Category Archives: Cell Therapy

A new technology for cellular immunotherapy developed by Abramson Cancer Center researchers at Penn Medicine showed promising anti-tumor activity in the lab against hard-to-treat cancers driven by the once-considered undruggable KRAS mutation, including lung, colorectal, and pancreatic.

The study, published in Nature Communications, successfully demonstrates using human cells that a T-cell receptor, or TCR, therapy could be designed to mobilize an immune system attack on mutated KRAS solid tumors and shrink them. The preclinical work has laid the groundwork for the first-in-human clinical trial now in the planning stages for the treatment of advanced pancreatic cancer in patients whose tumors harbor specific KRAS mutations and express a specific type of human leukocyte antigen, or HLA, the therapy is built to recognize.

Weve shown that targeting mutant KRAS immunologically is feasible and potentially generalizable for a group of patients with lung, colorectal, and pancreatic tumors, says senior author Beatriz M. Carreno, an associate professor of pathology and laboratory medicine in the Perelman School of Medicine and a member of the Center for Cellular Immunotherapies, the Abramson Cancer Center, and Parker Institute for Cancer Immunotherapy at Penn. We look forward to taking this research to the next level and closer to clinical study.

Read more at Penn Medicine News.

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New cell therapy shows potential against solid tumors with KRAS mutations | Penn Today - Penn Today

Bijal Shah, MD, MS, discusses the results of treatment with the chimeric antigen receptor T-cell therapy brexucabtagne autoleucel in the ZUMA-3 trial in patients with acute lymphoblastic leukemia.

Bijal Shah, MD, MS, an associate member in the Department of Malignant Hematology at Moffitt Cancer Center, discusses the results of treatment with the chimeric antigen receptor (CAR) T-cell therapy brexucabtagne autoleucel (Tecartus) in the ZUMA-3 trial (NCT02614066) in patients with acute lymphoblastic leukemia (ALL).

Shah hopes that he and his fellow investigators have done enough to show the FDA that this treatment is safe option to treat patients with ALL. Patients in the ZUMA-3 trial had a 71% complete remission (CR) rate with CAR T-cell therapy, which Shah says is unheard of in this population. These patients had progressed on previous therapies or after transplant. Patients achieved deep remissions and most had minimal residual disease negativity after treatment. These were real remissions, as opposed to cosmetic remissions, according to Shah.

This efficacy translated into pronounced benefits in relapse-free survival, duration of remission, and overall survival. Patients who achieved CR did not reach a median overall survival with CAR T. Shah feels that this is incredible for this patient population. CAR T-cell therapy was expected to be a meaningful treatment in ALL, and now the data show it can be used safely.

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ZUMA-3 Trial of CAR T Results in Impressive Efficacy for Patients With ALL - Targeted Oncology

PITTSBURGH, Aug. 04, 2021 (GLOBE NEWSWIRE) -- A new collaboration among UPMC Hillman Cancer Center, the University of Pittsburgh and New Jersey-based biotechnology company Avalon GloboCare Corp. (NASDAQ: AVCO) aims to develop new cancer immunotherapy approaches and streamline manufacturing processes to bring these powerful treatments to cancer patients within days instead of weeks.

Cancer immunotherapy, which stimulates and trains a patients own immune system to target and kill tumors while leaving healthy cells intact, is an effective treatment for many cancer patients. One of these therapies, chimeric antigen receptor (CAR) T-cell therapy, alters a patients own T-cells to kill their cancer cells. This approach has been successful for some patients with leukemias, lymphomas and more recently, multiple myeloma, but only a limited number of patients have been able to benefit from these therapies.

The new collaboration, led by Yen-Michael S. Hsu, M.D., Ph.D., director of the Immunologic Monitoring and Cellular Products Laboratory (IMCPL) at UPMC Hillman, seeks to develop next-generation CAR-based cellular therapies to make them accessible to a wider range of cancer patients.

CAR T-cell therapies approved by the U.S. Food and Drug Administration (FDA) are personalized therapies, made from the patients own cells. Current therapies use a DNA-based viral vector to engineer expression of the CAR against an antigen present on tumor cells. Patient cells are modified in the laboratory and infused back into the patient in a process that takes several weeks.

With Avalon GloboCare FLASH-CARtechnology, we will use an innovative messenger ribonucleic acid (mRNA)-based technology platform that will allow researchers to create CAR cellular therapies much faster than before -- in just one to two days, said Hsu. We also believe this approach will reduce toxicity and overall cost associated with current CAR T-cell therapies, meaning more cancer patients could be eligible for this type of cellular therapy.

The researchers are also using the technology to develop next-generation, personalized CAR T-cell therapies, including engineering cells that target more than one tumor antigen, enhancing their ability to target and kill cancer cells. Hillmans IMCPL and Avalon GloboCare are developing a treatment for patients with relapsed or refractory B-cell lymphoblastic leukemia and non-Hodgkin lymphoma. Human clinical trials are poised to begin in mid-2022.

Another goal, according to Hsu, is to make universal or off-the-shelf CAR-based cancer immunotherapies. Unlike personalized treatments, this universal cellular therapy will be derived from a healthy donor, manufactured in bulk and readily available to treat patients without delay

A clinician could order this cellular immunotherapy in the same way as antibody or oral cancer treatment, reducing the time a patient has to wait for treatment. Because this cellular therapy would be made in a large batch, the cost of manufacturing would be much lower, resulting in a lower cost of the final cellular therapy products for patients, said Hsu.

The researchers are also working to streamline and enhance the quality of CAR T-cell manufacturing with Avalons Point-of-Care Modular Autonomous Processing System onsite at UPMC Hillman, a National Cancer Institute-designated comprehensive cancer center.

As an FDA-registered, Foundation for Accreditation of Cellular Therapy-accredited laboratory, the IMCPL supports investigator-initiated research and technical expertise in translating laboratory research into clinical biologic products. The mission of the lab is to deliver high-quality and safe translation of cutting-edge scientific breakthroughs into cellular therapies for improving cancer care and human health.

About UPMC Hillman Cancer CenterUPMC Hillman Cancer Center is the regions only National Cancer Institute-designated Comprehensive Cancer Center and is one of the largest integrated community cancer networks in the United States. Backed by the collective strength of UPMCwhich is ranked No. 15 for cancer care nationally by U.S. News & World Reportand the University of Pittsburgh School of Medicine, UPMC Hillman Cancer Center has nearly 70 locations throughout Pennsylvania, Ohio, New York, and Maryland, with cancer centers and partnerships internationally. The more than 2,000 physicians, researchers, and staff are leaders in molecular and cellular cancer biology, cancer immunology, cancer virology, biobehavioral cancer control, and cancer epidemiology, prevention, and therapeutics. UPMC Hillman Cancer Center is transforming cancer research, care, and preventionone patient at a time.

About the University of Pittsburgh School of MedicineAs one of the nations leading academic centers for biomedical research, the University of Pittsburgh School of Medicine integrates advanced technology with basic science across a broad range of disciplines in a continuous quest to harness the power of new knowledge and improve the human condition. Driven mainly by the School of Medicine and its affiliates, Pitt has ranked among the top 10 recipients of funding from the National Institutes of Health since 1998. In rankings recently released by the National Science Foundation, Pitt ranked fifth among all American universities in total federal science and engineering research and development support.

Likewise, the School of Medicine is equally committed to advancing the quality and strength of its medical and graduate education programs, for which it is recognized as an innovative leader, and to training highly skilled, compassionate clinicians and creative scientists well-equipped to engage in world-class research. The School of Medicine is the academic partner of UPMC, which has collaborated with the University to raise the standard of medical excellence in Pittsburgh and to position health care as a driving force behind the regions economy. For more information about the School of Medicine, see http://www.medschool.pitt.edu.

About Avalon GloboCare Corp.Avalon GloboCare Corp. (NASDAQ: AVCO) is a clinical-stage, vertically integrated, leading CellTech bio-developer dedicated to advancing and empowering innovative, transformative immune effector cell therapy, exosome technology, as well as COVID-19 related diagnostics and therapeutics. Avalon also provides strategic advisory and outsourcing services to facilitate and enhance its clients' growth and development, as well as competitiveness in healthcare and CellTech industry markets. Through its subsidiary structure with unique integration of verticals from innovative R&D to automated bioproduction and accelerated clinical development, Avalon is establishing a leading role in the fields of cellular immunotherapy (including CAR-T/NK), exosome technology (ACTEX), and regenerative therapeutics. For more information about Avalon GloboCare, please visit http://www.avalon-globocare.com.

For the latest updates on Avalon GloboCare's developments, please follow our twitter at @avalongc_avco

Forward-Looking StatementsCertain statements contained in this press release may constitute "forward-looking statements." Forward-looking statements provide current expectations of future events based on certain assumptions and include any statement that does not directly relate to any historical or current fact. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors as disclosed in our filings with the Securities and Exchange Commission located at their website (http://www.sec.gov). In addition to these factors, actual future performance, outcomes, and results may differ materially because of more general factors including (without limitation) general industry and market conditions and growth rates, economic conditions, and governmental and public policy changes. The forward-looking statements included in this press release represent the Company's views as of the date of this press release and these views could change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of the press release.

Contact Information:Avalon GloboCare Corp.4400 Route 9, Suite 3100Freehold, NJ 07728PR@Avalon-GloboCare.com

Investor Relations:Crescendo Communications, LLCTel: (212) 671-1020 Ext. 304avco@crescendo-ir.com

Contact: Cyndy PattonMobile: 412-415-6085E-mail: PattonC4@upmc.edu

Contact: Asher JonesMobile: 412-639-6222E-mail: JonesAG@upmc.edu

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UPMC and Pitt Develop New Cancer Immunotherapy with Avalon - GlobeNewswire

The heavily regulated and complex biopharmaceutical industry has been slow to change and adopt automated practices. A shift toward automation is a vital step forward to making breakthrough therapies more scalable and viable to help save patients lives. While the hurdles must be carefully considered, it is now time to accelerate digital transformation to enable the application of smart technologies, and specifically, to advance the cell and gene therapy industry.Motivated by pandemic-driven changes in human resources and consumer behaviors, many companies in multiple sectors and regions were forced to accelerate their adoption of automated and other digital technologies. Many of these changes are here to stay.

In biopharma, automation and digital technologies are increasingly being incorporated throughout the entire drug development process, from the discovery of disease mechanisms to the industrial manufacture of regulatory approved therapies. Many laboratories are switching from paper records to electronic laboratory notebooks (ELNs) and digital laboratory information management systems (LIMS). This is the case in most biopharma labs as well as smaller, academic research institutions.

Many therapeutics begin in academic research laboratories, where basic research is performed to better understand disease pathways and identify novel targets for potential treatment. Transitioning a therapy from an academic lab to commercial scale can be particularly challenging for cell and gene therapies as academic teams may not focus on the scalability of production methods for industrial manufacturing.

Additionally, the requirements for transitioning from small-scale to large-scale manufacturing facilities can be stringent, especially when Good Manufacturing Practice (GMP) regulations must be met. To make the production methods originally devised at the research bench useable at scale, they may need to be radically altered, for example, by altering a batch production process into an automated continuous one.

Fortunately, the scale required for autologous cell therapies is not large. But not all cell therapies will be required only in small, single lots. The priority placed on developing allogeneic cell therapies, which are sourced from donors who are unrelated to the patient, means that large-scale production methods will also be needed, to produce large volumes of these therapies for multiple patients.

While genomic therapies, like traditional small molecule drugs, often include synthesis steps, they also often employ living cells in their manufacture and are relatively large, akin to the production and size of biologic drugs. These and other similarities may account for why pre-existing technologies and methods have largely been transplanted from the other two major drug modalities to discover and produce genomic medicines.

However, these transplanted technologies and methods are not always all that well suited to genomic medicines. Technologies and methods stemming from disparate processes and workflows can make it more difficult to incorporate digital transformation and automation, resulting in islands of automation. Clearly, there is a need for dedicated solutions designed specifically for gene and cell therapies.

These solutions can take many forms, including a digital approach. By focusing on key pain points expressed by their partners in the cell and gene therapy industry, cell therapy experts have determined that there are generally two kinds of solutions needed. One needs to address key bottlenecks within the various processes in the drug discovery and drug-making journey, and the other needs to address more overarching issues within the broader workflow. This includes solutions designed to better integrate various steps in the drug discovery, development and manufacturing processes, as well as enable more end-to-end visibility and control.

As more biopharma companies and academic institutions digitally transform their operations and infrastructure, automation, including that driven by artificial intelligence, can deliver much-anticipated improvements in terms of reducing hands-on time, resource utilization, and even risk, while at the same time increasing process and product consistency, scalability, and regulatory compliance.

Although hurdles remain, such as the continued need for bespoke solutions for this relatively new drug modality, the future is bright as the COVID-19 vaccines herald the advent of the genomic medicines age one where we hope to see realized the promise of cell and gene therapies to deliver long-term remission and even cures for patients with some of the most difficult-to-treat diseases.

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Automating Manufacturing Is Critical for Advancing Cell and Gene Therapies - Technology Networks

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SAN DIEGO, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer, today announced the appointment of Yuan Xu, Ph.D., to its Board of Directors as an independent director. Dr. Xu brings to Fate Therapeutics over 25 years of discovery, development, manufacturing, and commercial experience in the global biopharmaceuticals business, most recently serving as the Chief Executive Officer and Board Member of Legend Biotech Corporation where she led the companys efforts in advancing ciltacabtagene autoleucel (cilta-cel) from proof-of-concept in 2018 to BLA preparation in 2020.

Yuan is an accomplished leader and innovator with extensive experience in guiding development and scaling manufacture of novel therapies, including CAR T-cell therapies such as cilta-cel, saidScott Wolchko, President and Chief Executive Officer ofFate Therapeutics. We are excited to work with Yuan, and we look forward to benefiting from her deep domain expertise as the Company advances its off-the-shelf, iPSC-derived NK and T-cell product candidates further into clinical development, transitions to large-scale manufacture, and establishes technical operations to support commercial approval.

Dr. Xu joined Legend Biotech Corporation in March 2018 as chief executive officer and as a director, playing a leading role in the companys IPO, clinical development of the autologous CAR T-cell therapy cilta-cel, and partnership with Janssen until her resignation in August 2020. Prior to Legend, Dr.Xu was Senior Vice President at Merck from August 2015 to August 2017, where she led discovery, preclinical and technical development, and manufacture of the Biologics & Vaccines subdivision. Dr. Xu was Vice President at Gilead from March 2014 to August 2015, where she led biologics and vaccines development and oversaw all operational aspects of the companys Oceanside manufacturing facility as Site Head, and was Vice President at Novartis from 2008 to 2014, where she led several functions in the U.S. and Europe including the biotherapeutics development unit focusing on innovative medicines such as engineered cell therapies, gene therapies, and antibody drug conjugates. Dr. Xu currently serves as an independent director on the board of directors of Akero Therapeutics, Inc. (Nasdaq: AKRO).

Fate Therapeutics has pioneered the field of iPSC-derived cell therapy, and has established a clear leadership position in the development of off-the-shelf NK and T-cell cancer immunotherapy with its robust clinical pipeline, novel iPSC product platform, and high-value strategic collaborations, said Dr. Xu. I look forward to working closely with the Companys board and management team as we move into late-stage clinical development, scale manufacture, and seek to make these innovative cancer medicines more broadly accessible to patients.

Early in her career, Dr. Xu held positions at Amgen, Chiron, GlaxoSmithKline and Genentech. Dr. Xu received a B.S. in biochemistry from Nanjing University and a Ph.D. in biochemistry from the University of Maryland, and she completed her post-doctoral training in virology and gene therapy at the University of California, San Diego.

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that are designed to be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely designed to overcome numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 350 issued patents and 150 pending patent applications.

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for patients with cancer. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology pipeline includes off-the-shelf, iPSC-derived natural killer (NK) cell and T-cell product candidates, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens using chimeric antigen receptors (CARs). Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the advancement of, plans related to, and the therapeutic potential of the Company's product candidates, the Companys clinical development and manufacturing strategies, and the Companys plans for the clinical investigation and manufacture of its product candidates. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that results observed in studies of its product candidates, including preclinical studies and clinical trials of any of its product candidates, will not be observed in ongoing or future studies involving these product candidates, the risk that the Company may cease or delay clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, the amount and type of data to be generated, or otherwise to support regulatory approval, difficulties or delays in subject enrollment and continuation in current and planned clinical trials, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), and the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Contact:Christina TartagliaStern Investor Relations, Inc.212.362.1200christina@sternir.com

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Fate Therapeutics Appoints Yuan Xu to its Board of Directors - StreetInsider.com

The T-cell therapy market is anticipated to rise at a stellar growth rate for the forecast period between 2020 and 2030. The shift in medical practices from small molecule and protein-based therapies to adoptive therapies that has attracted strategic investments by both public and private agencies is creating opportunities in the T-cell therapy.

Key parameters based on which the T-cell therapy market is divided in this report are modality, therapy type, indication, and region.

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The report provides an in-depth analysis of demand drivers, trends, and opportunities in the T-cell therapy market for the 2020 2030 forecast period. Furthermore, the report throws lights on key segments along with their growth rate estimations for the aforementioned forecast period. Last but not the least, the report discusses the competitive landscape of the T-cell therapy market. This includes insights into growth strategies of key players along with their revenue share estimation in the T-cell therapy market for the 2020 2030 forecast period.

T-cell Therapy Market: Competitive Landscape

The T-cell therapy market is fiercely competitive due to the presence of some large players in the fray. With increasing approvals of T-cell therapy and expanding manufacturing capabilities, competition in the market is expected to intensify in the future. For example, in December 2020, the European Medicine Agency issued market authorization for Tecartus the only CAR-T therapeutic product from Kite Pharma for mantle cell lymphoma.

Key players operating in the T-cell therapy market include;

T-cell Therapy Market: Key Trends

First and foremost, substantial application of T-cell mechanism in cancer immunotherapy due to its high success rate fuels growth in the T-cell therapy market. As of June 2020, above 350 CAR-T clinical trials registered in China. This data indicates the increasing significance of Chimeric Antigen Receptor therapy for the treatment of cancer.

Besides this, expanding role of gene therapy for the treatment of a number of rare diseases is spawning demand for CAR-T therapies. With increasing adoption of gene therapy, the T-cell therapy market is expected to touch new frontiers over the forecast period from 2020 to 2030.

Over the COVID-19 pandemic, increasing investments to decipher the application of T-cell therapies for viral infection research is adding a new dimension to the growth of T-cell therapy market. In this context, a study published in December 2020 demonstrates the potential of T-cell therapy to treat high-risk COVID-19 patients.

In another similar case study, in September 2020, the U.S. FDA sanctioned the IND application for the use of ALVR109 to treat COVID-19 patients. This is likely to expand the adoption of T-cell therapies for viral infections.

T-cell Therapy Market: Regional Assessment

North America is the leader among other key regions in the T-cell therapy market. Factors such as a robust research infrastructure for clinical trials of T-cell therapies and a commercial base for T-cell therapies makes the region leader in the overall T-cell therapy market.

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The U.S. and Canada predominantly steer growth in the T-cell therapy market in the region. The increasing number of regulatory approvals along with changing reimbursement scenario in the U.S. and Canada have accelerated the uptake of T-cell therapies in these countries. This bolsters the T-cell therapy market in the region.

China has emerged as a key region in the CAR-T therapies market in recent years. The high number of clinical trials undertaken pertaining to these therapies is creating opportunities in the T-cell therapy market in China.

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T-cell Therapy Market Trends, Share and Future Growth Analysis Report to 2030 - BioSpace

Image credits: Bone Therapeutics

A few years back, the concept of altering genes to treat disease was considered science fiction. However, it is now a reality. Thanks to technological advancements, numerous other cutting-edge approaches are reshaping how we treat and cure diseases.

Cell therapy is one such approach that involves injecting new cells into a patients body to replace or repair damaged tissue to treat a disease. According to the report, a total of 1342 active cell-based therapy clinical trials have been identified and characterised based on cell type, target indication, and trial phase.

Recently, the winners of the 2021 Future Hamburg Award were announced.

Nowadays, various medical organisations use cell therapies that are clinically approved. Based in Gosselies, Belgium, Bone Therapeutics is a biopharmaceutical company focused on innovative cell therapy products to treat bone diseases.

Recently, the company secured a 16M loan from the European Investment Bank (EIB) to accelerate the clinical development of ALLOB, Bone Therapeutics scalable allogeneic cell therapy platform.

Further, EIB will also support and prepare Bone Therapeutics lead product, viscosupplement JTA-004, for future regulatory approval and commercialisation.

ALLOB is an allogeneic cell therapy platform consisting of human allogeneic bone-forming cells derived from ex-vivo cultured bone marrow mesenchymal stromal cells (MSC) from healthy adult donors. It is currently in two Phase I/IIA proof-of-concept trials for the treatment of delayed-union fractures and spinal fusion procedures.

The patient recruitment is expected to complete in the first half of 2022 and the results in the second half.

JTA-004 is Bone Therapeutics next generation of intra-articular injectable, which is currently in phase III development for treating osteoarthritic pain in the knee.

JTA-004 consists of a unique patented mix of plasma proteins, hyaluronic acid a natural component of knee synovial fluid, and a fast-acting analgesic. It intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain and inflammation.

The Belgian company plans to submit a marketing authorisation application to European regulatory authorities in the first half of 2022. Additionally, the company continues to engage with potential partners to develop and commercialise JTA-004 in Europe, the US, and Asia.

The EIB will be disbursing the loan in two tranches of 8M each, subject to conditions precedent.

The first 8M will be available upon approval of the issuance of associated warrants by Bone Therapeutics General Meetings before the end of August 2021.

The next 8M will be released when specific clinical and commercial milestones have been achieved.

The loan facility will be in the form of a senior loan, repayable to the EIB in a single payment five years following the disbursement of each of the two tranches. The loan carries a fixed interest of 2 per cent per year paid annually and a 3% capitalized interest, says the press release.

Founded in 2006, Bone Therapeutics has an extensive portfolio of cell and biological therapies at different stages ranging from pre-clinical programs in immunomodulation to mid-to-late stage clinical development for orthopedic conditions.

Bone Therapeutics is building towards a very important set of milestones, including moving towards potential regulatory approval and commercialisation of therapy for over 250 million patients, as well as continuing with the clinical development of its allogeneic cell therapy platform ALLOB. In addition, we are building on our success in orthopedics and moving our formidable MSC capabilities to target wider indications. The support of a major European financial institution such as the EIB will be an additional important component to this activity, says Jean-Luc Vandebroek, Chief Financial Officer, Bone Therapeutics.

This financing committed by the EIB will allow Bone Therapeutics to further advance the clinical development of its lead product candidates JTA-004 and ALLOB, further accelerating paths to approval and commercialisation, he adds.

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Belgium's Bone Therapeutics secures 16M loan from EIB to develop new cell therapy-based orthopaedic treatments - Silicon Canals

Abstracted presented recently at the American Society of Clinical Oncology annual meeting offered promising data for 2 chimeric antigen receptor (CAR) T-cell therapies, one in relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL) and one in R/R multiple myeloma.

Approved in 2020 for patients with R/R mantle cell lymphoma, brexucabtagene autoleucel, sold as Tecartus, is also now being studied in patients with R/R B-ALL, with the recent conference data suggesting the treatment offers a clinical benefit in these patients.1

After a median follow-up of 16.4 months, median overall survival (OS) was not reached among the patients who responded to Tecartus. These patients had a mean relapse-free survival of 14.2 months.

Across the 55 patients receiving Tecartus, 71% achieved a complete response (CR) or CR with incomplete hematologic recovery (CRi), 97% of which were negative for minimal residual disease.

The patients included in the study were heavily pretreated, with 45% having previously received blinatumomab, 22% having previously received inotuzumab ozogamicin, and 42% having previously received allogeneic stem cell transplant.

Ninety-five percent of patients experienced grade 3 adverse events. The most common adverse events included anemia (49%) and neutropenia (49%). Grade 3 cytokine release syndrome and neurologic eventscommonly reported among patients treated with CAR T-cell therapieswere reported in 24% and 25% of patients, respectively, with a median time to onset of 5 days and 9 days, respectively. According to the researchers, they were generally reversible.

Novel therapy in multiple myeloma. During the conference, researchers also offered data from 2 different time points of an ongoing phase 1 study of CART-ddBCMA in R/R multiple myeloma. CART-ddBCMA is an autologous CAR-T cell therapy that uses a novel BCMA-targeting binding domain and is designed to reduce the risk of immunogenicity and have high stability.

As of January 29, 2021, there were 9 evaluable patients, all of which responded to CART-ddBCMA. Four patients achieved a stringent CR (sCR), one achieved a very good partial response (VGPR), and 4 achieved a PR. One of the patients who achieved a PR had disease relapse and was retreated, while the rest of the patients had ongoing responses.

Similar findings were seen as of April 2021,3 with all 12 evaluable patients achieving a response, including 5 sCR, 1 CR, 3 VGPR, and 3 PR. Eleven of these responses are ongoing and data from the study suggests that responses deepen over time. According to the researchers, despite previously progressing on BCMA-targeted therapy, a patient still achieved a VGPR.

All 12 of these patients have received at least 3 prior lines of therapy, and 10 were penta-refractory.

References

1. Shah B, Ghobadi A, Oluwole O, et al. Phase 2 results of the ZUMA-3 study evaluating KTE-X19, an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, in adult patients (pts) with relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). J Clin Oncol. 2021; 39(Suppl 15): abstr 7002. doi: 10.1200/JCO.2021.39.15_suppl.7002

2. Frigault M. Phase 1 Study of CART-ddBCMA, a CAR-T therapy utilizing a novel synthetic binding domain, for the treatment of subjects with relapsed and refractory multiple myeloma. J Clin Oncol. 2021; 39(Suppl 15): abstr 8015. doi: 10.1200/JCO.2021.39.15_suppl.8015

3. Arcellx Announces Presentation of Positive Clinical Results from Ongoing Phase 1 Study of CART-ddBCMA at the 2021 ASCO Annual Meeting. News release. June 4, 2021. Accessed July 2, 2021. https://arcellx.com/arcellx-announces-presentation-of-positive-clinical-results-from-ongoing-phase-1-study-of-cart-ddbcma-at-the-2021-asco-annual-meeting/

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Studies Offer Promising Data on CAR T-cell Therapy in B-ALL, Multiple Myeloma - AJMC.com Managed Markets Network

This article was originally published here

Clin Rev Allergy Immunol. 2021 Jul 5. doi: 10.1007/s12016-021-08866-1. Online ahead of print.

ABSTRACT

Autoimmunity is caused by an unbalanced immune system, giving rise to a variety of organ-specific to system disorders. Patients with autoimmune diseases are commonly treated with broad-acting immunomodulatory drugs, with the risk of severe side effects. Regulatory T cells (Tregs) have the inherent capacity to induce peripheral tolerance as well as tissue regeneration and are therefore a prime candidate to use as cell therapy in patients with autoimmune disorders. (Pre)clinical studies using Treg therapy have already established safety and feasibility, and some show clinical benefits. However, Tregs are known to be functionally impaired in autoimmune diseases. Therefore, ex vivo manipulation to boost and stably maintain their suppressive function is necessary when considering autologous transplantation. Similar to autoimmunity, severe coronavirus disease 2019 (COVID-19) is characterized by an exaggerated immune reaction and altered Treg responses. In light of this, Treg-based therapies are currently under investigation to treat severe COVID-19. This review provides a detailed overview of the current progress and clinical challenges of Treg therapy for autoimmune and hyperinflammatory diseases, with a focus on recent successes of ex vivo Treg manipulation.

PMID:34224053 | DOI:10.1007/s12016-021-08866-1

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APEIRON Biologics launches next clinical trialwith innovative cancer therapy APN401Important development step for promising cell therapy

Vienna, Austria, 06 July 2021: APEIRON Biologics AG announced today the start of a Phase Ib clinical trial with its product candidate APN401 for the treatment of solid tumors. The principle of cell therapy with APN401 by inhibiting the immune checkpoint Cbl-b aims at the patient's own immune cells. These are modified to recognize and destroy cancer cells without being permanently genetically altered.

The open-label, multi-center Phase Ib clinical trial is expected to enroll approximately 60 patients at multiple sites in Austria. The study objective is to evaluate the safety, tolerability and immunological effects of the treatment on patients with various solid tumors. This will build on the experience of the two previous Phase I clinical studies, which already successfully demonstrated good tolerability and the first signs of clinical efficacy by activating the immune cells that are crucial to tumor defense.

The clinical study is divided into two parts. Part A of the study aims to determine the optimal dosing, i.e. the quantity of treated cells reinfused back to the patient. Patients will receive APN401 treatment every three weeks. In part B of the study, patients with specific tumor indications (three groups of 15 patients each) will be treated to generate further efficacy signals, which will be used to determine the tumor indication for a subsequent Phase II clinical study. The Phase I clinical study will start at the Medical University of Vienna (MUW) where the GMP-certified production of the cell therapy and treatment of the patients will take place.

For the treatment, the patient's own peripheral blood mononuclear cells (PBMCs) are collected, specifically modified outside the body using RNAi technology and then reinfused into the patient. APEIRON uses a specially developed system for this purpose, which enables an automated process from cell processing to reinfusion within just one day. This GMP-certified system increases patient safety and the reproducibility of the manufacturing process. APEIRON's technology thus enables personalized cell therapy in solid tumors.

Peter Llewellyn-Davies, CEO of APEIRON Biologics AG, says: "We are thrilled to be contributing a groundbreaking cancer treatment with this truly pioneering step in development of our APN401 cell therapy. The short outpatient administration process, which can also be used for solid tumors, plays a key advantage compared to other autologous cell therapies. APN401 could offer critically ill patients, with previously difficult-to-treat cancers, new individualized treatment options and thus new hope. The APEIRON team is highly motivated to develop much needed new treatment options with this major next step."

Dr. Romana Gugenberger, Chief Medical & Scientific Officer (CMSO) of APEIRON Biologics AG, explains: "The immune system is the most effective weapon against tumor disease and offers many advantages over conventional therapies such as chemotherapy. Cbl-b is a master checkpoint in the immune system that controls important processes of the immune response, especially in cancer. APN401, by blocking Cbl-b using RNA interference (RNAi), is designed to reactivate the patient's immune system, allowing it to fight solid tumors. The flexibility of RNAi technology could expand the applicability of cell therapy to additional immune checkpoints and holds enormous potential for new therapeutic approaches."

Prof. Dr. Nina Worel, Head of Cell Therapy at the Department of Transfusion Medicine at AKH / Medical University of Vienna and lead investigator of the study, adds: "Patients with advanced solid tumors urgently need new, safe and effective therapeutic options. Using cell therapy to enable the patient's immune system to directly attack the tumor is a very promising approach. APEIRON's APN401 could become superior in safety and efficacy to standard oncology therapies as well as to currently available cell therapies, due to its rapid applicability and central immune activation. We are excited to initiate this study here in Vienna and other sites and gain new insights."

About APN401

Immune checkpoints are receptors with immunoregulatory activity. Tumor cells can make use of these immune checkpoints to escape recognition by the immune system. Cbl-b represents a new class of intra-cellular immune checkpoints in contrast to the immune checkpoint molecules PD-1/PD-L1 and CTLA-4, which are localized at cell surfaces.

APN401, an autologous cell therapy, was designed to transiently, i.e. temporarily, inactivate Cbl-b ex-vivo in autologous PBMCs. These altered autologous PBMCs are then returned to the patient, with the entire procedure performed on an outpatient basis over one day. APN401 is well tolerated, has a good safety profile, and has shown early evidence of clinical activity in patients with advanced solid tumors in two Phase I studies.

More information on checkpoint inhibition of Cbl-b and APN401 can also be found on our website.

About APEIRON Biologics AG

APEIRON Biologics is a privately held European biotech company based in Vienna, Austria, focused on the discovery and development of treatments for respiratory diseases and novel cancer immunotherapies.

APEIRON received EU marketing approval for APN311 (dinutuximab beta, Qarziba(R)) in 2017 for the treatment of pediatric neuroblastoma patients and out-licensed global, exclusive rights for this product to EUSA Pharma Ltd.

APEIRON is developing a promising drug for COVID-19: APN01 (rhsACE2, alunacedase alfa), a soluble recombinant version of the SARS-CoV-2 cell entry receptor ACE2. APN01 has three distinct potential clinical benefits for COVID-19 and has completed a double blind, placebo-controlled Phase II trial in Europe and Russia. Based on promising results, APN01 was selected for a publicly funded, large-scale study in COVID-19 by the U.S. government which is scheduled to start in Q3 2021.

APN401's proprietary cellular therapy process brings in a paradigm change in cancer treatment to fight solid tumors. The clinical program is a first-in-class ambulatory autologous transient therapy to strengthen immune reactivity via an intracellular master checkpoint inhibitor, Cbl-b.

APEIRON Biologics' projects and technologies are based on a strong patent portfolio and partnerships with leading pharmaceutical companies and academic institutions.

Further information, visit http://www.apeiron-biologics.com and connect with us on twitter and LinkedIn.

For further information please contact:

APEIRON Biologics AGPeter Llewellyn-DaviesCEOEmail: investors@apeiron-biologics.comwww.apeiron-biologics.com

Media and Investor RelationsMC Services AGJulia Hofmann, Andreas JungferT +49 89 210 228 0Email: apeiron@mc-services.eu

FORWARD LOOKING STATEMENTS

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

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