The road to treatment 8
At the end of 2021, one of our contacts at Stoke Therapeutics sent us their financial results and company updates. This includes (translated from their page):
The company has nominated STK-002 as a clinical candidate for the treatment of ADOA, a serious and progressive disease of the optic nerve. There are currently no approved treatments for ADOA, which is primarily caused by loss-of-function mutations in the OPA1 gene, resulting in 50% OPA1 protein expression and disease manifestation. STK-002, a proprietary antisense oligonucleotide (ASO), is designed to increase OPA1 protein expression by utilizing the non-mutant (wild-type) copy of the OPA1 gene. Based on preclinical data generated to date, the Company believes that STK-002 has the potential to be a disease modifying therapy for people living with ADOA.
