구조모아 (StructureMoa)항암 chemical structure spider web
방문타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-6 · 마지막 9월 2일
현재 선택: 1개 · 임상 갱신 필요 1개
프로그램 상세에서 관심 등록 후 2개 이상 모으면 여기서 한 번에 비교할 수 있습니다.
표가 넓으면 좌우로 스크롤하세요. 핵심 비교 모드에서는 중요 항목만 표시됩니다.
| 항목 | |
|---|---|
Overview Program | 4D-710 |
Overview Company | 4D Molecular Therapeutics |
Overview Modality | CGT |
Overview Target | Cystic Fibrosis Lung |
Overview Indication | Cystic Fibrosis Lung |
Overview Phase | PHASE_2 |
Overview Status | RECRUITING |
Overview Content status | Standard Database |
Overview Data Confidence | Data Confidence · Medium |
Overview Development Signal | Development Signal · Emerging |
Overview Approval status | Investigational |
PK/PD Species | NHP |
PK/PD Animal (cat.) | Human, NHP |
PK/PD Experiment | Adeno-associated virus (AAV) vectors are the leading platform for the delivery of in vivo gene therapy; however, wild-type AAV vectors exhibit a limited capacity to transduce airway cells and evade pre-existing human neutralizing antibodies (NAb). |
Toxicology Species | NHP |
Toxicology Animal (cat.) | Human, NHP |
Toxicology Major finding | Design and Characterization of 4D-710, an Aerosolized Gene Therapy for Cystic Fibrosis Lung Disease.. Cystic fibrosis (CF) is an autosomal recessive disease caused by variants in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. Delivery of a functional CFTR transgene to airway epithelial cells (AEC) offers the potential to provide durable restoration of normal CFTR func… |
Clinical Safety signal | Design and Characterization of 4D-710, an Aerosolized Gene Therapy for Cystic Fibrosis Lung Disease.. Cystic fibrosis (CF) is an autosomal recessive disease caused by variants in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. Delivery of a functional CFTR transgene to airway epithelial cells (AEC) offers the potential to provide durable restoration of normal CFTR func… |
Clinical Program phase | PHASE_2 |
Clinical Trial activity | 1 recruiting |
타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-6 · 마지막 9월 2일
현재 선택: 1개 · 임상 갱신 필요 1개
프로그램 상세에서 관심 등록 후 2개 이상 모으면 여기서 한 번에 비교할 수 있습니다.
표가 넓으면 좌우로 스크롤하세요. 핵심 비교 모드에서는 중요 항목만 표시됩니다.
| 항목 | |
|---|---|
Overview Program | 4D-710 |
Overview Company | 4D Molecular Therapeutics |
Overview Modality | CGT |
Overview Target | Cystic Fibrosis Lung |
Overview Indication | Cystic Fibrosis Lung |
Overview Phase | PHASE_2 |
Overview Status | RECRUITING |
Overview Content status | Standard Database |
Overview Data Confidence | Data Confidence · Medium |
Overview Development Signal | Development Signal · Emerging |
Overview Approval status | Investigational |
PK/PD Species | NHP |
PK/PD Animal (cat.) | Human, NHP |
PK/PD Experiment | Adeno-associated virus (AAV) vectors are the leading platform for the delivery of in vivo gene therapy; however, wild-type AAV vectors exhibit a limited capacity to transduce airway cells and evade pre-existing human neutralizing antibodies (NAb). |
Toxicology Species | NHP |
Toxicology Animal (cat.) | Human, NHP |
Toxicology Major finding | Design and Characterization of 4D-710, an Aerosolized Gene Therapy for Cystic Fibrosis Lung Disease.. Cystic fibrosis (CF) is an autosomal recessive disease caused by variants in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. Delivery of a functional CFTR transgene to airway epithelial cells (AEC) offers the potential to provide durable restoration of normal CFTR func… |
Clinical Safety signal | Design and Characterization of 4D-710, an Aerosolized Gene Therapy for Cystic Fibrosis Lung Disease.. Cystic fibrosis (CF) is an autosomal recessive disease caused by variants in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. Delivery of a functional CFTR transgene to airway epithelial cells (AEC) offers the potential to provide durable restoration of normal CFTR func… |
Clinical Program phase | PHASE_2 |
Clinical Trial activity | 1 recruiting |
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