Boston-based biotechnology firm developing custom antisense oligonucleotide (ASO) therapies for ultra-rare genetic diseases. Founded by Julia Vitarello, who agreed on January 5th 2026 to become its chief executive.
In January 2026 Britain's Medicines and Healthcare products Regulatory Agency (MHRA) approved a novel clinical trial under a "master protocol" in which EveryONE Medicines will tailor a unique ASO for each of ten children suffering from ultra-rare genetic neurodegenerative diseases. If the trial succeeds, the MHRA will grant a "process approval" for making custom ASOs rather than approving each drug individually—a regulatory first.
The first patient, known as patient A, was treated in early 2026 at Great Ormond Street Hospital (GOSH) in London by paediatrician Paul Gissen. Patient A has Niemann-Pick disease type C (NPC), caused by a mutation in a gene encoding a protein responsible for clearing brain cells of surplus fats. The drug she received is called avasen; it binds to an RNA messenger in a way that masks the genetic error, allowing cells to produce healthy proteins.
The company reckons process approval could cut the cost of developing custom therapies from $2m-3m to below $1m and the time from two to three years to less than nine months. The MHRA's protocol specifies a particular type of ASO—whose length and chemical composition are well understood—as a "platform" molecule needing only tiny changes to customise it to a particular illness.
A professor is one who talks in someone else's sleep.