Our Science

Rocket’s pipeline is comprised of first-in-class gene therapies that incorporate both adeno-associated viral vector (AAV) and lentiviral vector (LVV) approaches to gene therapy.

Rocket selects indications driven by a single gene in specific cell types and develops therapies that directly target the genetic mutation in the affected cells. We are platform agnostic and choose each program’s gene therapy platform based on what is most practical for the disorder being targeted.

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Each program is intended to be transformative, enabling not only reversal of the disorder at molecular and cellular levels, but sustained relief from debilitating and potentially life-threatening symptoms. Science and scientific data drive all product development decisions to address patients’ unmet medical need. Additionally, the first-in-class nature of our programs allows us to make the right decisions in a thoughtful manner, without outside pressure.

With this foundation, Rocket is ideally positioned to launch a sustainable pipeline of therapies that soar beyond the current treatment options for these rare and devastating disorders.

In vivo and Ex vivo diagram
AAV

AAV

The AAV platform is ideal for disorders that affect the heart, liver, eye or central nervous system.

The AAV transduction process occurs in vivo (inside the body) with the Rocket team engineering each AAV construct to incorporate the corrected gene. The “therapeutic AAV” is injected directly into a patient, resulting in expression of a healthy, disease-modifying therapeutic protein in the targeted diseased cells.

Different AAV serotypes preferentially bind to specific receptors, which enables scientists to utilize specific serotypes to increase the likelihood of successful targeting of particular cell types. For example, the AAV9 serotype has been shown to have a particular propensity for heart muscle cells.

As with the LVV-platform, the ultimate goal is to allow for sufficient quantities of healthy protein to be produced by a patient’s own cells.

Rocket’s first AAV-based program is Danon Disease, a type of heart disease.

LVV

LVV

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.

Discovery

Rocket’s Research and Discovery group in the Cranbury, New Jersey facility utilizes approximately 20,000 square feet of state-of-the-art laboratories for designing, optimizing and advancing candidate therapeutics to clinical stage development.

Manufacturing

Rocket’s manufacturing facility in Cranbury, New Jersey was designed specifically for Rocket’s manufacturing needs. Approximately half of the newly constructed 103,720 square foot facility will be dedicated to adeno-associated virus (AAV) Current Good Manufacturing Practice (cGMP) manufacturing.
Rocket manufacturing facility in New Jersey

Publications

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