The FDA has approved XellSmart's IND applications for allogeneic iPSC-derived cell therapies targeting Parkinson's disease (XS-411) and ALS (XS228), with both therapies also receiving approval for Phase I trials in China.
uniQure's AMT-162, a gene therapy for SOD1-ALS, has received the green light from the IDMC to proceed with enrollment in the second dose cohort of its Phase I/II EPISOD1 trial.
The FDA granted breakthrough therapy designation to STK-001 for Dravet syndrome, highlighting its potential to improve upon current treatments by restoring NaV1.1 protein levels.
The American Society of Hematology (ASH) meeting showcased significant developments, including AstraZeneca's new data on venetoclax with Calquence for chronic leukemia and BeiGene's focus on Brukinsa for leukemia and lymphoma. Regulatory news featured uniQure's progress on Huntington’s candidate AMT-130, while Q32 Bio and Keros Therapeutics faced setbacks in their clinical trials.
The FDA has agreed that existing Phase I/II data for uniQure's AMT-130, compared to external controls, can support a Biologics License Application for accelerated approval.
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