Kinnari Patel, President and COO of Rocket Pharmaceuticals.
Kinnari Patel’s love for science began at a very young age as her traditional Indian parents may have strongly encouraged – or rather expected her to like science – it didn't take much convincing for her to follow her natural curiosity. That curiosity led Patel to the C-Suite at cutting edge biotech company Rocket Pharmaceuticals before the age of 40. Fast forward to today Patel is leading the way in finding gene therapy treatments for rare diseases in children.
“Science was often seen as a path to success and a field highly respected, which I fully appreciate now,” Patel explained. “As an undergraduate, I pursued a pre-med curriculum and enjoyed pharmacy courses, especially in how drugs were created, interacted with each other and impacted patients.” While pursuing her Doctorate of Pharmacy program, Patel shared that she was given the prestigious opportunity to do a rotation with the FDA’s Office of Orphan Drugs, an office dedicated to supporting and advancing the development and evaluation of treatments for rare disease. The most shocking revelation for Patel, was learning there were more than 7,000 rare diseases in the world, impacting 400 million people globally, including 3 in 10 children. According to Patel, this is more than cancer and AIDS combined.
“The fact that 400 million people face rare diseases in the world, yet with so few FDA approved treatments, had a big impact on me. At that moment, it became my goal to join the pharma and biotech industry and research potential treatments for rare diseases. I wanted to use science as a vehicle to provide a better life for patients and humanity.” Following a career at several top global pharma companies, Patel became one of the first executives of Rocket Pharma, a leader in biotech focused on seeking cures for rare, life threatening, pediatric diseases at their root cause at the genetic level through gene therapy - not just seeking a treatment.
Despite some of the major barriers in the industry, Patel saw a huge opportunity to apply her big pharma experience in a new visionary biotech company and potentially change the trajectory of people’s lives and for generations to come. Prior to taking on the role of President and Chief Operating Officer at Rocket Pharma, Patel had her own personal reservations about her abilities and shared that she even researched and turned over a list of candidates she felt may be better suited for the position. “When I first encountered the opportunity, my experience and background were geared towards regulatory affairs, drug development, and working closely with health authorities like the FDA – a world away from leading operations at a publicly traded company. Fortunately, Dr. Gaurav Shah, Rocket Pharma’s CEO, believed in my abilities to integrate science and business, and didn’t give up. It took about six months for me to convince myself, and in the nearly 5 years since, I’ve never looked back.”
Like most women she knew, Patel had also experienced gender discrimination in her past roles. From ideas being blatantly ignored by male colleagues to receiving feedback about her appearance. “In the beginning of my career, I went along with some of these suggestions regarding my appearance – I wore glasses to appear older, dressed formally, and smiled less,” Patel said. The more she focused on doing the work and making a difference Patel “quickly realized that these gender-based societal expectations of my appearance were so superficial.
Rocket Pharmaceuticals focuses on finding gene therapy cures for rare childhood diseases and ... [+]
Today at Rocket Pharma, Patel says they have built a very different type of company. “We are a company that values gender parity and celebrates diversity – people can be their best, and the best team members are selected for opportunities based on their work, regardless of gender.” Pushing aside her feelings of uncertainty or previous experiences with gender discrimination, Patel educated herself about the business opportunity for Rocket Pharma, and realized that her experience from the pharmaceutical industry could be invaluable to a first-of-its-kind, innovative biotech startup.
Since it launched in 2015, Rocket Pharma has advanced four gene therapy programs targeting rare diseases, going from initial concept to compelling data from human clinical studies in a rapid fashion without compromising quality or patient safety. In fact, Patel shared that one of Rocket Pharma’s gene therapies - for Danon disease- is now the first gene therapy targeting the heart to show early positive results in human studies.
Another one of the gene therapies that Rocket Pharma has developed is for a rare disease called Leukocyte Adhesion Deficiency (LAD-I), a rare genetic immune disorder that leaves young patients vulnerable to infections that often result in death within the first two years of life for most patients. In fact, after having lost a child to LAD-I, a family from India found out their second child also had severe LAD-I. The family, living in a remote village in India, was desperate to do whatever it took to give their daughter a chance at life. When Rocket Pharma learned about their story, the company helped them navigate complicated immigration processes, and helped them get passports in an effort to have her join the company’s LAD-I trial.
“Then came the unplanned roadblocks of COVID-19 which caused delays due to borders being shut down and then causing this young child to get hospitalized due to COVID-19.” Patel explained. “We knew the clock was ticking for this 2-year-old girl.” The team at Rocket Pharma never gave up. After a lot of hurdles, according to Patel, the young girl finally made it to the U.S. for LAD-I gene therapy treatment as part of its LAD-I trial. “Now, more than two years later, she is alive and thriving – zero hospitalizations and living a normal life,” Patel said. The company has since treated approximately 40 kids around the world.
Today, Rocket Pharma has more than 200 employees including some of the best minds in science and gene therapy, according to Patel. And as the company continues to forge a growing number of partnerships and trials happening around the world, Patel believes the company’s progress has been remarkable and is proud that she has had the opportunity to help lead Rocket Pharma to this point. The company is also preparing to submit its first filings for potential U.S. and European approvals for two programs in 2023.
