This drug entry is a stub and has not been fully annotated. It is scheduled to be annotated soon.
Identification
- Generic Name
- Tefidsogene civaparvovec
- DrugBank Accession Number
- DB16789
- Background
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 that inserts a correct copy of the Iduronate 2-Sulfatase (IDS) gene into the albumin locus in hepatocytes. Developed by Sangamo Therapeutics, tefidsogene civaparvovec is being investigated in mucopolysaccharidosis type II.1,2
- Type
- Biotech
- Groups
- Experimental
- Biologic Classification
- Gene Therapies
Other gene therapies - Synonyms
- 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)
- Tefidsogene civaparvovec
- External IDs
- SB 913
- SB-913
- SB913
Pharmacology
- Indication
Not Available
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Prevent Adverse Drug Events TodayTap into our Clinical API for life-saving information on contraindications & blackbox warnings, population restrictions, harmful risks, & more.Avoid life-threatening adverse drug events with our Clinical API- Pharmacodynamics
Not Available
- Mechanism of action
- Not Available
- Absorption
Not Available
- Volume of distribution
Not Available
- Protein binding
Not Available
- Metabolism
- Not Available
- Route of elimination
Not Available
- Half-life
Not Available
- Clearance
Not Available
- Adverse Effects
Improve decision support & research outcomesWith structured adverse effects data, including: blackbox warnings, adverse reactions, warning & precautions, & incidence rates. View sample adverse effects data in our new Data Library!Improve decision support & research outcomes with our structured adverse effects data.- Toxicity
Not Available
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.Not Available
- Food Interactions
- Not Available
Categories
- Drug Categories
- Classification
- Not classified
- Affected organisms
- Not Available
Chemical Identifiers
- UNII
- 2V7PRS3ISP
- CAS number
- 2143580-10-1
References
- General References
- Zapolnik P, Pyrkosz A: Gene Therapy for Mucopolysaccharidosis Type II-A Review of the Current Possibilities. Int J Mol Sci. 2021 May 23;22(11):5490. doi: 10.3390/ijms22115490. [Article]
- PR Newswire: Sangamo Announces Interim Results Of Phase 1/2 CHAMPIONS Study Showing Preliminary Evidence Of In Vivo Genome Editing In Patients With MPS II Treated With SB-913 [Link]
- External Links
- Not Available
Clinical Trials
- Clinical Trials
Phase Status Purpose Conditions Count 1, 2 Terminated Treatment Mucopolysaccharidosis Type II (MPS II) 1 Not Available Active Not Recruiting Not Available Hemophilia B / Mucopolysaccharidosis Type I (MPS I) / Mucopolysaccharidosis Type II (MPS II) 1
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
- Not Available
- Prices
- Not Available
- Patents
- Not Available
Properties
- State
- Not Available
- Experimental Properties
- Not Available
Drug created at May 10, 2022 20:05 / Updated at September 24, 2023 02:55