Broad Institute

JUNE 27, 2024

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.

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Drug Target Review

OCTOBER 16, 2023

How can PDOs revolutionise drug discovery and deepen our understanding of disease? Patient-derived organoids (PDOs) are proliferative 3D cell structures derived from tissue samples of both healthy and diseased tissue. Improved translatability to the patient and the disease state.

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Disease Immune Response Cell Biology Therapies 122

Broad Institute

OCTOBER 15, 2024

By Ari Navetta October 15, 2024 Credit: Allison Colorado, Broad Communications Eric Brown is studying the links between the microbiome and disease. The Xavier Lab looks at interactions between microbes in your gut and your immune system, and diseases such as arthritis and inflammatory bowel disease (IBD).

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Disease Immune Response Research Science 125

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

The Pharma Data

DECEMBER 4, 2021

The first peer-reviewed study in North America examining the timing between the first and second doses of COVID-19 mRNA vaccines shows that a longer dose interval leads to a stronger immune response. The study is funded by the Government of Canada through its COVID-19 Immunity Task Force (CITF).

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Broad Institute

JANUARY 19, 2024

By examining the genomes of bacteria collected from hundreds of people with persistent Salmonella infections, they discovered genetic mutations that both reduce the bacteria’s “virulence,” or ability to infect, and dampen the host’s immune responses, creating a kind of molecular camouflage that shields the bacteria from the immune system’s gaze.

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Immune Response Treatment Disease Research 119

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 24, 2023

1 Dr Ninh (Irene) La-Beck, from the Department of Immunotherapeutics and Biotechnology at the Texas Tech University Health Sciences Center (TTUHSC) Jerry H. The grant will enable her to investigate the interaction of nanoparticles with the immune system and cancer, supporting the development of new drug delivery systems.

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Immune Response Pharmacy Treatment Virus 111

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. We’re deeply grateful for that.”

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The Pharma Data

MAY 16, 2021

Adjuvanted recombinant COVID-19 vaccine candidate triggered strong neutralizing antibody responses in all adult age groups. High immune response after a single dose in patients with prior infection shows strong booster potential. For media and investors only. Issued: London UK. About the Phase 2 study. Sanofi, Empowering Life.

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Immune Response Vaccine Trials Treatment 52

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 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

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. It’s the first time they have seen the nervous system directly tamp down an allergic response.

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Immune Response Regulations Treatment Therapies 104

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. Chronic obstructive pulmonary disease Chronic obstructive pulmonary disease (COPD) is the third leading cause of global deaths.

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Immune Response Disease Science Therapies 113

PerkinElmer

JANUARY 25, 2023

Laboratory professionals in all areas of science have experienced significant changes in recent years, and 2022 was no exception. Additionally, chronic disease, cancer and neurodegenerative disorders like Alzheimer’s were top of mind last year for diagnostics and drug R&D teams alike.

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PLOS: DNA Science

MAY 16, 2024

These factors are converging to enable both identification of novel infectious diseases as well as microbial resistance, before these threats can impact public health, write a team from the European Society for Clinical Microbiology and Infectious Diseases in Frontiers in Science. COVID clearly caught us off guard.

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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.

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Small Molecule Therapies Treatment Disease 111

The Pharma Data

NOVEMBER 17, 2020

Lead Pharma has entered into a collaboration and license agreement with Roche , hoping to lead the way in the development of oral small molecules for immune-mediated diseases like rheumatoid arthritis, psoriasis and inflammatory bowel diseases. . The agreement encompasses Lead Pharma’s entire pipeline.

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Small Molecule Disease Immune Response Licensing 52

Drug Target Review

MARCH 26, 2024

Last summer, we published a first signal of efficacy in humans in the LANCET Infectious Diseases for our lead broad-spectrum influenza candidate, OVX836. DGG: Resilience: In science, we can’t predict and control everything. Sometimes science “speaks” and sometimes it doesn’t.

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Vaccine Virus Immune Response Science 110

DrugBank

MAY 15, 2024

This method was more about serendipity than science. Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. But as molecular biology has advanced, so too has our approach to finding new drugs.

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Small Molecule Molecular Biology Drugs Disease 98

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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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

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

Drug Target Review

APRIL 26, 2023

Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. 1 RNA-delivery particles have the potential to revolutionise the treatment of lung diseases such as cystic fibrosis.

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The Pharma Data

NOVEMBER 10, 2020

. P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 Oral and nasal administration routes are both physiologic approaches to stimulate the mucosal immune system to induce disease modifying benefits.”.

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Clinical Trials Science Disease Trials 40

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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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. 1, 2021 11:00 UTC. FOSTER CITY, Calif. &

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DrugBank

MAY 6, 2024

This category encompasses a broad spectrum of treatments including vaccines, blood components, gene therapies, and more, each tailored to combat diseases in highly specific ways. 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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FDA Approval Therapies Treatment Clinical Trials 96

Drug Target Review

JUNE 9, 2023

C ould you explain the specific mechanisms or techniques used in the pre-clinical research to translate the genetics of the adaptive immune system into clinical products for disease detection and treatment? What types of diseases or conditions were targeted in the pre-clinical research phase? a healthcare informatics company.

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Treatment Packaging Clinical Research Therapies 105

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. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. About BioNTech.

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Immune Response Vaccine Clinical Trials Virus 52

The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. However, if the immune system malfunctions, it can attack healthy cells, tissues and organs. Autoimmune disease impacts different parts of the body, weakening functionality. Autoimmune diseases affect more than 24 million people in the U.S.

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Disease Clinical Trials Small Molecule Trials 52

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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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

Codon

SEPTEMBER 29, 2024

The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. While mRNA data was often illuminating, their interpretation was tricky; more akin to art than science. Subscribe to Asimov Press.

Engineering 141

Engineering Virus DNA Therapies 141

Broad Institute

AUGUST 7, 2023

MIT biology PhD student Bingxu Liu and Rebecca Carlson PhD ’23, a recent graduate of the Medical Engineering and Medical Physics program through the Harvard-MIT Division of Health Sciences and Technology, are the lead authors of the paper, which appears in Science. Paper Cited Liu B, Carlson R, et al. Human STING is a proton channel.

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Regulations Clinical Trials Engineering Science 91

PLOS: DNA Science

OCTOBER 3, 2024

One of the oldest and most successful immunotherapies is simpler: a tamed version of a classic vaccine, against the infectious disease tuberculosis (TB). Their report appears in Disease Models and Mechanisms. Eventually, a strain emerged that seemingly protected against a host developing the associated infectious lung disease.

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Vaccine Immune Response DNA Treatment 52

The Pharma Data

DECEMBER 8, 2020

With the proceeds, Qu Biologics will complete stage 1 of the RESTORE Phase 2 clinical trial for patients with moderate to severe Crohn’s disease and its Phase 2 Study to assess activation of anti-cancer immune response in colon cancer. “We

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