PLOS: DNA Science

OCTOBER 19, 2023

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.

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Drug Target Review

JANUARY 23, 2024

Cell-based therapies, such as tumour-infiltrating lymphocyte (TIL) therapy, have also drawn significant attention following promising results in a melanoma trial. Such approaches apply artificial intelligence (AI) and machine learning (ML) to predict mutations most likely to be good targets for the immune system.

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Drug Target Review

DECEMBER 13, 2024

2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. These can include anti-tubulin compounds, potentially reducing side effects and improving quality of life.

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Therapies Treatment Clinical Trials Drugs 98

Drug Target Review

FEBRUARY 28, 2024

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.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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Therapies Immune Response DNA Virus 69

Broad Institute

OCTOBER 11, 2023

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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Research Immune Response Clinical Trials Therapies 137

Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Stem cell transplants primarily help the immune response through the “graft-versus-leukaemia effect,” and we have to manage the “graft versus host effect.”

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Therapies Treatment Vaccine FDA Approval 64

Drug Target Review

OCTOBER 16, 2023

This is particularly pertinent in immune-based cell therapies like CAR-T, where animals do not mimic the human immune response. 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.

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Disease Immune Response Cell Biology Therapies 122

Drug Target Review

DECEMBER 27, 2023

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 immune response and drive therapeutic efficacy.

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Immune Response Science Clinical Development Drugs 105

Alta Sciences

JUNE 11, 2024

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

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Therapies Molecular Biology Immune Response Science 52

Drug Target Review

OCTOBER 4, 2023

More recently, researchers have begun to look at conjugating immunomodulatory agents to antibodies to directly activate an immune response against the tumour. This approach – immune-stimulating antibody conjugates (ISACs) – uses a payload that stimulates the innate and adaptive immune responses, recruiting tumour-fighting T cells.

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Drug Target Review

APRIL 17, 2024

HLA are the critical molecules that our immune systems use to present peptides to T cells and facilitate recognition and killing in immune responses to pathogens. HLA-II presents peptides to CD4 T cells thought to be important for indirectly helping immune responses and facilitating antibody production.

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Immune Response Laboratories DNA Virus 105

Drug Target Review

OCTOBER 19, 2023

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.

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Immune Response Regulations Treatment Therapies 104

Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 9 In addition, host immune responses further add to the complexity of developing cell-specific AAV capsids for clinical applications.

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Drug Target Review

JUNE 9, 2023

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.

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Drug Target Review

MARCH 21, 2024

There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. AAV-based therapies often can’t be dosed more than once due to concerns about an immune response. Molecular Therapy-Nucleic Acids. Human molecular genetics.

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Therapies RNA Immune Response Treatment 64

Drug Target Review

NOVEMBER 6, 2023

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).

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Small Molecule Therapies Treatment Disease 111

LifeSciVC

MAY 9, 2024

These connections provide all-too-important ‘reasons to believe’ for further investment and movement into earlier lines of therapy. However, despite this long winter, new approaches, ranging from CD3 bi-specifics, to cell therapies, to cancer vaccines, and more recent novel intracellular mechanisms have started to turn the tide.

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Immune Response Vaccine Therapies Engineering 78

Drug Target Review

JULY 7, 2023

Antibodies protect us by attaching to molecules on those invaders (known as antigens) and triggering our body’s natural immune response to destroy them. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.

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Disease Immune Response Therapies Treatment 98

PLOS: DNA Science

FEBRUARY 8, 2024

The molecular tools of CRISPR were borrowed and developed from the natural immune response 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.

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Immune Response DNA Engineering Virus 98

Drug Target Review

JANUARY 15, 2024

Researchers from the Nara Institute of Science and Technology (NAIST) have used the reverse-blastocyst complementation (rBC) method to understand the conditions required to form lungs in rat-mouse chimeric models. Pluripotent Stem Cell-Based Cell Therapy – Promise and Challenges. The Pathology of Chronic Obstructive Pulmonary Disease.

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Immune Response Disease Science Therapies 113

DrugBank

MAY 6, 2024

Unlike conventional chemical-based medicines, biologics offer targeted therapies derived from living cells, which can result in more effective and personalized treatments. A Wave of Innovations The year 2024 has already proven to be significant for the field of biologics, witnessing a notable increase in FDA approvals.

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Drug Target Review

DECEMBER 5, 2024

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.

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The Pharma Data

APRIL 22, 2023

These “acute inflammatory lesions” then attract other myeloid cells, as well as self-reactive T and B cells that belong to the immune system’s second arm, known as the “adaptive immune system” and directly attack the myelin covering. “Current MS therapies do not specifically target myeloid cells.

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Codon

SEPTEMBER 29, 2024

While mRNA data was often illuminating, their interpretation was tricky; more akin to art than science. It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life. As such, they were quietly abandoned. Subscribe to Asimov Press.

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The Pharma Data

APRIL 15, 2022

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.

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Immune Response Vaccine Clinical Trials Virus 52

Codon

APRIL 9, 2023

“Computer science is to biology what calculus is to physics.” ” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Read Basic Science *mRNA decoding in human is kinetically and structurally distinct from bacteria.

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Therapies RNA Engineering Vaccine 52

Drug Target Review

MAY 14, 2024

The combination of P1 with antiPD1 more efficiently activated innate and adaptive immune responses 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.

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Engineering Immune Response FDA Approval Clinical Trials 59

Codon

JULY 14, 2024

While this departs from the reductionist, single-component therapies that have dominated transfusion medicine since World War II, emerging data underscores that whole blood transfusions—blood with all its parts—yield better outcomes following severe blood loss than transfusions involving discrete blood components. Always free.

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Production Clinical Trials Trials Therapies 140

Broad Institute

OCTOBER 4, 2023

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.

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Drug Target Review

APRIL 3, 2024

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.

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Science Clinical Development Treatment DNA 105

DrugBank

MAY 15, 2024

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.

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Small Molecule Molecular Biology Drugs Disease 98

The Pharma Data

JANUARY 31, 2021

–( 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.

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The Pharma Data

SEPTEMBER 8, 2020

About AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology.

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Olympian Clinical Research

MAY 6, 2024

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 immune responses causing the disease.

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Treatment Small Molecule Clinical Research Immune Response 52

The Pharma Data

SEPTEMBER 8, 2020

LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. About AstraZeneca.

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Science Vaccine Clinical Trials Pharmaceutical Companies 52

PLOS: DNA Science

OCTOBER 3, 2024

The idea to redirect an immune response against a pathogen to fight cancer goes back to 1891, when Manhattan bone surgeon William Coley became intrigued by a man’s neck tumor that melted away after he contracted a nasty Streptococcus skin infection. For many patients, BCG stops the cancer.

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