Rocket Pharmaceuticals
Rocket Pharmaceuticals is a biotechnology company dedicated to developing gene therapies aimed at treating rare pediatric diseases. The company's primary focus is on its lead program, a lentiviral-based gene therapy for Fanconi Anemia, a genetic disorder that leads to bone marrow failure. In addition to this, Rocket is advancing therapies for several other conditions, including Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis, all of which are currently in various stages of clinical and preclinical trials. The company is also engaged in an adeno-associated virus program targeting Danon disease, a serious multi-organ disorder. Rocket Pharmaceuticals collaborates with various academic and research institutions, enhancing its research capabilities and expanding its therapeutic portfolio. The company is based in New York, New York.
Renovacor
Acquisition in 2022
Renovacor, Inc. is a preclinical-stage biopharmaceutical company based in Philadelphia, Pennsylvania, dedicated to developing gene therapies for cardiovascular diseases. Founded in 2013, the company focuses on innovative treatments for dilated cardiomyopathy (DCM), particularly cases caused by mutations in the BAG3 gene. This condition affects over 3 million individuals in the United States, with around 35,000 patients estimated to have BAG3 mutations, classifying it as an orphan disease. Patients with BAG3-associated DCM, often younger and experiencing faster disease progression, currently have limited treatment options, primarily consisting of standard heart failure care. Renovacor's lead program involves a recombinant adeno-associated virus (AAV)-based gene therapy aimed at replacing the defective BAG3 gene, which holds the potential to prevent disease progression and improve outcomes for this vulnerable patient population.
Inotek Pharmaceuticals
Acquisition in 2017
Inotek Pharmaceuticals is developing molecules with novel mechanisms of action to fulfill major unmet medical needs in significant diseases of the eye. Complete loss of vision or vision impairment currently affects 3.3 million people over the age of 40 in the United States, with the most common diseases including age-related macular degeneration (AMD), glaucoma, cataract and diabetic retinopathy. With the continued aging of the population, the National Eye Institute projects that this number will grow to 5.5 million people by the year 2020.
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