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Pilot Feasibility Study of BCL11A as Gene Therapy Target in Sickle Cell Disease

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Preliminary results from pilot feasibility study suggest that a gene therapy construct targeting BCL11A is safe, effectively induces HbF expression, and improves sickling signs and symptoms when delivered to patients with severe SCD.

Released: December 13, 2019

Expiration: December 11, 2020

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Jointly provided by the Annenberg Center for Health Sciences at Eisenhower and Clinical Care Options, LLC
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Supported by educational grants from

AstraZeneca

Celgene

Dova Pharmaceuticals

Janssen Biotech, Inc., administered by Janssen Scientific Affairs, LLC

Karyopharm Therapeutics

Loxo Oncology subsidiary of Eli Lilly

Pharmacyclics an AbbVie Company