Article
Latest Developments in CRISPR and Genome Editing Clinical Trials
IQVIA Pipeline Link / Trial Link
Mar 21, 2025

Genome editing technologies have brought major breakthroughs in science and medicine leading to the invention of clustered regularly interspaced short palindromic repeats (CRISPR) as a gene editing tool to edit DNA. CRISPRs are repeating DNA sequences in the genome of prokaryotes, including bacteria, and were first discovered in E. coli in 1987. In the early 2000s, the role of the CRISPR and CRISPR-associated sequence (CAS) protein systems was identified as an adaptive immune response in bacteria against invading phage DNA (double-stranded DNA viruses). Later in 2012, the CRISPR-CAS9 system was adopted as a gene-editing tool for selectively editing the human genome. A Nobel Prize was awarded to the researchers who discovered the CRISPR-CAS9 genetic scissors in 2020. On 16 November 2023, the UK MHRA's approval of Vertex Pharmaceuticals and CRISPR Therapeutics' exagamglogene autotemcel (CASGEVY) marked the first time that marketing authorization has been granted to a CRISPR gene editing therapy.

This article provides an update on current approved CRISPR and other gene editing therapies and outlines the latest development activities including ongoing clinical trials of key experimental therapeutics with data sourced from IQVIA Pipeline Link and IQVIA Trial Link. 

Stay informed about the latest advancements in CRISPR gene editing therapies and other genome editing systems, including authorized treatments and ongoing clinical trials. Download the article now to gain strategic insights and stay ahead in the rapidly evolving field of pharmaceutical innovation!

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