Certain statements contained herein and subsequent statements made by and on behalf of the Company, whether oral or written may contain "forward-looking statements". Such forward looking statements are identified by words such as "intends," "anticipates," "believes," "expects" and "hopes" and include, without limitation, statements regarding the Company's plan of business operations, product research and development activities, potential contractual arrangements, receipt of working capital, anticipated revenues and related expenditures. Factors that could cause actual results to differ materially include, among others, acceptability of the Company's products in the market place, general economic conditions, receipt of additional working capital, the overall state of the biotechnology industry and other factors set forth in the Company's filings with the Securities and Exchange Commission. Most of these factors are outside the control of the Company. Investors are cautioned not to put undue reliance on forward-looking statements. Except as otherwise required by applicable securities statutes or regulations, the Company disclaims any intent or obligation to update publicly these forward-looking statements, whether as a result of new information, future events or otherwise.

SOURCE: Vitro Diagnostics, Inc.

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Stem Cell Therapy for the Coronavirus COVID-19 Pandemic - Yahoo Finance

CAR T cell therapy has been a game-changer for blood cancers but has faced challenges in targeting solid tumors. Now researchers from thePerelman School of Medicinemay have an alternative to T cell therapy that can overcome those challenges. Their research shows genetically engineering macrophagesan immune cell that eats invaders in the bodycould be the key to unlocking cellular therapies that effectively target solid tumors. The team was able to show these CAR macrophages can kill tumors both in human samples in the lab and in mouse models. They published their proof-of-concept paper inNature Biotechnology.

The approach in this study is closely related to CAR T cell therapy, in which patient immune cells are engineered to fight cancer, but it has some key differences. Most importantly, it centers around macrophages, which eat invading cells rather than targeting them for destruction the way T cells do; while T cells are more like a game of Space Invaders, macrophages are like Pac-Man.

Macrophages also have another key difference from T cells in that they are the bodys first responders to viral infections. This has historically presented challenges in trying to engineer them to attack cancer, since macrophages are resistant to infection by the standard viral vectors used in gene and cell therapy.

We have known how to engineer T cells to do this for years, but the fact that macrophages are innately resistant to the viral vectors that we use in our CAR T cells presented a unique challenge, which we show here we were able to overcome, says the studys senior authorSaar Gill, an assistant professor of hematology-oncology and a member of PennsAbramson Cancer Center.

Read more at Penn Medicine News.

See the article here:
Alternative immune cells may surpass T cells to fight solid tumors - Penn: Office of University Communications

Ever since the novel coronavirus, or COVID-19, was first reported in China's Wuhan city, the virus has spread to more than 196 countries and territories around the world with393,284 confirmed cases and17,161 deaths so far. In India, the maximum number of cases has been reported in the state of Maharashtra.The number of coronavirus cases in the country has risen to 519, with 10 deaths.

It is the need of the hour to find a solution for coronavirus.

Clinical trials in China are already testing the efficacy of stem cell therapies for COVID-19. Arecent clinical trialwith seven COVID-19 patients showed that a stem cell product improved patient outcome. According to research published in the peer-reviewed journalAging and Disease,mesenchymal stem cell (MSC) therapy could be effective in treating COVID-19.

Coronaviruses (CoV) belong to a large family of viruses leading to respiratory illnesses, such as common coldto more severe diseases such as Middle East Respiratory Syndrome (MERS-CoV) and Severe Acute RespiratorySyndrome (SARS-CoV).

Earlier this year, a new strain of coronavirus was discovered, which was not previously identified in humanbeings, also known as the novel coronavirus (nCov). The symptoms of the infection are respiratory issues, fever, cough, shortness of breath, and breathingdifficulties. More severe cases of COVID-19 can cause pneumonia, severe acute respiratory syndrome, and kidneyfailure.

In recent years, scientific research hasshown that MSCs have properties that maymake them very useful to repair damaged tissues in the patients respiratory system and promotefaster healing and recovery.Umbilical cord tissueis particularly rich inthese cells, which is why many parents arechoosing to store them at birth.

MSCs can reduce the overproduction of immune cells caused by a reaction to the virus and reduce excessive levels of inflammatory substances, thus regulating the immune system.

Currently, many vaccines or drugs are being tested to deal with coronavirus. There is widespread fear and phobia among the population. Why not use your own defence system rather than searching for drugs to tackle the virus?

MSCs are multi-potent cells that have been widely used for tissue regeneration and immunomodulation, and can be a potential solution. The infusion of autologous and allogenic MSCs has been proven safe and effective in tissue repair and disease modulation. MSCs have anti-inflammatory, antimicrobial properties; therefore, they have the potential to control inflammatory conditions, possibly viral diseases, and may reduce mortality.

Another interesting therapeutic avenue is immunotherapy. Natural killer (NK) cells, a component of our innate immune system, play an important role in tackling malignancies as well as virally infected cells. These cells serve to contain viral infections while the adaptive immune response is generating antigen-specific cytotoxic T cells that can clear the infection. Thus, NK cell therapy can be safe and effective in the management of COVID-19.

We need to ensure control of person-to-person transmission of the infection. Therefore, stringent isolation/quarantine measures are important until complete recovery of an infected individual.

(Disclaimer: The views and opinions expressed in this article are those of the author and do not necessarily reflect the views of YourStory.)

How has the coronavirus outbreak disrupted your life? Write to us or send us a video with subject line 'Coronavirus disruption' to editorial@yourstory.com

Continued here:
Can cell-based therapy be helpful in tackling coronavirus? - YourStory

TAMPA, Fla. & DUBLIN--(BUSINESS WIRE)-- Vycellix, Inc., an immuno-discovery cell & gene therapy company, and Avectas Limited, a cell engineering technology business, today jointly announced that the companies have entered into a collaboration agreement to develop proprietary approaches for cell-based immunotherapeutic products.

The companies will collaborate on the delivery of Vycellix's novel RNA immunomodulator VY-M using Avectas' cell engineering platform, Solupore. The collaboration will address current limitations for cell-based therapies, in particular with respect to the need to accelerate the manufacturing process, reduce the cost of manufacture, and ultimately improve patient outcomes.

"We are delighted to partner with Vycellix and join forces in the development of novel cell-based products," stated Michael Maguire, Ph.D., CEO of Avectas. "We believe Solupore will play a critical role in the manufacture of cell-based therapies and will support a path towards effective patient outcomes."

According to Vycellixs President, Douglas Calder, Solupore represents a new paradigm for delivery of transgenes, and our initial studies will evaluate Solupore to deliver our product candidate, VY-M, to T cells and natural killer (NK) cells. We expect to accelerate the expansion-time of T cells and NK cells by decreasing the non-dividing lag time, resulting in much shorter vein-to-vein delivery-time to patients. The studies will be conducted at Avectas Dublin-based facility and at Karolinska Institutet, Stockholm, Sweden.

Both Vycellix and Avectas are collaborative partners within NextGenNK, a newly established competence center for development of next-generation NK cell-based cancer immunotherapies based at Karolinska Institutet, Stockholm, Sweden. It is envisioned that Vycellix and Avectas will further expand their collaboration within the NextGenNK constellation.

We are excited to see the NextGenNK Competence Center catalyzing interactions among its industrial partners to advance NK cell-based immunotherapies, said Hans-Gustaf Ljunggren, M.D., Ph.D., Director of the NextGenNK Competence Center. The present collaboration may pave the way for similar collaborations among NextGenNK partners.

About Vycellix, Inc.: Vycellix is a private, immuno-discovery, life science company at the forefront of innovation in the development of cell & gene-based therapies targeting indications in, but not limited to, hematology/oncology, autoimmunity/chronic inflammatory diseases, and organ/tissue transplantation.

The Companys portfolio of transformational platform technologies encompass novel tools urgently sought after to enable broad global adoption of advanced therapies including: 1) the ability to generate Universal Cells (VY-UC), without the need to alter expression of any of the cellular components that control self-recognition (HLA Class I or II), obviating the need for immune-suppressive drugs and redefining the path towards off-the-shelf therapies; 2) the ability to amplify cell-potency through the upregulation of internal cytotoxic mechanisms (VY-X); 3) the ability to accelerate the expansion of cells for immunotherapy by near-elimination of non-dividing lag time to leap forward to shorter vein-to-vein time with expanded cells (VY-M); and, 4) the ability to markedly enhance gene transduction levels using viral vectors with implications for autologous and allogeneic CAR-T and CAR-NK cell development (VY-OZ).

The Companys platforms were all discovered by scientists at the world-renowned Karolinska Institutet (KI) in Stockholm, Sweden. KI is globally recognized for its Nobel Assembly, which awards the Nobel Prize in Physiology or Medicine. For more information, please visit the Companys website at: http://www.Vycellix.com and follow its Twitter feed at: @Vycellix.

About Avectas Limited: Avectas is a cell engineering technology business developing a unique delivery platform to enable the ex-vivo manufacture of our partners' gene-modified cell therapy products, which will retain high in-vivo functionality. Our vision is to be a leading non-viral cell engineering technology provider, integrated into manufacturing processes for multiple autologous and allogeneic therapies, commercialized through development and license agreements. For more information, please visit the Company's website at http://www.avectas.com.

Forward Looking Statements: This press release contains forward-looking statements. All statements other than statements of historical facts are forward-looking statements, including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as plan, expect, anticipate, may, might, will, should, project, believe, estimate, predict, potential, intend, or continue and other words or terms of similar meaning. These statements include, without limitation, statements related to the pre-clinical, regulatory, clinical and/or commercial development and all anticipated uses of VY-OZ, VY-X, VY-M and VY-UC, and the Companys plans for seeking out-licensing opportunities for these assets. These forward-looking statements are based on current plans, objectives, estimates, expectations and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with immuno-discovery product development, including risks associated with advancing products to human clinical trials and/or ultimately regulatory and commercial success which is subject to the uncertainty of regulatory approval, market adoption and other risks and uncertainties affecting Vycellix and its development programs. Other risks and uncertainties of which Vycellix is not currently aware may also affect Vycellixs forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. Vycellix undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200325005026/en/

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Vycellix and Avectas Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies - BioSpace

Introduction

Sangamo Therapeutics (SGMO) is a clinical-stage biotech company focusing on the research and development of genomic medicine across 4 distinct technology platforms: gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation.

Sangamo is best-known for developing its proprietary gene-editing technology, zinc finger proteins ("ZFPs"), which is a naturally occurring class of transcription factor proteins found in humans and other species. The company has used its internal know-how and technical expertise to develop a proprietary synthetic ZFP platform with potential clinical utility in ex vivo gene-edited cell therapy, in vivo genome editing, and in vivo genome regulation.

ZFPs also can be engineered to make zinc finger nucleases ("ZFNs") which proteins that can be used to specifically modify DNA sequences by knocking in or knocking out select genes, or genome editing, and ZFP transcription-factors ("ZFP-TFs") which are proteins that can be used to selectively increase or decrease gene expression.

Sangamo is developing a series of clinical programs, which are either wholly-owned or partnered with well-established pharma and biotech companies, to focus on 3 therapeutics areas in inherited metabolic disease ("IMDs"), central nervous system ("CNS), and inflammatory and autoimmune diseases. Its full list of clinical pipeline programs is listed in Figure 1.

Figure 1 Sangamo Therapeutics' Clinical Pipeline (Source)

The company's most advanced program is an investigational gene therapy for severe hemophilia A, SB-525. SB-525 is developed under a global collaboration with Pfizer (PFE), of which the rights of SB-525 have been transferred to Pfizer to run a phase 3 trial. In December 2019, both companies announced updated initial data from the phase1/2 trial of SB-525. SB-525 was generally well-tolerated and demonstrated a sustained increase in Factor VIII activity. SB-525 has been granted RMAT, Orphan Drug, and Fast Track designation by the FDA as well as Orphan Medicinal Product Designation by the European Medicines Agency ("EMA").

Beyond SB-525, the company is also investigating 2 wholly-owned gene therapy. ST-920 is being evaluated to treat Fabry disease, a rare inherited metabolic disease, in a phase 1 study in the US and UK. SB-920 has received Orphan Drug designation by the FDA. The company also plans to advance ST-101 into clinical trials in 2021 to treat phenylketonuria ("PKU") which is a rare inherited disorder that originates from a defect in the PAH gene and results in a harmful accumulation of phenylalanine in cells throughout the body.

Sangamo is working with Sanofi (NASDAQ:SNY) to develop ex vivo gene-edited cell therapies, ST-400 and BIVV-003, for transfusion-dependent beta-thalassemia ("TDT") and sickle cell disease ("SCD") respectively. Both ST-400 and BIVV-003 are related product candidates using the same technology involving gene editing of a patient's own hematopoietic stem progenitor cells using non-viral delivery of ZFN technology.

Sangamo is the phase 1/2 study of ST-400 in 6 patients with TDT while Sanofi is recruiting the phase1/2 study evaluating BIVV-003 in patients with SCD, and Sanofi is responsible for the subsequent development, manufacturing, and commercialization of both programs.

In Dec 2009, Sangamo presented interim results for the first 3 patients ST-400. As of the data cut date, 2 more patients have been enrolled although they were not included in the interim updates. The 3 patients treated with ST-400 experienced prompt hematopoietic reconstitution, demonstrating neutrophil engraftment in 14-22 days and platelet engraftment in 22-35 days. No emerging clonal hematopoiesis had been observed as measured by on-target indel pattern monitoring in the three treated patients. The downside of the data readout is that its treatment of TDT appears to be not as efficacious as other competitors such as bluebird bio (BLUE).

Sangamo also has a global collaboration and license agreement with Kite Pharma, a wholly-owned subsidiary of Gilead Sciences (GILD), for the development of engineered cell therapies for cancer. The company is working together with Kite to design ZFNs and viral vectors to disrupt and insert select genes in T cells and natural killer cells. The first program of this agreement expected to start a clinical trial in 2020 is KITE-307, which is an allogeneic anti-CD19 CAR-T cell therapy. Given the well-documented struggles of Kite's approved autologous CAR-T, Yescarta, the success of allogeneic CAR-Ts will be very beneficial.

Lastly, Sangamo is also evaluating the potential of regulatory T-cells ("Tregs") genetically modified with a CAR ("CAR-Tregs") in solid organ transplantation. CAR-Treg cell therapies are being conducted in several preclinical studies in autoimmune and inflammatory diseases such as multiple sclerosis ("MS") and inflammatory bowel disease ("IBD"). The most advanced CAR-Treg cell therapy is TX200, which is an autologous treatment for the prevention of solid organ transplant rejection and the clinical trial is expected to be initiated in 2020.

Sangamo is only planning to start a new clinical trial for its in vivo genome editing programs. SB-913 is a second-generation ZFNs program that will be used to treat Mucopolysaccharidosis type II ("MPS II") and a new clinical trial is planned to start this year.

The company had previous programs from first-generation ZFNs that have been halted as they did not demonstrate enough clinical benefits. The company plans to use data from the SB-913 study to definite the next steps for its in vivo genome editing programs.

The company also has several preclinical programs evaluating their ZFP-TF technology as a novel therapeutic approach for CNS diseases. In December, Sangamo announced a collaboration with Biogen (BIIB) to develop and commercialize ST-501 for tauopathies including Alzheimer's disease, ST-502 for synucleinopathies including Parkinson's disease, a third undisclosed neuromuscular disease target, and up to 9 additional neurological disease targets. Under the terms of the agreement, Biogen will pay Sangamo $350M upfront, inclusive of a license fee and equity investment, and Sangamo is eligible to receive up to $2.37B in future milestones.

Sangamo also has a partnership with Pfizer and Takeda (NYSE:TAK) to develop and evaluate ZFP-TFs. The company is working with Pfizer to evaluate ALS and frontotemporal lobar degeneration ("FTLD") that are linked to the mutations in the C9ORF72 gene. In the partnership with Takeda, the company is evaluating a preclinical program for Huntington's disease in which ZFP-TF is designed to differentially down-regulate the mutated disease-causing huntingtin gene ("HTT gene") while preserving the expression of the normal version of the gene.

As of 31 December, 2019, cash and equivalents on hand was $385M. The amount is excluding the $350M injection from the collaboration with Biogen, and when factored in, cash on hand should comfortably be in the range of high $600-700M. This should give them a comfortable runway to fund all operations well into 2021, an important point given that the recent stock market crash which limits any secondary offering options.

Impressively, the company has managed to strike several high-profile partnerships with 5 global biotech/big pharma companies. Such partnerships not only validate Sangamo's technology and capabilities, but they also provide future avenues of funding with as much as $6.34B royalties on net product sales and potential milestone payments due to the company.

Figure 1 Sangamo Therapeutics' Partnerships (Source)

In terms of competition, the company competes with several players, particularly in the cell and gene therapy space. bluebird bio has more advanced programs in both TDT and SCD and, to date, has shown much better efficacy. There are also other companies such as CRISPR Therapeutics (CRSP) that are using an alternative gene-editing method, CRISPR/Cas9 in gene therapy. Other companies such as Editas Medicine (EDIT) and Intellia Therapeutics (NTLA) are also developing CRISPR/Cas9 for treatments in TDT and SCD, although it must be noted that these are not their lead programs.

In terms of allogeneic CAR-T, there are more established players such as Allogene Therapeutics (ALLO), Cellectis (CLLS), and Precision Biosciences (DTIL). The main difference among these companies is primarily the choice of gene-editing tools with Allogene and Cellectis using TALEN while Precision is using ARCUS. All these companies are, currently, in a similar stage of clinical development.

In addition to healthy donors derived allogeneic therapies, Fate Therapeutics (FATE) is developing allogeneic therapies from induced pluripotent stem cells ("iPSCs") as a renewable cell source. The advantage of this is that product consistency and potency will be improved, and the manufacturing process will be akin to the well-established biologics where they are produced from a single cell line. It is notable to note that Allogene is also investigating using iPSCs as a renewable cell source. Also, Atara Biotherapeutics (ATRA) is developing an Epstein-Barr Virus ("EBV")-based allogeneic T cell therapy platform. Their lead program is in Phase 3 and a BLA filing is expected by the second half of the year. That should put them in the lead position of commercializing an allogeneic T cell therapy and the company is gradually moving into allogeneic CAR-T space as well.

Sangamo is, currently, trading at a market cap of around $700M, which is almost as much as its cash position. While its cash position will eventually deplete to fund operations and clinical trials, the current valuation means that there is also no value for its technology and intellectual position. I consider it a good time to take up a small position in Sangamo, especially if investors have a time horizon of at least a year to weather the COVID-19 black swan event and wait for further clinical updates from the company.

It must be cautioned though that investing in clinical-stage biotech can be extremely risky, given the binary nature of the field. This is especially so, given the market turmoil from the COVID-19 pandemic. The pandemic has also led to several countries announcing lockdowns, which have disrupted supply chain and operations. Several clinical trials have already been delayed globally and this may impact Sangamo negatively, as their cash burn will continue even if clinical trials are delayed.

Disclosure: I am/we are long ATRA, BLUE. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Sangamo Therapeutics: Market Cap Is At A Bargain Relative To Its Cash Position - Seeking Alpha

IASO Biotherapeutics (IASO BIO) today announced the completion of $60 million in series B financing led by GL Ventures, which is a venture capital fund of Hillhouse Capital focusing on early-stage innovative companies. Biomedicine and medical devices are its key investment areas. This new funding will go toward expanding the companys product pipeline, promoting the progress of current projects (including regulatory applications and clinical trials in China and the United States), improving the technology of a fully-human antibody development platform, expanding cooperation with leading international research and development institutions, and accelerating the construction of commercial facilities.

IASO BIO is a clinical-stage biotechnology company dedicated to the development of innovative cell therapies for cancer. Founded in March 2017, the company is developing over 10 pipeline products, focusing on autologous and universal CAR-T products for hematological tumors. All products are based on fully-human sequence. Additional development efforts include unique TCR-like CAR-T cell therapy products for solid tumor indications such as viral infection related gastric cancer and nasopharyngeal carcinoma.

IASO BIO owns a proprietary phage display library>2x1011that supports the demand for fully-human antibodies required for CAR-T and lays the foundation for the development of antibody drug pipelines as well. Its in-house plasmid, lentivirus and CAR-T production technology platforms can meet the requirements of IND submissions and clinical research for multiple products.

In September 2019, IASO BIO received NMPA approval of a phase Ib/II IND application for CT103A, an anti-BCMA CAR-T co-developed with Innovent Biologics for the treatment of relapsed refractory multiple myeloma (rr/mm). In addition, a number of new drug candidates have entered the pre-clinical stage or are involved in investigator-initiated clinical trials, with several of these drugs expected to enter clinical trials in the next two years.

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"IASO BIO welcomes the support and investment from Hillhouse Capital, and we view them as our long-term partner," said Brian Hall, co-Founder and Executive Vice President of Business Development, IASO BIO. "With their support and guidance, we are very confident in our ability to execute on our vision for the company. Their commitment enables us to accelerate the progress of research projects, enhance the team's capabilities globally, bring new hope to patients, and attract the right talent with ambition to join us."

Michael Yi, Co-CIO of Hillhouse Capital, stated, "Biomedicine is one of the areas that is most thoroughly investigated and heavily involved in by Hillhouse. We are dedicated to creating value in this industry and promoting the growth of companies with ingenuity, innovation, vision and devotion. We are deeply impressed by IASO BIOs diverse team and proactive culture. IASO BIO boasts a unique development platform for fully-human antibody, a solid CMC process for plasmid viruses and cell products, efficient corporate governance, and innovative products with excellent clinical data. IASO BIO also has strategic collaborations with leading international research institutions. What an achievement to have built up such capability and potential in a time span of just three years! It gives us great pleasure to cooperate with IASO BIO and we hope to contribute to their becoming an internationally competitive company with a portfolio of innovative drugs."

About GL Ventures

GL Ventures is a venture capital fund of Hillhouse Capital that focuses on early-stage innovative companies in four major areas: healthcare, software services, consumer Internet, emerging consumer brands and services. Founded in 2005, Hillhouse Capital is one of the largest investment institutions in Asia, focusing on discovering and creating value. Biomedicine and healthcare has always been Hillhouses key investment area. We are committed to creating a "people-oriented ecosystem, focusing on support for innovative healthcare." Built on professional investment capabilities and solid industry research, GL Ventures has invested in an array of innovative companies such as BeiGene, Innovent Biologics, Junshi Biosciences, I-Mab Biopharma, Frontage Laboratories, Nano-Micro, Medcaptain, Pinsmedical, Clinplus, Peijia Medical, Perfect Diary, Ijovo, Zoom, Deepexi, Xforceplus, Yunzhangfang, Moka, Hetao101, Codemao, Magic Ears, Kaochong, More Tickets, Beidian, and Shanzhen. In the future, GL Ventures will continue to find visionary early-stage entrepreneurs and help them grow into future industry leaders.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200324005225/en/

Contacts

Media Contacts SKC, Inc.Mary Placidomary@skc-pr.com (415) 218-3627

Sharon Y. Simsharon@skc-pr.com (415) 420-1889

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IASO Biotherapeutics Raises $60 Million in Series B Financing Led by GL Ventures to Advance Cell Therapies for Cancer Care - Yahoo Finance

Quote from Paul Lammers, MD, MSc., President and CEO of Triumvira:

"Given the challenges of manufacturing existing cell therapies, we believe it is critical to leverage innovative technology to automate manufacturing processes to improve consistency, accelerate logistics, reduce footprint, and reduce cost. Additionally, as our novel TAC technology exhibits a strong rationale for treating solid tumors, having a manufacturing process that, once commercialized, could be used at point-of-care would be vital to conveniently deliver cell therapy to patients given the high incidence of solid tumors."

Quote from Eytan Abraham, Head of Personalized Medicine, Lonza Pharma & Biotech:

"We are thrilled to be collaborating with Triumvira to accelerate and enable bringing novel cancer immunotherapies to the clinic and patients. We are leveraging the Cocoon Platform, coupled with the array of R&D and manufacturing capabilities and expertise that Lonza brings to the table, to accelerate the path to the clinic and commercial approval. Our focus is on doing this in a way that will allow these novel therapies to be manufactured reliably, reproducibly, at the needed scale and a sustainable cost."

AUSTIN, Texas; HAMILTON, Ontario & BASEL, Switzerland--(BUSINESS WIRE)-- Triumvira Immunologics, Inc. (Triumvira), a private, clinical-stage biopharmaceutical company developing a novel platform for engineering T-cells to attack cancers, and Lonza, a leading cell & gene therapy manufacturer, announced today that the companies have an agreement in place.

This collaboration, aimed at the development of Triumvira's TAC01-HER2 T-cell therapy for treating solid tumors, will leverage Lonza's expertise in process development and the usage of its proprietary Cocoon Platform. This automated patient-scale cell therapy manufacturing platform offers flexibility to execute a wide variety of protocols within a single system is enabled through a single-use, highly customizable cassette and custom programming.

Immunotherapy, particularly T-cell products, belong to the most potent drugs that could overcome the complex barriers encountered in solid tumors. However, current T-cell approaches remain insufficiently effective. The TAC receptor represents a significant advancement, providing a novel mechanism of T-cell activation, leading to improved anti-tumor responses in solid tumor models.

TAC01-HER2 has demonstrated encouraging and repeated safety and efficacy in multiple preclinical models of HER2-overexpressing tumors. Within this collaboration, TAC01-HER2 will initially be tested in a Phase 1/2 clinical trial examining different HER2-overexpressing solid tumors.

About Triumvira Immunologics

Triumvira Immunologics, Inc. (Triumvira) is a clinical stage immunotherapy company with the vision of developing novel T-cell therapies that are safer and more efficacious than current cell therapy cancer treatments, including chimeric antigen receptor (CAR) and engineered T-cell receptor (TCR) therapies.

Our proprietary T-cell Antigen Coupler (TAC) technology recruits the entire natural T-cell receptor and functions independent of the Major Histocompatibility Complex (MHC), allowing for the development of better therapies for a broader range of patients with solid or liquid malignancies and with diseases other than cancer. With operations spanning North America, our corporate offices are in Austin, Texas, with our research facilities in Hamilton, Ontario.

For more information, visit http://www.triumvira.com or send email inquiries to partners@triumvira.com.

About Lonza

Lonza is an integrated solutions provider that creates value along the Healthcare Continuum. Through our Pharma Biotech & Nutrition segment and our Specialty Ingredients segment businesses, we harness science and technology to serve markets along this continuum. We focus on creating a healthy environment, promoting a healthier lifestyle and preventing illness through consumers' preventive healthcare, as well as improving patient healthcare by supporting our customers to deliver innovative medicines that help treat or even cure severe diseases.

Patients and consumers benefit from our ability to transfer our pharma know-how to the healthcare, hygiene and fast-moving consumer goods environment and to the preservation and protection of the world where we live.

Founded in 1897 in the Swiss Alps, Lonza today is a well-respected global company with more than 100 sites and offices and approximately 15,500 full-time employees worldwide at the end of 2018. The company generated sales of CHF 5.5 billion in 2018 with a CORE EBITDA of CHF 1.5 billion. Further information can be found at http://www.lonza.com.

Additional Information and Disclaimer

Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited (SGX-ST). Lonza Group Ltd is not subject to the SGX-STs continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200324005026/en/

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Triumvira Immunologics and Lonza Announce Collaboration to Develop Innovative Point-of-Care Manufacturing for TAC T-Cell Therapeutic Targeting Solid...