Overview: Diseases That Gene Editors Potentially Could Cure

In just 10 years since CRISPR first emerged as a game-changing gene-editing technology, more than 100 gene-editing therapeutic candidates have reached the clinic, where they are being evaluated in trials for a growing range of diseases, including infectious diseases, blood disorders, blood and solid cancers, eye diseases, metabolic disorders, and a number of rare genetic conditions.

Within the CRISPR Medicine field in 2021 alone, we saw positive clinical data for the first ever in vivo CRISPR medicines, base editors made their way to the clinic, and therapies began to emerge in new disease areas such as diabetes and hereditary angioedema. You can view the full list of ongoing clinical trials here.

At CMN, we strive to cover all clinical and industrial news concerning therapeutic gene editing, as well as research and technological advances that solve major challenges and push gene-editing modalities towards new therapies, in many cases for incurable diseases that have limited treatment options.

CMN Disease Overview

To supplement our clinical trials listing, our regular CMN interviews and clinical news updates, we recently began building an overview of all of the diseases that are in scope for gene-editing therapy or diagnostics.

This includes diseases that are currently being targeted in gene-editing clinical trials, as well as diseases that are being investigated at research level. Each record contains the standard disease code (ICD, according to the International Classification of Diseases), general disease description, as well as information about mutations involved (when known), disease frequency, symptoms, treatment and a list of sources used. An excerpt from our cystic fibrosis disease record is shown below.