American Society for Gene & Cell Therapy (ASGCT)

The principal investigator at Seattle Children’s Research Institute discussed the continuing need to share preclinical research at conferences even as more gene therapies enter the clinic.

The chief scientific officer at Candel Therapeutics discussed progress in investigations with CAN-3110 and CAN-2409.

The principal investigator at Seattle Children’s Research Institute discussed the potential of new delivery methods and gene editing to overcome the limitations of AAV vector-based gene therapies.

The senior investigator at the National Cancer Institute Center for Cancer Research discussed unmet needs that remain and further research to be done.

The senior research fellow at Fred Hutch Cancer Center discussed research trends at the 2023 ASGCT meeting.

The professor of medicine at University of California San Diego discussed preclinical data on the LX2020 gene therapy.

The professor at Ohio State University and University of California – San Francisco discussed investigations with different delivery methods of GDNF gene therapy.

Betty Woo, the vice president and general manager of cell, gene, & advanced therapies at Thermo Fisher Scientific, discussed the challenges that exist in gene therapy and cell therapy manufacturing and potential solutions.

The PhD candidate from University of North Carolina – Chapel Hill discussed preclinical research presented at ASGCT 2023.

The scientist at Senti Biosciences discussed developing small molecule-regulated gene circuits.

The chief scientific officer of LocanaBio discussed preclinical data with an AAV-delivered, snRN, exon 51 skipping approach presented at ASGCT 2023.

The assistant professor of microbiology at Penn Medicine discussed research into characterizing type 1 interferon and EGR2 signaling and their effects on CAR T-cell activation.

Florian Eichler, MD, a neurologist at Massachusetts General Hospital, discussed efficacy data from the CANaspire clinical trial that he presented at ASGCT 2023 showing reductions in urine NAA levels.

The senior scientist at Tune Therapeutics discussed preclinical research presented at the ASGCT 2023 meeting.

There were no dose-limiting toxicities, serious adverse events, nor infusion-related reactions among 6 patients treated with ENCell’s investigational EN001 therapy.

The principal investigator at Seattle Children’s Research Institute discussed her lab’s preclinical research on nonviral delivery methods for gene editing tools in the context of treating hemophilia A.

Lena Winstedt, PhD, Hansa Biopharma, discussed imlifidase, which is being investigated in combination with gene therapies from Sarepta and Genethon.

The chief scientific officer at REGENXBIO discussed multiple presentations given by the company at the 2023 ASGCT meeting.

All evaluable patients had improvements in natriuretic peptides, left ventricular volume, and NYHA class.

The principal investigator at Seattle Children’s Research Institute discussed the preclinical research on delivery methods for genomic medicines that her lab presented at ASGCT’s 2023 meeting.

There were no serious adverse events reported and no patients discontinued from the study.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed updated data from the phase 1/2 CAMPSIITE trial presented at ASGCT 2023.

All evaluable patients had significant reductions in daily cornstarch intake at 1 year and at last visit during long-term follow-up.

The CEO, founder, and president of Siren Biotechnology discussed the preclinical data she presented at ASGCT’s 2023 meeting on the use of AAV immuno-gene therapy for high-grade gliomas.

The chief scientific officer at Candel Therapeutics discussed late-breaking data presented at ASGCT 2023.

The trial compared data from external controls including a recent domagrozumab null study and a propensity score weighted comparison.

The senior investigator at the National Cancer Institute Center for Cancer Research discussed updated data from a phase 1/2 trial presented at ASGCT 2023.

The chief scientific officer of Sangamo Therapeutics discussed the company’s AAV capsid evolution research.

The findings, presented at ASGCT’s 2023 conference, included an analysis of biomarker data in relation to disease severity.

The neurologist at Massachusetts General Hospital discussed BBP-812, an investigational AAV vector-based gene therapy being evaluated for Canavan disease in a phase 1/2 trial.