Drug Discovery Featured Articles & Applications
Beyond Nuclear Genetics: Let’s Think Differently About CNS Disease Targets
Nuclear genes with mutations remain the primary focus for selecting biological drug targets, but we have yet to see a drug come to market from a genetically validated target that can arrest or reverse diseases like Parkinson’s. We need to expand our research to explore the essential drivers, genetic or otherwise, that switch our cells from order to disorder or from function to dysfunction.
The Promise Of Gene Silencing To Treat Not-So-Rare Diseases
In this last article in the series on siRNA, I examine its potential for treating not-so-rare and common diseases. Unlike rare diseases, which are often caused by pathological genetic mutations, common diseases may be associated with genetic variants that are not pathological. I look at cardiovascular disease and iron flux redux in particular.
Attributable Data Integrity in Modern Biopharma Using ALCOA Principles
This article is the first in a five-part series providing contemporary, practical, and useful examples of data integrity within each of the five traditional principles of ALCOA. This article centers on A: Attributable.
ADCs Are The Future Of Precision Chemotherapeutics
Antibody-drug conjugates (ADCs) allow potent chemotherapeutics to be targeted to their intended site of action and offer a wider therapeutic window compared to traditional systemic chemotherapy. This article delves into how ADCs provide targeted treatments and shares emerging trends in ADC development.
The Potential Of siRNA To Treat Rare Diseases
siRNA is a precision, targeted medicine with the potential to treat many diseases that currently lack effective therapeutic options. In this second article of my series on the subject, I take a closer look at siRNA’s potential for treating rare diseases.
4 Learnings From A Career Of Drug Discovery
Drug discovery is a challenging, high-risk, high-reward endeavor. I’ve personally hit plenty of potholes on the drug discovery journey. That is just the nature of this enterprise. After being in the field for over 20 years, I have found that some key tenets emerge that can help increase one’s chances for success.
FDA Seeks Comment On Conducting Remote Regulatory Assessments
The FDA believes the use of both voluntary and mandatory remote regulatory assessments (RRAs) for all types of FDA-regulated products is in the interest of public health. The document describes the difference between voluntary and mandatory RRAs and shares information about the RRA report. Public comment ends on Sept. 30, 2022.
The Promising Emergence Of Venom-Derived Compounds
There are 100,000+ venomous animal species, each producing a chemical cocktail of up to 1,000 different components, including peptides, proteins, and non-peptide small molecules. Many in drug development view venom-derived compounds as simple peptides that will be easily digested, limiting their therapeutic potential. This article dispels that myth.
The Methylation Analysis Landscape In 2022
Within liquid biopsy, methylation has become a key area of interest due to its role in altering gene expression and its ability to provide information on tissue-of-origin for ctDNA. The explosion of methylation as a biomarker in oncology stems from its ability to serve as an indication for the presence of cancer early in development. This article also shares key market insights about the diagnostic companies currently working in this area.
The Promise Of Gene Silencing: A Primer On siRNA
Small interfering RNAs (siRNAs) are short sequences of RNA that interfere with gene expression, reducing or eliminating the expression of a disease-related protein by targeting its corresponding mRNA. siRNAs could treat a wide array of diseases. How does it differ from other therapeutic modalities such as gene therapy and gene editing?