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.

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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.

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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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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.

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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.

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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.

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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.

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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%

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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.

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FDA Law Blog: Biosimilars

APRIL 13, 2025

LYTGOBI was granted accelerated approval for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or rearrangements. DCR is defined as the sum of complete response (CR), partial response (PR) and stable disease (SD).

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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.

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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.

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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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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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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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

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The Pharma Data

FEBRUARY 24, 2022

Allergan, an AbbVie (NYSE: ABBV) company, today announced new data to be presented from Allergan’s leading portfolio of eye care treatments at the 2022 American Glaucoma Society (AGS) Annual Meeting being held in Nashville, TN and virtually from March 3-6. Robinson, M.D.,

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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.

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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.

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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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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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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.

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Drug Target Review

APRIL 14, 2025

With many healthcare systems prioritising cost-effectiveness, there is limited demand for novel treatments, even though they are crucial to combatting antibiotic resistance. This model not only promises a broader market but also presents tangible benefits for patients. coli and yeast, but spirulina presents a new set of considerations.

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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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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.

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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.

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National Institute on Drug Abuse: Nora's Blog

MARCH 6, 2025

Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&

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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?

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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.

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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.

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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.

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