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15 studies found for:    "gene therapy" OR "gene transfer" OR "virus delivery" | Hemophilia
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Rank Status Study
1 Active, not recruiting Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
Condition: Hemophilia B
Intervention: Biological: AskBio009
2 Recruiting Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
Condition: Hemophilia B
Interventions: Genetic: Gene Transfer;   Drug: scAAV2/8-LP1-hFIXco
3 Not yet recruiting Gene Therapy for Haemophilia A.
Condition: Hemophilia A
Intervention: Biological: AAV2/8-HLP-FVIII-V3
4 Recruiting Gene Therapy Study in Severe Haemophilia A Patients
Condition: Severe Haemophilia A
Intervention: Genetic: BMN 270
5 Recruiting A Gene Therapy Study for Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: SPK-9001
6 Recruiting A Gene Transfer Study for Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SPK-8011
7 Active, not recruiting LTFU for Gene Transfer Subjects With Hemophilia B
Condition: Hemophilia B
Intervention:
8 Terminated Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: Adeno-Associated Viral with Human Factor IX
9 Active, not recruiting Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: AAV5-hFIX
10 Not yet recruiting Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SB-525
11 Enrolling by invitation Non-Interventional Study to Capture the Patient Experience on Gene Therapy for Hemophilia Within the AskBio009-101 Study Through Patient and Investigator Interviews
Condition: Hemophilia B
Interventions: Other: Qualitative Interview - Participants/ Patients;   Other: Qualitative Interview - Investigators and Study Coordinators
12 Recruiting Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: AAVrh10FIX
13 Recruiting Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Condition: Hemophilia B
Intervention:
14 Terminated Hemophilia B Gene Therapy - Spark
Condition: Hemophilia B
Intervention: Biological: AAV8-hFIX19
15 Recruiting Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
Condition: Hemophilia B
Intervention: Biological: SB-FIX

Study has passed its completion date and status has not been verified in more than two years.