Meg Escobosa: Welcome to the Game-Changing Women of Healthcare, featuring exceptional women making an impact in healthcare today. Together, we dig into the many healthcare issues we face today and how these innovative leaders are working to solve them. We celebrate our guests’ accomplishments, setbacks, and the lessons they've learned throughout their careers. I'm Meg Escobosa. Join me in conversation with some of the many brilliant and courageous women on the front lines of the future of health.
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Hello everyone, and welcome back to the Game-Changing Women of Healthcare. I'm your host, Meg Escobosa. Today on the show, we have Kinnari Patel, President, Head of Research and Development, and Chief Operating Officer of Rocket Pharmaceuticals, a clinical stage company seeking gene therapy cures for patients with rare diseases.
Kinnari has more than 20 years of pharma experience, including stints at Bristol Myers Squibb, Novartis, Roche and Pfizer. Kinnari graduated with a BS in biology and a Doctor of Pharmacy degree from the University of the Sciences in Philadelphia. She completed an executive MBA from NYU Stern and the C suite Harvard Business School Advanced Management Program.
Outside of Rocket Pharmaceuticals, Dr. Patel is a member of the Alliance for Regenerative Medicine's Board of Directors and serves on the Healthcare Business Women's Association Global Advisory Board. Welcome to the show, Kinnari.
Kinnari Patel: Thank you so much for having me here. I'm excited to be part of this conversation.
Meg Escobosa: Well, we feel very lucky because you have obviously been doing a lot of great work. This particular focus is really important and it's going to make an impact in the world and that's what we're really interested in - celebrating and elevating women who are making an impact. So it's great for us to hear your story and how you're going to make that impact. We want to hear a little bit more about how you got involved in rare diseases and particularly how you got started with Rocket.
Kinnari Patel: Absolutely. So I was introduced to rare diseases back in over 20 years ago in the 2004 time frame. For starting a dual degree program at my university the dean was confused and he rewarded me by having it FDA rotation. So I did a rotation with the orphan products group at the FDA with Dr. Marlene Hefner, who was the head of the group there, and, to my surprise I never thought about drug development. I thought about clinical pharmacy care. I thought about developing oncology therapies to change the trajectory of cancers, but it wasn't until the FDA that I realized, even in America in early 2000s, there were patients that were dying with the rare diseases. There was not enough research being done in the rare disease space. There were not enough drugs approved. And even though the legislation was something I was familiar with and started in 1983, only a dozen drugs had been approved by then, or so, in a decade after and we weren't making that much traction so to me, I was fortunate in that rotation time at the FDA. They give me a great opportunity to meet patients, mom advocates, dad advocates and kids and the legislation in the US, in that time frame, kids were still an afterthought. It was done for adults and then if it was safe and effective enough, we could do it in kids. So I didn't realize at the time what I was in front of was a challenge that would become a passion for my life and life's mission. And here I am over 20 years later, having the good fortune of working at all the big pharmaceuticals and learning a lot of what to do and what not to do.
I realized the only way to truly start up and focus on the science, the patients, the unmet medical need, is to be part of a company that was started just on that foundation, which is Rocket. So I joined Rocket about nine years ago. I helped the CEO decide that should he find the company and should he be the CEO, founder and six months later, he got me involved and I ended up leaving my comfortable home in the Washington DC area, leaving AstraZeneca behind, but they made it easy for me. They said, “Hey, go. If you fail, you can always come back, right, and if you succeed, we'll acquire you,” and so my whole thing was, “No, I don't want to be acquired. If we're going to succeed, we're going to succeed the right way.” So, nine years later, still here, still loving what I do, and it's not just about the science and the patients and the drugs that we work on and diseases we work on. I've had the good fortune with our CEO and the culture at Rocket to champion rare diseases as a whole for all of health care improvement and not just the US, but globally and that has been such a fundamental difference to what I do today and why I keep going is there's a lot still to be done, but there's a lot we're doing together as a community.
Meg Escobosa: What a unique experience and very lucky experience to have the safety net of your former company willing to take you back, and that's such a testament to what kind of teammate and leader you are. They didn't want to lose you, and of course they envision acquiring you and maybe they still have those designs. It's interesting and I don't know if you are in a position to talk about this, but rare diseases, the reason they probably aren't getting attention is because of what you described is not a huge number of people who have them, and therefore, the market opportunity might be more challenging to make the case for the investment required. Can you talk a little bit about the thinking or the strategy that Rocket is using to address that challenge?
Kinnari Patel: Definitely. So, even, not just at Rocket, but all of my pharmaceutical experiences, even at Novartis, they knew me as the rare disease champion, right? So, rare disease by definition in the US means that when you apply for the designation, only 200,000 Americans, or less, have that disease impact. So if you think about it, metastatic melanoma, skin cancer, is actually a rare disease; renal cell carcinoma - rare disease, but we don't think about it from that perspective.
With the advancement of genetic testing and gene specific mutations, a lot of diseases could be sliced and diced as rare diseases because you have a personalized medication available. But what we realize is Gleevec and some of those multi-billion dollar blockbuster drugs, they were all developed for rare diseases and cumulatively the impact they've made is so significant that financial rewards are still there and a for-profit organization could still do well while doing well by the science and the patients.
So to me, the philosophies Rocket that we brought in is thinking about not just one disease, thinking about platforms, right? While we're a company started by drug developers, and we always think about what's the disease? What's the medical need? And we start from there. Is there a monogenic gene of interest that is dysfunctional, missing, that's causing these patients, unfortunately, to pass away or have a devastating life?
And if the answer to those questions are “yes”, then we will develop a gene therapy that's not personalized, but individualized medicine at times. So then we say, “What are the efficiencies we can achieve to make drug development costs cheaper so we can actually have multiple rare diseases being developed in a more cost-effective manner, more sufficient, efficient manner,” but at the same time, let's not forget because these are devastating diseases where patients don't have luxury of time, what do we do to do things right the first time, right? So even before having a clinical study, we'll talk to the patients, impacted caregivers, treating physicians, to know what's important to them.
So our first study could be informative not just for safety or dose ranging, but also gives us potential for benefit in these patients, in the patients with diseases. That leads us to a portfolio of getting drug development costs down. The second thing of recognizing patients and the COGS and revenues is this, if we can get to the patients before it's too late, and potential in Rocket that we have, and it's a beautiful potential is what we call ourselves - our mission is to make DNA no longer your destiny, meaning just because you're born with a genetic disease or mutation or missing gene, that shouldn't define who you are and for your loved ones. So if we can go to the root cause of the gene and change it, we can give the life back to that person, their family and loved ones, and exponentially, we can do really well. So what we realize and these are just public numbers a lot of rare diseases are no longer rare, but a rare disease has a community impact over 10 million just New Yorkers, New York City people, and over 300 million people worldwide, right? That's just the patients impacted, but if you think about it, when you have somebody in your family that has an illness they deal with, is it just the patient impacted or the person impacted or is it the whole family? So when you're trying to treat rare disease, you're treating billions of people's impact of life.
Meg Escobosa: So true, and actually, you're revealing quite an innovative way of thinking and just this notion of the platform mechanism of action, or how you can make an impact on the patient. That is something we're really interested in is that innovation, how did you come to it so thank you for sharing that. I think it's really fascinating, and has such incredible potential.
Why is genetic testing so important and what are some of the barriers around getting people tested?
Kinnari Patel: On one hand we feel like we grew up with DNA helix and we know what it looks like and we played with it, right? But in the human genome project, everyone's genetic profile, human genome wasn't even a project that was made public until 25, 30 years ago, right, it was not that long ago if you think about the totality of research and science. So to go from having that project come to life and I remember celebrating it with Watson and Creek on awards and when I was doing my genetics bachelor's degree, right? So it wasn't that long ago, it was 25 years or so ago.
From there to going to diagnostic testing, when I started initially working in the rare disease space, genetic testing would take so many hurdles from insurance companies to providers to finding a person or even a facility that carries a genetic test and it would cost thousands of dollars, right? We're fortunate nowadays that genetic testing is available for a few hundred dollars.
Most insurance companies in the US do cover it. In Europe, newborn screening and other things are becoming more and more a must, a requirement, not a nice to have, right? So we've come a long way, but unfortunately, the research and innovation hasn't caught up to day-to-day life. We still see most patients, it takes them about 7 to 10 years to get the right genetic diagnosis, the right diagnosis, and usually they have to go to three to five physicians to figure out what's even wrong with them.
Now, if you're thinking about Fanconi or Danon disease, where kids for Danon disease pass away by the age of 19 to 20, uniformly if they're boys. If you don't get diagnosed by the time you're symptomatic, it becomes too late. So to me, the battle against genetic testing is still a big battle.
And I think I'm really lucky that again, as I said, at Rocket, we get to do things that we think are meaningful. And one of the cool things is at Rocket, we were able to work with institutions like Invitae and Rady and become members of that. And we were able to leverage the relationships we have in the genetic testing that we made mission genome a possibility, and we launched that in November. This allows patients with cardiomyopathies to get free genetic testing and also genetic counseling to make sense of what they have and support to get them diagnosed earlier. And to me, individualized medicine or personalized medicine, if you don't know what's causing the issue that you have, how do you even go about treating it?
And what we realized with the Danon disease patients is, unfortunately, the co-founder of the Danon foundation is a mom. Oh, my gosh, she's a warrior mom. I admire her, respect her and look up to her so much. Her son was having issues walking from class to class in middle school so eventually, they got diagnosed. Found out that not only her two boys and she had Danon disease but she had 13 family members that had Danon disease and the importance of genetic testing kind of showcase right there.
We did a Rare Disease Day at Rocket. We celebrate Rare Disease Day, which I've been able to do this for 15, 20 years, but at Rocket, we do it with unlimited support and love and passion of the team, which is exponentially impactful. Two years ago, I want to say, at Rare Disease Day, we had it at a Make A Wish castle in New Jersey and one of our speakers, she was a young woman, and she has a cardiovascular genetic disease. And she got diagnosed because while playing a sport, she fell. And almost, luckily, she didn't pass away, but she was rushed off to the ER. They did genetic testing. She has a cardiomyopathy. They got all of their cousins and family members tested, and now they know that two other younger kids have it.
That knowledge means that when the kid is playing sports, when a therapy is available, when they develop symptoms, they can easily intervene before it's too late. So I think the value of genetic testing is so there, but I think we still need to make it not scary for people to understand: A - they can easily get access to it. B - what does it mean when you have the diagnosis? Their support system available is important. And three, the complexities of cost, if there are costs associated with that. The NIH has a program, companies like Rocket has programs that give people access for free to get genetic testing. So those resources, there are a lot of them available, but getting that out to patients is still a thing that we're fighting and we're trying to do as much as possible to amplify the value of it. So just uncovering the mystery of what's wrong with somebody's misdiagnosis and how do we get to the cause? So we know how to actually give them a treatment option.
Meg Escobosa: That's a great point, and it's true. Not until you actually come into symptoms, do you even, of course, why would you think you have a genetic issue? And a friend of ours just also went through the similar unfortunate scenario where they had symptoms of something, but nobody thought to do genetic testing. Their son had a cardiac arrest. He's doing fine. He's in recovery, but they had to go through many challenges on the journey. And it's true, you never think to say, “Oh, there's a genetic underlying disease impacting you.” You think there's everything else. So it's got to be a change in mindset to be thinking that's something we should look at right away. We have the tools, we have the information, and it'll lead to much clearer treatment paths. Even if we don't have the treatment today, it's coming.
Kinnari Patel: I was at a conference two weeks ago in Dallas and I met this guy and the passion, he was sitting across from me at a conference that I was presenting at, but his passion came through so much, I had to go up and say, “Hey, how are you? What do you do?” He's like, “I'm a CEO.” I'm like, “Oh, okay,” like that's not always sensing. What I learned - he's not a healthcare provider. He's CEO of a company. He started the company because his son got diagnosed with a rare disease and it didn't have treatment options available. So he launched this company to fight for his son's rare disease. And to me, that's beautiful. And that gives us so much hope.
Meg Escobosa: So true. That's amazing. And it's, and obviously you can't replace, as a parent, you just - who's going to advocate for your child more than you? Nobody.You're describing that passion. It's true. You've got to look out for them.
Can you share, you're in a really significant role at Rocket. You're a leader. Talk about what it's like to be in the leadership role and your philosophy about mentorship and supporting the next generation of leaders?
Kinnari Patel: Definitely. So, I forget that I'm in a leadership role. Sometimes people treat me differently and it takes me off guard because they're like, oh, you're the president of the company. I don't think of it that way, right? To me, when I joined Rocket, I was lucky to join a company that believed in the mission and the values that I believe in. And instead of fighting uphill, I got the honor of building something that makes sense for science, for patients, for our people in the company, right, and for the ecosystem. So it's been such a rewarding experience, but my philosophy is I wouldn't ask anybody to do something I wouldn't do, right?
So to me I consider myself a working leader. I think of myself as a team member. When I have team members, what I think they appreciate, but we don't really talk about it, but I think they appreciate the fact that we like to be in the details and in the trenches.
So if I have a patient - I actually used to stay up all night until we got results at two in the morning, six in the morning, and I would be on the team's WhatsApp after COVID days, like, “Where are we? What's going on?” And we would have like a virtual team's room and I would be in it every step of the way.
And to me, that's one of the benefits of a small biotech is you get to see and do and add value everywhere, being a working leader is so important. If I'm asking people to do X, Y, and Z, I should be able to do it.I've been really lucky that despite all the mistakes I've made, and despite being a first generation immigrant, and the first person in our family to be in a corporate America job, and I've done everything wrong you shouldn't do, I've never bought Christmas gifts for bosses. I've not, when people say, “speak up”, I actually thought they meant speak up even though I'm sitting in the back row and I spoke up and that wasn't a good thing, you know?
So the faux pas I've crossed every error that could and I'm still here, right? But I want to make it easy for people to know it's okay to be yourself. So to me at Rocket, the culture is I've made a lot of mistakes, and my goal is to never make the same one twice.
Let's talk about, to be on the forefront of innovation, people should really think out of the box. And it's okay. Let's celebrate the mistakes. We win as a team. We lose as a team. We learn as a team. When you started the conversation, you talked about elevate. Actually, elevate is the word that I had helped pick up as one of the Rocket values. So Rocket for values. It starts with the foundation of trust. When we talk about generosity, the core of generosity is really this: the most precious thing any one of us has is our time and our knowledge. So being generous with your time and your knowledge to support each other. It's not about the role and responsibility you have, but if your team member right and the left of you are struggling, supporting them is being generous.
So, generosity is our second value. Curiosity means lifelong learner. You can't be doing one and done gene therapy and have this promise of getting rid of rare diseases as a whole, but having a promise, a lofty promise of changing the human kindness as a whole, where genetic disease do not define your life and your loved one's life, requires us to be curious, requires us to be resilient and agile, right?
So constantly learning from each other, constantly making mistakes is so supportive. And I think if we all do that, we can elevate as a company, as an industry, as humanity. So those were the concepts. So to go back to it, my leadership style is to try my best every day to live to those values. How do I teach? How do I mentor? How do I help make sure others don't make the mistakes I made? How do I celebrate people that are at Rocket and what they do? But when they leave Rocket, there's so many people that I still have good fortune of hearing back from and get to mentor and guide them when they're starting their own companies and doing X, Y, and Z.
And so to me, the impact is exponential, that each person can have. So I'm really proud to be not just championing things for Rocket and the women at Rocket and diversity and equality here and equal pay and all these things is it's also the work I get to do with organizations like HBA and ARM organization where we're trying to make the entire cell and gene therapy industry.
Meg Escobosa: What a lovely philosophy for leadership and I love the values that are underpinning Rocket Pharmaceuticals' way of, culture and the organization. How different is it for you to be in a leadership role and how has your perspective changed as a result?
Kinnari Patel: It's very different. Being a leader of a private company is also very different than being a leader in a pharmaceutical where you're middle management or some sort of leadership role than being a leader that is trying to do hands-on work to move the science in the pipeline, to being publicly traded company officer.
So I think the layers of complexities are very different and unique and people would say certain things and I didn't appreciate what it meant. It's a lonely job, it's a complex job and it's a job that requires a whole life of yours. It's not a job. It's a lifestyle. It's a mission. Sometimes I wake up with a little night because I have dreams and I - certain things click for the better. Certain things make me so nervous, so I’m used to me waking up at two, three in the morning and writing things down so I don't forget those ideas or those lessons learned, right?
One of the things is this weekend. We had a really stressful meeting about something. We're trying to make some innovative things happen for patient treatment. And then I woke up Saturday morning at three in the morning and I wrote something down and then I wrote an email to everybody Saturday at work, like not everybody, but like the 10 people in the team.
I was like, “I have a solution. It came to me.” They're like, “How?” I'm like, “In my sleep!” Well, so they're like, “You're obsessed.” I'm like, “Probably. Yes, I'm obsessed.”
I do love what I do, but the leadership role at Rocket comes with a lot of responsibility because the decision I make is not just about me. It's not just about the company. It's not about just our shareholder either. Being one of the few cell and gene therapy companies that are still standing and trying to succeed at what we do, it feels like the responsibility is for the entire field.I'm lucky to have a CEO that is a great thought partner and we can think through stuff so it's a little less lonely cause at least we have each other to talk through a lot of the complex things, but it's it comes with the job. It comes with the territory. So I'm just saying it's complex and I didn't realize it till I was in the seat, how complex it is.
Meg Escobosa: And it's true. It's easy. You were very literally generous in the way that you lead and that you're in those team meetings, you're staying up till two, but the truth is there's way more going on for you that nobody else sees except for the CEO. A nd it's also, in the world of innovation and any problem solving, that lateral or passive thinking, you focus on the problem you're trying to solve. If you can't get to a solution, you have to walk away. But that passive, the brain is amazing and how it's continuing to work on this and it's waking you up at three in the morning with a solution. So you know, that's just part of also the innovation and creative process. So amazing that you have that ability. It's also not that common to be able to do it.
Kinnari Patel: It's probably not also healthy by day.
Meg Escobosa: Interrupting your sleep.
Kinnari Patel: It's a gift. I say it's a gift to find what you're passionate about so early in your career, and it's a gift to work in a field that allows you to do good and so much meaning absolutely and I'm fortunate that I have family that supports me, right?
And to have all three of them. Sometimes as I say this outlet I'm like, “I need to be more grateful and to win more points with my husband and my daughter.”
Meg Escobosa: But they know it yeah, I know it's true it never hurts to say “thank you”.
Kinnari Patel: Yeah, we have a great team at Rocket. It's not a company of one or two people. It's a company of 280 plus amazing people that care. And I think that's the thing. As a leader, everyone has a different leadership style. And to me, when somebody says, “Hey, is this person successful? Are they good?” And somebody called me from the company to hire Rocket team members saying, “Why did you let this person go? I heard you let them go.” I was like, “Here are their strengths, first and foremost, is that what you're looking for?” And if that's what you're looking for, they're going to thrive in that environment, right? Our environment is very different. We don't have a playbook. You have to know the science, you have to collaborate, you have to do certain things in certain ways.
And, the funky thing is when we're a company of 12 and one person got to do everything, they loved it. Now we're a company of 300, and they're like, “We're too big,” and I'm like, “What do you mean we're too big?” We're just, so it's all relative. Not everyone can thrive in every environment, in every cycle of a company.
So trying to find the people that have like-minded thoughts and like-minded plays, are willing to grow, develop, and keep up with agility, speed, and also be able to give back.
Meg Escobosa: Well, it's also, it's so interesting that is experience talking. That's also the company is evolving. The company's needs are evolving. When you look back over your career, are there some key milestones that, that you reflect on and realize, looking back, you may not have noticed it in the moment, what were some of those milestones that helped you prepare you for this particular role?
Kinnari Patel: At Rocket or in my life as a whole?
Meg Escobosa: I would say over the course of your career or your life. We're interested in all of it. All of it. Well, because you're a whole leader.
Kinnari Patel: I think I'll go back to this. My grandma, my mom, and my dad had this philosophy, my mom's mom. She always felt education and giving back to the community is more important than individual self, right?
So our life has always been about advancing knowledge and sharing knowledge like my mom wanted to be a teacher, right? That's what she was obsessed over. She would work in the farms in the morning to go to college and then help out so she could do a little bit of everything and be the only woman from her village to go to college, right?
So that's how I grew up in a lifestyle where, you know, my mom's like, “Just because you're a female doesn't mean you have less advantage than a male. You're going to make more mistakes and you're going to have to work four times harder to get there, but if you're willing to work hard, we're here to support you,” right?
So it was always about integrity. It was about trust and loyalty and it was about work ethics and being okay to roll with the punches, right? That's how I grew up. And then I think my defining moment was. I'm of Indian heritage, so my parents, of course, either you could be a doctor or a lawyer, and I didn't want to be a doctor, so when I got in, I did my MCATs, and I got into med school, and then I finally had enough guts to tell my dad, “I don't even like the sight of blood. I hated the cadaver, I didn't enjoy that experience. I'd rather be a pharmacist than a doctor.” I think my dad stopped talking to me for about a year, though.
So that was a little hard to go from his right hand boy, girl, I, the tomboy that used to watch football with him all the time and all the sports, to go from that and be like, “Oh my God, my dad is going to disown me because I won't be a doctor.” So that was, to me, hard, but I think doing the dual degree, I got to learn about genetic testing. I got to learn about rare diseases, applying pharmacy knowledge. And so I was very fortunate of that, plus my husband that I met who was, who saw so much more in me than I did in myself and it was having that combination really catapulted what I was doing and what I realized is I, all I had to do was be authentic to myself.
I couldn't emulate being somebody else. I was very fortunate that at Pfizer, when I was a high potential, they got me to go to two events. One was how to learn how to play golf so I can get better projects if I knew how to play golf. I’m horrible at it, but they paid for it, and then I got etiquette training. And so I learned how to eat caviar, but I'm telling them like, “I'm vegetarian. I'm never going to eat caviar.”
Meg Escobosa: I've never even heard of that. It's etiquette training.
Kinnari Patel: So I got all these like cool trainings along the way of what I should be doing. But I realized very early in my career is I needed to be myself so I can sleep well at night. So, learning one of the lessons of being authentic to myself and saying, “Yes, English is my third language. I can't write beautifully. I'll have grammar errors, but you know what, I'm going to make sure my team can write better than I can.” These are my flaws, but I'm going to make sure people around me have those strengths.
And what I succeed in my relentless, my passion that I won't give up, right?
Meg Escobosa: Problem solving, creativity
Kinnari Patel: Problem solving, crisis management, with crisis, I am at my A game, right? A+ game. So it's knowing yourself early on. I think there were examples and experiences that took me back in my career, which, and there were examples that have made things catapult forward, but what I've obsessed about is knowing and doing everything myself and learning at once. And if I learn it, see one, do one, teach one, is my philosophy. Every step of the way, whether I was in middle school teaching elementary school kids or high school teaching middle school, I got my, I almost got my TA certification, but I didn't get through the courses, but I was a system teacher for genetics and organic chemistry in college, right?
Meg Escobosa: Wow.
Kinnari Patel: And then I taught at Rutgers Pharmacy School and PCP Pharmacy School and other pharmacy school as a postdoc fellow and thereafter. So, the more I taught, the more I realized how much I needed to learn and get better.
The more I learned, the more I was able to make valuable decisions in healthcare in my area of passion. So, it's been an interesting cycle. So, I've been fortunate to start a postdoc fellowship program in R&D at BMS and then AZ and now at Rocket and internships. So, it's never been about just me.
And when I get done, it's never been a straight road. I've taken so many backstabs, right? To come to Rocket, I was like, “I'll take a pay cut. You want me to commute from DC by myself? I'll do it.” Why? I'm never going to get a chance to do something cool as this, money comes and goes. Title comes and goes. Those experiences and ability to sleep well knowing that you've done your best to make things better. That is what keeps me going. So I think for my life, that's been the key. For Rocket, the key successes has been just to starting the company in cell and gene therapy and monogenic diseases and working on diseases that no one else was able to figure out.
Just because Fanconi anemia is complex and researchers were researching over 20 years, right? One of the best decisions we made is there was a group, which is the NIH of Spain in Madrid, called CMAT, and there was a group at Fred Hatch. They were both researching Fanconi anemia for over 10 years and the Fanconi community, they were both like researching in parallel, but not collaboratively.
So, one of the best decisions at Rocket we made is we licensed both programs. And we said, how do we license both programs and how do we set up a contract so they can collaborate with each other so everyone succeeds, no matter which product we go with. And if we learn the best of both and we start a whole new product, everyone still succeeds, right?
That philosophy of collaboration, bringing innovator scientists together, I think, has been the hallmark of Rocket uniqueness from day one that academic communities really appreciated. Knowing that patients weren't after thought we were thinking about when we go to marketing or commercial launch, but we talked to patients and communities before even having an IND made a world of a difference and then transparently sharing what data we have, what hiccups we have, what, oh, we didn't get to the clinic when we wanted to, but it's gonna take us two more years. Keeping them informed in an honest, transparent way of the why as much as we can made a difference.
Deciding to go public in 2018, we were given an opportunity to go public through reverse merger. And all the data, we love data in our company, all the data says every reverse merger company fails, right? So we're like, “Okay, so we could do it the longer way.” The problem was this. I had my MBA and I was helping with the finance team. We had one finance person, at the time, and myself. How do we go public if we're all drug developers wanting to just do drug development? The easiest way to go public is reverse merger. So we have the least, right? So we said, “It's not about what's normal and what people do. What do we do based on the strengths we have?”
We decided to go public as a reverse merger. And fast forward, when I was at HBS professors, two of them are, they're still reaching out. We just haven't had time. They wanted to write a case study on Rocket, how it became the most successful reverse merger in healthcare. Which is pretty cool.
Philip Morgan uses it as a success story of reverse mergers, right? So I think that was important. And then the other big change for Rocket was we were working on ex vivo technology, which is basically we take a patient's cells, put the corrected missing gene in, and give it back to patients. So truly individualizes therapy.
All gene therapies were either CAR Ts, they all have one platform they work with, right? Essentially. And then we had an opportunity, we came across a disease and we said, “Oh, we want to work on the disease, but the technology we are familiar with doesn't work with it. AAV does.” So everyone said, you can't, you're not an AAV company.
So we said, “Why can't we?” We're drug developers. We're not technologists. We're not gene therapists. So we said, “We'll just bring a lot of experts around the table to augment what we don't know,” and then we, the pivotal moment, number three for WCAG was really when we decided to say, we're not a platform company, we're a platform agnostic company.
If the disease makes sense. The science makes sense. There's unmet medical need. We'll work with the technology that is available and de-risked from other companies and learn from it to make it available as a technology to make this drug have an option. So we became a cardiovascular AAV therapy and heme, non-oncology heme, ex vivalenti.
So to me, some of these were things that we were not afraid to do and shy to do. And that's what I really appreciate is we've been able to make some changes. Not because what's the norm and what's the data. But what makes sense because that's who we are, and how can we add the greatest value because of our uniqueness?
Meg Escobosa: Yeah, so you're not being limited by a certain definition of what kind of solutions or therapies you'll come up with. You're going to be open-minded to what's available in the market, what technology exists, and leverage it. Can we backtrack just a little bit about the success story of the reverse merger?
Can you look back and reflect on what you think led to the success so far.
Kinnari Patel: I think so far, what's led to success is, we've been so lucky to have investors that keep investing us from series. A investors are still in our docket of investors, right? When you have investors that believe in your long term story, but also we as a company have devoted a lot of time in talking about the science, the why we do what we do, the decisions we make.
And then when we have, you know, we've made mistakes. We've had faux pas, right? We've had misses on milestones, talking about those misses and owning what that miss meant individually, but then learning from that and how we're going to apply to the rest of our pipeline or rest of our future. I think that has led to an ecosystem where we're trusted by not just our patient communities and academic partners, which is really important, but also trusted by the investor community.
And I think that has led to the success that even last year, the markets in biotech and healthcare and cell and gene therapy have not been great, right? For three plus years now, everyone's expecting next year is going to turn things around. We don't know when it will turn around. Yeah, I actually think 2025 - I've coined it as a year of optimism across the board.I think it is a turning point for a Rocket for the industry and so many other things, but our success story has been whether we had $500,000 in our bank account, right? Or $500 million in the bank account? Our decision making structure is still about where does each dollar go and is it going towards a value creation for science for patients and therefore investors? I think that has helped us keep the relationship, the respect and the success of being a reverse merger company.
Meg Escobosa: Thank you for sharing that very helpful, very insightful and transparency obviously wins the day with your investors.
Kinnari Patel: Sometimes it's not easy to own things, but here's what we learned from it. We're working on it, we’re working on it.”
Meg Escobosa: Yeah. Well, congrats. And really, truly best of luck with the continued work. Kinnari tell me, I believe Rocket celebrates Rare Disease Day. Can you tell me what you have planned this year?
Kinnari Patel: Absolutely. So at Rocket, we're so fortunate that we're able to raise awareness for rare diseases. Not just in the US and the area we live in, but around the world. So we actually do two things. We do Rare Disease Day Light Up for Rare campaign. We started this about six years ago. And last year, I think over 120 monuments around the world lit up for rare diseases, including the Coliseum, to Empire State Building, always does it for us, to universities. And what we've heard overwhelmingly from the patient communities around the world is that one day these patients, no matter where they live, they feel like they're not alone, and they have hope.
So we do Rare Disease Day, Light Up for Rare, and Rare Disease Day celebration to give hope to these patients. That there's help, there's innovation, there's science, and there are people committed to making a difference. And for Rocket, every year we celebrate this in person, with at least individuals from the company, but also patient communities coming together.
And we've had good fortune from having Abbey Myers previously, who came up with the Rare Disease Orphan Drug Act, come and participate with us to all these amazing people. And this year, our team is putting together Rare Disease Day near Cranbury, New Jersey.
We're going to have patient voices. We're going to have challenges being discussed of the rare disease community. We're going to talk about genetic testing and the importance of genetic testing and what's available and what can be done more. And we're making this not just an in person event, but we have collaboration with Make-A-Wish Foundation and other organizations like NORD and other groups where we're going to make this available as a webcast for people to participate in.
But it's meant to be education, information, support system for rare diseases and what patients go through.
Thank you so much for asking about it, and it's important for us to just advocate for these patients everywhere in the world, with every disease that they face.
Meg Escobosa: It's a lovely gesture to represent and reflect the support for this community, so beautiful. Thank you for sharing and good luck with the event, and I hope people can participate.
Kinnari Patel: We're excited and I hope about 300 people will come in person, but we hope a lot more individuals around the globe get access to it. So thank you.
Meg Escobosa: Wonderful. Given your accomplishments and where you are, do you have any advice that you want to pass on to the next generation? We have a wonderful audience of young people, also career, people in your position, but people who are in school right now pursuing their education to join us in the field of health care, do you have any advice for them on how they should think about their opportunities and what's needed?
Kinnari Patel: I think the advice I would say is know who you are and what you love to do. Whatever passion you have, at the end of the day, even if you're pleasing your mom or dad, or whatever, you come back to that passion and when you're doing what you're passionate about, the impact you'll make and the happiness you'll achieve is going to be so much exponentially better that I think knowing that what you like and love to do is important.
Sometimes don't forget, though, to know that not everyone's fortunate to know that sorely. Know what you don't like. Know which environments you don't thrive in and ruling that out is just as important. So for me, retail pharmacy I did it. Everyone's like, “You're never going to like it.” I'm like, “But I'm not going to know unless I try.” So I tried to be a retail pharmacist for six months. Oh yeah, everyone was right. But now I know for myself, right? I try to be a clinical pharmacist. I try to, so keep trying things hands on yourself to understand what you like and don't like. That's number one.
Number two, be authentic to who you are, right? I would love to tell. Women out there, especially because it's a woman podcast of sort, right? What I want to tell women is the glass ceiling is still there. You're people that are breaking it. One of the coolest example, and I didn't appreciate how new was till people wrote about it after, right, because you don't realize anymore. This year for the first time at JP Morgan, Tuesday was designated as wear your favorite pink day, right? Biotech sisterhood day. And I was like, of course I wore my pink and I was in it. But I didn't realize it was the first time we've ever done that at JP Morgan, right?
Still in 2025, women are saying, “We can be good CEOs. We can be good innovators. We can - please invest in us,” right? To me, it is, the equality is still not there in every level, so that's one thing to remember. But second thing, what I say is, one of the women leaders early in my career that I had, and I learned so much from her, so I'm grateful to her, right?
But she said, “This is what it took me, so it has to take that or harder for the next person,” right? This is not pledging. We're not in a sorority. Our job is to make things easier for the next generation to do even better. So to me, it's remember, give back and give back in everything you've learned because you don't want this to be that much harder for people around you and behind you because to truly change the equation in healthcare specifically, the facts are this: most healthcare decisions are made by moms and women in the household, right? Most commercials out there are about guys and fishing and grandpas. Not the moms and the woman, right? We still have ways to go from commercial to internal, right?
But if the decision makers are women, for health care, a lot of times. Why aren't we actually supporting women to understand what's important to them? Why are we investing in caregivers to understand what's important to them and the patients themselves? So keep the north star of fighting for that.
And I would say the only other thing I would say is honestly, we can eradicate healthcare being a reason people pass away. Let it be car accidents and other, hopefully they'll get better. But not that we have capabilities that we have in advancement in the field that we have. Why should people leave with, live on with genetic diseases and fight hospital systems and in and out and all these things.
The reality is this, your or my generation. We can't make all this change ourselves. We need next few generations to be excited about health care, changing the health field, changing equality, equal opportunities to really get there. So to me, keep pursuing, keep following your passion and have a purpose that you live for, and don't be afraid to ask for help.
Meg Escobosa: Kinnari, thank you. That was so great. I appreciate your sharing your experience and your insights, your accomplishments are something to be so proud of and we will be cheering Rocket Pharmaceuticals from the sidelines. We really wish you the best and please come back and share more as you accomplish more. We really are interested in the journey you're on.
Kinnari Patel: Thank you so much. Really appreciate you amplifying the message for Rocket for health care and for women and the next generation. I think the work you do is great and more we can do to support everyone in this ecosystem. The better we're going to be. Thank you.
Meg Escobosa: Thank you.
Meg Escobosa: Thanks for joining us for the Game Changing Women of Healthcare, a production of The Krinsky Company. Today's episode was produced by Calvin Marty, Chelsea Ho Medina Sabich, Wendy Nielsen, and me, Meg Escobosa. This podcast is engineered, edited, mixed and scored by Calvin Marty. If you enjoy the show, please consider leaving a rating and review wherever you get your podcasts. It really does make a difference and share the show with your friends and colleagues. If you have any questions, comments, or guest suggestions, please email us at podcast@thekrinskyco.com and visit us on the web at www.thekrinskyco.com.
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