CGTLive's 2024 Pillars of Progress: Most-Watched Conference Interviews

Throughout 2024, the CGTLive^® team closely tracked advancements in cell and gene therapy, with a goal of spotlighting the ongoing developments toward treating a wide range of medical conditions. These efforts included holding in-depth conversations with experts in the clinical care of these individuals, as well as in cell and gene therapy development, culminating in our coverage of each step of progress that the most exciting cellular and genetic treatments have made along the pipeline.

From major data publications and presentations to FDA decisions and major medical meetings, the team spent all year bringing the latest information to the website's front page.

One of the key places to find the latest updates on these treatments is during major medical society meetings. In 2024, our team covered updates from meetings of organizations across the breadth of medical specialties in search of the biggest news in cell and gene therapy.

Here, we'll highlight some of the most-watched interviews from CGTLive's conference coverage this year. Click the buttons to view these videos.

Alexandra Collin de l’Hortet, PhD, on Evaluating Epigenetic Gene Therapy for the Treatment of FSHD

“The key takeaway is that we can rescue methylation on that particular locus in the genome and that correlates very well and that translates very well into a suppression of the toxic gene target that is the cause of that disease—a complete reduction of cell death or apoptosis and eventually a rescue of muscle function. Those are the key takeaways from the product that starts with the root cause, the methylation, and all of the positive effect that restoring methylation has on that disease.”
– Alexandra Collin de l’Hortet, PhD

Epic Bio is currently developing EPI-321, an investigational epigenetic gene therapy product intended to treat facioscapulohumeral muscular dystrophy by restoring methylation of the aforementioned DNA region to normal levels. EPI-321 is a CRISPR-based system delivered by an adeno-associated virus vector and functions by adding a methylation mark. The company presented preclinical results obtained in several different models at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD. At the conference, CGTLive spoke with Alexandra Collin de l’Hortet, PhD, the head of therapeutics at Epic Bio, to learn more about EPI-321 and the findings that were presented.

Georg Schett, MD, on Exploring CAR-T for Autoimmune Disease

“The relapse rate is very low, and there have been occasional relapses reported in the field by other studies, but also with low dose CAR T-cell therapy, because that was the starting dose. So, this has to be interpreted in the right way, and I would say that relapses occur, of course, nothing is 100%, but so far, it looks that most of the people enjoy a drug free remission for a long time."
–Georg Schett, MD

Safety and some preliminary efficacy were seen in a pair of clinical studies—the CASTLE basket study and a phase 1 clinical trial for CC-97540 (Bristol Myers Squibb)—that evaluated CD19-directed chimeric antigen receptor T-cell (CAR-T) therapies in patients with a variety of refractory autoimmune and rheumatic diseases. At the American College of Rheumatology (ACR) Convergence 2024 in Washington, DC, Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg, presented data from these 2 studies. At the conference, CGTLive®'s sister site HCPLive® spoke to Schett about the findings seen in both studies and how they add to the nascent and growing literature of CAR-T therapy use in the rheumatological field, namely the diseases included in the studies – systemic lupus erythematosus, systemic sclerosis, and myositis, among others.

Michael Severino on In Vivo Gene Editing With RNA Gene Writers

“Our programs are based on a platform that we call our RNA gene writers. So, our RNA gene writers modify DNA, but they do it using all RNA constructs. So, the protein that does the work is delivered as an mRNA, and the template which contains the instructions is an RNA, and that enables a number of advantages.”
–Michael Severino

Tessera Therapeutics is aiming to develop effective and safe in vivo, nonviral gene and cell therapies, which could eliminate issues inherent with current modes of approved gene therapies such as ex vivo chimeric antigen receptor (CAR) T-cell therapies and adeno-associated virus (AAV) therapies. The company presented on a number of these programs at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland. CGTLive spoke with Michael Severino, chief executive officer, Tessera, while at ASGCT to learn more about the company’s RNA gene writing platform and the promising advantages it might offer for a variety of different fields and indications.

Shankar Ramaswamy, MD, on Bringing Gene Therapy to Common Diseases

“The technology has really matured to a point where we can take that next leap as a field to bring gene therapies into a broader universe of diseases. I think we as a field need to figure out how we can scale the technology, how we can bring down the cost, and how we can improve throughput and quality of the products that we engineer and manufacture. But those are solvable challenges.”
– Shankar Ramaswamy, MD

One company currently seeking to bring AAV vector-based gene therapy to common diseases is Kriya Therapeutics. Kriya gave 6 presentations regarding their work on this endeavor at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD. At the conference, CGTLive sat down with Shankar Ramaswamy, MD, the cofounder, chairman, and CEO of Kriya Therapeutics, to learn more about the company’s efforts. Ramaswamy explained that gene therapy has significant potential to address unmet needs for relatively common diseases like type 1 diabetes and geographic atrophy, but that bringing gene therapy to such indications will require overcoming challenges such as scaling manufacturing, reducing costs, and characterizing products.

John Murphy, PhD, on Using Gene Editing to Tackle Primary Hyperoxaluria Type 1

“What we are able to do—we hope, if this product works as intended—is to provide a once in a lifetime treatment that will really allow patients to have normalized oxalate and avoid all the complications of the disease.”
– John Murphy, PhD

Arbor Biotechnologies is currently developing ABO-101, a lipid nanoparticle (LNP)-delivered Cas12i2-based gene editing approach to treating primary hyperoxaluria type 1. ABO-101 is intended to permanently inactivate the HAO1 gene in the cells of the liver via a one-time treatment. Preclinical data from mouse model and nonhuman primate (NHP) research regarding ABO-101 were presented by the company at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD. In an interview with CGTLive at the conference, John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies, spoke about the background behind ABO-101 and gave an overview of the promising data presented, which indicated potential for the approach to lower levels of oxalate in a safe manner.