Healthcare Professionals

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At Rocket, we are seeking gene therapy cures for patients with rare and devastating diseases. We are deeply committed to working closely with healthcare professionals as we advance cutting-edge gene therapy research and support patients with rare and devastating hematologic and cardiovascular diseases.

Importance of Genetic Testing

Rocket is committed to raising awareness of genetic testing programs that can shorten the diagnostic journey, including our sponsorship of the Invitae Unlock™ Cardiomyopathy and Arrhythmia testing program and role as a founding member of the BeginNGS™ consortium in partnership with Rady Children’s Institute for Genomic Medicine to facilitate newborn screenings using rapid Whole Genome Sequencing (rWGS®).

Expanded Access Policy

Access policy to experimental (pre-approval) medicines

We are committed to developing safe and effective gene therapies and making them available as quickly as possible because we know that patients are waiting. At this time, Rocket is not able to make its investigational gene therapy products available on an expanded (or early) access basis anywhere in the world.

Importance of clinical trials

Rocket understands that the generation of data through clinical trials is the globally recognized process to obtain approval and secure timely access for new therapies. Enrollment in a clinical trial ensures that necessary data is generated to inform the use of new treatment options. Information about our clinical trials can be found on the clinical trial tab of this Rocket website and by using health authority websites, including ClinicalTrials.gov.

Medical Information

Healthcare professionals may contact Rocket at medinfo@rocketpharma.com.

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