INS1202

Insmed is a large New Jersey company whose main products are lung medicines (Arikayce and Brinsupri). Gene therapy is a small, newer part of its pipeline; INS1202 is its second gene therapy to reach people, after one for Duchenne muscular dystrophy.

INS1202 uses AAV9, an emptied-out virus, to carry a short piece of genetic material (a short hairpin RNA) into nerve and support cells. Once there, it keeps destroying the instructions for SOD1, so less of the protein is made, possibly for years after a single dose. Lowering SOD1 is the same idea behind tofersen, which is approved for SOD1 ALS and needs repeated spinal injections. In SOD1 mice INS1202 improved movement and survival, and it protected nerve cells in lab tests with cells from some sporadic ALS patients, but most treatments that work in mice do not work in people.

The FDA cleared the trial in 2025 and the first person was dosed in January 2026. The first three people, all with sporadic ALS, got the lowest dose. By late October 2026 the trial had moved to a middle dose, enrolling people with sporadic and SOD1 ALS, with a higher dose possible later if it looks safe.

No results have been reported. ARMOR's main goal is safety over about one year. It will also look at SOD1 and the nerve-damage marker NfL in spinal fluid, muscle strength and the ALSFRS-R score, but with no placebo group and few people it cannot show whether the therapy slows ALS.

This is the most favourable result reported. See all key studies for the full picture.

The trial takes adults aged 18 to 79 who either carry a disease-causing SOD1 change or have no known ALS gene, have had ALS for no more than 42 months, have an ALSFRS-R score of at least 24 and breathing capacity of at least 50%. Participants take steroid pills around the dose to prevent immune reactions, so they must be able to swallow pills.

On its October 2026 earnings call Insmed said it expects to share initial data from the ALS programme in 2027.

The registry lists ARMOR as recruiting at seven US centres. Everyone in the trial gets the gene therapy; there is no placebo. In late 2026 the middle-dose group was enrolling both people with sporadic ALS and people with SOD1 ALS. We found no expanded access programme.

• ARMOR (Phase 1) (NCT07290062) recruiting · Baltimore MD, Columbia MO, Columbus OH, La Jolla CA, Palo Alto CA, Philadelphia PA, Worcester MA