Krystal Biotech's investigational topical gene therapy has shown promise in DEB. It is designed to deliver COL7A1 and restore C7 protein in patients with the rare disorder.
Heidi Anne Duerr, MPH
Advertisement
Articles by Heidi Anne Duerr, MPH
Advertisement
Latest Updated Articles
Uptake of Nononcology Gene Therapy Remains Slow in HematologyPublished: August 16th 2024 | Updated: August 19th 2024
Phase 3 Trial Seeks to Continue Supporting Arsa-Cel Gene Therapy for MLDPublished: March 18th 2024 | Updated: June 3rd 2024
Rapcabtagene Autoleucel Shows Promise for DLBCL in Updated Phase 2 DataPublished: December 7th 2024 | Updated:
Aimee C. Talleur, MD, on Predicting Response and Toxicity to Cell TherapyPublished: December 12th 2022 | Updated:
RPE Cell Therapy Shows Improvements in Early GA With AMDPublished: July 14th 2024 | Updated:
Multi-Characteristic Opsin Gene Therapy for Retinitis Pigmentosa Improves Multiple Measures of VisionPublished: May 18th 2023 | Updated:
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
