Category Archives: Cell Therapy

The comprehensive report published by Persistence Market Research offers an in-depth intelligence related to the various factors that are likely to impact the demand, revenue generation, and sales of the Animal Stem Cell Therapy Market. In addition, the report singles out the different parameters that are expected to influence the overall dynamics of the Animal Stem Cell Therapy Market during the forecast period 2017 2025.

As per the findings of the presented study, the Animal Stem Cell Therapy Market is poised to surpass the value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% over the assessment period. The report includes a thorough analysis of the upstream raw materials, supply-demand ratio of the Animal Stem Cell Therapy in different regions, import-export trends and more to provide readers a fair understanding of the global market scenario.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/14941

The report segregates the Animal Stem Cell Therapy Market into different segments to provide a detailed understanding of the various aspects of the market. The competitive analysis of the Animal Stem Cell Therapy Market includes valuable insights based on which, market players can formulate impactful growth strategies to enhance their presence in the Animal Stem Cell Therapy Market.

Key findings of the report:

The report aims to eliminate the following doubts related to the Animal Stem Cell Therapy Market:

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Key Participants

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

The report covers exhaustive analysis on:

Regional analysis for Market includes

Report Highlights:

In order to get a strategic overview of the market,Access Research Methodology Prepared By Experts athttps://www.persistencemarketresearch.co/methodology/14941

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Animal Stem Cell Therapy Market: Challenges and Opportunities Report 2017 2025 - Bulletin Line

A SICK schoolboy will be spending Christmas in Singapore to receive treatment as he continues to fight his battle against leukaemia.

Oscar Saxelby-Lee is still waiting to receive CAR-T cell therapy - which is only available in Singapore as no other treatment options were available on the NHS - to treat his T-cell acute lymphoblastic leukaemia.

The treatment was due to start today (December 24), according to the Hand in Hand for Oscar Facebook page, however it has been forced to be put on hold.

The five-year-old has been given steroids as his blood results have come back high.

Oscars family have also been told there is a high possibility of secondary haemophagocytic lymphohistiocytosis (HLH) reoccurring.

HLH is a rare immune disorder where the body reacts inappropriately to a trigger, usually an infection.

This means Oscars specialised white blood cells (known as T-cells) have become over-activated, causing severe inflammation and damage to tissues such as the liver, spleen and bone marrow.

The original Facebook post reads: We have movement around the bed today. Still spiking temperatures but they are slowly phasing out from being consistent.

We are opening more Christmas cards and his face says it all. Thank you to everyone who has sent a little something over and to his classmates at Pitmaston who each decorated a card.

READ MORE:

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Tom Jones' mum: We want to prevent others feeling this grief

The following post said: Back track..... Cell infusion is on hold.

Steroids have been administered as Oscars blood results and certain markers have come back high.

Theres a high possibility of secondary HLH reoccurring so we must treat first. Never ending.

Oscars parents, Olivia Saxelby and Jamie Lee, pleaded for help to raise 500,000 after being told that, despite a bone marrow transplant, Oscars leukaemia had returned.

The community backed the campaign and raised the huge sum in just over three weeks.

Read more here:
Poorly Oscar still waiting for CAR-T cell therapy as treatment is 'put on hold' - Worcester News

Expansion of T cells collected for use with chimeric antigen receptor T-cell (CAR-T) therapy can be significantly increased when phosphoinositide 3-kinase (PI3K) inhibitors are added to the culture, according to data presented recently at the 34th Annual Meeting & Preconference Programs of the Society for Immunotherapy of Cancer, or SITC 2019.1

CAR-T cells are generated from apatients own lymphocytes, which are genetically modified in a lab to express asynthetic antigen receptor that targets them to that patients own cancercells.

In order for CAR-T cells to be given topatients, they must expand in a lab for a period of 1 to 2 weeks, explainedstudy author Edmund K. Waller, MD, PhD, FACP, of Winship Cancer Institute,Atlanta, Georgia. In some patients who have had extensive prior chemotherapyexposure, their T cells do not grow to a satisfactory extent to allow them toreceive CAR-T.

Dr Waller worked with Christopher R.Funk, BS, a medical student at Emory, to define laboratory conditions thatcould enhance the expansion of T cells and generate more effective T cells foruse in CAR-T manufacturing.

Specifically, they explored the effect ofsmall-molecule inhibitors of the PI3 kinase to modify the behavior of T cellsduring laboratory expansion. This line of research was prompted by theclinical observation that patients treated with PI3 kinase inhibitors developedautoimmune disease as a side effect of drug therapy, Dr. Waller said. Wereasoned that PI3 kinase inhibitors might augment T-cell expansion when addedto cells in the laboratory.

The team showed that expansion of T cellscan be increased significantly when PI3K inhibitors idelalisib, a PI3K deltainhibitor, or duvelisib, a dual PI3K delta and gamma inhibitor, were added tothe culture.

In addition, they successfully expanded Tcells from lymphoma patients who were heavily pretreated with prior rounds ofchemotherapy and whose T cells would have ordinarily failed the manufacturingstep in CAR-T, Dr Waller said.

In these patients, PI3K inhibitorsincreased frequencies of CD8 cells and costimulatory molecule-expressing cells.The addition of idelalisib also resulted in a dose-dependent decrease in immunecheckpoint molecules LAG3, Tim-3, and PD-1.

Looking at the T-cell differentiationstate, the PI3K inhibitors increased the frequency of early, memory, andeffector memory cells. Finally, PI3K inhibitors also significantly increasedmitochondrial mass within total CD3 and CD8 subsets.

The end result is that we are able tomanufacture, in a shorter time, more potent T cells from patients who havereceived prior chemotherapy treatment, thus, facilitating CAR-T manufacturing,Dr Waller said.

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PI3K Inhibitors Increased T-Cell Expansion Ahead of CAR-T Cell Therapy - Cancer Therapy Advisor

CLEVELAND, Ohio Immunotherapy, which boosts a patients immune system to destroy cancer cells, is widely seen as the future of cancer care.

This kind of therapy uses substances made by the body, or in a laboratory, to improve or restore the immune system. It can slow the growth of cancer cells, help the immune system do a better job of destroying tumors and slow the spread of cancer to other parts of the body.

There are several kinds of immunotherapy, including CAR-T cell therapy and immune checkpoint inhibitors. In CAR-T, white blood cells called T cells are genetically modified to activate the immune system to recognize and destroy certain cancers.

Immune checkpoint inhibitors are drugs that block proteins made by some immune system cells. When these proteins are blocked, T cells are better able to kill cancer cells.

Cancer vaccines, which are used to prevent and treat cancer, also are a kind of immunotherapy.

Have you or a family member experienced immunotherapy treatment for cancer? Was the treatment effective or ineffective? We want to hear your story.

Please write a short email, no more than 500 words, about your experiences with immunotherapy. Include your full name, age, city and daytime phone number. Your name, age and city will be published with your comment, but your phone number will be kept private.

Comments must be received by Friday, Jan. 10.

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Have you tried immunotherapy to treat cancer? Calling All Readers - cleveland.com

Introduction

I have written about companies working on research and development of cell therapies as the next frontier in cancer treatment. Current approved treatments such as CAR Ts are autologous treatments and require complex personalised manufacturing and can't be scaled up, which, in turn, drive up costs and limit patient's accessibility. It has been well documented that the approved CAR Ts are facing manufacturing issues and underperformed commercially.

To circumvent this, companies such as Atara Biotherapeutics (ATRA) and Allogene Therapeutics (ALLO) are working on allogeneic T cell therapies, which are derived from healthy donors and are less costly to manufacture. Fate Therapeutics (FATE) is taking a novel approach by developing programmed cellular immunotherapies for cancer and immune disorders.

In this article, I introduce Fate Therapeutics and give an overview of their technology and clinical pipeline.

Fate Therapeutics is a clinical-stage company that is developing first-in-class cell therapy product candidates based on a therapeutic approach known as cell programming. Fate does this by using human induced pluripotent stem cells ("iPSCs") to generate a clonal master iPSC line with preferred biological properties. The clonal master iPSC line is then directed to create their cell therapy product candidate.

Similar to how biologics such as monoclonal antibodies are manufactured from master cell lines, Fate believes that clonal iPSC lines can be made and used as a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, which, in turn, can be mass-produced repeatedly at significant scale in a cost-effective manner and can be delivered off-the-shelf to increase patient's accessibility. Figure 1 lists the advantages of using renewable master cell lines over healthy donor cells in cell therapy.

Figure 1: Advantages of Renewable Master Cell Line as Cell Source in Immunotherapy (Source)

With this approach, Fate is programming blood and immune cells, including natural killer (NK) cells, T cells, and CD34+ cells to advance a clinical pipeline of programmed cellular immunotherapies in the therapeutics areas of immuno-oncology and immuno-regulation.

In other cases, Fate also uses pharmacologic modulators, such as small molecules, to enhance the biological properties and therapeutic function of healthy donor cells ex vivo before their product candidates are administered to a patient.

In 2012, the Nobel Prize in Physiology was awarded to John Gurdon and Shinya Yamanaka for their ground-breaking discovery that fully differentiated mature human cells can be induced to a pluripotent state. iPSCs, with their unique capacity to be indefinitely expanded and differentiated into any type of cell in the body, hold revolutionary potential for creating better cell therapies.

Fate believes that iPSCs can be used to overcome key limitations inherent in many of the cell therapy product candidates undergoing development today, such as the requirement to source, isolate, engineer and expand cells from an individual patient or healthy donor with each batch of production. These batch-to-batch manufacturing requirements are logistically complex and expensive and can result in variable cell product identity, purity, and potency as well as manufacturing failures.

Fate is targeting to utilize clonal master iPSC lines as a renewable source for manufacturing cell therapy products which are uniform in composition and can be repeatedly mass-produced at significant scale in a cost-effective manner, to increase patient's accessibility. Fate is applying their expertise in iPSC biology to genetically engineer, isolate, and select single cell iPSCs to be used as clonal master iPSC lines. Such master iPSC lines are subsequently directed to create immune cells such as NK cells, T cells, and CD34+ cells.

Beyond iPSCs, Fate is also working on programming hematopoietic cells ex vivo by using advanced molecular characterization tools and technologies to identify small molecule and biologic modulators that promote rapid and supra-physiologic activation or inhibition of therapeutically-relevant genes and cell-surface proteins, such as those involved in the homing, proliferation, and survival of CD34+ cells or those involved in the persistence, proliferation, and antitumor activity of NK cells and T cells. Fate believes this approach systematically and precisely program the biological properties and therapeutic function of cells ex vivo prior to adoptive transfer is a reproducible, scalable and cost-effective approach to maximize the safety and efficacy of cell therapies.

As discussed earlier, currently approved T cell therapies are autologous and rely on the use of a patient's own cells. Manufacturing such highly personalised treatments are complex and costly, which drives up costs and limits patient's accessibility. While companies are working on allogeneic platform which is derived from healthy donors, Fate is taking it one step further by working on renewable master iPSC cell lines. Fate believes that their approach has the potential to improve cell product consistency and potency, reduce manufacturing costs, shorten time to treatment and importantly, increase patient's accessibility.

Fate is advancing a series of programmed cellular immunotherapies from both iPSC derived and donor derived cell sources, in therapeutic areas of immuno-oncology and immuno-regulation. Figure 2 lists down Fate's clinical pipeline.

At the 62nd American Society of Haematology Annual Meeting & Exposition ("ASH 2019 meeting"), Fate provided updates to several of its clinical programs, which I will cover in more detail in the below sections.

Figure 2: Fate Therapeutics' Clinical Pipeline (Source)

Since 2014, checkpoint inhibitors such as Merck's (MRK) Keytruda and Bristol-Meyer Squibb's (BMY) Opdivo have been approved to treat several cancer indications. Unfortunately, more than 60% of patients treated with checkpoint inhibitors will not respond or will relapse. Consequently, there is a huge unmet need for therapeutic approaches to overcome resistance to checkpoint inhibitors. NK cells have shown some potential to overcome such resistance and as such Fate is developing FT500, which is an off-the-shelf- NK cell therapy derived from a clonal master iPSC line, for the treatment of advanced solid tumors, both as a monotherapy and in combination with checkpoint inhibitor. In November 2018, FT500 was cleared by the FDA to be the first-ever iPSC-derived cell therapy cleared for clinical investigation in the United States.

Fate believes that the use of a clonal master iPSC line to produce FT500 provides a large, homogenous population of NK cells that is well-defined and displays potent activity, while also being available for repeat clinical dosing. At the ASH 2019 meeting, Fate reported results from the ongoing Phase 1 trial of FT500. Out of 12 patients, no dose-limiting toxicities or FT500-related serious adverse events were reported.

CD16 mediates antibody-dependent cellular cytotoxicity ("ADCC") which is a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity. Numerous clinical trials with FDA approved monoclonal antibodies have demonstrated that patients homozygous for the 158V variant, have improved clinical outcomes. However, this patient group only accounts for 15% of the population. In addition, ADCC is dependent on NK cells maintaining active levels of CD16 expression, which has been shown to be considerably downregulated in cancer patients, which can significantly inhibit anti-tumor activity.

To this end, Fate is developing FT516, a targeted NK cell product candidate created from a master clonal iPSC line engineering to express a high-affinity, non-cleavable CD16 receptor, as an off-the-shelf immunotherapy for the treatment of haematological malignancies.

In February 2019, FT516 was cleared by the FDA as the first-ever clinical investigation of a cell product derived from a clonal master engineered iPSC line. A phase 1 trial evaluating FT516 as a monotherapy for the treatment of acute myeloid leukemia ("AML") and in combination with CD20-directed monoclonal antibodies for the treatment of advanced B-cell lymphoma is ongoing.

At the ASH 2019 meeting, Fate reported encouraging interim results from the phase 1 study. The first AML patient treated showed no morphologic evidence of leukaemia at Day 42 following treatment and evidence of hematopoietic recovery.

In 2017, 2 autologous CD19 CAR T were approved - Kymirah by Novartis (NVS) and Yescarta by Gilead (GILD) to treat relapsed/refractory ("r/r") leukemias and lymphomas. While both treatments have been revolutionary, not all patients respond to therapy and even for patient who initially respond, they may experience a relapse. The downregulation of CD19 from the tumor cell surface has been clinically demonstrated to be an important mechanism of resistance.

To over CD19 antigen escape, FT596 expresses a novel modified receptor to augment ADCC to enable coincident targeting of CD19 and additional antigens such as CD20. On top of a CAR targeting CD19, FT 596 also expresses a cytokine complex that promotes survival of NK cells. Combined, these features are intended to maximize potency of FT596.

In September 2019, FT596 was cleared by the FDA for clinical investigation as the first cellular immunotherapy engineered with three active anti-tumor components. At the ASH 2019 meeting, Fate reported encouraging preclinical results for FT596 and that it has managed to produce GMP doses of FT596 at approximately $2,500 per dose. This will enable it to overcome the limitations of the current generations of autologous CAR T. Phase 1 trial of FT596 is expected to be initiated in early 2020.

FT538 is an off-the-shelf NK cell therapy created from a clonal master iPSC line engineering to prevent expression of the cell surface protein, CD38, which is highly and uniformly expressed on Multiple Myeloma ("MM") cells. CD38 is also broadly expressed on NK cells, and as a result, NK cells can be significantly depleted in patients treated with anti-CD38 antibodies. However, NK cells are important for the antitumor activity of anti-CD38 antibodies via the ADCC mechanism.

FT538 is developed for use in combination with anti-CD38 antibodies for enhanced ADCC. FT538 is designed to provide a robust population of NK cells resistant to depletion when used in combination with anti-CD38 antibodies, with the aim of improving patient outcomes in MM.

FT819 is an off-the-shelf first-of-kind CD19 CAR T manufactured from a clonal master iPSC line. The technology was licensed from Memorial Sloan Kettering ("MSK"), led by Michel Sadelain, scientific co-founder of Juno Therapeutics. FT819 is designed to overcome current limitations in approved autologous CD19 CAR Ts, including safety, costs, scalability and patient's accessibility.

At the ASH 2019 meeting, Fate reported encouraging preclinical data on FT819, with plans to submit application to start clinical trials in the first half of 2020.

Beyond iPSCs derived immunology-oncology programs, Fate is also developing NK100, which is donor-derived NK cell therapy. NK100 is produced through a feeder-free, 7-day manufacturing process during which NK cells sourced from a healthy donor are activated ex vivo with pharmacologic modulators, inducing the robust formation of adaptive memory NK cells. NK100 is currently being evaluated in 3 separate Phase 1 studies in r/r AML, ovarian cancer and advanced solid tumors which have progressed on or failed available approved therapies.

Additionally, Fate is also developing ProTmune as a programmed cellular immunotherapy as a next-generation allogeneic hematopoietic cell transplantation ("HCT") cell graft. Thousands of patients with hematologic malignancies and rare genetic disorders seek curative outcomes through HCT. HCT is limited by the occurrence of graft-versus-host disease (GvHD) and severe infections. Around 50% of patients undergoing HCT die or experience relapse within the first two years after HCT. ProTmune is designed to optimize the therapeutic properties of the graft prior to administration to patient and Fate believes that it can reduce GvHD, severe infection and disease relapse. There are no approved therapies for prevention of GvHD in patient undergoing allogeneic HCT and Fate believe ProTmune is well positioned to solve a significant unmet clinical need. ProTmune is currently investigated in an ongoing phase 2 study and has been granted Fast Track and Orphan Drug Designation by the FDA and Orphan Medicinal Product Designation by the European Medicines Agency.

As of 30 September 2019, Fate's cash on hand was US$302.8M compared to US$201.0M at 31 December 2018. This was largely driven by the net proceeds of US$162.4M from a round of public offering of its common stock in September 2019. I expect this amount to be able to fund their clinical programs way into 2021 at the very least.

In addition, Fate also announced recently the completion of its GMP facility dedicated for iPSCs - derived cell therapies. The importance of manufacturing in cell therapies cannot be understated and having its own dedicated manufacturing facility is a step in the right direction.

Fate has made huge progress in the past 12 months, with 3 programs - FT500, FT516, FT596 being cleared by the FDA to start clinical trials. The market has also reacted positively to the encouraging recent updates at the ASH 2019 meeting, with stock price rising 41% for the period 6 November to 10 December 2019, which is the period where abstracts were announced and the last day of ASH 2019 meeting.

Arguably though, the biggest show of faith in Fate's technology comes from their competitor, Allogene Therapeutics. Allogene recently announced a collaboration with Notch Therapeutics to research and develop iPSC-derived allogeneic cell therapies. This clearly marks the belief of the importance of using a renewable cell source for cell therapies, which Fate is currently the market leader.

Investing in clinical-stage biotechnology companies is generally risky as such companies have no approved products to generate revenue. Fate's clinical pipeline is also relatively early stage, with no ongoing pivotal trials. Any clinical trial failure may cause huge downward swing in its share price.

Even if the event that its product eventually gets approved, there is no guarantee of commercial success. Many of Fate's competitors working on autologous cell therapy treatment such as Novartis, Gilead, and BMS have more resources and a stronger balance sheet and Fate may not be able to compete with them in the long run.

Allogeneic players such as Atara are also working on their own platform and are currently running a pivotal trial, to be completed by the second half of 2020. As stated above, Allogene is also working on iPSC-derived therapies and will compete with Fate. While I believe Fate is the current market leader in this space, circumstances may change in the future.

As always, investors should conduct their own due diligence before taking up any position. They should also have a relatively long-time horizon and risk appetite before investing in clinical-stage biotechnology companies.

In this article, I covered an overview of Fate's cell programming approach as well as their clinical pipeline. With the limitations of current generations of cell therapies, including high cost and complex manufacturing, Fate's cell programming platform offers a refreshing and alternative approach for future generations of cell therapies.

While they have made huge progress in recent months, Fate's clinical pipeline is still relatively early stage, with no pivotal trials ongoing. Investors should consider their time horizon and risk appetite before taking up any positions. While I am optimistic about Fate's technology, I personally will be waiting for more catalyst in 2020 before taking up any positions.

For investors who wish to invest in cancer immunotherapy, they can consider the CNCR ETF which offers a diversified portfolio in the field, reducing risks in selecting individual stock ticker. I have covered companies working in cell therapies/immunotherapy and will continue to cover more companies in the coming weeks and months.

Disclosure: I am/we are long ATRA. 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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Fate Therapeutics: Using Cell Programming To Produce Better Cell Therapies - Seeking Alpha

Charles River Labs is stumping up $380 million in cash to buy out cell therapy biomaterials producer HemaCare.

This will boost Charles Rivers work in cell therapy by adding HemaCares ability to produce human-derived cellular products for this growing market.

It supplies biomaterials, including human primary cell types, and cell processing services to support the discovery, development and manufacture of cell therapies, including allogeneic (donor-derived cells) and autologous (patient-derived cells) programs.

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This builds on the hope and hype around cell therapies like CAR-T and their capacity to hit back at certain oncology targets, namely blood cancers.

The CRO said that the deal will create a unique, comprehensive solution for cell therapy developers and manufacturers worldwide to help accelerate their critical programs from basic research and proof-of-concept to regulatory approval and commercialization.

James Foster, chairman, president and CEO Charles River, said: Cell and gene therapies are important new modalities, with an estimated 10 to 20 new product approvals per year within five years. In order to continue to enhance our ability to support our clients research efforts, particularly in biologics discovery and development, we are expanding our scientific capabilities in this emerging, high-growth market with the acquisition of HemaCare.

The addition of HemaCares innovative cell therapy products and services to our integrated, early-stage solutions will create a unique, go-to partner for clients to work with Charles River across a comprehensive cell therapy portfolio from idea to novel therapeutic.

RELATED: Charles River swoops on early-stage CRO Citoxlab

Pete van der Wal, president and CEO of HemaCare, added: We are very pleased to be joining the Charles River team, which is widely recognized as the industry-leading, early-stage contract research organization. Partnering with Charles River will strengthen the value proposition for our clients, enabling them to work seamlessly with one scientific partner to enhance the speed and efficiency with which they can advance their cell therapies. The transaction will offer compelling value to our shareholders. This is an exciting day that will usher in a new era for HemaCare and my talented colleagues.

Cell therapy is becoming a focus for biopharma, but the relatively new area requires cutting-edge tech to help nurture new research like CAR-T into an established market.

Charles River says its work in the area is currently making around $100 million a year, but it sees the addressable market for HemaCares products is expected to increase from approximately $200 million today to nearly $2 billion in 10 years and wants to be a part of that.

While spending nearly $400 million in cash (the company had a market cap of $257 million at the end of play last week, with Charles River paying a 33% premium), HemaCare is expected to immediately drive profitable revenue growth, with estimated revenue growth of at least 30% annually over the next five years, the CRO estimated.

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Charles River Labs snaps up HemaCare, eyeing the growing cell therapy market - FierceBiotech

In January 2019, then-FDA commissioner Scott Gottlieb ushered in the new year with a bold prediction: The agency, he said, would be approving between 10 and 20 gene and cell therapies per year by 2025. At the time, there were a whopping 800 such therapies in the biopharma pipeline and the FDA was aiming to hire 50 new clinical reviewers to handle the development of the products.

That momentum will no doubt start to pick up in 2020, as several companies in late-stage development of their gene and cell therapies achieve key milestones or FDA approval. Among the companies expected to make major strides in gene and cell therapies next year are Biomarin, with valoctocogene roxaparvovec to treat hemophilia A, Sarepta and its gene therapy for Duchenne muscular dystrophy, plus multiple players developing CAR-T treatments for cancer, including Bristol-Myers Squibb and Gilead.

But with such explosive growth comes challenges. Gene and cell therapies require enormous up-front investing in complex manufacturing processes, as well asinnovative approaches to securing insurance coverage for products that come with eye-popping price tagssuch as Novartis $2 million gene therapy Zolgensma to treat spinal muscular atrophy. Those are just a few of the obstacles that will be front-and-center in 2020 as more gene and cell therapies make their way towardthe finish line.

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Pharma companies will face challenges figuring out how to incorporate gene and cell therapies into their overall business, said Michael Choy, partner and managing director at Boston Consulting Group, in an interview with FiercePharma. They dont fit well into the normal paradigms of budgeting and decision-making. They require a different pace of evolution and specialized expertise. For now, companies are shoe-horning gene therapies into their current model, but over the long-term there will have to be changes.

That will become increasingly clear in 2020 as both Big Pharma and small up-and-comers move towardthe clinic with their gene and cell therapies. John Zaia, M.D., director of the Center for Gene Therapy at City of Hope, predicts there will be at least three gene and cell therapy FDA approvals in 2020. He also expects to see momentum among companies seeking to improve on the technology to address unmet needs in medicine.

For example, Zaia believes off-the-shelf CAR-T cancer treatments will show promise in early studiesand will be met with enthusiasm in the cancer community, he told FiercePharma in an email. The first generation of FDA-approved CAR-T treatments, Novartis Kymriah and Gileads Yescarta, take several weeks to make because they require removing T cells from patients and engineering them to recognize and attack the patients'cancers. Several companies are advancing off-the-shelf CAR-T treatments, including Precision BioSciences, which has been building out a manufacturing plant equipped to make 10,000 doses per year.

RELATED: Biotech building facility to make genome-edited, off-the-shelf CAR-T therapies

Gene therapies for inherited diseases will make strides in 2020, too, Zaia predicts. City of Hope is one of the participants in a phase 1 study of CSL Behrings gene therapy to treat adults with sickle cell disease. CSL will be racing against several companies working on the disease, including Bluebird Bio, which is testing its beta thalassemia gene therapy Zynteglo in sickle cell. There is a big push from many research centers to cure sickle cell diseaseand early results with the use of gene therapy look very promising, Zaia said. Years of research is finally coming to realization.

With such robust R&D underway in gene and cell therapies, its no surprise several players are stepping up their investments in manufacturing. In October, Sanofi said it would retrofit a vaccine plant in France so it couldbe used for gene therapy manufacturing. Pfizer shelled out $19 million for a North Carolina facility that will serve as its manufacturing hub for gene therapies. Even Harvard University is getting into the game, working with a consortium of contract manufacturers to build a $50 million facility dedicated to making cell therapies and viral vectors for gene therapies.

But how will the healthcare system pay for all of these complex therapies? Its a question that will continue to dog the industry, BCGs Choy said. Theres a lot of interest in outcomes-based payments and payments over time, but the issue is theyre very difficult to implementbecause the infrastructure to track outcomes over time doesnt really exist, he said.

Still, payers and pharma companies are hinting at their willingness to put that infrastructure in place. Pfizer, which is developing DMD and hemophilia gene therapies, said recently its brainstorming with payers on innovative strategies for reimbursement. Novartis and Spark have already pioneered payment strategies that deviate from the standard pay-everything-up-front system. Novartis has some pay-for-performance contracts in place for the $475,000 Kymriah. And in September, Cigna agreed to cover Novartis Zolgensma and Sparks Luxturna on a per-month, per-member schedule.

RELATED: Novartis, Spark gene therapies win a boost with soup-to-nuts Cigna coverage

Despite the many challenges in cell and gene therapy, some players are showing theres likely to be a robust market for these innovative treatments. In its first quarter on the market, Zolgensma brought in $160 million in salesfar surpassing analysts expectations.

The promise of huge returns on gene and cell therapies will likely drive acquisitions in 2020, Choy predicted. These treatments are so transformative for patients, and as the clinical proof of effectiveness continues to grow, youre going to see a lot more deal-making in this area, he said.

Buyers will likely show a willingness to invest in early-stage gene and cell therapies, especially if they come with technology platforms that allow for the development of many follow-up products, Choy added. For these types of therapies, the lifecycles will be much shorter than they are for traditional pharmaceuticals, particularly for rare diseases, he said. If you administer a one-time therapy, that revenue peaks quite quickly and then drops off. So to have a sustainable revenue from a gene therapy business, you need to replace that, which requires managing a pipeline.

Judging from recent events in the burgeoning gene and cell therapy industry, the news flow in 2020 will be generated not just by the industrys largest players, but also by its upstarts. In December, Ferring Pharmaceuticals spinout FerGene turned heads with data showing that its gene therapy to treat non-muscle invasive bladder cancer eliminated tumors in more than half of participants in a phase 3 trial. And Gileads Kite Pharma just applied for FDA approval for its mantle cell lymphoma CAR-T, KTE-X19, based on a 93% overall response rate in a phase 2 trial.

There were 75 gene therapy clinical trials initiated in 2018, nearly doubling the trial starts of 2016momentum thats likely to continue next year, BCG said in a recent report. The scientific foundation is in place, BCG analysts concluded, but there is still much to do to deliver the full benefit of gene therapy to patients."

Excerpt from:
Pharma's gene and cell therapy ambitions will kick into high gear in 2020despite some major hurdles - FiercePharma

SINGAPORE, Dec. 19, 2019 /PRNewswire/ -- Tessa Therapeutics (Tessa), a clinical-stage cell therapy company focused on the development of innovative cell therapies to treat cancer, today announced plans to open a 90,000 square foot commercial-scale cell therapy manufacturing facility in Singapore by end-2020, which will be one of the leading commercial-scale cell therapy manufacturing facilities in Asia.

Notably, the state-of-the-art facility will be built to run both clinical and commercial manufacturing of cell therapy products that is compliant with current Good-Manufacturing-Practice (cGMP) guidelines from the U.S. Food and Drug Administration, European Medicines Agency and key regulators in Asia.

"This facility will strengthen our ability to bring innovative cell therapies to thousands of patients around the world," said John Ng, Chief Operations Officer of Tessa Therapeutics. "As one of very few manufacturing facilities globally designed with the capabilities to meet clinical trial and registration requirements from multiple geographies, Tessa is poised to rapidly advance its role as a leading innovator of next-generation cancer cell therapies."

Operationally, the facility will integrate digital technology to monitor, in real-time, its manufacturing operations and vein-to-vein supply chain logistics. The ability to digitally manage the complex process involved in delivering cell therapies to patients worldwide will not only enhance the traceability and control of patient material, but also enable greater standardization, scalability and quality management across its operations. This ultimately translates into better decision-making and coordination at the hospital sites for improved patient care.

The new facility will expand on the company's proven record of successful global cell therapy manufacturing, shipments, and infusions to patients in a Phase III clinical trial setting. Currently, the Company has produced and delivered autologous, personalized cell therapies to more than 100 patients in 30 clinical sites across five countries. The new manufacturing facility will enable Tessa to deliver its cell therapies faster and more reliably.

The new facility will also build out Tessa's in-house process development capabilities and serve as a centre of excellence to advance CMC (Chemistry, Manufacturing and Controls) development efforts of its cell therapies from early phase clinical development through to commercialization.

The entire manufacturing hub, totalling 130,000 square feet, will include office space to house Tessa's Corporate Headquarters. Tessa has entered into a lease agreement to develop the facility within an existing high-tech industrial building in Singapore.

About Tessa Therapeutics

Tessa Therapeutics is a clinical-stage biotechnology company focused on the development of cell therapies for lymphomas and solid tumors. Tessa's Virus-Specific T cell (VST platform) has shown a strong safety profile and early efficacy in the treatment of solid tumors.

Tessa is currently conducting two pivotal studies of autologous cell therapies in nasopharyngeal cancer and classical Hodgkin lymphoma. Tessa also has an earlier-stage clinical pipeline of autologous and off-the-shelf, allogeneic therapies targeting a wide range of cancers.

The Company has built robust operational and supply chain capabilities, across Asia and the United States, to successfully deliver cell therapies on a global scale, creating a fully integrated approach to the treatment of cancer.

For more information on Tessa, please visit http://www.tessatherapeutics.com.

Tessa Therapeutics Media Contacts

Gladys Wonggladyswong@tessatherapeutics.com +65-6384-0755

Zara LockshinSolebury Troutzlockshin@troutgroup.com +1-646-378-2960

https://www.tessatherapeutics.com/

SOURCE Tessa Therapeutics

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Tessa Therapeutics to Open 90,000 Square Foot Commercial-Scale Cell Therapy Manufacturing Facility in Singapore in 2020 - PR Newswire UK

A geriatric assessment-guided multidisciplinary team clinic (MDC) approach for older hematopoietic cell transplantation (HCT) candidates appears to be feasible and may help reduce transplant-associated morbidity and mortality, according to a study published in Blood Advances. Researchers found that a greater adoption of this approach could lead to more widespread utilization of transplantation among older adults.

The team reported outcomes with an MDC approach that included a cancer-specific geriatric assessment and a team of providers who assessed candidacy of patients for HCT. The providers created an individualized optimization plan for allogeneic HCT candidates aged 60 years and older and for autologous HCT and adoptive T-cell therapy candidates aged 70 years and older.

The researchers treated 247 patients with this MDC model, with allogeneic HCT candidates comprising the majority of the cohort (60%), followed by autologous HCT candidates (37%) and older cellular therapy candidates (3%). Patients treated using the MDC approach experienced fewer inpatient deaths (P <.001), shorter length of stay (P <.001), and fewer discharges to nursing facilities (P =.0043) compared with patients treated prior to the implementation of this approach.

The 1-year rate of overall survival improved from 43% prior to implementation of the MDC approach to 70% with the MDC approach, and the 1-year nonrelapse mortality rate decreased from 43% to 18%. For 31 recipients of autologous HCT aged 70 years or older for whom treatment was optimized by the MDC approach, nonrelapse mortality and OS at 1 year were 0% and 97%, respectively.

The researchers noted that until recently, identifying risk factors for expected HCT complications aside from age had been difficult. The MDC approach outlined may offer a way to maximize resiliency to avoid biases of older age alone and more safely offer hematopoietic and cellular therapies.

However, the researchers cautioned that their findings have significant limitations because they come from a single institutional observational study of heterogeneous patients and transplant types. Thus, prospective studies are warranted that can more completely characterize the interventions and surrogate markers that may be of most benefit in this patient population.

Disclosures: Some authors have declared affiliations with the pharmaceutical industry. Please refer to the original study for a full list of disclosures.

Reference

Read more:
A Multipronged Approach to Geriatric Assessment Before Hematopoietic Cell Transplantation - Hematology Advisor

A multicenter phase 3 trial began enrolling patients earlier this year to test the safety and efficacy of tisagenlecleucel (tisa-cel/Kymriah) as a second-line therapy for aggressive B-cell non-Hodgkin lymphoma (NHL).

Tisa-cel, an anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy, has already been approved for use in patients who have relapsed after receiving 2 lines of therapy. But its possible that, if administered sooner across treatment regimens, CAR-T could help more patients avoid relapse. The new study on this topic, known as BELINDA, aims to answer that question.

The hypothesis is that CAR-T cells should improve upon progression-free survival as compared to standard of care, said Michael Bishop, MD, director of the hematopoietic stem cell transplantation program at the University of Chicago Medicine, Illinois, and one of the BELINDA coauthors. Dr Bishop presented the study protocol at the 34th Annual Meeting & Preconference Programs of the Society for Immunotherapy of Cancer, or SITC 2019, in National Harbor, Maryland.1

Around a third of patients with non-Hodgkin lymphoma (NHL) will relapse after receiving first-line immunochemotherapy, and another 10% to 15% do not respond to initial treatment. For these patients, the outlook is grim: median overall survival is less than 12 months. Second-line treatment consists of high-dose chemotherapy combined with autologous stem cell transplant, but fewer than half of patients will qualify for a transplant. Youve got half the patients who wont get the transplant, and the other half that do, only a quarter of those will have sustained remission, said Dr Bishop. Its a large unmet patient need.

Dr Bishop went on to explain that previous trials have indicated that some 30% to 40% of patients receiving CAR-T therapy for multiply relapsed or refractory NHL have achieved long-term remission. The other exciting thing about this trial is its moving CAR-T up the treatment algorithm, he said.

The BELINDA trial is a multicenter, phase 3, open-label trial, in which patients are randomly selected to receive treatment in 1 of 2 arms: tisa-cel, or standard of care. Similar to the ZUMA-7 trial,2 which tested another CAR-T therapy called axicabtagene ciloleucel (Yescarta), BELINDA is enrolling patients whose disease either does not respond to first-line therapy (rituximab and anthracycline) or has returned within 12 months, and who are eligible for autologous stem cell transplant.

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BELINDA Trial Tests Earlier Use of Tisa-Cel in Aggressive B-Cell Non-Hodgkin Lymphoma - Cancer Therapy Advisor