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First Gene-Editing Treatment Approved

The first ever gene-editing treatment for public availability was recently approved by the FDA. What’s its potential? 

The treatment uses something called CRISPR, a Nobel Prize-winning, gene-editing tool that works like genetic scissors to cut out and repair genomic problem areas. CRISPR technology is an efficient way to edit DNA, treat complex disease and profoundly improve patients’ health. 

Gene-editing treatments could be close to cures. 
This first breakthrough treatment is approved to treat sickle cell. Sickle cell is a blood disease impacting 100,000 Americans, nearly all of them Black. It limits patients’ red blood cells from carrying oxygen at full capacity, and it can cause chronic pain, damage organs and lead to disability.  

The CRISPR-based cure repairs sickle cell patients’ blood stem cells, then returns them to the patient via infusion. It helps alleviate not just pain for patients, but also their need for constant blood transfusions and progression of the disease. For some, the improvement in quality of life is akin to a cure.  

Public investment is required to optimize access. 

What if gene editing could treat other rare and debilitating diseases?  It’s possible, but it will require policy support and federal investment. And patients living with these conditions must be able to access treatments. 

Congress authorizing billions for health research and the “Cancer Moonshot Initiative” offer guidance. Applying similar funding to CRISPR-based treatments could accelerate treatments for common and rare conditions alike. 

Better research funding could also accelerate patients’ access to these life-transforming treatments. The current list price for the sickle cell gene-editing treatment is about $2.2 million per patient, and it can be administered at only a relatively limited number of authorized medical centers.  As gene-editing treatments become more developed and widespread, however, costs will likely moderate and geographic availability will increase. 

Approval of the first gene-editing treatment marks a pivotal moment in medical history. Now policymakers should work to advance these treatments and make them accessible to patients who need them. 

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