Hearing function was found to be normal for 4 of the patients (50%) at their most recent follow-up.
Annual Meeting of the European Society of Gene & Cell Therapy (ESGCT)
Advertisement
The DSMB has cleared Atamyo to go forward with a planned dose of ATA-100 for the second cohort that is 3 times higher than the dose used for the first cohort.
Advertisement
Excision plans to escalate the trial of EBT-101 to the second dose level in the fourth quarter of 2023.
The therapy has been well-tolerated in the first 3 participants which also showed GCase expression in the plasma.
The chief medical officer of Forge Biologics discussed the potential advantages of the investigational combination therapy approach.
The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT.
Advertisement
All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.
Advertisement
Advertisement
Trending on CGTlive®
1
Sanofi’s Neovascular Age-Related Macular Degeneration Gene Therapy SAR402663 Garners FDA Fast Track Designation
2
Around the Helix: Cell and Gene Therapy Company Updates – September 17, 2025
3
Ultragenyx's Glycogen Storage Disease Gene Therapy DTX401 Continues to Enable Decreases in Reductions in Daily Cornstarch Intake at 96 Weeks
4
