| (19) |  |
| (11) | EP 4 234 550 A3 |
| (12) | EUROPEAN PATENT APPLICATION |
| (88) | Date of publication A3: | | 06.09.2023 Bulletin 2023/36 |
| (43) | Date of publication A2: | | 30.08.2023 Bulletin 2023/35 |
| (22) | Date of filing: 22.05.2019 |
| | (51) | International Patent Classification (IPC): |
|
| (84) | Designated Contracting States: | | AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR |
| (30) | Priority: | 22.05.2018 US 201862674968 P
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| (62) | Application number of the earlier application in accordance with Art. 76 EPC: | | 19730621.0 / 3797106 |
| (71) | Applicant: Design Therapeutics, Inc. | | Solana Beach, California 92075 (US) |
| | (72) | Inventors: | | - ANSARI, Aseem
Solana Beach, California, 92075 (US) - SHAH, Pratik
Solana Beach, California, 92075 (US)
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| (74) | Representative: Carpmaels & Ransford LLP | | One Southampton Row
London WC1B 5HA
London WC1B 5HA (GB) |
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| | |
| (54) | METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE |
(57) The present disclosure relates to compounds and methods which may be useful for modulating the expression of
fxn and treating diseases and conditions in which
fan 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 GAA; 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 GAA; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.