For those suffering from the agonizing effects of sickle cell disease, hope has long remained elusive. The severe inherited blood disorder throttles the body's oxygen supply, unleashing bouts of excruciating pain and life-threatening crises which can claim lives prematurely. But a groundbreaking FDA approval today marks a major milestone that could fundamentally transform treatment options for this debilitating illness.
The newly FDA-approved treatment, called Casgevy, represents the first ever approved therapy using CRISPR gene editing. It works by harnessing the power of CRISPR to dial down production of hemoglobin in its adult form, while restoring levels of an alternative fetal hemoglobin that functions better for those with sickle cell disease.
Casgevy reprograms the process that normally shuts off fetal hemoglobin after birth, allowing the body to revert to making this alternate form into adulthood instead. This effectively counteracts the defective adult hemoglobin that contorts blood cells into the dysfunctional sickle shape.
By enabling patients to produce normal fetal hemoglobin again, Casgevy offers the promise of making sickle cell disease much milder, if not curing it altogether. The approval offers a “revolutionary” new option beyond the currently limited treatment alternatives for a disease affecting 100,000 Americans.
After decades of hopes pinned on gene therapy, the ability to precisely edit genes using CRISPR has finally unlocked its vast potential. Casgevy represents the first CRISPR treatment to gain FDA approval for any condition - but likely only the first of many advances to come.
REFERENCE:
•FDA: https://www.fda.gov/news-events/pre...-therapies-treat-patients-sickle-cell-disease
•NY Times: https://www.nytimes.com/2023/12/08/health/fda-sickle-cell-crispr.html
•NPR: https://www.npr.org/sections/health...rst-gene-editing-treatments-for-human-illness
•CHOP: https://answers.childrenshospital.org/sickle-cell-fetal-hemoglobin-timeline/
The newly FDA-approved treatment, called Casgevy, represents the first ever approved therapy using CRISPR gene editing. It works by harnessing the power of CRISPR to dial down production of hemoglobin in its adult form, while restoring levels of an alternative fetal hemoglobin that functions better for those with sickle cell disease.
Casgevy reprograms the process that normally shuts off fetal hemoglobin after birth, allowing the body to revert to making this alternate form into adulthood instead. This effectively counteracts the defective adult hemoglobin that contorts blood cells into the dysfunctional sickle shape.
By enabling patients to produce normal fetal hemoglobin again, Casgevy offers the promise of making sickle cell disease much milder, if not curing it altogether. The approval offers a “revolutionary” new option beyond the currently limited treatment alternatives for a disease affecting 100,000 Americans.
After decades of hopes pinned on gene therapy, the ability to precisely edit genes using CRISPR has finally unlocked its vast potential. Casgevy represents the first CRISPR treatment to gain FDA approval for any condition - but likely only the first of many advances to come.
REFERENCE:
•FDA: https://www.fda.gov/news-events/pre...-therapies-treat-patients-sickle-cell-disease
•NY Times: https://www.nytimes.com/2023/12/08/health/fda-sickle-cell-crispr.html
•NPR: https://www.npr.org/sections/health...rst-gene-editing-treatments-for-human-illness
•CHOP: https://answers.childrenshospital.org/sickle-cell-fetal-hemoglobin-timeline/
