A Gene Therapy for Sickle Cell Disease has Been Approved for an Investigational New Drug Application by the FDA

The United States Food and Drug Administration has accepted an Investigational New Drug application (IND) for an investigational gene therapy designed to be a treatment for sickle cell disease. Previously, the FDA had placed the IND under clinical hold, so its acceptance is a significant milestone in the drug’s development. For more detailed information, you can view the source press release at Vertex Pharmaceuticals’ website by clicking here.

About CTX001

CTX001 is an investigational gene therapy that is being developed as a potential treatment for beta-thalassemia and sickle cell disease. According to Vertex’s press release, it is designed to work by elevating levels of foetal haemoglobin (a type of haemoglobin that is present at birth but is replaced by adult haemoglobin), which may help to alleviate sickle cell crises. The therapy is being developed and commercialised through collaboration between CRISPR Therapeutics and Vertex.

The IND Application

CRISPR Therapeutics and Vertex initially applied to the FDA for an IND for CTX001 for the treatment of sickle cell disease in April this year, however, the application was placed on clinical hold while the FDA waited for questions to be resolved.

INDs are used to get around a US law that prohibits the transport of drugs across state lines before they are approved for marketing. Since most drug developers want to carry out studies in several states, they need the FDA to approve an IND for their drug to continue with their research.

Next Steps

The news about the IND acceptance follows a previous announcement that CTX0001 had received approval for its Clinical Trial Applications in several countries for beta-thalassemia and sickle cell disease. The next step will be to initiate a planned Phase 1/2 clinical study of the drug in patients with sickle cell disease later this year. In addition, a study of the therapy for beta-thalassemia is currently enrolling patients in Europe.


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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