Quality Grind Podcast

Navigating Cell & Gene Therapy: The Quality Path to a Bright Future

• MEDVACON Life Sciences • Season 1 • Episode 26

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In this episode of the Quality Grind Podcast, hosts Joe Toscano and Mike Kent conclude their four-part series on Cell and Gene Therapy with guest Ray Kaczmarek. They discuss the importance of building relationships and implementing solid quality systems across the development lifecycle. 

Ray shares his pragmatic views on ensuring quality and compliance from early stages, the significance of robust training programs, and the future prospects of the Cell and Gene Therapy field. Tune in for insightful discussions on maintaining regulatory standards, managing deviations, and fostering a culture of responsibility and innovation in the life sciences industry.

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Ep 26: Navigating Cell & Gene Therapy: The Quality Path to a Bright Future

Jessica Taylor: [00:00:00] This is the Quality Grind Podcast presented by Medvacon. Conversations that go beyond compliance. Sharing insights geared toward helping you navigate the everyday grind of regulated life science industries. Here are your hosts, Joe Toscano and Mike Kent.

Mike Kent: Welcome back to the Grind everyone! 

Ray Kaczmarek: I think Ray hit the nail on the head in Part Three. Having a solid relationship with all your partners, whether they be development partners, cogs in your supply chain, or anyone else who's helping you fulfill the needs of your organization, success really starts and ends with clearly understanding and agreeing on who's going to do what, how, why, and to what level of expectation. 

We round out our series on Cell and Gene Therapy in Part Four, where we identify strategies for implementing [00:01:00] quality systems and a compliance mindset across the development lifecycle. Now, Ray has a bit of a different approach to this, and I think you'll find it very pragmatic and even refreshing, and to me that's why it's effective and sustainable. We conclude the episode with a look forward to the exciting future of this dynamic genre of life sciences. 

Mike Kent: Let's rejoin the conversation with Ray, Joe, and me in Part Four of Cell and Gene Therapy here on The Grind.

Joe Toscano: One more critical area for you, because I really think we hit some really great points, right? Things people need to be cognizant of, look out for. Again, people in industry are gonna know this, but there's a lot of people looking into it or new to it, right? So hopefully a lot of this is helpful and gives a different perspective on approach. 

But then I go to the last thing, quality and compliance, right? How do you build that in early? How do organizations ensure that they're getting that foundation in [00:02:00] place? Because as we know, that comes back to bite people in later stages in a very big and very expensive way. 

What are your thoughts and recommendations about how organizations can implement that process early? 

Ray Kaczmarek: Yeah, it's, it is interesting because it's gonna be... I've come in where we've had too many people... 

Joe Toscano: You gotta be very careful... 

Ray Kaczmarek: ...in the Quality 

Joe Toscano: - right? yeah 

Ray Kaczmarek: - organization. 

Yeah.

Well, when I have more Quality people than I actually do have in Manufacturing and in QC, it's probably, it is either a really big red flag that I've got a lot of problems and I'm having to figure things out, or we're usually off or lopsided. That's telling me our processes are really not being adopted by the individuals that should be using it. It doesn't, again, it doesn't mean that I have bad people. It probably means I do have bad processes, because we're having to augment a lot of people with bad processes typically.

Mike Kent: To make up for it. 

Ray Kaczmarek: So when I see, when I first come in... so you're asking like when I come into an organization and I'm the guy at the [00:03:00] top, of course I'm gonna look at the balance of my organization because it tells me where a lot of problems sit, -- Right -- just because how people are utilized, how my organization probably will run, where the issues are there. So like when we start seeing that, where do you balance it? 

But I've also come in to late stage companies and they're trying to manage it with two or three people. I can tell you there is not enough time in the day for that to happen and be effective. And you can outsource to whoever you want to outsource. That is not an effective model when you start getting towards those submissions, the commercialization and what it is. The internal quality systems still have to be run even in a virtual company. Yes. You still have the other side of the business. You still have oversight requirements that may be outside of the internal requirements of your virtual company. Okay. So there's different skill sets and trades that are actually utilized and what you're looking at. So to have too little is the same thing. 

So what it comes [00:04:00] down to is, I've always looked at this, I try to grow Quality based on the needs of the business. And I may have a large Quality group in an early phase company because I have something that's later stage and something that's early stage. And the amount of work that's going into the early stage clinical does require Quality support. And early, early piece, sometime it gets overlooked and it has the Regulatory interface with Quality there. And that gets overlooked as well, is do I have a good Clinical-Regulatory and Quality-Regulatory setup that's gonna feed us into the later piece? They're almost separate jobs sometimes. Because when I get... 

Joe Toscano: ...But equally, equally important though.

Ray Kaczmarek: Yeah. Well I'm, if I'm manufacturing for clinic, but I'm also manufacturing potentially for a PPQ or moving the other portion, that's a full time job that it's not gonna go away anytime soon. And it's just gonna continue to build as I make more lots. And this one right here is sort of hit and miss, [00:05:00] but the demands and some of the earlier ones are quite different than demands in some of the later programs, but you still have to do change management. You still have to understand the revisions of the protocols. We still have to have, say, clinical material supply. We still have to release things. We still have to, all those things are still there. They may not be sitting in that realm right now because that realm is going off to start focusing on commercial supply a little bit. And you'll see, start seeing that.

So it depends on the stage, but you're gonna, you're gonna build it. I know you can contract some of these roles because they may not be full-time roles. So you could have Clinical Quality as a contract. You could, you can find very good consultants to do this work. I could do it partial, partial time, and integrate well. I've seen at early phase companies, even at times have some of their Heads of Quality are contractors, right, contract people. But what they're gonna have to do, though, is somebody in that organization still has a responsibility for Quality because a [00:06:00] consultant doesn't, right? 

So you've gotta map that, and understand well that if they're taking a charge and your CMC team and all that, is understanding and following things they're putting in, they're all quality players in a virtual company. They're quality players in their own standup company. That's just the roles could be a little bit different in a virtual piece because you have more of an oversight angle where you're more of the executor on the commercial side, or you're in an ownership side, right? So, you have the ability to potentially have a little bit of a hybrid piece there. But again, they still have to be trained. They still have to have a role, they still have to have a reporting structure. 

So they can carry Quality in an oversight position a little differently because they're actually doing oversight when they, right, when they go in and watch somebody manufacture, right? So they better have their quality hat on. But they have to have their technical hat on, right? So they can do a dual role. When I'm supervising, I gotta have my quality hat on when I'm doing that. When I'm supposed to be [00:07:00] exercising, yeah, I'm an operator, I'm supposed to have them both. It's just a little bit easier when I'm on the outside looking in and I'm observing than when I'm right there in the middle of things to realize what's right in front of you. Right? Sure. It's the whole, you know, when you're right in the middle of it, you miss the obvious, right? When you're outside in the periphery, you can start seeing the training wreck coming and it's much easier to go like that. You've got one or two choices, right? You could either nudge the guy beside and you, "Hey, watch this." Or you say, "Oh, hey, hey, have you guys ever thought about..." and usually prevent it. That changes, and usually that changes where it is. So I guess it's whether or not they like you or not is whether they, they let the train wreck happen. 

But the reality is, people want to come to work every day and do a great job. -- Yeah, absolutely. -- And not only that, they want to be good at what they do, but they also want to have good relationships with people. So the reality is most of those individuals will always choose the right [00:08:00] thing. And when you have individuals who choose not to, they just told you that they don't want to be on your bus. It makes it very easy. Yeah. And again, they self-select. You rarely have to actually fire somebody in our industry, they usually fire themselves. 

Joe Toscano: No, understood. And I don't want to bring up the next piece 'cause Michael will get very excited and, I'm gonna need to temper that.

Mike Kent: Never... must... Never do I ever get excited about anything. 

Joe Toscano: Exactly. Lemme, well, you know exactly where I'm going. You know, the importance of training and even talent acquisition. And, Mike, you're not allowed to speak.

Mike Kent: Wow. Wow. Wow. There goes the fun part of the podcast, right? 

Joe Toscano: No. Again... 

Mike Kent: I know we have Ray here, but... 

Joe Toscano: I know it's, I know it's a passion of yours. So, but it is so important, right, the training aspect throughout the various phases, regardless of the type of organization. A lot of people do it to check a box, right? Mike and I have this conversation all the time, and [00:09:00] it's a shame because it's such a lost opportunity.

Talk about the importance of training and the impact that it has and good training, not just, you know what, let's meet our yearly GMP, everybody sit in the room and, you know, read a bunch of stuff and then we're, we can all check the box.

Mike Kent: I know you said I couldn't speak, but that's the first check in the process, right? I mean, how you set up that environment and the expectations around training make a huge difference as to how it gets executed. 

Ray Kaczmarek: Yeah. It, and, this is also a tricky one to answer, right? And I, and I... 

Joe Toscano: Oh, you're not on the spot, don't worry. 

Joe & Mike: We're not recording this. Yeah, yeah. It's okay. We're not recording this. Yeah. Nobody will hear this, just the three of us. Yeah. 

Ray Kaczmarek: So here's the thing that over the years I've struggled with, right? I've had large training organizations and they've been successful and not so successful. I've had super small training organizations and they've kicked the pants off [00:10:00] my larger training organizations, right? It is really, it's the elements of training and understanding, and then how can we roll it out and execute, put the importance on it. It's like you were saying, what's the importance around it? 

At the site level, when I'm talking with anybody, whether it's a site head or department head or whatever else, what they would always hear, and they'll always hear today, even the other side when I'm working on the technical pieces, we do things safely, correctly, on time, every time, in that order and non-negotiable, right? You know, and that really comes down to, yeah, I just gave you cardinal rules, right? Safely and correctly and on time, and those are the things we're gonna do. But you never, ever put a person or patient in harm's way. Right? You're always gonna do the right thing thing, and damn it, go do something. Right? You're gonna do it, right? That really requires guidance, organization and training. So [00:11:00] training now becomes essential for you to do it safely, correctly, on time, every time. And if you're following that and you're thinking about from a compliance aspect, well, if I can't do this on time, right, every time, what's my gap? And that's where the operational discipline comes in, and that's where the guidance from the leader starts.

It's thou will do those things to train your people. If they don't know how to do it, is it a process issue or is it a people issue? If they're not trained, at first it's a process issue. If it continues to not get fixed, it becomes a people issue because you're not fixing your process. 

So we understand trying to put that in, well, how do you get that as part of the integration? When I'm fully commercial, it's so much actually easier than when I'm starting to start from scratch and build it, right? 

When I already have something in an SOP and a training history and somebody knows how to do that, well, it is around putting a process of [00:12:00] qualifications, standards, work standards. Sometimes it's training a process and then forcing people to do it a few times and then that actually starts to take off. And that's why I can have a smaller team sometimes just kick these large teams because the ownership gets put down into the SME level. And they help with these SMEs maintain the records and everything else, and these guys train you to a standard. And then they fill out that, they turn it in, and that guy helps them out. And then these large groups may come down and say, well, I'm going to teach you how to gown. When was the last time you actually worked on the manufacturing floor? Because man, you barely could put that shoe cover on. Because your balance is off and... 

But it's like saying, but back to the thing is, it's a delicate balance. But there has to be a mandate. And that mandate does have to start with, something like, 

I look at it safely, correctly, on time, every time. 

And my, it made it easy because it's... 

To me, I need to tell the guys it's cardinal rule. [00:13:00] That's it. If you do something and you violate safety, that's probably your last day working here. If you do something and you knowingly violate compliance, it's probably your last day working here. 

Again. But again, I also know that mistakes happen. So things do happen, but again, if we're not paying attention, you probably don't want to work in this industry. Our industry is very serious about what we do. I'm signing my name and a batch record because I said I did that step. Which means it may have a weight and a balance, which means I would expect that you did that weight, balance, checked it, saw it, and whoever has the verified by it was right there, or actually took it and verified the weight again. One way or the other, there better be a verification. Either it's through observation or physical check, right? Because it's a 'verified by'. Just because you got it out the door, if you don't follow that step, that is a serious issue. Right? Not just from a GMP perspective, but now I've just given you a cardinal rule.[00:14:00] 

This is what I tell the people. This is what my expectation. So when you start with an expectation of compliance, but you also say there's no... just because I have to be compliant and safe doesn't mean it's an excuse not to do the job. That's pretty much what I'm telling you. We'll find a way.

And that's it. Find a way. And that's where now my entrepreneurial people, my go-getters, people that actually love what they're doing, they start to come out of the woodwork and they start to show you ways to be very efficient, effective, consistent, putting those into procedures, fixing your processes. And you can go from a couple of hundred of 

'in our control' deviations one year to, in a multi-billion dollar vaccine process, down to three, and in an entire year, right? Because of of the people, not because of the leadership. I give them guidance, but then they took it and we worked together to figure out how we're gonna flush out bad processes and make people effective. Okay, and that's it. 

So I think it's, a hard thing to [00:15:00] answer because I can tell you what I do. Do I have to have 50 training people? No, I have to have at least a training mentality and a dedication to doing it right. I will tell you the earlier I can have somebody looking at some of these systems, the better off it is. And when I'm in a commercialized state, I've gotta have somebody that's responsible for Training. I've always found that is if I look at it and say, well, you six people are responsible, no one's responsible. And that's what I've found. 

Joe Toscano: And I agree, you need a point person, or in large organizations it's a department. But I think where there's a failure point sometimes, and we would be called in to help because it's a failure point, is that person responsible doesn't know how to conduct training. They're not very good at showing, demonstrating and teaching. Not everybody's a good teacher. A lot of people know how to do something, but they don't know how to demonstrate it well. 

Mike Kent: They may not even want to...

Ray Kaczmarek: ... A difference between a trainer. Yeah. But to me, there's a difference between a trainer and a process. [00:16:00] 

The best processes yield the best results for training. So I can have a trainer on every shift and, multiple trainers on every shift, but if I don't have a process that feeds right training material...

Yep. 

...how to train that piece... I agree... record it and how to do it. What's my, piece to cross reference it? More importantly, this was the thing that I remember, it was, it's been over a decade ago, conversation... 

Joe Toscano: It goes quick, doesn't it? 

Ray Kaczmarek: Yeah. I was gonna say, had a conversation around, well, how do you know the guy that's getting ready to work on those batch record steps is trained on everything that's in there. What's your printouts look like? How they, do they match to your records? Well, and this is just basic operations, right? But again, process, right? What allows the supervisor to truly know they are up to date to the latest compliance?

Okay. We can laugh more about it today because the way technology has gotten so good. Right? I mean, I didn't have these things integrated into iPads and tied to all my systems and [00:17:00] all that, where I didn't have that, you know, 12, 14, 15 years ago for sure. I had a system, I had to go out, print it out and check on things individually. But now technology's enabling us to be a lot more compliant with data and information. But again, I still have to have a process. We still have to be able to set it up. I still have to be able to train somebody to be effective. So that, again, I look at it, if I have nobody in charge of training, there's nobody in charge of training specifically, which means I can put 10 people there, it's probably not gonna be effective. I need to have that centralized point, which does gimme that. And again, if I find, bring a consultant group in or somebody else to in to establish that piece, there's fine with doing that. But somebody still has to have ownership to maintain it... absolutely... or it's gonna eventually fall apart. But the process itself drives good training.

And not everybody can train. They want to train, but not everybody can train. It's okay. [00:18:00] It's not a failure. I don't tell somebody, well, if you can't train, you can't be at this level. Right. Now if you're not a good trainer, don't train people. 

Mike Kent: Don't put that individual on that spot in the bus. Exactly. I'm a manager, therefore I should be expected to. But... 

Ray Kaczmarek: Not if you know if you how to do it. 

Mike Kent: Exactly. Or you don't want to do it. I've had scientists get promoted right into leadership, managerial roles, and they'll sit there and they'll fail. Not because they're not brilliant, by the way, but it's because that's not what they want to do.

The other piece of it, I know you told me I wasn't allowed to talk. Okay. But the other piece, and you brought it up, Ray, so I feel compelled to jump in. I'm a co-host on this podcast too, right? That's what the, that's what the title says. 

Joe Toscano: I, I just know how much you love this topic... 

Mike Kent: So I'll keep it brief, but the piece that I think comes out [00:19:00] of your comment about the technology. We didn't have access to information anywhere at any time under any circumstance. Because there's a record in a system somewhere and a checkbox next to it doesn't mean the individual is not competent now in that moment. So that's where I think that... and competency has technique and capability, but it also has an element of decision making as a part of that. 

So along the lines of training, I'm curious from your perspective where and how you've been successful throughout your career of influencing individuals to, when push comes to shove, this doesn't feel right. I know I'm supposed to, or I think I have a question of a concern. Doing the right thing and making the best [00:20:00] decision in the moment. What in your mind is leadership's role? How do you create that environment where people are compelled to make the right best decision, even though it may be the most difficult thing for them to do in the moment?

Ray Kaczmarek: Okay, so this is gonna, this is where the HR professionals have to shut off and go click elsewhere. 

So now what it comes into, and then, I mean, just in the last company that I ran, the first thing, I came out and I was talking to the entire team, the first week I was there, I said, listen, in our organization, we only hire fully developed adults.

And what does that mean? It means we don't have to tell you what's right and wrong, when you need to be at work, when you should go on break, and we don't need to tell you that you need to train on something before you do it. You're an adult, we're gonna hold you accountable. If we're babysitting, [00:21:00] you're not gonna work here. So first is I have to empower people to be people. And let them know, I expect you to make decisions and look at a process. 

The second thing is, I like to give things like, for, especially for site design, simple things like the cardinal rule, like safely, correctly, on time, every time, right? If you don't feel like you can do that, you have a compliancy stop, you have a safety stop, you elevate that. You talk through that issue. And people, when they start to use it, and they're not punished for using it, right? Yeah. The next thing you know, it's, oh, we're gonna fix this problem and we can talk about it. Well, I really can't do this because I can't get this to vent off. Well, I'm glad you called and didn't just try to take the thing off because it was 10 pounds of pressure on an 800 pound tank, right? Yeah. That would blow that sight glass through the ceiling, right? As long as you're not in front of it, we'll laugh about it later, right, but, because, but it's, it is gonna be a big hole.

Joe & Mike: Yeah. 

Ray Kaczmarek: Yeah. So the good news is they'll stop. They ask the [00:22:00] question, I can't get to do this, I can't get this cycle off. But rather than just thinking, oh, I can just crack that and go on. Oh no, they stopped. They made the, they made a, an adult decision in my mind. You do what, you did what you should do if you're acting like a fully developed adult. You look at it and say, well, that's not safe. Why would I do that?

Okay. And it's not pushing at all costs and not doing that, and just being a drone. You can't be in our organization. 

You listen to anything that you have from compliance talks and all that, they expect people to be able to understand what they're doing in their jobs. And that's training, that's the competency piece we're talking about. Yeah. But first it also starts with making sure you're looking for and bringing in people who want to do that type of work. So as we start looking at those things are two things that I've always, that people hear me say a lot. 

The third thing is it's, it is really around, forcing leaders to understand their role and making sure their employees are successful. And some of that is just looking at [00:23:00] simple things like metrics, right? So when I look at deviations, and then classifying deviations a little bit differently: is it in my control? Is it not in my control? And if it's in my control, what about it is in my control? Oh, well, you know, the batch record was wrong. Guess what? That's an 'in our control' deviation. There's no reason we should have a deviation for a bad batch record. That means it's either poor planning, didn't understand it, different process change, whatever it's, but it's in our control.

How do I eliminate those? I gotta do RCA. And once you start doing human factoring on 'in our control' deviations, and you start looking at the processes that are leading up to it, a lot of times it is process driven, right? More than it is people driven. Yes. They make bad decisions in bad processes. 

Okay, so now I'm back to, hopefully we're thinking about human controls based on doing RCA that we put [00:24:00] in place by looking at human factoring issues and those types of compliance, which drives the safely, correctly, on time model. 

You make a mistake, hopefully you learn from it. If you're, if you bring the mistake up, you know, there's things where if you're concerned about employees not wanting to report things, you know, for a deviation? Whether you want to call it an amnesty thing or not now. I looked at it a different way. Some people would do it that way. I said, well, it's actually the other way around. I hire a fully developed adult. So if something's not right, and you know, you're not acting as a fully developed adult not reporting it. Okay, I can't have you in my organization. I, that's where we look at it. 

So I look at it actually a little bit differently where it was like, well, you know, I don't want want to say anything. No, no. Then you're not a person in our organization. And it's okay to say something and it's okay to say something to, say it was you, Joe. And Joe, I saw this and this [00:25:00] didn't look right. Let's talk about it and let's go talk about it with the supervisor. You know, this is what what happened. And you can't be a punishment environment. There just needs to be a discussion. People make mistakes, it's gonna happen. And sometimes at two o'clock in the morning, you hit that spot. You may not realize you're writing your name sideways on the paper until 

Joe Toscano: Right. 

Ray Kaczmarek: ...an hour later when you look at it and go, wham, did I write that? You hit that stage. And hopefully your buddy beside you saw you there and fixed it and ushered you off and said, you need to go take a break because you're at a point where you're tired. Yeah. Because your body's natural rhythms, they're off. It happens in off shift. I don't care what anybody says. You see it and it, it's not always the same person at the same time, but when it hits, somebody can have a bad moment. Even on a day shift, you've been working there on and off for eight hours, you can hit a little spot where you're just sort of in front of the controller. Just didn't hit the button right away. And the other person's, "Hey, why don't you, come in and break", that's being buddies. But it's [00:26:00] also you now self-reflecting and trying to figure out how to stay out of that. Are you working too hard? Then having that discussion, Because that's again, the supervisory piece that's rotating people through. It's keeping them fresh. It's understanding your teams. That's the human factoring elements that aren't in play when you start seeing those things going. 

Mike Kent: Right. 

Ray Kaczmarek: Right. That bad process and management leads to poor decisions. 

Okay. So, again, looking at human factoring a lot, getting some rules and guidance, which just allows me to basically preach, right, and you know, same thing. I tell people, our industry doesn't fire them. They usually self-select eventually and they fire themselves if they want to get fired. But a lot of people really don't get fired. They usually quit.

But the reality is, doing a few of those things, they usually decide very quickly that's not the business they want to be in, and that's okay. It's not for everybody. I don't judge you, you looked [00:27:00] at it, this is not for you. That's fine. That's about being a fully developed adult and making that realization and decision for yourself. And okay, yeah, that's the thing. Go do something that is aligned. 

And that's where I always, I would tell people, especially as I've gotten higher in executive levels. You know, some of my senior leaders where they're, where they get into it is, you can see if they're not happy. You'd be like, you have those conversations. Is this really what you want to be doing? Because if it's not, then let's talk about what you want to be doing. And it could be another seat on the bus in the business, or it could be that it's not the right bus. 

Well, if that's the case, then we both need to be responsible about the discussion. That gets us back to, yeah, maybe we have to make a change. But the reality is, usually you have that discussion, it's usually something very simple and they're right back in the seat they were supposed to be in. Yeah. If you don't ask, you don't get, you don't get any information.[00:28:00] 

Joe Toscano: No. Understood. 

So one last question for you, if you if you mind. To me, viral vector, cell and gene therapy, exciting area, right? Look what's just happened in the last seven or eight years in that marketplace.

Where do you see it going? What, what gets you excited about this space? The innovation that we're seeing in regards to it? What do you see the future as being? 

 

Ray Kaczmarek: I can't tell you where it's gonna go, but I can tell you what I'm, what I feel like it's going to do in that I'm seeing different types of drug products coming out that can cure very specific things, or in some cases of other things, there's hundreds you know, hundreds of that lead to this outcome. And now they've figured out, well, I can't treat a hundred of these things, but I can treat the outcome. And now companies are looking at it from that sense, right? How can I take somebody who might be [00:29:00] handicapped for life or something like this or what have you? Or they, it's, there's many mutations that lead to this lack of protein somewhere, but it creates this health issue. I can treat the health issue and I can treat with a gene therapy. You may not solve the problem and cure that disease per se because there's still the mutation there, but you can keep that person's quality of life or extend their life greatly, right, by just treating that.

So I think you're gonna see more of this. I think you're gonna see a lot more. There's gonna probably be some platform designs that come out in the next decade that we never thought of. How can you deliver something like mRNA differently? And I mean, can you do it and deliver it in a different manner, which allows it to actually be effective, you know, right away and then disperse appropriately? Do you have to do some of these therapies today? They're talking about editing versus, [00:30:00] you know, and replacing things and the body does it that way. Or even, you know, I'm giving it a cold. Giving it a virus and I insert some stuff. Next thing you know, I create that protein that way. Do I get away from viral type work? Do we get away from different types of administrations because we find out the reactions to the body? 

I think some of it's to be seen. It's definitely not an exact science. There's more about it. And I remember when I first started stem cells back in, it was back in 2011, first time being in there, I was talking to the chief scientific officer and the head of development and we're doing the stem cell. We're finding the clinical result was interesting and that people with strokes were recovering much quicker. And I asked, what do you think causes that to happen in the cell? And the chief scientific officer smiled at me. He says, if I knew that we might be the only three people in the world that knows the answer.

Wow. 

[00:31:00] The reason I don't have it is 'cause we didn't have, we weren't advanced enough in that assay and that science. But it's not that people weren't looking, this cell had been around for quite some time. 

So it's now back to what is it really mean, and how is our industry moving? So I think our analytical capabilities will continue to grow and I think we'll see some innovation come out. What Cell and Gene brings out is a lot of entrepreneurial type people, and it is small classifications. I can treat this, I can do these types of things. But what it also brings is an opportunity for potentially platforms, and in a, and a drug delivery piece. There's more discussions around, we potentially have ways now to potentially get past, you know, the brain blood, the blood brain barrier, per se. How can I actually dose appropriately where I need these things? I look at something like, the theory of something like Lilly's Alzheimer's drug, [00:32:00] right? In theory, if it became a vaccine and I could give you that, I could clean plaque out every five years, and you have that piece, that would be an incredible opportunity. Well, right now it's not that way, it's, I think, a catheter injection piece. It does have a lot of risk to it. But the reality is, it's well, it's well understood, but just taking that and saying, if they could actually make that more like this, and then understand how to restore memory. Because right now it stops, right? We're seeing some products that like, say the cure, but we see a lot of things, we do see products that restore. But we also see, like this product, it stops the movement. But if you've lost a little bit, then you've lost a little bit. Yeah. And then that's where I'm hoping that gene therapy will be better tomorrow than it is today. That they'll understand a lot [00:33:00] more of that. They'll be able to analyze those things, will have more history, patient history. I still haven't seen an eyeball or appear in somebody's forehead, so, I know, but we have had deaths, because we've had things that did not react well. 

I think some of the things, there are still things about our business that are very scary, that can create that type of an outcome. But there are also a lot of things that we laugh about because, again, I can't imagine that I could grow an eyeball on my forehead, but we laugh about it because it just doesn't, I mean it doesn't make sense to us. Doesn't register. Yeah. But again, but there's some very real risks to what we do in our industry. Yep. And we see that. So again, I think it'll continue to grow. I think we will continue to understand those things that have occurred, those risks. And I think I go back to [00:34:00] this, you can't put a value on time. As you know, as I think about it, as I get older, I would like to think that I can extend my time, you can't put the value to it now. What you lose is what you lose. So it's, the biggest thing here is, I think that's an opportunity. It may also lead to other breakthroughs. Yeah. I, don't know how AI is going to play, but there's an opportunity. 

Joe Toscano: Yeah. There's a huge one there. 

Ray Kaczmarek: We can aggregate stuff and do all this, then we can spend hours on that thing too. But the reality is, you're gonna see science, technology, new pieces come in on how we're gonna utilize the newer technologies in our science. And I'm excited about the next 10 years. 

Yeah. Biggest thing is how we're gonna fund it, how we're gonna do it, and how we maintain some of the, right infrastructure that we have. But we have to break some of the old ways of doing things and look at newer ways of thinking sometimes. 

Joe Toscano: And I think the most exciting [00:35:00] areas here, right, are really for those populations that sometimes it was difficult to manufacture products for it because they were small, especially rare diseases.

And there's been so many great inroads there for treating some of those populations that before they might've been too small to really have companies make big investments in regards to trying to find, you know, a way to help those individuals. There's just been so many cool innovations in regards to treating some of those patients and individuals where it restores hope for people that are dealing with difficult situations. 

So, you know, Ray, this has been great. I mean, as always, love talking to you. We always have great conversations whenever we do get together, and this was no different. But, can't thank you enough for spending some time with us and being on the podcast and, and hopefully you had some fun with it. 

Ray Kaczmarek: I always do. I always love talk with talking with you, gentlemen. Thank you for having me. Again, it's an honor to be able to sit and talk to people about things. Yeah. I look at it, we've got [00:36:00] so much more to do yet. Today might be a drug that helps you see, tomorrow might be a drug that keeps you from having heart attacks.

Yeah.

I mean, it's the same thing. I'm excited about what we do in our industry. But again, there's a lot of unknowns. 

Yeah.

But that's, if you're a problem solver, that's why you sort of end up here. 

Yeah, exactly. 

But that's what makes it fun. But also what's, what drives you is the, is to keep moving those things forward.

Joe Toscano: Absolutely. Well, thank you so much. We really had a great time.

Ray Kaczmarek: Thank you. 

Mike Kent: Thanks very much. 

Well, we certainly hope you've enjoyed this four part series on Cell and Gene Therapy. We here at the Quality Grind Podcast would like to thank Ray Kaczmarek for sharing his time, experiences, and perspectives on so many aspects of this emerging field, and for being kind enough to meet us on location to have this tremendous conversation.

We'd also like to thank the good folks at the Courtyard by Marriott - Mission Valley in San [00:37:00] Diego for hosting us to record these episodes. And last, but certainly not least, we'd like to thank all of you for listening and/or watching. Your support and feedback allow us to continue bringing you these discussions with experts from across the life sciences landscape.

So from all of us to all of you, again, thank you very much, and we'll see you again next time here on The Grind.

Joe Toscano: If Medvacon can help you and your organization, we're happy to do so. We specialize in the following areas: Quality and Compliance, Validation and Qualification Services, Project Management, Tech Transfers, General and Specialized Training Programs, Engineering Services, and Talent Acquisition. If you have general questions as well, feel free to give us a call at any time.

We can easily be reached at 833 633 8226 or via our website at www.medvacon.com. Thanks so much, and we look forward to [00:38:00] speaking with you. 

Jessica Taylor: Thank you for listening to the Quality Grind Podcast presented by Medvacon. To learn more or to hear additional episodes, visit us at www.medvacon.com.