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Gene Therapy
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Gene Therapy trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Gene Therapy trials may be worth asking aboutNorth America
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This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficienc…
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to…
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD). In Phase I the participant…
The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the puta…
Background: Chemokine receptor 4 (CCR4) is a protein that is found on the surface of certain T-cell lymphoma cells and is common in mature T-cell cancers. Whit…
Subjects exposed to gene therapy products (GTPs) may be at risk for delayed or long-term adverse events. This is a long-term follow-up (LTFU) protocol designed…
This phase II MATCH treatment trial tests how well trastuzumab and pertuzumab work in treating patients with HER2-amplified non-breast, non-gastric/gastroesopha…
This phase III trial uses the Decipher risk score to guide therapy selection. Decipher score is based on the activity of 22 genes in prostate tumor and may pred…