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Lab study backs the NGN-401 gene therapy
Neurogene makes a gene therapy called NGN-401. A new lab study found it raised the MeCP2 protein within one week, and the level stayed steady for months. MeCP2 is the protein that is missing or not working in Rett. The therapy is now in a late-stage trial called Embolden.
Source: Rett Syndrome News
Longer-term update from the NGN-401 early trial
Neurogene shared data from its early trial of 10 people. The company says every person gained at least one new skill, and no new serious side effects tied to the treatment were reported since October 2025. These are company-reported results and still need to be confirmed.
Source: Neurogene press release
Brain stimulation helped memory in Rett mice
Scientists at Baylor College of Medicine and Texas Children's studied deep brain stimulation in mice with Rett. It helped new brain cells grow and improved learning and memory in the mice. This was in mice, not people.
Source: Baylor College of Medicine
Company reports results for a pill called NA-921
Biomed Industries says its drug NA-921, taken by mouth, did better than placebo in a 12-week study of girls and young women with Rett. The company plans a bigger study next. These are company results shared at a meeting.
Source: Biomed Industries
A different gene found in one girl with Rett
Doctors in Sweden found a change in a gene called GABBR2 in a girl with classic Rett signs whose MECP2 test was normal. They say doctors should think about this gene when MECP2 testing comes back normal.
Source: Rett Syndrome News
TSHA-102 gene therapy finishes dosing in its main trial
Taysha Gene Therapies finished giving its gene therapy, TSHA-102, to 17 girls and young women in its REVEAL trial. The company expects first results and FDA feedback in the first half of 2027.
Source: Taysha Gene Therapies