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

Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

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Therapies Disease Engineering Medical Schools 114

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

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

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.

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Disease Clinical Trials Therapies Science 111

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?

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Small Molecule Trials Treatment Disease 229

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

Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

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Trials Disease Clinical Trials Clinical Research 75

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.

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Disease Treatment Licensing Licensing 105

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

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 98

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

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.

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Disease Protein Expression Engineering Treatment 80

Conversations in Drug Development Trends

OCTOBER 29, 2024

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?

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Clinical Research Disease Research Clinical Trials 72

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.

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Disease Research Drugs 59

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.

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Disease Clinical Trials Therapies Science 59

Conversations in Drug Development Trends

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

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Clinical Trials Trials Disease Drug Development 52

KIF1A

MARCH 2, 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 has created a platform to learn about autism-associated genes, identify risk factors for subgroups, and better inform new interventions.

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Laboratories Cell Biology Disease Research 119

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.

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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

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 117

Broad Institute

FEBRUARY 16, 2024

According to the study's first author, Soo Heon Kwak, a visiting scientist at Broad Institute in Flannick’s lab and now a clinician based at Seoul National University Hospital in Korea, the study's findings challenge our current way of thinking about T2D and other complex diseases, which buckets patients into black-and-white symptomatic categories.

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Medical Schools Hospitals Disease DNA 133

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

Drug Target Review

JANUARY 12, 2024

How does the integration of Real-World Data (RWD) with genomic biomarker data contribute to a more comprehensive understanding of disease progression and treatment response? Based on the drugs mechanism of action, we can make informed decisions. Most drugs do not work in all people.

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Doctors Clinical Trials Treatment Therapies 111

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

Science Daily: Pharmacology News

DECEMBER 13, 2023

Their findings uncover detailed information that could help overcome the limitations of today's immunotherapies and identify new drug targets for a wide range of diseases, including autoimmune diseases and cancer.

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Immune Response Government Disease Drugs 95

Drug Hunter

JUNE 25, 2023

Maze’s Oral GYS1 Inhibitor, MZE001 Sanofi is paying $150 million plus up to $600 million in milestones for Maze Therapeutics’ glycogen synthase 1 (GYS1) program, including MZE001, which aims to reduce glycogen substrate build-up in Pompe disease. Get ahead now by requesting a trial. already a member?

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Targeted Protein Degradation Computational Chemistry Licensing Licensing 130

Drug Target Review

SEPTEMBER 19, 2024

Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). The treatment often initially provides good symptom relief and those affected by the disease can live a largely normal life.

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Disease Drugs Treatment Drug Development 52

Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

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Small Molecule Cell Based Assays Disease Assay Development 105

KIF1A

FEBRUARY 24, 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. Theoretical and Experimental data KIF1A is so small, and moves so quickly, that it is very difficult to get information about individual steps.

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Disease Research Treatment Science 111

Broad Institute

OCTOBER 23, 2024

They knew that discovering the genetic cause of her disorder would help them find other people like her, help get the condition formally recognized as a new disease, and help them better advocate for research into new treatments. This is the first human disease caused by loss of one copy of a lncRNA gene.

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DNA RNA Disease Hospitals 128

KIF1A

OCTOBER 28, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research Genetic overlap between ALS and other neurodegenerative or neuromuscular disorders How do you define a disease?

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Disease Treatment Research Hospitals 105

Drug Hunter

MAY 22, 2023

These tools show remarkable selectivity over other caspase family members and high proteome selectivity overall, enabling rigorous interrogation of caspase-6’s role in a range of disease settings. Drug Hunter Premium is drug discovery, distilled, so you can quickly catch up and make informed decisions based on industry examples.

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Drugs Trials Disease 133