It’s fitting that a company called Rocket Pharmaceuticals would want to light up the sky. Well, not the sky exactly, but for the last five years the gene therapy pharma has been lighting up landmarks and buildings around the world to shine a light on Rare Disease Day.
Rocket has partnered with the European Organization for Rare Diseases and the National Organization for Rare Diseases counterpart in Europe, and forms part of the Global Chain of Lights campaign, which unites the rare disease community across the globe.
Fittingly, the first Rare Disease Day was Feb. 29, 2009, to highlight the millions of people around the world living with a one of the many rare diseases. On non-rare-leap years the day falls on the last day in February.
This year Rocket Pharmaceuticals will also be hosting an event at the Make-A-Wish Foundation’s Wishing Place castle, a sanctuary for gravely ill children. The event brings together music, science and drug development
“A lot of rare diseases are genetic in origin and unfortunately many of them impact kids. Many of the times, unfortunately they have such severity, such high mortality, that we said you know, let's partner with them to have our event at Make-A-Wish,” said Kinnari Patel, M.D., president and chief operating officer of Rocket Pharma.
The goal of lighting up the 100-plus monuments in the green, blue, pink and purple of rare diseases throughout the U.S. and Europe is to get people wondering what these colors are for and to Google why they are lit up this way, which will, of course, lead them to information about the project.
“When people Google the colors because they think, ‘Hey, this is so beautiful, this is so different than the usual,” it gives them an opportunity to learn about rare diseases,” Patel said. I think our goal is kind of simple and through lights and we want to give hope to patients with rare diseases. Because of the 7000 genetic diseases that are out there.
"Unfortunately, only 5% of them have therapies available. So it's the 500 plus diseases, where there are no treatment options available, and many of them impact kids. So these families may feel alone …we want to give them hope that a lot of people around the community are thinking about them and we are trying to do something to change that equation.”
The idea of lighting up iconic buildings started with none other than Jay Z and Alicia Keys. Patel and her husband were watching the video for “Empire State of Mind” and the many shots of the lit-up Empire State Building made them think, "If this can be done for a music video, why not for science and rare diseases?"
While the initial response was “no,” later after the pharma moved into the iconic building, they tried again, and this time was met with an affirmative and a green, blue, pink and purple Empire State Building. Once this landmark agreed to participate, convincing other sites got much easier.
Rocket Pharma is working towards its first regulatory filings for two late-stage programs in Leukocyte Adhesion Deficiency-I (LAD-I) and Fanconi Anemia (FA) this year. If approved, these could bring gene therapy cures to over 8,000 rare disease patients across the world.