Rocket Pharmaceuticals

#TeamRocket is lacing up our shoes this Sunday for the Hypertrophic Cardiomyopathy Association’s (HCMA) Bighearted 5k!    We’re proud to join HCMA in supporting patients, families and caregivers affected by hypertrophic cardiomyopathy and other thick heart muscle disorders.    Our commitment to the cardiac community extends beyond the 5k – we're actively advancing #genetherapies for heart conditions through our innovative AAV platform.    Hope to see you there!     https://bit.ly/4nEQDnt

We’re off to Boston for the American Society of Human Genetics (ASHG) Annual Meeting - the largest human genetics and genomics meeting and exposition in the world.      Stop by the Medelian Phenotypes Poster Session on Friday, October 17th at 2:30 pm ET to hear from our Senior Medical Director & Head of Scientific Communications Susan Manganaro present about A Hypertrophic Cardiomyopathy-Based Model Estimate of the Prevalence of Danon Disease in the United States.     Learn more:  https://bit.ly/3Vv2U22      #ASHG25 

#TeamRocket came together on #WorldHeartDay for our 4th Annual Route for Hearts Walk!     Every step we took together represents our shared commitment to addressing genetic heart conditions at their source and supporting patients and families who inspire our work. To everyone who walked with us: thank you for making a meaningful difference in #cardiovascular health.      Learn more about our commitment to genetic heart health: https://lnkd.in/gzbvd46

#Rocket is proud to support St. Jude’s Children’s Research Hospital in recognizing National Children’s Health Day!    Today, we celebrate the incredible resilience of children facing health challenges and honor the dedicated healthcare professionals, researchers, and families who work tirelessly to improve pediatric care. We at Rocket Pharmaceuticals are dedicated to our mission of developing life-changing gene therapies for children with #rarediseases like Danon disease and more.     Learn more: https://bit.ly/47ZxyrH  

#TeamRocket is proud to be a sponsor of the European Society of Gene and Cell Therapy 2025 Congress where our team is looking forward to connecting with the global gene and cell therapy community      Our Chief Science and Gene Therapy Officer, Jonathan Schwartz, will present on October 10th at 9:00 AM CEST on “Transformative Treatment of Devastating Cardiomyopathies: Safety and Efficacy in AAV Clinical Development of RP-A501 (Danon Disease) and RP-A601 (PKP2-ACM)."    We’re looking forward to hearing about the collaborative efforts driving this field forward.    See the full program:  https://bit.ly/3VBWEpc

It’s National Manufacturing Day, and we’re shouting out Elizabeth Valente, Rohini Mitra, Jessica Miller, Zalikatu Kamara, Alivia Gallo, Sneah Sukhadia, Bill Pavlik, and Cesar Dela Rosa—the incredible people behind the scenes who are involved in developing potentially curative therapies for patients and their loved ones to fully live in a world without of genetic disease.     Our teams support innovation, precision, and the relentless pursuit toward our vision of a world where DNA is not destiny and #geneticdisease is history. 

#TeamRocket is committed to deeply understanding each #raredisease we research through Natural History Studies.  These observational studies differ from clinical trials in that they capture the full patient journey: how diseases progress over time, what symptoms patients experience, and how conditions impact quality of life under standard care.  Currently, #Rocket is sponsoring two global Natural History Studies. The first is tracking patients with PKP2-related arrhythmogenic cardiomyopathy (also called ARVC/ARVD), and the second focuses on #DanonDisease. The studies are gathering data to better understand how these rare heart conditions progress, helping advance future treatments. Learn more about Natural History Studies: https://bit.ly/3KfsWny 

This #WorldHeartDay, #TeamRocket is holding our 4th Annual Route for Hearts Walk. By walking, we support the World Heart Federation’s mission to make every heartbeat count and raise awareness for #hearthealth.     At Rocket, we're advancing cardiac gene therapies to address genetic heart conditions at their source. As part of our commitment to cardiovascular health, we support research, genetic testing, and patient-centered approaches.     Hear from Joe Walter in Commercial about our commitment to heart health ⬇️ 

Join #TeamRocket tomorrow at 7:30 am at #HFSA2025 for our sponsored presentation, led by Ahmad Masri, MD, Cara Barnett, MS, CGC, Allison Cirino, MS, CGC, Chloe Reuter, MS, LCGC and Daniel Judge, MD, on Genetic Testing for Cardiomyopathies and Heart Failure. See you there!     Learn more: https://bit.ly/3Iz2yEI

As we wrap up Children's Cardiomyopathy Awareness Month, Jillian Pogorzelski, from Quality Assurance, reminds us of our dedication to developing life-changing gene therapy treatments for patients and families. The incredible strength and courage of these patients fuel our mission and drives our commitment to advancing genetic #hearthealth.     Through collaboration with patients, families, researchers, and clinicians, we can accelerate progress toward potential gene therapy treatments for those living with cardiomyopathies. Thank you to everyone who joined us in raising awareness this month and beyond.      Children's Cardiomyopathy Foundation