Gene therapy

By Dr Phil Jones The mission at The University of Texas MD Anderson Cancer Center is to eradicate cancer, which remains a major cause of mortality worldwide. It is the second leading cause of death in the United States and globally, claiming more lives than malaria, HIV/AIDs and tuberculosis combined (or ~1 of every 7 […]

Genetically-Engineered Models: The Value of Precious GEMs By Dr Megan M. MacBride Genetically-engineered animal models (GEMs) are critical tools for drug discovery and development, but intellectual property (IP) concerns can complicate access. As pharma and biotech increasingly outsource R&D activities to universities, careful scrutiny is required to ensure adherence to IP restrictions on research tools […]

Current Trends in RNA Therapeutics Development By Dr Xiaoqiu Wu and Dr Andrew P. Turnbull Cellular RNAs play crucial roles during disease progression and represent a diverse and largely untapped class of biomolecules that can be exploited for drug development. RNA species include messenger RNAs (mRNAs) that are translated into proteins, long non-coding RNAs including […]

Integrated Platform Drug Discovery and Development Companies – Part 1 By Dr Urban A. Kiernan and Dr Stephen Naylor Technology was lauded as the saviour of the drug discovery and development (DDD) process during the late 1990s. A myriad of independent companies developed and offered ‘innovative’ tools, technologies and platforms (TTPs) to pharmaceutical companies in […]

Cell Therapy – Preserving Cell Integrity and Viability To Drive Discovery Mary Kay Bates Cell therapy is poised to play a pivotal role in the development of precision and personalised medicines that will transform healthcare for millions of patients worldwide. Involving the direct use of whole cells or cellular material in patients, cell therapy is […]

Analytical Biophysical Tools To Accelerate Discovery of Therapeutic Antibodies By Dr Yasmina Noubia Abdiche The innate ability of antibodies to bind their targets with high affinity and exquisite specificity is leveraged in the discovery of therapeutic antibodies to treat a broad range of diseases including cancer, autoimmune disease and heart disease. In addition, antibodies are […]

Disruptive Approaches To Accelerate Drug Discovery and Development (Part 2) By Dr Anton Fliri, et al. Part 2: Emergent intelligence, a new paradigm for drug discovery. (You can read part 1 of disruptive approaches to accelerate drug discovery and development linked here.) The staggering clinical trial failure rate of experimental drugs indicates that despite huge […]

Astrazeneca R&D – Improving Drug Development Productivity With Better Predictivity By Dr Menelas Pangalos & Steve Rees et al. Changing demands in global healthcare over the past 15 years have led to greater complexity and spiralling costs in drug development. The average price tag of taking a new drug from discovery to completion of Phase […]

Precision Medicine Drugs – Pleonasm or Reality? Dr Urban Kiernan and Dr Stephen Naylor The complexity and uniqueness of human metabolism and physiology in the past decade has been highlighted and dissected. Arguments have been made to suggest that our lack of understanding about human individuality has led to the limited efficacy and safety of […]

How High-Throughput CRISPR is set to Deliver Knockout Performance in Drug Discovery By Dr Paul Avery and Dr Richard Massey CRISPR-Cas9 has rapidly transformed our ability to perform targeted gene editing. While the technique has received much interest for its potential in the field of gene therapy, advances in its high-throughput use mean it can […]