All 12 Rett syndrome patients treated with Taysha's TSHA-102 gene therapy in the REVEAL Phase 1/2 trial achieved at least one developmental milestone within one year, with functional gains continuing up to 30 months. The company has completed dosing in the Phase 3 portion and expects top-line data and FDA feedback in early 2027.
ChemT Biotechnology closed a $4 million seed round led by Wavemaker Ventures to advance its AI-driven biomanufacturing platform. The company's CelMo™ platform demonstrated a 50% increase in antibody output in CHO cells. ChemT plans to expand its technology to stem cells, NK cells, and HEK cells.
The FDA agreed uniQure's existing trial data could support an accelerated approval filing for Huntington's therapy AMT-130, a reversal from earlier positions. Meanwhile, UK-based Harness Therapeutics selected HRN001 as its lead experimental candidate, targeting the FAN1 protein to address somatic expansion of Huntington's-causing genetic repeats.
The FDA has granted accelerated approval to Denali Therapeutics' Avlayah (tividenofusp alfa-eknm), the first therapy targeting neurological symptoms of Hunter syndrome. The approval was based on a surrogate endpoint measuring heparan sulfate reduction in cerebrospinal fluid, with confirmatory study results required for full approval. The global Hunter syndrome treatment market is projected to reach $2.6 billion by 2033.
Biotech stocks Ocugen, Kodiak Sciences, and CytoDyn are experiencing market movements driven by clinical data and regulatory milestones, particularly Biologics License Application (BLA) filings. Ocugen saw a surge in short interest, Kodiak reached a 52-week high on positive trial results, and CytoDyn's stock remains volatile as it advances its monoclonal antibody pipeline.
Sangamo Therapeutics has retained Raymond James to evaluate strategic alternatives to advance its pipeline and maximize stakeholder value. Key assets include the BLA-ready Fabry disease gene therapy ST-920, the STAC-BBB capsid platform generating $88M in fees to date, and multiple neurology programs. No transaction has been agreed and no timetable has been set.
The FDA fast-tracked an inhalable gene therapy for advanced lung cancer after an early 11-patient trial showed tumour shrinkage in three patients and disease stabilization in five. Larger studies involving about 250 patients are underway.
Two Louisiana parents published a children’s book to raise awareness of Infantile Neuroaxonal Dystrophy, a rare disorder affecting two of their daughters. They are also helping raise money for a gene therapy trial.
Sensorion appointed Fred Chereau chief executive effective June 1, 2026, while reporting six-month Audiogene trial data for SENS-501. The company said early hearing improvements in cohort 2 persisted at six months and no serious adverse events were reported.
Benitec Biopharma's gene therapy BB-301 improved swallowing ability in all four OPMD patients followed for at least one year in a Phase Ib/II trial. One patient showed continued improvement at two years. The company plans to meet with the FDA in mid-2026 to discuss a pivotal study.
