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Rocket Pharmaceuticals Inc   (RCKT)
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What are Rocket Pharmaceuticals Inc's Business Segments?



Rocket Pharmaceuticals Inc is a clinical-stage biotechnology company that focuses on developing gene therapies for genetic disorders, utilizing lentiviral vectors to deliver genetic material to cells. The following is an extensive description of the segments, products, and services offered by Rocket Pharmaceuticals Inc:

Segments:

Rocket Pharmaceuticals has three main segments:

1. Rare Pediatric Genetic Diseases: Rocket Pharmaceuticals Inc focuses on developing gene therapies for rare pediatric genetic diseases. These genetic disorders are life-threatening, and they affect a small percentage of the population. Rocket Pharmaceuticals has identified four therapeutic areas to focus on, which are:

e Hematologic Disorders: disorders of the blood, such as severe combined immunodeficiency (SCID) and Fanconi anemia.

e Metabolic Disorders: disorders that affect the body's metabolism, such as Crigler-Najjar syndrome and Fabry disease.

e Immune Disorders: disorders that affect the immune system, such as leukocyte adhesion deficiency (LAD) and pyruvate kinase deficiency (PKD).

e Neurologic Disorders: disorders that affect the nervous system, such as Danon disease and Tay-Sachs disease.

2. Oncology: Rocket Pharmaceuticals focuses on developing gene-based therapies for cancer. They use lentiviral vectors to deliver genetic material to cancer cells to increase their susceptibility to chemotherapy and other drugs. Rocket Pharmaceuticals is currently working on developing therapies for hematologic malignancies and solid tumors.

3. Gene Editing: Rocket Pharmaceuticals is developing gene editing technologies to correct genetic mutations that cause disease. They use the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system to edit genes.

Products:

Rocket Pharmaceuticals has developed several gene therapies, including:

1. RP-A501: This gene therapy is being developed to treat Danon disease, which is a rare genetic disorder that affects the heart, skeletal muscles, and intellectual disability. RP-A501 uses lentiviral vectors to deliver functional copies of the LAMP2 gene, which is mutated in individuals with Danon disease.

2. RP-L102: This gene therapy product is being developed to treat a hematologic disorder called LAD, which is a rare genetic disorder that affects the immune system. RP-L102 uses lentiviral vectors to deliver functional copies of the CD18 gene, which is mutated in people with LAD.

3. RP-L201: This gene therapy is being developed to treat the blood disorder Fanconi anemia. It uses lentiviral vectors to deliver functional copies of the FANCA gene, which is mutated in people with Fanconi anemia.

4. RP-L301: This gene therapy is being developed to treat PKD, which is a genetic disorder that affects red blood cells. RP-L301 uses lentiviral vectors to deliver functional copies of the PKLR gene, which is mutated in people with PKD.

Services:

Rocket Pharmaceuticals offers the following services:

1. Clinical Development: Rocket Pharmaceuticals offers clinical development services, including clinical trial design, monitoring, and management for gene therapy products.

2. Manufacturing: Rocket Pharmaceuticals offers manufacturing services to produce gene therapy products for clinical trials and commercialization.

3. Regulatory Affairs: Rocket Pharmaceuticals offers regulatory affairs services, including support for regulatory submissions and interactions with regulatory agencies.

4. Scientific Advisory: Rocket Pharmaceuticals offers scientific advisory services to support the development of gene therapies for rare pediatric genetic diseases.

In conclusion, Rocket Pharmaceuticals Inc has three main segments, Rare Pediatric Genetic Diseases, Oncology, and Gene Editing. They have developed several gene therapies to treat rare pediatric genetic diseases such as Danon disease, LAD, Fanconi anemia, and PKD. Rocket Pharmaceuticals also offers clinical development, manufacturing, regulatory affairs, and scientific advisory services.
   

Rocket Pharmaceuticals Inc Tax Rate Companies within the Major Pharmaceutical Preparations Industry



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