Drug Target Review

JULY 7, 2023

Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. Antibodies protect us by attaching to molecules on those invaders (known as antigens) and triggering our body’s natural immune response to destroy them.

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Disease Immune Response Therapies Treatment 98

Drug Target Review

NOVEMBER 17, 2023

NK cells are among the front line of protection from infected and abnormal cells as part of the ‘innate immune response’. 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).

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Therapies Cell Biology Clinical Trials Trials 118

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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Therapies FDA Clinical Trials DNA 111

Drug Target Review

OCTOBER 2, 2024

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.

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Therapies Disease Engineering RNA 98

Plenge Gen

APRIL 7, 2020

Pharmacologic intervention has the opportunity to impact disease progression in the SARS-CoV-2 / COVID-19 crisis. Repurposing of approved therapies is the fastest way to impact patients today, as these medicines have regulatory approval to enable investigator-initiated trials and have a manufacturing process to ensure drug supply.

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Immune Response Disease Clinical Trials Drugs 52

Drug Target Review

JULY 4, 2023

Among the many remarkable components that make up this complex defence network, T cells have emerged as powerful guardians, orchestrating an array of immune responses with great precision.

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Immune Response Engineering Treatment Therapies 52

Drug Target Review

JANUARY 23, 2024

In April 2023, we saw the first randomised data applying an individualised vaccine in combination with checkpoint inhibition for resectable melanoma, demonstrating benefit in terms of time to disease recurrence. Espen integrates clinical and research expertise as Director of Medical Affairs.

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Treatment Immune Response Vaccine Therapies 122

Drug Target Review

NOVEMBER 17, 2023

NK cells are among the front line of protection from infected and abnormal cells as part of the ‘innate immune response’. 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).

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Therapies Clinical Trials Immune Response Trials 103

Drug Target Review

MARCH 1, 2024

How significant are the results in terms of advancing TCR-T cell therapy and AMP immunotherapy for the treatment of solid tumours? We have already applied this approach to several important tumour targets where enhancements in TCR-T therapy may lead to important gains in clinical benefit, including mutated KRAS, NY-ESO, and HPV E7 antigens.

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Immune Response Therapies Vaccine Small Molecule 105

Drug Target Review

JANUARY 2, 2024

In research published in Scientific Reports , 1 investigators focused on mesenchymal stem cells (MSCs), known for their potential in treating cell defects and regulating immune responses. We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun.

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RNA Immune Response Therapies Disease 128

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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Therapies Engineering Molecular Biology Science 105

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

The Pharma Data

AUGUST 16, 2021

Better activation of innate and adaptive immune responses 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.

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Immune Response Vaccine Virus RNA 52

Drug Target Review

SEPTEMBER 9, 2024

functions to reset the immune system and potentially provide remission for allergic diseases? 1104 acts upstream in the inflammatory response, targeting one of the first key players: antigen-presenting cells (APCs). In allergic diseases, APCs identify and present unknown antigens to the immune system.

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Disease Immune Response Clinical Trials Treatment 64

Drug Target Review

DECEMBER 28, 2023

In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. These neoantigens are identified by T cells of the immune system as foreign proteins and thus trigger an immune response. Neoantigens are recognised as non-self and trigger an immune response.

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Bioinformatics Immune Response DNA Vaccine 105

Broad Institute

OCTOBER 11, 2023

He also read everything he could about her disease, including emerging evidence that suggested that the immune system could recognize and kill Merkel cell carcinoma. His mother had a presentation of the disease that suggested her immune system was already on the job.

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Research Immune Response Clinical Trials Therapies 137

Drug Target Review

JANUARY 15, 2024

Cancer is a disease driven by variable genetic mutations. Our innate immune system evolved to clear genetically diverse pathogens and limit host toxicity, raising the possibility that it can produce similar effects in cancer. The preclinical results demonstrated significant immune responses and tumour regression.

Treatment 105

Treatment Immune Response Therapies Clinical Trials 105

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

MARCH 21, 2024

Dystrophin is mutated in Duchenne muscular dystrophy (DMD), a rare disease that causes progressive muscle weakness and atrophy. 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. Of note, mRNA is redosable as opposed to AAV.

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Therapies RNA Immune Response Treatment 64

The Pharma Data

OCTOBER 14, 2021

Biogen today announced results of a new analysis of immune response to the COVID-19 vaccine among people with multiple sclerosis (MS). Biogen today announced results of a new analysis of immune response to the COVID-19 vaccine among people with multiple sclerosis (MS). Using data from the MS PATHS network in the U.S.,

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Vaccine Therapies Disease Immune Response 52

Drug Target Review

DECEMBER 13, 2023

Combinatorial analytics approaches identify combinations of features that together are associated with the disease phenotype in patient sub-groups, capturing the non-linear effects of interactions between multiple genes. The combinatorial approach is considerably more sensitive than GWAS and requires much smaller patient populations.

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Bioinformatics Disease Regulations Immune Response 145

Drug Target Review

OCTOBER 19, 2023

It’s the first time they have seen the nervous system directly tamp down an allergic response. A potential game-changer in medicine The implications of this research are vast, potentially transforming the way we treat a variety of diseases involving both the immune and nervous systems.

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Immune Response Regulations Treatment Therapies 104

Drug Target Review

NOVEMBER 7, 2024

Introduction Therapeutic antibodies have proven to be indispensable medicines for addressing the most debilitating diseases. 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.

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Disease Immune Response Regulations Engineering 52

Drug Target Review

JANUARY 15, 2024

Chronic obstructive pulmonary disease Chronic obstructive pulmonary disease (COPD) is the third leading cause of global deaths. The pathogenesis of COPD is based on the innate and adaptive inflammatory immune response to the inhalation of toxic particles and gases. The Pathology of Chronic Obstructive Pulmonary Disease.

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Immune Response Disease Science Therapies 113

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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Treatment Packaging Clinical Research Therapies 105

Drug Target Review

NOVEMBER 6, 2023

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. 2 It is this complexity that necessitates powerful, targeted combination therapies. 2 It is this complexity that necessitates powerful, targeted combination therapies.

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Small Molecule Therapies Treatment Disease 111

Crown Bioscience

AUGUST 2, 2021

In this blog we will explore immune-mediated inflammatory diseases (IMIDs) —which represent a diverse group of conditions characterized by an excessive and/or inappropriate immune response.

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Disease Drug Development Immune Response Drugs 52

Alta Sciences

JANUARY 11, 2024

It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure. However, there are significant challenges associated with the successful development of these complex, leading-edge therapies.

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Therapies Immune Response Disease Virus 52

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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Therapies Virus DNA Compliance 52

SugarCone Biotech

JUNE 10, 2024

Targeting 4-1BB remained of interest to the immuno-oncology field as cell culture experiments and tumor models in mice suggested that robust anti-tumor immune responses could be triggered by anti-4-1BB antibody therapies. This is the T cell type most closely associated with anti-tumor immune responses.

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Immune Response Production Regulations Clinical Trials 88

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.

Immune Response 105

Immune Response Science Clinical Development Drugs 105

Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. 15 ISACs use small immuno-agonist molecules conjugated to the antibody to boost the immune response while minimising toxicity.

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Small Molecule Drugs Immune Response Targeted Protein Degradation 126

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

DECEMBER 13, 2024

These types differ in their etiology, natural history, and present distinct challenges in disease management. 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.

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Therapies Treatment Clinical Trials Drugs 52

The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

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Therapies Clinical Trials Small Molecule Immune Response 52

Drug Target Review

SEPTEMBER 11, 2023

Their findings, published in Nature Biotechnology, have broad applications as mucins are associated with many diseases, including cystic fibrosis, respiratory diseases, and viruses. This sugar-coated mucin binding to checkpoint receptors signals that the cancer cell poses no threat, consequently blocking the immune response.

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Immune Response Research Engineering Virus 116

Drug Target Review

JULY 11, 2023

In the quest to combat and eradicate diseases, scientists and researchers are increasingly turning to the immense potential of artificial intelligence (AI) as a tool. It was here that the Impact Medicine Fund was raised as a concept around wellness for those with disease, with the ambition of unlocking some of this vast potential.

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