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

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

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

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

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? Treatment with mesdopetam counteracted both these features of the psychosis model, ie, restored normal levels of synchronisation and reduced HFOs. Visual hallucinations are common in PD.

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Disease Treatment Licensing Licensing 105

Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes.

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Treatment RNA Hospitals Disease 96

Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

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Biochemical Assays Treatment Bioinformatics Disease 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

Conversations in Drug Development Trends

AUGUST 14, 2024

By: Simran Padam, Medical Director, Medical Affairs Personalized treatment approaches have emerged as pivotal in improving outcomes for hematological cancers. Heterogeneity in Hematological Cancers Due to their disease heterogeneity, hematological cancers are well-suited for personalized approaches.

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Treatment Therapies Disease Protein Expression 78

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

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

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. Cardiac tissue damage and fibrosis caused by 20 Gy XR were significant improved after NG treatment.

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Disease International Regulations Treatment 100

Drug Target Review

OCTOBER 25, 2023

Their findings may result in new treatments for reproductive conditions. He stated that this could inform new treatments for reproductive conditions like recurrent pregnancy loss and preeclampsia. A human foetus contains genetic material from both parents, which makes it partly foreign to the pregnant person’s body.

Treatment 105

Treatment Virus Research Science 105

DrugBank

JULY 20, 2023

of the global population) suffer from rare conditions without effective treatments or cures. Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. At any given time, millions of people ( 3.5–5.9%

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Disease Clinical Trials Trials FDA 75

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. These patterns can foretell which treatments will likely work and which won’t, how likely and quickly the cancer will spread, and its path in the body.

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Disease DNA Virus RNA 98

DrugBank

JULY 8, 2024

Revolutionizing Treatment Through the Skin with Transdermal Drug Delivery Systems The human skin, often perceived as a passive barrier to the external environment, holds potential as a route for drug administration. These fluctuations can lead to unwanted side effects or compromise the effectiveness of the treatment.

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Treatment Drugs Clinical Trials Engineering 79

Broad Institute

DECEMBER 18, 2023

But a new path for bringing treatments to patients is starting to emerge. 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.

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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. This can lead to a new treatment being offered to the patient.

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Doctors Clinical Trials Treatment Therapies 111

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. Carr : Cells that are near one another talk to each other.

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Disease Protein Expression Engineering Treatment 80

Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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Disease Clinical Trials Clinical Pharmacology Trials 59

Broad Institute

OCTOBER 3, 2024

Largest-ever genetic study of epilepsy finds possible therapeutic targets By Ari Navetta October 3, 2024 Breadcrumb Home Largest-ever genetic study of epilepsy finds possible therapeutic targets Scientists have uncovered new genetic links to different types of epilepsy, which could lead to more tailored treatments.

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DNA Doctors Treatment Disease 133

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

Drug Target Review

FEBRUARY 21, 2024

This is a time when several promising treatments – including mRNA vaccines, BiTE therapies and CAR-T cell therapy, are essentially in competition with each other – they all have a common goal of treating the same disease, but they are approaching the objective from different angles.

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Therapies Treatment Vaccine FDA Approval 64

Fierce BioTech

JULY 25, 2023

How combining datasets from multiple sources can inform and improve commercial strategies Hundreds of rare disease treatments have entered the market over the past decade—thanks largely to a combination of government incentives, strong urging from patient advocacy groups and advances in cell and gene therapies.

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Pharma Companies Government Therapies Disease 52

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

Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. But even when this approach succeeds, the candidates often don’t work in other species, and the approach doesn’t provide information about how the AAVs reach their targets.

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Therapies FDA Approval Engineering Disease 137

Drug Target Review

SEPTEMBER 5, 2024

I was part of building up this small biopharma company and learn a lot about rare diseases, regulatory interactions and challenges related to that, challenges related to out licensing drug candidates and having new people joining the company every month as we were growing so fast.

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Pharma Companies Licensing Licensing Disease 111

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.

Disease 59

Disease Clinical Trials Therapies Science 59

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

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

JUNE 21, 2023

Now, researchers at the Broad Institute of MIT and Harvard have found that treatment with continuous hypoxia — low-oxygen conditions comparable to levels at a Mount Everest base camp — restores balance and coordination in a mouse model of Friedreich’s ataxia. The study appeared recently in Human Molecular Genetics. “If

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Treatment Disease Molecular Biology Medical Schools 98

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 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.

Therapies 52

Therapies Treatment Clinical Trials Drugs 52

Chemical Biology and Drug Design

JANUARY 7, 2024

Abstract Atherosclerosis (AS) is one of the main risk factors of ischemic cardiovascular and cerebrovascular diseases. This study aims to clarify the molecular mechanism of BYHWT in treatment of AS through network pharmacology and in vitro experiments. BYHWT in AS treatment was associated with 46 signaling pathways.

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Treatment Regulations Disease Research 100

Chemical Biology and Drug Design

JANUARY 29, 2024

sPLA 2 inhibitors have been developed for the treatment of inflammatory and other conditions such as cardiovascular disease, arteriosclerosis and rheumatoid arthritis. Due to their roles in inflammation, the sPLA 2 enzymes are of much medicinal interest.

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Small Molecule Immune Response Treatment Disease 100

PPD

OCTOBER 12, 2023

These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type. The post Informed Design of Bioanalytical PCR Assay Testing Parameters appeared first on PPD Inc.

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Laboratories Protein Expression Clinical Trials Pharmacokinetics 52

Drug Target Review

OCTOBER 11, 2023

This momentous discovery has the potential to reshape how we approach the diagnosis, treatment, and prevention of this deadly disease. Prostate cancer is a complex and heterogeneous disease, and the quest to tailor treatment to individual patients has never been more crucial.

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Research Disease Treatment DNA 98