Monthly Archives: July 2021

What is Stem Cell Research? – WorldAtlas

Posted: July 6, 2021 at 2:49 am

Stem cells are those that don't have a specific purpose.

A stem cell is a cell in a living body with the potential to develop into different types of cells during the early stage. Most cells in a living organism are differentiated cells, meaning they are found in a specific organ and perform particular functions. Red blood cells, for example, are specifically designed to transport oxygen through the blood. Human beings start out as a single cell known as a zygote, which is a fertilized egg. The zygote undergoes cell division into two, then four, eight, sixteen, and so on. The cells begin to differentiate and specialize in specific functions in the body as the zygote develops. The cells that haven't acquired specific purpose are known as stem cells; they can replicate indefinitely unlike differentiated cells that begin to break down after replicating. Once a stem cell divides, it remains as a stem cell or turns into a differentiated cell. This makes them especially intriguing for scientific research.

There are two types of stem cells: embryonic and adult stem cells. Embryonic stem cells are derived from embryos developed through in vitro fertilization in a fertilization clinic. The fertilized eggs are then donated for research purpose with the consent of the donors. Embryonic stem cells develop into specialized cells as the embryo develops. Adult stem cells are found among differentiated cells in an organ or tissue. Their main functions are to repair and maintain the tissue. Unlike embryonic stem cells that are produced by the embryo, researchers are still trying to understand the source of adult stem cells.

Stem cells have three distinct properties regardless of their source; they replicate and renew themselves infinitely, they are unspecialized, and they give rise to differentiated cells. Unlike a nerve cell, blood cells, or muscle cells, stem cells can proliferate. Research conducted in laboratories have revealed that stem cells can yield millions of unspecialized cells with the properties of the parent stem cell.

The properties of stem cells make them excellent and intriguing candidates for research. The embryonic human stem cells provide information concerning the complex process that occurs during the development of life. The primary objective of the research is to understand how undifferentiated cells end up with specific functions. Human stem cells are used to test new drugs and observe how a human body would respond to the medication. Stem cells are also helping researchers study diseases such as cancer and diabetes and how they can be treated. The immediate potential application of stem cell research is the generation of tissues and cells that replace organs once destroyed or removed. A breakthrough would eliminate the dependency on organ transplant, but instead, patients would receive stem cells that would generate the organ. Preliminary research in rodents shows that transplanted stem cells from the bone marrow can generate heart muscle tissues and repair the heart. A bone marrow transplant is already being used as a treatment for some form of cancer in humans.

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New global guidelines for stem cell research aim to drive …

Posted: July 6, 2021 at 2:49 am

The International Society for Stem Cell Research (ISSCR) today released updated guidelines for stem cell research and its translation to medicine.

Developed in response to recent scientific and clinical advances, the revised guidelines provide a series of detailed and practical recommendations that set out global standards for how these emerging technologies should be harnessed.

Stem cell research has huge potential it could help pave the way for new therapies for ailments ranging from Parkinsons disease to childhood kidney failure. But scientific advances in this field can present unique ethical and policy issues beyond that seen in other areas of medical research.

The science is advancing at breakneck pace. Just in the past couple of months, we have seen model human embryos grown from skin cells, and the creation of human-monkey embryos for use in research.

The ISSCR has long recognised the need to set clear ethical boundaries for stem cell research. Previous guidelines have provided advice on techniques such as the use of human embryos to create stem cells, and set the required standards when using these technologies to create new medicines.

They have also explicitly banned certain practices, such as reproductive cloning and the sale of unproven therapies that claim to be made of stem cells.

The 2021 guidelines an update on the previous version, released in 2016 aim to set standards for the many recent advances in stem cell and human embryo research. These include chimeric embryos containing cells from humans and other animals, organoids grown from stem cells to create tissue that resembles particular human organs, and models of human embryos arrangements of human cells that mimic the early stages of embryo development.

The guidelines contain a clear requirement for certain new stem cell research approaches only to be conducted after a specialised review process. This review should be independent of the researchers, and include community members as well as people with expertise in the relevant science, ethics and law.

This is beyond what is typically required by a university or research institute where medical research is conducted. Besides evaluating the merit of the proposed research, the new reviews should also consider whether there are alternative ways to do the research, the source of stem cells and how they were obtained, and the minimum time required to reach the research goals, particularly in relation to human embryo and related research.

Specialised review is not a new concept. The previous guidelines required it when researchers made stem cells from human embryos or sought to culture human embryos in the lab. But now researchers will now also be required to seek higher review when they create model embryos such as blastoids, or study the development of animal-human embryos in animal wombs.

Researchers developing new therapies for mitochondrial disease will also be required to seek higher-level review before attempting to transfer to the uterus of a woman human embryos in which affected mitochondria (a part of the cells energy-production apparatus) have been replaced.

Importantly, the revised guidelines also clearly rule out certain activities. These continue to include reproductive cloning and attempts to form a pregnancy in a woman from genetically edited human embryos or from model embryos made from stem cells. Prohibited activities also now include using eggs and sperm made from human stem cells for reproduction, or transferring a human-animal chimeric embryo into the uterus of a woman or an ape.

Read more: China's failed gene-edited baby experiment proves we're not ready for human embryo modification

The guidelines also call for a public conversation about whether we should allow limited lab research on human embryos beyond the existing limit of 14 days development. Historically, it has not been possible to support human embryonic development outside the body beyond this stage. However, recent advances in human embryo culture raise the possibility that this may now be technically feasible.

Extending the amount of time in culture - in terms of days - could potentially yield new treatments for developmental conditions or infertility, but will also raise concerns about whether possible benefits justify this research. Any decisions to overturn this long-held signpost would need to be carefully deliberated and take into consideration existing law, community values and discussion around what the new limit should be.

The revised guidelines also reinforce the need for informed consent for the collection of human material and participation in stem cell clinical trials, and reiterate that no new stem cell treatment should be made available before it is tested for safety and effectiveness in well-designed and publicly visible clinical trials. The ISSCR continues to condemn the commercial use of unproven stem cell treatments.

While stem cell science holds much promise, it is paramount that research is scientifically and ethically rigorous, with appropriate oversight, transparency and public accountability.

The fact these guidelines are driven by experts including stem cell scientists, doctors, ethicists, lawyers and industry representatives from across 14 countries indicates a deep sense of responsibility and integrity within the research community, and a desire to ensure science remains in step with community values.

However, these guidelines are recommendations, not laws.

Researchers will need to abide by their respective national or state regulations and ethical standards. Some countries already have regulatory frameworks that are consistent with the new recommendations. In other places there is no national guidance around laboratory and clinical stem cell research at all, or existing law touches on some but not all of the emerging applications of stem cell research.

Read more: As scientists move closer to making part human, part animal organisms, what are the concerns?

For example, in Australia there is already an established pathway for higher-level review of embryo models created from stem cells. However, the same legislation currently bans any attempt to use mitochondrial transfer techniques to create embryos for research or to achieve a pregnancy both of which are permissible under the new ISSCR guidelines.

Rather than attempting to impose a set of hard-and-fast rules on an ever-evolving research field, the new guidelines attempt to address emerging issues and drive important discussions at domestic level. Ultimately, it is the public and the regulators who will need to set the standards.

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$6.3 million for ground-breaking stem cell research projects – News – The University of Sydney

Posted: July 6, 2021 at 2:49 am

The Federal Minister for Health and Aged Care, the Hon Greg Hunt, has announced$18.7 million in funding for the 2020 Stem Cell Mission, which focuses on research that develops and delivers innovative, safe and effective stem cell medicines to improve health outcomes.

Researchers from the University of Sydney have been awarded $6.3 million for three projects which will address blindness in adults, chronic heart failure, and help to improve decisions about access to stem cell interventions.

Deputy-Vice-Chancellor (Research), ProfessorDuncan Ivisoncelebrated the funding success.

This funding will allow our health and medical researchers to undertake important research for the benefit of many Australians and their families, through trials that use stem cell grown heart muscle in patients with no option end-stage heart failure," he said.

Medical Research Future Fund (MRFF) 2020 Stem Cell Mission grants awarded to Sydney researchers include:

The Stem Cell Mission is a priority of the Morrison Governments $20 billion Medical Research Future Fundand will provide $150 million towards research over 9 years.

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$6.3 million for ground-breaking stem cell research projects - News - The University of Sydney

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Limits for human embryo research have been changed: this calls for public debate – The Conversation CA

Posted: July 6, 2021 at 2:49 am

For 40 years, research into early human development has been guided by the principle that after 14 days, an embryo should not be used for research and must be destroyed. This rule has been part of the law of more than 12 countries. But new guidelines released by the International Society for Stem Cell Research have removed this rule. This makes it possible to conduct research on human embryos that are at more advanced stages of development.

Now, countries must revise their laws, policies and guidelines to reflect this change. But first, public debate is crucial to determine the limits of what sort of research should be allowed.

Over the decades human embryo research has allowed us to understand normal and abnormal human development, as well as early genetic diseases and disorders. Studying human embryos, as the earliest forms of human life, can give us insight into why miscarriages occur, and how our complex body systems develop. Human embryos are also important for stem cell research, where researchers try and create cell-based therapies to treat human diseases.

Often, extra embryos are created during in vitro fertilisation procedures. These extra embryos may be donated for research. They are cultured (or grown) in a laboratory and can be studied until they reach day 14 post-creation.

The 14-day rule has served as an international standard since 1990 when it was included in the Human Fertilisation and Embryology Act in the UK. At the time that it was introduced, it was not possible to keep human embryos alive in a laboratory for more than a few days. However, scientists have been recently been able to keep embryos alive for longer periods, between 12 and 13 days. The ethical, legal and social consequences of such research were also important considerations.

Although the 14-day rule has been criticised as being arbitrarily decided, there are a number of reasons for the time frame.

After an egg cell is fertilised by a sperm cell, the resulting embryo consists of a few identical cells. Most embryos will implant in the uterus after the 14th day. After this point, the primitive streak appears, which is the first sign of an embryos developing nervous system. The rule also identified the point at which the embryo shows signs of individuation, because it is no longer possible for the embryo to split into twins after 14 days. Some people reason that due to these events, it is at this stage that a moral being comes into existence, and it would not be ethical to perform research on embryos after this time.

There has been increasing pressure from some researchers to remove the 14-day rule, or at least extend it, as it prevents critical research from being undertaken. Extending the rule would allow important research into early human development to be done. The new guidelines make it possible to do research on embryos older than 14 days if the approval processes of the relevant ethics committees are followed.

A significant problem, however, is that there is no longer any limit on the time frame for research. Would it be permissible to do research on human embryos that are 20 days old or 40 days old? The guidelines specify no limit. The longer a human embryo is allowed to grow, the more recognisably human it becomes. At what point would we regard the research unethical, and at what point does the moral cost outweigh the benefits of research?

Countries around the world take a variety of approaches to human embryo research. Some like Italy and Germany dont allow it at all. Others, like the UK, allow research to continue until the embryo is 14 days old, after which it must be destroyed. There are also some which permit embryo research without identifying a limit. Some, like the US, do not have any law regulating it (but there are guidelines which contain reference to the 14-day rule).

In South Africa, reference to the rule is found in the National Health Act (2003), which states that human embryo research may only be done with permission of the minister, and that the embryos must not be older than 14 days.

International guidelines are not legally binding. But the effect of the revised guidelines is that the international standard for best practice in scientific research has now changed. This means that countries which have implemented the rule in their laws will need to revise them so that they are in line with best practice in science.

Human embryo research is a sensitive topic because people are divided on the moral status of the human embryo. Some people believe that the embryo, as the earliest form of human life, should be protected and not subjected to research at all. Others believe that while an embryo has some moral status, it cannot be protected in the same way as humans are, and may be used for some important research which could ultimately benefit people.

The decision to discard the 14-rule appears to have been made without public input. That does not encourage the public to trust in science, and public engagement should have come before such an an important rule was changed.

There are a number of approaches to working with the revised guidance. Bioethicist Franoise Baylis has suggested that project-specific time limits should be identified, based on the minimum amount of time required to address the stated research objectives. This would mean that some research would still be subject to the 14-day limit, while other studies would be permitted to exceed it. Another approach would be to keep the 14-day limit as the norm, and consider applications to exceed it case by case. Or the limit could be extended to 28 days.

The coming conversations surrounding embryo research will prove to be very important. The proverbial genie is out of the bottle, and public debate is crucial.

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Limits for human embryo research have been changed: this calls for public debate - The Conversation CA

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

Posted: July 6, 2021 at 2:45 am

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

Posted: July 6, 2021 at 2:45 am

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

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

Posted: July 6, 2021 at 2:45 am

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

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Improving the Efficacy of Regulatory T Cell Therapy – DocWire News

Posted: July 6, 2021 at 2:45 am

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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Combination of CRISPR/Cas9 System and CAR-T Cell Therapy: A New Era for Refractory and Relapsed Hematological Malignancies – DocWire News

Posted: July 6, 2021 at 2:45 am

This article was originally published here

Curr Med Sci. 2021 Jul 3. doi: 10.1007/s11596-021-2391-5. Online ahead of print.

ABSTRACT

Chimeric antigen receptor T (CAR-T) cell therapy is the novel treatment strategy for hematological malignancies such as acute lymphoblastic leukemia (ALL), lymphoma and multiple myeloma. However, treatment-related toxicities such as cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) have become significant hurdles to CAR-T treatment. Multiple strategies were established to alter the CAR structure on the genomic level to improve efficacy and reduce toxicities. Recently, the innovative gene-editing technology-clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated nuclease9 (Cas9) system, which particularly exhibits preponderance in knock-in and knockout at specific sites, is widely utilized to manufacture CAR-T products. The application of CRISPR/Cas9 to CAR-T cell therapy has shown promising clinical results with minimal toxicity. In this review, we summarized the past achievements of CRISPR/Cas9 in CAR-T therapy and focused on the potential CAR-T targets.

PMID:34218353 | DOI:10.1007/s11596-021-2391-5

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Combination of CRISPR/Cas9 System and CAR-T Cell Therapy: A New Era for Refractory and Relapsed Hematological Malignancies - DocWire News

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APEIRON Biologics launches next clinical trial with innovative cancer therapy APN401 – Yahoo Eurosport UK

Posted: July 6, 2021 at 2:45 am

DGAP-News: APEIRON Biologics AG / Key word(s): Study06.07.2021 / 08:00 The issuer is solely responsible for the content of this announcement.

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.

06.07.2021 Dissemination of a Corporate News, transmitted by DGAP - a service of EQS Group AG.The issuer is solely responsible for the content of this announcement.

The DGAP Distribution Services include Regulatory Announcements, Financial/Corporate News and Press Releases. Archive at http://www.dgap.de

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APEIRON Biologics launches next clinical trial with innovative cancer therapy APN401 - Yahoo Eurosport UK

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