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...

DUBLIN and TAMPA, Florida, March 24, 2020 /PRNewswire/ -- Avectas, a cell engineering technology business andVycellix, Inc. an immuno-discovery cell & gene therapy company, today 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," says Michael Maguire, PhD, 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 Vycellix's President, Douglas Calder, "Solupore represents a new paradigm for delivery of transgenes, and our initial studies will evaluate Solupore to deliver our new product candidate, VY-M, to T cells and 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 Avectas and Vycellix 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 Avectas and Vycellix will further expand their collaboration within the NextGenNK constellation.

"We are excited to see the NextGenNK competence center catalysing interactions among its industrial partners to advance NK cell-based immunotherapies," says Hans-Gustaf Ljunggren, MD PhD, Director of the NextGenNK competence center. "The present collaboration may pave the way for similar collaborations among NextGenNK partners."

In February 2020, Avectas announced that it had entered an agreement with the Centre for Commercialization of Regenerative Medicine (CCRM) based in Toronto, Canada to accelerate the translation of Avectas' non-viral cell engineering platform (Solupore) into the clinic.

About Avectas: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

About Vycellix:Vycellix, Inc.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 Company's platforms were all initially 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 Company's website at http://www.vycellix.com

View original content:http://www.prnewswire.com/news-releases/avectas-and-vycellix-announce-collaboration-to-advance-next-generation-solutions-for-the-optimized-manufacture-of-cell--gene-therapies-301028561.html

SOURCE Avectas; Vycellix

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

The positioning of the Global Cell Therapy Technologies Market vendors in FPNV Positioning Matrix are determined by Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) and placed into four quadrants (F: Forefront, P: Pathfinders, N: Niche, and V: Vital).

New York, March 25, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Cell Therapy Technologies Market - Premium Insight, Competitive News Feed Analysis, Company Usability Profiles, Market Sizing & Forecasts to 2025" - https://www.reportlinker.com/p05872139/?utm_source=GNW

The report deeply explores the recent significant developments by the leading vendors and innovation profiles in the Global Cell Therapy Technologies Market including are Ge Healthcare, Lonza Group, Merck KGaA, Terumo Bct, Inc., Thermo Fisher Scientific, Inc., Beckman Coulter, Inc., Becton, Dickinson and Company, Miltenyi Biotec GmbH, Sartorius, and Stemcell Technologies.

On the basis of Product, the Global Cell Therapy Technologies Market is studied across Consumables, Equipment, and Systems & Software.

On the basis of Process, the Global Cell Therapy Technologies Market is studied across Cell Preservation, Distribution, and Handling, Cell Processing, and Process Monitoring and Quality Control.

On the basis of Cell Type, the Global Cell Therapy Technologies Market is studied across Animal Cells and Human Cells.

On the basis of End User, the Global Cell Therapy Technologies Market is studied across Life Science Research Companies and Research Institutes.

For the detailed coverage of the study, the market has been geographically divided into the Americas, Asia-Pacific, and Europe, Middle East & Africa. The report provides details of qualitative and quantitative insights about the major countries in the region and taps the major regional developments in detail.

In the report, we have covered two proprietary models, the FPNV Positioning Matrix and Competitive Strategic Window. The FPNV Positioning Matrix analyses the competitive market place for the players in terms of product satisfaction and business strategy they adopt to sustain in the market. The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. The Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines the optimal or favorable fit for the vendors to adopt successive merger and acquisitions strategies, geography expansion, research & development, new product introduction strategies to execute further business expansion and growth.

Research Methodology:Our market forecasting is based on a market model derived from market connectivity, dynamics, and identified influential factors around which assumptions about the market are made. These assumptions are enlightened by fact-bases, put by primary and secondary research instruments, regressive analysis and an extensive connect with industry people. Market forecasting derived from in-depth understanding attained from future market spending patterns provides quantified insight to support your decision-making process. The interview is recorded, and the information gathered in put on the drawing board with the information collected through secondary research.

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on sulfuric acid offered by the key players in the Global Cell Therapy Technologies Market 2. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments in the Global Cell Therapy Technologies Market 3. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets for the Global Cell Therapy Technologies Market 4. Market Diversification: Provides detailed information about new products launches, untapped geographies, recent developments, and investments in the Global Cell Therapy Technologies Market 5. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players in the Global Cell Therapy Technologies Market

The report answers questions such as:1. What is the market size of Cell Therapy Technologies market in the Global?2. What are the factors that affect the growth in the Global Cell Therapy Technologies Market over the forecast period?3. What is the competitive position in the Global Cell Therapy Technologies Market?4. Which are the best product areas to be invested in over the forecast period in the Global Cell Therapy Technologies Market?5. What are the opportunities in the Global Cell Therapy Technologies Market?6. What are the modes of entering the Global Cell Therapy Technologies Market?Read the full report: https://www.reportlinker.com/p05872139/?utm_source=GNW

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

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The Global Cell Therapy Technologies Market is expected to grow from USD 10,423.12 Million in 2018 to USD 25,712.23 Million by the end of 2025 at a...

CAMBRIDGE, Mass & HOUSTON--(BUSINESS WIRE)--AlloVir, a late-clinical stage T-cell immunotherapy company, today announced the expansion of its research and development collaboration with Baylor College of Medicine to include the discovery and development of allogeneic, off-the-shelf, virus specific T-cell therapies to combat SARS-CoV-2, the virus that causes COVID-19. With AlloVirs proprietary technology, in addition to targeting SARS-CoV-2, the investigational virus specific T-cell therapy may also address other coronavirus (CoV) strains including SARS-CoV, MERS-CoV, and also the endemic CoVs that commonly afflict immunocompromised patients. AlloVir aims to develop a therapy for CoVs that can be used as a stand-alone treatment or incorporated into the companys multi-respiratory virus investigational therapy, ALVR106, which is designed to address other devastating and life-threatening community-acquired respiratory viruses.

Given the worldwide coronavirus pandemic and risks to immunocompromised patients now and in the future, we believe it is our responsibility to leverage our scientific expertise and allocate resources for an allogeneic, off-the-shelf, coronavirus-specific T-cell program, said Ann Leen, Ph.D., AlloVir Co-Founder, Chief Scientific Officer, and Professor of Pediatrics at Baylor College of Medicine. Together with Baylor College of Medicine we have already advanced two highly innovative allogeneic, off-the-shelf, multi-virus specific T-cell investigational immunotherapies. We believe we can apply this same approach to develop a cell therapy to treat and prevent coronavirus infections and diseases in immunocompromised patients.

AlloVir and Baylor College of Medicine are leading the way in the clinical development of novel immunotherapies designed to restore natural T-cell immunity to fight off viral infections and diseases in immunocompromised patients, including recipients of stem cell and solid organ transplants, said Michael Dilling, Executive Director of Baylor Licensing Group within Baylor College of Medicine Ventures. Expanding upon this collaboration and technology platform to treat and possibly prevent other emerging life-threatening community-acquired virus infections and diseases is a natural extension of our partnership.

About AlloVirs Approach:

T cells are vital to the immune systems ability to detect and kill virus-infected cells. In healthy individuals, virus-specific T cells form a critical component of the bodys natural defense system and provide protection against thousands of disease-causing viruses.

However, these viruses can go unchecked in immunocompromised patients, such as those undergoing hematopoietic stem cell transplantation (HSCT), solid organ transplantation, and cancer treatment; in patients with HIV infection; and in the elderly. Typically, when viruses attack immunocompromised patients, standard of care therapies do not address the underlying problem of a weakened immune system and, therefore, many patients suffer with life-threatening outcomes such as multi-organ damage and failure, and even death.

AlloVir uses natural immune stimulant proteins called cytokines combined with non-harmful fragments of the virus to activate and expand naturally occurring cells against target viruses. These cells are then provided to immunocompromised patients in order to restore natural T-cell immunity to prevent and/or treat associated viral infections and diseases. AlloVir has developed a proprietary manufacturing process that allows for a bank of virus-specific T cells to be developed from a small number of carefully chosen, healthy, virus-immune, third-party donors. AlloVirs virus-specific T-cells therapies do not require exact immunological matching to patients, allowing hundreds of patients to be treated with virus-specific T-cells manufactured from a single donor. AlloVirs therapies can be stored in a frozen state and thus supplied rapidly and globally as an off-the-shelf therapy for patients suffering from, or at risk for, one or more viral infections and diseases.

About AlloVir

AlloVir, formerly ViraCyte, is an ElevateBio portfolio company that was founded in 2013 and is the leader in the development of novel cell therapies with a focus on restoring natural immunity against life-threatening viral diseases in patients with severely weakened immune systems. The companys technology platforms deliver commercially scalable solutions by leveraging off-the-shelf, allogeneic, multi-virus specific T cells targeting devastating viral pathogens for immunocompromised patients under viral attack. AlloVirs technology and manufacturing process enables the potential for the treatment and prevention of a spectrum of devastating viruses with each single allogeneic cell therapy. The company is advancing multiple mid- and late-stage clinical trials across its product portfolio.

AlloVirs lead product Viralym-M (ALVR105) is in late-stage clinical development as an allogeneic, off-the-shelf, multi-virus specific T-cell therapy targeting six common viral pathogens in immunocompromised individuals: BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, human herpesvirus 6, and JC virus. In a positive Phase 2 proof-of-concept study, published in the Journal of Clinical Oncology (Tzannou, JCO, 2017), greater than 90% of patients who failed conventional treatment and received the companys lead allogeneic T-cell therapy, Viralym-M, demonstrated a predefined criteria for a complete or partial clinical response, most with complete elimination of detectable virus in the blood and resolution of major clinical symptoms. The company plans to initiate pivotal and proof-of-concept studies with Viralym-M in 2020 for treatment and prevention of severe and life-threatening viral infections and diseases.

AlloVirs second allogeneic, off-the-shelf, multi-virus specific T-cell therapy, ALVR106, targets four common and devastating community-acquired respiratory viruses: respiratory syncytial virus, influenza, parainfluenza virus, and human metapneumovirus (Vasileiou, Haematologica, 2019). The company anticipates initiating clinical studies with ALVR106 in 2020.

AlloVirs investors include Fidelity Research and Management Company, Gilead Sciences, F2 Ventures, The Invus Group, Redmile Group, EcoR1, Samsara Biocapital, and Leerink Partners Co-investment Fund, LLC.

For more information visit http://www.allovir.com

About ElevateBio

ElevateBio, LLC, is a Cambridge-based biotechnology company, established to create and operate a broad portfolio of cell and gene therapy companies with leading academic researchers, medical centers and entrepreneurs. ElevateBio builds companies by providing scientific founders with fully integrated bench-to-bedside capabilities including world-class scientists, manufacturing facilities, drug developers and commercial expertise. ElevateBio BaseCamp, a company-owned Cell and Gene Therapy Center of Innovation, will serve as the R&D, process development and manufacturing hub across the entire ElevateBio portfolio while also supporting selected strategic partners. For more information, visit http://www.elevate.bio.

About Baylor College of Medicine

Baylor College of Medicine (www.bcm.edu) in Houston is recognized as a health sciences university and is known for excellence in education, research and patient care. It is the only private medical school in the greater southwest and is ranked 22nd among medical schools for research and 4th for primary care by U.S. News & World Report. Baylor is listed 20th among all U.S. medical schools for National Institutes of Health funding and No. 1 in Texas. Located in the Texas Medical Center, Baylor has affiliations with seven teaching hospitals and jointly owns and operates Baylor St. Lukes Medical Center, part of CHI St. Lukes Health. Currently, Baylor has more than 3,000 trainees in medical, graduate, nurse anesthesia, physician assistant, orthotics and genetic counseling as well as residents and postdoctoral fellows. Follow Baylor College of Medicine on Facebook (http://www.facebook.com/BaylorCollegeOfMedicine) and Twitter (http://twitter.com/BCMHouston).

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AlloVir Expands Its Research Collaboration with Baylor College of Medicine to Discover and Develop Allogeneic, Off-the-Shelf, Virus-Specific T-Cell...

SEATTLE, March 25, 2020 /PRNewswire/ -- According to Coherent Market Insights, the global viral vector and plasmid DNA manufacturing market is estimated to be valued at US$ 427.2 million in 2019, and is expected to exhibit a CAGR of 22.8% over the forecast period (2019-2027).

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Key Trends and Analysis of the Global Viral Vector and Plasmid DNA Manufacturing Market:

Key trends in the market include increasing incidences of cancer, rising number of product launches, and increasing collaboration and acquisition activities by key market players.

According to World Health Organization (WHO), in 2018, around 9.6 million cancer deaths occurred globally. Over the last decade, development of gene therapy for the treatment of the cancer has increased significantly. Gene therapy treatment for cancer include transfer of foreign genetic material in the targeted cancer cell in the host's body. Various types of viral vectors and plasmid DNA such as retrovirus and HGF plasmidare used in the development of gene therapy.

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Increasing product launches and approvals by regulatory authorities are expected to drive growth of the global viral vector and plasmid DNA manufacturing market over the forecast period. For instance, in December 2017, Spark Therapeutics received the U.S. FDA approval to launch the LUXTURNA in the U.S. market. It is the first FDA approved gene therapy for treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the U.S.

Furthermore, key players operating in the market are focused on adopting acquisition, agreement, and collaboration strategies, in order to expand their product offerings in markets. For instance, in December 2017, Merck KGaA entered into a commercial supply agreement with bluebird bio, Inc., a clinical-stage biopharmaceutical company. According to the agreement, Merck agreed to manufacture viral vectors for bluebird's gene therapy products targeting the rare genetic disorders.

Key Market Takeaways:

Key players operating in the global viral vector and plasmid DNA manufacturing market include

Lonza Group AG, FinVector Vision Therapies, Cobra Biologics and Pharmaceutical Services, Sigma-Aldrich Co. LLC, VGXI, Inc., VIROVEK, SIRION Biotech GmbH, FUJIFILM Diosynth Biotechnologies U.S.A., Inc., Sanofi, Cell and Gene Therapy Catapult, Brammer Bio, and MassBiologics.

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Market Segmentations:

Did not find what you were looking for? Here are some other topics:

DNA AND RNA SAMPLE PREPARATION MARKET

DNA and RNA samples are necessary for variety of applications in drug research and development and cancer studies. High quality DNA and RNA samples are important for a wide variety of research and clinical applications. Biological studies require purified and isolated nucleic acids as the first step and in all recombinant DNA techniques. The extraction of nucleic acids from biological material requires cell lysis, inactivation of cellular nucleases, and separation of the desired nucleic acid from cellular debris.

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LIFE SCIENCE PRODUCTS MARKET

Life science products include laboratory supplies & accessories, cell culture & fermentation processes, cell therapy technologies, chromatography products, bioprocess filtration, fixed and live cell research through imaging and analysis, sample collection products, recombinant proteins, cell lines, and antibodies. These products are used for drug discovery, tissue engineering, drug screening, forensic testing, and genetic analysis.

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GLYCOBIOLOGY MARKET

Glycobiology involves study of structural aspects, biosynthesis, and biology of polysaccharides and how they function in an organism. Study of glycobiology has variety of application in areas such as drug discovery and development, diagnostic applications, therapeutic application, and industrial applications.Complex structure of glycan's and difficulty in its study, high costs of spectrometry and high performance liquid chromatography are expected to hinder growth of the market.

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Global Viral Vector and Plasmid DNA Manufacturing Market to Surpass US$ 2,205.6 Million by 2027 - CMI - Yahoo Finance

The global Canine Stem Cell Therapy market registered a value of ~US$ xx Mn/Bn in 2019 and is spectated to grow at CAGR of xx% during the foreseeable period 2019-2029. In terms of product type, segment holds the largest share, while segment 1 and segment 2 hold significant share in terms of end use.

The Canine Stem Cell Therapy market study outlines the key regions Region 1 (Country 1, Country 2), region 2 (Country 1, Country 2), region 3 (Country 1, Country 2) and region 4 (Country 1, Country 2). All the consumption trends and adoption patterns of the Canine Stem Cell Therapy are covered in the report. Prominent players, including player 1, player 2, player 3 and player 4, among others, account for substantial shares in the global Canine Stem Cell Therapy market.

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Market Taxonomy

The global canine stem cell therapy market has been segmented into:

Product Type:

Application:

End User:

Region:

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The Canine Stem Cell Therapy market research answers important questions, including the following:

The Canine Stem Cell Therapy market research serves a platter of the following information:

Why go for Canine Stem Cell Therapy Market Research?

Our analysts work irrespective of the time-zone, the result, we are being recognized worldwide. We abide by the notion that each client has his/her own set of requirements. With extensive primary and secondary research, our experts churn out the most accurate information regarding the desired the Canine Stem Cell Therapy market.

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Canine Stem Cell Therapy Market top key players, size, Analysis, growth, research, Types, Regions and Forecast from 2019-2024 - Packaging News 24

Patients with certaintypes of relapsed/refractory leukemia or lymphoma showed a favorable responsewhen treated with natural killer (NK) cells modified to express an anti-CD19receptor, according to a recent study from the University of Texas MD Anderson CancerCenter in Houston.

In the study, 11 patientswith CD19-positive chronic lymphocytic leukemia (CLL) or non-Hodgkin lymphomareceived the modified cells, known as chimeric antigen receptor-NK (CAR-NK)cells. Seven of the patients experienced complete remission, while in 1 otherpatient, treatment reduced the aggressiveness of their disease. The responsesoccurred within 30 days, and treatment was well tolerated. Administration ofCAR-NK cells did not lead to cytokine release syndrome, an inflammatoryresponse commonly seen after CAR-T cell therapy. The results were published inthe New England Journal of Medicine.1

Im super excited aboutour data, and what weve seen in the patients, but Im cognizant of the factthat this was a handful of patients, said Katy Rezvani, MD, PhD, the studyssenior author. We are making great strides, but we also have to be cautious interms of what conclusions we can draw.

Adoptive cell therapyusing CAR-T cells has shown promise against CD19-positive cancers, but itshindered by several important drawbacks. For one thing, it requires isolatingand modifying the patients own cells, which eats up valuable time. Because thepatient will have already endured multiple rounds of therapy, it may not bepossible to generate useful doses of CAR-T cells from the patients depletedsupply. CAR-T cells also inflict some unpleasant side effects, includingcytokine release syndrome (CRS), an inflammatory response that causes fatigue,body aches, and fever.

Some researchers areturning to NK cells as an alternative. The aptly named natural killer cellshave the innate ability to hunt down and kill cancer cells, which makes them anenticing tool for potential therapies. Unfortunately, they dont do wellagainst some leukemias without a little help. Professor Dario Campana, MD, PhD,of the National University of Singapore was among the first to express CARs inNK cells.2 We found at that time that acute lymphocytic leukemiaswere resistant to NK cells, recalled Dr Campana, who was not involved in thecurrent trial. When you add the CAR, then the killing is tremendous. The CARoverrides any inhibitor signals that the NK cells have.

In the current phase 1/2trial, Dr Rezvani and her team created CAR-NK cells from banked cord bloodcells. Because they lack the T-cell receptors that cause graft-vs-host disease,in which the transplanted cells attack the hosts own body, NK cells can comefrom an unrelated donor and dont have to be human leukocyte antigen-matched.This means CAR-NK cells could one day be manufactured as an off-the-shelfproduct, rather than created specifically for each patient.

For this trial, eachpatient received CAR-NK cells developed from a separate cord blood donor, butDr Rezvani said that, in theory, 1 donor could provide CAR-NK cells to multiplepatients.

None of the 11 patientsdeveloped the CRS or neurotoxicity that have been observed in patientsadministered CAR-T cells, and they the researchers never reached the maximumtolerated dose. No increase in inflammatory cytokine levels was observed.

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CAR-NK Cells Make Their Debut in Hematologic Cancers - Cancer Therapy Advisor

Five real-life cases of adult patients with relapsed/refractory CD19-positiveB-cell acute lymphoblastic leukemia (ALL) were recently detailed in an articlein Blood along with the rationalesfor selecting CD19-directed CAR-T therapy or the CD3/CD19 bispecific antibody,blinatumomab, as the first CD19-targeted treatment approach for each patientcase.

While both blinatumomab and CD19-directed CAR-T therapy (ie,tisagenlecleucel in patients 25 years or younger) are approved by the US Foodand Drug Administration (FDA) for the treatment of relapsed/refractory B-cellALL, the mechanisms of action of these therapies are very different: the formerdrug activates T cells by linking them through their CD3 receptor to the CD19surface antigen on B cells, whereas CD19-directed CAR-T therapy uses autologousT cells that have been genetically modified to express the CD19 receptor.

Nevertheless, both treatment approaches are considered tolerable and potentiallycurative in the setting of relapsed/refractory B-cell ALL. Furthermore, it maybe possible to subsequently offer the alternative CD19-targeted treatment ifdisease progression occurs following treatment with either CD19-directed CAR-Ttherapy or blinatumomab. However, some patients will become ineligible for subsequenttreatment with the alternative approach due to loss of B-cell expression ofCD19.

Oncethe decision to use CD19-targeted immunotherapy to treat a patient withadvanced ALL has been made, the physician faces the challenge of selectingbetween blinatumomab and CAR T cells, the study authors noted, adding that itis crucial to weigh all considerations for each individual patient beforeselecting one immunotherapy over another.

Inthe patient cases highlighted in this article, multiple factors were consideredin making individualized treatment decisions.

Forexample, initial treatment with blinatumomab was selected for an older patientwith low-burden disease, given its FDA approval across all age groups, itslower associated risks of severe cytokine release syndrome and neurotoxicitycompared with CAR-T therapy, and its demonstrated efficacy in patients withlow-burden disease. Furthermore, because allogeneic hematopoietic stem celltherapy (allo-HCT) was planned for this patient who had a matched siblingdonor, another factor weighing in favor of blinatumomab was the avoidance of delaysassociated with CAR-T manufacturing.

Factorsassociated with selection of CD19-directed CAR-T therapy as the initialCD19-directed approach included the presence of extramedullary disease in the centralnervous system (CNS), as there is evidence supporting CNS penetration by CAR-Tcells, as well as promising antileukemic activity in patients with extramedullarydisease.

Inaddition, CAR-T therapy was preferred for a patient who experienced diseaseprogression following allo-HCT and was unlikely to receive a second allo-HCT,given evidence for long-lasting remissions even without consolidation allo-HCTfollowing treatment with CAR-T therapy.

Inthis context, the study authors stated that blinatumomab in this setting isbetter used as a bridging therapy rather than a definitivecurative treatment.

The study authors concluded that treatment with blinatumomab and CD19 CAR T cells holds promise in advanced ALL, allowing more patients to attain remission and possible cure with and without additional therapies. Both treatments have unique limitations and advantages, and the treating physician should be discerning when selecting treatment of each case.

Reference

Aldoss I, Forman SJ. How I treat adults with advanced acute lymphoblastic leukemia eligible for CD19-targeted immunotherapy.[published online March 12, 2020]. Blood. 2020;135:804-813. doi:10.1182/blood.2019002132

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Rationales for Selecting CD19-Targeted Therapy in R/R B-Cell ALL - Cancer Therapy Advisor