The 28th American Society of Gene & Cell Therapy (ASGCT) meeting kicks off in mid-May in New Orleans. The annual event, which is on pace toward a goal of 8,000 attendees, will be a whirlwind of sessions, keynotes, fireside chats, posters, and exhibitors.
Before the conference, GEN spoke with David Barrett, JD, the CEO of ASGCT, and Paula Cannon, PhD, current ASGCT president and a Distinguished Professor of Microbiology and Immunology at the Keck School of Medicine of USC, to ask their perspectives on the event, what attendees should be looking out for, and what they, personally, are most looking forward to.
An attorney by training, Barrett has been the CEO of ASGCT since 2016. He told GEN that he has had the “fortunate and unique position to see gene therapy go from pre-approval status to in the clinic and watch this tremendous growth within the field, and within the broader industry.”
Cannon has been a long-time leader in the field of gene therapy, working in gene therapy, hematopoietic stem cells, and human immunodeficiency virus (HIV). Her research group has developed genome editing techniques for human hematopoietic stem cells and is currently developing anti-HIV gene therapy technologies that target the CCR5 gene.
This interview has been edited for length and clarity.
LeMieux: David, what do you enjoy about your role as CEO of ASGCT?
David Barrett, JD
CEO, ASGCT
Barrett: What I love about the field, and what I love about the people who are involved in the field, is that it takes much more than a group of scientists and a group of clinicians to move this forward. The people working in gene therapy are not looking at expanding the current paradigm of tried-and-true science and medicine like small molecules or surgical approaches. Rather, they are developing something that is entirely new that takes a cooperative effort from scientists and physicians, hospital administrators, nurses, lawyers and lawmakers, policy makers, and patients and patient advocates to move this thing forward. That truly multidisciplinary approach to advancing an entirely new way of practicing medicine is an extraordinary gift to be a part of.
LeMieux: Meetings are always important to the field that is hosting them. But what is the importance of this particular meeting to this field?
Barrett: This is the one opportunity where those diverse audiences come together and, in one fell swoop over the course of one week, share what has happened over the course of the past year and converge on what is about to happen over the course of the next.
And with ASGCT, it is an egalitarian meeting; what I mean by that is every session is open to every attendee (for the most part). That approach really opens the science and the policy aspects to everybody in the building and to the people who are there virtually. It is such an exciting activity when you get all of these people together to share their knowledge.
On the scientific side, the common denominator of the traditional multidisciplinary approach—where you’ve got hematologists, oncologists, neurologists, immunologists, and every other “ologist” under the sun—is that there are commonalities and learnings that can be appreciated by all. They might not have any other point where they are converging on what is going on in gene therapy more broadly. Sharing that information, I think, is what has allowed for some of the broad advances in gene therapy that we’ve seen since the late 1990s.
LeMieux: What are some of the major themes at the meeting this year?
Paula Cannon, PhD
President, ASGCT
Cannon: We are seeing a shift towards the field embracing new tools: in vivo engineering, nonviral approaches based on LNPs and modified nucleic acids, and manipulation of the epigenome as well as the genome. And of course, we continue to see exciting updates from the clinical trials that have been reporting in the last few months.
LeMieux: What will you be most looking forward to (professionally and personally)?
Cannon: I’m excited by the exponential expansion in our collective imagination for what cell and gene therapies can do. It’s clear that we are moving beyond the classic one-gene disorders to considering more complex disorders, we are harnessing immunotherapies for diseases beyond cancer, and we are making inroads into treating neurological conditions —even prions!
Our keynote speakers are especially exciting. We cover the continuing expansion of the cell and gene therapy toolbox with Nobel laureate Drew Weissman giving an update on RNA technologies, Kiran Musunuru discussing rapid development of highly personalized therapies, and an update on in utero technologies from Tippi MacKenzie. Our final speaker, Mike McCune from the Gates Foundation, will discuss global considerations about how to deliver cell and gene therapies.
Barrett: I really look forward to the keynote lectures in the general session. I have worked for several years with each president, and the keynote lectures that they invite often represent the things that they are most interested in. So, I’ve had the opportunity to talk about these topics with our president over the course of several years. I love to be able to hear the lectures that they’ve wanted to play a role in inviting, and to hear these keynote speakers talk about their work so passionately to such a broad audience. Selfishly, because I’m not a scientist, I can understand a lot more of what they discuss because it’s more broadly applicable. So, I’m looking forward to that. The other thing that I’m looking forward to is walking out of the first general session and being led into the exhibit hall for the very first time: as that rope drops, and the curtain pulls back, and we can see what an extraordinary display of the industry there is on that trade show floor. It is always very exciting.
LeMieux: Is there anything new this year at the meeting?
Barrett: We received more than 2,100 abstracts this year, which represents the largest response to our call for abstracts. We have more fireside chats, more sponsored symposia, and different committee proposals than we’ve had before. But really the backbone of the scientific conference is the scientific exchange. So, having more than 2,100 new pieces of research submitted to our meeting and presented—either through oral presentation or poster presentation—is exciting. And you just can’t get that anywhere else.
LeMieux: Gene therapy is challenging, in part, because of the heartbreaking stories of adverse events including a recent death from Sarepta Therapeutics’ gene therapy for DMD, Elevidys. How do you keep this in mind when you’re working on putting the meeting together?
Barrett: We do a couple of things to make sure that we keep that in mind. What I mean by that is considering the willingness of patients and their families to engage in gene therapy, either clinical trials or approved drugs, because these are for serious, life-threatening, and in many cases, life-ending conditions that don’t have good options or any options. And so, although I guess we can’t really understand, we appreciate the hardship and the sacrifice that patients and their families go through when they decide to pursue a gene therapy or not.
And in a lot of instances, these kids are really sick. And we acknowledge that for some, this is lifesaving, and for many, they won’t be able to access these for a variety of reasons. And for some, it won’t provide a lifesaving cure. It is necessary for us to understand why we’re doing this and the importance of the work that our scientists and physicians are performing. And every gene therapy, every trial, every patient counts. And we cannot ever lose sight of that.
LeMieux: How do you think the challenges that the scientific community is facing right now, specifically with the news coming out of the government and lack of funding, will play out at the meeting? Do you have anything specific planned at the meeting to touch on these concerns?
Barrett: We have a number of fireside chats where we will be working to approach some of those issues. And we’re hopeful that some of our invited speakers will be able to talk about some of the challenges that we’re facing in science and medicine right now. And they’re very real. There’s no doubt about that. We are actively engaging every day with legislators, their staffers, regulators, and other members of the executive administration to represent the interests of our members. And I do think that there will be some content that speaks to that.
Cannon: Of special interest this year will be our fireside chats, which we are hoping to include an interview with NIH Director Jay Bhattacharya. I look forward to hearing his opinions on how NIH can continue to support the basic research that has given rise to so many of the cell and gene therapy innovations we are now seeing come to fruition.
Barrett: Gene therapy is a field that has responded to changes in funding and changes in practices before. It has responded to real tragedies. And it continues to move the science forward with understanding, respect, and appreciation for all that has come before and for those who have sacrificed before. It has found ways to move forward. And I have every confidence that we will continue to find ways to move forward despite what’s happening in the federal funding space and questions about what’s happening coming from the administration.
LeMieux: You’re right: gene and cell therapy is a tenacious field that has kept moving forward despite strong headwinds.
Barrett: It does, it keeps pushing forward. And I will tell you that it is not just the researchers and the physicians. It is the patient community. And while gene therapy is not just for rare conditions, many gene therapies target rare disease. The rare disease community of patients and their advocates is the strongest and most vocal and most capable group of people I have ever encountered. They will literally move mountains.
LeMieux: What do you hope people take away from the meeting?
Barrett: I hope that they take away an appreciation for the growth in the space over the course of the last year and the advancements scientifically and clinically in cell and gene therapy. I hope that they take away the fact that this is a booming industry with extraordinary opportunity, both scientifically and clinically. I hope that they learn something. And I hope that they take something away and implement it into their daily life.
The post Gene Therapy in the Big Easy: Society CEO and President Preview ASGCT 2025 appeared first on GEN - Genetic Engineering and Biotechnology News.
Before the conference, GEN spoke with David Barrett, JD, the CEO of ASGCT, and Paula Cannon, PhD, current ASGCT president and a Distinguished Professor of Microbiology and Immunology at the Keck School of Medicine of USC, to ask their perspectives on the event, what attendees should be looking out for, and what they, personally, are most looking forward to.
An attorney by training, Barrett has been the CEO of ASGCT since 2016. He told GEN that he has had the “fortunate and unique position to see gene therapy go from pre-approval status to in the clinic and watch this tremendous growth within the field, and within the broader industry.”
Cannon has been a long-time leader in the field of gene therapy, working in gene therapy, hematopoietic stem cells, and human immunodeficiency virus (HIV). Her research group has developed genome editing techniques for human hematopoietic stem cells and is currently developing anti-HIV gene therapy technologies that target the CCR5 gene.
This interview has been edited for length and clarity.
LeMieux: David, what do you enjoy about your role as CEO of ASGCT?
David Barrett, JD
CEO, ASGCT
Barrett: What I love about the field, and what I love about the people who are involved in the field, is that it takes much more than a group of scientists and a group of clinicians to move this forward. The people working in gene therapy are not looking at expanding the current paradigm of tried-and-true science and medicine like small molecules or surgical approaches. Rather, they are developing something that is entirely new that takes a cooperative effort from scientists and physicians, hospital administrators, nurses, lawyers and lawmakers, policy makers, and patients and patient advocates to move this thing forward. That truly multidisciplinary approach to advancing an entirely new way of practicing medicine is an extraordinary gift to be a part of.
LeMieux: Meetings are always important to the field that is hosting them. But what is the importance of this particular meeting to this field?
Barrett: This is the one opportunity where those diverse audiences come together and, in one fell swoop over the course of one week, share what has happened over the course of the past year and converge on what is about to happen over the course of the next.
And with ASGCT, it is an egalitarian meeting; what I mean by that is every session is open to every attendee (for the most part). That approach really opens the science and the policy aspects to everybody in the building and to the people who are there virtually. It is such an exciting activity when you get all of these people together to share their knowledge.
On the scientific side, the common denominator of the traditional multidisciplinary approach—where you’ve got hematologists, oncologists, neurologists, immunologists, and every other “ologist” under the sun—is that there are commonalities and learnings that can be appreciated by all. They might not have any other point where they are converging on what is going on in gene therapy more broadly. Sharing that information, I think, is what has allowed for some of the broad advances in gene therapy that we’ve seen since the late 1990s.
LeMieux: What are some of the major themes at the meeting this year?
Paula Cannon, PhD
President, ASGCT
Cannon: We are seeing a shift towards the field embracing new tools: in vivo engineering, nonviral approaches based on LNPs and modified nucleic acids, and manipulation of the epigenome as well as the genome. And of course, we continue to see exciting updates from the clinical trials that have been reporting in the last few months.
LeMieux: What will you be most looking forward to (professionally and personally)?
Cannon: I’m excited by the exponential expansion in our collective imagination for what cell and gene therapies can do. It’s clear that we are moving beyond the classic one-gene disorders to considering more complex disorders, we are harnessing immunotherapies for diseases beyond cancer, and we are making inroads into treating neurological conditions —even prions!
Our keynote speakers are especially exciting. We cover the continuing expansion of the cell and gene therapy toolbox with Nobel laureate Drew Weissman giving an update on RNA technologies, Kiran Musunuru discussing rapid development of highly personalized therapies, and an update on in utero technologies from Tippi MacKenzie. Our final speaker, Mike McCune from the Gates Foundation, will discuss global considerations about how to deliver cell and gene therapies.
Barrett: I really look forward to the keynote lectures in the general session. I have worked for several years with each president, and the keynote lectures that they invite often represent the things that they are most interested in. So, I’ve had the opportunity to talk about these topics with our president over the course of several years. I love to be able to hear the lectures that they’ve wanted to play a role in inviting, and to hear these keynote speakers talk about their work so passionately to such a broad audience. Selfishly, because I’m not a scientist, I can understand a lot more of what they discuss because it’s more broadly applicable. So, I’m looking forward to that. The other thing that I’m looking forward to is walking out of the first general session and being led into the exhibit hall for the very first time: as that rope drops, and the curtain pulls back, and we can see what an extraordinary display of the industry there is on that trade show floor. It is always very exciting.
LeMieux: Is there anything new this year at the meeting?
Barrett: We received more than 2,100 abstracts this year, which represents the largest response to our call for abstracts. We have more fireside chats, more sponsored symposia, and different committee proposals than we’ve had before. But really the backbone of the scientific conference is the scientific exchange. So, having more than 2,100 new pieces of research submitted to our meeting and presented—either through oral presentation or poster presentation—is exciting. And you just can’t get that anywhere else.
LeMieux: Gene therapy is challenging, in part, because of the heartbreaking stories of adverse events including a recent death from Sarepta Therapeutics’ gene therapy for DMD, Elevidys. How do you keep this in mind when you’re working on putting the meeting together?
Barrett: We do a couple of things to make sure that we keep that in mind. What I mean by that is considering the willingness of patients and their families to engage in gene therapy, either clinical trials or approved drugs, because these are for serious, life-threatening, and in many cases, life-ending conditions that don’t have good options or any options. And so, although I guess we can’t really understand, we appreciate the hardship and the sacrifice that patients and their families go through when they decide to pursue a gene therapy or not.
And in a lot of instances, these kids are really sick. And we acknowledge that for some, this is lifesaving, and for many, they won’t be able to access these for a variety of reasons. And for some, it won’t provide a lifesaving cure. It is necessary for us to understand why we’re doing this and the importance of the work that our scientists and physicians are performing. And every gene therapy, every trial, every patient counts. And we cannot ever lose sight of that.
LeMieux: How do you think the challenges that the scientific community is facing right now, specifically with the news coming out of the government and lack of funding, will play out at the meeting? Do you have anything specific planned at the meeting to touch on these concerns?
Barrett: We have a number of fireside chats where we will be working to approach some of those issues. And we’re hopeful that some of our invited speakers will be able to talk about some of the challenges that we’re facing in science and medicine right now. And they’re very real. There’s no doubt about that. We are actively engaging every day with legislators, their staffers, regulators, and other members of the executive administration to represent the interests of our members. And I do think that there will be some content that speaks to that.
Cannon: Of special interest this year will be our fireside chats, which we are hoping to include an interview with NIH Director Jay Bhattacharya. I look forward to hearing his opinions on how NIH can continue to support the basic research that has given rise to so many of the cell and gene therapy innovations we are now seeing come to fruition.
Barrett: Gene therapy is a field that has responded to changes in funding and changes in practices before. It has responded to real tragedies. And it continues to move the science forward with understanding, respect, and appreciation for all that has come before and for those who have sacrificed before. It has found ways to move forward. And I have every confidence that we will continue to find ways to move forward despite what’s happening in the federal funding space and questions about what’s happening coming from the administration.
LeMieux: You’re right: gene and cell therapy is a tenacious field that has kept moving forward despite strong headwinds.
Barrett: It does, it keeps pushing forward. And I will tell you that it is not just the researchers and the physicians. It is the patient community. And while gene therapy is not just for rare conditions, many gene therapies target rare disease. The rare disease community of patients and their advocates is the strongest and most vocal and most capable group of people I have ever encountered. They will literally move mountains.
LeMieux: What do you hope people take away from the meeting?
Barrett: I hope that they take away an appreciation for the growth in the space over the course of the last year and the advancements scientifically and clinically in cell and gene therapy. I hope that they take away the fact that this is a booming industry with extraordinary opportunity, both scientifically and clinically. I hope that they learn something. And I hope that they take something away and implement it into their daily life.
The post Gene Therapy in the Big Easy: Society CEO and President Preview ASGCT 2025 appeared first on GEN - Genetic Engineering and Biotechnology News.