Broad Institute

SEPTEMBER 6, 2023

Diabetes and cardiac fibrosis are two conditions in dire need of new therapies,” said Todd Golub, director of the Broad Institute. For both patient populations, there are no safe and effective therapies for reversing disease. Florez is an institute member at the Broad, where he directs the Diabetes Research Group.

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Broad Institute

JANUARY 25, 2024

They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We

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Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

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Science Daily: Pharmacology News

MAY 16, 2024

In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

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Science Daily: Pharmacology News

AUGUST 29, 2024

For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. But a major hurdle has been finding a safe and efficient way to deliver those genes.

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Therapies Disease Research 117

Science Daily: Pharmacology News

JULY 17, 2024

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

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Therapies Treatment Disease Research 124

Broad Institute

APRIL 2, 2024

Scientists link certain gut bacteria to lower heart disease risk By Allessandra DiCorato April 2, 2024 Breadcrumb Home Scientists link certain gut bacteria to lower heart disease risk Study finds several species of cholesterol-metabolizing bacteria in people with lower cholesterol levels.

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Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Goodsell, RCSB Protein Data Bank and Scripps Research. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Always free.

Therapies 117

Therapies DNA Virus Clinical Trials 117

Chemical Biology and Drug Design

MAY 11, 2023

The figure depicts the neuroprotective role of crocin in Parkinson's disease. Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects.

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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

Drug Target Review

FEBRUARY 9, 2024

What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? These characteristics position ADSCs as promising candidates for addressing a wide range of medical conditions, from degenerative diseases to traumatic injuries.

Therapies 105

Therapies Immune Response Clinical Trials Treatment 105

Broad Institute

JANUARY 8, 2024

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Delivery dilemma Gene editing approaches promise to treat a range of diseases by precisely correcting genetic mutations that cause disease.

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Engineering Research Virus DNA 144

Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

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Drugs.com

DECEMBER 29, 2023

29, 2023 -- A new cellular therapy improved learning and memory in mice with Alzheimer's disease, researchers report.The therapy -- developed at the University of Nebraska Medical Center (UNMC) -- relies on both the immune system to. FRIDAY, Dec.

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Broad Institute

AUGUST 7, 2024

Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy By Allessandra DiCorato August 7, 2024 Breadcrumb Home Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy The method could accelerate the development of more effective adeno-associated viruses (AAVs).

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Therapies Research Engineering Packaging 84

BioPharma Drive: Drug Pricing

JULY 1, 2024

Like many of its cell therapy peers, Artiva, which originally sought an IPO in 2021, has shifted its strategy in hopes of riding a recent wave of investor interest in autoimmune disease research.

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Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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Therapies Cell Biology Clinical Trials Trials 118

Broad Institute

FEBRUARY 7, 2024

However, researchers lack efficient ways to explore how these genetic variants are molecularly connected to cardiovascular disease, limiting efforts to develop therapeutics. In a study published in Nature , the researchers applied this technique to endothelial cells, which line blood vessels.

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Disease Hospitals Medical Schools Therapies 105

Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.

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Disease Treatment Small Molecule Therapies 113

Drug Target Review

JUNE 27, 2023

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.

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Therapies Immune Response Engineering Drugs 105

Drug Target Review

JULY 31, 2023

Our inaugural report is a groundbreaking exploration of the remarkable advancements in cell and gene therapy that are revolutionising the field of drug discovery. Expert Insights We are honoured to have collaborated with renowned experts in the field of cell and gene therapy, who have generously shared their invaluable insights.

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Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. So, what makes these therapies unique?

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Broad Institute

NOVEMBER 21, 2024

Researchers identify source of a brain cancer’s deadly transformation By Corie Lok November 21, 2024 Breadcrumb Home Researchers identify source of a brain cancer’s deadly transformation Slow-growing glioma tumors become more aggressive when their cells shift identities and acquire new genetic mutations.

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Research Medical Schools DNA Hospitals 124

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

Chemical Biology and Drug Design

NOVEMBER 4, 2024

Alzheimer's disease (AD) is a progressive age-related neurodegenerative brain disorder that destroys memory and other important mental functions. ABSTRACT Alzheimer's disease (AD) is a progressive chronic age-related neurodegenerative brain disorder characterized by the loss of memory and other cognitive functions.

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ASPET

DECEMBER 22, 2023

This study provides a unique translational research opportunity to help both humans and dogs diagnosed with diseases that carry dismal prognoses in both species: histiocytic sarcoma (HS), hemangiosarcoma (HSA), and disseminated mastocytosis/mast cell tumor (MCT). Standard-of-care therapeutics broadly synergize with PTL.

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Clinical Trials Disease Drugs Therapies 189

FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs.

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FDA Disease Clinical Pharmacology Science 105

Fierce BioTech

NOVEMBER 21, 2023

Cell and Gene Therapy (CGT) has the potential to transform the life trajectory of children with genetic conditions amendable to gene modification with advances in treatment and possible cures. Pediatric CGT trials have the potential to cure diseases and prolong lives.

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Therapies Research Trials Treatment 91

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. 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

Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? There are a lot of drug development challenges that are, in some cases, unique to neuroscience, including neurological diseases, psychiatric disorders, and rare diseases affecting the nervous system.

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PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

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Advarra

MARCH 10, 2023

Having a one in four chance of developing sickle cell disease (SCD) is uniquely, and overwhelmingly, familiar to the African American population. Considered a rare disease, research in finding better therapies – and possibly even a cure – for SCD may be competing for funding with conditions impacting broader populations.

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Broad Institute

OCTOBER 11, 2023

Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. 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

Science Daily: Pharmacology News

NOVEMBER 13, 2023

Researchers have discovered the mechanism by which cholesterol in our diet is absorbed into our cells. This discovery opens up new opportunities for therapeutic intervention to control cholesterol uptake that could complement other therapies and potentially save lives.

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Therapies Disease Research 95

BioPharma Drive: Drug Pricing

JUNE 22, 2023

The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.

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Therapies FDA Trials Disease 132

Chemical Biology and Drug Design

APRIL 19, 2023

Abstract Alzheimer's disease (AD) is a progressive neurodegenerative disorder caused due to the damage and loss of neurons in specific brain regions. The current research aims to find natural substances that can be used as drugs to treat AD. It is the most common form of dementia observed in older people.

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Disease Therapies Research Drugs 100

Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

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