Tag: Gene Therapy
Lentiviral Gene Therapy Shows Sustained Clinical Efficacy for ADA-SCID
Overall and event-free survival of 100 and 95 percent, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy
Exa-Cel Gene Therapy Improves Quality of Life in Severe Sickle Cell Disease
Exa-cel also shows beneficial effects on health-related quality of life for adults and adolescents with transfusion-dependent β-thalassemia
Revakinagene Taroretcel Reduces Ellipsoid Zone Area in Macular Telangiectasia Type 2
Reduced ellipsoid zone area seen with revakinagene taroretcel compared with sham procedure
Gene Therapy Effective in Patients With Recessive Dystrophic Epidermolysis Bullosa
One-time surgical application of prademagene zamikeracel improves wound healing and pain
FDA Approves Zevaskyn for Rare, Genetic Skin Disorder
Zevaskyn is the first and only autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa
Sarepta’s Gene Therapy Under Scrutiny After Patient Death
FDA Approves Kebilidi for Aromatic L-Amino Acid Decarboxylase Deficiency
Approval represents first gene therapy for treatment of AADC deficiency
Lentiviral Gene Therapy Beneficial for Early Cerebral Adrenoleukodystrophy
No major functional disabilities observed at month 24; 81 percent of patients had no major functional disabilities at median of six years
Hematologic Cancer Develops in Some Patients Receiving Eli-Cel
Hematologic cancer developed in seven of 67 patients with cerebral adrenoleukodystrophy receiving eli-cel gene therapy
Fidanacogene Elaparvovec Superior to Prophylaxis for Hemophilia B
Adeno-associated virus gene-therapy vector for hemophilia B superior for reducing annualized bleeding rate
