Orchard Therapeutics’ Gene Therapy OTL-203 Stabilizes or Improves Sensory Symptoms in Patients With MPS-IH
Hearing function was found to be normal for 4 of the patients (50%) at their most recent follow-up.
Limb-Girdle Muscular Dystrophy Gene Therapy Trial Set to Enroll Second Cohort Based on Initial Positive Results
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.
CRISPR Therapy Well Tolerated, Detectable in First 3 Participants With HIV
Excision plans to escalate the trial of EBT-101 to the second dose level in the fourth quarter of 2023.
Gaucher Disease Type 1 Gene Therapy Shows Promising Initial Data
The therapy has been well-tolerated in the first 3 participants which also showed GCase expression in the plasma.
Maria Escolar, MD, on Combining HSCT With Gene Therapy in Krabbe Disease
The chief medical officer of Forge Biologics discussed the potential advantages of the investigational combination therapy approach.
Maria Escolar, MD, on Addressing Unmet Needs in Krabbe Disease With Gene Therapy
The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT.
Patients With Fabry Disease Withdrawn From ERT After Isaralgagene Civaparvovec Treatment
All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.
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