Category Archives: Cell Therapy

The FDA cleared the investigational new drug (IND) application for the use of CYNK-001 in adults with coronavirus disease 2019 (COVID-19), according to Celularity, the agents manufacturer.1

The company also announced that it will immediately begin a phase I/II clinical study of CYNK-001 in collaboration with Sorrento Therapeutics, which will include up to 86 patients with COVID-19.

We are confident that our strategic relationship with Sorrento will help assure our ability to meet the scale requirements for our efforts in COVID-19, Robert Hariri, MD, PhD, CEO for Celularity, said in a press release.

Individuals who are enrolled in the trial will receive infusions of natural killer (NK) cells, which the company believes could kill cells infected with the virus and address the resulting inflammation caused by the immune system.

Infectious Disease Research Institute (IDRI), a nonprofit based in Seattle, says it is coordinating the trial, which will take place at medical centers in the US.2

To date, efforts to treat COVID-19 cases have been primarily focused on antiviral medications, Corey Casper, MD, MPH, clinical professor of global health and medicine at the University of Washington and interim president and CEO at IDRI, said in a press 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.

CYNK-001 is the only cryopreserved allogeneic, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells. The company suggests that using such cells eliminates the risk of an immune system reaction that other kinds of donor cells can cause.

The therapy is already being tested in patients with acute myeloid leukemia and multiple myeloma in early-stage studies, and as a potential treatment option for various solid tumors.

In January, the FDA cleared Celularitys investigational new drug (IND) application for CYNK-001 in patients with glioblastoma multiforme (GBM).3The clinical trial is anticipated to be the first clinical trial in the US to investigate intratumoral administration of an allogeneic NK cell therapy.

The FDA clearance of our IND validates the versatility of our allogeneic, off-the-shelf, placental-derived NK cell therapy platform to generate novel clinical candidates against a broad range of devastating cancers, Hariri said in a press release. This IND represents a significant step toward a potential immunotherapy option that is more accessible and tolerable to patients with glioblastoma multiforme.

According to the company, they expect to initiate first-in-human clinical testing of CYNK-001 administered either intravenously or intratumorally. The study is anticipated to evaluate the safety, feasibility, and tolerability of multiple doses of CYNK-001 in patients with relapsed GBM.

Nonclinical safety and efficacy data were presented at the 2019 Society for Neuro-Oncology (SNO) Annual meeting, demonstrating that a single administration of CYNK-001 was well-tolerated and showed enhancedin vivoanti-tumor activity against GBM.

References:

1. Sorrento to Provide Manufacturing Support to Celularity as CYNK-001 NK Cell Trial for COVID-19 Begins Enrolling Patients [news release]. San Diego, CA. Published April 2, 2020. globenewswire.com/news-release/2020/04/02/2010998/0/en/SORRENTO-TO-PROVIDE-MANUFACTURING-SUPPORT-TO-CELULARITY-AS-CYNK-001-NK-CELL-TRIAL-FOR-COVID-19-BEGINS-ENROLLING-PATIENTS.html. Accessed April 2, 2020.

2. Xconomy National. Celularity to Test Natural Killer Cell Therapy for Cancer Against COVID. Xconomy National website. Published April 2, 2020. xconomy.com/national/2020/04/02/celularity-to-test-natural-killer-cell-therapy-for-cancer-against-covid/. Accessed April 2, 2020.

3. Celularity Announces FDA Clearance of Landmark IND for CYNK-001, an Allogeneic, Off-the-Shelf Cyropreserved NK Cell Therapy [news release]. Warren, NJ. Published January 22, 2020. businesswire.com/news/home/20200122005061/en/Celularity-Announces-FDA-Clearance-Landmark-IND-CYNK-001. Accessed April 2, 2020.

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FDA Accepts IND for NK Cell Therapy CYNK-001 to Treat Patients with COVID-19 - Cancer Network

Executive Summary

Introduction

Chimeric Antigen Receptor T-cell therapyreferred to as CAR-T in this pieceis a form of immunotherapy that utilizes the patients own immune system to attack cancer and is considered the fifth pillar of cancer therapies.[1] By modifying the patients T-cells with an engineered, synthetic receptor specific to their cancer, the patients body becomes capable of recognizing and attacking the tumor while leaving normal cells alone.

One of the first applications of CAR-T in adults is Yescarta, which treats relapsed large B-cell lymphoma. Fairly quickly, however, CAR-T has been investigatedand is under developmentfor the treatment of multiple myeloma, a cancer disproportionately affecting Medicare patients.[2]

CAR-T promises to save lives while limiting the side effects of more conventional cancer therapies, but the inadequate reimbursement of such a personalized treatment approach is proving to be prohibitive, particularly for Medicare patients. Because hospitals do not receive full reimbursement for the cost of CAR-T, they are reluctant to offer or prioritize it. To solve this problem, Medicare can create a new billing category specifically for CAR-T, although this category will require some exceptions due to the overwhelming proportion of the total cost of treatment being the drug product itself.

Current Status of CAR-T Medicare Reimbursement

Acute care hospitals are reimbursed for Medicare patients through the Inpatient Prospective Payment System (IPPS), which bundles payments for hundreds of clusters of conditions according Medical Severity Diagnosis-Related Groups (MS-DRG, or DRG for short). IPPS typically works in the following way. A Medicare patient is admitted to the hospital and placed into a DRG based on their principal diagnosis. From there, a series of adjustments are performed based on their geographic area, complexity of the case, and hospital-specific characteristics. For more information on determining payment rates under the IPPS, please see this American Action Forum primer.

The process of CAR-T reimbursement is typical of any case under the IPPS but requires a fair bit of patchwork to reach the current but insufficient rates, yielding some degree of payment uncertainty for hospitals. If a patient is to receive CAR-T therapy, they are grouped not into their own DRG but rather designated under DRG 016: Autologous Bone Marrow Transplant with Complications or Major Complications (CC/MCC) or T-Cell Immunotherapy because it is the closest DRG that the Centers for Medicare and Medicaid Services (CMS) has to CAR-T in terms of clinical characteristics and the medical resources used for treatment.[3] From there, a new technology add-on payment (NTAP) for the CAR-T therapy is made along with an additional outlier case payment due to its high cost.

Issues with Current Reimbursement Scheme

The problems with the current reimbursement system are three-fold. First, current levels of reimbursement are inadequate for hospitals, leading to monetary losses for each treatment. Second, the ability to extend the NTAP for CAR-T treatments is expiring in April, 2020 andbecause it has been three years since CAR-T received approval by the Food and Drug Administration (FDA)CMS does not have the legal authority to provide a one-year temporary extension.[4]

Hospital Reimbursement

The list price CAR-T therapy runs at about $373,000.[5] In fiscal year 2020, the average CAR-T case payment under the IPPS is about $353,000 which includes the NTAP, outlier payments, and all hospital-specific adjustments. Once the operating and capital payments are estimated and factored in, the average total DRG payment come short of covering the full price of treatment and administration by about $50,000 as seen in Table 1. This results in serious losses for the hospital for each CAR-T treatment and impedes the ability of patients to receive this life-changing therapy.

*These numbers are averages and may fluctuate based on hospital and clinical-specific characteristics. More information on payment rate calculations can be found here.

In addition to the current losses of about $50,000 per case, in fiscal year 2021should a new DRG not be created and CAR-T remain in DRG 16the expiration of the NTAP will result in the average payment under the IPPS dropping to around $280,000. As a result, hospitals will face additional losses of about $73,000.

NTAP Extension

NTAPs are designed to be a temporary bridge between new technologies and more stable payment options through a new DRG. As stated above, however, the NTAP for CAR-T is expiring and incapable of being extended due to it passing the three-year FDA approval period. Typically, the process for transitioning these NTAPs to stand alone DRGs is straightforward, but in this case transition is complicated by the fact that most cases under DRG 16 are standard autologous bone marrow transplants, not CAR-T cases. Since DRG payment rates are set based on data from prior years, the small proportion of CAR-T cases paid under DRG 16 do not shift the payment up significantly from year-to-year, leading to an inability of DRG 16 payment rates to catch up to the reimbursement levels needed for CAR-T. All of these issues taken together logically demonstrate the need for a permanent split of CAR-T reimbursement from DRG 16.

Solution

The straightforward solution to this issue of CAR-T therapy reimbursement is to create a new, specific cell line therapy DRG, separating CAR-T from DRG 16 entirely. This new designation would significantly boost hospital reimbursement without requiring an NTAP, ensuring hospitals can more favorably consider CAR-T in the treatment line, all to the substantial benefit of the patient. Establishing a new DRG would require CMS to consider, however, that the principal cost of treatment is the therapy itself and neither operational nor capital-related costsfactors that affect the reimbursement, as noted below. CMS would have to accommodate for that fact in the final rule for 2021.

CMS Authority

CMS has the authority to establish new MS-DRG codes for the following fiscal year, tying them to specific codes in the International Classification of Diseases, 10th Revision, Procedure Coding System (ICD-10-PCS) and/or the ICD-10, Clinical Modification (ICD-10-CM).[6]

Tying the New DRG to the ICD-10

In the case of CAR-T therapy, a new DRG should be established using the ICD-10-PCS codes XW033C3: Introduction of Engineered Autologous Chimeric Antigen Receptor T-cell Immunotherapy into Peripheral Vein, Percutaneous Approach, New Technology Group 3 and XW043C3: Introduction of Engineered Autologous Chimeric Antigen Receptor T-cell Immunotherapy into Central Vein, Percutaneous Approach, New Technology Group 3. Using these two codes would cover all billable procedures involving CAR-T therapy, thus divorcing it entirely from DRG 016: Autologous Bone Marrow Transplant with Complications or Major Complications (CC/MCC) or T-Cell Immunotherapy. DRG 016 would also need to be modified to prevent overlap between the two.

Other Considerations

To reiterate, the primary cost of CAR-T therapy is not its administration within a hospital system, but the cost of the treatment itself. The DRG payment determination for CAR-T must take this high cost into account by establishing a sufficient adjusted base payment rate. As a result, IPPS mechanisms such as the wage index will have limited utility for tailoring the final payment to the specific patient or hospital. Due to this fact, the wage index should either not be applied to the new DRG, or the DRG should be established such that the wage index minimally impacts reimbursement.

With the cost of CAR-T in mind, the other major consideration is the impact of including clinical trials on the perceived cost per treatment. Since the cost of drugs are not factored into a case treated through a clinical trial, including clinical trials involving CAR-T in payment rate determination skews the true cost. Payments for CAR-T must be set solely for non-clinical trial cases in order to accurately capture the price. While this would be a deviation from the normal payment rate-setting process under the IPPS, the high cost of CAR-T necessitates CMS make an exception in order to ensure reimbursement is adequate and thus patients more readily receive it.

Conclusion

CAR- T therapy is among the most innovative and astounding medical advancements in the past couple of decades. Cancer patients both young and old stand to have their lives saved after a single treatment course, circumventing some if not much of the suffering imposed by multiple rounds of chemotherapy and radiation therapy. Seeing as the technology is becoming more established and applicable by the year, it is appropriate to guarantee adequate provider reimbursement for this therapy under the IPPS through the establishment of its own MS-DRG.

[1] https://www.cancer.gov/news-events/cancer-currents-blog/2017/car-t-cell-multiple-myeloma

[2] https://www.cancer.gov/news-events/cancer-currents-blog/2017/car-t-cell-multiple-myeloma

[3] https://www.cms.gov/icd10m/version37-fullcode-cms/fullcode_cms/P0044.html

[4] https://www.healthaffairs.org/doi/full/10.1377/hlthaff.27.6.1632

[5] https://www.ashclinicalnews.org/online-exclusives/cms-proposes-coverage-car-t-cell-therapies/

[6] https://www.cms.gov/Medicare/Medicare-Fee-for-Service-Payment/AcuteInpatientPPS/MS-DRG-Classifications-and-Software

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A Path Forward for CAR-T Therapy Reimbursement Under the IPPS - American Action Forum

NEW DELHI :Researchers at Queen's 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 O'Kane, both researchers from the Wellcome-Wolfson Institute for Experimental Medicine at Queen's 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 O'Kane 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, Queen's 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.

"We're 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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Britain starts clinical trial of cell therapy treatment for Covid-19 patients - Livemint

HAMBURG, GERMANY, AND TORONTO, ON / ACCESSWIRE / April 2, 2020 / Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) and the innovative biotechnology company panCELLa Inc. announced today that the companies have entered into a licensing and investment agreement.

Under the terms of the agreement, Evotec will receive a non-exclusive licence to access panCELLa's proprietary iPS cell lines "iACT Stealth Cells(TM)", which are genetically modified to prevent immune rejection of derived cell therapy products ("cloaking"). Furthermore, Evotec will also have access to a new-generation cloaking technology known as hypoimmunogenic cells. In addition, the "FailSafe(TM)" mechanism effectively addresses a key challenge in iPSC-based cell therapy, potential tumour formation by residual undifferentiated cells.

Using the cell lines, Evotec will be able to develop iPSC-based, off-the-shelf cell therapies with long-lasting efficacy that can be safely administered to a broad population of patients without the use of medication to supress the patients' immune system. With a growing portfolio of iPSC-based cell therapy projects at Evotec, access to research as well as GMP-grade iPSC lines modified with one or both of the panCELLa technologies significantly accelerates Evotec's cell therapy discovery and development efforts. Modified iPSC lines will be available for the development of cell therapy approaches across a broad range of indications by Evotec and potential partners. Furthermore, Evotec has made an investment to take a minority stake in panCELLa and has nominated Dr Andreas Scheel to join panCELLa's supervisory board.

Dr Cord Dohrmann, Chief Scientific Officer of Evotec, commented: "Cell therapies hold enormous potential as truly regenerative or curative approaches for a broad range of different diseases with significant medical need. Integrating panCELLa's technology and cell lines into our ongoing proprietary research and development efforts strengthens Evotec's position in cell therapy. It is our goal to provide safe highly-effective cell therapy products to as many patients as possible. In addition to small molecules and biologics, cell therapy will become yet another major pillar of Evotec's multimodality discovery and development platform."

Mahendra Rao, MD, PhD, CEO at panCELLa, added: "We welcome the partnership with Evotec. Evotec's widely recognised expertise and existing portfolio of iPSC-related technology platforms will allow panCELLa to rapidly advance its own therapeutic interests in NK cell therapy, pancreatic islet production and iPSC-derived MSC platform, in addition to enabling panCELLa to make its platform technologies widely available. I believe that the investment by Evotec in our company is a strong validation of the leading role of panCELLa in the field of regenerative medicine and in the utility of its platform technologies. We welcome Dr Andreas Scheel to our Board."

No financial details of the agreement were disclosed.

About Evotec and iPSCInduced pluripotent stem cells (also known as iPS cells or iPSCs) are a type of pluripotent stem cell that can be generated directly from adult cells. The iPSC technology was pioneered by Shinya Yamanaka's lab in Kyoto, Japan, who showed in 2006 that the introduction of four specific genes encoding transcription factors could convert adult cells into pluripotent stem cells. He was awarded the 2012 Nobel Prize along with Sir John Gurdon "for the discovery that mature cells can be reprogrammed to become pluripotent". Pluripotent stem cells hold great promise in the field of regenerative medicine. Because they can propagate indefinitely, as well as give rise to every other cell type in the body (such as neurons, heart, pancreatic and liver cells), they represent a single source of cells that could be used to replace those lost to damage or disease.

Evotec has built an industrialised iPSC infrastructure that represents one of the largest and most sophisticated iPSC platforms in the industry. Evotec's iPSC platform has been developed over the last years with the goal to industrialise iPSC-based drug screening in terms of throughput, reproducibility and robustness to reach the highest industrial standards, and to use iPSC-based cells in cell therapy approaches via the Company's proprietary EVOcells platform.

Story continues

About cell therapy and panCELLa's FailSafe(TM) iPSC technologyCell therapy, one of the most promising regenerative medicine approaches, replaces a patient's missing or broken cells with functioning cells from a range of different sources, either from a donor, from the patient's own material, or from stem cells. The advent of induced pluripotent stem cells ("iPSC") has opened up stem cells as an almost unlimited source of consistent-quality material for such cell therapies. At the same time, differentiating cell therapies from a single validated source circumvents critical risks of contamination associated with administering both donor and patient cell material.

However, the patient's immune system will treat such iPSC-based transplants as "foreign" and use the body's immune system to counteract the therapy, thus undermining its long-term efficacy. While organ transplants require an often lifelong regimen of immunosuppressants, iPSC-derived cells used for cell therapies can be cloaked to make them undetectable by the patient's immune system, thus avoiding rejection and enabling effective long-term relief of the patient's symptoms.

To increase the safety of such iPSC-derived cell products, panCELLa's proprietary FailSafe(TM) technology is able to inactivate any iPSC-derived proliferating cell before and after transplantation through the use of a readily available anti-infective medication. FailSafe(TM) is the only quantifiable "safety switch" on the market which is expected to be critical for regulators, clinicians and patients to make informed decisions when evaluating treatment options.

ABOUT PANCELLA INC.Incorporated in August 2015, panCELLa (www.pancella.com) was founded by Dr Andras Nagy and Dr Armand Keating based on Dr Nagy's ground-breaking work in the area of stem cell research. Through panCELLa, Drs Keating and Nagy are seeking to create an effective cell therapy derived from stem cells, which are modified to provide a sufficient and very high level of safety before and after the cells are introduced to the patient. panCELLa serves those companies developing products from stem cells. panCELLa seeks to create universal "off the shelf" FailSafe(TM) Cells and to assist pharmaceutical and biotechnology sectors to achieve such with their own cell lines. Targeted medical applications include deadly, debilitating, or aggressive diseases requiring immediate treatment where there is no time to cultivate a customized stem cell treatment from the patient (i.e. cancer, cardiac infarct, diabetes, stroke and spinal cord injury).

ABOUT EVOTEC SEEvotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies, academics, patient advocacy groups and venture capitalists. We operate worldwide and our more than 3,000 employees provide the highest quality stand-alone and integrated drug discovery and development solutions. We cover all activities from target-to-clinic to meet the industry's need for innovation and efficiency in drug discovery and development (EVT Execute). The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuronal diseases, diabetes and complications of diabetes, pain and inflammation, oncology, infectious diseases, respiratory diseases, fibrosis, rare diseases and women's health. On this basis, Evotec has built a broad and deep pipeline of approx. 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate). Evotec has established multiple long-term alliances with partners including Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CHDI, Novartis, Novo Nordisk, Pfizer, Sanofi, Takeda, UCB and others. For additional information please go to http://www.evotec.com and follow us on Twitter @Evotec.

FORWARD LOOKING STATEMENTSInformation set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of Evotec as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.

Contact Evotec SE:Gabriele Hansen, SVP Corporate Communications, Marketing & Investor Relations, Phone: +49.(0)40.56081-255, gabriele.hansen@evotec.com

SOURCE: Evotec AG

View source version on accesswire.com: https://www.accesswire.com/583603/Evotec-Expands-Its-IPSC-Based-Cell-Therapy-Platform-Evocells-Through-Licensing-Agreement-with-panCella

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Evotec Expands Its IPSC-Based Cell Therapy Platform Evocells Through Licensing Agreement with panCella - Yahoo Finance

CAMBRIDGE, Mass., April 03, 2020 (GLOBE NEWSWIRE) -- TCR2 Therapeutics Inc. (TCRR), a clinical-stage immunotherapy company developing the next generation of novel T cell therapies for patients suffering from cancer, today announced that Alfonso Quints Cardama, M.D., Chief Medical Officer of TCR2 Therapeutics, will present an update on company progress during the panel Emergence of TCRs and Next-Generation Therapies at the Goldman Sachs Cell Therapy Day on April 6, 2020 at 2:00pm ET using a virtual platform.

About TCR2 Therapeutics

TCR2Therapeutics Inc.is a clinical-stage immunotherapy company developing the next generation of novel Tcell therapies for patients suffering from cancer.TCR2sproprietary T cell receptor (TCR) Fusion Construct Tcells (TRuC-T cells) specifically recognize and kill cancer cells by harnessing signaling from the entire TCR, independent ofhuman leukocyte antigens (HLA). In preclinical studies, TRuC-T cells have demonstrated superior anti-tumor activity compared to chimeric antigen receptor T cells (CAR-T cells), while exhibiting lower levels of cytokine release. The Companys lead TRuC-T cell product candidate targeting solid tumors, TC-210, is currently being studied in a Phase 1/2 clinical trial to treat patients with mesothelin-positive non-small cell lung cancer (NSCLC), ovarian cancer, malignant pleural/peritoneal mesothelioma, and cholangiocarcinoma. The Companys lead TRuC-T cell product candidate targeting hematological malignancies, TC-110, is currently being studied in a Phase 1/2 clinical trial to treat patients with CD19-positive adult acute lymphoblastic leukemia (aALL) and with aggressive or indolent non-Hodgkin lymphoma (NHL). For more information about TCR2, please visitwww.tcr2.com.

Investor and Media Contact:

Carl MauchDirector, Investor Relations and Corporate Communications(617) 949-5667carl.mauch@tcr2.com

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TCR Therapeutics to Present at the Goldman Sachs Cell Therapy Day - Yahoo Finance

Seeking European Commission Funding to Fight Against Corona

Stempeutics Research, a group company of Manipal Education and Medical Group (MEMG), recently announced that it has partnered with Global Consortium of cell therapy companies seeking European Commission Funding to Fight Against Corona! (FAC!). Under this partnership, Stempeutics will export its stem cell product Stempeucel (subject to regulatory approvals) for treating critically ill COVID-19 patients with lung disease. First the product will be clinically tested and upon successful outcomes, it intends to export the product on a regular basis. In this connection it is signing up an alliance with Educell, Slovenia.

Currently, no specific drugs or vaccines are available to cure the patients with COVID-19 infection. Mortality in COVID-19 infected patients with the inflammatory lung condition ARDS (Acute Respiratory Distress Syndrome)is reported to approach 50 per cent, and is associated with older age, co-morbidities such as diabetes, cardiovascular disease, COPD (chronic obstructive pulmonary disease), higher disease severity, and elevated markers of inflammation. Current therapeutic interventions (with the exception of ventilators / respirators which are in very short supply) do not appear to be improving in-hospital survival. Hence, there is a large unmet need for a safe and effective treatment for COVID-19 infected patients, especially in severe cases. A promising new therapy for the ARDS, the terminal stage of COVID-19, using MSCs can quickly (2-4 days) reduce inflammation of the lung tissue, and allow patients to more quickly come off of the ventilatory support and hopefully fully recover with less significant lung damage.

Stempeucel is an allogeneic, off the shelf, pooled mesenchymal stromal cells having anti- inflammatory and immune-modulatory properties which prevents the over activation of the immune system. Stempeucel product exhibits a wide range of potent therapeutic properties. The product exhibits potent immunomodulatory and anti-inflammatory properties which could help in reducing the inflammation caused due to the cytokine storm elicited by the bodys immune cells in response to SARS-CoV-2 (COVID-19) related infection in the lungs. Also, the growth factor, Angiopoietin-1 (Ang-1) is effective in reducing alveolar epithelium permeability in the lung. Hence it is envisaged, Stempeucel will reduce the fatal symptoms of COVID 19 induced pneumonia and its progression to ARDS.

Commenting on this initiative, Dr Miomir Knezevic, Leader, Global Consortium and Founder, Educell said, We are happy to partner with Stempeutics since its product Stempeucel is already designated as an ATMP [1] in Europe and also Stempeucel technology has been patented in many countries in Europe. Stempeutics manufacturing process is scalable and the product is affordable which are key to meet the demands of COVID-19 patients.

BN Manohar, CEO, Stempeutics said, From the clinical data using Stempeucel in different clinical trials in other indications it may be postulated that Stempeucel has the potential capability for treating COVID-19 infection. Together with the safety profile observed from DCGI approved clinical trials involving more than 350 patients injected with Stempeucel by different routes of injection, this therapy may help in mitigating the lung tissue damaging effects of COVID-19 infection.

Dr Stephen Minger, Scientific Advisor, Global Consortium and ex Global Director of R&D, Cell Technologies GE Healthcare added, The most severely affected CV-2 infected patients will often go on to develop ARDS which necessitates assisted ventilation to preserve breathing and lung function. Moreover, many ARDS patients will also experience an acute but severe life-threatening inflammatory response (cytokine storm) which can result in long-term damage to lung tissue and lung function. Treating ARDS patients with allogeneic expanded bone marrow derived MSCs could alleviate and ameliorate lung inflammation and compromised lung function and significantly reduce the time required for patients to be ventilated.

Dr Raviraja NS, Sr Director, Business Development and Innovation, Stempeutics, said, Given the severe shortage of ventilators in the world, and the high mortality rate of patients who develop ARDS (approx. 50 per cent), the clinical use of MSCs in COVID-19 ADRS patients could drastically impact on the healthcare burden currently occurring due to very large patient numbers, limited equipment and overworked medical personnel.

Reference:

[1] ATMP is Advanced Therapy Medicinal Product regulated by EU Act December 7, 2007.

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Stempeutics partners with Global Consortium of cell therapy companies - Express Healthcare

The latest headlines in your inbox

A group of scientists are reportedly in talks with the Government on a potential treatment for the coronavirus using immune cells from young and healthy volunteers.

Researchers from TC Biopharm near Glasgow, responsiblefor cloning Dolly the Sheep,have used the new therapy which uses immunity-building cell transfusions to successfully treat cancer.

They are now hoping it will also work against the coronavirus, and are in talks with the Government to trial the therapy for that purpose, the Daily Telegraph reports.

It is hoped the treatment will be made available in NHS hospitals by July.

Dr Brian Kelly, senior strategic medical adviser to TC Biopharm, told the paper: 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 bodys natural defences 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 donor T-cells differ from normal immune cells as they do not identify invaders in the body based on alien protrusions on the surface of cells, but by detecting the unusual metabolism of viruses.

When the donor cells do detect a virus, they begin to destroy while also signalling it to the rest of the immune system as an alien intrusion requiring eradication.

Dr Kelly said with this approach, even if the virus mutated and returned to a body, the infusion exercise could be repeated and would still work.

Listen to The Leader: Coronavirus Daily podcast

TC Biopharm was founded by Angela Scott, who was part of the team who cloned Dolly the Sheep in Edinburgh in 1996.

Additional reporting by Press Association.

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Cell therapy treatment mooted as potential way to beat coronavirus - Evening Standard

Lev Gerlovin, vice president in the Life Sciences Practice at Charles River Associates (CRA), and Pascale Diesel former vice president in the Life Sciences Practice at CRA, discuss how pharma companies are changing their strategies when it comes to cell and gene therapies.

In recent years, drug development has been transitioning from a one pill fits all approach to a focus on more targeted and personalised therapies, including many cell and gene therapies. These innovative treatments often bring the promise of high efficacy and even curative benefit and, in many cases, are options for patients with diseases that previously had limited or no treatments available. With several cell and gene therapies now approved and many more advancing toward late-stage clinical development, patient communities and healthcare systems are rapidly recognising their potential benefits, whilst pharmaceutical companies are considering opportunities including mergers, acquisitions, licensing agreements, and partnerships. The deal-making landscape for cell and gene therapies is already very active and positioned to grow exponentially in the years ahead.

While the pace of deal-making is accelerating, the unique qualities of cell and gene therapies are also requiring dealmakers on both sides to consider some innovative and previously untried strategies designed to optimise returns and reduce risk. They are structuring deals to address many factors, including the lack of commercial benchmarks for these products and limited long-term safety and efficacy data often associated with cell and gene therapies. We conducted an analysis of more than 30 deals executed in the cell and gene therapy sector recently and compared them to deals executed for monoclonal antibodies (mAbs) between 1999-2013. Emerging cell and gene therapies are often considered magic bullets in the treatment of many serious diseases a claim that was applied to many mAbs when they were first introduced to the market about 20 to 25 years ago and the level of interest in deal-making for these therapies is a reflection of the level of optimism companies have regarding their clinical and commercial potential. Findings from our analysis show that the pace of deal-making in cell and gene therapy is faster and occurring much earlier in the drug development process compared to deals seen in the past for breakthrough therapies including mAbs.

When the first mAb was approved in the mid-1980s, deal-making interactions were mostly limited to collaborations and licensing agreements between smaller biotechnology firms. It took more than 20 years for big pharma to become significantly interested in mAbs and start proactively pursuing deals for these drugs. Conversely, deals within the cell and gene therapy sector have shown a much faster uptake, quickly growing in momentum since approval of the first cell and gene therapies in the early 2010s. More than 50 cell and gene therapy-related partnerships and investments were established between 2010-2016, with big pharma involved in many deals from the beginning. In one example, Novartis initiated a collaboration with GenVec in 2010 for clinical development of adeno-based gene therapies a deal worth potentially $213 million (excluding royalties). That same year Novartis also formed a strategic alliance with GlaxoSmithKline and the Telethon Institute of Gene Therapy to pursue additional gene therapy research and development.

Larger pharmaceutical companies such as Novartis seem more eager to embrace cell and gene therapies compared to prior innovative therapies including mAbs, but the types and values of related deals are evolving compared to what has historically been seen. While the largest mAb deals were often characterised by the acquisitions of late-stage and marketed products, which present more robust data and potentially lower risk to acquisition or licensing partners, most major cell and gene therapy acquisitions involve product pipelines, platform technologies, and manufacturing capabilities rather than one single product. Larger pharmaceutical companies are also targeting earlier stage opportunities, with a higher proportion of partnerships involving Phase 1 or even preclinical stage assets. For example, Pfizer established a collaboration deal with Spark Therapeutics for the development and potential commercialisation of a Phase 1/2 gene therapy for the treatment of haemophilia B and also acquired Bamboo Therapeutics based on a promising gene therapy portfolio including one Phase 1 and several preclinical assets for the treatment of rare diseases impacting the nervous system.

As more companies work to establish a presence in this sector, both larger and smaller companies now often prefer innovative licensing and collaborative agreements, whereas the majority of deals executed for mAbs were straightforward mergers or acquisitions. Given that cell and gene therapy development is still in its nascent stages and many stakeholders, including payers, have questions related to their long-term clinical benefit and budget impact, the fact that mergers or acquisitions are deemed less attractive is unsurprising. Innovative partnerships can be a lower risk option for licensors or investors who want to expand their cell and gene therapy portfolios without assuming full financial responsibility. Partnership and licensing deals can also help reduce the risk of possible disruptions in business operations and productivity, which often occur following M&A deals and the subsequent restructuring of company resources and teams.

The opportunity to access complementary business capabilities is another factor that is driving interest in innovative licensing and collaborative agreements. With licensing arrangements, a licensor might be seeking a partner who can offer specialised expertise in a specific indication whereas a licensee might see the benefit of collaborating with a company with technical abilities or assets that align with their own product pipeline. The partnership between Neurocrine Bioscience and Voyager Therapeutics reflects this type of synergistic collaboration, where Neurocrine provides expertise in central nervous system (CNS) drug development as well as financial support for the clinical development of gene therapies for Parkinson's disease and Friedreichs ataxia. In return, Neurocrine gains the opportunity to co-commercialise and potentially obtain global rights to portfolio-compatible therapies.

As new cell and gene therapies emerge and others progress to late-stage clinical development and regulatory approval, most industry stakeholders anticipate that both the structure and value of deals in the sector will mature. Companies will exercise the option to follow up on previous collaborative deals, punctuating the space with potentially massive exclusive licensing agreements and acquisitions.

*The views expressed herein are the authors and not those of Charles River Associates (CRA) or any of the organisations with which the authors are affiliated.The authors wish to acknowledge the contributions ofSil Collins and Alex Davidto this article.

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How pharma companies are shifting deal strategies for cell and gene therapies - EPM Magazine

A group of Korean researchers said they have confirmed a gene responsible for inherited retinal disorders (IRD) among Koreans.

IRD is a combination of several rare diseases that usually develops at a young age and progresses slowly over the lifetime. The patients gradually lose their sight, and most of them eventually lose their vision entirely due to continuous retinal cell degeneration.

The Seoul National University Bundang Hospital (SNUBH) Department of Ophthalmology and Seoul National University Hospital (SNUH) Department of Laboratory Medicine jointly conducted the study.

Currently, antioxidant therapy, artificial retinal transplantation, and stem cell therapy are being used to treat the disorder regardless of mutations, but the only viable treatment is gene therapy. Even when gene therapy is possible, only less than 1 percent of all IRD patients can be treated with it.

In the West, genetic abnormalities of these retinal diseases have been studied and known well. However, researches on Korean cases are still lacking, and the joint research team tackled the subject to find the causative gene for IRDs with 86 domestic patients, the team said in a news release on Wednesday.

The team studied and identified the gene responsible for the disorders by using the latest technique of gene analysis with the most number of patients who have been reported so far.

The study revealed that only 44 percent of the patients, 38 out of 86, possessed the causal gene for IRDs. Even among the patients with retinitis pigmentosa, the most common disorder among the IRDs, only 41 percent had the causative gene.

The causative genes could be quite diverse even in the same disorder. The patients can find a responsible gene only when they receive genetic counseling very actively and can receive gene counseling, too, the research team explained.

Differences were found in the type and frequency of causal gene mutations between Korean and Western cases. However, there were similarities between those of Korean and other Asian nations, including Japan.

The research and diagnosis environment for IRDs has been very poor until now, and our study has significance as a basic data for diagnosis and treatment for Korean patients with IRDs, SNUH Department of Ophthalmology Professor Woo Se-joon said.

Patients need to receive causal gene tests actively to provide the domestic medical communities with sufficient data, and a list of patients who can be treated. By doing so, clinical trials and new drug development in gene therapy will progress smoothly, he added.

Previously, only a few hospitals could diagnose the causative gene for IRDs and afford to test and treat IRD patients due to the high cost of genetic testing. Recently, however, the chance of diagnosis has increased as more hospitals are conducting genetic tests amid the lowered cost thanks to insurance benefits.

Also, the therapeutic opportunity for IRD patients is likely to get broadened, as the retinal pigment epithelium 65 gene (RPE65) therapy won approval from the U.S. Food and Drug Administration for the first time in the world.

Although we do not have a clear way to prevent IRDs at the moment, the prediction of risk and their early detection are developing through the discovery of family history and causative genes, Professor Woo said. Early diagnosis can prevent impaired vision by gene therapy and vision correction, and the patients will be able to choose appropriate jobs with social activities.

Also taking part in the research team were Professors Joo Kwang-sic and Park Kyu-hyung of SNUBH and Professors Seong Moon-woo and Park Sung-sup of SNUH.

The results of this study were published in the Journal of Korean Medical Science.

shim531@docdocdoc.co.kr

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SNUH team finds causal gene of inherited retinal disorder - Korea Biomedical Review

AlloVir and Baylor College of Medicine have teamed up to develop T-cell therapies against the novel coronavirus behind the COVID-19 pandemic outbreak. The alliance builds on existing work to create off-the-shelf cell therapies that identify and eliminate specific viruses.

Since setting up shop in 2013, AlloVir has built up a body of evidence that it can help people with weakened immune systems cope with viral pathogens. The approach entails exposing donor T cells to cytokines combined with viral fragments, thereby equipping the immune cells to recognize and help eliminate certain pathogens.

The most advanced expression of the approach is Viralym-M, a T-cell therapy designed to take out six viruses that commonly affect immunocompromised patients. Baylor moved that drug into phase 2 in 2014, coming away with data that encouraged AlloVir to plan a late-phase program.

Now, AlloVir and Baylor are applying the same approach to the SARS-CoV-2 virus sweeping across the world. AlloVir is aiming to create an off-the-shelf therapy that is capable of targeting SARS-CoV-2 and potentially similar viruses such as SARS-CoV, MERS-CoV and endemic coronaviruses.

The plan is to position the SARS-CoV-2-specific T cells for use as a monotherapy and incorporate the coronaviruses into ALVR106, a preclinical asset aimed at community-acquired respiratory viruses. In doing so, AlloVir thinks it can improve outcomes in immunocompromised patients who are exposed to SARS-CoV-2 and other viruses with the potential to overwhelm their weakened immune systems.

Neither AlloVir nor Baylor has sketched out a timeline for the COVID-19 program. Other companies, notably those such as Gilead Sciences that repurposed existing drugs, have more advanced assets, but T cells in development at AlloVir could still fill a gap in the treatment landscape. Equally, AlloVirs interest in coronaviruses other than SARS-CoV-2 means its therapies could still be relevant even if it misses the window in which treatments for the pandemic pathogen are in high demand.

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AlloVir, Baylor ally to develop COVID-19 T-cell therapy - FierceBiotech