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Scientists engineer CRISPR enzymes that evade the immune system By Allessandra DiCorato January 9, 2025 Breadcrumb Home Scientists engineer CRISPR enzymes that evade the immune system The new genome-editing tools could lead to safer, more efficient gene therapies. Lisa Yang and Hock E.
To convert this immunosuppressive environment into one amenable to an immuneresponse, investigators engineered a novel oncolytic virus that can infect cancer cells and stimulate an anti-tumor immuneresponse.
Confo Therapeutics , led by CEO Dr Cedric Ververken, is at the forefront of developing innovative GPCR-targeted therapies using its proprietary ConfoBody ® platform. The MC4R-based ConfoGen was used to immunize llamas, resulting in an immuneresponse that generated a highly diverse panel of MC4R-specific agonists.
This ebook delves into the intricate world of cancer therapies, immune system breakthroughs, therapeutic genetic engineering, and groundbreaking advancements in gene editing, featuring insightful interviews with renowned experts in the field.
We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. Generally, IO has been focused on harnessing the anti-tumour activity of certain cancer-fighting T-cells , a key cell type involved in the adaptive immune defense system.
NK cells are among the front line of protection from infected and abnormal cells as part of the ‘innate immuneresponse’. They recognise ‘cell stress molecules’ on the surface of infected, old, injured and cancerous cells without the need for complex pre-stimulation signals of the adaptive immune system (eg, T cells).
Among the many remarkable components that make up this complex defence network, T cells have emerged as powerful guardians, orchestrating an array of immuneresponses with great precision.
Agonist antibodies of immune checkpoint regulators These represent a groundbreaking class of immunotherapeutic agents that mimic the natural function of endogenous ligands by binding to specific cell-surface receptors. Often immunological diseases involve deficiencies or dysregulation of immune function.
Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.
Better activation of innate and adaptive immuneresponses was achieved with CV2CoV, resulting in faster response onset, higher titers of antibodies, and stronger memory B and T cell activation as compared to the first-generation candidate, CVnCoV. “In Induction of innate immunity was investigated via specific cytokine markers.
In research published in Scientific Reports , 1 investigators focused on mesenchymal stem cells (MSCs), known for their potential in treating cell defects and regulating immuneresponses. We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun.
2D cell models are often engineered to over-express the target of interest and so lack its temporal behaviour in its native or disease state. This is particularly pertinent in immune-based cell therapies like CAR-T, where animals do not mimic the human immuneresponse.
This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.
The molecular tools of CRISPR were borrowed and developed from the natural immuneresponse of bacteria to viruses – bacteriophages – that infect them. A recent paper in BioDesign Research , CRISPR-Cas-Based Engineering of Probiotics , from investigators at several Chinese research institutions, contributed to the report.
This sugar-coated mucin binding to checkpoint receptors signals that the cancer cell poses no threat, consequently blocking the immuneresponse. Tender elaborated that these results in immune cells ignoring the cancer rather than eliminating it, as they normally would.
Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.
“The T-cell and NK-cell target discovery approach complements our portfolio aimed at turning cold tumors hot and redirecting the innate immune system to elicit a sustained and durable immuneresponse against tumors.
These disease signatures capture both the linear and non-linear effects of genetic and molecular interaction networks and enable the identification of associations including those that are only relevant to a subgroup of patients that influence disease risk, prognosis and/or therapyresponse.
Researchers from the Tumor Immunotherapy Discovery Engine ( TIDE ) at the Broad Institute of MIT and Harvard, AbbVie, and Calico Life Sciences report that the molecule simultaneously makes tumors more sensitive to immune attack and boosts the activity of immune cells to fight tumors in mice.
Merck will select cancer targets and harness Janux’s proprietary Tumor Activated T Cell Engager (TRACTr) technology to engineer novel, T cell engager drug candidates. Janux technology can be applied to immunotherapies that target all three stages of an anti-tumor immuneresponse.
The Agreement extends an existing relationship between the companies and gives Pandion the option to use standard phage display or mammalian display as the engines for the discovery of next generation antibodies. For further information, please visit: [link]. View source version on businesswire.com: [link]. Source link.
Nasdaq: SYBX ), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced SYNB1891 has advanced into the combination therapy stage of the ongoing Phase 1 trial. CAMBRIDGE, Mass. , 14, 2020 /PRNewswire/ — Synlogic, Inc. Synlogic’s President and Chief Executive Officer.
Anderson Professor of Chemical and Biomolecular Engineering, and his colleagues, are reporting in iScience the event of an intranasal subunit vaccine that gives durable local immunity against inhaled pathogens. A fundamental limitation of intramuscular vaccines is that they’re not designed to elicit mucosal immunity.
Early clinical results are consistent with preclinical studies and suggest THOR-707 (SAR444245) may promote an anti-tumor immuneresponse without alpha-mediated side effects, both alone and in combination with anti-PD-1. We will continue to explore the molecule’s potential for best-in-disease combinations.”. About THOR-707 (SAR444245).
The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immuneresponse. The study showed this broad immuneresponse led to the complete clearance of the virus in a matter of days after infection of previously-vaccinated primates. link]
1. About ImmunityBio.
(Nasdaq: ACET), a biotechnology company discovering and developing allogeneic gamma delta T cell therapies for cancer and other diseases, today announced that the U.S. T cell therapy expressing a chimeric antigen receptor (CAR) targeting CD20 for treatment of non-Hodgkin’s lymphoma (NHL). T cell therapy in NHL patients.
Coined immunotherapy, this umbrella term describes several types of treatment, including immune checkpoint inhibitors, immune system modulators, and adoptive cell therapies. Each triggers the immune system in a different way to fight cancer.
Quantitative imaging of CD8 T cells enables quicker identification of drug efficacy, therefore potentially reducing the length of clinical trials, reducing costs and helping new therapies to advance to market faster, which ultimately will improve treatment and care of cancer patients. About ImaginAb. ImaginAb Inc.
Former Sarepta Therapeutics executive Bo Cumbo left to launch his new gene therapy company, AavantiBio, with a $107 million Series A. AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. AavantiBio . Sirnaomics.
Syngeneic tumor animal models play a critical role because they use standard inbred mice that have a competent immune system, which is required to evaluate immune-modulating therapies. One consideration in using syngeneic models is that some drug candidates can cause a negative immuneresponse not seen in humans.
New research from first-in-class marketed and investigational therapies in hemophilia, immune thrombocytopenia and acquired thrombotic thrombocytopenic purpura will be presented. Sanofi’s two marketed extended half-life factor replacement therapies shifted a two-decades-old treatment paradigm when launched in 2014. ePoster.
Merkin Prize in Biomedical Technology for developing chimeric antigen receptor (CAR) T-cell therapy, a groundbreaking form of personalized cancer immunotherapy that turns T cells into tumor killers and has led to durable remissions in tens of thousands of patients with previously incurable blood cancers.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime.
This is how many medical studies begin, said biomedical engineering professor Phil Bourne, who serves as founding dean for the School of Data Science. One of the most dangerous phenomena COVID-19 can trigger in your body is something called a cytokine storm, which is a potentially fatal amplification of an immuneresponse.
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chPD1 will be used in the Company’s proprietary chimeric antigen receptor therapy (CAR-T) platform using gamma-delta T-cells (GD-T).
Barber’s work will give Kiromic a significant acceleration in the clinical development of its therapy platform and an even more significant advantage over its competitors.
(Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the observational PRECISION study (TNX-C002), to examine the immuneresponses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic.
Draft guidance on potency assays for CGT products garners extensive stakeholder input Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapy products, unveiling a major shift in approach to the issue. How does “potency” apply to cell and gene therapy (CGT) products?
Using single-cell RNA sequencing to analyze gene expression in individual cells, the researchers have found how 86 major cytokines affect 17 immune cell types in mice. NH: This is the first single-cell resolution dictionary of each major immune cell type responding to each major cytokine in vivo at an unprecedented scale.
Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. Credit: Neumann EN, Bertozzi TM, et al.
The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. The core of the CRISPR immuneresponse is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Collectively, these repeat-protospacer regions are known as CRISPR arrays. Visit our website to get a copy.
Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.
The combination of P1 with antiPD1 more efficiently activated innate and adaptive immuneresponses than either P1 or antiPD1 alone by re-energising T cell effector functions, thus providing the remarkable reduction of tumour growth in solid tumours and extended survival period.
His mother had a presentation of the disease that suggested her immune system was already on the job. But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way.
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