Shenzhen Geno-Immune Medical Institute
Gene-modified autologous stem cells(Shenzhen Geno-Immune Medical Institute)은 Beta-Thalassemia 표적 세포·유전자 치료로, Fanconi Anemia 영역에서 개발·상용화 중입니다. 작용기전: targets a pan-leukocyte antigen. 전임상 단계입니다. 대표 임상 효능: ORR 1% (ClinicalTrials.gov NCT01352286). 차별점: unique setting of autologous GT for ADA SCID, and these dosing principles may be applied to future GT trials using low-dose BU.
Beta-Thalassemia
Fanconi Anemia
Active
ADC) conditioning to explore optimal curative treatment of SCID
s
cleavable (SG3249) or non-cleavable (SG3376) linkers
lentivirally transduced HSPCs
humanized NSG mice, the ADCs completely ablated human HSCs without toxicity to non-hematopoietic tissues, ena
unique setting of autologous GT for ADA SCID, and these dosing principles may be applied to future GT trials using low-dose BU
targets a pan-leukocyte antigen
T-cell and NK cell cytotoxicity
DNA damage in either setting following this treatment, suggesting that ACK2 does not induce immediate genotoxicity, providing cruc
downregulation, effectively modulated the MAPK pathway with Fos downregulation in wild-type and FA mice
차트 로드 중…
임상시험이 많습니다. 임상시험 탭에서 Phase/Status 필터·검색을 사용하세요.
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다.
Shenzhen Geno-Immune Medical Institute
Gene-modified autologous stem cells(Shenzhen Geno-Immune Medical Institute)은 Beta-Thalassemia 표적 세포·유전자 치료로, Fanconi Anemia 영역에서 개발·상용화 중입니다. 작용기전: targets a pan-leukocyte antigen. 전임상 단계입니다. 대표 임상 효능: ORR 1% (ClinicalTrials.gov NCT01352286). 차별점: unique setting of autologous GT for ADA SCID, and these dosing principles may be applied to future GT trials using low-dose BU.
Beta-Thalassemia
Fanconi Anemia
Active
ADC) conditioning to explore optimal curative treatment of SCID
s
cleavable (SG3249) or non-cleavable (SG3376) linkers
lentivirally transduced HSPCs
humanized NSG mice, the ADCs completely ablated human HSCs without toxicity to non-hematopoietic tissues, ena
unique setting of autologous GT for ADA SCID, and these dosing principles may be applied to future GT trials using low-dose BU
targets a pan-leukocyte antigen
T-cell and NK cell cytotoxicity
DNA damage in either setting following this treatment, suggesting that ACK2 does not induce immediate genotoxicity, providing cruc
downregulation, effectively modulated the MAPK pathway with Fos downregulation in wild-type and FA mice
차트 로드 중…
임상시험이 많습니다. 임상시험 탭에서 Phase/Status 필터·검색을 사용하세요.
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다.