Category Archives: Cell Therapy

After receiving CD19 chimeric antigen receptor T-cell therapy (CART19), patients with relapsed or refractory large B-cell lymphomas (rrLBCL) can have long-term remission. But more than 50% of patients do not respond to therapy. In order to identify high-risk patients who could benefit from alternative or consolidative therapy, methods were required.

Prior to CART19, researchers assessed low-pass whole-genome sequencing (lpWGS) of cell-free DNA (cfDNA) as a fresh method for risk categorization. In order to identify DNA copy number alterations (CNAs), they conducted lpWGS on pretreatment plasma samples from 122 patients at the time of leukapheresis who received standard-of-care CART19 for rrLBCL. High focal CNA scores (FCS), which indicated genomic instability, were the most important pretreatment factor in the multivariable selection and were negatively correlated with 3-month complete response rates (28% vs. 56%, P=.0029), progression-free survival (PFS; P=.0007; hazard ratio, 2.11), and overall survival (OS; P=.0026; hazard ratio, 2.10).

In 108 (89%) patients, they found 34 distinct focal CNAs; the deletion of 10q23.3, which results in the loss of the FAS death receptor, was most strongly related to poor outcomes, resulting in worse PFS (P<.0001; hazard ratio, 3.49) and OS (P=.0027; hazard ratio, 2.68) in both patients. They developed a straightforward risk model that could accurately risk stratifying patients by integrating FCS with conventional indicators of increasing tumor mass (elevated lactate dehydrogenase and >1 extranodal site).

As a result, lpWGS of cfDNA was a minimally invasive assay that might quickly identify high-risk patients and help determine which patients to choose from and which targeted medicines to test in upcoming clinical trials.

Reference: ashpublications.org/blood/article/140/5/504/485178/Risk-assessment-with-low-pass-whole-genome

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Risk Assessment with lpWGS of cfDNA Before CD19 CAR T-cell Therapy for LBCL - Physician's Weekly

Seasoned pharmaceutical industry executive to lead Tessas corporate and development strategy built around its proprietary autologous and allogeneic CAR-T platforms

Appointment follows Tessas recent US$126 million financing and precedes several clinical and data milestones expected during the second half of 2022 and early 2023

SINGAPORE, Aug. 22, 2022 (GLOBE NEWSWIRE) -- Tessa Therapeutics Ltd. (Tessa), a clinical-stage cell therapy company developing next-generation cancer treatments for hematological malignancies and solid tumors, today announced the appointment of Thomas Willemsen as its President and Chief Executive Officer effective October 1, 2022. Mr. Willemsen has also been named as a member of Tessas Board of Directors.

Mr. Willemsen brings more than 25 years of experience to Tessa with extensive international management and leadership experience in the pharmaceutical industry. He joins Tessa from Takeda Pharmaceuticals, where he served as Senior Vice President, Asia Pacific, leading the transformation of Takeda across 10 markets and enhancing its focus on rare and genetic diseases, oncology, and vaccines.

As President and CEO, Mr. Willemsen will lead Tessas corporate, business and development strategy focused on maximizing the value potential of the companys proprietary autologous and allogeneic CAR-T platforms, including ongoing clinical programs involving its autologous CD30-CAR-T therapy (TT11) and allogeneic CD30.CAR EBVST therapy (TT11X). Mr. Willemsen will lead Tessa from its global headquarters in Singapore. John Ng, who had served as Tessas acting CEO since November 2021, will continue as the companys Chief Technical Officer.

Tessa is extremely pleased to add an executive of Thomas caliber as our new President and CEO, coming at a time of significant optimism at the company as we pursue a multi-tier development strategy built around our proprietary CAR-T technologies and fueled by the recently completed US$126 million financing, stated Gran Ando, M.D., Chairman of the Board of Tessa Therapeutics. Thomas career is highlighted by senior leadership positions at several global pharmaceutical companies, including Takeda, GlaxoSmithKline and Merck KGaA. His combination of executive experience at global pharma companies and deep understanding of commercialization and oncology makes Thomas an ideal executive to lead Tessas next stage of growth.

Prior to Takeda, Mr. Willemsen held the position of Vice President, Oncology, at GlaxoSmithKline (GSK) for its Intercontinental & Emerging Markets business, where he was assigned to develop the business strategy for Asia & Emerging Markets, including Access Strategy and Commercial Structure design. Prior to that, Mr. Willemsen served as Chairman and General Manager for GSK in China, and as General Manager of GSK Taiwan. He also spent 12 years with Merck KGaA in various commercial and regional roles in the Asia Pacific region, and as the Head of its German Oncology business unit.

Mr. Willemsen graduated with an MBA from Trier University, Germany, and attained a Chinese Language Degree from Sun Yat-Sen University, Guangzhou, China. He speaks German, English, and Mandarin.

Tessa is at the forefront of developing the next generation of CAR-T therapies, including our allogeneic off-the-shelf EBVST technology, which has demonstrated very encouraging safety and efficacy data in the ongoing Phase 1/2 clinical trial in CD30 positive lymphomas, said Mr. Willemsen. I look forward to working with the entire Tessa team and continuing the positive progress with our CAR-T programs as we strive to ultimately bring these important therapies to patients with high unmet medical needs.

Dr. Ando concluded, On behalf of Tessa, I would like to commend John Ng for serving as our acting CEO during the past several months. Under Johns leadership, Tessa achieved numerous clinical and business successes, including the close of the Series A financing and the recent initiation of a clinical trial of TT11 in combination with nivolumab, which has the potential to introduce TT11 as a second-line therapy for relapsed and refractory CD30+ classical Hodgkin lymphoma patients.

About Tessa Therapeutics

Tessa Therapeutics is a clinical-stage biotechnology company developing next-generation cell therapies for the treatment of hematological cancers and solid tumors. Tessas lead clinical asset, TT11, is an autologous CD30-CAR-T therapy currently being investigated as a potential treatment for relapsed or refractory classical Hodgkin lymphoma as both a monotherapy (Phase 2) and combination therapy (Phase 1b). TT11 has been granted RMAT designation by the FDA and access to the PRIME scheme by European Medicine Agency. Tessa is also advancing an allogeneic off-the- shelf cell therapy platform targeting a broad range of cancers in which Epstein Barr Virus Specific T Cells (EBVSTs) are augmented with CD30-CAR. A therapy using this platform is currently the subject of a Phase 1 clinical trial in CD30-positive lymphomas. Tessa has its global headquarters in Singapore, where the company has built a state of the art, commercial cell therapy manufacturing facility. For more information on Tessa, visit http://www.tessacell.com.

Cautionary Note on Forward Looking Statements

This press release contains forward-looking statements (within the meaning of the Private Securities Litigation Reform Act of 1995, to the fullest extent applicable) including, without limitation, with respect to various regulatory filings or clinical study developments of the Company. You can identify these statements by the fact that they use words such as anticipate, estimate, expect, project, intend, plan, believe, target, may, assume or similar expressions. Any forward-looking statements in this press release are based on managements current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the Companys financial results, the ability to raise capital, dependence on strategic partnerships and licensees, the applicability of patents and proprietary technology, the timing for completion of the clinical trials of its product candidates, whether and when, if at all, the Companys product candidates will receive marketing approval, and competition from other biopharmaceutical companies. The Company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made, and disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the Companys views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The Companys products are expressly for investigational use pursuant to a relevant investigational device exemption granted by the U.S. Food & Drug Administration, or equivalent competent body.

Tessa Therapeutics Investor Contact

Wilson W. CheungChief Financial Officerwcheung@tessacell.com

Tessa Therapeutics Media Contact

Tiberend Strategic Advisors, Inc.Bill Borden+1-732-910-1620bborden@tiberend.com

Dave Schemelia+1-609-468-9325dschemelia@tiberend.com

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Tessa Therapeutics Appoints Thomas Willemsen as President and CEO - GlobeNewswire

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.

SOURCE IASO Biotherapeutics

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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 Culture Marketby Product (Consumables (Media, Serum, Reagent, Vessels), Equipment (Bioreactor, Centrifuge, Incubator)), Application (Vaccines, mAbs, Diagnostics, Tissue Engineering), End User (Pharma, Biotech, Hospital) - Global Forecast to 2026

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