Coming Together for the Nano-rare Patient
hi I'm kierden one of the producers of the patient empowerment program podcast as this series audience has grown tremendously over the past year we decided to bring back one of our highest rated episodes and one of my favorites for everyone to hear this aired back in October of 2022. it's called coming together for the Nano rare patient with John maraganore when Dr Stan crook ran ionis and Dr John maraganori ran an island they were partners that turn into Rivals and not always friendly ones as they dedicated a pursuit to an entirely new therapeutic space RNA targeted drug Discovery and development since then the two drug Discovery Titans have set aside their differences and come together for one reason helping patients so sit back relax and enjoy a conversation between two Biotech Industry leaders and this revived episode of the patient empowerment program our podcast is proudly sponsored by a nylon Pharmaceuticals since 2002 a nylon has worked to Pioneer rnai Therapeutics an Innovative new class of medicines that silence the genes that cause disease and in doing so help people live longer healthier lives [Music] I'm Stan crook welcome to the end alarm podcast Series today I'm honored to welcome Dr John Morgan Oreos Our Guest for most of our audience John needs no introduction John is one of the best known and most widely admired CEOs in the history of biotechnology and though John and I were on occasion directly competitive account myself as one who admires John immensely until late last year John was the founder and CEO of Ellen Island CEO he led the conversion of s-i-rnas from fascinating cell biology to a powerful platform for drug discovery in addition to all the business successes he had he has made important contributions to the to the science and he's also been very broadly involved in in the senior leadership of our industry and served as a mentor to many uh John grew up in Chicago and completed his undergraduate studies and PhD at the University of Chicago in 1986 and immediately entered the the industry first uh being a scientist at Simon genetics and then moving on to Biogen where once again he demonstrated his his capabilities in advance steadily and then on to Millennium and again the same pattern that's typical of somebody who's so talented and left Millennium as vice president of market and business development and in 2002 he became the founding CEO of Alan Island and and the rest as they say is history today John serves on a number of boards of directors is involved in several investment funds in continues to play an important role in the industry and and continues to serve as a mentor to to many in the industry John has been a colleague he's also been a very tough competitor and a friend for good many years so it's a real pleasure to welcome John margonori today John it's uh it's it's good to see you it's great to see you Stan and thanks for all those kind words you are you are a um an amazing colleague and friend and also a fierce competitor yeah yeah we we'll we'll try not to dwell on that too much here John okay so you know John I I know your your biographical history well but what I realized is I was getting ready for this that I really don't know much about your background uh I know you your parents were Greek what what sort of family do you come from well you know my father was a physician and my mother was a nurse she stopped you know being a nurse when you know we were born so she can raise the kids but they were immigrants that came to uh Chicago from Greece and um brought us up in a typical uh you know Greek American household and you know we ate well of course and um you know we were motivated by our by my by my father mostly and and my mother to um you know keep trying harder and harder and working harder but you know my father drove my passion for science uh and medicine and um my mother I credit her with giving me the gift of optimism which has been pretty helpful as a CEO for sure in biotechnology when you're pioneering an entirely new technology if you're not optimistic you don't even try right you you got it you got it very key yeah so um I'm interested in in when you realize that that you were interested in not just basic research but research applied to Bringing Therapeutics to to patients yeah I mean I always love science Dan I I you know was the typical nerd who had the chemistry set the biology set and did all that stuff and it was my passion for for sure and when I went to the University of Chicago I I um entered a research lab um you know in my sophomore year and started working on you know after school hours and on the weekends and basically doing basic research on on structure function of uh proteins and enzymology and I just loved it I just enjoyed being in a laboratory and and um doing things it it always occurred to me when you were doing science that you know you're doing something that nobody else in the world is really doing and you're finding something out um you know in your in your work that could illuminate a whole um you know level of understanding scientifically so I I just enjoyed that um completely but you know in um in 1986 when I when I got my PhD I went to the Uptown company um and I went there because my thesis advisor was recruited to the option company to basically become under early scientists and I did a postdoc at upjohn which really illuminated for me the power of the biotechnology and pharmaceutical industry working together you know in a team-like manner to basically identify you know potential therapies or cures and it really became my um uh my calling at that point in time to stay in the industry and to be driven by what the industry can do it is a it is an enormously complex and joyous experience that too few people get to experience right you're right absolutely right so I I know that you you grew and you grew at both Biogen and and Millennium and there are two companies that at least from the outside seem very different to me uh how would you how would you describe your experience in those two places and what were the big take-home things that put you ready for for doing being so successful on Ireland yeah well they were really very two very very different companies of course baijin's one of those one of those early Pioneers in the biotechnology industry one of the admired still independent um you know biotechs and I went there in in 1987 um and um and went there as a scientist bench scientist and began doing some early work initially in the HIV field when HIV was just getting up and starting I also discovered a drug advision that ultimately made it to the market by valaruden which is a anticoagulant direct Ramen inhibitor and um at Biogen I learned about I really learned about drug Discovery because I I took this invention I was the program leader I led the multi-disciplinary team all the way into phase three clinical development and then in 1994 we decided to stop the development of that program with the phase three trial ended up with mixed results and it was a very important important learning experience for me the drug ended up getting approved in the hands of another company but for me to actually lead that program and do the drug development was really one of the funnest things I've ever done to this day I then transitioned at baijin into the business side and became the head of Business Development advisor for the last three years that I I stayed at bojang and at Biogen Stan you may remember Jim Vincent I I really benefited from that person you know me uh May arrest in peace but Jim was a notorious leader in the industry because of his stubbornness and his thickheadedness and but he did teach me about the critical nature of just having a very high bar for everything you do and the the management skills I learned from Jim um to this day I I think were really very helpful but then I went to Millennium in 1997 and work for a great Visionary leader Mark Levin one of the you know biggest thinkers biggest thinking leaders I've ever met and worked for very colorful individual as you as you know and um you know Millennium was a company that was driven off of a big Vision a big idea and I learned the the the the benefits and the challenges of working in a company under that type of uh that type of roof but really was a fantastic experience of course so we're focused on the genome and solving the genome and helping make drugs out of out of genomic research well it's fascinating experience and very different from mine of course I suppose that you would probably agree that that for the last 20 years you've been a a Dream Merchant that you've that you've that what you did was conceive a dream and sell the potential of that dream and then convert that to you know the daylight the reality of actually doing it talk to me a bit about how that felt yeah well I mean I I uh you know I I made the decision in in 19 or 2002 to to leave Millennium to go start almalum as the founding as the founding CEO and you know Stan I um you know at the time I had to say I was very happy a millennium I I would all often get you know phone calls to go be a CEO but I was really very happy at Millennium I love working with Mark Levin he was a tremendous leader and a friend but I I became convinced as as you know the recruiters and you know people like Phil sharp and other people were trying to persuade me to go to almylum I became convinced that if we're able to understand how to make drugs out of these small interfering rnas that that I could actually be involved in helping create a whole new class of medicines and of course at the same time I was looking at the scientific progress that was being done at ionis and and you know ultimately Al Milam stood on the shoulders of ionis to be able to advance the medicines that we did but you know there was a foundation there of Reason to Believe In in developing very high impact medicines and so I had to take the plunge I I remember I I was just getting married the same year I talked to my fiance I said look I think this is going to be the right thing to to do you know obviously you know she she was very happy with me doing the transition along with getting married the same very year uh in fact my my offer letter for the job was sent to my honeymoon suite and Mauritius um and uh I was pleased to accept it um but I really believed that with album Islands technology and what the founders have put together that we could really create a whole new drug class and I I really believe that that joining the company at that time would be um you know a remarkable Endeavor and it turned out to be the case and and of course you focused meaningfully on rare diseases um your pipeline of course is much larger than just rare diseases what drove you into the rare disease space John yeah well it's driven by the patient uh stand first and foremost I mean the the the disease burden for people living with the rare diseases is enormous and at the same time there is incredible genetic data that really shines a light on how science can solve the problem and so when you have this um situation patient with a rare disease combined with the genetic understanding of how you can treat it and then you have a technology that can be applied to it there's nothing else you can do then go down that path it it it it's a Guiding Light it's a clear direction of travel that um you know you really have to take on and we're really proud we brought three rare disease medicines to the market and we have others in registration right now uh and in late stage development but the impact that we've been able to have in some of these rare diseases is really um you know to me one of the greatest Thrills and joys of the elm Island story for sure yeah um it is a powerful way to to to be to do work and do and to feel good about the work you do and appreciate it outside the industry I'm sure you would agree totally agree totally great we hope you're enjoying program podcast provides support to our podcast listeners the best way that we can there's no better way for us to do that than to ask you directly do you have questions you want to ask Stan crook Stan will be taking questions directly from you and other podcast listeners and dedicating an entire episode towards answering your questions AMA style if you're a nano rare disease patient family member friend physician rare disease Advocate or you just enjoy the podcast we want to hear questions from you please don't be shy all questions are important and may end up helping other listeners so don't miss a great opportunity to get your questions answered by the patient empowerment program host CEO of enlarum and the father anti-sense technology himself Dr Stan crook to submit a question for the upcoming q a episode email podcast at nlorum.org that's podcast at nlorem.org with the subject line podcast question if you wish to be identified mention your name in the email if not we'll keep your submission Anonymous we can't wait to hear from you now back to the episode [Music] well I remember um uh sitting in my office in Carlsbad and you and and Barry green showing up um looking for a license as I recall at that time there were a lot of other sir would-be companies and we were entertaining proposals from from all of them and uh and and then I met you and Barry and I said I don't know whether sirna is ever going to be a drug but if it's going to be a drug it will be with these guys and so we ended up doing that license uh and and then from there of course we collaborated on a large on a large number absolutely including uh co-founding and co-funding uh regulus of micro RNA company so we had a long positive experience even though um we were seeing in in in the rest of the world as as direct competitors in in terms of the technology space um but um you know I I do think that that working together uh helped us both achieve the ends that we sought to in to receive to do would you say that too absolutely Stan I mean the deal that we did back in 2004 and I'll I'll never forget flying over to Carlsbad and visiting with you and Lynn and and and putting that Arrangement together um was frankly one of the smartest things that we ever did at Elm Island because it created and I think also for ionis it created this um I like to call it you know Pax oligo uh like the Pax Romana that occurred but this this sense of of peace between these two technology competitors but frankly to technology pioneers and it also created this remarkable Synergy between the companies that allowed us to do some really good science together we had very frequent engage engagements with each other as you know we started the company together as you mentioned we also found ways in which we could avoid you know competition with each other on specific programs which was smart and and a clever thing to do um but it really was an important arrangement for sure it you know for us to have tried to find a way around the remarkable um you know accomplishments that ionis had done from an intellectual property standpoint would have cost us years and hundreds of millions of dollars to do it so it it made from a business standpoint it made a lot of sense and then at the same time I think ionis had a partner in the rnai space that they can count on and um and benefit from I mean ionis uh benefited economically from our success as well which is really terrific so it was a good arrangement and it was one that I'm really proud of at the end of the day SMI and so we did all all kinds of great stuff together and then we had a couple of drugs that were directly competitive and we discovered uh that we're both uh immediately competitive and we had a falling out uh and um and um you know and each of us has his own perspective about that but we didn't talk to each other for for a few years and as I thought about the patients that we're trying to serve at an alarm and I realized that I need all the help I could get and you could be an incredibly helpful uh person for these patients and and it made no sense to hang on to whatever bitterness I felt and but I was very very worried when I wrote that email to you asking you if you'd like to talk and you were immediately gracious um so I I know how it felt for my end what about you I'll tell you Stan I I I never ever um stopped admiring uh you um and and you know that and I also was watching the Ann lorem story from from afar from the east coast and really thought it was powerful um you know in the talmud there's a phrase whoever says a single life saves the whole world and it's a phrase that really comes to mind when I think about what you did in this new effort with enlarum because the power of of of of treating just a single patient with the disease with the technology that can address it is just is just awe-inspiring and and and so I was seeing all this and I was excited about what you were doing so when you reached out I I I I was so happy to respond and and um and get back to you and listen when when when we had this competitive period of two three years together um you know we ultimately got back together so to me it reminded me of a big brother and a younger brother they get a fight at the they get into a fight and but they still come together at the end because they're family and I really felt the same way as uh We've now been working together on in Laurel well I think we did come together uh around a family and the family is the nanor rare patient exactly and and in the end it's vital to these patients that the two of us participate in this because we're the people who created this opportunity and so it was certainly well I'm I'm embarrassed that I waited as long as I did and you've been just extraordinarily helpful already and and and and so um why don't we spend some time now talking about in lorem and the patients and and and in the model that that we're we're proceeding with which is a non-profit model and and how you see all that potentially unfolding yeah well listen I think I think the the starting with the the the the power of the technology the anti says technology and and maybe other technologies that come into it in the future but being able to um selectively design a medicine for a a patient uh or a small very small group of patients with a very discrete um genetic uh defect is um incredibly powerful being able to deliver that achieve a desired therapeutic effect do it safely and then on top of it have the regulatory framework the the framework from the FDA that enables all that that's not trivial um you know the FDA obviously had to think a lot about how does it enable the advancement of these type of medicines for for patients so one aspect of it to me is the technology which is really oppressive and and the regulatory framework for the technology the other aspect of it is is really the of course the patient and the ability of giving hope to a patient with a nanowrare disease at a family that surrounds that patient as well and then frankly the generations of of other patients with nanow rare diseases who can see what we can do one patient at a time it is a very powerful thing and it really does go back to that top ludic quotation that I gave a moment ago about how you can really save a world of virtue of saving just one life you bet and for me it's very much like returning to the practice of medicine which I still miss because it is that intimate experience of one patient one family one hope at a time and um and of course I'm looking forward to your involvement and getting you more involved with some of the patients and families and so on and we've both been Trailblazers we've both taken an opportunity that was a blank piece of paper and wrote a textbook and we both then took steps that that meant we were always in Uncharted Territory and the the name that Jefferson gave to the Lewis and Clark expedition has always been something I admire a lot and he called it the core of Discovery and uh I think of in Laura Mountain Island and ionis as cores of Discovery focused on on the inner landscape of health and disease and and and helping patients and um and and so it's a great it's a great uh tremendous value to these patients to be able to have you on the team and helping us uh uh do the things we need to do and I firmly believe that the next technology that will be ready to to to take on this task and in in industrialize this task so that we have quality at every step and and scale will be s-i-rnas uh yeah and and so it also made sense from my point of view that we would that we would bring the two people together who knew those Technologies better than anyone else so that when the technology had makes one more step or two then our patients could benefit from that and obviously even though you've retired uh from Allen Island uh this this collaboration extends to Allen Island now and there is no better organization to discover an sirna and develop it in Allen Island and you know I don't know exactly when that's going to happen but it will happen that's the next thing yeah it will happen it will happen and and Stan that it I couldn't agree with you more in terms of how you see it all coming together and I also to your point about being on the frontiers of Discovery you know doing it together with the with the with a colleague a brother an older brother a younger brother whatever the case might be is just that much more powerful at the end and I and I think that we can bring um ASO and and srna Technologies together help this uh amazingly important cause um and and help first and foremost his patients with these nanow rare diseases well I uh welcome you to all that and I guess the final comment that I'll make and leave it open for you to sort of finish is I believe we've made great progress at in Lauren but what we have left in front of us is an enormous task and one of the biggest questions that we we must answer is a non-profit model like we're developing sustainable yeah as you thought about in Lauren how did you think about solving that and improving indeed that it it is sustainable industrializable and scalable well I think I think it is sustainable Stan and and I think you're beginning to prove that it is one of the ways that it it you create sustainability is by creating relationships with other companies whether it's the AL nylons of the world but also the clinical trial networks of the world and the and other stakeholders in the system then work with patient groups that that obviously have a vested interest in advancing medicines for nanor diseases so these are all the different communities that that together we can create and then with the generosity of philanthropy and others that could contribute we can do this in a non-profit Manner and and ultimately deliver cures for for patients that otherwise have no hope they really have no hope they are hopeless and and you're right uh hope is a powerful powerful uh thing to lose and an important thing to recover not just for the patient but for the family so absolutely right it's been a great pleasure chatting with you and I I want to end then giving you the floor anything that you would like to say to the community that's interested in in what we're doing banana rare patients yeah well I would just say this I I I I hope that people take a look at what's already been done within Lauren but then also think about the power of enlarum going forward I I have no uh doubt that as people get to understand uh the stories of of uh patients that have been impacted already by and Lauren and then the the framework that's been put together to help patients going forward they will truly understand the impact and potential power of of this approach and so I really encourage everybody out there to take a really close look um this is this is a remarkable effort here to help a patient community in a high impact way that is what we are all about as a biotechnology industry that's all what we're all about is scientists and Physicians and it's something which of course um you know Stan you have done a brilliant job I've been leading thus far and I'm happy to be on the on the boat with you this time and look forward to many years of working together I do too and I think it is showing the True Heart of our industry and I know we share that passion that heartfelt passion to see people who are suffering be better yeah agreed thanks so much John for joining us and it's been a great privilege to know you all these years and to get to know you a little better in this conversation as well terrific great Stan thank you so much our podcast is proudly sponsored by a nylon Pharmaceuticals since 2002 an island has worked to Pioneer rnai Therapeutics an Innovative new class of medicines that silence the genes that cause disease and in doing so help people live longer healthier lives foreign [Music] is a non-profit committed to discovering and providing personalized experimental treatments for free for life to patients with genetic diseases that affect one to Thirty patients worldwide referred to by and Lauren as Nano rare many of these patients progress and die without ever achieving a diagnosis this is where an alarm comes in they do The Impossible by providing hope and for those that they can help free lifetime treatment for more information about nlorum or today's episode visit nlorum.org any questions can be sent into podcast at nlorum.org search nlorum on Twitter Instagram YouTube LinkedIn and Facebook to connect with us this video is hosted by Dr Stan Crook and produced with the help of the following professionals thank you for watching
2023-05-12 14:37
