[EN] METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE<br/>[FR] PROCÉDÉS ET COMPOSÉS POUR LE TRAITEMENT D'UNE MALADIE GÉNÉTIQUE
申请人:DESIGN THERAPEUTICS INC
公开号:WO2021158707A1
公开(公告)日:2021-08-12
The present disclosure relates to compounds and methods for modulating the expression of dmpk, and treating diseases and conditions in which dmpk plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence CAG or CTG; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence CAG or CTG; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.
Tubulysin analogs of the formula (I)
where R
1
, R
2
R
3
, R
4
, R
5
, R
6
, R
7
, and Y are as defined herein, are anti-mitotic agents that can be used in the treatment of cancer, especially when conjugated to a targeting moiety.
[EN] 1H-PYRAZOLO[4,3-d]PYRIMIDINE COMPOUNDS AS TOLL-LIKE RECEPTOR 7 (TLR7) AGONISTS<br/>[FR] COMPOSÉS DE 1H-PYRAZOLO[4,3-D]PYRIMIDINE EN TANT QU'AGONISTES DU RÉCEPTEUR 7 DE TYPE TOLL (TLR7)
申请人:BRISTOL MYERS SQUIBB CO
公开号:WO2021154669A1
公开(公告)日:2021-08-05
Compounds according to formula I or II are useful as agonists of Toll-like receptor 7 (TLR7). Such compounds can be used in cancer treatment, especially in combination with an anti-cancer immunotherapy agent, or as a vaccine adjuvant.
[EN] LINKED DIBENZIMIDAZOLE DERIVATIVES<br/>[FR] DÉRIVÉS DU DIBENZIMIDAZOLE LIÉS
申请人:ENANTA PHARM INC
公开号:WO2010091413A1
公开(公告)日:2010-08-12
The present invention discloses linked dibenzimidazole derivatives, or pharmaceutically acceptable salts, esters, or prodrugs thereof, which inhibit RNA-containing virus, particularly the hepatitis C virus (HCV). Consequently, the compounds of the present invention interfere with the life cycle of the hepatitis C virus and are also useful as antiviral agents. The present invention further relates to pharmaceutical compositions comprising the aforementioned compounds for administration to a subject suffering from HCV infection. The invention also relates to methods of treating an HCV infection in a subject by administering a pharmaceutical composition comprising the compounds of the present invention.
[EN] HEPATITIS C VIRUS INHIBITORS<br/>[FR] INHIBITEURS DU VIRUS DE L'HÉPATITE C
申请人:ENANTA PHARM INC
公开号:WO2010099527A1
公开(公告)日:2010-09-02
The present invention discloses compounds or pharmaceutically acceptable salts, esters, or prodrugs thereof, which inhibit RNA-containing virus, particularly the hepatitis C virus (HCV). Consequently, the compounds of the present invention interfere with the life cycle of the hepatitis C virus and are also useful as antiviral agents. The present invention further relates to pharmaceutical compositions comprising the aforementioned compounds for administration to a subject suffering from HCV infection. The invention also relates to methods of treating an HCV infection in a subject by administering a pharmaceutical composition comprising the compounds of the present invention.