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

Science Daily: Pharmacology News

OCTOBER 24, 2024

A strain of the bacterium harbored in the children's gut microbial communities possessed a previously unknown gene capable of producing and metabolizing key molecules involved in regulating many important functions ranging from appetite, immune responses, neuronal function, and the ability of pathogenic bacteria to produce disease.

Immune Response 259

Immune Response Regulations Disease Research 259

Science Daily: Pharmacology News

NOVEMBER 29, 2023

A study identified the epigenetic regulator MSL2 an 'anti-monoallelic' factor that maintains biallelic gene dosage. This discovery not only reveals a communication system between parental alleles but also points to potential therapeutic strategies for diseases associated with haploinsufficient genes.

Regulations 226

Regulations Disease Research 226

Chemical Biology and Drug Design

JANUARY 23, 2024

Abstract Nonalcoholic fatty liver disease (NAFLD) is a chronic liver disease associated with lipid deposition in liver cells and/or subsequent inflammation, excluding other known causes. Among the many factors that shape these transitions, uncoupling protein 2 (UCP2) may be involved in every stage of the disease.

Production 100

Production Disease Regulations 100

Drug Target Review

DECEMBER 20, 2024

Their work focuses on creating ‘digital twin generators’ – AI-driven models that predict how a patients disease may progress over time. AI-powered digital twins A key component of Unlearns work is its focus on digital twin technology, which uses AI to create personalised models of disease progression for individual patients.

Clinical Trials 128

Clinical Trials Trials Pharmaceuticals Drug Development 128

BioPharma Drive: Drug Pricing

AUGUST 15, 2024

The approval of Niktimvo in graft-versus-host disease hands Incyte another immune system-regulating medicine that analysts see as valuable, especially if use is broadened through additional clearances.

Regulations 176

Regulations Disease Drugs 176

Covalent Modifiers

MAY 9, 2024

4c00220 Multiple sclerosis (MS) is a chronic disease with an underlying pathology characterized by inflammation-driven neuronal loss, axonal injury, and demyelination. Hopkins Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.4c00220

Treatment 208

Treatment Clinical Development Disease Regulations 208

Covalent Modifiers

JUNE 29, 2024

Phosphorylation of their C-terminal RS domains by SR protein kinases (SRPKs) regulates their localization and diverse cellular activities. Dysregulation of phosphorylation has been implicated in many human diseases, including cancers.

Regulations 200

Regulations Disease 200

ASPET

JUNE 6, 2024

MAP4K4 has been involved in regulating multiple biological processes and a plethora of pathologies, including systemic inflammation, cardiovascular diseases, cancers, metabolic and hepatic diseases. Recently, multiple reports have indicated the upregulation of MAP4K4 expression and signaling in hyperglycemia and liver diseases.

Disease 100

Disease Regulations 100

ASPET

APRIL 2, 2024

Bromine domain protein 2 (BRD2), a member of the Bromodomain and extraterminal domain (BET) protein family, is a crucial epigenetic regulator with significant function in various diseases and cellular processes.

Disease 100

Disease Regulations Virus Research 100

BioPharma Drive: Drug Pricing

JUNE 28, 2024

European drug regulators voted for a third time not to renew the approval of Translarna. Elsewhere, Sanofi invested in a brain disease biotech and Esperion sold royalties to help pay off a loan.

Biosimilars 167

Biosimilars Regulations Drugs Disease 167

Chemical Biology and Drug Design

SEPTEMBER 30, 2023

Abstract Epigenetic regulation of genes through posttranslational regulation of proteins is a well-explored approach for disease treatment, particularly in cancer chemotherapy. Structural classification and regulatory functions of histone deacetylases and their inhibitors.

Regulations 100

Regulations Treatment FDA Disease 100

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 69

Clinical Development Clinical Trials Clinical Pharmacology Trials 69

Science Daily: Pharmacology News

AUGUST 28, 2023

The stakes of their findings are high: a ruptured nucleus can lead to elevated DNA damage that is characteristic of many diseases, including cancer.

DNA 189

DNA Regulations Disease Research 189

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

Advarra

JULY 26, 2022

Importantly, the 2018 Farm Bill preserved FDA authority to regulate products with cannabis or cannabis-derived compounds under the Federal Food, Drug, and Cosmetic (FD&C) Act and Section 351 of the Public Health Service Act. The post CBD Research: A Dive into the Regulations of Cannabis Research appeared first on Advarra.

Regulations 97

Regulations Research FDA FDA Approval 97

SCIENMAG: Medicine & Health

AUGUST 16, 2023

Medications to treat various chronic diseases may hinder the body’s ability to lose heat and regulate its core temperature to optimal levels.

Regulations 60

Regulations Disease Treatment 60

Broad Institute

FEBRUARY 7, 2024

However, researchers lack efficient ways to explore how these genetic variants are molecularly connected to cardiovascular disease, limiting efforts to develop therapeutics. The team found that a key biological mechanism involved in a rare vascular disease may influence CAD risk.

Disease 105

Disease Hospitals Medical Schools Therapies 105

Drug Target Review

MARCH 12, 2024

The study’s findings offer new insights into the biochemical language microbes use to influence distant organ systems, which could revolutionise the way researchers approach disease. Also, they help to explain how microbes in the gut can influence distant organ systems. This study was published in Cell Reports. Gut Microbes.

Disease 111

Disease Biochemical Pharmacology FDA Approval Pharmacy 111

Chemical Biology and Drug Design

DECEMBER 13, 2024

ABSTRACT Severe fever with thrombocytopenia syndrome (SFTS) is a severe emerging infectious disease caused by Dabie bandavirus (DBV). Tectorigenin could reduce the expression levels of inflammatory cytokines in both DBV-infected THP-1 cells and plasma samples of IFNAR / mice infected with DBV.

Regulations 100

Regulations Disease Research 100

Drug Target Review

JULY 13, 2023

In this article, we will delve into the world of condensate biology and explore the groundbreaking research projects undertaken to discover the potential they hold for diseases such as amyotrophic lateral sclerosis ( ALS ) and colorectal cancer. Klein notes the importance of Dewpoint’s disease-agnostic approach.

Disease 98

Disease Small Molecule Cell Biology Drugs 98

Drug Target Review

SEPTEMBER 8, 2023

This receptor, referred to as retinoic acid receptor alpha (RARα), is recognised for its regulation of gene expression within the nucleus. Remarkably, these findings propose that the presence of a cytoplasmic version of RARα is indispensable for T cells to initiate their defence against diseases.

Regulations 98

Regulations Research DNA Immune Response 98

Antidote

JUNE 16, 2023

They also are responsible for producing hormones and regulating various salts and minerals that are necessary for bodily functions. Despite being relatively small organs, the kidneys are extremely important to the functioning of the human body. Their main function is to filter the blood, eliminating wastes, toxins, and excess fluids.

Disease 96

Disease Regulations 96

Chemical Biology and Drug Design

FEBRUARY 15, 2024

Abstract Dry eye (DE) is a multifactorial ocular surface disease characterised by an imbalance in tear homeostasis. However, the DE disease pathogenesis remains unclear, thereby affecting its clinical treatment. This study aimed to explore the mechanism through which prostaglandin E2 (PGE2) affects DE inflammation by regulating Th17.

Disease 100

Disease Protein Expression Regulations Treatment 100

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

Chemical Biology and Drug Design

JULY 17, 2023

The overall findings suggested that quercetin might regulate AKT in the treatment of NAFLD-related liver cancer. Abstract Nonalcoholic fatty liver disease (NAFLD) is the most common liver disease in the world. There were 455 intersection targets of NAFLD-related liver cancer, and 65 drug-disease common targets.

Disease 100

Disease Treatment Regulations Drugs 100

Covalent Modifiers

DECEMBER 11, 2023

Mutations that impair nuclear protein association with chromatin are implicated in numerous diseases. Using covalent scout fragments, KB02 and KB05, we identified ligandable cysteines on proteins involved in spindle assembly, DNA repair, and transcriptional regulation, such as Cys101 of histone acetyltransferase 1 (HAT1).

Small Molecule 162

Small Molecule DNA Regulations Disease 162

Drugs.com

FEBRUARY 19, 2024

coli bacteria has sickened 10 people in four states, hospitalizing four, federal regulators warn. Centers for Disease. MONDAY, Feb. 19, 2024 -- Raw milk cheese tainted with E. The cases have been tied to Raw Farm brand raw cheddar cheese, the U.S.

Hospitals 147

Hospitals Regulations Disease 147

ASPET

SEPTEMBER 6, 2023

However, aberrant TLR7/8 activation by endogenous ligands has been implicated in the pathogenesis of autoimmune diseases including systemic lupus erythematosus. TLR activation and Type-I interferon (IFN) were shown recently to impart resistance to glucocorticoids (GC), which are part of the standard of care for multiple autoimmune diseases.

Disease 100

Disease RNA Treatment Regulations 100

Covalent Modifiers

JANUARY 11, 2024

These two discrete activities are tightly regulated by both environmental stimuli and redox reactions. Ubiquitously expressed in humans, TG2 has been implicated in numerous disease pathologies that require extensive investigation.

Regulations 173

Regulations Disease 173

Chemical Biology and Drug Design

MAY 11, 2023

Abstract Ginsenoside Rg5 has been implicated in a variety of diseases. Western blot was used to detect the expression of apoptosis-related proteins Bcl-2, Bax and Cleaved caspase 3 in the hypoxia group, hypoxia+Rg5 group, and the hypoxia +Rg5 + MK2206 group. * 05, ** p  < .01, 01, p  < .001.

Regulations 100

Regulations Disease Research 100

FDA Law Blog: Drug Discovery

JANUARY 30, 2025

This condition was included in the final rule for the original regulations, 21 CFR 314 Subpart H (see the December 11, 1992 Federal Register, 57 FR 58958 ) as well as when the pathway was codified in the Federal Food, Drug, and Cosmetic Act by FDAMA (21 USC 356(c)) in 1997. rare disease settings).

FDA 105

FDA Trials Disease Drugs 105

Drug Target Review

MAY 18, 2023

In sickle cell disease, however, this function is impaired, leading to a range of potential health consequences. The irregularly shaped and sticky red blood cells produced by the disease can obstruct small blood vessels, causing recurring painful attacks. Hydroxyurea, the first sickle cell drug approved by the U.S.

Disease 80

Disease Clinical Trials Drugs Trials 80

SCIENMAG: Medicine & Health

JANUARY 9, 2024

In addition, they also protect against cardiovascular diseases. Brown fat cells convert energy into heat – a key to eliminating unwanted fat deposits.

Regulations 72

Regulations Hospitals Disease Research 72

Drug Target Review

DECEMBER 13, 2023

Combinatorial analytics approaches identify combinations of features that together are associated with the disease phenotype in patient sub-groups, capturing the non-linear effects of interactions between multiple genes. The combinatorial approach is considerably more sensitive than GWAS and requires much smaller patient populations.

Bioinformatics 145

Bioinformatics Disease Regulations Immune Response 145

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.

Disease 100

Disease International Regulations Treatment 100

Broad Institute

MARCH 12, 2025

By Leah Eisenstadt March 12, 2025 Credit: Broad Communications Scientists in the Spatial Technology Platform at the Broad develop, use, and share spatial -omics approaches, such as Perturb-FISH, that can reveal the roles of genes in various cell types and the impact of intercellular interactions on health and disease.

RNA 99

RNA Science Research Disease 99