Dr Michael Krohn and Dr Dirk Sombroek of Brain Biotech introduce a specially developed nuclease , which is the first of a new class of genetics-based cancer therapies tool
An alternative CRISPR-Cas nuclease for biotech & pharma
Dr Michael Krohn and Dr Dirk Sombroek of Brain Biotech introduce a specially developed nuclease , which is the first of a new class of genetics-based cancer therapies tool
Figure 1 - Workflow that led to identification of more than 2,000 novel Class 2 CRISPR systems
The discovery of gene technology ’ s most precise tool CRISPR ( Clustered Regularly Short Interspersed Palindromic Repeats ) won a Nobel Prize in 2020 . CRISPR is a gene-editing technology originally derived from prokaryotes . What makes it so unique and fascinating is the ease and versatility of its application in life sciences , allowing for the targeting or editing of almost any nucleotide sequence in a given living organism .
This still sounds crazy to molecular biologists , since they dreamed of it for decades . Today modern sequencing and CRISPR methods can be used to read , re-write and control changes in the code of life , making it probably as meaningful as the historic Gutenberg event of book printing .
In this technology , CRISPRencoded enzymes known as CRISPR-associated ( Cas ) nucleases are programmed with a small ribonucleotide stretch - a guide RNA ( gRNA ). This allows the complex of nuclease and gRNA to scan , identify , bind and cut at the site of the gRNA ’ s pendant sequence in the genome of a cell .
Today CRISPR is pervasive in the life sciences and is used in diagnostics and agriculture or for more sustainable microbial production in industrial applications . It is also used ex vivo to design or correct patient cells for re-introduction and for curative clinical goals , and it has entered clinical trials and therapies .
However , one facet of CRISPR ’ s inherent potential is still missing : applying it as a smart , programmable anti-cancer cytotoxic drug . The vision is to rely on a patient ’ s genomic make up to selectively destroy cancerous cells identified and targeted by its malicious genomic cancer sequence code . Here we describe such a novel mode of action ( MoA ) that we believe has the potential to become a programmable anti-cancer drug for oncotherapies .
Diversity is key
The IP landscape for CRISPR applications has been described at best as complex , at worst ‘ a nightmare ’. The major licensors of CRISPR technologies around the Cas9 and Cpf1 ( Cas12a ) nucleases , BROAD / MIT and ERS Genomics / Berkeley , remain in a multi-year legal battle .
As a result , interested parties often either have to license CRISPR from both institutions or at least face a painful and time-consuming process to identify the right licensor or even the right patents for their business . In addition , the licensing fees are prohibitive for many applications . This was our main motivation
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