Rocket Pharmaceuticals
Rocket Pharmaceuticals, Inc. is a biotechnology company dedicated to developing innovative gene therapies for rare and severe pediatric diseases. The company specializes in lentiviral vector and adeno-associated virus platforms to target genetic disorders. Its lead program focuses on treating Fanconi Anemia, a serious condition that leads to bone marrow failure. In addition to this, Rocket is actively researching therapies for several other bone marrow-derived disorders, including Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis. Furthermore, the company is developing an AAV-based gene therapy for Danon Disease, a multi-organ disorder associated with increased risk of cardiovascular complications. To advance its research, Rocket Pharmaceuticals maintains numerous collaborations and license agreements with prestigious institutions, enhancing its capabilities in gene therapy development. The company is headquartered in New York, New York.
Top Stages
2 past transactions
Renovacor
Acquisition in 2022
Renovacor, Inc., established in 2013 and headquartered in Philadelphia, Pennsylvania, is a biopharmaceutical company dedicated to developing transformative gene therapies for cardiovascular diseases. Currently in the preclinical stage, Renovacor's primary focus is on creating a recombinant adeno-associated virus (AAV)-based gene therapy targeting patients with dilated cardiomyopathy (DCM) caused by mutations in the Bcl2-associated athanogene 3 (BAG3) gene. This condition affects approximately 35,000 individuals in the United States and Europe, qualifying it as an orphan disease. Renovacor aims to improve patient outcomes by potentially preventing disease progression through a BAG3 gene replacement therapy, given the current five-year survival rate of only 50% for DCM patients despite standard care.
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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