COO Liftoff With Rocket Pharmaceuticals' Kinnari Patel, PharmD - podcast episode cover

COO Liftoff With Rocket Pharmaceuticals' Kinnari Patel, PharmD

Jul 17, 202346 min
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Chief Operating Officer is arguably the toughest job in the emerging biopharma C-suite. HR, CMC, regulatory affairs, legal, PR, you name it, the COO touches it, and practically everything rolls up to her. This week's episode of the Business of Biotech brings us to a president and COO who's also a mom, a volunteer, and an advocate for diversity and equality in biopharma. That advocacy isn't just lip service, by the way. Here, Rocket Pharmaceuticals' Kinnari Patel, PharmD, shares on the real, practical, replicable steps Rocket takes to build a diverse and equitable work environment, how the fruits of that effort have proven important to the company's success, and where Rocket is taking its high-flying gene therapy act next.

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Transcript

Speaker 1

We recently asked a couple hundred of you , emerging biotech leaders , about your go-to sources of information when you face tough professional challenges . Your top response wasn't webinars , it wasn't scientific journals , it wasn't trade shows , it wasn't even consultants Far and away . You said you most often turned to your peers for trusted insight .

Enabling a community of peers is what the Business of Biotech podcast is all about . It's also what our new Business of Biotech newsletter is all about Peer driven content , no strings attached , delivered to your inbox once a month . Go to bioprocessonlinecom backslashbob to subscribe .

The Business of Biotech is produced by Bioprocessonline , part of the Life Science Connect community , with support from Citeva . Citeva also demonstrates its commitment to the leaders of new and emerging biopharma at Citevacom backslash emerging biotech Check that out .

Dr Kinnery Patel is president and chief operating officer at Clinical Stage Gene Therapy Company Rocket Pharmaceuticals , and I'm not going to assume that she never tires , but her attack of those dual roles and of her role as an advocate for women in biotech is seemingly tireless .

On today's episode of the Business of Biotech , dr Patel is here with me to discuss her professional roles at Rocket , whose pipeline constitutes a complex mix of AAV and LVV gene therapies for several rare and devastating inherited genetic diseases .

We'll also discuss the role she embraces as a woman in STEM advocate and a mom , the experiences with a who's who of big pharma companies that led her to Rocket , and how she maintains the altruism that led her to biopharma in the first place and hopefully get a grip on how she balances it all so seemingly effortlessly .

Dr Patel , it's an honor to have you on the show .

Speaker 2

Thank you so much for having me . I'm so excited to be here and to talk about things that are near and dear to my heart .

Speaker 1

Yeah , as am I . I'm excited to have you as well and I want to start sort of at the highest level with just a general question about why you , why you started inside , why science I mean you and I . So full disclosure for the audience , you and I .

Dr Patel and I were just chatting before we started recording and I learned that she has like six nieces and nephews who graduate with pharma and other medical degrees from Pitt . So I'm assuming it was in the blood . But you tell me why science ?

Speaker 2

You know , being a first generation American from that had parents come immigrate from India right , the passion was always about science . The passion from the beginning was I saw so many devastating diseases that took away loved ones around us and to me it was mind boggling . There has to be more . We can do this day and age .

So passion for science came very early from you know , working on different test tube experiments in like LA Merty School , and then really over time I realized the power of science and medicine could make a difference in humanity as a whole . So older , the older I got , the more I became even more motivated to pursue a career in science and make a difference .

Speaker 1

Yeah , yeah , that's outstanding . Plus , I'm not going to discount the fact that there was probably some family pressure .

Speaker 2

You look around like yeah , I mean either Indian culture , either you're going to be a doctor or you're going to be a lawyer , you know . So those were the two options being a tennis player . Other things were not the options .

Speaker 1

That's fantastic . You started out like from the outset , coming out of school sort of in what I would call the big leagues .

You worked at Sanofi , pfizer , roche , novartis , bms , astrazeneca Like I don't know , I'm running out of big league names to throw out there and then in 2016 , you joined Rocket Pharmaceuticals , which is like on the heels of its incorporation .

So I'm always intrigued when I talk with you know big pharma alumni who joined new and emerging biopharma opportunities about the motivation to do that . What was it that led you to make that jump ?

Speaker 2

Wow , I didn't realize there were so many companies I have . My career in pharmaceutical and biotech really started with the passion of making a difference in very disease patient lives and I was very fortunate to do a postdoc fellowship program at Pfizer and Johnson Johnson where I met some great leaders .

So my motivation in my career has always been follow great science and products that can make a difference in patient lives and follow great leaders you can learn from them .

So when Rocket Pharmaceutical was started by Dr Gaurav Shah and our chairman , dr Rod Wang both of them I had known separately in my life and thought the world of from not only how intelligent and science driven they were , but how kind they were . So to have people .

That's kind and in a seasoned and willing to mentor and develop people and allow them to be their best selves . So when I got a call from them saying hey , we're thinking about starting a company , will you join us ? Initially I laughed and I said listen , the smallest company I've been at which I loved was BMS , because it was only 26,000 team members .

Now you're telling me you're one . I'm like what are you ? So it took about six months to get there , but the science they were working on was so brilliant .

I mean , they told me that , after working on oncology transplant , hiv and other medications , they were developing therapies that can be one and done , curative therapies for patients with rare diseases that may not live to the age of five or 10 or 20 . And that was just an amazing opportunity that I couldn't give up .

Speaker 1

Yeah , was that gene and gene therapy and rare disease space new to you at the time ? I mean all that extensive experience in the big leagues . Did you work on gene therapy or rare diseases in any of those experiences ?

Speaker 2

I can't be in rare disease quite a bit at Novartis and at Breastome R , Scrib and AstraZeneca , but it was never in gene therapy . One of the products that I worked on which were new to innovation in early 2010 , was immunoconcology .

So I worked on your Royal N Updivo at BMS , taking up D Bo from an IND to heading towards a BLE filings , so that was the most innovative forefront I was part of .

So to do gene therapy and to do it where it was just really becoming something that everyone started talking about CAR-T , X-U-L-N teaching therapy and to be part of that forefront of innovation was really challenging , exciting , and I knew one thing was short . I will never be bored .

Speaker 1

For sure . Was there any apprehension ?

I mean , it's the human assumption that jumping from the securities of big companies and big pharma and successful roles , mind you , at those companies into a fledgling company that was looking for money I mean , these are non-revenue generating companies I can only imagine that there's got to be a little bit of apprehension and if so , how did you kind of overcome

that ?

Speaker 2

I was very nervous and , honestly , there were two or three things that made this all possible . One , my husband is just such a supportive person and he knows me so well and he said listen , you're always trying to fight for this , this , this .

Why not just be in a company where you can form a company culture to focus on things and patients and be able to be part of that storytelling or building something brand new that you , instead of trying to change everything around you ? That was great .

The second component was having companies like AstraZeneca and BMSA hey , try it , and if it doesn't work , you can always come back to us . It was a safety net . Worst case , I could always go back to big pharma , which was a great thing .

And the third thing was I knew that during this opportunity even if it was six months that I was going to try rocket and it didn't succeed the amount of learnings I would have . We're doing something different . As scary as it was , I felt like I didn't want to be comfortable in doing what I was doing .

So , it was a challenge worth taking and I had a family to support me to do that .

Speaker 1

Yeah , very , very good . When you joined in 2016 , did you come right on as president and COO or did you start in a different role ?

Speaker 2

No , you know , I I the CEO at the time was like hey , do you want to start as a president ? And I'm like I don't know what a president qualified . I'm like my as a woman leader . I've always been one of those individuals that said , if I haven't done something and done it well , I'm not going to take on the responsibility in the title .

So I said you know , what I can do is regulatory safety , quality compliance . So I started as a vice president of regulatory quality , safety and compliance at Rocket . It's not what normally people do and that's not what I'm advocating for individuals to do , but for me , experiences allowed me to have confidence to do something right .

Speaker 1

Yeah , yeah . And now I mean those experiences I'm sure have been added to your leadership as COO , which you know , I would argue , is probably one of the most challenging positions in particularly new and emerging biopharmals , because you cover so many bases .

I mean your , your , your role calls on you to oversee regulatory clinical execution , global program teams , alliance management , cmc , quality and development and , I'm sure , more right . I mean , those are the , those are the official bullet points , but I'm sure there's more .

So just give us , give us some insight in that role what the keys are to to embracing and , I guess , successfully executing on such a diverse range of responsibilities . That's a big , that's a big , unfair question to ask .

Speaker 2

No , I love the question and actually , you know , when we were going to go public in 2018 , in 2017 , mid 2017 , our CEO said hey , we're going to go public in the next six months . You should be the official CEO . And I'm like I don't know what the CEO does . What do you mean ? Ceo is like you're doing .

You know you're doing IT , hr , finance and everything else . That's the CEO job . You're doing everything anyways . You just don't have the title and the compensation for it . And that took me a bit back and I said listen , wait , we're a company of 10 individuals . I'm just doing things that need to be done in order for us to succeed .

Right , so I think that's the mind frame . What I realized when I started researching CEO is every CEO has a different background and you can really make the role . What is what you think is needed for the company and what your strengths are . So for me , I realized that my strength is I can integrate different functions really well .

My head works like I have a dummy on my head . Where any new information , I know what the risk and rewards and opportunities are . I know ongoing basis . I just can't turn off my brain from working that way . I go to a shop right , and I know what is the easiest steps number of steps you need to take to get to the front door Right .

That's how I think A to B is the most efficient way . So for CEO , for me , I just kept doing what I needed to . So initially I did HR IT . As we became a more established company with more clinical proof of concept , stronger investor base , more investor money , we started hiring experts that can do these jobs better than us .

So I think what I realized the success of CEO is you fill in the gaps where you need to to help make sure that in a biotech and pharmaceutical company , your products get to the patients that need it as fast as possible and the highest quality product possible . And I think when you do that , you define your own role .

So to me , the lessons learned if I had I have few colleagues that have reached out for my prior big firm I will say I'm thinking about going to become a CEO in biotech . What should I think about ? What should the role be ? I said look at your own authentic self right . What are the things you enjoy doing ?

What are the things that are your strengths from your prior learnings , and do those two , bridge well with the company's needs and if they do for 80% or 70% of the time , go for it , because you're going to be great at it .

Speaker 1

That's interesting and it's super insightful advice , dr Patel , because it's not . I love the point that you make about it not being necessarily a prescriptive role , so organizationally dependent . So I think the takeaway to do research on the needs of the company and that CEO , it's not like being a lead scientist where it's pretty prescriptive what you're working on .

So super insightful advice .

The other thing that strikes me about the role of COO at Rocket and the levels of responsibility that you have is that you see so much See the good , you see the bad , you see the ugly , you see the promising science , you see the scientific failures , you see the financial wins , you see the financial losses like you see it all , and I can only imagine that

that would be at times . As I said in my intro , you're seemingly tireless , but it seems like that might be something that would make one weary right Having access and exposure to all that .

And I read a story about an experience you had I failed to mention that you also worked for the FDA's Office of Warfarin Products Development and I read a story about an experience you had when you were there that left you wondering why candidates with promising early data would not be further developed . Because it might not .

That candidate might not make enough money to overcome its R&D costs , and that's you know . I mean , that's something that I think weighs on a lot of people in this industry . At the time when you're with the office of or from products and development , you are a young , energetic , aspiring scientist .

I can see where that sort of altruism would lead you to that conclusion . Like my God , I need to do something to further these candidates that are otherwise left by the wayside . Now that you've been in the industry for so many years , I see you maintaining that altruism and that energy and that expectation . How do you do that ?

Again , sort of an unfair question to ask , but what's kind of built into Dr Patel that enables you to continue the pursuit of that ideal ?

Speaker 2

When I did that rotation with FDA or from products , it was such an honor . I worked with Dr Marlene Hefner and she was a division director for years and she helped develop orphan drug regulations and bring them from US to EU , japan and other parts of the world .

I was in the fight , she was fighting , and then I met the patients and parents and the story you're talking about . I was devastated that if there is a drug available , why can't a patient have access if they need it and why is the revenue stopping somebody from getting access to a therapy that could save a person's life , especially a kid's life ?

This all comes into my idealism of making a difference in humanity comes from , honestly , my parents and my grandma .

No matter how much money they had or how much access to education or resources they had , they devoted their life to making their communities better every step of the way , and so I grew up with the notion that if you're a good person , just try to do two things right Be a good person and give back what you have to others and make a difference in this world

. So I grew up with those two philosophies every step of the way , and my husband is one of the kindest person in the world . So he said hey , if you're making a difference in all these kids' lives , let's make sure you have the support to do it . And he absolutely has been my anchor in the rock that has kept me going when days are tough .

Right , there are tough days , but I , on one hand , feel excited and humbled to do something that I love to do and have this family support to do it . That's one .

The other thing I do is I honestly believe that if we can seek cures , one-in-one potential cures for devastating diseases that people are born with , I mean , we are going to change the trajectory of healthcare and how patients live their life and be in a place where a patient's DNA that they're born with or damaged DNA doesn't define their life or their loved

one's life and caregiver's life . So , to be part of that , I just honestly remind myself on tough days that it's such an honor to do what I do and I feel so privileged and I think that's what keeps me going . And in a biotech world , you know , there's just so much energy and passion .

So we're lucky that at Rocket , so many like-minded people are around us that if one person has a bad day , the other person's passion and positivity kind of lifts you up and that makes a difference in having that culture of collaboration , family-oriented environment and just a support system we have for each other .

Speaker 1

Yeah , fantastic . Another recent story that I saw I don't know if it was , I think I saw it on LinkedIn . I don't know if it was on your post or someone posting about you , but it was this it was a picture of you on a television screen .

You're doing an interview , I think , for women in STEM , on like Cheddar News , and the picture was not like the picture that the television was like up on a you know like in an entertainment center , and there was a picture of your daughter in front of the television screen looking up at you and her arms were like wide open , right Like she was .

Her arms are wide open , like she wanted to embrace you on the on the screen and like as if she were rooting for you right In this interview . And it's struck me .

It's struck me as interesting and , I guess , inspirational a little bit , because you know there you are and there's your daughter looking on and cheering you on and it begs the question how your , I guess how personal it is for you to be a crusader for women in STEM and women in biofarm in particular .

Speaker 2

It's that picture . Oh my God , I . That picture is just something , one of the best memories I will have for my rest of my life . Right , and I am actually quite an introvert , I would love to just dive in work on the science , put pen to paper , and so TV interviews these types of things is not my comfort zone . Our CEO is amazing at it .

He's a Grammy nominated musician . So I always say , hey , you should be the face of rocket and I'll just do the back end things right .

But I think picture like that reminded me that , as uncomfortable as I am with my communication outwards , there are women that need role models and if I can serve as an inspiration or role model to you , can pursue your passion in your dream and you don't have to fit a particular mold right , you can just do what you love to do and succeed at it and make a

difference . And remember . I think that picture reminded me that no matter what I do at home or at work or in you know , debates and discussions I have on panels , on Congresses , there are other people looking up .

So I need to make sure that not only do I do the right thing for what's meaningful , for what I believe , but also be an inspiration , because we are still paving the path in the biotech healthcare industry .

There are not that many women leaders that have succeeded to an important jobs that can make a big difference in this industry , and I think that's a disservice to the healthcare industry .

So if there's anything I can do on sharing my lessons learned , giving hope or just saying how can I mentor you , develop you and be your executive sponsor to get at that , I would love to do that . It's something that we just need more of to be better as an industry leader .

Speaker 1

Yeah , I guess there are follow up questions I want to try to unpack in that statement . One of them would be like what experiences as a female executive in bio pharma have you , what have you experienced that have sort of underscored the importance of you embracing that role ?

And I'm not looking for , you know , I'm not looking to do an expose here on you know , potentially you know damaging experiences . I'm just curious , like in the daily course of your transition from you know through big pharma and into rocket and through even back into your academic days , what sort of inspires you to want to continue that advocacy ?

Speaker 2

I always feel like I learned from the best around me of what works really well and what doesn't . And what I saw were , you know , early in my career I saw that lot of women that were putting in two X the hours and effort just to get a name on a publication .

Instead of being the lead publishing person they were getting , they were just glad and grateful to have a name of their contribution on a publication . That's what I saw when I was in academics and I didn't understand that concept . I'm like you're doing all the work , you're doing twice the work .

Why are you just happy of getting a name on the on the paper , like , well , it's better than not getting recognized at all , right ? So I realized very early in pharmacy school when I was doing my genetic research and publications , that this was a problem and it's something that's a norm .

And I think when I initially started the pharmaceutical industry career , I was working hard to work on a filing . In my early days to work on a new drug getting to the market was like the most challenging , coolest thing a regulatory person could do , right ?

And I was really doing everything under the sun to show that I'm qualified and could have an opportunity to do this and one of my colleagues he was . He comes in . He's like , oh , I got assigned to this project . He's like is this what you wanted ? I'm like , when did that happen ?

I've been like working my behind the first six one , trying to show I can do ad value . And he's like , oh , I actually didn't want it , but at the poker night last night they were talking it'll be fun for me to be involved , it'll be good exposure for me . So they assigned me to it and I think that's kind of this experience is ahead .

And all of those experiences the good ones , where I got championed by women leaders and other executives that were mindful , intentionally providing opportunities , and organizations like HBA that were supporting how to communicate better , how to get sponsorship , how to get the projects that will help you showcase what you can do to the things that may not have been great

. What we've done at Rocket is incorporate all of those best practices here , so we let people's work speak for itself , and that's kind of been the passion at Rocket is hey , if you do , you get opportunities . Not because I don't know if you play poker or golf or football team that you're a fan of or you know cooking night . It doesn't matter .

Either way , you get opportunities because you deserve it and you can add value to the pipeline , or you're the future potential individual that can grow into an opportunity Right .

So that's been intentionally done at Rocket very part of the cultural reason , which has the best of everything we've seen and eliminated some of the things that we thought was challenging to provide equal opportunity to individuals with different backgrounds and diversity .

Speaker 1

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Yeah , two words that struck me in your response to that question mindfulness and intentionality . You know I have a lot of conversations with I shouldn't say a lot , because we're 160 , probably episodes into this podcast and a small percentage of them have been with , to your point , executives who are female in biopharma . So not a lot .

But we intentionally attempt to go down that road and in a lot of those conversations I have it's a conversation about championing and having the being a crusader . But when we drill down to what you're doing , that's intentional and mindful , to be that crusader and to be that champion , specifics are sometimes lacking .

Can you share , like , some thoughts on , like what specific forums , avenues , actions that women can take to support one another in bio , or maybe some of the you know some of that stuff that's going on at Rocket .

Speaker 2

Absolutely so . One of the things we saw I saw a research in early 2010 that showed that a lot of scientists are women disproportionate . Higher than 50% .

Scientists start off in the labs but they never make it to director or higher level because they're not able to communicate their scientific concept , because they're not exposed to opportunities to present their ideas in presentation skills right PowerPoint or verbal or papers , things along those lines .

So what we do at Rocket is , when individuals join at Rocket , they have opportunities to present in breakfast and lunch and learn opportunities of what they do , and it could be things that they do content device at Rocket or it could be something that they love doing . Like you know , we had during COVID .

We hosted cooking shows right , where people would just be in their kitchen and being able to verbalize what you love to do is a great way to just tangibly advance your communication skills and become visible as a leader , as a person , as a whole person , not just a person at work . So that's one thing we do .

The second thing we've been very mindful of in my lessons learned is , from the beginning , every six months , we look at everyone's compensation right and make sure there's equality and compensation based on work , contributions , titles , et cetera .

And this helps not just women and individuals with diverse backgrounds , but it also helps individuals with the longest tenure at the company . I don't know if you knew this , but individuals that have the longest tenure in a company often get paid the least in that role , right , because the new profits keep changing .

So what I saw in one of the Baker Pharmaceuticals I was working in supporting my functional leader is individuals that were director for 10 years were getting paid about 30 , 40% less because they weren't advocating with the market value of what existed in the current market .

So new hire that would come into director with even maybe less experience will get a higher compensation . So to me it didn't make sense . If you're in the company longer , you know , more you can add a greater impact . Why are you getting paid less ?

So what we try to do is , course , correct for individuals that have been here longer , because the market data of how you negotiate keeps changing , right , the inflation and everything else allows people to keep up to current . So us , every six months we look at everyone's compensation .

So people that have been here five years at this title and a contribution get paid same as the newest person that negotiated their compensation . So I think these are some of the things we intentionally do behind the scenes .

The other components we do is what we found is a lot of times when individuals don't understand the full context of what they're doing , why they're doing it , it doesn't allow them to be as impactful and have more barriers right Of being able to influence and bring their creative thought process to a problem or solution to a problem that others may not have thought

about . So at Rockets we developed gene therapy , drug dopamine training certification program . So every six months every new team member that joins Rocket gets to training in every single one of our products our science , our HR , our legal . How do you do contract negotiations ?

So they get a full training over two weeks and if they finish the quizzes they can have these . You know , impasse them , of course , which is relatively easy to do . You get the certificate that says that , even if you're a manufacturing associate or somebody in HR or in regulatory , you understand the full picture , what Rockets trying to accomplish .

When you understand the full picture , what you're trying to accomplish , your role , you can understand the work you do day to day , how it impacts the bigger picture . And what we found is when we do that .

It allows AP people to understand the context , ask the questions that they may not have been comfortable asking , or knowing and presenting the ideas that they have that they've not thought about presenting because they're like , hey , I'm too shy , maybe I don't know something somebody else does .

So there's subtle ways that we've kind of made an environment where we open dialogue to have more constructive , direct feedback and have the best ideas rise to the decision making .

Speaker 1

I am so happy with that response , dr Patel , because I alluded in the question I alluded to . Like you know , I try to get into these conversations on many occasions and there's sort of some soft , fluffy answers . That was super concrete . I mean , that's beautiful , like . Those are very specific examples and I love it .

When I think about your response , though , I think like and full disclosure . So Garav has been my guest on the show , so I know him a little bit right and I know his like , I know his leadership style and I fully get that he is an embracer of diversity and inclusion and equality .

But at the same time , some of the things that you just mentioned the opportunity to hone presentation skills , training programs and super specifically like compensation review you don't even a COO . You don't just go in and say , okay , this is how we're gonna do it .

We're gonna do these things , like , not without buy-in , both upstream and downstream , right and peripheral buy-in . And again , I know I'm not gonna say that's been easy for you , but I know that Garav probably hasn't been a stumbling block for you in that effort .

But I'm curious about your thoughts on how to solicit and win buy-in from your colleagues and , as I said , the folks perhaps upstream who need to sign off on some of these initiatives .

Speaker 2

Well , I am so lucky that Garo has been this amazing , inspirational leader . His philosophy is everyone has a zone of genius and our job is to make sure that we help them find it and make the most out of that zone of genius . So I think that's a very positive outlook . So he's allowed and enabled me to really bring these ideas and make it easier .

So I think , for individuals that are looking to get buy-in and to make their ideas be known , what I found is whether you're trying to get a buy-in idea to be bought in within your own team , your colleagues or your boss or boss's boss , you have to talk about the why .

First and foremost , I think , the value proposition of , for example , people are used to normally having org charts when they join the company . So when they join Rocket , they're like where's the org chart ? I wanna know who I report and who reports to whom . Well , at Rocket we don't have an org chart right . It's something every six months .

I'm asked and I keep coming with the rationale why we shouldn't put out an org chart . I'm sure it will change one day to make the company run more efficiently . We have accountability chart right . The thinking here is my articulation of this example is if we what do we need to know people do ? Right , it's what are they accountable for ?

What are the expertise that they have ? Which projects are they involved in ? What is their role and responsibility ? If you can find that , without knowing who they report to , what their titles are right , what's the value versus the downside ?

Right , if we have the best minds and if we really have the philosophy that it doesn't matter who you report to and what your title and what the hierarchy is . We want you to give your ideas and be equal to us . How better to do that than not to put out this barrier of titles and hierarchy ? Yes , it exists , but why have it as a prominent thing ?

So what we tell team members is when you're invited to a meeting , that means you have value to add . If you're in a meeting with Garo and you're a scientist or a director of VP , doesn't matter . You have just as much opportunity to speak your mind as Garo or I do . And that's how we've done a little subtle things to break down their barriers .

So in this proposal , every time we have new individuals that come and join a different personal organization saying just give it up . What's the , what's the permanent right , there's no harmony . But is there a different way we can do things , where we can cultivate our culture that shows collaboration , that shows oneness , right and unity ? These are little things .

So what I would tell individuals if you have an idea you want to pursue , provide the why , provide the vibe versus the alternative and what are the pros and cons associated with it . To your best of your ability and I think , at the minimum , even if your ideas aren't agreed to or supported , people know how you're thinking about it .

So to me , a lot of times it's not the answer , it's the reason behind the question and the problem that makes a difference in getting to a better solution . So that's kind of been the philosophy that I've talked about the why and the value proposition .

Speaker 1

Yeah , the why and the value proposition . So I want to kind of segue into an opportunity to talk about some of the work that Rocket is doing .

But I'm wondering if you can take a stab at making a connection between why this conversation that we're having about equity and inclusion and diversity is important to the North Star at Rocket Does that make sense , completely so .

Speaker 2

For us , the North Star is really we want to make as many therapies available to patients that need it around the world , so less patients suffer from devastating leptorhidic diseases . And it's not just the patient that suffer .

The diseases we work on are so severe it really takes the whole village of not just the parents , the grandparent , siblings , the entire village of their friend circle and everyone supporting them through this devastating disease being in and out of hospitalization or , unfortunately , kids passing away in their single digit years . So to eliminate that is our goal .

And if you think about caregivers , if you think about the population that's been impacted by the genetic disease we work on , these patients , these individuals that are impacted , are all over the world , with all kinds of different backgrounds , different barriers , different economic situations .

So to us , if the people we're treating are represented in the company that we have , it allows us to really make a thoughtful decision and make it a thoughtful and as easy as possible way for patients to get access to therapy . For example , we had a family from Middle East that came in for a clinical study in the US .

One of our team members , on his own , said you know , I know , they have dietary restrictions . This is the first time they're in America . In the US they're going to be scared .

So him and his kids baked all these cookies and bought all these groceries from their Middle Eastern store that they went to and bought all this so they can feel comfortable in their transition because they knew that they were about to embark on a gene therapy study .

And that's hard to begin with and let alone to do it in a new country where you don't speak the language or even understand the food options . So those kind of things you can train people to do that as part of their job . This team member did that because he's like I understand , because my background .

I understand the challenges when you move to a different country you go through and what little things can make a big difference in overcoming this hurdle . So to us it's representing our science , where it's going to make an impact , and having that representation of women leaderships .

If you think about it , most of the you know there were surveys out there and I'm blanking on the percent , but most of the women in the house are actually the one that go to pharmacy and take even their husbands and parents , grandparents and kids to the doctors .

Right If case , having more women in different decision making paradigm of science , of data , of drug development , having their perspective is so valuable because they'll know how , what is important to the patients that need it and the caregivers . As a caregiver and as a scientist , they can bridge that much better .

And I think that diversity of thought process they bring to the table and the questions they ask if this was my child , if this was my loved one , what would I do differently ? Right , it kind of makes a big difference . It seems subtle , but it makes such a big difference in how we do what we do . So when days get tough , I look at my daughter .

I'm like if she was sick , I mean you , the mountains I would climb through and make a therapy available for her . If I do that for her , other kids need that opportunity , right , and other individuals are champions . So that's kind of the philosophy we have at Rocket .

Speaker 1

Yeah , I was going to ask you how your role as mom influences your role as COO at Rocket , but I think you , just you just answered that beautifully , yeah .

Speaker 2

People thought once I had a kid . They're like oh you're , you're going to forget the rocket and pursuing rare diseases . I came back after my my having the baby , and even more motivated and more passionate by doing this faster , better and for more diseases , for more patients .

Speaker 1

Yeah , yeah . So that's a that's a perfect segue into the diseases and the patients that the rockets serve and give us . I want to give you an opportunity , before we wrap up here , to share with us what rockets working on and and maybe give us an update on where the company is in terms of its clinical progress .

Speaker 2

Definitely so . At Rocket we're seeking gene therapy cures . We're trying to advance a very integrated pipeline through two different types of technology in gene therapy .

One technology ex-revolentees where we use patients own cell putting the corrected gene of interest and give it back to the patient so talk about individualized medicine to the core patient gets their own cells with the corrected gene back to them .

The other technology we use , aav , is an ex-revolute program where we infuse the corrected gene or the missing or the damaged gene back to the patient .

In either way , what we're doing is we have a pipeline that's quite robust of him or of blood disorders , and we have three programs in the blood disorders and then we have three programs in the cardiac diseases and so our pipeline is really focused on heme , which , phanconiumemia , is a rare disease that's . Actually .

We've already finished our phase one to global study across US , europe and UK .

We're heading end of this year to the FDA and EMA to get this drug approved because there are hundreds of patients that are waiting for this therapy and there have been decades of research done to support how gene therapy could be a really great treatment option for these patients with devastating disease .

The second one is you know , everyone at Rocket champions has their own favorite . So the second one is LED one leukocyte adhesion deficiency . This is a program that I've championed from beginning , from licensing it to now we're about to submit this to the FDA for approval .

This disease unfortunately two thirds of the kids die by the age of two , and what we've shown in the nine kids we've treated is all nine kids . Not only are they alive , but they're thriving . They're living normal kids' life of going to daycare , no more hospitalization , antibiotic needs , et cetera .

So what you see is a drastic change from before to after it and we're really passionate about bringing this therapy to patients because it's really pursuing excellence in science . And the second hematology disease we're working on is pyruvate kinesthetfficiency . So , as you know , normal hemoglobin for us is around 12 .

And that means we have enough oxygen that goes around the body so we can do what we need to do daily . In PKD's severe disease , unfortunately , the patients were treating their hemoglobin levels between six to eight .

They need transfusions just to keep going day to day and even with the transfusion dependencies that they have , unfortunately they're not able to live a normal life . And what we've done is we've treated patients , and we've treated three patients to date , two of them . We've had two years data .

Their hemoglobin went from six to eight at baseline to normal 12 to 14 range and these individuals are transfusion independent , getting a job and going back to a life that they had never had before , which is remarkable . So we're excited about our heme pipeline going to the patients across the US and Europe . In cardiovascular gene therapy .

For cardiovascular had been tried so many times before and unfortunately the science is complicated for gene therapy to begin with , and diseases like heart disease are complicated . For the first time we've been able to solve that equation . So for Danone disease , this is a disease heart disease where unfortunately young boys get diagnosed at the age of eight to 12 .

And unfortunately , if they don't get a heart transplant by the age of 1920 , they pass away . So talk about devastation in a whole different way . This cardiac disease also impacts females and females pass away , unfortunately in the mid 30s . So there is just as much devastation in males and females and truly one of the worst heart disease up there .

What we've shown in patients we've treated is these patients , after gene therapy , are going into their 20s without needing a transplant and going to college . And one of them got his own apartment . This other kid wasn't able to trick or treat .

We found out from a mom that , hey , last year he needed to be put into a wagon and just taken from a house to house and few houses just to participate as a normal kid .

This time , after the two hour time I guess six to eight tick or treating timeslot that they had in the township expired and he was running door to door and didn't want to give up tick or treating all night long because he was like I can live a normal kid's life . And that's the transformation we've seen .

We have other two programs that are also going to the clinic . One of them is in a clinic called Pegasus , pkp2 , which is another devastating cardiac disease and a BAC3 . So what our goal is is this is our starting point Just as many programs that are publicly known , which are six . We have just as many programs as ideas and discoveries .

And what we found at Rocket is , if we do drugged up inefficiently , if we follow science , if we've learned lessons learned from ourselves but other companies and other investigators around the world that are brilliant minds we get to work with and collaborate with , we can bring an idea to an actual drug product that can go to patient in about two years and then ,

for the first time , we're seeing for LED1 , we went to clinic for the first time in patients in 2018 , 2019 early timeframe . Now we're taking it all ready to commercialization . The drug document takes 15 to 20 years .

We're able to consolidate that with using the best practices of following the science , focusing on patients and medical needs , having patient feedback involved from day one in designing the right study that would be meaningful for them , and also the physicians that treat these diseases , getting their voices to be heard of what's important to them .

So I think when we do this in a collaborative , integrated fashion , we're able to accelerate drug development , especially working with FDA and EMA transparently every step of the way . So we hope that what we've done in these six programs we can bring some repeat for more diseases and help hundreds of thousands of patients around the world .

So , as you see , I can keep going all the time because I love what we do at Rocket and I think we can truly be a transport transformation in healthcare .

Speaker 1

Yeah , that's fantastic work and , as you're talking and sharing some of those stories , I asked you about your perception as a mom and COO at Rocket .

I'm a dad and , as you're telling these stories , like I'm feeling the same thing you're feeling , I'm feeling the value You're very compelling and I appreciate the work that you're doing as an advocate for women in bio as well .

I mean , it's like I said , sometimes these are tricky conversations to have , but you put a lot of concrete examples behind that advocacy and I like that . So what haven't I asked you , dr Patel , that I should have asked you or that you feel is part of the story that I haven't given you an opportunity to tell ?

Speaker 2

Well , I think you've covered all the components . I think , honestly , for us , it's just we chip away at good science and collaboration and teamwork , and I think the only additional thing I would add is , for Rocket , for what we do . It's not just about what we do , but how we do make a difference , right ?

Because if you're trying to create a legacy , if you're trying to get next generation of science to be developed and passionate to make a difference in healthcare , and if you're trying to be role models , the what is gonna matter but also the how's gonna matter even more .

So I think for Rocket , our value is really about giving trust and informing that trusted relationship from beginning right , having generosity and curiosity . If we're generous with our not just knowledge , our time , our energy and our passion and bring our whole selves to work and everything we do , I think we can make a difference .

But no matter how much you feel like you know , being humble and being curious to say , I know this , but here are all the things I don't know , and it's a puzzle of expertise that we need together .

I think that puzzle piece is what's made Rocket really special is , while we have some brilliant minds like Garo and others and David Riggs and other scientists that are working in this company , right , the fact is , every step of the way they're like I know this , but I don't know X , y and Z , and we learn from each other continuously , and I think if we

continuously have those conversations based on generosity , curiosity and the foundation of trust , we can elevate each individual person working at Rocket , the therapies we work on and the ecosystem . And so , to us , it's all about lifting up what we do and how we do around the world .

And so , to me , I think that's the take home message I would give to individuals follow your passion , but how you follow it is gonna matter If you do it collaborative versus sharp elbows , right , it differentiates how much difference you can really make in your whole life , not just for that one moment in time .

Speaker 1

Yeah , that's a fitting place to end , Dr Patel . I appreciate it . I really enjoyed having you on the show . It's been a great conversation .

Speaker 2

Thank you so much , really appreciate being able to talk about these things and thank you for really caring about how we do things that we do at Biotech Science and in Advocacy . We really appreciate your support in this .

Speaker 1

For sure . Yeah , so that's Rocket Pharmaceuticals President and COO , dr Kennery Patel . I'm Matt Pilder and this is the Business of Biotech . We're produced by Bioprocess Online and sponsored by CITIVA , whose support of new and emerging biopharma companies is on full display . It's ITIVAcom backslash emerging biotech .

If you like listening in on conversations like this one with Dr Patel , subscribe to the Business of Biotech podcast and sign up for our newsletter at bioprocessonlinecom backslashbob . Also , be sure to leave us a review , let us know how we're doing and , as always , thanks for listening . Country music .

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