Making Sense of Antisense Oligonucleotide-Based Therapies in Muscular Dystrophies

Good news for the Duchenne Muscular Dystrophy (DMD) community. On June 8th, BioMarin announced the filing of a Marketing Authorization Application to the European Medicines Agency for Drisapersen, an antisense-mediated exon 51-skipping compound able to target the most prevalent genetic mutations responsible for the lack of production of functional dystrophin. The European filing follows the submission of a New Drug Application to the US FDA for Drisapersen back in April 2015.

Quantifying in vivo Biodistribution and Kinetics of Your Biologic or Nanomaterial

With the ability to provide non-invasive, three dimensional, longitudinal, and quantitative data—PET, SPECT, and fluorescence imaging (FMT) enable translational imaging of:

Are you ready for ISO 15189:2012 to make a difference in your bottom line?

Is it possible to get more efficiency in your conventional and specialty tests while maintaining ongoing quality? ISO 15189:2012 accreditation answers this question by delivering a comprehensive approach to quality management in medical, central and referral laboratories. Not only can these standards ensure quality, but they can reduce your risk of costly delays and ultimately save money in your trials.

Get Glowing Results From Your Cell Lines – With Luciferase

Firefly (P. pyralis) luciferase is an enzyme that produces bioluminescence in the presence of D-luciferin. The DNA for the luciferase 2 (luc2) gene, along with a puromycin resistance gene, is incorporated into the genome of the cells using a lentiviral transduction system.

Image Analysis

Analysis of biomedical imaging data is no less a task than the image acquisition itself. Due to the complex nature of such data and variability across different subjects, information extraction has largely remained a time-consuming process. Vendors of imaging instruments and specialty software offer a variety of tools; however, a user still needs to work through a series of complicated and time sapping procedures for image analysis involving extensive manual operations. It is not just tedious, it is prone to error. The evolvement of image analysis has seriously lagged behind the rapid advancement of imaging technology.

Choosing the Best Pathway for CDx Development and Commercialization

Drug developers are increasingly incorporating a CDx development (companion diagnostic) strategy into their programs to deliver safer, more effective and appropriate therapies to the right patients. While the end result can be significantly beneficial for patients, and development partners, the path from analytical and clinical validation to demonstrating clinical utility and obtaining regulatory approval is often challenging. It requires a comprehensive understanding of clinical trial design, regulatory submission strategies both for the therapy and the diagnostic assay, and also a line of sight for successful commercialization.

Evaluating New Antiviral and Antibiotic Drug Candidates

The development of anti-infective treatments to combat illnesses caused by viral and microbial agents has witnessed many unique advances, most notably in the fights against bacterial infections, hepatitis and HIV. The momentum in this exciting area continues with new tools and methods to better support the development journey. 

Addressing the Drug Development Gap in China

Each year, new medicines are launched globally but few are available to Chinese patients,  in comparison to those in the US or EU. Drug developers are working to reduce this gap and expand the reach of new molecular entities (NMEs) so that they can improve healthcare to patients in need. Confronting this issue requires navigating the evolving regulatory environment in China and capitalizing on parallel development opportunities in the global market.

PET Quantification of Receptor Occupancy

The use of PET imaging to detect and quantify receptor occupancy (RO) has become a translational standard in CNS discovery and clinical trials. For example, 18F radiolabeling of targeted small molecules enables a PET tracer-based PD biomarker. The tracer can be used for dynamic, non-invasive quantification of percentage RO.

The Evolution of Open-Label Extensions and Rare Disease Studies

When running early development studies, sponsors must consider whether or not to provide open-label, long-term treatment at the end of their randomized, placebo-controlled trial. In the past, sponsors have been hesitant to offer open-label extensions. But with our industry’s increasing focus on patient-centric care, sponsors are now weighing their options to incorporate more patient input into early development, especially when working with promising novel treatments in rare disease studies.