What other genetic therapies for ALS are in development?

According to Tofersen, other genetic therapies targeting other known genetic causes of ALS are currently in development. The C9orf72 mutation is the most common genetic cause of ALS and frontotemporal dementia (FTD).

Researchers are developing antisense oligonucleotides (ASO) and other molecular therapies aimed at reducing toxic RNA repeat segments or influencing protein formation. In patients with a mutation in the FUS gene, specific genetic therapies are being developed to correct the defective function of the FUS protein or to prevent the formation of harmful protein accumulations. In addition to ASO technologies, other RNA-based approaches, such as RNA interference (RNAi), are also being developed to suppress the expression of harmful genes.

Another research approach is to activate or introduce protective genes or proteins into the cells in order to halt neuronal degeneration. These genetic therapies are in clinical trials or in even earlier stages of development. In the medium term, with a perspective of 3-5 years, medical and scientific progress can be expected, which will lead to the availability of further genetic drugs.