Rocket Pharmaceuticals, Inc. focuses on developing gene therapies aimed at curing rare genetic diseases by addressing their root causes. The company employs a multi-platform approach, utilizing ex vivo lentiviral and in vivo adeno-associated viral technologies to create targeted treatments for conditions with significant unmet medical needs. Its primary customers include patients and caregivers affected by these rare diseases, and it operates within the biotechnology sector, specifically in the field of gene therapy.
