Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Cancer Immunotherapy

Posts tagged ‘spliceosome’

Molecular biology was a hot new topic back in its infancy in the late 1980’s just as I was finishing my doctorate – cue moment of realising you’ve missed a big wave before it really even started!

Springtime in DC

These days scientists now delve in the realm of deeper molecular biology and go much further than mere genes… it’s all about transcription factors, super enhancers, chromatin complexes, bromodomains, and even chromodomains. In the past, many of these drivers were often considered ‘undruggable’ – think MYC or RAS, for example.

The world of molecular biology is rapidly changing as researchers understand pathways and processes associated with carcinogenesis better, thereby enabling new approaches to evolve and with it, valid new targets for therapeutic intervention.

This field is always one of my favourite ones to cover at AACR, where we not only learn about exciting new research from investigators, but also up and coming young biotech companies that are doing good work who deserve to be highlighted.

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ASH16 in San Diego

Today we resume our coverage from the recent American Society of Hematology (ASH16) annual meeting with a look at some fascinating and highly compelling science that was presented in an obscure and hard to find tiny hall in San Diego.

This story is also about how a small biotech company that many casual observers may not even be aware of, is taking advantage of advances recent research to grab a clinical lead in a very specialised field in oncology that may yield a novel approach worthy of taking notice of..

Genomics is increasingly becoming a core element of cancer research. Think of it as the alphabet soup of molecular biology concerned with the structure, function, evolution, and mapping of genomes.

Once we understand and identify the genomic landscape in health and diseases such as cancer, it allows numerous platforms to evolve whereby those unique differences can be identified (as driver vs. passenger mutations, for example), explored in depth, and later key ones targeted with therapeutics. Inevitably, there are many ways to do this.

Much of the focus in genomics has been on DNA, but what about RNA?

RNA is important because a mistake – even a single nucleotide – can be devastating to the cell, and a reliable, repeatable method of RNA processing is necessary to ensure cell survival. Mis-splicing can thus lead to the development of new point mutations and genomic instability deep in the cell nucleus, potentially causing the evolution of certain cancers.

Paradoxically, these aberrations also offer novel therapeutic targets – but are they druggable?

What we are exploring here is a completely different approach, both in terms of how a fledgling company is funded and also the type of research that is conducted.

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