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New Advancements in Cell and Gene Therapy Implementing Precision Medicine


Welcome to the cell culture dish podcast new advancements in Cell Gene Gene therapy implementing precision medicine. I'm brand new sergeant editor cell culture dish joining me. Today is Dr Paul. WOTTON chief executive officer Obsidian Indian Therapeutics. Dr Watson Brings Significant Experience Spanning Scientific Research Product Development and corporate growth gained over thirty year career. Most recently he served as the founding president and CEO of Leon Therapeutics He also serves on the board of directors first of several pharmaceutical companies and was named the Ernst and Young Entrepreneur of the year in two thousand fourteen Wanted to start out today by asking about the origin of the name Obsidian. Where does the name come from? So the name is derived from a Senate Roque Upset in which is found across the world and and The points of it radio's ninth companies that we're bringing precision to cell gene therapies in terms of allowing seven gene therapies. Eh to be controlled In a way that hasn't been done before and city is a rock that he used to make the most precise scalpel used in surgery surgery. So it reflects the precision The second thing which I think is recognized as a company We have now fifty people obsidian and we count come from everywhere in the world. It's just like Obsidian rock so you'll find him city in the volcanic logger flows Across the world on park trump's reflects diversity as much as anything else. That's a great a great reason to choose that name. I think that's that's really unique. I wanted to move on and ask why. And how did you begin I'm wondering kind of what your personal story is. And what inspired you or motivated you to move into the cancer space. Brian Yorkey involved with A number of cell therapy companies. I think the Senate gene therapies are extremely impulsive technology. Extol the future and it does represent the future of medicine and my first exposure to this was with a company called Casa which was working on back to the soldiers which was actually purchased by Stella's a few years ago and they're building a Development Machine here Massachusetts to develop both cell and gene syrupy through that. Let's talk to the company with flagship pioneering Here in Cambridge based on what had been done in lapsed mit. by Bob. Langer Dan Anderson We identified there. There was no paternity to improve. So I'm gene therapies that particular the cell therapy's by allowing us to put him into the body which were that. She consists of cells that were hidden from the immune system. They can produce replacement in proteins for Diseases on enzyme replacement Talk Diabetes or things hemophilia during the cool so that cheap Ed Greg Nut Company My wife ended up being treated for cancer going through chemotherapy and I was lucky that we wrote the transitional transitional leadership that company to Jerry of the Valley Just fifteen months ago and then obsidian approach me about the styles this year and I'll be interested in working here. A couple of things was firstly the prime sort of targets as the deal. That you've done with cell gene slash ban was in oncology. And I'm very interested in in oncology as a field now and secondly the the technology platform itself was extremely relevant to wear. This type of therapy is going in the future. During the course of my work over the past five or six years is the missing link him up also on gene. Therapy has been the ability to control Sakai activity And the let's due to have gene and cell Pho- piece modulated in the same way that you see with a typical small molecule truck whereas you change the drug You get get greater effect but now it will do to make both seven gene therapies for swimsuit to changing the dose of small molecule drugs. They can want you like no regulate. Yeah I think that's really powerful meanness course when something like that touches you personally. And you're in the healthcare industry than I completely understand that murder that you'd have blocked to learn Warren lent to pursue that area. It becomes like a passion even to get more involved so I think that's something that's our whole that that happens to people are and really a lot of times when you look people. Stories you find there's some impetus here move them forward in a particular area nearly drive them to succeed in a in an area that control personal experience. No actually. I think you're right the treatment that we give patients today. For example in oncology settings is quite. I'm GONNA use the word crews in some respects that some of the medications used for being used fifty or sixty years ago when you look at the success of self therapy's of had retreated the Some forms of cancer would be Cox for example the outcome and some patients these are the sickest patients nations They've been through therapies. Fail does but what you see is an many passion complete response which to demeaning means good if we can stop begin to make this Touch with more accessible and using the wide assessing but at some point hopefully my lots on so she's would become the mainstay of treatment rather than the lost points a cold during that treatment. Yes I think that's very accurate and I think you know. It is kind of amazing. When you say about therapies today that they're definitely does seem to be a crucial element to it just because of like you said it's the same thing we've been using in it's time for some innovation? I think we're starting to see that Cartesian and the cell orange therapy solutions that currently perfectly into my next question which was I wanted to talk to you a little bit about how cancer therapy has evolved in last decades and then if you can elaborate a little bit on what we've touched on in terms of what are some of the most recent Advanced in his face. Soften the probably in the last decade. Also the the main at bounce has been the impact to school. Immuno oncology approaches which used the body's own immune system in Palo that two sites choose. We've seen the success of the checkpoint inhibits as strict simple which have evolution is the way that Some Kansas and treatment trees and also last week and I think he was on the BBC where melanoma patients for example and ask them by being will be on five years and fifteen fifteen twenty years ago. That was an outcome of definitely both expected. Just because of the success of these checkpoint inhibitors this change in the treatments offset in Kansas and the success or the treatment of Sun Kansas. It's immune on college. He space which has been the most significant development And then of course menaul seeing the successes of the The county therapies as well and I actually believe we just started his revelations relations so that stood alone whites ago but for me. At least it's the last ten years from the impacts of immune oncology. That's probably the biggest thing yeah it's quite astounding. The results that they're seeing that space. What are some of the limitations of SARS gene therapies? We've talked about the promise. I don't know that. But what are some of the limitations that we still have that. Were still working through in your mind. Well I think that Today for example one of them is most of the cell. Therapies used to treat passions. Tense Day all what's known as autologous. Which means that you'll using the patient's own cells taking it out Engineering them and then giving the back to the patient once. They've been Purified Kaci for example simple therapies. Do views patient's own cells and what we're starting to see now is because of the expense of that she being the president's those sales and manufacturing before you give them back to the patient as a couple of things so it takes a while for that so the patient can be waiting weeks given the therapies and secondly autologous cell therapy so expensive to to make and I think one of the things that could happen in the future as we start to see a shift towards off the shelf therapies and those will be more accessible. I'm probably more affordable healthcare system. Something that's one. Limitation is just as a price point of these therapies and the second thing has been something. We've been focused on here at the city. In which happen we actually make them more controllable and make them improve their performance. The cogs he's been so I need sales papa cell therapy by giving the the cells sort of more robust appearance right so allowing them to persist longer All Up to survive much longer within the micro environment and we can do that. HAIRED OBSIDIAN bought applying at technology to control signs of kind Activity within cells the used to treat cancer. Also using immuno oncology top approaches. I I'd like to hear more. I'm could you describe your platform than how it can be applied specifically on onto what we're talking about in terms of improving assange therapy. Yes show the platform here was actually developed. Initially by professor I'm onless spaced and Stanford University and Tom Hood's enabled to create a technology technology platform. Where you could control signed connectivity using Commercially viable small molecule drugs on the way he was able to do. This was to Basically genetically engineer cells which is a fusion protein. Like while we use I L two. There's an example into into and then what he could do is creates a fusion protein that was tagged with what we call the drunk responsive to Maine and so that drug responsive faced fusion protein is manufactured overtime by the cell resulted this genetic engineering and it's essentially really randomly structured so the proteins in a sort of random folding Appearance on the cell recognizes those being mis folded and takes off to the purchase on all the trash can in the cell and those kinds on attack to when that fusion protein is exposed to a small molecule drunk responsive domain phones is properly in the presence of small molecule and then the sides kind can be used by themselves or it's no compasses and as soon as drug molecules legit stuffed with disappeared from the from the circulation and the protein starts to go back into its natural state which is to be taken off so the trash. Kansas allows the drug is there. We can rescue the Unfolded proteins if you will and turn them into two proteins uh-huh and when the drug is gone goes back to his resting state which is normally and Because of that we can control the time. Course all the action of the protein we can also control the level because it does change depending on how much drug he puts into. The systemic circulation says controlling so activity or protein activity by administering a small molecule truck. And it's time that anyone has done this particular with commonly-available small molecule drugs that have been widely used in therapies for decades. Sort of very safe The they drug we actually have right now is to set the mind which is a very old diuretic drug with a very big dosing rain. So it's perfect for trying to do And we've seen that we've been able to control numerous sides cards and numeral numerous different cell types using this drug and in combination with the appropriate sponsor. Tonight that's really interesting. I'm unique approach. I wanted to kind of dig into that a little bit. What differentiates the platform choir from other comparable companies say? That's a very good question because other companies have tried to turn on and off protein activity but so he's approaches a Jimmy thing an all or nothing approach attends on and then you have to turn it off. I think the big differences us this is the we can not only just turn the protein activity on but we can also. It's more like a dimmer switch on off. Switch the best way to think about technology because we can multi like that protein. You'll science connectivity often down on a scale of say once ten And it's responsive responsive to the small molecules. I think that's really level of precision now that we can apply to both cell. Gene therapies is very similar to do traditional Those just WanNa talk pharmacology with Older drugs have been used in the clinic for a long time. We've talked a lot about of the cancer therapeutics and the approach to cancer therapy. But I wanted to find out a little bit about How your platform platform could be used for other selling gene therapy? Applications are there other indications that you're looking at and having that be applied for example whole Right now the application on just describe pry was too much science kind activity by within the cell by tanking with these trump responsive remains we can also tag transcription factors with this talk of technology which allows you to control Secreted protein output from a cell. which takes us then into being so much like like gene therapy? So that's an interesting area for us to apply this technology to Providing more precise those control with the gene syrupy Which is actually one of the biggest challenges facing that type of technology and We can also see the applied to for example Disorders at the back of the highway could apply this technology to be so to manufacture grow taxes and things like that which could potentially take the applied to patients with macular degeneration and a future where you control the amounts that are back to being produced At will else they wouldn't necessarily have the thing crystal time. But you might just won't soon ship a patient to tablet. They can use it say once a week and then you get. That protein expressed flunks a week. So that we can so that the cells all the ways exposed to growth factors One of the recent regulations and pulled all the applications vacations we're talking about is because if you continually expose So things like Iot Fhu. I'll sit inside kinds. which which is y'all Houston settings than the The cells and cells talk to wear out continual exposure to those taxes. So not being up to give them into mitchely now. She's getting up just best to control. But also the cells over the long time developing robustness on the mall sense. It's too and More active within Within the size of action intended full so I think that we can see numerous application. So I can see this technology benching being used and things like also disorders matching its generation. I disabled as Gene therapies we can see them being used and things like replacement replacement therapy which could be ends on replacements or for example in the future The list is very very long. And so I I I I try. Focusing on oncology where the use of cell therapeutics to treat patients. That Schumer has been tremendously successful us over the mode of good clinical outcomes onto we believe we can make those. That really sounds great. I wanted to see if you could share a specific example example of a current therapy that you could that could benefit from this approach. You touched on it a little bit in the last answer but is there one that you can have in mind that would be really. We have a perfect fit for this actually This is breaking news. So you've actually also the question of the right day we're making some Wall Street analysts after this this discussion we're having brandy but we've identified the first Clinical programs we we've been looking at Notice chew infiltrating lymphocytes which Missile Technology has been used to treat patients. We've things the head neck cancer and melanoma And this is where they take the patients on Memphis thoughts from within a chamber and then grow up and then get back. The patients in these lymphocytes already trained to attack the tumor. What you do basically is just to get back to the patient and much greater numbers they can actually do a job and to use the patient's own T. cells? They recognize that she already. Because it will ready. And they've they've identified the necessarily antigens clinically. They've had great success in Diseases like melanoma had cats and so What we believe is we can make those cells Moore wrote bossed for use in patients by giving them their own? You can choose on demand by giving Molecule one example. Why would we can write dispatches today if you go into a clinic and you have to be treated with this till therapy Manufacturing process can take weeks to complete for you to give it back to the patient patient secondly patients in this setting Cells being given to them. They're reponse promos known as into Therapies therapies high dose Interleukin two and that's therapy is quite challenging a little bit because it does have a number of side effects and and there's the number size in this country that Able to Minister I'll suits who cancer patients because they they need to have support Rooms necessary just in case. There's a problem with therapy But we believe we should eliminate over Juiston for that oil oil to dosing By giving the sells their own sauce about song to kind of similar sites. Kind which means that. The patient doesn't doesn't have any systemic exposure to that size to con- it's so the needed that she has sought to come and present and they can go to work on their own without leaving the patient additional doses of constantly keep those cells alive the longest so we cannot eliminate that can do that. We can for example the treatment more accessible because you can treat the patients that don't need those filed suit supports necessarily for treating patients We can actually remove that piece of the equation that she making a much better people as patients at the same time providing those cells with their own POW pack that we cannot those far on demand that they small molecules the more. I'm going to be using Ruby centers on my one We can also make those sales more road. Boston persistent of the Cheema seismic spent more active and chainsaw as well else that'd be kidding chambers. And secondly they need that sought console which some patients have difficulty Managing about doc so patients central patients who would normally be eligible for shelter. These go into that because of the conducive science concepts meditation. I picked the right day to ask that question. That's great Wonderful News it's really interesting and look forward to reading more about that. As that progresses to Kinda follow along or get back to the cancer piece that we were talking about. I'm wondering if you could talk a little bit. Broadly about what role you see for precision medicine in cancer care overall. Will I think it's it's something that is going to be full. The future and Forty seamless accessible college. He's just about to syrup. Approach that we're looking at here They're all the cell types for example is natural killer cells where if you to be able to and help them persistent grow In the body and control that persistence with this kind of known as I o fifteen which we at. Let's see all working on their city. We can control that activists you I'll fifteen so. For example we can control the activity activity of natural killer cells and improve that for patients and the number of cell types that we can use to treat reeks patients with some Schumer's and I think it just comes the approving of the patient the become eligible for cell therapy treatment because of precision medicine. But also you're gonNA see a broadening in touch the cells that can be used to To treat these patients which gives the more accessible I think ultimately making this medication more accessible for patients go that we want to achieve. That's great what is next for Obsidian near term and then you talked about obviously the this on your big announcement and what's going on today. But what else awesome horizon. What other indications are you looking out? What's coming up for the company? So that's a good question One of the Nice things about a platform like right. This is it. It sounds really we build a lot of opportunity to choose Diseases where we believe that Regulating seventeen so he's can make a difference You know you can look at things like you can look at Mulji We can look at other cancers dances apart from the ones have been looking at right now. so I I can see that. There's quantum number because he states that we can We can talk at the White House. Think about technologies as a pro team that you can make we can regulate it And we can also regulate it than any cell tide. Whether it's the patient's own cells or describes Elian off the shelf sell time so we have a very drooled platforming centers of APPLICA- ability. Actually triaging that so those opportunities you set right now. It's not just by her grounds for our own internal pipeline. So I think you'll see more announcements about what we're doing with our own internal pipeline. Second thing is we all in discussions with numerous other companies in the SANJIV. Alan team coach he space about trying to ply type outfit today technology and vice versa. Eventually we'll be doing selected taller ships as we move toward the WHO wants to focus on what's important full pipeline as much as possible We making great progress to that still slash ashby. Ns Lines right now. So I expect you'll see some Milestone updates on on this removes towards and then a company they like that just require financing so we expect to be doing another financing in the next twelve months prior to taking this company out to and probably the public markets about two to three years older company which you set internally to be in the clinic with this technology well she by twenty twenty two so that's not just I would see as a way so we're very focused now on taking this technology into the clinic firm you too and also on great goal to have and wish you the best in that where would you be in five years and kind of is there precipitate strategy that you're taking moving forward. I know you talked about. Obviously there's so many opportunities of this platform is there anything specific that you will be utilizing in terms of strategy to to manage all that I'm a huge believer in strategic planning. So we've the management team working on that right now The opportunities that we have in five years time I think will include Having a commercial products on the market to treat patients with But Kansas so this is the program that could be coming within about two years time. uh-huh moving five years time should be at the point where it could be used to treat patients in a commercial setting so we have to stop thinking about how we GonNa do that We gotta be broiled NIA pipeline. That's for sure I think we'll have multiple partnerships in place in this country and potentially other countries as as well and we should probably be a publicly traded company at that point in time the company's. I'd like to look out as sort of role models for a company like city where they have a really strong platform play develop products include companies like L.. Nine on which is a local company. Me Here is quite famous company and we met you just picked up ahead of human resources. Came to this from El Nine and so we're going to build a team aimed to replicate does success in the next few years. I've really appreciate your time today. This is really interesting. Thanks so much for sharing your story and Obsidian Story. I just like to close by asking. There's anything answer to like to add for our listeners. Before we say goodbye well if the bank's prime one thing I would say is that business is all about people They all the county of life and we built a really strong team here. I'm sitting. We've had a lot of new team members. Join us in policy amongst and everyone's really energized and working very hard to bring this Technology she into the clinical setting as quick as possible. We have an obligation to do that. And we've assembled a team that's really moving very quickly now. Working very hard to make that happen The second thing is that you know these benches like this require Somebody would willingness to place bets. Let's on this type of zone out season in fantastic tools atlas have been tremendous we have google ventures in the company. Anthony AMGEN TAKEDA BUT SEX. I mean and others but it's actually really nice to have a very high level support not just from the tables from that board as well if you're working tonight. This is really about thanking over people to help put into place On the team here sitting as well that we're here before I joined Able to develop his platform to a point where we were ready to pull the trigger on identifying the candidates and development development programs and some of those guys have been hip in eighty four years and to now they're saying the dream son inch reality which is just tremendous. Padilla came about because awkward. That's exciting to be able to see. All your hard work come to fruition. I'm sure for those folks. It's just really thrilling. I couldn't agree more. It's all about the people so I think that's great Thank you so much again for your time today. I really appreciate it and look forward to keeping up on obsidian and following what. You're you're working on moving forward. Thanks Brandon Good Luck. Thank you thank you for joining us for this edition of the cell culture dish podcast to learn more about this and other stem cell bio manufacturing related topics topics please visit us at. WWW DOT cell culture dish dot com or for downstream bio manufacturing topics www dot downstream. ON-STREAM COLUMN DOT Com.

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