Alternative splicing modulating antisense oligonucleotides (ASOs) targeting novel identified alternative splicing changes associated with RBM10 mutations in bladder cancer.
We are developing lead ASOs to treat bladder, and eventually other, cancers.
Technology:
Bladder cancer is the 9th most common malignant disease worldwide and 13 most frequent cause of cancer death. 80 % of those cancers are non-muscle invasive bladder cancers (NMIBC) while 20 % are muscle invasive (MIBC). While the survival rate after diagnosis in the earlier NMIBC stages is good, it is low for MIBC cancers. Even for NMIBC, patient lives are severely affected because cancer relapses in 60–70 % of the cases and regular check-ups are necessary. These check-ups are not straightforward and have not only a high impact on the patients personal and sex life but are also very costly. Bladder cancer treatment has the highest cost per patient from diagnosis to death.
The alternative splicing factor RBM10 has been found to be frequently mutated in both, NMIBC and MIBC. Through an extensive sequencing and genetic analysis, the CRG team explored, identified and validated the consequences of RBM10 mutations on specific alternative splicing events. The team developed and validated ASOs that correct those RBM10-induced changes of those specific alternative splicing events that can be used to treat bladder, and eventually other, cancers.
Available for licensing / partnering / co-development, etc.