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

Review top news and interview highlights from the week ending May 26, 2023.

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.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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 investigational gene therapy, also known as SRP-9001, was recently backed by the agency’s advisory committee in a tight decision. Its new deadline for review is June 22, 2023.

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.

Alliance for Cancer Gene Therapy (ACGT) recently hosted their 2023 Summit, a scientific gathering of cancer experts to discuss challenges and opportunities with cell and gene therapy.

The approval was based on data from the phase 1/2 EPCORE NHL-1 clinical trial.

The professor of internal medicine at UT Southwestern Medical Center shared his perspective on the current landscape of CAR-T cell therapy in the care of patients with myeloma.

M. Peter Marinkovich, MD, and Alfonso L. Sabater, MD, PhD, offered their respective perspectives on the recent FDA approval of the topical and redosable gene therapy for DEB.

Diane M. Simeone, MD, discussed the phase 1/2 EVEREST-1 trial, which seeks to assess the novel CAR T-cell therapy A2B530 in patients with solid tumors—hopefully progressing one-and-done engineered therapies in this population.

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

May is Brain Cancer Awareness Month, and the ACGT is raising awareness of the potential of the human immune system when combined with cell and gene therapy to defeat brain tumors.

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.

The senior research fellow at Fred Hutch Cancer Center discussed findings from the PLAT-08 trial presented at ASGCT 2023.

All 8 patients with light chain amyloidosis who received HBI0101 responded.

The most robust transduction in nonhuman primates was seen with ICV delivery of pooled PM4.AAV.mRFP and AAV2.mNG gene therapy,