Business:
Antisense Oligonucleotide Medicines
Drug notes:
STK-002 Clin1 autosomal dominant optic atrophy; undisclosed RD SYNGAP1 syndrome; undisclosed RD Rett syndrome; undisclosed RD CNS condition
About:
Stoke Therapeutics is using new insights in RNA biology to address the underlying cause of severe diseases. Many diseases arise when one copy of a gene functions normally and the other is mutated, called haploinsufficiency. Using their Targeted Augmentation of Nuclear Gene Output (TANGO) platform, Stoke is identifying ways to restore missing proteins by increasing protein output from healthy genes, thereby compensating for the non-functioning copy of the gene. The platform involves the design of antisense oligonucleotides (ASOs) that bind to pre-mRNA and help the target genes produce more protein. Stoke is currently applying their technology to patients with Dravet syndrome, a severe and progressive genetic epilepsy.
Scientist I, Discovery Biology Bedford, MA|5 days ago
Scientist I, Formulation Bedford, MA|5 days ago
Associate Director/Director, Clinical Science- Dat... Bedford, MA|5 days ago
Director, Statistical Programmer Bedford, MA|5 days ago
Principal Scientist, Oligonucleotide Chemistry Bedford, MA|5 days ago
Senior Manager, GCLP Compliance Bedford, MA|15 days ago
Senior Scientist, Bioinformatics Bedford, MA|17 days ago
Director, Regulatory Affairs, Global Regulatory St... Bedford, MA|23 days ago
Senior Director, Regulatory Labeling Development a... Bedford, MA|23 days ago
Executive Director, Field Medical Affairs Cambridge, MA|45 days ago
Director/Senior Director, Medical Affairs - Midwes... Home, KS|52 days ago
Executive Director/Vice President, Commercial Oper... Bedford, MA|55 days ago
Senior Director, Global Medical Information Bedford, MA|58 days ago
Director, Engineering & Facilities Bedford, MA|62 days ago
Research Associate, Biology Bedford, MA|Posting date unknown
Senior Analytical Scientist Bedford, MA|Posting date unknown