Dublin, June 16, 2022 (GLOBE NEWSWIRE) -- The "Global CAR T Cell Therapy Market Opportunity & Clinical Pipeline Insight 2028" drug pipelines has been added to ResearchAndMarkets.com's offering.

Global CAR T cell therapy market opportunity is expected to surpass US$ 15 Billion by 2028

The development of CAR T cell therapy will gather momentum from the increasing number of investors, designers, and analysts in the guide therapy.

Rising frequencies of cancer across the globe together with the relentless technological advancement for dependable and effective cancer treatment is expected to support the growth of the worldwide CAR-T cell therapy market. Furthermore, heavy investments in clinical research and development in cell therapy are estimated to boost the market for CAR-T cell therapy in the years to come.

The report provides information on the globally approved CAR T cell therapies along with their price, dosage, and sales analysis. The report analyzes and forecasts the size of the global CAR-T cell therapy market.

The report delivers a full-scale exploration of market dynamics and factors that are manipulating the growth of the global CAR-T cell therapy market. The report also gives a competitive analysis of major market players that will give a competitive advantage to customers in their respective businesses.

Chimeric antigen receptor (CAR) T cell therapy is a novel immunotherapeutic approach that is indicated for the management of cancer. CAR T-cell therapy is a type of therapy in which the T-cells are taken from the blood of the patient, which is then modified in a lab by the addition of a gene for a man-made receptor (chimeric antigen receptor).

This aids in better recognizing of specific cancer cell antigens. The CAR T-cells are then given back to the patient. To date, 6 CAR T-cell therapy products including Kymriah (tisagenlecleucel), yescarta (axicabtagene), Tecartus (brexucabtagene autoleucel), Breyanzi (lisocabtagene maraleucel), Carvykti (ciltacabtagene autoleucel), and Abecma (idecabtagene vicleucel) have been approved by FDA for the management of wide range of cancers. In addition to this, Relmacabtagene autoleucel have been approved in China.

The introduction of CAR T cell therapies has shown high adoption rates in a short span of time has propelled further research and development activities in this domain. Apart from hematological malignancies, the researchers are also expanding the role of CAR T cell therapies in solid tumors which will propel the growth of the market.

For instance, a recent research study demonstrated that infusing immune cells into patients has shown early promise by shrinking tumors in digestive system. The interim results demonstrated that tumors in nearly 50% of the 37 patients so far reduced in size after the therapy.

The global CAR T cell therapy market is highly consolidated with several key players. The pharmaceutical giants in the market are increasingly partnering or collaborating with technology companies to integrate advanced technologies for the betterment of CAR-T therapy or to expand their geographical presence.

For instance, Bayer and Atara Biotherapeutics entered into a worldwide license agreement for the development of next-generation ATA3271 which is mesothelin-directed CAR-T cell therapy. The agreement also aims to develop ATA2271, for the management of high mesothelin-expressing tumors such as malignant pleural mesothelioma and non-small-cell lung cancer.

Among drug types, Yescarta is currently dominating the market. Yescarta (axicabtagene ciloleucel) is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma.

Recently in 2022, regulatory bodies expanded the approval of drugs for the management of large B-cell lymphoma (LBCL) that is refractory to first-line chemoimmunotherapy or relapses within a year of first-line chemotherapy. The wide range of therapeutic indications as well as the global availability of the drug is the major factors which are the major factors aiding in the dominance of these factors.

Report Highlights:

Key Topics Covered:

1. Research Methodology

2. Chimeric Antigen Receptor (CAR) T Cell Therapy - Next Era in Immuno Oncology

3. Evolution of Chimeric Antigen Receptor (CAR) T-Cell Design3.1 Structure of CAR-T Cell3.2 1st Generation Chimeric Antigen Receptor3.3 2nd & 3rd Generation CAR-T Cell3.4 Principle of Chimeric Antigen Receptor Design

4. Approaches to Improve the Efficiency of CAR-T Cell Therapy

5. CAR T Cell Therapy Manufacturing Cost Analysis

6. Emerging In-Vivo Induced CAR T Cell Therapies6.1 Need of In-Vivo CAR T Cell Therapy6.2 In-Vivo CAR T Cell Platform; VivoVec, TumorTag, & RACR/CAR6.3 Key Drug in Research & Development

7. Global CAR T Cell Therapy Market Outlook 2022 - 20287.1 Market Overview7.2 Future Market Opportunity

8. Globally Approved CAR T Cell Therapies - Pricing, Dosage & Sales Analysis 20288.1 Tisagenlecleucel (Kymriah)8.2 Axicabtagene Ciloleuce (Yescarta)8.3 Brexucabtagene Autoleucel (Tecartus)8.4 Lisocabtagene maraleucel (Breyanzi)8.5 Idecabtagene Vicleucel (Abecma)8.6 Relmacabtagene autoleucel (Carteyva)8.7 Ciltacabtagene autoleucel (Carvykti)

9. US CAR T Cell Therapy Market Outlook9.1 Approved & Key CAR T Cell Therapy Products in US9.2 Market Overview9.3 Current Reimbursement Scenario

10. Europe CAR T Cell Therapy Market Outlook10.1 Approved & Key CAR T Cell Therapy Products in Europe10.2 UK10.3 Germany10.4 Spain10.5 France10.6 Rest of Europe10.7 Current Reimbursement Scenario

11. China CAR T Cell Therapy Market Outlook11.1 Approved & Key CAR T Cell Therapy Products in China11.2 Current Market Size & Forecast

12. Japan CAR T Cell Therapy Market Outlook12.1 Approved & Key CAR T Cell Therapy Products in Japan12.2 Market Overview12.3 Current Reimbursement Scenario

13. South Korea CAR T Cell Therapy Market Outlook13.1 Approved & Key CAR T Cell Therapies in South Korea13.2 Future Market Opportunity

14. Australia CAR T Cell Therapy Market Outlook14.1 Approved & Key CAR T Cell Products in Australia14.2 Australia CAR T Cell Therapy Market14.3 Current Reimbursement Scenario

15. Taiwan CAR T Cell Therapy Market Outlook

16. Global CAR T Cells Clinical Pipeline Overview16.1 By Company16.2 By County16.3 By Indication16.4 By Phase

17. Global CAR T Cells Clinical Pipeline By Company, Indication & Phase17.1 Research17.2 Preclinical17.3 Clinical17.4 Phase-I17.5 Phase-I/II17.6 Phase-II17.7 Phase-III17.8 Registered

18. Marketed CAR T Cell Therapy Clinical insight18.1 Breyanzi18.2 CARVYKTI18.3 Yescarta18.4 Kymriah18.5 Abecma18.6 Tecartus

19. Global CAR-T Cell Therapy Market Dynamics19.1 Favorable Parameters19.2 Market Challenges

20. Competitive Landscape20.1 Autolus20.2 Bellicum20.3 Bluebird20.4 Celgene20.5 Cellectis20.6 Celyad20.7 Eureka Therapeutics20.8 Fortress Biotech20.9 Immune Therapeutics20.10 Juno Therapeutics20.11 Kite Pharma20.12 Novartis20.13 Sorrento therapeutics20.14 TILT Biotherapeutics20.15 Ziopharm

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Global CAR T Cell Therapy Market Research 2022: Opportunity & Clinical Pipeline Insights to 2028 - Insight On More Than 600 CAR T Cell Therapies...

JERSEY CITY, N.J., June 15, 2022 /PRNewswire/ --InsightAce Analytic Pvt. Ltd. announces the release of market assessment report on "Global Cell Therapy Packaging Products and Services Market (Therapy (T-cell Therapies, Dendritic Cell Vaccines, Stem Cell Therapies, NK Cell Therapies, and Other ATMPs), Package Engineering Design (Primary Packaging and Secondary Packaging), Scale of Operation (Clinical and Commercial)) By Trends, Industry Competition Analysis, Revenue and Forecast Till 2030"

According to the latest research by InsightAce Analytic, the global cell therapy packaging products and services market is expected to reach US$ 1,252.14 Million in 2030, recording a promising CAGR of 20.32% during the period of 2022-2030.

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Cell therapy aims to modify genetic material to treat different chronic diseases. Cell and gene therapy is the most significant medical advance in recent history. The increasing investments by key players in the development of promising therapies and advanced packaging technologies are anticipated to fuel the market growth over the forecast period.

Biopharmaceutical companies are investing in developing and manufacturing new customizable "patient-centered medicine" and modernizing their supply chains. Although biopharmaceutical firms' primary focus is on the drug product (DP) research and production and delivery methods (e.g., syringes), packaging and labelling are crucial to ensuring product quality and efficacy. The packaging of cell and gene therapy products must maintain closure integrity and product stability and allow simple access to the product while remaining functional during heat and mechanical loads experienced. The packaging must be designed to endure cryogenic temperatures without compromising the quality of the biological material or its longevity. For instance, In February 2022, Sharp, a leading provider in contract packaging and clinical supply services, has designed new purpose-built production suites to fulfil the rising demand from producers of gene treatments for dedicated and specialized packaging capacity. The innovative secondary packaging by Sharp aims to give an appropriate environment for tackling the challenges of gene treatments, notably at low temperatures and distribution in cold and ultra-cold supply chains. In summary, packaging technology and engineering, graphics, and labelling design are vital components of the development and marketing of gene and cell therapy programs.

Major driving factors of the cell therapy packaging products and services market are the increasing need for cell therapies, advancements in packaging and labelling, high prevalence of cancer diseases. Furthermore, advanced medical technologies, the rising trend of outsourcing in the healthcare industry, and the ongoing efforts of service providers to further improve their portfolios are enhancing the growth of the cell therapy packaging products and services market. However, the high cost of manufacturing systems, lack of standard therapy protocols, and complex procedures are restraining the growth of this market.

Geographically, the North America region is the primary revenue holder of this market due to rising awareness about cell and gene therapies, increasing government investments in the research and development of cell therapies, stringent regulations and an increasing number of human chronic diseases. On the other hand, Europe will also dominate the market during the forecast period due to advancements in the biopharmaceutical field and stringent regulations. The Asia-Pacific market is expected to grow faster in the future due to the growing cell therapy manufacturing industries and the adoption of new technologies.

Major key players in the cell therapy packaging products and services market areAlmac, Catalent Pharma Solutions, Cryoport Systems, Core Cryolab Inc., Yourway, Lufthansa Cargo, Saint-Gobain Life Sciences, Thermo Fisher Scientific, Sharp, West Pharmaceutical Services, Chart Industries Inc., and Other Prominent Players. Leading manufacturers in this field focus on novel therapy innovations, partnerships, collaborations, mergers, and agreements. These strategies will help to boost their growth opportunities in this market.

Key Developments:

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Market Segments

Market Size (Value US$ Mn) & Forecasts and Trend Analyses, 2022 to 2030 based on Therapy

Market Size (Value US$ Mn) & Forecasts and Trend Analyses, 2022 to 2030 based on Package Engineering Design

Market Size (Value US$ Mn) & Forecasts and Trend Analyses, 2022 to 2030 based on Scale of Operation

Market Size (Value US$ Mn) & Forecasts and Trend Analyses, 2022 to 2030 based on Region

North America cell therapy packaging products and services market revenue (US$ Million) by Country, 2022 to 2030

Europe cell therapy packaging products and services market revenue (US$ Million) by Country, 2022 to 2030

Asia Pacific cell therapy packaging products and services market revenue (US$ Million) by Country,2022 to 2030

Latin Americacell therapy packaging products and services market revenue (US$ Million) by Country, 2022 to 2030

Middle East & Africa cell therapy packaging products and services market revenue (US$ Million) by Country, 2022 to 2030

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Other Related Reports Published by InsightAce Analytic:

Global Cell and Gene Therapy Bioassay Services Market

Global Vaccine Cold Chain Logistics Market

Global Cell and Gene Therapy Drug Delivery Devices Market

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InsightAce Analytic is a market research and consulting firm that enables clients to make strategic decisions. Our qualitative and quantitative market intelligence solutions inform the need for market and competitive intelligence to expand businesses. We help clients gain competitive advantage by identifying untapped markets, exploring new and competing technologies, segmenting potential markets and repositioning products. Our expertise is in providing syndicated and custom market intelligence reports with an in-depth analysis with key market insights in a timely and cost-effective manner.

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Cell Therapy Packaging Products and Services Market worth $1.25 Billion by 2030 - Exclusive Report by InsightAce Analytic - PR Newswire

ACE1831 is a potential antibodyarmed allogeneic gamma delta T cell therapy developed using Acepodia's unique antibody-cell conjugation (ACC) technology as an optimized T cell engager platform to treat patients with non-Hodgkin's lymphoma

ALAMEDA, Calif. and TAIPEI, Taiwan, June 20, 2022 /PRNewswire/ -- Acepodia, a clinical-stage biotechnology company developing first-in-class cell therapies with its unique Antibody-Cell Conjugation (ACC) platform technology to address gaps in cancer care, today announced that it has received clearance of its Investigational New Drug (IND) application from the US Food and Drug Administration (FDA) to initiate a Phase 1, first-in-human, multi-center clinical study of its ACE1831 in patients with non-Hodgkin's lymphoma.

"The FDA clearance of our IND application for ACE1831 is a significant milestone for Acepodia as we move into the clinic with a first antibody armed allogeneic gamma delta T cell product candidate through our unique ACC platform. Based on ACE1831's encouraging preclinical data, we believe that our antibody armed gamma delta T cell therapy has the potential to provide additional treatment options for patients with NHL," said Sonny Hsiao, Ph.D., chief executive officer of Acepodia. "The ACC approach allows us to circumvent the limitations of current T cell engager therapies. Meanwhile, we can also significantly reduce manufacturing costs and has the potential to generate a cost-effective cancer treatment for patients. We look forward to advancing ACE1831 into its first clinical trial," said the chief executive officer.

About Gamma-Delta () T CellsAcepodia's gamma delta T cell program harnesses the unique properties of gamma delta T cells to develop a new class of allogeneic cell therapies for the treatment of cancer. Gamma delta T cells have characteristics of both the innate and adaptive immune systems that make them an ideal chassis for the development of cell therapies. This cell type can recognize and attack cancerous cells as well as coordinate a broad antitumor immune response by recruiting other immune factors and cells to the site of disease. Gamma delta T cells have also been shown to preferentially traffic to distinct tissues and could be ideally suited for more targeted treatment of certain types of cancers.

About AcepodiaAcepodia is a clinical-stage biotechnology company developingfirst-in-class cell therapies with its unique Antibody-Cell Conjugation (ACC) platform technology to address gaps in cancer care. Leveraging its ACC technology, the company links tumor-targeting antibodies to its proprietary immune cells, such as gamma deltaT cells to create novel antibody cell effector (ACE) therapies, which have increased binding strength against tumors that express low levels of tumor antigens.

Acepodia is made up of seasoned leadership and scientific experts dedicated to advancing its robust pipeline of ACE therapies with the potential to bring innovative, effective and affordable cell therapies to a broad population of patients across a variety of solid tumors and hematologic cancers. For more information, visithttps://www.acepodia.comand follow Acepodia onTwitterandLinkedIn.

About non-Hodgkin's lymphomaNHL is the most common cancer of the lymphatic system that develops in white blood cells called lymphocytes. Because lymph tissue is found all through the body, lymphoma can begin almost anywhere. The most common type of NHL in adults is diffuse large B-cell lymphoma, which is usually aggressive, accounting for about 30% of NHL diagnoses. Most types of NHL are incurable with available therapies, and more than 500,000 new cases of NHL are diagnosed each year worldwide.

Investor ContactAcepodiair@acepodiabio.com

Media Contactpr@acepodiabio.com

Gina LeeDirector of Communications and Public AffairsT. +886-2-2697-6100 ext.131M. +886-919-259-599E. gina@acepodiabio.com

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Acepodia Announces FDA Clearance of IND Application for ACE1831, an Anti-CD20 Armed Allogeneic gamma delta T-cell Therapy Candidate to Treat Patients...

We all love to reminisce about the good old days! Memories are part of our life and everyone reflects upon the good and bad times spent. Unfortunately, with a steadily increasing incidence of Alzheimers disease globally, many are losing their memoriesin fact even losing the ability to remember basic activities that do not as such require conscious thought.What is Alzheimers disease?Alzheimers disease (AD) is one of the major causes of dementia, and as of the last decade, there are approximately 3.7 million Indians with the condition. The lack of awareness regarding the early symptoms of the disease and knowledge about the associated risks poses challenges in early diagnosis and prompt interventions. Moreover, one cannot prevent or reverse the disease process.AD begins in individuals around the age of 40-50 years when certain unwanted proteins accumulate in the brain. These protein deposits interfere with the connections in the brain and disrupt the transmission of informationconsider them roadblocks that cannot be crossed.Thus, with time and depending on the extent of protein accumulation, a patient begins to forget things and gradually fails to form a link between the sensory information gained and the required action. For example, in advanced stages, apart from being unable to recognise people (including family members) a patient might not know what to do with a toothbrush or how the lock on a door should be fastened, although these are things he/she would have been doing for years together. It is a sad sight to watch a family member deteriorate in this manner. Since such episodes affect the overall quality of life of the patient, caregivers also tend to face the burden.How can this be treated?Unfortunately, there are no medications to treat the disease. Thus, the least we can do is try to slow down the progression of the condition. To begin with, annual health check-ups must be advised to everyone over the age of 45 years, adding brain imaging where required. By this we might be able to identify AD early and initiate activities and treatment that can slow the progression, says Dr Pradeep Mahajan, Regenerative Medicine Researcher.We have stem cells and growth factors in our body, which function to control inflammation, enhance the functions of other cells, and provide a constant pool of healthy cells to regenerate lost tissues. Through cell-based therapy, we are only providing these molecules at the required location in the appropriate quantity, explains Dr Mahajan.He continues, There are several other molecules, for example, chaperones (helper proteins) and exosomes (cell-associated packets of genetic material, proteins, immune cells, etc.) that act as messenger molecules, and also help in de-tangling nerve fibres and protein deposits in the brain. These can be utilised to create a healthier environment in the brain and restore internal balance (homeostasis).With more targeted therapies being researched, it might be possible to reverse AD in the future. For now, though, regenerative medicine can be a beacon of hope for patients to help lead an independent life for as long as possible in a natural, minimally invasive manner, concludes Dr Mahajan.

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How stem cell therapy can help with Alzheimer's disease - Times of India

BOSTON & SEATTLE & EMERYVILLE, Calif.--(BUSINESS WIRE)--Affini-T Therapeutics, Inc., a biotechnology company unlocking the power of T cells against oncogenic driver mutations, and Metagenomi, Inc., a genetic medicines company with a versatile portfolio of next-generation, wholly-owned gene editing tools, today announced a partnership to enable Affini-Ts next generation ex vivo T cell receptor (TCR) cell therapies for solid tumor patients using Metagenomis novel proprietary gene editing systems.

We are delighted to announce our partnership with Metagenomi and to apply their next-generation gene editing systems to our cell therapies targeting oncogenic and viral driver genes, including KRAS and p53, to provide the greatest impact for patients across a variety of hard-to-treat solid tumor types, said Jak Knowles, M.D., Co-founder, President and Chief Executive Officer, Affini-T Therapeutics. By working with Metagenomi, we will gain access to powerful, novel gene editing tools to make precise and multiplex edits to immune cells, thereby optimizing the effector function of our cell-based therapeutics.

With this collaboration, Metagenomi is executing on its ex vivo therapeutics partnering strategy in the immuno-oncology space. We are especially excited to partner with the outstanding team at Affini-T and its scientific founder Dr. Phil Greenberg, who has fundamentally advanced this field. We believe that cell therapy, combined with our powerful gene editing tools, is the future of immuno-oncology, treating patients with the highest unmet medical need, said Brian C. Thomas, Ph.D., Founder and Chief Executive Officer of Metagenomi. Our strategy in cell therapy strives to create a broad pipeline of licensed, partnered and in-house development programs with leading scientific teams.

The partnership will leverage Metagenomis proprietary gene editing systems to complement Affini-Ts state-of-the-art TCR discovery and synthetic biology platforms to generate groundbreaking cell therapy products. Affini-T will have the option to exclusively license Metagenomis technology to make gene edits in autologous TCR T cell therapies for specific tumor targets, with the option to expand non-exclusively to editing certain allogeneic approaches. In the future the parties will discuss further targets for co-development and co-commercialization.

Under the terms of the agreement, Metagenomi will be entitled to receive tiered payments for each optioned cancer target plus additional milestone and royalty payments.

About Metagenomi

Metagenomi is a gene editing company committed to developing potentially curative therapeutics by leveraging a proprietary toolbox of next-generation gene editing systems to accurately edit DNA where current technologies cannot. Our metagenomics-powered discovery platform and analytical expertise reveal novel cellular machinery sourced from otherwise unknown organisms. We adapt and forge these naturally evolved systems into powerful gene editing systems that are ultra-small, extremely efficient, highly specific and have a decreased risk of immune response. These systems fuel our pipeline of novel medicines and can be leveraged by partners. Our goal is to revolutionize gene editing for the benefit of patients around the world. For more information, please visit https://metagenomi.co/.

About Affini-T Therapeutics

Affini-T is unlocking the power of T cells and targeting core oncogenic drivers to develop potentially curative therapies for solid tumor cancers. Our differentiated cell therapy platform harnesses state-of-the-art engineering and synthetic biology capabilities to target even the most devastating cancer-driving mutations, beginning with KRAS. We leverage these tools to optimize T cell functions and rewrite the rules of the solid tumor microenvironment, enabling the potential for sustained clinical outcomes in patients. Building on the world-class innovation inherent in our leadership team, founders and technologies, we are powered to develop transformational medicines that last. Follow us on LinkedIn and Twitter.

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Affini-T Therapeutics & Metagenomi Announce Next-Generation Gene Editing Partnership to Advance Cell Therapies for Solid Tumor Patients - Business...

Sponsored Research Agreement (SRA) with Penn advances multichain KIR-CAR platform targeting solid tumors

PHILADELPHIA, June 21, 2022 /PRNewswire/ -- Verismo Therapeutics, a recent University of Pennsylvania spin-out company behind the novel KIR-CAR platform technology for CAR T-cells, today announces that Penn and Verismo entered into a Sponsored Research Agreement (SRA). Under the SRA, Verismo is funding additional KIR-CAR T-cell focused preclinical research programs at Penn to advance new potential treatment options for solid tumor indications. The research funded under the SRA is expected to be conducted over four years, from 2021 to 2025, in the Penn laboratories of Michael C. Milone, MD, PhD, an associate professor of Pathology and Laboratory Medicine, and Donald L. Siegel, MD, PhD, a professor of Pathology and Laboratory Medicine.

Dr. Milone, with Dr. Carl June, is the co-inventor of the CTL019 CAR T product that became the first FDA-approved CAR T cell therapy, and he is the lead inventor of the KIR-CAR technology. Under the SRA, Dr. Milone and his lab are working to advance the KIR-CAR platform engineering and are performing key preclinical research for the purpose of enabling an IND for future clinical studies involving the KIR-CAR platform.

Dr. Siegel is a leading innovator in phage display technology. Under the SRA, Dr. Siegel and his lab are utilizing this technology to generate novel antibodies to cancer targets, which will expand Verismo's product pipeline and potentially enable the treatment of additional tumor indications.

"Our ongoing collaboration with Penn brings us closer to our goal to treat solid tumors. Dr. Milone and Dr. Siegel have a clear vision of what scientific advances are needed to advance our progress and pipelines. I am hopeful that the collaboration between Penn and Verismo will accelerate the development of the KIR-CAR therapy and ultimately bring a new much needed treatment option to patients with solid tumors," said Dr. Bryan Kim, CEO of Verismo.

Editor's Note: Drs. June, Milone, Siegel, and Penn are equity holders in Verismo. Penn has licensed certain Penn-owned intellectual property to Verismo and Drs. June, Milone, and Siegel, along with Penn, may receive financial benefits under the license in the future. Penn'sPerelman School of Medicine receives sponsored research funding from Verismo to support certain research and development programs at Penn.

ABOUT VERISMO THERAPEUTICS

Verismo Therapeutics is a pioneer in dual-chain KIR-CAR T technology, on track to bring its first asset into first-in-human clinical trials in 2023. Verismo is the only company developing the KIR-CAR platform, amodified NK-like receptor designed to improve persistence and efficacy against aggressive solid tumors. The KIR-CAR platform technology was developed specifically for advanced solid tumors, an area of high unmet medical need. For more information, visit:www.verismotherapeutics.com

ABOUT KIR-CAR PLATFORM

The KIR-CAR platform is a dual-chain CAR T cell therapy and has been shown in preclinical models to be capable of maintaining antitumor T-cell activity even in challenging solid tumor environments. Based on natural killer cell receptors, KIR-CAR provides a natural on-and-off stimulation to the T-cell, without triggering T cell exhaustion. DAP12 costimulatory chains aid additional T-cell stimulating pathways, further improving cell persistence. This continued function and persistence can lead to ongoing regression of solid tumors in preclinical models, including those refractory to traditional CAR T-cell therapies. Furthermore, the KIR-CAR platform can be combined with many additional emerging technologies, such as in-vivo gene editing, advanced T cell selection, combination therapies, and even allogeneic cellular therapies to provide an adaptable tumor-targeting therapy for patients in need.

Contact: Alyson Kuritz, 908-892-7149, alyson@0to5.com

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Verismo Therapeutics Announces Research Partnership with the University of Pennsylvania - BioSpace

SYDNEY, June 21, 2022 (GLOBE NEWSWIRE) -- With the once dominant amyloid hypothesis of Alzheimer's disease suffering yet another crushing blow this week, the field is desperate for a ray of hope. Enter S2N, a new Australian biotech pioneering an all-new neurorestorative approach, aiming to rebuild and replace the lost brain cells in Alzheimer's that underlies clinical symptoms.

In a world first, a veterinary trial led by S2N suggests the audacious concept may work. S2N's new form of cell therapy reversed the dementia-like syndrome that strikes down many older pet dogs with Alzheimer's.

Co-founder Professor Michael Valenzuela explains, "Because of deep parallels between the canine brain and human brain, and canine Alzheimer's and human Alzheimer's, I started this trial 10 years ago with the assumption that if it's going to work in humans, then it needs to work in dogs first. And the results exceeded my wildest expectations."

Dementia was reversed in more than half of the canine patients, with a clinically meaningful improvement in 80%. For many of the carers, it was a life-changing turnaround, some at the point of considering euthanasia before treatment.

Fiona Gibbs, carer of Leo, a 12-yo Pomeranian in the trial, describes the impact: "Before treatment, Leo was really bad, forgetting who we were, getting lost, and having these unpredictable episodes where he would growl and snap - it was really scary and we just couldn't go on. A few months after treatment, he started getting better, and then he was back to his normal self, and we look back at the movies and think, 'Wow, was he really that bad?'"

Leo's life-changing improvement lasted almost two years, typical of clinical recovery in the trial.

And when Valenzuela looked in the brain, the findings were remarkable, "The hippocampus, the memory centre of the brain, was packed with baby neurons and new synapses, precisely where we delivered the cells. Compared to untreated dogs, it was like night and day".

Importantly, microscopic analysis confirmed the dogs had classic Alzheimer pathology. In other words, the cell therapy worked in the setting of natural disease, a first of its kind.

"Given our doggie patients also had many of the same health issues that older people face, it gives me even greater confidence," says Valenzuela.

Stem cell pioneer Professor Brent Reynolds of the University of Florida, not connected to the study, considers it a landmark in the quest to treat brain degeneration. "Alzheimer's is an area of medicine that needs new thinking. What stands out are clinically meaningful outcomes in a natural canine model of this devastating disease. Also, the company's approach to generating cells from the same patient could solve many of the problems facingcell therapies."

The study helps pave the way for S2N to launch a world-first human trial in 2024.

Contact

Professor Michael Valenzuela

Co-founder & CEO, Skin2Neuron Pty Ltd.

mvalenzuela@skin2neuron.org

+61 413 603 784 (AEST)

Related Images

Image 1: Figure 1/2

Hippocampus (memory centre) of an older dog with dementia-like syndrome successfully treated with S2N's cells. It is packed with green cells that are new neurons (brain cells) and yellow dots, new synapses (connections between brain cells).

Image 2: Figure 2/2

Same brain area in an aged untreated dog. There are no new neurons (no green cells), a few red dots (old synapses), but no new synapses (no yellow dots).

This content was issued through the press release distribution service at Newswire.com.

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New Alzheimer's Treatment on Horizon as Dementia Reversed for First Time in Dogs - GlobeNewswire

At the 25th Annual Meeting of The American Society of Gene & Cell Therapy in May 2022, UNC Ophthalmology Associate Professor Matt Hirsch, PhD, was among four established gene therapy researchers across the U.S. to be honored as recipients of the ASGCTs 2022 Outstanding New Investigator Award.

At the 25th Annual Meeting of The American Society of Gene & Cell Therapy in May 2022, UNC Department of Ophthalmology Associate Professor Matt Hirsch, PhD, was among four established gene therapy researchers across the U.S. to be honored as recipients of the ASGCTs 2022 Outstanding New Investigator Award.

Hirsch is a leading genetic engineer at UNC-Chapel Hill with a strong investigative record in using Adeno-Associated Virus (AAV) Vectors to treat rare degenerative hereditary diseases via therapeutic gene delivery and gene editing.

At UNCs Gene Therapy Center (GTC), Hirsch has been a long-time lead investigator of collaborative, cross-disciplinary translational studies of AAV Vectors to treat a range of ocular genetic disorders, such as hereditary lysosomal storage diseases leading to corneal blindness and retinal disease. His investigative contributions helped transform the Carolina Eye Research Institute (CERI) into a world-renowned cell and gene therapy center that houses large-scale, cross-disciplinary clinical trials to promote ophthalmic scientific innovation in restoring and preventing loss of eyesight. At the GTC, the Hirsch Lab has also developed targeted genetic therapy for treating muscular dystrophies and evaluated AAV large gene delivery contexts in disease models of dysferlinopathy and hemophilia A.

Hirsch has co-founded three UNC GTC-affiliated start-ups, including RainBio, a biotechnology company focused on restoring vision in patients who have experienced corneal blindness by replacing the missing Mucopolysaccharidosis type 1 (MPS1) gene via (AAV) Vector gene therapy. Over secured seven U.S. patents, led 24 federally and industry-funded investigations, published 10 textbooks and chapters, authored 45 refereed articles, and mentored eight PhD candidates.

Within the gene delivery and cell therapy research community, Hirsch and his lab colleagues are known for their investigative development of integrating novel and existing approaches to exploit host DNA repair pathways for intracellular large transgene reconstruction. Their work enables others to pursue further identification of host DNA repair proteins and pathways necessary for episomal genetic engineering at the basic science level, toward advancing generation of safer, more efficient clinical reagents in lab experimentation and testing.

Annual winners of the ASGCTs Outstanding New Investigator (ONI) Award are leaders in academia, research foundations, government and industry. At the May 2022 ASGCT Annual Meeting, the contributions of each award winner to the field of gene and cell therapy were recognized, and each awardee presented a plenary lecture highlighting their scientific accomplishments that led to their Award.

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Hirsch Among Outstanding New Investigators at ASGCT 2022 Annual Meeting | Newsroom - UNC Health and UNC School of Medicine

Anita Kumar, MD, medical oncologist with Memorial Sloan Kettering Cancer Center, led a session at the 2022 Annual Meeting of the American Society of Clinical Oncology (ASCO), on New Directions in Mantle Cell Lymphoma.

In the United States, about 4000 people per year are diagnosed with mantle cell lymphoma (MCL), a cancer that develops from malignant B-lymphocytes in an area of the lymph node known as the mantle zone.1

As Anita Kumar, MD, a medical oncologist with Memorial Sloan Kettering Cancer Center explained to lead off a session at the 2022 Annual Meeting of the American Society of Clinical Oncology (ASCO), MCL is associated with chronic activation of the B-cell receptor complex. This, she said, has allowed for the development of Bruton tyrosine kinase (BTK) inhibitors, such as ibrutinib, as well as PI3 kinase inhibitors.

Many factors guide decisions on initial treatment after diagnosis, such as the patients age, fitness, and especially transplant eligibility. In recent years, the chemoimmunotherapy combination of bendamustine and rituximab has become the standard of care for patients who are not eligible for autologous stem cell transplant (ASCT).

However, mantle cell lymphoma is both clinically and biologically heterogeneous, Kumar said. And this really challenges the one-size-fits-all approach using chemoimmunotherapy across the board. We know this from our clinical practice.

Kumars talk on approaches for treatment-nave MCL anchored the ASCO session she chaired, New Directions for Mantle Cell Lymphoma in 2022. The June 6 session also featured talks by Toby Eyre, MBChB, MRCP, consultant hematologist at the Department of Hematology, University of Oxford, who discussed prognostic markers in MCL; and Chan Cheah, clinical professor of medicine, University of Western Australia. Cheah focused on novel therapies in relapsed/refractory settings, including the use of chimeric antigen receptor (CAR) T-cell therapy and bispecific antibodies to treat MCL.

Eyre offered an overview of current risk stratification of MCL patients, which highlighted the importance of the TP53 mutation as an extremely strong predictor of inferior overall survival (OS) in trials across several new therapeutic classes. Clinical factors can be useful, but not at the expense of testing for TP53 mutations, he said.

The use of the MCL International Prognositic Index (MIPI), combined with a measure of proliferation of the Ki-67 protein allows clinicians to group MCL patients into one of 4 risk categories for diagnostic, treatment, and clinical trial purposes, he said.

Kumar followed with a case for stratification, as she discussed the range of conditions among newly diagnosed MCL patients. There are patients with non-nodal leukemic MCL whose disease is indolent; often, these patients can be monitored for years. And on the other end of the spectrum, we see blastoid mantle cell lymphoma that's highly aggressive with high proliferation; oftentimes with evidence of chemoresistance and inferior survival, she said.

Investigators are learning more about the biology of these different presentations, Kumar said. More conventional MCL is associated with greater genomic instability and TP53 mutation, along with inferior OS. In addition, minimal residual disease assessment has allowed for novel treatment paradigms to be explored in mantle cell lymphoma, she said, as she reviewed studies that linked MRD status to remission duration. MRD, she said, has the potential as a surrogate end point, as a marker to predict relapse during surveillance or therapeutic resistance, and to potentially help doctors decide when treatment can cease or less treatment is needed.

A case in point: in the Cooperative Group Study EA4151, newly diagnosed transplant eligible patients are randomized after induction therapy to ASCT with rituximab maintenance or just rituximab.2 This is an exciting clinical trial design, because it leverages MRD assessment as a prognostic biomarker, and potentially in patients who achieve a deep molecular remission investigates whether we can omit upfront autologous stem cell transplant, she said.

As with other types of lymphoma, theres discussion whether to move newer targeted therapies and other non-chemotherapy treatments into earlier lines of treatment, where they might be more effective with fewer toxic effects. Kumar outlined this discussion in light of results from the SHINE trial, presented earlier at ASCO, which showed adding ibrutinib to bendamustine and rituximab offered a progression-free survival (PFS) benefit but no OS benefit.3

She said the Triangle study will examine 870 younger, fitter patients examine 3 arms: one with alternating regimens of well-known chemotherapy combinations followed by ASCT, one with ibrutinib added to one of the chemotherapy combinations followed by ASCT, and one with the chemotherapy combinations and ibrutinib only.4 Again, Kumar said, the trial may demonstrate an ability to achieve better outcomes without ASCT.

Chemotherapy-free combinations, including those that include BTK inhibitors, are a major area of study. Kumar reviewed these findings:

Concern about the cardiovascular effects of ibrutinib has led to trials with zanubrutinib, a second-generation BTK inhibitor. A 500-patient, phase 3 clinical trial of 2 different regimens of zanubrutinib with bendamustine vs zanubrutinib alone is under way.6

Triplet combinations are now being studied in MCL. Kumar reviewed the results for OASIS, which studied ibrutinib, obinutuzumab, and venetoclax in 15 mostly low-to-intermediate MIPI patients, of whom 2 had a TP53 mutation.7 The idea here is to leverage dual BTK and BCL2 inhibition, which is known to be synergistic in MCL, Kumar said. The PET complete response (CR) at 6 months was 86.6%; after a median follow-up of 14 months, 1-year PFS was 93%.

This establishes this triplet combination as a highly active treatment program in mantle cell lymphoma, and it was also demonstrated that this was well tolerated, she said. Of great interest, we see that this combination was active in patients who had a TP53 mutation as well as blastoid disease.

Kumar concluded with 2 other trials of interest to payers:

We really recommend enrollment in a clinical trial for patients who have a TP53 mutation, Kumar said. Insights into mantle cell lymphoma disease biology have improved our biologic risk stratification, and certainly identification of a TP53 mutation at time of initial diagnosis is of great importance.

References

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Challenging the One-Size-Fits-All Approach in Early MCL Treatment - AJMC.com Managed Markets Network

Several treatment options are being explored for patients who have residual disease or who relapse after CAR T-cell therapy, including a possible second infusion with the same CAR T-cell product.

Young patients with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) who had a suboptimal response or relapse after chimeric antigen receptor (CAR) T-cell therapy could benefi t from reinfusion, according to a retrospective review (NCT03827343).

Our study provides important insights into [second CAR T-cell infusion] outcomes, including with antigen targets beyond CD19, the study authors, including corresponding author Nirali N. Shah, MD, MHSc, of the National Cancer Institute (NCI), wrote in their report published in the Journal for ImmunoTherapy of Cancer.

Several treatment options are being explored for patients who have residual disease or who relapse after CAR T-cell therapy, including a possible second infusion with the same CAR T-cell product.

The retrospective review looked at patients with B-ALL who received a reinfusion in 1 of 3 phase 1 NCI-based clinical trials (NCT01593696, NCT02315612, NCT0344839). At fi rst CAR T-cell therapy infusion, patients received either an anti-CD19, anti-CD22, or an anti-CD19/CD22 agent. If the patients had relapse after complete remission (CR) or suboptimal response and CAR expansion, they were offered a second infusion (n = 18).

The primary aim of the review was to assess CR rates of second infusions and the rate of adverse events (AEs). Secondary goals included the characterization of CAR expansion and antigen expression as well as the impact of the intensity of lymphodepletion.

A total of 136 patients were treated across the 3 studies, but only 18 (13.2%) went on to receive a second infusion with the same CAR product. As of the time of the second infusion, the median age of patients was 19 (range, 8-31). A majority of the patients were male (88.9%), White (66.7%), and of non-Hispanic ethnicity (83.3%). As of the second infusion, 88.9% had active medullary disease, 22.2% had low marrow involvement, 66.7% had high disease burden, and 11.1% had isolated CNS disease.

Patients had received a median of 6 lines of prior therapy (range, 2-13), including prior hematopoietic stem cell transplantation (HSCT) in 77.8%, prior immunotherapy in 44.4%, and prior alternate CAR T-cell therapy in 50%.

Seven patients (38.7%) had a suboptimal response to the fi rst infusion of either partial response or stable disease, including 4 who did not have a CR from first infusion, and 11 patients (61.1%) had an antigen-positive relapse; all 18 patients had CAR expansion. The median time between the fi rst and second infusion was 116.5 days (range, 35-373), and 38.9% received therapy in between.

The second infusion was administered at the same dose for 15 patients (83.3%), at a lower dose in 2 patients (11.1%) and a higher dose in 1 (5.5%).

Seven patients (38.9%) achieved an objective marrow response to the second infusion, with 5 of these patients (71.4%) achieving a minimal residual disease (MRD) negative CR, and 6 (33.3%) had a morphologic CR.

Long-term survival with second CAR infusion was limited. All patients eventually relapsedincluding 2 with CNS disease, 3 with medullary relapse, and 1 with both medullary and extramedullary relapse and none were eligible for HSCT. The median duration of remission was 77 days (range, 54-292).

Our results suggest that diminished CAR T-cell expansion alongside antigen downregulation and loss impeded robust responses to CART2. Further exploration of the mechanisms underlying CART2 response is needed, Shah et al wrote.

Cytokine release syndrome (CRS) was observed in 83.3% of patients with fi rst infusion, including severe CRS of grade 3 or higher in 27.8%. Only 22.2% reported CRS with second infusion at a maximum severity of grade 1. Neurologic toxicity was reported with fi rst infusion in 22.2% and with second infusion in 11.1%.

Observed peripheral blood CAR T-cell expansion was higher with the fi rst infusion than the second (median, 24.05 vs 1.69 cells/mL, respectively; P = .03). Five patients (27.8%) showed no CAR expansion post second infusion. CAR expansion tended to be higher with the CD22-targeted agent than the other CAR T-cell products.

REFERENCE:

Holland EM, Molina JC, Dede K, et al. Efficacy of second CAR-T (CART2) infusion limited by poor CART expansion and antigen modulation. J Immunother Cancer. 2022;10(5):e004483. doi:10.1136/jitc-2021-004483

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Second CAR T Infusion Could Lead to New Response in B-ALL - Targeted Oncology