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Rocket Pharmaceuticals, Inc.
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7 products found

Rocket Pharmaceuticals, Inc. products

Adeno-Associated Viral Vector (AAV) Therapy

The LVV platform is ideal for modifying hematopoietic stem cells (HSCs) to address disorders affecting the bone marrow. The LVV transduction process occurs ex vivo (outside the body), which ensures that the gene has been properly integrated before the therapy is given to the patient. The process involves collection and isolation of a patient’s hematopoietic stem cells (HSCs), insertion of the corrected gene into the HSCs via a lentiviral vector outside the body, and the infusion of modified HSCs back into the patient. The goal is to enable sufficient quantities of a healthy, disease-modifying therapeutic protein to be manufactured by the patients’ own hematopoietic cells. Rocket’s LVV-based programs include: Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency and Infantile Malignant Osteopetrosis.

Lentiviral Vector (LVV) Therapy

The LVV platform is ideal for modifying hematopoietic stem cells (HSCs) to address disorders affecting the bone marrow. The LVV transduction process occurs ex vivo (outside the body), which ensures that the gene has been properly integrated before the therapy is given to the patient. The process involves collection and isolation of a patient’s hematopoietic stem cells (HSCs), insertion of the corrected gene into the HSCs via a lentiviral vector outside the body, and the infusion of modified HSCs back into the patient. The goal is to enable sufficient quantities of a healthy, disease-modifying therapeutic protein to be manufactured by the patients’ own hematopoietic cells. Rocket’s LVV-based programs include: Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency and Infantile Malignant Osteopetrosis.

Pipeline Products

Fanconi Anemia (FA)

FA is a rare, genetic disorder affecting DNA repair. Approximately two-thirds of FA cases are caused by genetic defects in the FANCA gene, which results in the FA subtype known as FA Complementation Group A (FA-A). FA patients may develop bone marrow failure (very low blood counts), cancers of the blood or other cancers.

Pyruvate Kinase Deficiency (PKD)

PKD is a rare, genetic blood disorder. PKD is caused by a defect in the PKLR gene which is responsible for energy production in red blood cells (RBCs). RBCs carry oxygen to the rest of the body. When the PKLR gene has a defect, RBCs are limited in their ability to produce energy and remain intact. As a result, patients with PKD frequently have anemia, chronic fatigue, yellowing of the skin and eyes (jaundice) and enlarged spleens.



Danon Disease (DD)

Danon Disease (DD) is a rare genetic disorder that is characterized by severe and primarily hypertrophic cardiomyopathy. Skeletal muscle weakness and mild cognitive impairment are also common.  The causative mutation has been identified in the gene encoding for lysosome-associated membrane protein (LAMP2), and in particular the LAMP2B version of the gene which is primarily expressed in heart, skeletal muscle and brain tissue.  Affected cells can no longer efficiently digest and recycle protein and other materials, leading to dysfunction in heart, muscle and brain cells.