article thumbnail

The rising impact of biomarkers in early clinical development

Drug Target Review

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.

article thumbnail

Medicinal Chemistry In The Age Of Artificial Intelligence

LifeSciVC

In 2001, I was at Millennium Pharmaceuticals, one of the major players in the nascent field of genomics drug discovery, and that time has strong parallels to the changing state of drug development today. In fact, the Millennium of 2001 (pre-Takeda acquisition) had one drug approved which was Velcade (bortezomib).

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Fishing for Answers in Human Disease

NIH Director's Blog: Drug Discovery

Caption: Researcher Zhaoxia Sun, at Yale, uses the zebrafish to study Polycystic Kidney Disease, which affects more than 600,000 Americans. This is a vital resource for understanding human health and disease. 2001 Dec 1;15(23):3217-29. How does the genetic blueprint of a fish help us or accelerate drug discovery?

Disease 52
article thumbnail

Why Sarepta’s most recent failure in DMD was entirely predictable

DrugBaron

Here is a disease that is caused by the lack of functional dystrophin in the muscles of these boys. The underlying assumption of most gene therapy procedures, irrespective of the disease being targeted, is that restoration of a wild-type DNA sequence will alone be sufficient to normalise the phenotype of the individual.

DNA 100
article thumbnail

Gianluca Pirozzi, MD, PhD, Celebrated for Dedication to the Field of Immunology and Rare Disease Research

The Pharma Data

Pirozzi began his career as a Medical Advisor for Servier in Italy in 2001. Dr Pirozzi subsequently was inspired to pursue a career in rare diseases after his daughter Valentina was diagnosed with a rare chromosomal syndrome, called Smith-Magenis Syndrome. positions he assumed in 2020. positions he assumed in 2020.

Disease 52
article thumbnail

BIOGEN ANNOUNCES TWO NEW NOMINEES FOR ELECTION TO BOARD OF DIRECTORS

The Pharma Data

Freire is President and Executive Director and member of the board of directors of the Foundation for the National Institutes of Health, a nonprofit organization committed to accelerating biomedical research and advancing breakthrough discoveries to fight critical diseases. About Maria C. a drug discovery and development company.

article thumbnail

Targeted protein degradation: turning undruggable targets into druggable targets

Drug Target Review

TPD is a rapidly evolving therapeutic modality to degrade a disease-causing proteins, thereby eliminating their function specifically. Undruggable versus druggable targets As elucidated by Shitaka, it is estimated that about 80 percent of the proteins involved in disease are undruggable targets. Expert Opinion on Drug Discovery.