Kiran Musunuru, MD, PhD, and Rebecca Ahrens-Nicklas, MD, PhD, physician-scientists at CHOP, discussed an n-of-1 clinical trial for a CRISPR gene-editing strategy.

The senior research scientist at Seattle Children’s Research Institute discussed preclinical work on using dimerizing agent-regulated immune-receptor complex T-cells to target plasma cells.

Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia discussed a case he presented at the American Society of Gene and Cell Therapy’s 2025 meeting.

The senior principal scientist at Regeneron also discussed the problem of preexisting immunity caused by natural exposure to AAVs.

Nathan Yozwiak, PhD, the head of research at the Gene and Cell Therapy Institute at Mass General Brigham, discussed how innovative research can be held back by financial considerations.

The CEO of Mission Bio discussed the company’s single cell assay platform in the context of the broader developing field cell and gene therapy field.

The physician-scientists at CHOP discussed long-term future expectations for personalized gene editing therapy.

The associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia discussed a case he presented at the American Society of Gene and Cell Therapy’s 2025 meeting.

The data presented came from 3 patients treated in the trial who had a follow-up time of up to 12 months.

Review top news and interview highlights from the week ending May 16, 2025.

The physician-scientists at CHOP discussed what lies on the horizon after KJ’s treatment with a CRISPR-based gene editing therapy for CPS1 deficiency.

The director of the Mass General Brigham Gene and Cell Therapy Institute also discussed the Institute’s presentations at ASGCT’s 2025 conference.

The PDUFA action date for the BLA has been set as November 9, 2025.

The physician-scientists at CHOP discussed an n-of-1 clinical trial for a CRISPR gene-editing strategy.

The senior principal scientist at Regeneron discussed an advanced approach to enabling gene therapy redosing.

The head of research at Mass General Brigham’s Gene and Cell Therapy Institute discussed how innovative research can be held back by financial considerations.

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

The new data were presented in a poster at the American Society of Gene and Cell Therapy’s 2025 meeting.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed treatment considerations for patients already living with SMA.

The president of MAGIC Clinic and CEO of Discovery DNA discussed the implications of gene therapy data presented at the 2025 WORLDSymposium.

James Beck, PhD, chief scientific officer of the Parkinson’s Foundation, discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed treatment decisions in newly diagnosed SMA.

In observance of World Lupus Day, held annually on May 10, we took a look back at recent progress in bringing cell therapy to this autoimmune disease.

Review top news and interview highlights from the week ending May 9, 2025.

In a mid-cycle review meeting, the agency also noted that the review committee found no major deficiencies with the BLA package.