{"id":"ada-gene-transfer","safety":{"commonSideEffects":[{"rate":null,"effect":"Gene therapy-related adverse events"}]},"_chembl":{"chemblId":"CHEMBL6068567","moleculeType":"Antibody"},"_dailymed":null,"mechanism":{"_ai_source":"groq-llama-8b","explanation":"This approach aims to restore the patient's immune system by providing a functional ADA enzyme, which is essential for the breakdown of toxic substances in the body. The healthy ADA gene is introduced into the patient's hematopoietic stem cells, which then produce healthy immune cells that can function properly. This treatment has shown promise in treating patients with ADA-SCID, a rare and severe immunodeficiency disorder.","oneSentence":"ADA gene transfer involves the introduction of a healthy copy of the ADA gene into a patient's hematopoietic stem cells to treat ADA-SCID.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T03:19:04.920Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency"}]},"trialDetails":[{"nctId":"NCT04049084","phase":"","title":"An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID","status":"ENROLLING_BY_INVITATION","sponsor":"University of California, Los Angeles","startDate":"2019-09-26","conditions":"Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)","enrollment":70},{"nctId":"NCT03645460","phase":"NA","title":"Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)","status":"RECRUITING","sponsor":"Shenzhen Geno-Immune Medical Institute","startDate":"2024-06-30","conditions":"Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)","enrollment":10},{"nctId":"NCT05432310","phase":"PHASE1, PHASE2","title":"Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector","status":"RECRUITING","sponsor":"University of California, Los Angeles","startDate":"2023-01-04","conditions":"Adenosine Deaminase Severe Combined Immune Deficiency","enrollment":20},{"nctId":"NCT03311074","phase":"PHASE4","title":"Retroviral Insertion Site Methodology Study","status":"WITHDRAWN","sponsor":"Fondazione Telethon","startDate":"2020-06-25","conditions":"Immune System Diseases","enrollment":""},{"nctId":"NCT00598481","phase":"PHASE2","title":"ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID","status":"COMPLETED","sponsor":"Fondazione Telethon","startDate":"2002-10-02","conditions":"Immunologic Deficiency Syndromes","enrollment":12},{"nctId":"NCT03478670","phase":"","title":"Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)","status":"ENROLLING_BY_INVITATION","sponsor":"Fondazione Telethon","startDate":"2017-03-28","conditions":"Immunologic Deficiency Syndromes","enrollment":50},{"nctId":"NCT04959890","phase":"","title":"Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy","status":"UNKNOWN","sponsor":"Fondazione Telethon","startDate":"2021-04-23","conditions":"Severe Combined Immunodeficiency Due to ADA Deficiency","enrollment":15},{"nctId":"NCT03765632","phase":"PHASE1, PHASE2","title":"Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID","status":"COMPLETED","sponsor":"Great Ormond Street Hospital for Children NHS Foundation Trust","startDate":"2018-01-03","conditions":"Severe Combined Immunodeficiency Due to ADA Deficiency","enrollment":13},{"nctId":"NCT04140539","phase":"PHASE2, PHASE3","title":"A Clinical Study to Enable Process Validation of Commercial Grade OTL-101","status":"WITHDRAWN","sponsor":"University of California, Los Angeles","startDate":"2019-10-15","conditions":"Severe Combined Immunodeficiency Due to ADA Deficiency","enrollment":""},{"nctId":"NCT01852071","phase":"PHASE1, PHASE2","title":"Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene","status":"COMPLETED","sponsor":"University of California, Los Angeles","startDate":"2013-08-02","conditions":"ADA-SCID","enrollment":46},{"nctId":"NCT02999984","phase":"PHASE1, PHASE2","title":"Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID","status":"COMPLETED","sponsor":"University of California, Los Angeles","startDate":"2016-12-16","conditions":"Severe Combined Immunodeficiency Due to ADA Deficiency","enrollment":10},{"nctId":"NCT01380990","phase":"PHASE1, PHASE2","title":"Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency","status":"COMPLETED","sponsor":"Great Ormond Street Hospital for Children NHS Foundation Trust","startDate":"2012-11-15","conditions":"Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)","enrollment":36},{"nctId":"NCT00794508","phase":"PHASE2","title":"MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID","status":"COMPLETED","sponsor":"Donald B. Kohn, M.D.","startDate":"2008-11","conditions":"Severe Combined Immunodeficiency","enrollment":10},{"nctId":"NCT01346150","phase":"","title":"Patients Treated for SCID (1968-Present)","status":"UNKNOWN","sponsor":"National Institute of Allergy and Infectious Diseases (NIAID)","startDate":"2011-05-15","conditions":"SCID, ADA-SCID, XSCID","enrollment":1007},{"nctId":"NCT01186913","phase":"","title":"Natural History Study of SCID Disorders","status":"ENROLLING_BY_INVITATION","sponsor":"National Institute of Allergy and Infectious Diseases (NIAID)","startDate":"2010-09-02","conditions":"Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome","enrollment":690},{"nctId":"NCT01182857","phase":"","title":"Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents","status":"WITHDRAWN","sponsor":"National Human Genome Research Institute (NHGRI)","startDate":"2010-08-05","conditions":"ADA-SCID","enrollment":""},{"nctId":"NCT00018018","phase":"PHASE1","title":"Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency","status":"COMPLETED","sponsor":"National Human Genome Research Institute (NHGRI)","startDate":"2001-06-20","conditions":"Severe Combined Immunodeficiency Syndrome","enrollment":8},{"nctId":"NCT02022696","phase":"PHASE1","title":"Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector","status":"COMPLETED","sponsor":"National Human Genome Research Institute (NHGRI)","startDate":"2013-12-16","conditions":"Adenosine Deaminase Deficiency, ADA-SCID","enrollment":1},{"nctId":"NCT01279720","phase":"PHASE1, PHASE2","title":"Gene Therapy ADA Deficiency","status":"COMPLETED","sponsor":"Great Ormond Street Hospital for Children NHS Foundation Trust","startDate":"2003-10","conditions":"Adenosine Deaminase Deficiency","enrollment":8},{"nctId":"NCT00001255","phase":"","title":"Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study","status":"COMPLETED","sponsor":"National Human Genome Research Institute (NHGRI)","startDate":"1990-09","conditions":"Severe Combined Immunodeficiency","enrollment":10},{"nctId":"NCT00599781","phase":"PHASE1, PHASE2","title":"Gene Therapy for ADA-SCID","status":"COMPLETED","sponsor":"IRCCS San Raffaele","startDate":"1992-03","conditions":"Severe Combined Immunodeficiency Syndrome","enrollment":8}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":10,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"phase_2","status":"active","brandName":"ADA gene transfer","genericName":"ADA gene transfer","companyName":"Donald B. Kohn, M.D.","companyId":"donald-b-kohn-m-d","modality":"Biologic","firstApprovalDate":"","aiSummary":"ADA gene transfer involves the introduction of a healthy copy of the ADA gene into a patient's hematopoietic stem cells to treat ADA-SCID. Used for Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}