Active Gene Programs
NOTE: The active gene programs list is updated quarterly and may not accurately represent recently initiated gene programs
| Gene Name | ASO Strategy | Status |
|---|---|---|
| ADCY5 | Allele-selective | Active ASO Program |
| ANXA11 | Allele-selective | Active ASO Program |
| ASXL3 | Non-allele-selective | Therapy available for a nano-rare patient with a mutation in the ASXL3 gene |
| ATL1 | Allele-selective | Active ASO Program |
| ATM | Splicing modulation | Active ASO Program |
| ATN1 | Non-allele-selective | Therapy available for a nano-rare patient with a mutation in the ATN1 gene |
| CACNA1A | Allele-selective | Active ASO Program |
| CACNA1E | Allele-selective | Active ASO Program |
| CAMK4 | Allele-selective | Active ASO Program |
| CHCHD10 | Non-allele-selective | Therapy available for a nano-rare patient with a mutation in the CHCHD10 gene |
| CLCN7 | Non-allele-selective | Active ASO Program |
| DHDDS | Allele-selective | Active ASO Program |
| DNAJC5 | Allele-selective | Active ASO Program |
| DNM1 | Allele-selective | Active ASO Program |
| DYRK1A | Upregulation | Active ASO Program |
| EIF2AK2 | Allele-selective | Active ASO Program |
| ELP1 | Splicing modulation | Therapy available for a nano-rare patient with a mutation in the ELP1 gene |
| FBXO31 | Allele-selective | Active ASO Program |
| FLVCR1 | Splicing modulation | Therapy available for a nano-rare patient with a mutation in the FLVCR1 gene |
| GARS1 | Allele-selective | Therapy available for a nano-rare patient with a mutation in the GARS1 gene |
| GBE1 | Splicing modulation | Active ASO Program |
| GNAO1 | Allele-selective | Active ASO Program |
| GRIA1 | Allele-selective | Active ASO Program |
| GUCA1A | Allele-selective | Active ASO Program |
| H3F3A | Non-allele-selective | Therapy available for a nano-rare patient with a mutation in the H3F3A gene |
| HNRNPH2 | Non-allele-selective | Therapy available for a nano-rare patient with a mutation in the HNRNPH2 gene |
| KCNB1 | Allele-selective | Active ASO Program |
| KCNH1 | Allele-selective | Active ASO Program |
| KCNQ2 | Allele-selective | Active ASO Program |
| KCNT2 | Non-allele-selective | Active ASO Program |
| KIF1A | Allele-selective | Therapy available for a nano-rare patient with a mutation in the KIF1A gene |
| LMNB1 | Non-allele-selective | Therapy available for a nano-rare patient with a duplication in the LMNB1 gene |
| MAPK8IP3 | Allele-selective | Therapy available for a nano-rare patient with a mutation in the MAPK8IP3 gene |
| NALCN | Allele-selective | Active ASO Program |
| NARS1 | Allele-selective | Active ASO Program |
| NEFH | Allele-selective | Active ASO Program |
| PACS1 | Non-allele-selective | Therapy available for a nano-rare patient with a mutation in the PACS1 gene |
| PACS2 | Allele-selective | Active ASO Program |
| PFN1 | Allele-selective | Active ASO Program |
| PLA2G6 | Splicing modulation | Active ASO Program |
| PLP1 | Splicing modulation | Active ASO Program |
| PRPH2 | Allele-selective | Therapy available for a nano-rare patient with a mutation in the PRPH2 gene |
| RHOBTB2 | Allele-selective | Active ASO Program |
| SAA1 | Non-allele-selective | Therapy available for a nano-rare patient with a mutation in the SAA1 gene for treatment in the liver |
| SAMD9L | Allele-selective | Active ASO Program |
| SCN2A | Allele-selective | Therapy available for a nano-rare patient with a mutation in the SCN2A gene |
| SCN8A | Splicing modulation | Active ASO Program |
| SERPINI1 | Non-allele-selective | Therapy available for a nano-rare patient with a mutation in the SERPINI1 gene |
| SETX | Allele-selective | Active ASO Program |
| SLC12A6 | Allele-selective | Active ASO Program |
| SLC37A4 | Allele-selective | Active ASO Program |
| SPTAN1 | Allele-selective | Active ASO Program |
| SPTLC1 | Allele-selective | Active ASO Program |
| TARDBP (TDP43) | Allele-selective | Therapy available for a nano-rare patient with a mutation in the TARDBP (TDP43) gene |
| TUBB4A | Non-allele-selective | Therapy available for a nano-rare patient with a mutation in the TUBB4A gene |
| UBTF | Allele-selective | Therapy available for a nano-rare patient with a mutation in the UBTF gene |
What do I do if my gene isn’t listed?
The list above reflects active gene programs or available ASO therapies. To learn more, please visit one of the following links:
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