Seeking Gene Therapy Cures

Advancing an integrated pipeline of genetic therapies that correct the root cause of complex and rare life-threatening disorders

Mission, Vision & Values

Our mission is to develop first-in-class and best-in-class, curative gene therapies for patients with devastating diseases. Our vision is “Seeking Gene Therapy Cures”. Our company values of Trust, Be Generous, Be Curious and Elevate are at the heart of all that we do.


Trust is given and trust is earned – it’s a balance. The word "trust" comes from the Proto-Indo-European word "deru" which means “to be firm, solid, steadfast.” Trust is the ground and foundation for everything we do.


Being generous means following up, sharing our best ideas, forgiving ourselves and others, asking who needs us, treating our word as gold, taking time to truly see others, and so many other things. The word "generous" has the same root as the word “gene” – which meant “to beget.” Genes thrive on the generosity of others. What more is there to say?


The wonder of a child staring up at the night sky. Humility, egolessness. No single one of us can do this job alone and it is ok to ask for help. Curiosity is derived from the Latin word “cura” which gave birth to the word “care” as well as “cure.” Generosity is to curiosity what gene is to cure.


Derived from the Latin word "levis," which means “light” as opposed to heavy. How can we bring trust, generosity and curiosity to elevate ourselves, each other, the pipeline and ultimately the life experience of patients and their families?

Patient Community

Our team strives to put patients and their families first when developing treatments for rare and complex inherited disorders. We aim to create a community where rare disease patients and their families can come together and feel supported through treatment and beyond.

hand painting at Rare Disease Day Event
scientists working lab

Our Science

Our strategic approach to gene therapy has positioned us to launch a sustainable multi-platform pipeline of treatments that directly target the genetic mutation in the affected cells for rare, life-threatening disorders, including Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Danon Disease, Pyruvate Kinase Deficiency (PKD), PKP2 arrhythmogenic cardiomyopathy (ACM), BAG3-associated dilated cardiomyopathy (DCM) and multiple undisclosed diseases.

Our People

The Rocket team possesses decades of experience and leadership in the biotech industry. This expertise, combined with our passion, creativity, a commitment to patients and curiosity, has enabled us to grow at an unprecedented rate and move four programs into the clinic in just two years.

Our People - Rocket Pharmaceuticals team members
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