CGTLive's 2023 Pillars of Progress: Most-Watched Conference Interviews

For all of 2023, the CGTLive^™ team was following along the clinical development of targeted and novel engineered approaches to the treatment of patients with various medical disorders. 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 2023, our team covered updates from more than 30 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.

ASGCT — Jennifer Kown, PhD, on Reducing LDL Cholesterol in NHPs With Epigenome Editing

• Epigenome editing, explored by Tune Therapeutics, shows promise in durable gene silencing and therapeutic impact.
• Tune Therapeutics' research on non-human primates reveals stable PCSK9 repression, reducing LDL cholesterol by 50%.
• Jennifer Kwon, PhD, highlights the potential of epigenome editing for targeted gene modulation in therapeutic applications.

Preclinical data on reducing low-density lipoprotein cholesterol with epigenome editing in nonhuman primates were presented in a late-breaking session at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California, by Jennifer Kwon, PhD, senior scientist, Tune Therapeutics.

"As we learn more about sequencing technologies, we reveal what the side effects are from actual genome editing. And so, epigenome editing does offer an alternative, where, instead of cutting, we can actually just tune the DNA expression.”
— Jennifer Kwon, PhD, of Tune Therapeutics

MDA — Stacy Lindborg, PhD, on the Upcoming FDA Advisory Committee Meeting for NurOwn in ALS

• BrainStorm Cell Therapeutics prepared for an FDA Advisory Committee Meeting on NurOwn's ALS therapy.
• The meeting addressed challenges in measuring disease progression, particularly the floor effect in advanced ALS cases.
• Stacy Lindborg, PhD, co-CEO, discusses trial data, positive biomarkers, and the need for regulatory flexibility.

Data showing a treatment effect favoring NurOwn over the placebo in a prespecified group of patients who had less-advanced ALS at baseline were presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held in Dallas, Texas, March 19-22, 2023.

"We are encouraged by the regulatory flexibility the FDA is showing in ALS... We are confident in the effectiveness of NurOwn and we're grateful to the FDA for the opportunity to review the full body of clinical evidence.”

— Stacy Lindborg, PhD, of BrainStorm Cell Therapeutics

ASCO — Maria Pia Morelli, MD, PhD, on Promising Efficacy of Sleeping Beauty TCR-T Therapy in Solid Tumors

• Alaunos Therapeutics' Sleeping Beauty TCR-T therapy shows efficacy and safety in mutant KRAS, EGFR, TP53-expressing solid tumors.
• Phase 1/2 trial data presented by Maria Pia Morelli, MD, PhD, at ASCO 2023 indicate promise.
• Morelli discusses potential advantages and positive outcomes in diverse solid tumor indications.

Data on sleeping beauty T-cell receptor T-cell therapy in patients with solid tumors solid tumors were presented by investigator Maria Pia Morelli, MD, PhD, assistant professor, department of gastrointestinal (GI) medical oncology, division of cancer medicine, The University of Texas MD Anderson Cancer Center, Houston, TX, at the 2023 American Society of Clinical Oncology (ASCO) meeting, held June 2-6, 2023, held both virtually and in Chicago, Illinois.

"It's definitely relevant, it’s definitely specific, because it's something that is expressed on the cancer cell only on that patient so it's patient specific and very exciting in that sense. And we hope that we can bring immunotherapy also for cold diseases like colon cancer where immunotherapy isn’t currently used.
— Maria Pia Morelli, MD, PhD, of MD Anderson

AHA — Roger Hajjar, MD, on Bringing Gene Therapy to Cardiology

• Roger Hajjar, MD, discusses the importance of gene therapy in cardiology and its evolving history.
• He addresses the challenges, historical context, and future directions in cardiac gene therapy.
• Hajjar emphasizes the need for innovative vector types and delivery methods to enhance safety and specificity.

Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, gave a talk on the importance of gene therapy in cardiology at the American Heart Association’s (AHA) Scientific Sessions 2023, held November 10-13 in Philadelphia, Pennsylvania. The talk, entitled “The Quest to Overcome the Challenges in Cardiac Gene Therapy".

"With the emergence of novel, more cardiotropic vectors and minimally-invasive techniques to deliver locally, we are now able to deliver much lower amounts of vector—100 times less. That will basically allow us to have a safer vector when we deliver these next generation capsids.”

– Roger Hajjar, MD, of Mass General Brigham

ASTCT/CIBMTR — Matthew Frank, MD, PhD, on Investigating CD22 CAR for LBCL After CD19 Relapse

• Investigational CD22 CAR-T therapy shows promise in large B-cell lymphoma patients who relapsed after CD19-directed CAR-T.
• Dr. Matthew J. Frank discusses the therapy's efficacy, safety profile, and the unmet needs it addresses.
• CD22 CAR offers hope for patients with CD19-negative disease or those relapsed after CD19 CAR-T.

Data on CD22 CAR, from a phase 1 clinical trial (NCT04088890), were presented at the 2023 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in Orlando, Florida, February 15-19, 2023, by Matthew J. Frank, MD, PhD, assistant professor of medicine, division of BMT & Cellular Therapy, Division of Stanford Cancer Institute.

"This appears to be a very promising emerging option for folks. We're excited about the high response rates, the durable response rates, and the relatively lower frequency of grade 3 events. So overall, we're very excited about this CD22 CAR therapy.”

– Matthew J. Frank, MD, PhD. Stanford Cancer Institute

WORLDSymposium — Samiah Al-Zaidy, MD, on Gene Therapy for Infantile GM1 Gangliosidosis

• Investigational gene therapy PBGM01 shows promise in addressing GM1 gangliosidosis's unmet need.
• Dr. Samiah Al-Zaidy discusses promising interim results, including positive efficacy signs and encouraging safety profiles.
• PBGM01 aims to offer a potential treatment for patients with infantile GM1 gangliosidosis, currently lacking viable therapeutic options.

Passage Bio presented updated interim safety and efficacy data from patients with infantile GM1 gangliosidosis in the phase 1/2 Imagine-01 clinical trial (NCT04713475) treated with PBGM01 gene therapy at the WORLDSymposium 2023, held February 22-26, in Orlando, Florida.

“Two years after dosing, high dose fordadistrogene movaparvovec in this phase 1 study supports a clinically meaningful difference in motor function in ambulatory participants with DMD versus external controls with similar pretreatment characteristics. The change 2 years after dosing varied by age and was largest in those aged 6-7 years.”

– Samiah Al-Zaidy, MD, of Passage Bio