STOK Profile
Stoke Therapeutics Inc. (STOK) is a biotechnology company headquartered in Bedford, Massachusetts, USA. The company is focused on developing therapies for genetic diseases by modulating RNA expression.
Stoke's lead product candidate is STK-001, an investigational therapy for the treatment of Dravet syndrome, a rare and severe genetic epilepsy. STK-001 is designed to upregulate the expression of a healthy copy of the SCN1A gene, which is mutated in most cases of Dravet syndrome. STK-001 is currently being evaluated in a Phase 1/2a clinical trial, and Stoke is also planning to initiate a Phase 2b clinical trial in the first half of 2022. Stoke is also developing other product candidates for the treatment of various genetic diseases, including STK-002, an investigational therapy for the treatment of genetic diseases caused by haploinsufficiency of a specific gene.
In October 2021, Stoke announced positive interim data from its Phase 1/2a clinical trial of STK-001 in patients with Dravet syndrome. The data showed that STK-001 was generally well-tolerated and demonstrated encouraging signs of clinical activity, including a reduction in seizure frequency and an improvement in seizure severity. The company plans to continue the development of STK-001 and expects to initiate additional clinical trials in the coming months.
As of September 30, 2021, Stoke had cash, cash equivalents, and marketable securities of $329.4 million. The company has incurred significant research and development expenses as it continues to advance its pipeline of product candidates.
Stoke's stock is listed on the NASDAQ stock exchange and has a market capitalization of approximately $1.1 billion as of February 21, 2023.
|
