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Noah Stansfield
Articles
Fabry Disease Gene Therapy Clinical Trial Moves Onto Dosing Second Cohort
October 07, 2022
The FDA recently cleared Freeline Therapeutics to begin treating patients in the United States.
Gene Therapy Shows Promise in Leber Congenital Amaurosis
October 06, 2022
Significant improvements were reported in full-field stimulus testing.
Around the Helix: Cell and Gene Therapy Company Updates – October 5, 2022
October 05, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Gene Therapy Trial Makes Headway in Canavan Disease
October 04, 2022
Data from the first 3 of the 8 patients dosed indicates that the patients improved on the GMFM and the MSEL.
Allogeneic Anti-CD7 CAR-T Demonstrates Promising Results in Hematological Malignancies
October 02, 2022
Among the 11 patients evaluated for efficacy, 9 patients (81.8%) had objective responses at 28 days.
BioMarin Resubmits BLA for Hemophilia A Gene Therapy Val-Rox
September 30, 2022
A prior resubmission in June 2022 was delayed with requests for additional durability and safety data.
GPRC5D-Targeted CAR-T Shows Promise in Heavily Pre-treated Multiple Myeloma
September 30, 2022
Of the 17 patients treated, 12 patients achieved a response, including 6 who achieved a complete response.
Around the Helix: Cell and Gene Therapy Company Updates – September 28, 2022
September 28, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
New Leber Hereditary Optic Neuropathy Gene Therapy Trial Doses First Patient
September 28, 2022
NR082 has been evaluated in 3 previous investigator-initiated trials involving 186 patients.
DMD Cell Therapy Shows Promise in Early Clinical Trial Data
September 27, 2022
The 3 patients demonstrated a mean significant improvement of up to 200% of baseline in motor unit potential.