Broad Institute

SEPTEMBER 28, 2023

By Allessandra DiCorato September 28, 2023 Credit: Strittmatter Laboratory, Yale University Amyloid beta clusters (red) builds up among neurons (green) in a mouse model of Alzheimer’s disease. The scientists identified a suite of changes in cells unique to the early stages of Alzheimer’s, including some not seen before in animal studies.

Disease 144

Disease Medical Schools Research Hospitals 144

Chemical Biology and Drug Design

SEPTEMBER 20, 2023

Ovarian cancer is the most lethal gynecologic cancer with recurrent or resistant disease observed in 70% of patients. The combined treatment successfully enhanced cisplatin cytotoxicity and helped to overcome cisplatin resistance in CR-Caov3 cells.

Treatment 190

Treatment Disease Research 190

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.

Clinical Development 59

Clinical Development Clinical Trials Clinical Pharmacology Trials 59

Covalent Modifiers

JULY 25, 2024

Our results highlight advantages in developing covalent inhibitors, such as USP30-I-1, for targeting USP30 as treatment of disorders with impaired mitophagy.

Biochemical Assays 182

Biochemical Assays Small Molecule Treatment Disease 182

Chemical Biology and Drug Design

MAY 11, 2023

The figure depicts the neuroprotective role of crocin in Parkinson's disease. Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects.

Disease 100

Disease Research Clinical Trials Treatment 100

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

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 Treatment RNA 98

Chemical Biology and Drug Design

APRIL 3, 2024

The present review explores the intricate relationship between autophagy and proteostasis in the pathogenesis of neurodegenerative diseases along with the therapeutic interventions to mitigate the same. The common pathological features of these diseases are associated with the accumulation of misfolded or aggregation of proteins.

Disease 100

Disease Treatment Research 100

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.

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Treatment Packaging Clinical Research Therapies 105

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

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

Chemical Biology and Drug Design

FEBRUARY 25, 2025

A combination of network pharmacology and invitro validation was used to reveal the potential targets and molecular mechanisms of resveratrol for CRS treatment. The potential targets and pathways of RES against CRS disease were predicted by network pharmacology and molecular docking.

Treatment 100

Treatment Disease Drug Research Research 100

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 Treatment Research 105

PPD

MARCH 11, 2025

While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.

Drug Development 52

Drug Development Clinical Trials Disease Trials 52

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 116

Therapies Disease Engineering Medical Schools 116

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

DECEMBER 13, 2024

These types differ in their etiology, natural history, and present distinct challenges in disease management. 3,4 However, inter- and intra-tumoural heterogeneity is widespread in lung cancer, leading to multiple mechanisms through which these tumours can and do develop resistance to treatment.

Therapies 98

Therapies Treatment Clinical Trials Drugs 98

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 98

Disease Clinical Trials Trials FDA 98

Codon

NOVEMBER 3, 2024

In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. million people every year.

Vaccine 102

Vaccine Trials Therapies Treatment 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.

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

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

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 97

FDA Approval Treatment FDA DNA 97

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

FDA Law Blog: Drug Discovery

JANUARY 30, 2025

They also present a more intense focus on the timelines for confirmatory study initiation and completion as well as the feasibility of those studies to verify clinical benefit within those timelines. rare disease settings). Additionally, sponsors should consider factors that may adversely affect accrual, including the approval itself.

FDA 105

FDA Trials Disease Drugs 105

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

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

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

Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations.

Therapies 72

Therapies Clinical Supply Clinical Trials Trials 72

Elrig

MARCH 25, 2025

It will also cover recent advancements in broadening their application, including innovative approaches to ensure their safety and efficacy, the use of targeted delivery to reach disease-relevant tissues as well as success stories in bringing therapeutic oligonucleotides to the clinic.

RNA 59

RNA Bioinformatics Science Pharmacokinetics 59

The Premier Consulting Blog

NOVEMBER 6, 2024

Inhaled combination products (ICP) have emerged as a significant advancement in the treatment of respiratory diseases such as asthma, chronic obstructive pulmonary disease (COPD), and other pulmonary conditions. However, these products present unique challenges from a Chemistry, Manufacturing, and Controls (CMC) standpoint.

Production 52

Production FDA Compliance Disease 52

Drug Patent Watch

DECEMBER 9, 2024

High-Throughput Screening: Modern Technology Meets Natural Products Advanced technologies now allow researchers to rapidly test thousands of natural compounds against specific disease targets. Artemisinin: Nobel Prize-Winning Malaria Treatment Artemisinin, derived from the sweet wormwood plant, has become a crucial treatment for malaria.

Drugs 97

Drugs Drug Development Pharmaceuticals Production 97

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.

Clinical Trials 52

Clinical Trials Disease Trials Therapies 52

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 Treatment FDA 52

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

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

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