Rocket Pharmaceuticals is a clinical-stage company developing gene therapy treatments for rare childhood disorders. Life-threatening childhood disorders such as Fanconi Anemia, Danon Disease and Leukocyte Adhesion Deficiency are caused by genetic mutations in single genes. Rocket is developing therapies that directly target these mutations in affected cell types using both adeno-associated viral vectors and lentiviral vectors. Rocket’s state-of-the-art laboratories are built for designing, optimizing and advancing candidate therapeutics to clinical stage development.

<p>Rocket Pharmaceuticals is a clinical-stage company developing gene therapy treatments for rare childhood disorders. Life-threatening childhood disorders such as Fanconi Anemia, Danon Disease and Leukocyte Adhesion Deficiency are caused by genetic mutations in single genes. Rocket is developing therapies that directly target these mutations in affected cell types using both adeno-associated viral vectors and lentiviral vectors.</p>
<p>Rocket’s state-of-the-art laboratories are built for designing, optimizing and advancing candidate therapeutics to clinical stage development.</p>


Rocket Pharmaceuticals

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