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- A Science study found that the three-dimensional organization of the genome differs in brain cells of people with Alzheimer's disease versus unaffected individuals. - Researchers combined single-cell GAGE-seq, spatial transcriptomics, and a new deep learning model called Hicformer to link genome folding with gene activity. - Alzheimer's cells showed 'increased compartment mingling,' more long-range and fewer short-range DNA contacts, and reduced overall gene activity. - The findings position higher-order chromatin alterations alongside amyloid-beta plaques and tau tangles as part of Alzheimer's molecular pathology.
- A phase Ib clinical trial demonstrated that adding investigational antibody ianalumab to ibrutinib therapy allowed 17 out of 39 chronic lymphocytic leukemia patients to discontinue daily treatment for 12-24 months. - The combination achieved a 60% overall response rate and 43.6% of patients reached undetectable measurable residual disease, indicating deep remissions that could enable treatment-free intervals. - The therapy showed a favorable safety profile with no dose-limiting toxicities and lower infection rates compared to single-agent ibrutinib, potentially transforming CLL from lifelong to fixed-duration treatment.
- GenSight Biologics received FDA authorization and IRB approval for expanded access treatment of one patient with its gene therapy GS010/LUMEVOQ® at the University of Pittsburgh School of Medicine in November 2025. - The authorization represents the first regulatory approval for the candidate since the company withdrew its European marketing authorization application in 2023. - GS010/LUMEVOQ® targets Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial genetic disease that causes irreversible vision loss and legal blindness in teens and young adults. - The company is preparing for a pivotal Phase III study RECOVER in H2 2026 while completing technology transfer to manufacturing partner Catalent to produce new batches for clinical and early access needs.
- OCU410 gene therapy demonstrated a 21.4% reduction in geographic atrophy lesion growth in low-dose patients after 6 months compared to untreated fellow eyes. - The phase 1/2 trial showed no serious adverse events during 12-month follow-up, with patients experiencing more than 40% decrease in lesion size and four-line improvement in low luminance visual acuity. - Ocugen's subretinal gene therapy requires only a single surgical intervention, offering a potential "one-and-done" alternative to repeated monthly injections. - Phase 2 enrollment has been completed and phase 3 trials are planned to begin in the first quarter of 2026.
