The first FDA filing of a therapy based on gene-editing technology

Joao Incio
By Joao Incio

What better day than World DNA Day to celebrate advancements in gene-editing technology! Our resident Bio sector lead, Joao Incio, explores CRISPR gene-editing medicine and what this means for the global market and wider industry.

Fifty years after the Nobel Prize was awarded to Francis Crick, James Watson, and Maurice Wilkins for the discovery of the double helix structure of DNA molecules, the labs of Jennifer Doudna at UC Berkeley and Emmanuelle Charpentier at the Max Planck Institute in Berlin were honoured with another DNA-linked Nobel Prize — for the CRISPR/Cas9 gene-editing technology, captured within the landmark study published in Science in 2012. Within seven years, the first CRISPR therapy started clinical testing and earlier this month, Vertex Pharma and CRISPR Therapeutics have filed their joint marketing application for FDA approval. This very first commercially available CRISPR gene-editing medicine is expected to be approved later this year.

A potential cure for two blood disorders, sickle cell disease (SCD) and beta thalassaemia — with the intricate name of exagamglogene autotemcel (exa-cel) — is an ex vivo application of gene editing, in which the technology is used as a one-time therapy to modify a patient’s own cells outside the body.

Patients with beta thalassaemia and SCD present with a mutation resulting in faulty haemoglobin, the iron-containing protein in red blood cells that carries oxygen to cells throughout the body. In the case of SCD, the abnormally shaped red blood cells can cause painful and debilitating vaso-occlusive crises, while thalassaemia patients may require blood transfusions. The change induced by gene editing reverts the faulty haemoglobin to a foetal haemoglobin type that serves as a substitute to regular protein in both SCD and beta thalassaemia. As a result, patients produce normal, healthy red blood cells, which means that the therapy can potentially cure these conditions.

The global gene-editing market was valued at US$3.9 billion in 2021, with the CRISPR-Cas9 segment representing its largest share. Driven by an overall increase in R&D expenditure in this space and the demand for personalised therapies, the overall gene-editing market is expected to grow at a compound annual growth rate of 6.7% to double in size by 2031, with CRISPR technology continuing to cement its dominance. This means that there are exciting times ahead for patients with severely debilitating conditions that can be targeted by CRISPR and other innovative technologies.

Read more from our resident sector experts and scientific content writers in their latest blogs…

Shona Richardson – Trends Changing Biopharma: Cell & Gene Therapy

Joao Incio – ramarketing review: March

Geoff Potjewyd – The rise of ‘cli-fi’ and real zombie viruses

Related news, insight and opinion