Gene Therapy May Eliminate Need for ERT in Late-Onset Pompe Disease

All study participants met criteria to remain off enzyme replacement therapy through 104 weeks after treatment.

Gene Therapy for Knee Cartilage Lesions Receives RMAT Designation

NeoCart was originally developed by the company Histogenics.

Andras Heczey, MD, on Antitumor Activity of CAR NKTs in Solid Tumors

The director of the Liver Tumor Program at Texas Children’s Hospital discussed a phase 1 study of CAR NK T-cells in neuroblastoma.

EtranaDez Accepted for Priority Review in Hemophilia B

A final analysis of the pivotal HOPE-B study was presented at ASGCT 2022.

Cell Therapy Modulates Tumor Microenvironment in Glioblastoma

The cell therapy was tolerated although there was a SUSAR of GGT elevations.

Carsten Bönnemann, MD, on Identifying At-Risk Genotypes for DMD Gene Therapy

The senior investigator from NINDS discussed findings investigating serious AEs across different trials.