Shots:

• The US FDA granted Orphan Drug Designation to Stoke’s STK-001 with its expected onset of P-I/II clinical study and IND submission to the US FDA in H1’19 and H1’20 respectively
• The FDA’s ODD is granted supporting the development of drugs for safe and effective treatment- diagnosis or prevention of rare diseases or disorders that affect fewer than 200-000 people in the US. Additionally- the designation also provides various development incentives- including tax credits for qualified clinical testing- an exemption from FDA application fees- and a 7yrs. period of marketing exclusivity in the US- if approved
• STK-001 is an investigational antisense oligonucleotide candidate- designed to upregulate NaV1.1 protein expression from the non-mutant (wild type) copy of the SCN1A gene restoring physiological NaV1.1 level in patients with Dravet syndrome

Click here to­ read full press release/ article | Ref: Stoke Therapeutics| Image: LinkedIn