Stoke Therapeutics إيبيت 2024

ماذا تفعل Stoke Therapeutics؟

Stoke Therapeutics Inc is a biopharmaceutical company founded in 2018 by experienced scientists to develop innovative therapeutic approaches for rare genetic diseases. The company's headquarters is located in Bedford, Massachusetts, and since its inception, it has continuously invested to expand its business and its development process. In 2019, the company announced a financing round of $90 million to advance the development of its pipeline and clinical trials. Stoke Therapeutics specializes in the development of therapeutics for the treatment of rare genetic diseases caused by mutations in haploinsufficiency genes. These diseases are caused by mutations in one of the two genes that code for a specific protein. Since a single defective gene is not sufficient to maintain the normal functions of a protein, serious health problems arise. Stoke's therapeutics aim to replace the missing gene product by targeted stimulation of the healthy gene. This is achieved through the development of antisense oligonucleotide-based therapeutics. Antisense oligonucleotides are small DNA-like molecules that are able to address, recognize, and bind to specific genes. By binding to the healthy gene, they can promote its expression and thereby replace the missing protein. The company's pipeline currently includes a range of products at various stages of development, including preclinical and clinical trials. The following products are the most important: STK-001: A product developed for the treatment of severe forms of Dravet syndrome. Dravet syndrome is a rare genetic disease characterized by seizures, developmental delays, and other neurological symptoms. STK-002: A product for the treatment of haploinsufficiency in STING1-associated vasculopathic syndrome (SAVS). SAVS is a rare genetic disease characterized by cardiovascular disorders and increased susceptibility to infections. STK-005: A product for the treatment of haploinsufficiency in adrenoleukodystrophy (ALD). ALD is a rare disease characterized by inflammatory and degenerative changes in the nervous system. Due to its innovative technology and focus on rare genetic diseases, Stoke Therapeutics has attracted the attention of many investors and potential partners. In 2020, the company entered into collaboration agreements with Roche Holdings and Ribometrix Inc to accelerate the development and commercialization of its therapeutics. Overall, Stoke Therapeutics has a promising future as it is able to provide a much-needed treatment option for patients with rare genetic diseases who currently have no effective therapies available. Stoke Therapeutics ist eines der beliebtesten Unternehmen auf Eulerpool.com.