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. In 2025, I think well see breakthroughs that allow us to make significant advances in rare diseases, where the data is small.

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Clinical Trials Trials Pharmaceuticals Drug Development 128

On Medicine

FEBRUARY 28, 2024

What is Perthes’ disease? Perthes’ Disease is a rare condition that affects the developing femoral head (the “ball” of the “ball and socket” hip joint) of a child due to poor blood supply. This in turn can cause significant pain for children with Perthes’ disease and limit their activity levels significantly.

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Disease Doctors Treatment Trials 109

Broad Institute

OCTOBER 23, 2023

By Makenzie Kohler October 23, 2023 People of South Asian ancestry around the world have more than double the risk of developing cardiometabolic diseases like diabetes, heart attack, and stroke compared to other populations. They will also have the opportunity to inform the researchers on whether their health status changes over time.

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Disease Research Hospitals DNA 145

Antidote

JANUARY 12, 2023

Alzheimer’s disease is the most common type of dementia in the United States, impacting around 6.2 Since it was first observed in the early 1900s, a lot of information has been uncovered through medical research. million people above the age of 65.

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Disease Research 98

Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Our team of chemists then uses this information to develop novel therapies.

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Therapies Disease RNA Engineering 102

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

Disease 124

Disease Research Science Medical Schools 124

Broad Institute

OCTOBER 30, 2024

Researchers have typically analyzed these cells as a mixed-together group, but this approach can miss rare cell types, and makes it difficult to draw conclusions about how cells interact to drive the disease. Eventually, we hope that these methods can help inform the development of drugs or even guide patients’ individual treatments.”

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Research RNA Medical Schools Treatment 127

Drug Target Review

MARCH 12, 2024

The study’s findings offer new insights into the biochemical language microbes use to influence distant organ systems, which could revolutionise the way researchers approach disease. microbeMAAST: a taxonomically informed mass spectrometry search tool for microbial metabolomics data. This study was published in Cell Reports.

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Disease Biochemical Pharmacology FDA Approval Pharmacy 111

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

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

Covalent Modifiers

MARCH 19, 2024

3c01661 Dysregulation of the ubiquitin-proteasome systems is a hallmark of various disease states including neurodegenerative diseases and cancer. Additionally, we report a ligand-bound crystal structure of the most potent molecule in complex with UCHL1, providing insight into the binding mode and information for future optimization.

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Fragment Screening Disease Treatment 195

Conversations in Drug Development Trends

MARCH 12, 2024

Biomarkers provide a rapid way to inform many clinical oncology study development decisions, including study endpoints. They can inform intervention outcomes, serve as study endpoints, and are advantageous across the spectrum of cancer care. What are they, how do they work, and how can they benefit your oncology study?

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Clinical Trials Immune Response Trials Disease 72

Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

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Disease Clinical Trials Trials Pharmaceutical Companies 81

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.

Disease 112

Disease Clinical Trials Therapies Science 112

Drug Hunter

AUGUST 15, 2023

LRRK2 has been a hotly pursued drug target for Parkinson’s Disease based on human genetics. Drug Hunter Premium is drug discovery, distilled, so you can quickly catch up and make informed decisions based on industry examples. Get ahead now by requesting a trial. already a member?

Small Molecule 229

Small Molecule Trials Treatment Disease 229

Science Daily: Pharmacology News

JUNE 2, 2023

Researchers from 24 countries have analyzed the genomes of 809 individuals from 233 primate species, generating the most complete catalog of genomic information about our closest relatives to date. In addition, using a specially designed AI algorithm, the genomic data enable new insights into the genetic causes of human diseases.

Disease 199

Disease Research 199

Broad Institute

AUGUST 17, 2023

Chao joined the NIH as a Post-Baccalaureate Intramural Research Training Award fellow, where she processed and analyzed genomic data in the context of rare disease. In that role, Chao continued to work in rare disease genomic analysis, identifying sections of duplicated or deleted genomic sequences called copy number variants.

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Disease Production Science Research 98

Drug Target Review

APRIL 4, 2024

What are the key findings of the preclinical study regarding the neurophysiological brain state in a Parkinson’s disease psychosis (PDP) model? The animal model is based on creating a dopamine-deficient state, by a specific lesion of dopaminergic cells, and then exposing it to repeated substitution therapy with L-DOPA.

Disease 106

Disease Treatment Licensing Licensing 106

SCIENMAG: Medicine & Health

AUGUST 7, 2023

Philadelphia, August 8, 2023 – There is a need to better deliver information on medical nutrition therapy for patients with Parkinson’s disease (PD).

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Disease Therapies Engineering 87

Broad Institute

SEPTEMBER 5, 2024

In doing that, we lost information about the uniqueness of each cell, and we had limited information about the spatial heterogeneity of tissues that plays a decisive role in disease. And we can derive meaningful biological information by doing this.

Disease 85

Disease Protein Expression Engineering Treatment 85

PLOS: DNA Science

JULY 27, 2023

That information led, thanks to vaccine shelved from the first SARS circa 2003, to the rapid development and deployment of mRNA vaccines against the new infectious disease. which spit out information guiding the trimming of tumors in real time, a bit reminiscent of its ancestor Hal the computer from 2001: A Space Odyssey.

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Disease DNA Virus RNA 95

FDA Law Blog: Drug Discovery

JANUARY 30, 2025

This target completion date is informed by the natural history of the disease, availability of alternative treatment, anticipated recruitment timeline, and the projected timeline for efficacy analysis(es); 2) the sponsors progress and plans for postapproval conduct of the trial provide sufficient assurance to expect timely completion of the trial.

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FDA Trials Disease Drugs 105

Drug Target Review

JANUARY 14, 2025

provide more general information. ChatGPT can also aid in deciding which visualisation options are available and what information each will provide, based on the data and your research question. By posing questions such as, ‘Which database should I use to search for data on the effects of longevity drugs in rodents?’

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Drugs Protein Expression Science Research 69

Broad Institute

MARCH 12, 2025

By Leah Eisenstadt March 12, 2025 Credit: Broad Communications Scientists in the Spatial Technology Platform at the Broad develop, use, and share spatial -omics approaches, such as Perturb-FISH, that can reveal the roles of genes in various cell types and the impact of intercellular interactions on health and disease.

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RNA Science Research Disease 92

Drug Patent Watch

DECEMBER 12, 2024

Consider drugs targeting rare diseases or specialized conditions. It allows us to make more informed decisions and identify opportunities that may have been overlooked using traditional methods.” Staying informed about future trends, such as biosimilars and personalized medicine, is crucial for adapting strategies.

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Licensing Licensing Drugs Biosimilars 96

Chemical Biology and Drug Design

JANUARY 15, 2024

The expression levels of endoplasmic reticulum (ER) stress-related protein and silent information regulator 1/nuclear factor kappa-B (Sirt1/NF-κB) signaling pathway were used to observe the therapeutic effect of naringin.

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Disease International Regulations Treatment 100

Agency IQ

JUNE 21, 2024

BY ALEXANDER GAFFNEY, MS, RAC This week, PETER MARKS, director of FDA’s Center for Biologics Evaluation and Research (CBER), spoke at several sessions of the Drug Information Association (DIA) annual meeting. AgencyIQ has the highlights, with new information on a “Rare Disease Hub” modeled as a sort of Center of Excellence for rare diseases.

Disease 59

Disease Research Drugs 59

Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

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Clinical Trials Disease Trials Therapies 52

Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing. We believe we are at a breakthrough moment in Alzheimer’s disease research.

Disease 59

Disease Clinical Trials Therapies Science 59

Broad Institute

FEBRUARY 13, 2025

The Primer on Medical and Population Genetics is a series of weekly lectures on genetics topics related to human populations and disease. If you'd like more information about the primer or have suggestions for future topics, please contact seminar series organizers Michelle McNulty and Nicole Rockweiler.

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Bioethics Disease Research 52

Advarra

NOVEMBER 14, 2024

The draft guidance highlights several key points pertinent to international trials: Diversity of expertise: KOLs with varied backgrounds and expertise enhances the DMC’s ability to address complex clinical and safety issues, and is crucial for interpreting data accurately and making informed decisions. local standards of care).

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Clinical Trials Trials International Clinical Research 97

Covalent Modifiers

MAY 1, 2023

Golosov, and Viktor Hornak Journal of Chemical Information and Modeling 2023 63 (7), 2170-2180 DOI: 10.1021/acs.jcim.3c00004 3c00004 Accurate estimation of the pKa’s of cysteine residues in proteins could inform targeted approaches in hit discovery. Ernest Awoonor-Williams*, Andrei A.

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Disease Drugs 130

Broad Institute

OCTOBER 28, 2024

Scientists then annotate the cells with this information, a process that can take days or even weeks, depending on the number of cells being labeled, and requires labor-intensive literature and database searches. Let’s say you have a therapeutic that is targeting a specific cell state identified in the context of a certain disease.

Engineering 119

Engineering Cell Biology Disease RNA 119

Broad Institute

MAY 16, 2024

The enormous challenge of getting therapies past this barrier — a highly selective membrane separating the blood from the brain — has stymied the development of safer and more effective gene therapies for brain diseases for decades. This can make it difficult to translate a gene therapy using these AAVs from animals to humans.

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Therapies FDA Approval Engineering Disease 137

Broad Institute

DECEMBER 18, 2023

To achieve this, the authors called for more genetics and longitudinal data types from patients of diverse ancestries, more data- and tool-sharing across sectors, the development of quantitative biomarkers for measuring disease mechanisms, and more. The following conversation was edited for length and clarity.

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Drug Development Clinical Trials Drugs Disease 130

NIOSH Science Blog: Drugs

JUNE 29, 2023

In this blog, the Centers for Disease Control and Prevention’s National Institute for Occupational Safety and Health (NIOSH) and National Center for Environmental Health teamed up with the Environmental Protection Agency (EPA) to provide employers, workers, and the general public information to help stay protected from wildfire smoke.

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Disease 112

Drug Hunter

MAY 21, 2023

These findings highlight the versatility of DCAF1 for TPD and offer a new avenue for developing therapies for diseases where catalytic inhibition is not an option. Drug Hunter Premium is drug discovery, distilled, so you can quickly catch up and make informed decisions based on industry examples. Get ahead now by requesting a trial.

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Targeted Protein Degradation Trials Therapies Disease 131