Rebisufligene etisparvovec ( DrugBank: Rebisufligene etisparvovec )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病2

19. ライソゾーム病


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
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agemin
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PhaseCountries
1EUCTR2019-002979-34-ES
(EUCTR)
18/05/202025/05/2020A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH) MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, with deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatmentcurrently available for the disease.
MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: scAAV9.U1A.hSGSH
Product Code: ABO-102
INN or Proposed INN: Rebisufligene etisparvovec
Abeona Therapeutics IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
34Phase 1;Phase 2Germany;United States;France;Spain;Australia
2EUCTR2015-003904-21-FR
(EUCTR)
23/12/201911/09/2019Gene transfer clinical trial for Mucopolysaccharidosis IIIAPhase I/II gene transfer clinical trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA MPS IIIA is a devastating lysosomal storage disease, caused by a N-sulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, with deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatment currently available for the disease.
MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: scAAV9.U1A.hSGSH
Product Code: ABO-102
INN or Proposed INN: rebisufligene etisparvovec
Other descriptive name: scAAV9.U1A.hSGSH
Abeona Therapeutics IncNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
22 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;France;Brazil;Spain;Australia;Germany;United Kingdom