CGTLive®’s Weekly Rewind – January 10, 2025

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

1. Ultragenyx Puts BLA for MPSIII Gene Therapy UX111 in Front of FDA

Ultragenyx is seeking accelerated approval for the BLA based on the use of CSF heparan sulfate data from the phase 1/2/3 Transpher A clinical trial.

2. Manali Kamdar, MD, on Sequencing of Treatment in Third-Line R/R LBCL

The clinical director of lymphoma services at the University of Colorado discussed the choice between treating patients with liso-cel or bispecific T-cell engagers.

3. Arbor Biotechnologies’ CRISPR-Based Gene Editing Therapy ABO-101 Cleared for US Trial in Primary Hyperoxaluria Type 1

Arbor noted it has designed redePHine, a phase 1/2 clinical trial (NCT identifier pending) that will evaluate ABO-101 in adults and children with PH1.

4. Tenaya’s Hypertrophic Cardiomyopathy Gene Therapy TN-201 Well-Tolerated in Early Clinical Data

The data comes from 3 patients treated at the trial’s low dose.

5. First Patient With Multiple Myeloma Dosed in EsoBiotec's Trial for In Vivo CAR-T ESO-T01

According to EsoBiotec, this is the first time a patient has been treated with an in vivo BCMA-directed CAR-T therapy in a clinical trial.