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Genome engineering company launches with $43 million investments

02 December 2013

By Dr Rachel Brown

Appeared in BioNews 733

A biotech start-up that will develop genome-editing therapies has received US $43 million investment from three venture capital firms.

Editas Medicine, 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/Cas9.

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 piece in'.

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.

However, there 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.

Fierce Biotech | 25 November 2013
Boston Globe | 25 November 2013
Editas Medicine (press release) | 25 November 2013
MIT Technology Review | 27 November 2013


08 February 2016 - by Cait McDonagh 
A biotechnology company co-founded by two leading scientists involved in developing the genome-editing technology, CRISPR/Cas9, has gone public, raising over $94 million in its initial share offering...
23 March 2015 - by Meghna Kataria 
Scientists have called for a worldwide moratorium on the use of the gene-editing technique CRISPR/Cas9 in human embryos and germ cells...

11 November 2013 - by Dr Naqash Raja 
The Children's Hospital of Philadelphia has invested US $50 million in a biotech start-up in an effort to commercialise gene therapy...
11 November 2013 - by Dr Anna Cauldwell 
A molecular technique that enables any part of the human genome to be altered with extreme precision has been hailed by scientists as a breakthrough in genetics...
05 November 2012 - by Dr Lucy Spain 
The European Medicines Agency (EMA) has approved Glybera, a gene therapy to treat a rare genetic condition, lipoprotein lipase deficiency (LPLD), for sale across Europe. Glybera is the first gene therapy to be approved for the Western market and offers the first therapeutic treatment for people with LPLD....
08 April 2005 - by BioNews 
A gene therapy technique that repairs faulty genes by 'editing' their DNA could lead to new treatments for inherited blood disorders and HIV, say US researchers. The approach could help address safety concerns over current gene therapy methods, many of which rely on specially adapted viruses to deliver working genes...

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