This week in drug discovery (1-5 July)
News round-up for 1-5 July by DDW Senior Digital Content Editor Diana Spencer.
The last few days have seen some interesting developments related to gene editing, including the discovery of a new mechanism for genetic programming and evidence in favour of redosing CRISPR-based therapies, as well as significant investment and new indications for gene therapies.
The top stories:
Gene therapy halts progression of rare genetic condition
The findings from a single-patient gene therapy trial in Canada show promise in halting the progression of spastic paraplegia type 50 (SPG50).
Evidence supports redosing with an in vivo CRISPR-based therapy
Intellia Therapeutics has shared the first-ever clinical data demonstrating the potential for redosing with an investigational in vivo CRISPR-based therapy.
Beyond CRISPR: New mechanism for genetic programming discovered
A team of researchers have discovered the bridge recombinase mechanism, a precise and powerful tool to recombine and rearrange DNA in a programmable way.
Gene therapy trial starts in early-onset dementia
AviadoBio has launched its Phase I/II ASPIRE-FTD clinical trial at the Ohio State University in the US.
Beacon Therapeutics raises $170 million in a Series B financing
Life science investors Syncona have committed $42.5 million to Beacon Therapeutics’ $170 million Series B financing round.