Take a look at the stories 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-read content from CGTLive's conference coverage this year. Click the buttons to read further into these stories.
Data from a phase 1/2 clinical trial (NCT04179643) of AB-1002 (AskBio; NAN-101) gene therapy were presented at the American Heart Association’s (AHA) Scientific Sessions 2023. Investigators found that 3 of 6 patients in cohort 1, those that completed 12-month follow-up, showed clinically meaningful improvements in left ventricular ejection fraction, NYHA Functional Class, Minnesota Living with Heart Failure Questionnaire, cardiopulmonary exercise test, and 6-minute walk test compared to baseline. The remaining 3 patients were still within 3 months of treatment and were not yet evaluable.
"We believe these encouraging early results in patients with advanced heart failure are important for the congestive heart failure community, as they bring hope to a sub-population where treatment options are needed."
— Litsa G. Kranias, PhD, of University of Cincinnati
PRGN-3005, an investigational MUC16-directed CAR-T therapy intended for the treatment of patients with platinum-resistant ovarian cancer, is currently being assessed in a phase 1/1b clinical trial (NCT03907527). The CAR-T was designed with a feature expected to improve persistence and overcome limitations that have held back the success of trials for other CAR-T therapies directed at ovarian cancer. Interim results from the study evaluating PRGN-3005 were presented at the American Society of Clinical Oncology (ASCO) 2023 Annual Meeting.
"This CAR T-cell was engineered not only to express the antigen, but also to express a cytokine that would keep the T-cells alive for a long period of time. We think this overcomes 1 of the main reasons why CAR T-cells may not work in solid tumors."– Mary 'Nora' Disis, MD, of University of Washington
EBT-101 (Excision Biotherapeutics) was well-tolerated and detectable in all participants with human immunodeficiency virus type 1 (HIV-1) treated so far in a phase 1/2 trial (NCT05144386). Updated data from the first-in-human trial were presented at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), in which the therapy was shown to remove large sections of HIV proviral DNA in preclinical studies and was granted Fast Track designation by the FDA in 2023.
"We believe that sharing this initial safety and biodistribution data is important for the HIV/AIDS community, the larger infectious disease community, and for gene therapies in development for other indications."
— Daniel Dornbusch, of Excision
GCC19CART (Innovative Cellular Therapeutics), an investigational autologous CAR-T therapy being evaluated in an investigator-initiated clinical trial (ChiCTR2000040645) in China for the treatment of relapsed/refractory metastatic colorectal cancer (r/r mCRC), and demonstrated efficacy improvements over standard of care (SOC) third-line therapies, according to data presented at the American Association for Cancer Research (AACR) Annual Meeting 2023.
"The new AE specific to this product is diarrhea because the target is GCC, which plays a role in intestinal homeostasis... So, that's expected, theoretically. Most patients treated with this product experienced diarrhea, but it can be controlled and because of the diarrhea management most of the patients actually recovered very quickly."– Victor Lu, MD, PhD, of Innovative Cellular Therapeutics
The administration of the investigational treatment UX111, an intravenously administered self-complementary AAV9-based gene therapy vector encoding hSGSH in development by Ultragenyx to treat patients with mucopolysaccharidosis IIIA (MPS IIIA)—also known as Sanfilippo syndrome type A—is associated with rapid and sustained reductions of relevant cerebrospinal fluid (CSF) biomarker levels in those treated prior to advanced neurodegeneration. The data were presented at the annual WORLDSymposium 2023.
"[MPS IIIA] is caused by a deficiency in the lysosomal enzyme N-sulfoglucosamine sulfohydrolase, resulting in toxic accumulation of lysosomal HS in the brain and other tissues. Neurodegeneration occurs in children with MPS IIIA, and that preclinical data have thus suggested that administration of UX111 results in expression of SGSH in the brain in MPS IIIA animal models.”– Kevin M. Flanagan, MD, of Nationwide Children's
Fordadistrogene movaparvovec (Pfizer; PF-06939926) was associated with clinically meaningful preservation of motor function in participants with Duchenne muscular dystrophy compared with external controls in a phase 1b clinical trial (NCT03362502). Data from the trial were presented in a late-breaking session at the the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting.
“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.”– Perry B. Sheih, MD, PhD, of UCLA