For World Hemophilia Day, CGTLive takes a look at how the hemophilia field has adapted to the introduction of gene therapy to the treatment landscape.

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

Aimee C. Talleur, MD, a physician at St. Jude Children’s Research Hospital, discussed obstacles to understanding late effects of CAR-T.

An interim analysis of a phase 1/2 study suggest the REGENXBIO therapy also showed efficacy signals.

The specific autoimmune disease(s) that will be evaluated in the trial is as of yet undetermined, but the company hopes to initiate the trial in Q4 of 2024.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed highlights from the meeting.

In light of the IND clearance, Tr1X anticipates initiating a phase 1 clinical trial within the second quarter of this year.

The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed takeaways from an MDA session on rare diseases.

The more recent data were presented at the 2022 ASGCT Meeting.

Tiffany Chen, PhD, the vice president of discovery at GentiBio, discussed the general and unique challenges of transitioning cell therapies to clinical trials in autoimmune disease.

The director of the Stem Cell Transplant & Cellular Therapy Program at St. Louis Children’s Hospital discussed recently approved and still-upcoming gene therapy options for SCD.

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed the NIH’s genome editing program and more.

Amar Kelkar, MD, a stem cell transplantation physician at the Dana-Farber Cancer Institute, discussed his research on the value of advanced therapeutics.

The company plans to initiate a pivotal trial of 4D-710 in the second half of 2025.

The director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine discussed novel innovations that lie on the horizon.

One participant experienced a possibly related serious AE of pigmentary changes in the macula with severe vision reduction.

Review top news and interview highlights from the week ending April 12, 2024.

Miguel-Angel Perales, MD, shares background on the unique design of a real-world analysis of liso-cel in LBCL in which he and his co-investigators prospectively collected retrospective data.

The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed research his lab is pursuing and its applications.

Shalini Shenoy, MD, MBBS, discussed when the choice should be made to transition from symptom management to curative therapies.

The data comes from 2 phase 1/2a clinical trials and their respective open-label extension studies.

The best response was very good partial response in 3 of 5 participants.

The associate professor in the Translational Science and Therapeutics Division at Fred Hutch Cancer Center discussed her lab’s research on making TCR T-cell therapies more effective.

Paul Harmatz, MD, professor, UCSF, discussed benefits in neurocognitive outcomes and ERT therapy usage after RGX-121 gene therapy for Hunter syndrome.

Adipose-derived MSCs did significantly increase salivary flow rate from baseline in patients with previous head and neck cancer.