Rocket Pharmaceuticals celebrated its sixth annual Rare Disease Day event bringing together leaders in the biotech community along with patients, patient advocates and Rocket employees. Every year the event highlights the progress Rocket is making towards finding gene therapy cures for rare and devastating diseases while offering hope to patient communities. Approximately 400 million people worldwide and 30 million people in the US live with a rare disease.
Rare diseases impact more people and families than all forms of cancer and AIDS combined and nine million children living with a rare disease pass away before their fifth birthday every year. Other rare diseases include Cystic Fibrosis, Hemophilia, Duchenne Muscular Dystrophy (DMD), and Sickle Cell and Ehlers-Danlos Syndrome (EDS), to name a few. It takes an average of six to eight years for patients to receive an accurate diagnosis of a rare disease. Symptoms vary not only between diseases but also among patients with the same condition. Approximately 72% of rare diseases are genetic, and only about 5% have an FDA-approved drug treatment.
DCM (Dilated Cardiomyopathy) patient “Becky” took to the stage during the daylong event at the Liberty Science Center to share how her rare disease has affected her life and how she’s found support from her community, “This is a chronic illness. There is no cure. I’m on tons of medication. I suffer from fatigue and brain fog, low blood pressure and some other unpleasant side effects. I think it’s made harder because I don’t necessarily present as sick to the outside world, so it makes it challenging to ask for help” She added,”I joined the DCM support groups and it’s crazy how these people from all across the globe, all genders, ages, races…all walks of life, have quickly become my friends and my confidantes.”
In a panel moderated by Forbes Editor-At-Large, Maneet Ahuja, Rocket CEO Dr. Gaurav Shah explained how gene therapy can potentially cure genetic-based diseases. “Gene therapy is one of the rare modalities in medicine that has emerged in the history of our species that is truly potentially curative because you’re replacing the core DNA that’s the basis of who we are as physical beings, you’re replacing faulty DNA with correct DNA so there’s no better cure to genetic diseases than gene therapy,” he shared. “We’ve cracked the door open. Our goal is to push the door open all the way.”
Rocket President and COO Kinnari Patel also suggested getting genetically tested sooner rather than later if you are having trouble with receiving a diagnosis. “I recommend everyone get tested. Don’t wait for the symptoms to appear, for you to feel uncomfortable, or having to go to the fourth or the fifth hospital to figure out what’s wrong with you. The beauty of genetic testing now versus twenty years ago when it cost ten or twenty thousand dollars, it now costs a few hundred dollars, if not less, and most insurance companies cover it.”
Rocket’s long-term vision is utilizing gene therapy to cure genetically modulated diseases, starting with diseases with single gene defects. Rare diseases may be rare individually, but collectively they are not rare. They also hope to unlock the ability to pursue cures for polygenic diseases as well. Currently, Rocket is working on gene therapies for LAD-I, FA, PKD, Danon, PKP2-ACM, and BAG3-DCM.
