Tominersen
Highest phase reached
Phase 3
Drug type
Oligonucleotide
- Listing at source
- Listed
- Highest phase reached
- Phase 3
- Drug type
- Oligonucleotide
- Drug name
- Tominersen
- ionis httrx
- Ionis httrx
- isis 443139
- Isis 443139
- isis 443139 10 mg
- ISIS 443139 10 mg
- ISIS 443139 120 mg
- ISIS 443139 30 mg
- ISIS 443139 60 mg
- ISIS 443139 90 mg
- isis 443139 free acid
- ISIS-443139 FREE ACID
- rg 6042 free acid
- RG-6042 FREE ACID
- rg6042
- Rg6042
- RG6042
- rg6042 free acid
- RG6042 FREE ACID
- ro7234292
- Ro7234292
- RO7234292
- RO7234292 (RG6042)
- tominersen
- Tominersen
And 6 more, listed on the interactive page.
- CAS registry numbers
- 1709886-74-7
- ChEMBL ID
- CHEMBL4297581
- FDA ingredient codes (UNII)
- 7QI41X9QWC
- Patents
- 0
- Designations
- 2
- First in class
- No
- Fast track
- No
- Breakthrough designation
- No
- Orphan designation
- No
- Priority review
- No
- Orphan exclusivity
- No
- Accelerated approval
- No
- Drug name stem
- -rsen
- Mechanism
- Huntingtin mRNA antisense inhibitor
- Action type
- ANTISENSE_INHIBITOR
- Target
- Huntingtin mRNA
- Protein identifier
- ENSG00000197386
- Mechanism
- Huntingtin mRNA antisense inhibitor
- Action type
- Inhibitor
- Target
- Huntingtin mRNA
- Protein identifier
- ENSG00000197386
Open Targets mechanisms
- Mechanism
- Huntingtin mRNA antisense inhibitor
- Action type
- ANTISENSE INHIBITOR
- Target
- Huntingtin mRNA
- Condition
- Pyoderma Gangrenosum
- Highest recorded phase
- 1
- Condition identifier
- EFO:0006835
- Subject identifier
- D017511
- Condition
- Hidradenitis Suppurativa
- Highest recorded phase
- 1
- Condition identifier
- EFO:1000710
- Subject identifier
- D017497
- Condition
- Huntington Disease
- Highest recorded phase
- 3
- Condition identifier
- MONDO:0007739
- Subject identifier
- D006816
- Condition
- Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
- Highest recorded phase
- 1
- Condition identifier
- MONDO:0015492
- Subject identifier
- D056648
- Condition
- Inflammation
- Highest recorded phase
- 1
- Condition identifier
- MP:0001845
- Subject identifier
- D007249
- Condition identifier
- EFO_0006835
- Condition identifier
- EFO_1000710
- Condition identifier
- MONDO_0006559
- Condition identifier
- MONDO_0007739
- Highest recorded phase
- 3
- Condition identifier
- MONDO_0015492
- Condition identifier
- MP_0001845
- NCT02519036
- NCT03342053
- NCT03761849
- NCT03842969
- NCT04000594
- Prodrug
- No
- Chirality
- Single enantiomer
- Inorganic
- No
- Natural product
- No
- Component
- Light chain
- Type
- Protein
- Sequence
- DIVLTQSPASLAVSLGQRATISCKASQSVDYDGDSYMKWYQQKPGQPPKLLIYAASNLQSGIPARFSGSGSGTDFTLNIHPVEEEDAATYYCQQSNEDPYTFGGGTKLEIKRTVAAPSVFIFPPSDEQLKSGTASVVCLLNNFYPREAKVQWKVDNALQSGNSQESVTEQDSKDSTYSLSSTLTLSKADYEKHKVYACEVTHQGLSSPVTKSFNRGEC
- Component
- Heavy chain
- Type
- Protein
- Sequence
- QVQLQQSGPQLVRPGTSVKISCKASGYSFTTFWMDWVKQRPGQGLEWIGRIDPSDSESRLDQRFKDRATLTVDKSSSTVYMQLSSPTSEDSAVYYCARGNDGYYGFAYWGQGTLVTVSSASTKGPSVFPLAPCSRSTSESTAALGCLVKDYFPEPVTVSWNSGALTSGVHTFPAVLQSSGLYSLSSVVTVPSSSLGTKTYTCNVDHKPSNTKVDKRVESKYGPPCPSCPAPEFLGGPSVFLFPPKPKDTLMISRTPEVTCVVVDVSQEDPEVQFNWYVDGVEVHNAKTKPREEQFNSTYRVVSVLTVLHQDWLNGKEYKCKVSNKGLPSSIEKTISKAKGQPREPQVYTLPPSQEEMTKNQVSLTCLVKGFYPSDIAVEWESNGQPENNYKTTPPVLDSDGSFFLYSRLTVDKSRWQEGNVFSCSVMHEALHNHYTQKSLSLSLGK
- Component
- Glycosylation sites (N)
- Sequence
- Asn-296 $ Asn-296''''
- Component
- Sequence
- Type
- Nucleic Acid
- Sequence
- (3'-5') mC=mU-mC-A-G=d(T=A=A=mC=A=T=T=G=A=mC)=A-mC-mC-A=mC
- Component
- Disulfide bridges
- Sequence
- 22-96 $ 22''-96'' $ 23'-92' $ 23'''-92''' $ 133-218' $ 133''-218''' $ 138'-198' $ 138'''-198''' $ 146-202 $ 146''-202'' $ 225-225'' $ 228-228'' $ 260-320 $ 260''-320'' $ 366-424 $ 366''-424''
- Component
- Glycosylation sites (N)
- Sequence
- Asn-296 $ Asn-296''
Related records
Trials (6)
- A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease
- A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)
- A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease
- An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies
- GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease
- Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease
In development for (4)
- Anti-neutrophil cytoplasmic antibody-associated vasculitis Phase 1
- Hidradenitis suppurativa Phase 1
- Huntington disease Phase 3
- Pyoderma gangrenosum Phase 1
Targets (1)
- HTT Huntingtin mRNA antisense inhibitor