Seeking #GeneTherapy cures for patients with rare and devastating diseases.

Joined November 2019
At last week’s Meeting on the Mesa, our CEO Gaurav Shah, MD, joined fellow industry leaders to share lessons in clinical safety, manufacturing and patient access, and discuss how those lessons can strengthen the path from development to treatment. Thank you to the @alliancerm and our fellow panelists for sharing their experience and perspectives. At Rocket, our purpose remains clear: translate scientific progress into meaningful treatment options for patients and families who need them. #CGMesa26 #GeneticMedicines
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October is #SuddenCardiacArrestAwarenessMonth. Sudden cardiac arrest can affect anyone, but genetic heart conditions like PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM) and BAG3-associated dilated cardiomyopathy (BAG3-DCM) can put some individuals at greater risk. That's why Rocket remains committed to advancing #GeneticMedicine for #CardiovascularDisease.
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September marks Pediatric Cardiomyopathy Awareness Month, and #TeamRocket is proud to stand with the @CCFheartkids in shining a light on the children and families living with this disease. #Rocket is focused on advancing #GeneticMedicine for cardiovascular diseases like Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM). This month and every month, we're inspired by the courage of patients and caregivers, and we remain committed to seeking a world where DNA is no longer destiny and #GeneticDisease is history.
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#TeamRocket is headed to the @escardio’s 2026 Congress in Germany, starting August 28th! Rocket is looking forward to connecting with experts to advance science, improve patient lives, and discuss #GeneticMedicine at one of the world’s premier cardiovascular medicine conferences. #ESCCongress
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#TeamRocket is headed to the San Diego Heart Failure Symposium on July 9th! Our team is looking forward to sharing updates on our #GeneTherapy pipeline and connecting with other experts in the field.
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#TeamRocket is at @escardio’s #HeartFailure26, engaging with the cardiovascular community to share our #GeneTherapy research and build ongoing relationships aimed at earlier diagnosis, advancing research, and strengthening collaboration in addressing inherited cardiomyopathies.
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#TeamRocket is heading to the @ASGCTherapy Annual Meeting next week!  We look forward to connecting with experts on the latest in #GeneTherapy for rare cardiovascular diseases. #ASGCT2026
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#TeamRocket is attending @escardio’s upcoming Heart Failure 2026 Congress, where we will connect with experts on rare cardiovascular diseases. Join us on May 11th at 3:30 CEST for a presentation on our AAV gene therapies for genetic cardiomyopathies. #HeartFailure26
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#TeamRocket is heading to Rome for the ARM Cell & Gene Therapy Meeting on the Med! Our CEO, Gaurav Shah, MD, will be joining a panel about removing barriers in cell and gene therapy. We look forward to engaging with the regenerative medicine community about our #GeneTherapy solutions for rare cardiovascular diseases.
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#TeamRocket is heading to @hrsonline’s Heart Rhythm 2026 to discuss the latest in cardiovascular care and research with fellow leaders in the field. As we advance our #GeneTherapy pipeline for rare genetic heart conditions, including #DanonDisease, we look forward to connecting with the #cardiovascular community. #HRS2026
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#NEWS: Rocket Pharmaceuticals today announced the U.S. FDA approval of the first #GeneTherapy for pediatric patients with severe Leukocyte Adhesion Deficiency-I (LAD-I). More: bit.ly/3NRnGsh
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#TeamRocket is at @ACCinTouch 2026 Annual Scientific Sessions engaging with leading cardiologists about researching genetic medicines for the heart — including our #GeneTherapy pipeline for #DanonDisease & other rare genetic heart conditions. Learn more:  rocketpharma.com
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Last month, we celebrated #RareDiseaseDay—but for #TeamRocket and the rare disease community, advocacy continues throughout the whole year. Rocket is proud to work alongside advocates like the @EveryLifeOrg for Rare Diseases in our shared mission to bring transformative treatments to patients with rare diseases. Check out these photos of our team at our Rare Disease advocacy events last month. ⬇️
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At 12 pm ET tomorrow,hear Donald B. Kohn, MD and Jordan Orange, MD discuss #GeneTherapy for inborn errors of immunity as part of @Info4PI’s Speaker Series. Register here: bit.ly/4cnm3N1
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#TeamRocket is presenting at the 2026 Primary Immune Deficiency Treatment Consortium Annual Workshop. Join us on 3/19 at 4:20 pm MT to hear more about LAD-I. Learn more about our science:  rocketpharma.com/our-science…
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Happy #InternationalWomensDay! This year's theme #GiveToGain speaks to everything #TeamRocket believes in. When we give our knowledge, support, & mentorship to those around us, we all gain. Celebrating the women advancing therapies for rare #cardiovasculardiseases every day. Learn more:  rocketpharma.com
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This #RareDiseaseDay, Rocket stands with the 300+ million people living with #RareDiseases. We’re proud to join @rarediseases in #LightUpforRare & continue our pursuit of a world where DNA is no longer destiny. Learn more about our commitments: rocketpharma.com @NasdaqExchange
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#TeamRocket is proud to have participated in #RareDC2026, supporting @EveryLifeOrg’s event that brings together patients, caregivers, advocates, policymakers and more to advance solutions for the #RareDisease community.
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#TeamRocket is proud to sponsor the @globeevents Rare Disease Summit tmrw! Join us in-person or virtually to hear patient stories, learn about advances in research & connect w/the #RareDisease community. Register: bit.ly/4r8y3WL
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