CGTLive's 2024 Pillars of Progress: Most-Watched Expert 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. Our coverage included exclusive insights from leading experts in clinical care and therapy development, offering a comprehensive view of the rapidly evolving landscape.

From pivotal FDA approvals to major conference highlights and innovative research publications, we delivered updates on the work that's shaping the future of cellular and genetic treatments. Beyond our coverage of medical meetings, we also engaged with experts year-round to explore company announcements, mark disease awareness milestones, and delve into the progress of emerging therapies, ensuring you, our readers, stayed informed every step of the way.

Here, we'll highlight some of the most-watched interviews from CGTLive's Videos page this year that were not directly related to ongoing medical conferences. Click the buttons to watch.

Paul Y. Song, MD, on Taking a Holistic Approach to Treating Parkinson Disease

“We believe it's a much more holistic approach—that you're not only removing the proteins, but you're also removing the neuroinflammation. We believe this will lead to a much more potentially comprehensive improvement in patients’ symptoms and condition versus just targeting the proteins or [another] symptom." – Paul Y. Song, MD

NKGen Biotech is currently developing SNK01, an autologous natural killer (NK) cell therapy, with the intention of addressing both the accumulation of α-synuclein and the neuroinflammation associated with PD. This year, the company received clearance from the FDA for a phase 1/2a clinical trial for SNK01 in PD. Shortly after, we spoke with Paul Y. Song, MD, the chairman and chief executive officer of NKGen, about the aspects of PD pathophysiology that have come to light in recent years, such as the impact of T-cells on neuroinflammation in an autoimmune disease-like fashion. He also spoke about how SNK01, which consists of patient’s own NK cells that have been expanded and enhanced outside the body before readministration, might function in treating PD. Song also noted that SNK01, which was originally developed for oncology indications, has also shown potential in the treatment of Alzheimer disease.

Shankar Musunuri, PhD, on Continuing Research With Gene Therapy in Inherited Retinal Diseases

“We have data to suggest [OCU410] can actually go after all those different pathways or causes of the disease, and could be a potential, one-time, curative therapy. Current therapies you have to continuously take, every month or so, intravitreal injections, and the degeneration is continuing. So, if we can show stabilization or improvement over those products, that's gold. So, we believe this gene therapy has that potential.”
–Shankar Musunuri, PhD

This year, CGTLive also spoke with Shankar Musunuri, PhD, chairman, chief executive officer, and cofounder, Ocugen, to learn more about its investigational agent OCU400 as well as other research the company is pursuing for inherited retinal diseases. He discussed the OCU410 program, which is treating patients with dry age-related macular degeneration (AMD) in a phase 1/2 trial (NCT06018558), and the unmet needs that remain in the population with dry AMD that gene therapy may address. He also shared plans to evaluate OCU410 in patients with Stargardt disease.

Bruce Cree, MD, PhD, MAS, on Selecting the Right Neurologic Autoimmune Disease Patient Populations for CAR T

"Don't target the patients that are doing well on [an approved] therapy or haven't been treated yet with the best possible [approved] therapy. The group to focus on—and this is just my opinion, of course—is the patients who have refractory disease for whom there really are no other therapeutic options. That's the unmet need. That's what you have to go after."
–Bruce Cree, MD, PhD, MAS

In early 2024, CGTLive^® sat down with Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center, to ask about advice for colleagues considering CAR-T clinical trials as a potential option for their patients. Cree gave examples of various situations where CAR-T may be a good or not-so-good option for patients with specific neurologic autoimmune diseases, emphasizing that it could be a great choice in situations where patients are refractory to standard of care therapies for their disease and have no FDA-approved treatment options remaining. He also called attention to the importance of carrying out randomized controlled clinical trials for CAR-T therapies in autoimmune diseases to determine whether these therapies truly provide benefit in these indications.

Arun Upadhyay, PhD, on the Potential of Gene Therapy in Inherited Retinal Disease

“Our approach is more gene-agnostic in nature, which not only enhances the function of the photoreceptor, but also helps with preserving the surviving retinal cells through cellular and molecular homeostasis.”
– Arun Upadhyay, PhD

In June, one of the Ocugen's gene therapy products based on this platform, OCU400, entered into its phase 3 clinical trial (liMeliGhT; NCT06388200), with the first patient dosed that month. Furthermore, findings from a phase 1/2 clinical trial (NCT05203939) in retinitis pigmentosa and Leber congenital amaurosis were recently presented at the 24th EURETINA Congress held September 19 to 22, 2024, in Barcelona, Spain.

In light of this milestone, CGTLive® reached out to Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, to learn more about the company’s approach. Upadhyay described the current landscape of care for RP and the unmet needs that remain. He then spoke about how OCU400 works and the design of the new phase 3 trial.

Brett Kopelan, MA, on the Future Treatment of Epidermolysis Bullosa With Advanced Therapeutics

“This is just amazing and we are new to the immunotherapy era where more CAR Ts are being tested in trials, and also the bispecific antibodies and T cell engagers are also being tested. It's an exciting time for our patients with mantle cell lymphoma.”
– Michael Wang, MD

On May 30, 2024, the FDA approved Bristol Myers Squibb’s lisocabtagene maraleucel (liso-cel, marketed as Breyanzi), another autologous CD19-directed CAR-T therapy, for the treatment of adults with r/r MCL who have been previously treated with at least 2 lines of systemic therapy, including a Bruton tyrosine kinase inhibitor. Following the FDA’s decision, CGTLive® spoke with Michael Wang, MD, a professor in the Department of Lymphoma/Myeloma at MD Anderson Cancer Center, who served as lead investigator on the phase 1 TRANSCEND NHL 001 clinical trial (NCT02631044) that led to liso-cel's approval in r/r MCL. In the interview, Wang expressed enthusiasm regarding the approval and emphasized that liso-cel boasts lower rates of high grade CRS and neurotoxicity compared to brexu-cel, but retains a comparable CR rate. As such, he highlighted that it should provide an important new option for older and frailer patients with r/r MCL. He also spoke about areas of interest for further research regarding liso-cel in MCL.