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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.
This absence of objective measures contributes to the variability in patient response to treatments, complicating efforts to produce standardised therapies. Moreover, regulatory pathways also change considerably based on the type of therapy – and guidance is sparse. Their development requires complex testing strategies.
In the ever-evolving landscape of immuno-oncology, managing the side effects of advanced therapies has become just as important as enhancing their efficacy. Existing therapies, such as tocilizumab, work by blocking specific inflammatory pathways but often come with limitations.
These CCR8+ Tregs are known to suppress immuneresponses in the tumour microenvironment (TME), allowing cancers to grow unchecked. “DT-7012 DT-7012: targeting Tregs where it matters most DT-7012 is Domain’s Treg depleting anti-CCR8 monoclonal antibody designed to deplete tumour-infiltrating regulatory T cells (Tregs).
Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation. FDA Orphan Drug Designation for Antibody-Mediated Rejection in Solid Organ Transplantation The U.S.
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.
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.
Recent years have witnessed remarkable advancements in cellular therapies, particularly in the development of treatments that harness the power of stem cells and the immune system. At the forefront of these innovations is Dr Thomas Ichim, a distinguished expert in biotechnology and stem cell therapy at Immorta Bio.
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.
The scientists suggest that new therapies that target these genes could directly address fibrosis and potentially be more effective for this complication than existing drugs, which are primarily focused on reducing inflammation. But now we can potentially think about additive therapies that more directly target the fibrotic process.”
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. BCMA and CD19 are the protein targets of the seven approved CAR-T therapies. You can unsubscribe at anytime.
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.
Researchers have discovered a protein variant that serves as a knob for regulating the body's innate immuneresponse. The findings could lead to new therapies for Long COVID, autoimmune disorders, and more.
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).
Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.
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. Vicebio was formed by European life sciences investment firm Medicxi in 2018. You can unsubscribe at anytime.
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.
This is particularly pertinent in immune-based cell therapies like CAR-T, where animals do not mimic the human immuneresponse. The full potential of PDOs would be to shorten the time and costs associated with medicine creation, and then use PDO therapies to treat patients effectively.
This interaction inhibits inflammation and immune activation, and in particular prevents macrophages from phagocytosing and destroying cancer cells. By blocking CD24 with an antibody drug, our goal is to powerfully reactivate the anti-cancer immuneresponse and drive therapeutic efficacy.
Their research centres around a remarkable immune protein called IL-31, which not only induces itching but also plays a crucial role in regulating inflammation. In the absence of IL-31, these nerve cells failed to control the immune system, leading to unchecked inflammation.
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.
2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).
My Attendance at the 2024 Boston Society Cell and Gene Therapy Conference pmjackson Tue, 06/11/2024 - 20:49 , via Wikimedia Commons" data-entity-type="file" data-entity-uuid="c7a7fa8b-b2fe-4d84-a75e-d1ba3a4e2caf" src="[link] width="742" height="249" loading="lazy" /> Ecm85, CC BY-SA 3.0
HLA are the critical molecules that our immune systems use to present peptides to T cells and facilitate recognition and killing in immuneresponses to pathogens. HLA-II presents peptides to CD4 T cells thought to be important for indirectly helping immuneresponses and facilitating antibody production.
The molecular tools of CRISPR were borrowed and developed from the natural immuneresponse of bacteria to viruses – bacteriophages – that infect them. CRISPR may make sense for replacing a single mutant gene, like the one behind sickle cell disease, for which a CRISPR-based gene therapy was recently approved.
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.
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. Biopharmaceutical New Technologies is a next generation immunotherapy company pioneering novel therapies for cancer and other serious diseases.
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. Online October 4, 2023.
Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. Being an only child of non-academic parents, I learned to work and study independently early on.
–( BUSINESS WIRE )– Gilead Sciences, Inc. While HIV treatment has advanced dramatically over the past three decades, people living with HIV still face a lifetime of therapy,” said Diana Brainard, MD, Senior Vice President, Virology Therapeutic Area, Gilead Sciences. About Gilead Sciences. Gilead Sciences, Inc.
This method was more about serendipity than science. 5 This period also saw the rise of biologics in the 1980s, which have played a crucial role in modern therapies, particularly for diseases like cancer. The Evolution of Drug Discovery: From Dark Rooms to Precision Targets In the past, drug discovery often felt like a lucky guess.
As medical understanding grew, so did the sophistication of the therapies. These targeted therapies revolutionized treatment, offering relief to severe cases by specifically addressing the underlying immuneresponses causing the disease.
It is not known whether CD24 on cancer cells has a unique epitope that can be specifically targeted for cancer therapy. Combination therapy is the best approach in immunotherapy. Our extensive data showed that CD24 expressed on normal cells and cancer cells can be differentiated with our novel anti-CD24 antibody, ONC-781.
Located in Oss, the Netherlands, Lead Pharma is a clinical-stage biopharmaceutical company focused on the development of novel small-molecule therapies for patients suffering from autoimmune diseases (a subset of immune-mediated diseases) and cancer. . This includes therapies against the nuclear receptor ROR?t,
Weiner, chairman of the scientific advisory board of Tiziana Life Sciences, commented:
“We understand that this will be the first-ever study with ‘take-home’ capsules of any mAb for immunotherapies for human diseases.
Phase1b/2 clinical study to be conducted in the United States and several European countries. and Europe.
Strong Th1 cell-mediated immuneresponses were also observed for the vaccine candidates with either adjuvant. We are encouraged by the high level of neutralizing antibodies in combination with the strong Th1 response which we believe could play an important role in controlling infection.
Researchers decipher how a gut bacterium influences immunity By Corie Lok July 26, 2022 Breadcrumb Home News Researchers decipher how a gut bacterium influences immunity Study finds a molecule made by the bacterium that helps moderate immuneresponses. Study after study had suggested that A. muciniphila ’s cell membrane.
Both Jerry and Jeff have proven and successful track records in the design and execution of early and late-stage clinical trials that have evaluated novel immunetherapies. The backbone of NexImmune’s approach is its proprietary Artificial Immune Modulation (AIM TM ) nanoparticle technology platform. .
GAITHERSBURG, Md.,
In a Phase 2/3 clinical trial, the Pfizer-BioNTech COVID-19 Vaccine elicited a strong immuneresponse in this age group Three 3-µg doses had favorable safety profile similar to placebo in young children ages 6 months through 4 years in Phase 2/3 clinical trial Pfizer-BioNTech COVID-19 Vaccine now authorized in the U.S.
16, 2020 /PRNewswire/ — Cytiva , a global life sciences leader, is supporting Clover Biopharmaceuticals , a global clinical-stage biotechnology company, to help accelerate the development and manufacturing of a protein-based S-Trimer subunit vaccine candidate.
SHANGHAI , Nov. About Cytiva.
Due to the innovative design of deucravacitinib, Bristol Myers Squibb earned recognition with the 2019 Thomas Alva Edison Patent Award for the science underpinning the clinical development of deucravacitinib. Many patients have an inadequate response or do not respond at all to currently available therapies.
Gilead Strengthens Early Pipeline in Oncology and Inflammation Through the Acquisition of XinThera Gilead Sciences, Inc. Executive Vice President, Research, Gilead Sciences. About Gilead Sciences Gilead Sciences, Inc. The company was founded in 2021 by Stephen Kaldor, Ph.D., Qing Dong, Ph.D., and Gene Hung, M.D.,
Dr Ashley Seifert, an associate professor in the Department of Biology at the UK College of Arts and Sciences, and his research group have pioneered the use of spiny mice and other animal models to understand how complex tissue can regenerate, bridging regenerative biology and medicine. The Innate ImmuneResponse to Infection [Internet].
Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immuneresponses in a phase 1 trial.
GSK is a science-led global healthcare company with a special purpose: to help people do more, feel better, live longer.
CHENGDU, China , Feb.
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