Drug Target Review

JULY 3, 2023

There is a significant unmet need for effective treatments to alleviate suffering and prevent premature death across the spectrum of genetic diseases. Unfortunately, the majority of genetic diseases lack a cure, leaving limited therapeutic options available.

Disease 98

Disease Therapies DNA Treatment 98

Drug Target Review

JANUARY 15, 2024

Cancer is a disease driven by variable genetic mutations. Could you provide insights into the driving factors behind exploring novel treatments for cancer types? Additionally, in evaluating the effectiveness of our initial pipeline asset, we sought tumours suitable for intratumoural injection that present surface-accessible lesions.

Treatment 105

Treatment Immune Response Therapies Clinical Trials 105

Drug Target Review

AUGUST 22, 2023

This Q&A explores how pre-clinical research is being used to identify potential therapies for Huntington’s disease, a devastating condition that currently lacks disease- modifying treatments. What makes the potential therapeutic for Huntington’s disease unique compared to other treatments?

Therapies 98

Therapies Disease RNA Treatment 98

Conversations in Drug Development Trends

NOVEMBER 21, 2023

Since the advent of modern medicine, the scientific community has developed more than 3,000 drugs for thousands of diseases. Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. There are so many patients suffering from diseases that have no treatments.

Disease 78

Disease Treatment Clinical Trials Clinical Development 78

Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

Treatment 105

Treatment Packaging Clinical Research Therapies 105

ASPET

JUNE 4, 2024

The goal of the present study was to determine the antiarthritic potential of the minor cannabinoid 8 -THC using the collagen-induced arthritis (CIA) mouse model. The 8 -THC treatment (30 mg/kg) reduced paw swelling and qualitative signs of arthritis.

Treatment 186

Treatment Disease Production 186

Drug Target Review

AUGUST 31, 2023

In the pursuit of a remedy for Alzheimer’s disease, a frontier in medical science is illuminating a glimmer of hope. Chronic inflammation involving microglia has been linked to the disease, as the release of inflammatory molecules triggers an increase in β-amyloid production.

Therapies 105

Therapies Disease Research Treatment 105

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. One diagnostic example that I discussed in my presentation is autism.

Trials 75

Trials Disease Clinical Trials Clinical Research 75

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.

Therapies 114

Therapies Disease Engineering Medical Schools 114

PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The gene presents a complex landscape. million DNA bases.

FDA Approval 98

FDA Approval Treatment FDA DNA 98

ASPET

JANUARY 23, 2024

Patients with sickle cell disease (SCD) display priapism, a prolonged penile erection in the absence of sexual arousal. The current pharmacological treatments for SCD-associated priapism are limited and focused on acute interventions rather than prevention.

Disease 100

Disease Therapies Treatment Research 100

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.

Disease 98

Disease Immune Response Therapies Treatment 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. At present, we test discarded human livers deemed not good enough for transplant.

Therapies 102

Therapies Disease RNA Engineering 102

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 111

Disease Clinical Trials Therapies Science 111

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.

Biochemical Assays 98

Biochemical Assays Treatment Bioinformatics Disease 98

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. Preeclampsia may present at any gestation but is more commonly encountered in the third trimester.

Treatment 105

Treatment Virus Research Science 105

FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

Clinical Development 69

Clinical Development Production Clinical Trials Treatment 69

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. –5.9%

Disease 75

Disease Clinical Trials Trials FDA 75

National Drug & Alcohol Research Centre Blog

FEBRUARY 12, 2024

Key findings: Alcohol is the seventh leading risk factor for mortality, disease, and disability. While lower prevalence drugs, may be well known to present a risk of overdose and death, this is not necessarily recognised for alcohol, which is much more widely used. Only seven people were in a treatment programme at the time they died.

Disease 110

Disease Treatment Drugs Research 110

Drug Target Review

SEPTEMBER 9, 2024

functions to reset the immune system and potentially provide remission for allergic diseases? 1104 acts upstream in the inflammatory response, targeting one of the first key players: antigen-presenting cells (APCs). In allergic diseases, APCs identify and present unknown antigens to the immune system.

Disease 64

Disease Immune Response Clinical Trials Treatment 64

The Pharma Data

MAY 22, 2023

Teva to Present Data Demonstrating UZEDY™ (risperidone) Extended-Release Injectable Suspension Significantly Prolonged Time to Impending Relapse for Adults with Schizophrenia at the American Psychiatric Association’s 2023 Annual Meeting Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. once-monthly dosing) and 2.7

FDA Approval 52

FDA Approval Pharmaceuticals FDA Treatment 52

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.

DNA 133

DNA Doctors Treatment Disease 133

Chemical Biology and Drug Design

AUGUST 7, 2023

Xanthine oxidase (XO) inhibitors are commonly used to treat gout, nephropathy, and renal stone diseases related to hyperuricemia. The present review discusses the many synthetic and natural XO inhibitors that have been developed which are found to have greater potency.

Disease 100

Disease Treatment Research 100

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?

Disease 59

Disease Clinical Trials Clinical Pharmacology Trials 59

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. The three-dimensional structures presented in this review have been published by various research groups and have been deposited in the Protein Data Bank.

Small Molecule 100

Small Molecule Immune Response Treatment Disease 100

Chemical Biology and Drug Design

APRIL 26, 2023

Figure created with BioRender.com Abstract IgA nephropathy (IgAN) is the most common glomerular autoimmune disease and has severe long-term consequences for patients, with 40% of the patients eventually progressing to end-stage renal disease. Despite the severity, no causal treatment is currently available.

Therapies 100

Therapies Disease Treatment Research 100

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?

Clinical Research 72

Clinical Research Disease Research Clinical Trials 72

Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

Science 145

Science Disease FDA Approval Clinical Trials 145

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.

Drug Development 130

Drug Development Clinical Trials Drugs Disease 130

biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Zebrafish Pioneers Advance in Pediatric Cancer Research and Treatment Increasing the survival rate is the primary goal when screening for cancer drugs.

Disease 59

Disease Medical Schools Pharma Companies Clinical Trials 59

Drug Target Review

JANUARY 24, 2024

Scientists from Northwestern Medicine have demonstrated that RNA interference could have a crucial role in the onset and development of Alzheimer’s disease (AD). Alzheimer’s disease Alzheimer’s is the most common cause of dementia. The team found very short sequences present in some of these sRNAs.

RNA 64

RNA Disease DNA Engineering 64

Drug Target Review

JANUARY 15, 2024

Monoclonal antibodies (mAbs) targeting tumour-specific antigens play an essential role in the treatment of many cancers. An important limitation of antibodies against tumour antigens is that these agents direct responses to molecular targets present on the surface of the cell.

Engineering 110

Engineering DNA Virus Immune Response 110

Drug Target Review

APRIL 11, 2024

Targeting pathways involved in SMC phenotypic modulation holds promise for developing novel therapeutic approaches for cardiovascular diseases. Can you explain the methodology used to investigate the genetic variants regulating extracellular matrix (ECM) secretion in SMCs, and predict proteins associated with vascular disease?

Disease 59

Disease Engineering Regulations Clinical Research 59

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.

Therapies 117

Therapies Pharmaceutical Companies Disease Treatment 117

Broad Institute

FEBRUARY 16, 2024

We didn’t expect that frequency of genetic variants would correlate with clinical presentation, but that amazingly seems to be what we’re seeing.” Their analyses demonstrated that the combination of common and rare genetic variants in youth-onset T2D have a greater influence on disease risk than in the adult-onset form.

Medical Schools 133

Medical Schools Hospitals Disease DNA 133

Broad Institute

SEPTEMBER 5, 2024

In recent years, increasingly sensitive mass spectrometers have paved the way for enormous progress: scientists are now able to detect and analyze nearly all proteins expressed in a sample, including ones present only in miniscule amounts. It's a matter of how much of that protein is present relative to its environment.

Disease 80

Disease Protein Expression Engineering Treatment 80

KIF1A

SEPTEMBER 30, 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 In the rare disease space, a common and straightforward question is: How rare is the disease? What’s a pre-print?

Disease 111

Disease FDA Clinical Trials Therapies 111