Category Archives: Cell Therapy

Celularitys subsidiary, LifebankUSA, maintains a repository of placental and cord blood, containing stem cells for potential therapeutic use. (LifebankUSA / Robert Hariri via Twitter)

Seattles Infectious Disease Research Institute and a New Jersey company called Celularity have been cleared by the Food and Drug Administration to start trials of an experimental cell-based therapy for COVID-19.

The immunotherapy treatment makes use of natural killer cells, or NK cells, which play a key role in the bodys natural defense against viral infections. Celularitys NK cell product, known as CYNK-001, is derived from placental stem cells for treating viral diseases as well as some types of blood cancers and tumors.

CYNK-001 has been safely given to patients in early trials for treating leukemia and multiple myeloma. Now the FDA has given investigational new drug clearance for its use to be extended to COVID-19, the disease caused by the coronavirus outbreak.

To date, efforts to treat COVID-19 cases have been primarily focused on antiviral medications, Corey Casper, who is CEO of the Infectious Disease Research Institute as well as a clinical professor of global health at the University of Washington, explained in a news release.While these are important, patients with serious disease may not respond completely to antiviral medications because they are experiencing damage already inflicted on the bodys vital organs.

About 80 to 100 patients diagnosed with pneumonia due to a COVID-19 infection will be enrolled at medical centers in the U.S., potentially including sites in Washington state. Participants in the study will be provided with infusions of CYNK-001.

The hypothesis is that administering NK cells to patients with moderate to severe COVID-19 will allow the immune cells find the sites of active viral infection, kill the virus, and induce a robust immune response that will help heal the damage and control the infection, Casper said. Beyond its promise as a critically needed treatment for COVID-19, the biology of NK cells indicates a possibility that this immunotherapy could be used as an off-the-shelf treatment for future pandemic infections.

Initial results are expected about 30 to 60 days after the first patients receive the cells. If the results are encouraging, a more rigorous clinical trial would follow.

Last weekend, Rudy Giuliani, President Donald Trumps personal attorney, touted the treatment on Twitter, saying that this therapy has real potential. This week, the FDA gave its clearance for the preliminary tests on an expedited basis.

In a statement issued today, Celularitys chairman and CEO, Robert Hariri, thanked the FDAs Center for Biologics Evaluation and Research for their tireless effort to guide us through the process of clearing the drugs use for COVID-19.

The New York Times quoted Paul Knopfler, a stem cell researcher at the University of California at Davis, as saying that Celularitys approach carried some risk. Its conceivable that the NK cells could exacerbate problems by massive killing of the patients respiratory cells, he told the Times.

The FDA has cleared testing of several other therapies for COVID-19, including use of the drugs hydroxychloroquine and remdesivir as well as blood plasma from recovered patients. In addition, the first U.S. clinical trial of a COVID-19 vaccine is underway at Seattles Kaiser Permanente Washington Research Institute.

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Celularity and Infectious Disease Research Institute will try out COVID-19 cell therapy - GeekWire

At least one outside expert said the approach could present safety risks. Paul Knoepfler, a stem cell researcher at the University of California, Davis, said that patients with coronavirus can develop severe reactions where their immune systems go too far in attacking cells in their lungs, causing damaging inflammation. Other cell therapies tested in China are designed to dampen the immune response. He said one risk with the natural killer cells is they could go in the other direction, exacerbating respiratory problems by massive killing of the patients respiratory cells.

Despite the scant evidence, Mr. Giuliani has become an early booster, interviewing Dr. Hariri on a podcast published on his website Saturday, and praising the treatment on Twitter, saying, this therapy has real potential. In a tweet on Saturday, he added, Lets hope F.D.A. can recognize that their cumbersome process designed to keep us safer, if it is not altered dramatically in times of great need, can result in unimaginable loss of human life.

Around the same time, Twitter deleted a post by Mr. Giuliani that it said violated its rules. The tweet, from March 27, made unfounded claims about the malaria drug hydroxychloroquine, one of the treatments that Mr. Trump has supported.

Dr. Hariri said that he has known Mr. Giuliani for years and that the appearance on his podcast was a friendly chat between people who know each other and who share a common interest in this particular response to this disease.

He said that he has no business relationship with Mr. Giuliani, and that Mr. Giuliani is not representing him in any way, either paid or unpaid. I dont have anything to do with what the mayor tweets or whatnot, and I dont agree or disagree with anything, he said.

Dr. Hariri said the company would follow the established process for testing whether a drug works. We have waited for the F.D.A. to complete their review, which they did in a heroic and quick fashion, he said.

On Wednesday evening the same day the F.D.A. approved his trial Dr. Hariri praised the appearance by the agencys commissioner, Dr. Stephen Hahn, on the conservative Fox News talk show The Ingraham Angle.

We are fortunate to have Dr. Hahn at the helm, he tweeted.

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Unproven Stem Cell Therapy Gets OK for Testing in Coronavirus Patients - The New York Times

New Delhi:Researchers at Queens University Belfast are leading a UK-wide clinical trial, offering an innovative cell therapy treatment for Covid-19 patients with acute respiratory failure.

This clinical trial led by Professor Danny McAuley and Professor Cecilia OKane, both researchers from the Wellcome-Wolfson Institute for Experimental Medicine at Queens is investigating the use of allogenic Mesenchymal stromal cells (MSCs) in patients with a complication known as acute respiratory distress syndrome (ARDS) caused by coronavirus.

In the most critically unwell patients with Covid-19, many develop a complication known as ARDS.

In ARDS, the lungs become inflamed and leaky so they fill with fluid. This causes respiratory failure and patients may require admission to intensive care and a ventilator to support their breathing.

A recent statement from the four UK Chief Medical Officers outlined the importance of clinical trials amid the Covid-19 crisis.

Professor Cecilia OKane said: It is only through clinical trials will we be able to determine if new treatments are effective and safe in critically ill patients.

The trial involves the use of MSCs, a type of cell derived from human tissue such as bone marrow or umbilical cord (which is otherwise discarded after the baby is born), to treat the injury to the lung caused by Covid-19.

MSCs are a novel treatment that has been shown in experimental models to reduce inflammation, fight infection and improve the repair of injured tissue.

Patients in this trial, which is known as Realist Covid-19, will be treated with a purified population of MSCs derived from umbilical cord tissue called ORBCEL-C.

The ORBCEL-C therapy has been developed by scientists at Orbsen Therapeutics in Galway, Ireland.

The ORBCEL-C therapeutic is manufactured under licence by the UK NHS Blood and Transplant Service for the Realist Covid-19 trial.

The trial is being introduced as part of an existing programme of research investigating the use of MSCs in patients with ARDS.

The first patient has now been recruited with plans to recruit at least 60 patients throughout the Covid-19 pandemic at multiple sites across the UK, including Belfast, Birmingham and London.

Professor Ian Young, Clinical Professor at the Centre for Public Health, Queens University Belfast, Director of HSC R&D and Chief Scientific Advisor at the Department of Health, said: The Health and Social Care Research & Development Division has been working with researchers across HSC to address the global problem of coronavirus.

The vital research which will provide important evidence regarding a potential new treatment for respiratory failure, a leading cause of mortality in Covid-19.A

We will continue to support health research and encourage people to participate in research trials and other studies so patients can get the best possible treatment to help tackle the spread of Covid-19.

The trial has been identified by the National Institute for Health Research (NIHR) as a national urgent public health study.

It is one of the many Covid-19 studies that have been given urgent public health research status by the Chief Medical Officer and the Deputy Chief Medical Officer for England.

The study is funded by the Health and Social Care Research & Development Division and the Wellcome Trust, sponsored by the Belfast Health and Social Care Trust and supported by the NI Clinical Trials Unit, the NIHR Clinical Research Network and the Northern Ireland Clinical Research Network.

Orbsen CSO Steve Elliman noted: While there are over 100 vaccines and therapies in development targeting the SARS-CoV-2 infection at present there are no disease modifying therapies approved for ARDS.

Were delighted the Realist trial was approved and listed by NIHR as an Urgent Public Health Research Study so we can continue assess the safety of the ORBCEL-C therapy in patients with ARDS.

Sir Professor Alimuddin Zumla of University College London, a global coronavirus and infectious diseases expert said: This is an exciting and important trial which targets rectifying the underlying causes of lung damage and has great potential of saving many lives from Covid-19.

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Trial in Britain for cell therapy treatment of COVID-19 - The Siasat Daily

At one-year follow-up, phase 2 clinical trial results showed that most patients with relapsed or treatment resistant mantle cell lymphoma responded to chimeric antigen receptor (CAR)-T cell therapy.

KTE-X19, a type of CAR-T cell therapy, was given to 68 patients who were a median age of 65, most (84%) were men and more than 80% had stage 4 disease. In addition, intermediate to high-risk mantle cell lymphoma was diagnosed in more than half of the patients.

Patients had all received up to five therapies prior to being given KTE-X19, including a Brutons tyrosine kinase (BTK) inhibitor. BTK inhibitor therapy has greatly improved outcomes in patients with relapsed or refractory mantle cell lymphoma, yet patients who have disease progression after receiving the treatment are likely to have poor outcomes, with median overall survival of just six to 10 months, stated a press release from MD Anderson.

However, after receiving the CAR-T cell therapy, 93% of patients responded to KTE-X19, including 67% who saw no signs of cancer following treatment. At a median one year-follow up, 57% of patients were in complete remission and overall survival was 83%. Progression-free survival, or the time after treatment without a patients disease worsening or death, was estimated to be 61%. At the time of the analysis, 76% of all treated patients in the study were alive, noted the researchers.

ZUMA-2 is the first multi-center, phase 2 study of CAR T-cell therapy for relapsed/refractory mantle cell lymphoma, and these efficacy and safety results are encouraging, Dr. Michael Wang, professor of lymphoma and myeloma, said in a press release. Although this study continues, our reported results, including a manageable safety profile, point to this therapy as an effective and viable option for patients with relapsed or refractory mantle cell lymphoma.

The most common side effects were neutropenia and thrombocytopenia. Cytokine release syndrome, or a rapid release of cytokines into the blood, occurred in 15% of patients and neurologic events in 31%. Both side effects are common when using CAR-T cell therapy, but all were managed in patients and no one died.

CAR-T cell therapy involves removing a patients T cells, genetically reengineering them in a lab and then infusing them back into the patient with a hope that these new T cells will attack the cancer cells. Two CAR-T therapies have already been approved by the Food and Drug Administration (FDA) Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) for the treatment of some adults and younger patients with certain blood cancers.

In February, the FDA granted a priority review to KTE-X19 for the treatment of adult patients with relapsed or refractory mantle cell lymphoma and is set to make its approval decision by Aug. 10, 2020.

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CAR-T Cell Therapy Shows Promise in Patients with Mantle Cell Lymphoma - Curetoday.com

Dublin, April 07, 2020 (GLOBE NEWSWIRE) -- The "CAR T-Cell Therapy for Multiple Myeloma - Market Insights and Market Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.

This report delivers an in-depth understanding of the CAR T-Cell Therapy use for Multiple Myeloma as well as the CAR T-Cell Therapy market trends for Multiple Myeloma in the 6MM i.e., United States and EU5 (Germany, Spain, Italy, France and the United Kingdom).

The Multiple Myeloma CAR T-Cell Therapy market report provides current treatment practices, emerging drugs, CAR T-Cell Therapy market share of the various CAR T-Cell Therapies for Multiple Myeloma, the individual therapies, current and forecasted Multiple Myeloma CAR T-Cell Therapy market Size from 2017 to 2030 segmented by seven major markets. The Report also covers current Multiple Myeloma treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

Scope of the Report

Report Highlights

Key Questions

Market Insights:

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

Reasons to Buy

Key Topics Covered:

1 Key Insights

2 Executive Summary

3 CAR T-Cell Therapy Market Overview at a Glance3.1 Market Share (%) Distribution of CAR T-Cell Therapy for MM in 2030

4 CAR T-Cell Therapy Background and Overview4.1 Introduction4.1.1 CARs Generations4.1.2 Genetic Engineering of T-Cells4.1.3 How CAR T-Cell Therapy Works4.2 The promise of CAR T-cell targeting B cell maturation antigen (BCMA) in multiple myeloma4.3 Current challenges in CAR T4.3.1 Therapeutic side effects4.3.2 CAR T-cells lack of success4.4 CAR T-cell therapy: Route to reimbursement4.5 Unmet needs

5 CAR T-Cell Therapy for Multiple Myeloma (MM): 6 Major Market Analysis5.1 Key Findings5.2 Market Size of CAR T-Cell Therapy in 6MM5.2.1 Market Size of CAR T-Cell Therapy by Therapies

6 Market Outlook

7 Emerging Drug Profiles for Multiple Myeloma7.1 bb2121: Celgene Corporation7.1.1 Product Description7.1.2 Research and Development7.1.3 Product Development Activities7.2 JNJ-68284528 (LCAR-B38M): Janssen Research & Development7.2.1 Product Description7.2.2 Research and Development7.2.3 Product Development Activities7.3 P-BCMA-101: Poseida Therapeutics7.3.1 Product Description:7.3.2 Research and Development7.3.3 Product Development Activities7.4 CAR-CD44v6: MolMed S.p.A.7.4.1 Product Description7.4.2 Research and Development7.4.3 Product Development Activities7.5 JCARH125 (Orvacabtagene autoleucel): Celgene Corporation7.5.1 Product Description7.5.2 Research and Development7.5.3 Product Development Activities7.6 Descartes-08: Cartesian Therapeutics7.6.1 Product Description7.6.2 Research and Development7.7 CT053 : CARsgen Therapeutics)7.7.1 Product Description7.7.2 Research and Development7.7.3 Product Development Activities

Companies Mentioned

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

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

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Outlook on the Worldwide CAR T-Cell Therapy for Multiple Myeloma (2017 to 2030) - Featuring MolMed, Celgene & Cartesian Therapeutics Among Others...

Ziopharm Oncology(ZIOP) has been under major pressure after their recent follow-on-offering as well as the collapse in the overall market, explains biotech expertJohn McCamant, editor ofThe Medical Technology Stock Letter.

Many biotech stocks have been under relentless selling pressure as the broad markets suffer a broad lack of confidence and uncertainty due to COVID-19.The recent offering has not helped as the added dilution has very difficult for the market to absorb; on the other hand the company now is well financed.

More from John McCamant: Biomarin: Gene Therapy and Hemophilia

The company also contributed by missing their timeline to start the Phase II TCR-T NCI trial in solid tumors with Dr. Rosenberg.Originally forecast to occur in Q4 of last year, the timeline has now been pushed out to the second quarter of this year.While the delay is disappointing, improved manufacturing processes are one of ZIOPs advantages.

Once the Phase II NCI trial manufacturing is given the okay by the FDA, Dr. Rosenberg will begin patient enrollment. The same manufacturing improvements will be used in the ZIOP controlled TCR-T trial at MD Anderson that will begin as soon as manufacturing receives the FDA go ahead.

After the recent offering, the company has $177 million in cash which is projected to last until 2022.

2020 is a very important year as the company needs to deliver data to show that they have the potential to deliver third- or next-generation cell therapy technology that can deliver life changing T-cell therapy for solid tumors and at a price that will make the therapy broadly available.

The MD Anderson relationship only gets better for Ziopharm as they will start a CD19 CAR-T trial in Q2 there in which they will incorporate their cutting edge manufacturing process that can infuse a patient as soon as the day after gene transfer on site.The company is also establishing their own cGMP manufacturing infrastructure at MD Anderson.

Story continues

The company has three potential upcoming catalysts as will have three controlled IL-12 data readouts in Q2, two in combo with PDL-1s and one monotherapy.

We also could get a press release from the NCI trial Phase II trial whenever Rosenberg thinks the therapy is working, which could be as soon as just weeks after the initial treatment. That will be a major positive event that we believe will be the inflection point for the company and the stock.

See also: Market History and Waterfall Declines

With T-cell therapy the theory is that you know pretty fast whether the T-cells are activated or not. In our view, the stock has multiple upcoming potential catalysts and the stock is significantly undervalued at current levels.

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Ziopharm- Catalysts in T-Cell Therapy - Yahoo Finance

WASHINGTON, April 2, 2020 /PRNewswire/ -- An article published in Experimental Biology and Medicine (Volume 245, Issue 6, March 2020) (https://journals.sagepub.com/doi/pdf/10.1177/1535370220910690) examines the safety of stem cell therapy for the treatment of colon cancer.The study, led by Dr. J. Liu in the State Key Laboratory of Bioreactor Engineering and Shanghai Key Laboratory of New Drug Design at the East China University of Science and Technology in Shanghai (China), reports that mesenchymal stem cells from a variety of sources promote the growth and metastasis of colon cancer cells in an animal model.

Mesenchymal stem (MSCs), a category of adult stem cells, are being evaluated as therapy for numerous cancers.MSCs are excellent carriers for tumor treatment because they migrate to tumor tissues, can be genetically modified to secrete anticancer molecules and do not elicit immune responses.Clinical trials have shown that MSCs carrying modified genes can be used to treat colon cancer as well as ulcerative colitis. However, some studies have demonstrated MSCs can differentiate into cancer-associated fibroblasts and promote tumor growth.Therefore, additional studies are needed to evaluate the safety of MSCs for targeted treatment of colon cancer.

In the current study, Dr. Liu and colleagues examined the effects of mesenchymal stem cells (MSCs) from three sources (bone marrow, adipose and placenta) on colon cancer cells.MSCs from all three sources promoted tumor growth and metastasis in vivo. In vitro studies demonstrated that MSCs promote colon cancer cell stemness and epithelial to mesenchymal transition, which would enhance tumor growth and metastasis respectively.Finally, the detrimental effects of MSCs could be reversed by blocking IL-8 signaling pathways. Dr. Ma, co-author on the study, said that "Mesenchymal stem cells have a dual role: promoting and/or suppressing cancer. Which effect is dominant depends on the type of tumor cell, the tissue source of the MSC and the interaction between the MSC and the cancer cell. This is the major issue in the clinical application research of MSCs, and additional preclinical experimental data will be needed to evaluate the safety of MSCs for colon cancer treatment."

Dr. Steven R. Goodman, Editor-in-Chief of Experimental Biology & Medicine, said: "Lui and colleagues have performed elegant studies on the impact of mesenchymal stem cells (MSCs), from various sources, upon the proliferation, stemness and metastasis of colon cancer stem cells (CSCs) in vitro and in vivo. They further demonstrate that IL-8 stimulates the interaction between colon CSCs and MSCs, and activates the MAPK signaling pathway in colon CSCs.This provides a basis for the further study of MSCs as a biologic therapy for colon cancer."

Experimental Biology and Medicine is a global journal dedicated to the publication of multidisciplinary and interdisciplinary research in the biomedical sciences. The journal was first established in 1903. Experimental Biology and Medicine is the journal of the Society of Experimental Biology and Medicine. To learn about the benefits of society membership, visit http://www.sebm.org. For anyone interested in publishing in the journal, please visit http://ebm.sagepub.com.

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Stem Cell Therapy for Colon Cancer - Yahoo Finance

The scientists who developed Dolly the sheep in 1996 is, currently, developing a new therapy to treat the deadly coronavirus infection using immune cells from young and healthy volunteers.

Using Your Bodys Natural Defenses against the Virus

Such an immunity-building cell transfusion has already been used to treat cancer. The researchers believe that it will be effective against the COVID-19 as well.

One of the key challenges of fighting viral infection is to develop something that is going to attack the infected cells and not the normal cells. So the solution that we came up with was to look at the body's natural defenses to viral infection. In patients who have successfully fought a viral infection, they have expanded their own immune system and that persists after that to stop them becoming infected again," the Mirror quoted Dr. Brian Kelly, the studys senior strategic medical adviser.

According to the researchers, their treatment sees donor T-cells differ from normal immune cells as they do not identify invaders in the body based on an alien protrusion on the cells surfaces but detects the unusual metabolism of the viruses.

When the donor cells detect a virus, it starts to destroy alongside giving signals to the rest of the immune system that an alien intrusion has occurred and that it needs to be eradicated. Such an approach might be effective even when the virus is mutated or returned to a body.

Experimental Stem cell therapy by Celularity

Derived from human placentas, this new stem cell therapy will undergo early testing in COVID-19 patients. Although it hasnt yet been used on any patientwith the coronavirus symptoms, it has caught the attention of U.S. President Trumps personal lawyer Rudy Giuliani, who had tweeted that it had real potential.

There is no proven treatment yet for COVID-19 and health experts worldwide have been carrying out several experimental approaches including the old malarial drug, anti-HIV drug, and the Japanese flu drug.

Celilarity has announced its early-stage trial for COVID-19 treatment known as Cynk-001 and has described the development as the first FDA-approved COVID-19 cell therapy. It is important to note that the agency has only approved the treatment to be used in a clinical trial and not to be widely prescribed to patients.

The objective here is preventative. If the timing of giving this can prevent those patients who have early disease from progressing to the more serious, life-threatening form, it could be a very, very useful tool, The New York Times quoted Dr. Robert Hariri, Celularitys founder and chief executive.

Researchers are racing to develop treatments for the coronavirus as hospitals battle its spread Photo: AFP / STR

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Coronavirus Update: Cell Therapy Reportedly Appears Promising To Beat COVID-19 - International Business Times

XconomySan Diego

Parkinsons disease patients experience progressive breakdown of their neurons, cells essential to movement and mental function. Aspen Neuroscience, a biotech working to develop a therapy that would reprogram those patients own living cells in ways that would allow the cells to reconstruct their damaged neural networks, has raised $70 million in a Series A financing round.

The 33-person company managed to land the funding amid tenuous economic times by shifting into high gear directly after the JP Morgan Healthcare Conference in San Francisco in January, the same month the first COVID-19 cases in the US were reported, CEO Howard Federoff (pictured) said in a phone interview.

I have a better understanding of how these emerging infections might impact on infrastructure, he said. The whole team came together in such a tightly aligned manner that there was no opportunity that we missed to be able to interact, accelerate, and, where possible, get investors on board.

OrbiMed led the funding for the San Diego-based company, which raised a seed round of $6.5 million late last year.

Aspen is advancing two product candidates to treat Parkinsons patients. The companys lead program, ANPD001, is in preclinical development as a treatment for sporadic, or spontaneous, forms of Parkinsons diseasethe most common type.

The companys second product candidate, ANPD002, combines autologous neuron therapy with gene editing to treat inherited forms of the condition. About 15 percent of people with Parkinsons disease have a family history of the disorder, and a number of genes have been identified as causing it if mutated.

Parkinsons affects more than 1 million people in North America and more than 4 million people worldwide, according to the National Institutes of Health. In the United States, the disease occurs in about 13 per 100,000 people, and about 60,000 new cases are identified yearly.

Aspen says the fresh cash will allow it to finish preclinical work on ANPD001 and, with the FDAs blessing, move it through Phase 1 testing. The financing will also allow it to recruit and screen Parkinsons patients for trial enrollment, make the cells they will need for those patients, and get enough data to advance into a Phase 2 study. Some of the money will also support ongoing research and development, including the ANPD002 program and R&D into possible treatments for neuroinflammatory disorders.

Available treatments for Parkinsons disease focus primarily on restoring dopamine, the chemical produced by the cells that are degraded as the disorder progresses. Aspen aims to reprogram cells collected from a patient into immature cells, or iPSCs, further engineer them to become predisposed to mature into neurons, then return those cells to the brain. Using a cell therapy product developed autologously, or from the patients own cells, would avoid the risk of an immune system reaction that could occur with the use of donor cells.

Aspen says it employs proprietary genomics tools that use machine learning to reduce the time and cost of the manufacturing process needed to produce its personalized cell therapies.

The work that underpins the company was conducted in the lab of Jeanne Loring, the companys co-founder and chief scientific officer, who also directs the Center for Regenerative Medicine at the Scripps Research Institute.

Led by OrbiMed, Aspens Series A round included investment from ARCH Venture Partners, Frazier Healthcare Partners, Domain Associates, Bill Mariss Section 32, and Sam Altman, the former president of Y Combinator and CEO of OpenAI.

As part of the deal, OrbiMeds Peter Thompson and ARCH Ventures Tom Daniel join its board of directors. Aspen also announced that Faheem Hasnain, a veteran biotech executive who most recently co-founded San Diegos Gossamer Bio (NASDAQ: GOSS), has joined the board as its chairman.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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Aspen Neuro Adds $70M to Test Parkinson's Cell Therapy in the Clinic - Xconomy

Shares of Fate Therapeutics were up more than 21% in premarket trading after the company announced a multi-billion dollar deal with Janssen Biotech to develop cell therapies for the treatment of cancer.

The partnership will leverage San Diego-based Fate Therapeutics induced pluripotent stem cell (iPSC) product platform and Janssens proprietary tumor-targeting antigen binders to create novel CAR NK and CAR T-Cell product candidates that will be developed against up to four tumor-associated antigens for hematologic malignancies and solid tumors. Fate will advance the different candidates through preclinical studies to the point of the filing of an Investigational New Drug Application with the U.S. Food and Drug Administration. When that time comes, Janssen will have the right to exercise its option for an exclusive license for the development and commercialization of collaboration candidates targeting the tumor-associated antigens.

Fates iPSC platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses, according to the company. The companys approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass-produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment, the company said.

Under terms of the agreement, Fate will receive $50 million in upfront cash, as well as a $50 million equity investment by Janssen. Janssen will also cover the funding costs of the R&D of the collaboration candidates. Once Janssen takes over the program, Fate will then be eligible to receive payments of up to $1.8 billion upon the achievement of development and regulatory milestones and up to $1.2 billion upon the achievement of commercial milestones. Fate will also be eligible to receive double-digit royalties on worldwide commercial sales of products targeting the antigens. Additionally, Fate has the right to co-commercialize the candidates should they be approved and share equally in profits and losses, the company said in its announcement.

Scott Wolchko, president and chief executive officer of Fate Therapeutics, touted the partnership with Janssen. He said the collaboration combines Janssens deep domain expertise in oncology, along with their proprietary technologies for targeting and binding certain tumors with Fates iPSC product platform to develop novel off-the-shelf CAR NK and T-cell cancer immunotherapies.

The collaboration strengthens our financial and operating position through a focused effort of developing cell-based cancer immunotherapies utilizing Janssens proprietary antigen-binding domains while enabling us to continue to exploit our deep pipeline of wholly-owned product candidates and further develop our off-the-shelf, iPSC-derived cell-based immunotherapies, Wolchko said in a statement.

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Fate Therapeutics Strikes Multi-Billion Dollar Cell Therapy Deal with Janssen - BioSpace