Rocket: Seeking gene therapy cures
Case Study

Rocket: Seeking gene therapy cures


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. They are platform agnostic and choose each program’s gene therapy platform based on what is most practical for the disorder being targeted.

The need

Rocket is a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders.

The mission

Rocket’s mission is to develop first-in-class and best-in-class, curative gene therapies for patients with devastating diseases.

Our progress

Rocket current has five programs in the clinical trials, with two programs in registration-enabling Phase 2.

Identifying unmet patient need

We seek to invest and build companies developing transformative therapies. Thanks to genome, disruptive innovation of new modular technologies, such as RNA medicine and gene therapy, can addressed undruggable before by older modalities like small molecules and antibodies.

Forming and building Rocket

Rocket was born out of more than a year-long study in gene therapy. In late 2015, Rocket was formed around a single academic license from a European academic institution. RTW hired a world-class management team, including CEO Dr. Gaurav Shah, COO Kinnari Patel, and CMO Dr. Jonathan Schwartz, and continued to identify additional targets and licensed four more academic programs.

Supporting Rocket through the lifecycle

RTW completed two private financings, syndicating both the Series A and Series B rounds, and took Rocket public through a reverse merger in January 2018. We believe opportunities exist to license additional gene therapy academic assets into the Rocket pipeline in the future. In addition to our board representation in the company, Rocket’s generous pipeline diversification of now five clinical programs creates an attractive risk reward opportunity, giving us comfort in owning an outsized position in the company.

Developing first in class gene therapies

Five of Rocket’s clinical programs include four lentiviral vector-based gene therapies for the treatment of:

  • Fanconi Anemia, a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer;
  • Leukocyte Adhesion Deficiency-I, a rare genetic disorder of immunodeficiency in young children;
  • Pyruvate Kinase Deficiency, a rare genetic disorder affecting red blood cells;
  • Infantile Malignant Osteopetrosis, a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure;
  • and an adeno-associated virus-based gene therapy for Danon disease, a devastating, paediatric heart failure condition.

Rocket’s goal is to have all five clinical programs become approved first-in-class gene therapies. The company is aspiring to become the next “Genentech of gene therapy” and we are looking forward to supporting them on this journey.