Deos is advancing a pipeline of preclinical and clinical programs focused exclusively on CNS indications with high unmet medical need.
Program Code Indication Modality Delivery Stage
DEOS-101 LBSL, AAV Gene Replacement Intrathecal IND-Enabling
DEOS-204 LBSL, ASO Intrathecal IND-Enabling
| Program Code | Indication | Modality | Delivery | Stage |
|---|---|---|---|---|
| DEOS-101 | LBSL | AAV Gene Replacement | Intrathecal | IND-Enabling |
| DEOS-204 | LBSL | ASO | Intrathecal | IND-Enabling |
Gene therapies aim to cure disease by altering the initial gene varients. This is achieved by inserting correct or functional genes and proteins to replace variants.
While some therapies, such as Antisense Oligonucleotides (ASOs) and CRISPR target certain variants, gene therapies generally address all variants in the target gene. gene therapies were first theorized to be a method of disease cure in the 1960s, and the first sucessful use on a patient was in 1990.
While gene therapies have a lot of promise, and have historically been sucessful, they are not always guaranteed to work. There can also be unexpected side effects. There are also undeterminable long term effects (some positive, some negative.)
You can help further research in several ways! If you are a patient with LBSL, you can participate in clinical study. And, everyone is welcome to donate and fund research.