Rocket Pharmaceuticals’ Research and Development (R&D) and Manufacturing Facility, Cranbury

Rocket Pharmaceuticals will build a new research and development (R&D) and manufacturing facility in Cranbury, New Jersey, US to support the production of its pipeline of lentivirus and adeno-associated virus (AAV) gene therapies.

The facility will house R&D and chemistry, manufacturing and control (CMC) operations. Announced in January 2021, it will also serve as the new headquarters and accommodate 150 staff members in Cranbury, New Jersey.

Rocket Pharmaceuticals plans to begin the first current good manufacturing practice (cGMP) production at the facility in 2021.

Rocket Pharmaceuticals’ Research and Development and Manufacturing Facility details

Spanning 103,720ft², Rocket Pharmaceuticals’ new state-of-the-art facility will dedicate approximately half of the entire space to AAV cGMP production. The remaining area will house R&D laboratories to support CMC operations for process and analytics.

Furthermore, the company intends to use the facility for a phase two study to evaluate the company’s first AAV-based gene therapy, RP-A501, for the treatment of Danon disease, a heart-related disease.

The Cranbury facility will play a vital role in the company’s objective to deliver five curative gene therapies for the treatment of rare diseases by 2025. It will support the company’s pipeline of genetic therapies.

Lease agreement and existing facility details

Rocket Pharmaceuticals signed a lease agreement for approximately 92,000ft² of rentable space in Cranbury to support process development, office, research and manufacturing activities.

The company’s Research and Discovery group uses 20,000ft² of labs at the site for the development of therapeutics.

Funding details

Rocket Pharmaceuticals secured approximately $300m through a public equity offering of 6,035,714 shares of its common stock in December 2020.

The company will use a portion of the funds raised through the public equity offering to build-out the new facility and develop Rocket’s gene therapies pipeline for rare diseases filing for the marketing authorisation of RP-L201 in the US and Europe.

Investment banking firms, including JP Morgan, BofA Securities, Piper Sandler and SVB Leerink served as the joint book-running managers for the public offering.

Rocket’s RP-A501 AAV-based gene therapy for Danon disease

Rocket reported positive interim Phase 1 clinical trial results for RP-A501 in December 2020. The gene therapy candidate expressing lysosomal associated membrane protein 2 (LAMP2B) for the treatment of Danon Disease showcased increased gene expression and positive biomarkers.

Preliminary data indicated that the low dose RP-A501 cohort remained well-tolerated and offered early evidence of clinical benefit.

Marketing commentary on Rocket Pharmaceuticals

Rocket Pharmaceuticals is a clinical-stage biotechnology firm focused on the development of a pipeline of genetic therapies for complex and rare childhood diseases.

In March 2020, Rocket Pharmaceuticals entered a research partnership agreement with Forty Seven, a clinical-stage immuno-oncology company, for the treatment of Fanconi Anaemia, a rare paediatric genetic disorder that results in bone marrow failure.

Rocket’s genetic therapies pipeline includes both adeno-associated viral vector and lentiviral vector (LVV) approaches for the treatment of rare genetic disorders.

The company leverages LVV-based programmes for the treatment of Fanconi Anemia, Leukocyte Adhesion Deficiency-I, a rare genetic disease that leads to malfunctioning of the immune system, Pyruvate Kinase Deficiency, a rare red blood cell disorder and Infantile Malignant Osteopetrosis, a rare, genetic bone disorder. The company announced that its first AAV-based programme targets Danon disease.