start-up that will develop genome-editing therapies has received US $43 million investment from three venture capital firms.
based in Massachusetts, plans to develop a new class of drugs that can
precisely correct disease-causing mutations in DNA, using a technology called
CRISPR can be used to edit single
base pairs and larger stretches of DNA, leading researchers to believe its versatility
can be used to target any mutation.
'The way to think about it
is molecular surgery… you can go in and make
changes to the DNA', Dr J Keith Joung, co-founder of Editas Medicine, told the Boston Globe. 'If there's a
defect, you can fix it; if there's a missing piece, you can put the missing
CRISPR/Cas9 is a gene-editing system initially
identified in bacteria, where it protects against viruses (see BioNews 730). A guide RNA
molecule brings the Cas9 protein to a specific stretch of DNA, which it then
cuts in a precise way. Unwanted DNA can
therefore be 'deleted' and replaced with versions of DNA to correct the faulty gene.
Dr Douglas Cole is a general partner at Flagship Ventures, one of the venture
capital firms alongside Polaris Partners and Third Rock Ventures that have invested
in Editas. He said: 'The gene editing approaches on which Editas is based
represent some of the most exciting and promising scientific breakthroughs in
recent years, making it possible, for the first time, to correct the genomic
defects responsible for a broad range of diseases'.
Editas Medicine has
not announced the conditions they will be aiming to treat. But their focus will
be on diseases that are proving especially hard to treat with traditional gene
therapy or gene knock-down efforts that do not actually remove the faulty gene
from cells. These traditional efforts have associated safety concerns and cannot
correct diseases that arise from faulty genes producing toxic proteins; such is
the case in Huntington's Disease.
Therapies based on CRISPR could overcome
these problems by precisely and accurately replacing the
faulty gene in its normal chromosomal region.
are technological and ethical barriers still to overcome, in addition to the
normal clinical trials, so it is likely to be years before a precise
genome-editing therapy will be available on the market.