Take a look at the interviews that stood out as pillars of progress and success from all of CGTLive's coverage of major 2023 medical society meetings and conferences.
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.
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
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
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
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
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
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