Gene Therapy for AADC Deficiency Receives Positive CHMP Opinion

The positive opinion was based on findings from studies conducted in Taiwan.

Ami J. Shah, MD, on Reaching Normalized Hemoglobin With Gene Therapy in PKD

The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.

Gene Therapy for LAD-I Posts Impressive Survival Data

In an interview with CGTLive, study primary investigator Donald Kohn, MD, discussed the safety and efficacy observed in the phase 2 trial.

CGTLive’s Weekly Rewind – May 20, 2022

Review top news and interview highlights from the week ending May 20, 2022.

Florian Eichler, MD, on Promising Efficacy of AXO-AAV-GM2 in GM2 Gangliosidosis

The director of the leukodystrophy service at Mass Gen discussed progress dosing in the phase 1/2 trial.

DMD Gene Therapy Shows Continued Microdystrophin Expression

Motor and pulmonary function have stabilized or improved in participants treated with SGT-001.