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WSCS 2015: Leading Nations: Development Strategies For Regenerative Medicine

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WSCS 2015: Leading Nations: Development Strategies For Regenerative Medicine

This Panel Of Global Leaders In The Regenerative Medicine Field From China, The Uk, Israel, Canada, And Switzerland Will Share Their Insights On The Strategies That Have Been Most Successful For Advancing The Field To Date, And On The Strategies That Are Most Promising For 2016 And Beyond.

Video Transcript

MODERATOR

► MICHAEL N. DRUCKMAN, HOGAN LOVELLS



SPEAKERS

► YIJIA LI, PHD, YUNNAN SHUNXI STEM CELL AND REGENERATIVE MEDICINE RESEARCH CENTER

► MICHAEL H. MAY, PHD, CENTRE FOR COMMERCIALIZATION OF REGENERATIVE MEDICINE (CCRM)

► CHRIS MASON, MBBS, PHD, FRCS, UNIVERSITY COLLEGE LONDON

► AVI TREVES, PHD, SHEBA MEDICAL CENTER

► ALAIN VERTES, PHD, NXR BIOTECHNOLOGIES



So welcome thank you for my name is Mike drunk men on the part of the global law firm. Hogan Loveless and I'm honored to check this morning's panel leading nations Field man strategies for regenerative medicine. We have a great panel here today of world leaders in this field we're gonna share their views on the most successful strategies around the world for advancing regenerative medicine. So turn off your cell phones take out your notes yet rated at a furious notes because everything they say may change your life and help you change the world but more importantly so I'm just gonna say a few words very brief about each speaker you learn more and join the conversation and this is you can see this PowerPoint so we want this to be dialogue they're gonna ask each other questions we won the audience to speak up so just a few words the new to the panel this year I'm very happy to say we have doctor usually he comes all the way from China 22 our trip and is going back tonight 22 hours. So we're we're quite honored dead said to have him here with us doctor lee is the director of the Yunnan Province stem cell bank and the Yunnan Sheng Qi stem cell & Regenerative medicine research center. He leads several stem cell research projects using both government and private funding in China and Australia. So welcome doctor lee new also to the panel this year doctor at L&S he's the managing director of NXR bio technologies which is a business development consulting firm in Basel Switzerland Dr. Robert S has expertise in innovative funding and commercialization strategies and in technology deployment he has worldwide experience in partnering in licensing large pharmaceutical companies while conducting the test returning happy to have Dr. avi Travis coming here from Israel he's the deputy director of the Cancer Research Center at the Sheba Medical Center he operates the stem cells transnational laboratory. He has served as a scientific advisory board member for over 14 bio pharmaceutical companies and he founded the successful start-up you meet us sell limited in 1998. Welcome back Travis also returning this year I have to have Dr. Chris Mason professor of regenerative medicine bio processing and advanced center for biochemical engineering at University College London. The world stem cell regenerative medicine congress named him the third most influential person in the stem cell feels. So we're very very lucky to have Dr. Mason and last but certainly not least. Dr. Michael may is the CEO of the Center for commercialization of regenerative medicine CCR am a Canadian federally incorporated not for profit organization whose mission is to bridge. The valley of death between discovery and commercialization. Which we all know this very difficult problem see CRM schools to bring five hundred million dollars in new funding and risk capital to stem cell development over 10 years welcome back. Doctor may turn into the players let's get to it I'm gonna start the ball rolling but I'd welcome people to ask questions as we go because of the taping. If you ask a question I'm just going to repeat it. So we'll just give a pause for that so my opening question doctor tests so you're a newcomer and you've got broad experience across many countries all start with you on this question in your view what are the best development strategies for Regenerative Medicine innovators around the globe okay thanks for this question let's elect one says he's been waiting for using terms of strategy first of all a very high level view of the strategy and I think that when we look at strategy it's important to understand what are the fundamentals of what it is we're trying to do and also democracy is at which an emerging technology and energy means. We have resistance to change we are very stones to other so that's very important to understand because what this is what it is that we need to do as an industry is to create a win means that a particular up will deliver benefits that no conversion Americans can deliver and as critical to bring essentially. These technologies to serve for other pharma companies and investors second part of the second angle to to consider his goals and it's very important. I think to companies in the standings up and there's a manufacturing cost of goods. They can't really control the logistic Chechen catheter don't have a product and we've seen this happening in Sumter predicts with advanced but in terms of manufacturability we have issues. I would say there is a subpoena to understand is to consider what would be silly strategy partnering with pharma companies. But also it's very important that we as an industry create shareholder value so based on this where could we i think is the best opportunities for emerging regenerative medicine I would say in geographies but you can enjoy their fees that the system to promote as its technologies to stability of data. But also regulatory environment that is really interesting that we can really accelerate the development and the pace of development and not forgetting patient advocacy and as well investments. We thank you I'll move on unless anyone has some immediate questions for dr. Bert s alright I'll move on doctor lee I'm fascinated to hear about stem cell development in China I know that your projects you utilize funds both private and public in China and Australia and I understand the government is preparing a special fun yes so I'd love to hear more about that and please tell us how those funds will be employed what role international collaboration can play supporting those efforts in what will the strategic approaches that Dr. protest mentions might might play also. So I think I just want everybody back wrong because China is really huge nation and has 1.3 billion people seriously is only one child policy so now it's changed is changing to talk out so everybody is very exciting worse so that's a lot of them cell preservation and stem cell research and based on our bank we are going to have a nearly 8,000 to 10,000 samples each year. So there's only one bank in one promised so consider there's more than twelve thirteen stem cell banks in China so huge and then try now takes stem cell research that's a China science catch up on maybe leave you know you know work and because we seemed to reject in medicine. It's really important though China is now just released their new regulations three days ago in the status of December regulations on how to do themselves clinical research and the regulations there under how stem cell donors to produce vendors. Then try is going to use this one hundred meeting USC daughters especially those stem cell research and increasing 21 BTUs $1 each year so based on that is a lot of support from the government fun. They want to do a lot of research on stem cells tenders multi center clinical trials and they are used for regenerative medicine and also from the privately funded because we there's a huge market a huge market for the stem cell preservation and application so each for its really likely to put a lot of research for example of our company is already spent nearly 12 media USA dollars privately Research Fund posts I'm a cease and use of cord blood tissues so I think this is the whole background picture continues. Chinese taking this very seriously and i think is really good opportunity so I think they still gave the mall its national corporation opportunities and we said so we really like to explore internationally. Because I really think this is a good opportunity for application for human house so we think it is really be product and then China want to play a bigger role in this year for us is lots of dollars and there's new regulations just out a few days ago so we welcome you to explore that alright we'll move on Dr Mason will turn to you I'd be very interested to hear professor. What grade would give academia and fostering regenerative medicine development in 2015 and what what do you believe academia academic institutions can and should do in 2016 particularly in the international collaborations with industry government long lines were talking here obviously. If I could see me right now I think academia as a crucial wrote like we shouldn't be doing the development was coming up with the step changes the future role in gene therapy is very much seal gene therapy and we see that we might go see CRM taking a great sometimes out to the Canadian academic world. I'm translating it to how I could be doing what we're doing I think exceptionally well if you look at the vast majority of the gene therapies. The ex vivo gene therapies that are coming straight up academia incubation period in academia now do they bring really is class results and then those would be interpreted into the companies we do know quite where they've been told that academic base. So I think I think we doing very well and we've got quite away to go I think we have to focus academia has to think just about the actual therapies but also the tools and technologies now for example how'd you manufacture these therapies I think we have a long way to go in the air at the moment we using the pharmacies could approach of centralized manufacturing with CMOS and I think we now need to be thinking much more about it distributed mode where you have many manufacturers did you see a therapist local basis like think that's where the blue skies research and academia to drive the field goal with thank you alright I turn to Dr. Travis. So Israel's biotechnology industry has been powerful a powerhouse of innovation even though it's a very small country would love you to share with us some thoughts and secrets behind their success particularly in the regenerative medicine industry including the in the impact of international collaborations and the role of the Israeli government's funding is where I don't have my bad I don't know what's a book issue a couple of years ago was very nice and not as these why though so many stops in the book is called up start-up nation and systematic analysis with some good examples about success those of technology startups in addition there is substantial government on I'll indeed in many stages in early stage like I deal with a few preliminary experiment you can already apply for certain program of funding schools the incubator program centrally. There are many opportunities for international collaboration actually working on R&D joined on the development. I think with every nation in the world and with china for example we have not only international organs but we sentence if he stands owns me a specific example I just know he'll come with Syngent a smoke down sixteen million people in the South China. These blogs please engine companies marketing support to both in Tucson not really sums of money but for startup companies very very supportive an important breakthrough now just out of curiosity audience. How many of you either are are in startup companies are working with start-up companies and even here so here's some insights and people will need some time for that excellent all right thank you don't trust me CSIRO has been making ground breaking new ground in the regenerative medicine field for a number of years already and increase the funding in the research particularly collaborations be interested to hear how CCRN makes decisions about balancing the question of local activities investment versus global and priorities and what role financing and sustainability plays in those decision interesting so first of all CRM approach has been to build the right kind of networks and ecosystem for commercialization as its kind of foundation principles so starting with academics and access to their intellectual property and institutions in regenerative medicine and being able to pull those discoveries together into new companies is where we started in then built an industry consortium on the back of that activity and are now building a third network which is an investor network that can fuel this model as opposed to just prime it with a little bit of government money that we started from but this this network or network is then supported by infrastructure a lot of it coordinated infrastructure from academia billions of dollars invested into infrastructure that can be used and then specialized teams that can advance these technologies and companies so CCR M's approaches to is to utilize all of that and like an investor invests in opportunities. So that's where you know the the basis of our evaluation of opportunities is really like into what an investor would look for and our sustainability is all about taking a small piece of each of those opportunities so we're not in granting agency that gets money from the government matches up with industry and then deploys it we actually evaluate IP from around the world not just Canada now and we take an investment in those opportunities and that an exit from those opportunities in the future will be part of our sustainability model now the last piece of this is is that no one jurisdiction we believe can actually have the scale for that sustainability so we have a global mission and we are building relationships all over the world to not just for the benefit of Canada because it's a global industry but for the benefit of all the areas in the world that have excellence in regenerative medicine to create a global network of clubs do similar activity to see CRM to create that tipping point for that scale we're truly sustainability can be achieved in a major part of achieving that sustainability is leveraging all of this success into-specialized funds that can focus on regenerative medicine because without that without again in a Canadian context were doing that in the near term to demonstrate it but when you think of a global network you need major investment and that can you can only sell to investors if you have something to sell you can't just put your hand is a world trade this industry is wonderful give us billions of dollars so the the CCR models about a step wise process for ultimate ultimately attracting billions of dollars into the space to go to China to build businesses in America and Europe and every excellent thank you doctor Aisha lots of good advice there for those thinking about coming to see CRM and others in the in the environment for investment all right now is the time I said I was going to quiz you thought I was kidding. I really am gonna take some fun for the audience so let me ask you a question you want to ask the question well so I was just in China but weeks ago and when asked about Germany relieved you're relieved get ready because. I am gonna ask a quiz but you may ask so in in in China I mean it's a place where you go in and you're in complete awe what's going on in China but obviously very big and very complex is is it possible to do anything in China without a good local partner are no longer a series of calls to China is quite so easy to find at January partner they know what you are doing and they have a rather it's also one big problem in China I think most people will find out he's IPU seems so if you have a really good products three months later maybe something new comes out and then these really takes a lot of money and the time to deal with two through the jurisdiction of the Justice caught those quick one is to officially the government so it's better to have one good results and have they have to know what a government one end and they have a result there and then they can using this power to appeal to Protect IP in to protect your products so finding a real good local partner is essential so I think this is my great thank Yolanda other questions are removed my quiz right my quiz with pics online right there gentlemen the front row please stand up this is Socratic Method alright so please tell us how much money has the government of China put aside for special for stem cell researches and you get a prize all right now that we've all pay attention so I wanna we have nonsegregated Reed here author well-known author of the book stem cell Dallas so it's on sale here also on Amazon so I encourage you all to thank you don t want to just give us a two-word soak up a couple of words about the book just tell us little bit about the book great thank you done Chicken Soup for the stem cells is heard it right hereon now will get back to get back to us a little bit more serious business alright so I move to questions about international clinical trials we handsome discussion about that but I'd like to talk a little bit more in depth and doctor lee I think we'll start with you and you have some experience with global standards on how to carry out regenerative medicine research and like to know your view first on what our priorities should be in 2016 for advancing those standards and for facilitating international clinical trials and research and then I didn't cause the panel is panelists tell their own experience and and also a staunch ally questions so at a plenary session this morning about the a refund the bricks through therapy approaches really good and then I also know something about Australia because Australia stem cell research stem cell clinical charge if you are doctor and then you are free to use of Genesis themselves like in 10 patients also and then to see how it goes and then but in China seriously everything is illegal because there's no regulation but only the stake a treatment but this year just three days ago the regular season comes out so they know they have a routine say how to the regulation but the problem misunderstand his two different from Australians then there's different from American Sanders so i think is better to find a better way to farm international consulting menthol to say how to set up their standards and the other really Really talked with some external scientists suggest they really don't believe China's clinical data so this is a situation but we really want to change it because we small justify an open access and think china should be accepted by the National work and is that one of the reasons thank you doctors that one of the reasons you think for the recent issuance of these yes interchangeability yes so we're not doing the research only in China so the Chinese government support for international collaboration so you can do multiple Center clinical trial likely join the WTO so everybody knows now following up with vendors so I believe in our data only days he's just because some black sheep in China and a doing really bad stopped and everybody is challenged people stop and I think Wednesday multi center clinical trial they know Chinese scientists are following the rest and there's then they believe it thank you I just wanted to jump in and say Canada was the first to approve this the initial tissue engineering skin product sand actually was the first to approve the first time cell treatment so many people may not realize the very close to yous neighbor to the north has a very open minded and efficient approval process and clinical trial infrastructure so one of the companies that we launched in the summer is a cord blood expansion company and just received approval for our clinical trial in very efficient 30 days and its global expansion so we should talk about it but it might be happening before your eyes conducting clinical trials and in Canada is is less expensive than in the USA and I think Chris could could back that up and the regulatory approval is based very much on risk benefit so it's open to conditional approval like is being considered in Japan less formal than the Japanese policy but is is a place that that many people may not considered as an alternative place to do clinical trials and get approval thank you other questions but just over 10 points and the scenes with Japanese wooden place to to accelerate with genetic medicine in a safe manner as well that's critical and the first successful the approval by p.m. the of Pokemon actually by mister + Partners al-jazeera pharmaceuticals so it's an hour pulled in Japan for a cute GVHD in both pediatric and another sort of our first product lines with a new process and that's appealing to them not to do this and we're going to see more posts but I think it's it's not file feel no just have fun but but 2011 thank you doctor may send a question and i think is extremely good because international clinical trials are more essential and I think we should think more than one country involved in the clinical trial I think they are essential for two reasons one its the patient populations alternatively smoking some of the indications were looking at the Red diseases it's hard to find a decent number of patients that operate brutal the right conditions multinational trials are essential components that you need reinvestment in the small companies that sell very well having conditional approval initiate reimbursing you just giving the public a way that the extent that they might be an example of the UK so we all like to be working with the Japanese regulators a UK regulators Japanese right nice looking so you run the two together to get the best out of each so the proposal that we've got getting foods that you could do a second control in the UK preschool trial generates a today to take that Japan get your condition approval which point you get to reimbursement Smith do a small British study but it was a little too few patients you then generating revenues in Japan you learn about the health technology assessment process whilst you do your pivotal clinical trials in Europe or USA where are you going to do it and then you have a position where you have great day to to get full approval in Japan from your USB or European they tell you had reimbursed them pull the period of the trial from Japan and you a much better shape than the catalytic the company you trust overthrowing the road with no real both of them to other places and say we're very keen on trying to get home until I get integrated regulations in different countries to the benefit both countries it's a great show you know it hasn't been done right now the discussions all of the apt imagine that HW all negotiations that makes her a high level and the we have a fleshed-out plan of how about looks you can do them pulled once you have conditional approval in Japan the product manufactured outside of Japan so that's a big plus so the ones you actually get a full approval for what you put approval will be happy to do that and you can take your data roam say kind of WS Europe with a very small breeding study to do a little bit Japan to get that initial approval that indicates a key and an indication of accuracy so yeah we are progressing that and companies are actively looking at both UK and USA companies Exxon anyone else have anything that practice yesterday I just want to end up in the united states you can get the pool for investment in clinical studies not for challenging course but it's a special program many knows about it in in the various phases you can get an investment on costs right because you know the only challenge without you have to declare which will cost of goods is always your cost is like I'm not attractive to companies use Aim or look at reinvestment slightly different therapy that could well be a woman dumb so I would caution against the night analyst say hi actually reduce some of those when I was an FDA and they are fairly extensive because FDA does expect you to show you're not making any profit it's OK in certain cases of you can justify covering your costs but they really expect a showing that you're not gonna make a profit now there are some exceptions you want to know more about that you can talk to me after condemning situation or just because that's the only way to know not the company and I know hospitals down to something example while using this so much certainly that some that that's where it's most successful I don't know profit companies of the top of my head I don't know they have but it's certainly more in academia doctor may and I know we have an audience question and then we'll go back to doctor lee so we're driving a similar model of this phase 1 safety data a broader than going to japan is one of the things that's really important to consider is especially with respect to reimbursement is the importance of tackling the manufacturing issues along that process because if you leave the optimization of manufacturing too late in the clinical trials it becomes very expensive to go backward sand you have an inefficient system and some companies have failed because of that and of course you don't want to optimize too early because you don't have the efficacy it's not worth it so there's there's an optimal point and that's eCRM we have a very strong focus on manufacturing and in optimizing manufacturing because that drives ultimately the reimbursement costs of goods will drive the equation for reimbursement so I think one of the challenges with all of this is if you go into a clinical trial and get reimbursement is that a real justified reimbursement because the optimization on costs may not have happened yet and so there will have to be an iterative process globally as we harmonized all of this where the manufacturing innovations line up with the clinical trial investments by other species or the government like Japan which is investing in their clinical trials to ultimately come up with a you know products that are economically feasible ran errands I just wanna give well OK look legit two more panelists questions and will turn to the audience just want to add this information for the audience if you are going to clinical trial in Australia is good for you because if you ambassador 100 bucks government give you 45 back so that means you are getting 145 in a research grant grannies caught R&D tax incentives so just you spend time money and then submit the report and it's a it's a tax credit on future revenue that it seemed in Canada you actually getting a grant a refund within one quarter of the yearned of fifty fifty cents on every dollar you spend on research before very very positive tax benefit I know in the USA there are tax benefits or certain things orphan drugs and other things but it seems that this is an additional manage Dr Mason I just wanna go out to all my goods and I think if we look back historically Google biotech biotech we recently reimburse the cost of goods plus that's how it was done and they were very happy to do that with some great stories up by about genteelness I should be very happy with that arrangement because they knew they could bring the cost down very quickly by an order of magnitude that they would get stuck they will be left to the high price is it was good to good plus now I think for us it it's probably tougher to get that food or money to save a monk who sold his right but I think it's important to remember that Coats goods plus he is an option on these therapies where they're approved very early you don't actually have a lieutenant data example you may come up with the gene therapy that you claim to use the disease but you don't know into the go by and it's very big challenge for the reinvestment agencies to give youth full price it was a kid but the company doesn't want it if it's later than one year they think that suggests the child's life but let's get those expectations particularly for some of the things I gene therapy where it'shard to know it'll be a lifelong care or not this point wrote your name from the dates you have and what is a time so I want to open up to the audience questions in the audience who started talking and then write history and I'm gonna repeat the question so give it to me a little slow rightly said summarized so I'll it to summarize the question is from personal experience Mr. Reid mention that the public has a lot of misinformation or non really scientific information what are the panelists views on how to educate the public particularly children but the whole public on that I get questionnaire we'll give each other in a cell phone no I just just want to see education as stem cell education also so sassy CRM and the UK catapult and the Californians to avert general medicine have actually created a children's museum exhibit around stem cells in it's actually on its way to California to do it to her and it's an interactive exhibit to educate kids and their parents about the history of stem cells in the power of it so you know doing things like that different kind of education is one example of something so our stem cell bank on behalf of everyone around 200 square meters for coming into with it and have a look and I also write a book called stem cell and regenerative medicine Chinese for little children to see the reason why about doing this because we are both public banks and family banks we have to first Italian the parents why you reserve your calls that and then was to application of your call the black T cells and stem cells so this is really cold in this offer our education and that's so we have to do this because the most people know what caused blood can do what what a surreal we got medicine East attend to and support most of the research excellence panel should I think I think it's a good question I think that we've been very bad as a sex actually exciting the public about technology we played used anytime we could be some serious work themselves no one understands I think that we are moving we we assessed removing it with we moving and I'm pretty gonna get a stake in the back of his but we move it to become a joke gene therapy industry is no doubt as we moved all my engineering 217 too much more gene and cell gene which is ultimately when we go to a top ten years from now that's what this conference will be about the gene communities done good job in the synthetic biology unity still a good job babe excited young people are competitions such as I JM whether you building block routing breaks to create a normal organisms and also supposed to put accelerating things that kids like and i think is drawing that excitement and it was a neighboring that the gene communities Dunlap thin know we are converging with the team community think we would enjoy riding on the back of that along with a great success make making it the audience alright and again I'm going to repeat the question so take a shot at that and welcome from the Bahamas so welcome welcome from the Bahamas and London nice nice to have you and and I'm in a very broadly characterized it as given the Grand possibilities of these therapies there may be some risk of it getting too narrowly focused on gene therapy in particular areas what can we do to leverage the technology in the learning in particular area so that it can be more broadly applied to is my close X alright so we have the question now for the answers Dr Mason the school but I think the questions very good and the regenerative medicine program time we truly regenerated not there's been some small molecules and biologic doubted a great success regenerating prejudgments in his term that we've used to long will I i would say is worn out useless in the US much good at the moment where cell and gene therapy I'm trending towards gene and that gives us the right way way more scope just regeneration regeneration really important but the technology will walkabout to this room is much more powerful it can affect things like cancer for example and things that are not regenerative things where maybe it's disease where the child is born with a genetic defect that corrected that's the genitive when you look at the whole host of the spectral what we can do with 17 limited as we look at our generation great but it's best to very small subset what we can do with 17 not to mention the understanding of the biology of healing has really driven much of the science behind the cell and gene therapy and regenerative medicine innovations that are happening but I would say there's no indication whatsoever that there is a narrowing of anything in this field the therapies that are will emerge in the future are a combiner combination therapies are small molecules needed you know genetic modification cells will be needed you know and and what we're here on this panel about it is about international collaboration and I told you about CCRN as model is academics coming together with investors with industry with patient advocates there is no trend that would suggest that we are not collaborating at ever larger scale sat ever greater inefficiencies in ever faster timelines towards therapies that we can't even imagine today that today that will emerge to in three years from now doing things that we can't predict so I don't think it's a narrowing it all and I think you know Chris is comments about you know trending towards gene therapy is not actually about narrowing the field it's gets its expanding and much more clever thank you Dr. test I just would like to add that we have a brand new technology still emerging and as an average ten innovation we need to understand that we're going to go through several s coastal development ride and what to write this way the efficient way to do this is to go from a little simpler to the more complicated from the new to the better and to do that we have to go through the standout processes that we understand and compare right so the standout approaches that we are having now is a correct one because it's from simple to better and here's around they think back to your question and i think is very very important to understand the impact of this technology easy to use technology to transform healthcare but you sure about this but not alone is going to being combination with all types of technologies particularly this particularly the rationalization of medicine and what we are going to witness I think next few years as these technologies get proven because it still needs to be done as its technology get proven women to give more services based complement more rationalization in Boniface d research and clinical center sand he's going to avoid essentially how we deliver a more personalized more precise more energy and so this revolution is only starting but we need to start from the simple to go to the mall complex from the new design but Iknow that FDA asked now earlier and earlier what bio markers you'll be helping really early stage questions they ask that are you going to need companion diagnostic or is likely labs efficient and so those are very important questions to raise early on in the process particularly for startup companies to think about dr. Travis went to add another perspective I think the best educational and promotional tool is to have good clinical data look what's happening today in the county technology immuno therapy of cancer every child in high school can lead in a newspaper what's a mechanism and what it's doing because alot of applications so I think once this field lack is a grouping good clinical data once we have that we don't need special girls Chinese President don't say when there is a bike at all if these cash them I say so in China have a saying is that what are you sorry Jen Madison what are stem cell whether it is GE gene therapy if he's cures that is easy one to cure the school so we have a support our different kind of technologies and because different diseases different cure so stem cell may be good in one pot and a lot of hogties Chris Burke us tonight maybe good so they've got developing so many different areas and in the real situation but there will besom consolidation at some point for example you know the human body is the best bio-reactor ever and you know your your bone marrow produces two million red blood cells as second we can't come anywhere near that kind of production incinerators so this idea that eventually gene therapy or a cocktail of molecules being consumed like a future drug to take advantage of the regenerative capacity within our own bodies could be that endpoint and and so cell therapy cell and gene therapy may be just intermediary you know steps along the way because in fact our bodies do this you know very very well thank you for that question it generated a lot of discussion other audience I'm gonna try to repeat it so all this is is the breakthrough queen and for those of you so that was the was it three of the five 305 oncology indications breakthrough therapies that come through I'm sorry give us your name again something great you have a big smile if you have a blacks William 18 denied denied personally I so be careful cost-recovery that yes I am from the chief counsel's office you're in Cedar religious summarizes spit's very important for companies are considering cost recovery that FDA has two considerations before we get to the actual cost of goods in the cost therein an initial threshold question about the potential for this product to provide a substantial benefit over existing therapy and that's what the good doctor here to sign and then the second is there's another group that deals with that financials right okay so now the question doctor lee and have two terms so this this question was what is the football comes back so the question is what is the of the for a colleague who had the six billion dollar answer whats the source of that six billion dollars a day early so you know pierce china has two major different scientific fine wine is called poly 1863 night 730 the 973 has stopped last year and now the nice I'm sorry changing to cause major scientific breakthrough file and then this sex free fun has seven different levels so that one is done some research the second one is digital you Weiss this is about the environment the falsies and wants to manufacture machine so all this and so there's no nice 73 now I'll turn you into this caused major breakthrough research stem cell bodies on the table and that's also the reason I have to go back tonight because of how to apply the really big grand gotta get some great you have a second question okay one more question so you so the question is if I have it right what is China's plan to globalize what what's the FDA's plan for globalizing standards is it a missed the last part of question the standards regulation before they even for a research done with their re- at the research done with their funds or you mean global OK so this is a question of Carmen is generally harmonization China and the globe doge why now is called a cfda nowadays great thank yous why is because China is it really be country and the government is be so they have to department and they are overlapping and there's another really recently to step in this field in the first phase the Ministry of house the second needs and the third is caused by china's reform and development Bureau so you can understand why the FDA cannot be the only one to rip that China and internationally because there are so many people and then wipe years later China's regulation has been staggered for well because this house up in three and also the staff here are competing so which one should the lead on regulation and now how many because china's reform and development Bureau has taken the lead and they say you to join together and then to the regulations I think in the future will be representative you need to feel that this is the regulations comes out and i think is mainly because the two different party they just political reasons why not scientifically they have been unified and then we will be the leader by the Department of reform and development just want to go out Google movies Asian between the USFDA and the European community so the question is yes then no motivation between the FDA and they eat she said yes I think building but the doctor man and i know in just gonna say point came up that think is really valuable for startup companies there are a lot of compartmentalization companies represented here is that you know the regulatory approval is safety and efficacy but the reimbursement is cost benefit and when and and often we asked the question at the last moment was gonna pay and and the problem is is that that caused banish it gets assessed against standard of care and the stand if you think of the companies that are represented here and we're doing it takes maybe five ten years to develop a product I think it's really important to be constantly assessing the cost benefit because the standard of care changes in the background and is nothing more frustrating than landing seven years in the future and you find out that actually that the standard of care what your product out of business because it doesn't have the benefit of benefits outweigh the costs and so you know we need we need to evaluate these things over time and in and and in terms of international harmonization reimbursement is done differently in different places in the world so and I hope I'm cracked year but in the us- it still driven a lot by lobby and in other places it's a Valium system where they they you know that's the the the benefit versus the cost and so but in either case you have to justify the cost benefit and if that baseline standard of care is changing it can be disastrous for just one is going to have a directives and regulations but member states of certain level of freedom for generations so to try to summarize in these international clinical trials one of the biggest challenges manufacturing sites because of the differing standards and the question is is it feasible to take Dr.Mason suggestion about local manufacturing separate manufacturing and globalize it that a fair summary Dr Mason you wanna take that one in the first instance opened his own new standards for you to manufacture a present and therefore we do have a blank sheet of paper so there is no reason at all this very much what I'm thinking if that it would be do this internationally he would try and get the bully in regulators because there are steps which are complete change their work in practice their example you busy just think its black box where the Commission takes some sort of senator from the patient that in the box where have all the unit operations such as genetic manipulation of cells expand wash and prepare them right here back in the patient so it's complete patient patiently whatever you want to call it that is a big step in the regulators now presentations that how how would you go about that for example what about things like the quality assurance and quality control in the final release all those things need unpacking carefully and I think this is a real opportunity to work with the regulators joined up man across all the regulator agencies so we don't end up becoming greatest where we have different levels of particle filtration on different GMP units preventing European and USA products going each way not just doesn't like said the patient is not good industry so I think we have a blank sheet and I hope people who all seem to think about making these medicare or distributed manufacturing devices take that on board and to work with multiple regulators not to me so I think having international collaboration among translation centers and driving that will will start to create at least that that alignment at the phase 1 phase two levels which is the starting point for this the second is that you know when we talk about engaging industry everyone always focuses on the pharmaceutical companies thinking that they're gonna you know they're going to enable this but I think we have to remember the infrastructure companies the companies that make the tools and devices as being important partners in achieving this harmonization among tools and therefore the ennoblement these this district distributed model so actually I think this is really good questions and then told us that he is able to do the unified one I think at London yesterday. I spent one hour into the world of coca-cola and then I see a movie called The Secret of coca-cola what's their secret uniform distribution you can find I will wear the same case of coke a cola why it is called Koch because he's cold so I think in the future can learn from coca-cola stem cell treatment of the house of the media at as top to the unified and then can produce I'm as the HSE seen any kind of situation I think this is something we can learn from the city of the purse I'll be curious to hear whether that's become is one thing but me senchymal stem cell is that her test product approved by one company so you want to distributed computing and also several centers for manufacturing date as well so you would like one in the us1 in Europe and one in Asia this way you can really mitigate risk capacity region as well but also other way a good level of distribution but what is critical when you are free at least 30 manufacture and I'm talking about what's important obviously is to have reprehensibility of lots and and complete product equivalents but this is a typical properties of so we need to think of fabricating biotech companies what has made a big pharmaceutical company successful or other generic company that makes a great lesson controversy is flawed we have no imagery we do not produce and we will continue to produce identical to what I mean by that by spring breeze on there is nothing has been more agree well let's let's take something like gene therapy what is a dose of Well there's maybe depends on the poem the potency of your cell viability of the cell when she put it back in the patient to reprobate so I'm not convinced we all producing identical the rethink we have to do is produce high-quality see at a price that is affordable within the therapeutic window i think that's quite different producing uniform product every single time I think that reproduce a uniform products going to hurt us because it's become more minutes later still has more genetically just cannot achieve that but I think we now need to still thinking very differently nothing to promote that to the regulator is wealthier things that those which you can even define how can you possibly create a consistent with him and then when the Canadian thing and try to get you guys to agree you're saying the same thing in fact quality systems whether as coca-cola or drugs or future sell products it's all the same principles you have aspect aspect doesn't have to be as tight as something for coca-cola but eventually have to produce produce a product within a range of potency and and and dose and that may be different for sales but in fact they will have to be quality systems in place consistently across these problems with its criminal justice policy is correct what i'm saying is do you produce the same cell number in the bile her patient who you know what you put into them will replicate I sell ten times more impatient I didn't taste you create a different product yeah but but it's the definition of this pact that defines that you might have between 500,000 anda million cells is okay in terms of the therapy and so you're you're not what you don't want him to put a billion cells in the patient or two but the process has to get you in a range multi-generational I would argue that as we get more sophisticated with these where patients will be very different and we already going to get down to your position or us life within basis he said he's gonna be very different we have to be able to make these spoke there to actually makes the genetic diseases that all will use the gene to get great results so I think they're quite different from using a consistent product every time I think about the kind of 25 reviews go to agree on that but I think the products in the near future opening very very different well what what we have is a specification issues as well because as you know the particulars who invented when they have any need to because of the statistics you need to produce a product reproducible Now in terms of energy and property can we all know the process is our products to find any notice it's also true and particularly so so we have really and I think this is a key issue here we are really a problem in terms of here see show can we didn't qualify as lots and I think as an industry we need to refine our knowledge and capabilities here and I will say and I'm not a scientist but the one question that I know the agency's certainly FDA will ask is howto I know how do I know that the quality they have to have to accept a certain amount of hydrogen AZI think that's just inevitable but the question is how do we know the spec is small enough and other Valley either accepted standards of validation at this point or is that doctor lee something that you think we need more if you want to use really big steel you have to prove to them that they are different batch of cells have the same quality of the same things so this is also one of our research may ready to fight and to produce we caught off the shelf products these products each from each each batch same to you all it says is that if you want to use one in your cell to cell we just gave you three million cell and then the doctor can decide whether to use half of them are just so I don't think three million unless three million cells puts it out of the economic feasibility I mean if he can just but as many as you want there's a cost of that but I think the problem and then the other parties you can you have a small batch and then you meet someone needs to Minnesota have a passion that the standard is really important also I know a company called many post so many policies used to treat they called cars them but they can create and now he's getting the approval and also did not have a proven in Australia so they are you from one batch from the one thing go baby when they have that can grow these won backing to eight hundred different batches and then they used to treat patients and they are not proven successful in a future one cannot identify all similar approach unimportant full to scale up and gave different person you were a say they seized procedure Madison your first tattoo that says and then you give them but it will be a long time the car cheap you are using another of the shop product you have to spend one night also rejecting to patients and your day for AML patients only it's date so they don't have the chance to use the card in the future so we are really thinking to find some to produce a life like a coke sprite they have been had they have and they can have a coke 0 you can have a different cell lines prepared for different patients but they were so I think this is something we should be friends outlines new stems the good professor from London so let me try to summarize that I i take it this is a challenge that suggests we need a paradigm shift in how we examine standardization and that you really can't fall but coke model and that we need to have a different sort of approach to both scale and to quality to have that pretty close let let me react and energy and I think actually I would really like our analogy with a computer not only are any product any consumer good analogy they way you treated in Denmark France Switzerland Geneva in in in in Chin awhile he's in the world you want to do the same treatment you want the same qualities you want to see importance you want us in city what we're talking about is no different morning adding a better right it's a famous chickens we have issues are challenges many things that we don't yet know in terms of potency is here from his own human stem cell phone this is still very very important done and some people think in terms of using interferon-gamma responsiveness for example as one measure and separately will be more in the future but as young's a day and I standardization but that has been selling many other industries and we need to use Powell announces the recipes from don't apply for stem cells was thinking that I think was incomplete maybe I can be used it I think we could be heard an elegy is producing territory you want to use them all the same potency they think corporation I have no no disagreement I think well I'm disagreed is where you are using so I could pull yourself so for example Think at the moment there is no regulatory system that would allow me to meet you there where I have a variable potency and ability to divide in vivo that one could reduce the same impact on the patient by having which is they got X opens the and tactics or potency expansion of the same number themselves in vivo and potency as combined you need to see them bot hand yet the regulatory process won't do that that's one thing we need to change any regulator would allow this moment in time to think through these criteria Need i think that's potency over within allows me a division ratio why and I think that's what we think and I'm giving you a very very simple example is that shot in the complexities of greater than that and you will have these moving sliding scale and that you completely different product is going to be different individual and let sit alone in this room is identical loyalty pool that you produced one bit right I don't see it as a little people would have been through my very unique individual genome requirement different been inside the standard isn't about a ideal we're not talking about a reference standard we're talking about ways to measure the quality of the product and that's what we're talking about with Coke is that it's the same everywhere not that there is one soft drink that everyone in the world should drink and there are you know there are competitors I think one principle that is pretty uniform across the world is is that we measure safety and efficacy and then we measure cost benefit and different countries measure those things differently in different processes when you need standards in order to to to measure consistently safety and efficacy cost and benefit and the more we agree on those standards built into them and it's more manufacturing comes in I think that's really where the coke analogy came from was manufacturing coke the same everywhere in the world and having the same product that that will apply to do our to whether the personalized Minister not to cells I think there's a difference i think one is metrology Wanna standards and I think I think the difference i i agree with ecology for example you again exactly how many cells do you want to get consistency across all I'm not sure that the same as the standard it's more than a trilogy mini standby control and I would think that's why they're so I actually answered my question is do different cell types talking about Andres the first one-many people talk about lawyers so that's missing is the standard standard procedure the hotel real Purdue was and how do I didn't reject but I'm talking about genetics so we are trying to produce I think the most salient Lisa had a five or six years and then you know exactly what they sell I A hayesand for allogeneic cell lines in the future we want to use them first you have to know this talent while you cannot using some other different batchand then I think the FTA will allow you you have to test again and again in four different batches if you use of one cell and they say it's all came from patient and then we produce and unmodified and then weekly pizza and then this kind of product is unified and then people like to receive it because they know exactly what you need genetic pot and what can trade and the wider looking for a suitable to use stem cells so I think this next them self feel that really the party because from our research from a message when your trip next time around seven to 10 days you know about it then also down to assess they may be current turning tool cancer or other pots and we don't know because he's hard to find but if it's out of genetic system will be cleaned them out after 72 days and it seems to me much safer well there's an old adage that you can always leave the audience wanting more so we're out of time but we do it we need to go to other places to go but for those that still have questions Encourage you to come up the find out first of all let's give them the hand are really great job and the audience thank you very much great panel so I'm gonna end up here but enjoy the rest of the conference thanks to dpi and tornadoes group and enjoy the rest thank you very much.

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