Author: Frank Staedtler

What are genome editing and CRISPR-Cas9?

Genome editing (also called gene editing) is a set of technologies that enable scientists to change the genetic code in the living cells of an organism. They make it possible to make specific changes to a particular location in the genome in a controlled way. One of the approaches is known as CRISPR-Cas9. The “Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9”, or CRISPR-Cas9 for short, was discovered by a team of CRISPR pioneers led by biochemists Emmanuelle Charpentier...

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First in vivo CRISPR medicine administered to patients in clinical trial

On 4 March 2020, the collaborating companies Allergan plc and Editas Medicine, Inc. announced the dosing of the first patient with the CRISPR-based medicine AGN-151587 (EDIT-101) in a phase 1/2 clinical trial (BRILLIANCE trial).[1]   Treatment for Leber Congenital Amaurosis 10 AGN-151587 (EDIT-101) is an experimental CRISPR-based medicine currently under development. The medicine is delivered via sub-retinal injection under development for the treatment of Leber congenital amaurosis...

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