RCKT Profile
Rocket Pharmaceuticals Inc (RCKT) is a clinical-stage biopharmaceutical company focused on developing gene therapies for rare genetic diseases. The company’s lead product candidate is RP-L102, a gene therapy for Fanconi anemia (FA), which is currently in a Phase 2 clinical trial. RP-L201, a gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I), is also in Phase 1/2 clinical development. The company is also developing gene therapies for Danon disease, pyruvate kinase deficiency, and infantile malignant osteopetrosis.
Rocket’s gene therapies are designed to deliver healthy copies of the defective genes that cause these rare diseases into patients’ cells. The company uses a lentiviral vector system to deliver the corrected gene to the target cells, with the aim of achieving long-term therapeutic benefits.
Rocket has partnerships with some leading research institutions, including the National Institutes of Health (NIH) and Memorial Sloan Kettering Cancer Center (MSK). The company has also been actively expanding its intellectual property portfolio, with more than 100 patents and patent applications covering its technology and product candidates.
In addition to its gene therapy programs, Rocket has a clinical-stage small molecule program, which includes a development program for a c-kit inhibitor for the treatment of gastrointestinal stromal tumors (GIST). The company has also established a manufacturing facility to support its gene therapy programs.
Rocket Pharmaceuticals was founded in 1999 and is headquartered in New York, NY.
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