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---
description: Find out why Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy is one of the best inventions of 2024
title: Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy: the 200 Best Inventions of 2024
image: https://static.time.com/v3/assets/bltea6093859af6183b/blt0a19ea45e7118927/698a8a12b3fce39edd0d22ee/vertex-pharmaceuticals-crispr-best-inventions.jpeg?branch=production&amp;width=3840&amp;quality=75&amp;auto=webp&amp;crop=16:9
---

Oct 30, 2024

# Cutting Edge Gene Therapy

by 

[Jessica Klein](/author/jessica-klein/)


## Jessica Klein


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DNA biotechnology medical research of double helix in human genetics for health - 3D Illustration Render; Shutterstock ID 1551196166; purchase\_order: Best Inventions; job: ; client: Kaya Lee Berne; other:

DNA biotechnology medical research of double helix in human genetics for health - 3D Illustration Render; Shutterstock ID 1551196166; purchase\_order: Best Inventions; job: ; client: Kaya Lee Berne; other:Shutterstock / Immersion Imagery

Combing through scientific literature, Casgevy’s creators found that adults with genes that would normally produce sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), but who also had higher levels of fetal hemoglobin—produced by babies under 1 year, with a higher affinity for oxygen than adult hemoglobin—didn’t suffer as much from symptoms. For those with no fetal hemoglobin, SCD causes painful blockages and anemia—known as pain crises—while TDT necessitates monthly blood transfusions, costing an[ estimated $5.4 million](https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9429534/ "undefined") over a lifetime. Using CRISPR technology, Casgevy harvests a patient’s blood-forming stem cells, and edits them (outside the body) to spur fetal hemoglobin production. Reinserted in the patient, they work to keep SCD and TDT symptoms at bay. In[ clinical trials](https://www.casgevyhcp.com/sickle-cell-disease/efficacy "undefined"), 93.5% of patients with SCD didn’t experience pain crises for at least a year after infusions. In Dec. 2023, the therapy became the first CRISPR-based therapy to receive FDA approval. 

These are independent reviews of the products mentioned, but TIME receives a commission when purchases are made through affiliate links at no additional cost to the purchaser.


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