Category Archives: Cell Therapy

SHANGHAI, Aug. 18, 2022 /PRNewswire/ -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, announces that a case report, titled "Long Term Complete Response of Advanced Hepatocellular Carcinoma to Glypican-3 Specific Chimeric Antigen Receptor T-Cells plus Sorafenib, A case report", has been published in Frontiers in Immunology (https://www.frontiersin.org/articles/10.3389/fimmu.2022.963031/full).

Hepatocellular carcinoma (HCC) is the most common histologic subtype of primary liver cancer, which is the sixth most common cancer type worldwide. Clinical efficacies of existing therapies for unresectable HCC are still unsatisfactory. CAR T-cell therapy has been approved for a variety of hematological tumors, but there are still great challenges for CAR T-cell therapies to treat solid tumors. We firstly reported GPC3 as a reasonable target for CAR T-cell therapy and thereafter advanced it into clinic.[1,2] In order to further enhance the efficacy of GPC3 CAR T cells, we proposed a new strategy by combining the GPC3 CAR T cells with sorafenib for the treatment of hepatocellular carcinoma[3]. To further validate this strategy in clinical setting, we conducted an investigator-initiated clinical trial at the First Affiliated Hospital of Wenzhou Medical University. The published case reported a patient with advanced HCC who achieved a complete response (CR) and a long survival period after the combination therapy of CAR-GPC3 T-cell plus sorafenib.

The case showed a 60-year-old Asian male patient with hepatitis B virus (HBV)-related HCC who underwent surgery in May 2018. In August 2018, the recurrence of liver cancer and pulmonary metastasis occurred after the operation, and then he received transarterial chemoembolization (TACE) to treat liver lesions and interventional ablation to treat pulmonary metastases. Two months later, he progressed and was enrolled into the clinical trial. After the enrollment, the patient underwent leukapheresis for CAR-GPC3 T-cell manufacturing. Seven days after leukapheresis, the patient started to receive 400 mg of sorafenib twice daily. The patient received 4 cycles of CAR-GPC3 T cells (CT011) treatment and each cycle was divided into two infusions. Prior to each cycle of CT011 treatment, lymphodepletion was performed. A total of 4109 CAR-GPC3 T cells were infused.

The CT011 plus sorafenib combination therapy was well tolerated. This patient obtained partial responses (PR) from the 3rd month and achieved CR in the 12th month after the first cycle of CT011 infusion. The tumor had no progression for more than 36 months and maintained the CR status for more than 24 months after the first infusion.

To the best of our knowledge, this is the first reported case with a CR after the combination therapy of CAR T cells with tyrosine kinase inhibitors. The clinical outcome demonstrated that the combination therapy of GPC3 CAR T-cell and Sorafenib may be a new promising approach for GPC3+ advanced HCC patients.

Dr. Zonghai Li, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics Holdings Limited, commented that, "There is great expectation for CAR T cells to provide curative potential in treating solid tumors. When enrolled into the clinical trial, the patient in this reported case had undergone local therapies such as TACE and interventional ablation but had not received systemic therapies such as anti-angiogenesis inhibitors. Based on the finding of our earlier preclinical research, we adopted the combination therapy of sorafenib and CT011 as treatment regimens. It was very encouraging to see that the patient achieved a complete response and a long survival period without recurrence for more than two years. While directly indicating that GPC3 CAR T may be used for early-line treatment of HCC, this case report also provides new evidence supporting the adoption of CAR T cells in the early-line treatment of other solid tumors."

About CT011

CT011 is an autologous CAR T-cell product candidate with proof-of-concept clinical data for the treatment of hepatocellular carcinoma (HCC) and has the potential to be the first-in-class globally. Dr. Zonghai Li Founder, Chairman of the Board, Chief Executive Officer and Chief Scientific Officer of CARsgen Therapeutics led the world's first successful effort in identifying, validating, and reporting GPC3 as a tumor-associated target for the development of CAR T-cell therapies to treat HCC. CARsgen has completed enrollment of a Phase I trial in China.

About CARsgen Therapeutics Holdings Limited

CARsgen is a biopharmaceutical company with operations in China and the U.S. and is focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. The Company has built an integrated cell therapy platform with in-house capabilities that span target discovery, antibody development, clinical trials, and commercial-scale manufacturing. CARsgen has internally developed novel technologies and a product pipeline with global rights to address major challenges of CAR T-cell therapies, such as improving the safety profile, enhancing the efficacy in treating solid tumors and reducing treatment costs. The Company's vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable.

References

[1]. Gao H, et. al. Clin Cancer Res. 2014 Dec 15;20(24):6418-28

[2]. Shi D, et. al. Clin Cancer Res. 2020 Aug 1;26(15):3979-3989

[3]. Wu X, et. al. Mol Ther. 2019 Aug 7;27(8):1483-1494

For more information, please visit https://www.carsgen.com/

SOURCE CARsgen Therapeutics

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Case Report of Long Term Complete Response in Hepatocellular Carcinoma to CARsgen's GPC3 CAR T Cells (CT011) Published in Frontiers in Immunology - PR...

MD Anderson Cancer Center and a Massachusetts biotechnology company received clearance from the Food and Drug Administration to begin testing a new cell therapy treatment on humans for metastatic melanoma, the most serious type of skin cancer. Clinical trials will begin next month.

The treatment, developed with Obsidian Therapeutics of Cambridge, Mass., aims to avoid life threatening complications associated with similar cell therapies.

It is a type of tumor infiltrating lymphocyte (TIL) therapy, in which doctors remove tissue from the tumor, isolate the cancer fighting T cells, and infuse in the patient to concentrate their attacks on the cancer cells and reduce the tumors. Typically, patients also receive an infusion of interleukin-2, which promotes the growth of those T cells, but can cause heart, lung and kidney dysfunction.

MD Anderson and Obsidian researchers hope they found a technique that is more effective without harsh side effects.

On HoustonChronicle.com: MD Anderson launches joint venture with biopharma manufacturer, creating a potential launching pad for Houstons biotech ambitions

The researchers modified the T cells to produce their own interleukin, but a slightly different version known as interleukin-15. They modified the T cells so they can be activated by a pill called Acetazolamide, which is used to treat a variety of diseases from from glaucoma to epilepsy.

If patients have adverse reactions to the treatment, they can be advised to stop taking the pill for a day or so. This wouldnt be the case with a one-time infusion.

It's an on-off switch, Dr. Rodabe Amaria, the lead researcher on the study said. Well be able to regulate the function of the T cell, which we really havent been able to do before in our T cell therapy.

Dr. Paul Wotton, chief executive officer of Obsidian Therapeutics, said he believes the therapy has the potential to significantly transform the treatment landscape for patients, bringing broader access to (tumor infiltrating lymphocyte therapy).

The cell therapies will be manufactured at CTMC, a joint venture between a San Diego biomanufacturer and MD Anderson to develop and manufacture cell therapies, aiming to build a niche in biologic treatments.

becca.carballo@chron.com

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FDA gives MD Anderson the green light to test skin cancer treatment on humans - Houston Chronicle

Clearly, CGTs have not taken the same development path as MAbs, and those paths are unlikely to converge unless one of the modalities shows differentiated benefits for productivity, purity, and applicability. However, manufacturing processes have improved dramatically since production of the worlds first CGT products, as has industry and regulatory understanding about the safety profiles and characterization requirements for such products. But the CGT industry has much further to go.

The State of the CGT Industry

A major question that lingers along the development path is whether gene therapies will continue addressing monogenic targets or expand to multigenic indications. Despite efforts to match the productivity of processes for other biopharmaceuticals, improvements to CGT-process productivity have been stepwise rather than transformative, and such changes generally have not met expectations. One result is that after 20 years, CGTs largely remain focused on treating small patient populations with specific medical needs. Small production volumes also have led to high prices, which have been justifiable for early entrants to the CGT space but will be unsustainable in the long term.

Until the past few years, suppliers of raw materials, cell-culture media, and bioprocess equipment largely have ignored the CGT space. The market had been perceived as too small and unproven to provide profitable opportunities. Luckily, commercial success has driven most suppliers to recognize the CGT industry as a viable market that needs special attention. Today, equipment is designed solely for cell therapy applications or viral vector manufacturing. Raw materials such as plasmids have increased both in quality and availability. Most suppliers have awakened to the potential of CGT commercial success, especially considering that clinical timelines for most such products are condensed compared with those for MAbs and other biologics.

The Story of Oxford Biomedica

My employer, Oxford Biomedica, also has been involved in gene therapy production for over 25 years. Oxford Biomedica has been a biotechnology company for much of its history, and weve built lentiviral (LV) manufacturing technology to support in-house production of our products. Like many other candidate therapies, our lead programs did not progress through clinical studies, and weve moved on to other indications. But our hard work in gene-therapy development, manufacturing, analytics, quality control (QC), and regulatory affairs has paid off nicely because other companies have recognized the depth of our expertise. Weve forged licensing partnerships so that our technology can be used by multiple companies rather than just one.

We capitalized on that business model in January 2022 when we worked with Homology Medicines to form a new entity called Oxford Biomedica Solutions to focus on adenoassociated virus (AAV) development and manufacturing. The hard work that was put into developing AAV manufacturing expertise now is readily available to partner organizations rather than held closely within one company. The decision to form Oxford Biomedica Solutions speaks to the importance of sharing lessons learned from taking products from early clinical phases through commercial manufacturing with partner companies that want to benefit from such experience.

Personal Reflections on CGT Development

From a business perspective, CGT companies have needed to navigate a series of ups and downs. Such a process isnt for the faint of heart.

Early in my career, only academic centers, a few biotechnology companies, and a large pharmaceutical company or two engaged with gene-therapy technologies. In 1999, the tragic death of Jesse Gelsinger, the first person to die during a clinical trial for gene therapy, dropped the industry to its knees. Most large pharmaceutical companies stopped funding gene therapy projects, but a small number of true believers persisted with the technology, developing safer and better vector systems and improving clinical trial design and support.

It was only 10 years ago that two fundamental technologies were found to be effective for gene therapy: AAV vectors were found to be effective in delivering therapeutic genes for certain monogenic rare diseases, and patients treated with CAR-T products showed spectacularly improved rates of complete responses to blood cancers. Companies and investors started to take notice. In 2019, when AveXis/Novartis began commercializing Zolgensma (onasemnogene abeparvovec-xioi) as a treatment for spinal muscular atrophy (SMA), I was among many scientists who felt that the drugs regulatory approval was just the validation that the CGT industry needed.

My work in CGT is personal. My 21-year-old daughter suffers from Rett syndrome (RTS, cerebroatrophic hyperammonemia), a rare, monogenic neurological disease. Sadly, a cure is not yet available but developers are still trying. The challenges that my family has encountered while seeking out RTS treatments mirror some of the obstacles that face the CGT industry. Manufacturing processes remain small and fragmented, although they have moved largely to mammalian cell cultures in suspension bioreactors, which will provide scalability advantages. AAV vectors work best when the serotype used matches to a target organ, but results have been inconsistent. Increasingly, the US Food and Drug Administration (FDA) insists on characterization of AAV products, even for early phase clinical trials. A number of clinical holds have occurred for AAV-based gene therapies. Thus, Oxford Biomedica believes, as do many drug companies, that AAV needs to be manufactured using a scalable process that can deliver high levels of full, infectious particles; that qualified analytics should be established at the earliest possible stage; and that impurities, including empty capsids, must be closely monitored and reduced.

Guarded Optimism for the CGT Industry

Ive worked for both drug developers and service providers within the CGT industry, so I am in the novel position of having been on both sides of the table. The macrodynamics of outsourcing have changed dramatically over the past five years. What had been a largely underserved marketplace now has become inundated with large multinational pharmaceutical companies, specialty providers, and academic centers. Companies that suffered through 1824 months of waiting in a manufacturers queue have decided to build their own capacity. And the few manufacturing specialists with CGT capabilities have found themselves becoming critically important as the talent pool has thinned out among the industrys many offerings. This current situation, when combined with our uncertain economic environment, is putting internal and external manufacturing capacity under renewed focus.

I am hopeful but realistic about the next 10 years of the CGT industry. The COVID-19 pandemic has shown us that under certain circumstances, new technologies such as mRNA can be developed and scaled rapidly. Cell therapies are likely to improve in quality and scale through automation of production processes. Improvements also might come from transitions from autologous to allogeneic processes or from novel applications such as in vivo delivery of CAR T cells. I hope that gene therapies will move to even more scalable processes, with well-considered regulatory guidelines particularly for AAV-based therapies established to help minimize patient and manufacturing risks. And just maybe, we can treat not only rare diseases such as what my daughter has, but also larger indications. Doing so will support the future of the industry.

David Backer is chief commercial officer at Oxford Biomedica in Oxford, UK; d.backer@oxb.com; https://www.oxb.com.

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Taking a Distinctive Path Reflections on the History of Gene Therapy Development - BioProcess Insider

DUBLIN--(BUSINESS WIRE)--The "Leukapheresis Market Research Report by Type (Leukapheresis Devices and Leukapheresis Disposables), Application, End-user, Region (Americas, Asia-Pacific, and Europe, Middle East & Africa) - Global Forecast to 2027 - Cumulative Impact of COVID-19" report has been added to ResearchAndMarkets.com's offering.

The Global Leukapheresis Market size was estimated at USD 46.88 million in 2021, USD 51.30 million in 2022, and is projected to grow at a CAGR 9.61% to reach USD 81.31 million by 2027.

Competitive Strategic Window:

The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

FPNV Positioning Matrix:

The FPNV Positioning Matrix evaluates and categorizes the vendors in the Leukapheresis Market based on Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Market Share Analysis:

The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others. Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

The report provides insights on the following pointers:

1. Market Penetration: Provides comprehensive information on the market offered by the key players

2. Market Development: Provides in-depth information about lucrative emerging markets and analyze penetration across mature segments of the markets

3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments

4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, certification, regulatory approvals, patent landscape, and manufacturing capabilities of the leading players

5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and breakthrough product developments

The report answers questions such as:

1. What is the market size and forecast of the Global Leukapheresis Market?

2. What are the inhibiting factors and impact of COVID-19 shaping the Global Leukapheresis Market during the forecast period?

3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Leukapheresis Market?

4. What is the competitive strategic window for opportunities in the Global Leukapheresis Market?

5. What are the technology trends and regulatory frameworks in the Global Leukapheresis Market?

6. What is the market share of the leading vendors in the Global Leukapheresis Market?

7. What modes and strategic moves are considered suitable for entering the Global Leukapheresis Market?

Market Dynamics

Drivers

Restraints

Opportunities

Challenges

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/2l9hhj

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Insights on the Leukapheresis Global Market to 2027 - Leukapheresis for CAR or Adoptive Cell Therapy Presents Opportunities - ResearchAndMarkets.com -...

SHANGHAI, NANJING, China and SAN JOSE, Calif., Aug. 19, 2022 /PRNewswire/ -- IASO Biotherapeutics (IASO Bio), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, announced today that the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) approved its investigational new drug (IND) application (Acceptance No.: CXSL2200233CXSL2200234) for the new extended indication of Neuromyelitis Optica Spectrum Disorder (NMOSD) for a fully human BCMA chimeric antigen receptor autologous T (CAR-T) cell Injection (Equecabtagene Autoleucel, CT103A). This is the world's first IND approval for CAR-T in NMOSD treatment.

The new IND application is based on clinical data from an investigator-initiated clinical study of Equecabtagene Autoleucel. Subjects in the study were NMOSD patients with poor symptom control who had at least one year of treatment with at least one immunosuppressant. The study's primary objective was to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetic (PK) and pharmacodynamic (PD) profiles in NMOSD patients treated with Equecabtagene Autoleucel. As of March 20, 2022, 12 subjects received Equecabtagene Autoleucel cell infusion, including three in the 0.5106 CAR-T cells/kg dose group and nine in the 1.0106 CAR-T cells/kg dose group. The study data initially showed that the Equecabtagene Autoleucel injection was safe in patients with relapsed/refractory NMOSD in the 0.5106 CAR-T cells/kg and 1.0106 CAR-T cells/kg dose groups. All patients experienced Grade 1-2 CRS (Cytokine Release Syndrome) and no Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) events. In terms of efficacy, all subjects observed improved Expanded Disability Status Scale (EDSS) scores after infusion. Fifty percent experienced improved visual acuity, 67% improved their walking ability, and 75% experienced improved bladder function. After a median follow-up of 5.5 months, 11 / 12 (92%) subjects did not observe any disease recurrence.

Wen (Maxwell) Wang, M.D., Ph.D., Chief Executive Officer and Chief Medical Officer of IASO Bio, said, "As one of the first companies to conduct research on CAR-T to treat autoimmune diseases worldwide, our BCMA CAR T-cell therapy represents a significant milestone for Investigator Initiated Trial (IIT) data of relapsed and refractory NMOSD, an autoimmune disease with serious complications, blindness, and paralysis."

The existing treatment of NMOSD can only decrease the number of relapses within a certain period and has little effect on the functional recovery of sensory, nervous, and motor systems. BCMA CAR T-cell therapy can reduce the disability score and improve the functions of sensory, nervous, and motor systems, providing a milestone proof-of-concept for CAR-T therapy to treat autoimmune diseases caused by auto-antibodies produced by plasma cells. The IND's approval demonstrates further momentum for IASO Bio to promote the expansion of CAR-T therapy and launch products beyond the treatment of malignant tumors to the treatment of autoimmune diseases. IASO Bio will initiate and complete the clinical study per submitted protocol to prepare for new drug application (NDA) and to bring hope to NMOSD patients.

About Equecabtagene Autoleucel (CT103A)

Equecabtagene Autoleucel (CT103A) is a BCMA chimeric antigen receptor autologous T cell injection, a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3 activation domains. Based on strict selection and screening, utilizing a proprietary in-house optimization platform, and integrated in-house manufacturing process improvement, the construct of the BCMA CAR-T is potent and Equecabtagene Autoleucel shows prolonged persistency in patients. The NMPA accepted the New Drug Application for Equecabtagene Autoleucel for the treatment of elapsed/refractory multiple myeloma (R/R MM).Equecabtagene Autoleucel also received Breakthrough Therapy Designation by the NMPA in February 2021 and Orphan Drug Designation (ODD)by the U.S. FDA in February 2022.

In addition to multiple myeloma, the NMPA has received IND application of Equecabtagene Autoleucel for the new expanded indication of Neuromyelitis Optica Spectrum Disorder (NMOSD).

About Neuromyelitis Optica (NMO)

Neuromyelitis Optica (NMO) is an acute or subacute inflammatory demyelination disorder of the central nervous system, an antibody-mediated idiopathic inflammatory disease of the nervous system. NMO Spectrum Disorders (NMOSD) are marked by NMO-IgG antibodies in the serum, covering NMO and NMO-related diseases. The first onset of NMOSD is seen at all ages, mostly in young and middle-aged people, with a median age of 39 years. The prevalence of NMOSD is high in Asian populated areas, and middle-aged women are the most prevalent group. According to Frost & Sullivan, the number of NMOSD cases in China was around 49,300 and 173,000 worldwide in 2021. AQP4-Ab is a foremost pathogenic antibody of NMOSD, and many clinical studies have confirmed that this antibody may cause pathological damage to the central nervous system in animals and humans. Its diagnostic specificity is up to more than 90%, with the positive rate of AQP4-Ab in NMOSD patients ranging from 40% to 90%. NMOSD is a highly recurrent disease with a high disability rate. More than 90% of patients have a multitemporal course, 60% relapse within one year and 90% relapse within three years, with sequelae found in most patients such as severe visual impairment, physical dysfunction, and urination and defecation disorders.

About IASO Bio

IASO Bio is an innovative biopharmaceutical company specializing in the development and manufacture of cellular therapeutics and antibody drugs. The company is expanding into solid tumors and autoimmune diseases with the development of hematologic oncology cell-based drugs and antibody drugs as the cornerstone of innovation. It offers a complete platform from early discovery, registration, and clinical development to commercial production. IASO Bio owns many technology platforms, including a fully human antibody discovery platform, a high-throughput CAR-T drug preference platform, a general CAR technology platform, a production technology platform, and a clinical translational research platform. It has more than 10 products at different stages of development, including Equecabtagene Autoleucel (CT103A)fully human BCMA chimeric antigen receptor autologous T cell injection, which received NDA acceptance of the China NMPA for the treatment of elapsed/refractory multiple myeloma (R/R MM).Equecabtagene Autoleucel also received Breakthrough Therapy Designation by the NMPA in February 2021 and Orphan Drug Designation (ODD) by the U.S. FDA in February 2022. In addition to multiple myeloma, the NMPA has received IND application of Equecabtagene Autoleucel for the new expanded indication of Neuromyelitis Optica Spectrum Disorder (NMOSD). Additionally, its product CT120 (fully human CD19/CD22 dual-target CAR-T cell injection) has entered the clinical research stage for the treatment of CD19/CD22-positive relapsed/refractory B-cell non-Hodgkin lymphoma (B-NHL) and relapsed/refractory acute B-lymphoblastic leukemia (B-ALL) and granted FDA Orphan Drug Designation (ODD).

Leveraging its strong management team, rich product pipeline, cutting-edge R&D, and business model, and with the introduction of innovative drugs that truly solve clinical pain points and open new treatment paths, IASO Bio is becoming one of the industry's most influential and innovative pharmaceutical companies. For more information, please visit http://www.iasobio.comor http://www.linkedin.com/company/iasobiotherapeutics.

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World's First CAR-T for NMOSD Treatment, IASO Biotherapeutics' Equecabtagene Autoleucel, Receives IND Approval by NMPA - PR Newswire

RoslinCT, a cell and gene therapy contract development and manufacturing organization (CDMO) developing life-changing therapies in Edinburghs BioQuarter, and Lykan Bioscience, a CDMO focused on cell-based therapies, have entered into a business combination agreement to form a global advanced therapies CDMO.

The combined group will offer process development expertise and cGMP manufacturing for a range of autologous and allogeneic cell therapies, with expertise in gene editing and induced pluripotent stem cell (iPSC) capabilities.

The group will benefit from expanded capacity, with process and analytical development laboratories and cGMP manufacturing facilities in Edinburgh, Scotland, and in Hopkinton, Massachusetts.

Lykan has a 64,000 sq. ft. cell therapy manufacturing facility and innovation/development laboratories with 16 cGMP processing suites running by the end of 2022. Further laboratory and cGMP capacity expansion in Scotland is planned to build on RoslinCTs existing 40,000 sq. ft facilities, including eight cGMP suites.

The companies said the pairing will shorten development and manufacturing timelines for advanced therapy sponsors, facilitating clinical and commercial GMP product release on both sides of the Atlantic.

Earlier in 2022, Global Healthcare Opportunities, or GHO Capital Partners LLP (GHO), a European investor in global healthcare, announced its investment in RoslinCT. As part of the new agreement, GHO is making a majority investment in Lykan and is backing the funding of the combined entity. WindRose Health Investors, previously the majority owner of Lykan Bioscience, has reinvested in the new combined group along with Lykan Management.

RoslinCT CEO Peter Coleman, and Lykan president and CEO Patrick Lucy, will remain in their current roles. Together, the new entity will have a global headcount of around 300 employees.

Coleman said: This combination puts us in a strong position as a leading global CDMO in the process development and manufacturing of advanced cell therapies, and we look forward to working with our new colleagues at Lykan to fuel future growth and meet the increasing demand for innovative therapies.

Lucy added: We are delighted to combine with RoslinCT to better serve the growing demand for manufacturing capacity and expand the range of innovative services we can provide our partners to support the development of advanced cell and gene therapies.

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RoslinCT and Lykan Bioscience combine to create cell therapy CDMO - Labiotech.eu

Creating Possible

Stories@Gilead - August 04, 2022

In February 2019, Eleonora was preparing to go out with a friend when she suddenly fainted. She woke up in the hospital near her home in Italy and didnt remember a thing. But she will never forget the news the doctors delivered to her.

They did a CT scan and it lit up like a Christmas tree, recalls Eleonora. They found several large tumors and told me I had non-Hodgkin lymphoma.

After losing her father to cancer a year earlier, she knew she had to overcome the cancer to spare her family any more pain.

She started chemotherapy, and a CT scan later revealed that the tumor had not responded to initial lines of treatment. During that moment of discouragement, a small voice rose inside of her saying, Do not give up.

A hospital in Genoa offered Eleonora another round of chemotherapy, but she decided not to pursue it. Instead, she was inspired after watching a program on a Swiss TV channel where they discussed a form of therapy that uses the bodys own immune system to fight cancer: chimeric antigen receptor (CAR) T-cell therapy.

A whole series of lights went off in my head that this might be my solution, Eleonora says.

She contacted a lymphoma specialist to discuss her options and started CAR T-cell therapy in December 2019. Two months later, she had a follow-up PET scan and it showed she had responded to treatment, with no evidence of cancer cells showing up in the scan.

Im not talking about months anymore, instead Im talking about a life ahead of me.

Watch the video above to see Eleonoras full journey.

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Overcoming Cancer with CAR-T Cell Therapy - Gilead Sciences

CHICAGO, Aug. 3, 2022 /PRNewswire/ --Cell Therapy Technologies Marketis projected to grow from USD 4.0 billion in 2022 to USD 8.0 billion by 2027, at a CAGR of 14.6% from 2022 to 2027, according to a new report by MarketsandMarkets.Growth in the market can be attributed to number of cell therapy clinical trials related to cancer. Furthermore, increasing incidence of communicable diseases and the growing risk of pandemics are also expected to fuel the market growth.

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Browse in-depth TOC on "Cell Therapy Technologies Market"202 Tables48 Figures218 Pages

The cell therapy equipment segment accounted for the second largest share of the product segment in the cell therapy technologies market in 2021.

The second largest share of cell therapy equipment segment can be attributed to the growing demand for these equipments. Cell therapy equipment is used in cell processing (such as cell isolation, expansion, and harvesting), cell preservation and handling, and process monitoring and quality control. The segment market is further sub-segmented into cell processing equipment, single-use equipment, and other equipment (flow cytometers, cell counters, microscopes, etc).

The stem cells segment accounted for the second largest share of the cell type segment in the cell therapy technologies market in 2021.

Rising awareness regarding the use of stem cells in the treatment of various diseases and the growing focus of players on stem cell research are driving the growth of this market segment. Rising collaboration between universities and biotechnology & biopharmaceutical companies for stem cell research and government support (availability of funding) are other important drivers.

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The Asia Pacific region is the fastest-growing region of the cell therapy technologies market in 2021.

The Asia Pacific is estimated to be the fastest-growing segment of the market. The growth of the market of the region is mostly driven by their low labor and manufacturing costs, which has drawn huge investments by biopharma giants to these countries. The increasing disposable income, growing prevalence of lifestyle and age-related chronic diseases also contribute to the high growth of the regional market.

Key players in the cell therapy technologies market include Thermo Fisher Scientific, Inc. (US), Merck KGaA (Germany), Danaher Corporation (US), Lonza Group (Switzerland), Sartorius AG (Germany), Terumo BCT (US), Becton, Dickinson and Company (US), Fresenius SE & Co. KGaA (Germany), Avantor, Inc. (US), Bio-Techne Corporation (US), Corning Incorporated (US), FUJIFILM Irvine Scientific (US), MaxCyte Inc. (US), Werum IT Solutions GmbH (Germany), RoosterBio Inc. (US), SIRION Biotech GmbH (Germany), TrakCel (UK), L7 Informatics, Inc. (US), Miltenyi Biotec GmbH (Germany), STEMCELL Technologies (Canada), GPI Iberia (Spain), MAK-SYSTEM (US), OrganaBio, LLC (US), IxCells Biotechnology (China), and Wilson Wolf Manufacturing Corporation (US).

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Cell Therapy Technologies Market worth $8.0 billion by 2027 - Exclusive Report by MarketsandMarkets - PR Newswire UK

Marker Therapeutics

Company expects to initiate Phase 1 trial of MT-601 in r/r NHL in 2023

HOUSTON, Aug. 04, 2022 (GLOBE NEWSWIRE) -- Marker Therapeutics, Inc.(Nasdaq: MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for MT-601, a multi-tumor-associated antigen (multiTAA)-specific T cell product targeting six antigens, for the treatment of patients with relapsed/refractory non-Hodgkin lymphoma who have failed or are ineligible to receive anti-CD19 CAR T cell treatment.

This new clinical trial will build upon results that were observed in the Phase I/II TACTAL study conducted by BCM, which assessed the safety and efficacy of five-antigen-directed multiTAA-specific T cell product, stated Dr. Mythili Koneru, Markers Chief Medical Officer. In the TACTAL study, BCM observed long-term CR rates that were comparable to recently approved CD19 CAR-T therapies, even at very low cell doses. Unlike CD19 CAR-T cell therapies, patients receiving multiTAA-specific T cell product had superior durability of response, without the severe toxicities that commonly occur with other adoptive cell therapies, such as cytokine release syndrome or neurotoxicity. Based on these results, we believe that multiTAA-specific T cell products can be easily administered in an outpatient setting without hospitalization.

In the TACTAL study, patients were treated with five-antigen-directed multiTAA-T cell product. Based upon the safety profile observed with multiTAA-specific T cell therapies containing WT-1 in multiple cancer indications, the FDA cleared in the IND the addition of WT-1 as the sixth tumor-associated antigen to the MT-601 product that will be used to treat patients in the Marker sponsored study. In addition, the FDA has cleared Marker to initiate its study at a dose level of 200 million cells per infusion, versus the dose range of 10-40 million cells per infusion used in the TACTAL study. This increase in the cell dose will be possible due to Markers development and adoption of a 9-day manufacturing process, which also accelerates the time to treatment.

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Dr. Koneru continued: We believe that the most important finding of the TACTAL study was that the administration of multiTAA therapy consistently drove an enhanced immunological response from the patients own immune system, which we believe was due to the lack of lymphodepletion which allowed the patients own immune system to play a part. We believe that this phenomenon, known as epitope spreading, was critical in driving more durable responses than have been observed with other cell therapies like TCRs and CAR-Ts. It is notable that none of the patients who developed a CR in the TACTAL study relapsed during the follow up period, and several patients have been in CR for over five years at their last follow-up. This contrasts strongly with the experience of CD-19 CAR-Ts, where up to 40% of patients are expected to relapse within 12 months of developing a complete response.

Markers MT-601 Phase 1 trial will focus on r/r NHL patients who have failed CD19 CAR-T therapy, or those who are ineligible for treatment with those therapies. MT-601 targets a series of tumor antigens other than CD19, offering patients a therapeutic alternative even if their tumor has escaped by downregulating the expression of CD19. For patients who cannot access CD19 therapies, MT-601 has the potential to generate objective responses, with tolerability and potentially longer duration of response.

FDA clearance of our IND for MT-601 is a significant milestone as we advance our pipeline in a number of Company-sponsored trials, saidPeter L. Hoang, Markers President and Chief Executive Officer. We believe that MT-601, which targets six tumor-associated antigens highly expressed in lymphoma, has the potential to build upon results of the TACTAL study. We look forward to initiating our Company-sponsored Phase 1 study next year.

About Marker Therapeutics, Inc.Marker Therapeutics, Inc. is a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications. Markers cell therapy technology is based on the selective expansion of non-engineered, tumor-specific T cells that recognize tumor associated antigens (i.e. tumor targets) and kill tumor cells expressing those targets. This population of T cells is designed to attack multiple tumor targets following infusion into patients and to activate the patients immune system to produce broad spectrum anti-tumor activity. Because Marker does not genetically engineer its T cell therapies, we believe that our product candidates will be easier and less expensive to manufacture, with reduced toxicities, compared to current engineered CAR-T and TCR-based approaches, and may provide patients with meaningful clinical benefit. As a result, Marker believes its portfolio of T cell therapies has a compelling product profile, as compared to current gene-modified CAR-T and TCR-based therapies.

To receive future press releases via email, please visit: https://www.markertherapeutics.com/email-alerts.

Forward-Looking Statements This release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Statements in this news release concerning the Companys expectations, plans, business outlook or future performance, and any other statements concerning assumptions made or expectations as to any future events, conditions, performance or other matters, are forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: our research, development and regulatory activities and expectations relating to our non-engineered multi-tumor antigen specific T cell therapies, including MT-601; the effectiveness of these programs or the possible range of application and potential curative effects and safety in the treatment of diseases; and the timing, conduct and success of our clinical trials, including the Phase 1 trial of MT-601. Forward-looking statements are by their nature subject to risks, uncertainties and other factors which could cause actual results to differ materially from those stated in such statements. Such risks, uncertainties and factors include, but are not limited to the risks set forth in the Companys most recent Form 10-K, 10-Q and other SEC filings which are available through EDGAR at http://www.sec.gov. Such risks and uncertainties may be amplified by the COVID-19 pandemic and its impact on our business and the global economy. The Company assumes no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Investors and Media Contacts

Marker Therapeutics:

Neda SafarzadehVice President/Head of Investor Relations, PR & Marketing(713) 400-6451Investor.Relations@markertherapeutics.com

Solebury Trout:

MediaAmy BonannoAbonanno@soleburytrout.com

Continued here:
Marker Therapeutics Announces FDA Clearance of IND for MT-601, the six-antigen targeted T Cell Therapy for the treatment of relapsed/refractory...

A Beam Therapeutics cell therapy whose precise genetic edits are intended to make it a better cancer fighter is now under an FDA clinical hold before a single patient has been dosed with the experimental treatment.

Beam submitted its investigational new drug application to the regulator in late June. On Monday, Cambridge, Massachusetts-based Beam said it received an FDA email notifying the company about the clinical hold placed on the application for the therapy, BEAM-201. Beam disclosed little else, other than that the agency said it would provide an official clinical hold letter within 30 days.

Beam develops medicines using base editing, a technology that enables it to target and edit a single base in the genome without making a double-stranded break in DNA. Whereas CRISPR has been described as molecular scissors, Beam likens its base-editing technology to a pencil that erases a genetic error and writes in the correct letter.

BEAM-201 is made from T cells sourced from healthy donors to create a therapy that is allogeneic, or off-the-shelf. Among the edits are changes that minimize the ability of tumors to suppress these engineered cells, and to prevent the cell therapy from targeting the patients T cells, a phenomenon known as fratricide.

Beam is developing BEAM-201 as a treatment for advanced acute lymphoblastic leukemia and T cell lymphoblastic lymphoma. The therapy is made with four simultaneous edits, which the company believes makes it the first experimental cell therapy with that many edits. After those multiplex edits, a lentivirus is used to introduce genetic material that gets the cells to produce a chimeric antigen receptor (CAR) that targets a particular cancer antigen. In the manufacturing process for clinical trials, the four base edits were achieved in 91% of cells, the company said in its 2021 annual report. An estimated 77% of cells achieved the four edits plus the modification to produce the CAR. In lab tests, Beam reported that the BEAM-201 cells killed cancer cells. In mice, the cell therapy showed a dose-dependent ability to clear or control cancer cells.

Other companies developing allogeneic T cell-therapies offering multiplex editing include Allogene Therapeutics and Cellectis. In a research note sent to investors Monday, William Blair analyst Raju Prasad noted that those companies have also received clinical holds, making us believe that this update is likely due to questions surrounding multiplex base editing of a T cell product (given the new gene-editing modality). Allogenes clinical hold was resolved in about three months, which signals a potentially similar timeframe for Beam, Prasad added.

Beams technology is also used by Verve Therapeutics under a licensing agreement. Verve employs base editing for an in vivo therapy designed to turn off the gene that codes for PCSK9, a liver protein that contributes to an inherited form of high cholesterol. Last month, Verve dosed the first patient in a clinical trial testing its base-editing medicine, VERVE-101, as a treatment for heterozygous familial hypercholesterolemia. That study is underway in New Zealand; Verve expects to receive regulatory clearances to begin human testing in the U.K. and the U.S. sometime in the second half of this year.

FDA lifts clinical hold on Celyads cell therapy

In other clinical hold news, the FDA has cleared Celyad Oncology to resume tests of CEL-101, its experimental cell therapy for advanced colorectal cancer.

The deaths of two patient in a Phase 1b led Belgium-based Celyad to voluntarily pause a Phase 1b test in February. Soon after, the FDA formally placed the study under a clinical hold. CEL-101 is made from T cells that come from healthy donors. Those cells are engineered in a way that reduces the risk of graft-versus-host disease, a complication in which the transplanted cells attack native cells of the patient.

The Phase 1b test of CEL-101 is evaluating the cell therapy in combination with Mercks mega-blockbuster cancer immunotherapy, Keytruda. Celyad said that the FDA lifted the clinical hold after the company changed eligibility criteria for the study.

Photo by Flickr user K-State Research and Extension via a Creative Commons license

Original post:
FDA hits pause on Beam Therapeutics' off-the-shelf cell therapy for blood cancers - MedCity News