DB16789InvestigationalGene Therapies
Tefidsogene civaparvovec, formerly known as SB-913, is an investigational therapy for genome editing. It consists of an adeno-associated virus (AAV) type 2/6 vector and a zinc finger nuclease (ZFN) system... Developed by Sangamo Therapeutics, tefidsogene civaparvovec is being investigated in mucopolysaccharidosis type II.
- Also known as
- A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/6 (RAAV REP2-CAP6) VECTOR, WHICH CONTAINS A PROMOTER-LESS HUMAN IDURONATE 2-SULFATASE (HIDS) TRANSGENE CASSETTE, ENCODING PARTS OF EXON 1 PLUS EXONS 2-9 FROM HIDS AND A SPLICE ACCEPTOR SITE (SA)
- Code names
- SB-913
Resolves to
Compound
What you can answer from here — as of April 23, 2025
Which companies are running trials of Tefidsogene civaparvovec, in which indications and phases, and which of those programs are still active?
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