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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Disease Research Science Medical Schools 124

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. By Sarah Wicks & James E.

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FDA Disease Clinical Pharmacology Science 105

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. The collaboration will focus on advancing three programs over the next three years.

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Disease Research Therapies Treatment 136

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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Therapies Engineering Treatment Molecular Biology 116

PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here.

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Therapies Clinical Trials DNA Trials 95

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 98

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

Fierce BioTech

SEPTEMBER 5, 2024

How can life sciences organizations effectively identify and recruit participants with specific genetic variants? How can life sciences organizations effectively identify and recruit participants with specific genetic variants? How can they engage providers and raise awareness of gene therapies for the patients who need them most?

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Trials Disease Clinical Trials Therapies 87

National Institute on Drug Abuse: Nora's Blog

FEBRUARY 1, 2024

50 years after founding, NIDA urges following science to move beyond stigma area Thu, 02/01/2024 - 11:20 Nora's Blog February 1, 2024 Image NIDA Image In 2024, NIDA celebrates its 50th anniversary. The knowledge generated by NIDA research has also led to a robust and rigorous science of drug use prevention.

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Science FDA Approval Treatment Research 127

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 109

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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Therapies Engineering Disease Research 112

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 101

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 100

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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Therapies Drug Development Disease Clinical Development 104

Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

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Disease Clinical Trials Therapies Science 111

Science Daily: Pharmacology News

AUGUST 9, 2023

Promising preclinical results show hematopoietic stem cell therapy was effective in rescuing memory loss, neuroinflammation and beta amyloid build-up in a mouse model of Alzheimer's disease.

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Therapies Disease 72

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

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

SCIENMAG: Medicine & Health

JULY 28, 2023

Lynch, lead author, is now a research fellow in the Department of Veterinary and Animal Sciences. Pearson, co-author of the paper, is now a NEWVEC post-doctoral researcher at UMass Amherst. Credit: UMass Amherst […]

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Research Therapies Disease Doctors 82

SCIENMAG: Medicine & Health

OCTOBER 2, 2023

Researchers at the University of Cologne’s CECAD Cluster of Excellence for Aging Research and the CEPLAS Cluster of Excellence for Plant Sciences have found a promising synthetic plant biology approach for the development of a therapy to treat human neurodegenerative diseases, especially Huntington’s disease.

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Therapies Disease Science Research 52

Science Daily: Pharmacology News

AUGUST 30, 2023

Scientists have made a `paradigm shifting' discovery on the mechanisms required for learning and memory that could lead to new therapies for Alzheimer's disease and potentially Down syndrome.

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Therapies Disease 74

Science Daily: Pharmacology News

JULY 12, 2023

They also found that co-transplantation of neuronal cell therapy with host regulatory T cells resulted in effective suppression of needle trauma and significant improvement in the survival and recovery of grafts. These findings suggest a path for the 'realistic' use of cell therapy to treat neurodegenerative disorders.

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Therapies Immune Response Disease Research 71

PLOS: DNA Science

FEBRUARY 1, 2024

Five people treated for pituitary dwarfism decades ago with human growth hormone (hGH) pooled from cadavers have shown cognitive decline reminiscent of early-onset Alzheimer’s disease. None had mutations that cause Alzheimer’s disease, ruling out genetics as a cause. More than 85 animal species develop prion diseases.

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Disease Treatment DNA Research 97

Drug Target Review

NOVEMBER 23, 2023

elegans be applied to the development of targeted therapies for neuropsychiatric conditions like eating disorders, OCD, and PTSD? If so, the concepts we develop in simpler animals should help us understand the more complex systems in the human brain and make good hypotheses about how brain diseases arise and can be treated.

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Therapies Molecular Biology Laboratories Government 98

Drug Target Review

FEBRUARY 29, 2024

From a very young age, I was drawn to science and medicine. This early exposure to bench science had a long-lasting impact. Through further research and work as a principal investigator on clinical studies for several central nervous system indications, I laid the groundwork for a career in gene therapy drug development.

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Therapies Disease Clinical Trials Science 111

Drug Target Review

DECEMBER 20, 2024

Their work focuses on creating ‘digital twin generators’ – AI-driven models that predict how a patients disease may progress over time. As Smith puts it, “now the viewpoint is that computational science is very powerful, and these AI/ML methods are going to be transformative in a lot of ways.”

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Clinical Trials Trials Pharmaceuticals Drug Development 126

FDA Law Blog: Drug Discovery

MAY 19, 2024

By Sarah Wicks — On May 21, 2024, the EveryLife Foundation for Rare Diseases (ELF) will host a Scientific Workshop at the National Press Club in Washington, D.C. aimed at identifying and characterizing the challenges in developing therapies for ultra-rare diseases and conditions that affect exceedingly small populations.

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Science Daily: Pharmacology News

FEBRUARY 22, 2024

A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.

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Therapies Clinical Trials Treatment Disease 81

Science Daily: Pharmacology News

NOVEMBER 13, 2023

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

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Therapies Disease Research 83

Drug Target Review

JUNE 15, 2023

Interested in a more disease-focused training for his postdoc, he pursued work in the National Institutes of Health (NIH), particularly in the National Center for Advancing Translational Sciences (NCATS), US. The core of their platform aims to modulate disease-relevant mRNAs that have been previously thought to be undruggable.t.

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RNA Small Molecule Disease Assay Development 96

Drug Target Review

AUGUST 6, 2024

A number of areas represent significant opportunities for Antibody Drug Conjugates (ADCs) beyond oncology, leveraging their ability to deliver therapeutic agents specifically to diseased cells or tissues while minimising off-target effects. Oncology also offers high returns and the opportunity to address severe, life-threatening conditions.

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Therapies Pharmaceutical Companies Disease Treatment 117

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

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FDA Approval Therapies FDA Disease 52

BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

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Science RNA Therapies Disease 57

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. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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Therapies Treatment Vaccine FDA Approval 64

Fierce BioTech

JUNE 6, 2023

Accelerating the Development of Nucleic Acid Therapies at Harwell Science and Innovation Campus Harwell Campus offers rich opportunities for pioneering NAT technology on all fronts, and the campus is attracting biotechnology companies of all sizes – from Moderna to start-ups to Government research institutes and accelerators.

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Science Therapies Molecular Biology Government 52

KIF1A

FEBRUARY 10, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. This highlights the heterogeneity of KAND, and the importance of identifying KIF1A mutations in other rare disease groups.

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