NEWS

Proud to annouce that functionalgenomics.pl won the 2026 Biomedical Engineering call for joint projects leveraging synthetic biology for biomedicine and tackling the critical barrier of safe and efficient gene therapy delivery into the human brain. Our project unites leading academic and industry partners to develop a next-generation virus-like particles platform for precise gene editing in the brain. The consortium’s ultimate goal is to translate these breakthroughs into clinical application with Weill Cornell Medicine, NYC, driving the path toward first-in-human studies. 

functionalgenomics.pl joined TOUGH GENES foundation for IRF2BPL research as scientific consultant. More information about TOUGH GENES and IRF2BPL involvement you can find here: https://www.toughgenes.org/

We are proud to annouce that functionalgenomics.pl is a partner of the first in the world IRF2BPL gene therapy clinical trial at Weill Cornell Medicine, NYC. After succesfull first dose of AAV9 based gene augumentation this technology will be further developed by https://functionalgenomics.pl  in collaboration with https://alcyonetx.com Lowell, Massachusetts and NextGen Precision Health, University of Missouri.

We are happy to annouce that first in the world in vivo mocel of IRF2BPL (NEDAMSS) was sucessfully generated by functionalgenomics.pl. Such transgenic mouse now cen be used for in vivo phenotypic study, drug testing and gene therapy testing including further development of the next versions of IRF2BPL gene therapy and delivery challange.

Meet the brilliant young researchers who are breaking boundaries as part of Bayer Foundation’s 2025 cohort of scientific fellowship awardees. Their cutting-edge projects in Drug Discovery and Medical Sciences:

Sofia Notopoulou, CRISPaR: Gene editing to target Parkinson’s disease, Centre for Research and Technology Hellas, Greece  to Pawel Lisowski, Berlin Institute for Medical Systems Biology, Germany in the season ov 2025/2026.

 We are happy to annouce that we have launched MDC BOOST engineering project aimed in gene therapy technology developement for diseases with complex gene architecture like Huntington’s disease and IRF2BPL related disease under umbrella of BlueRock THERAPEUTICS LP part of BAYER AG responsible for cell and gene therapies technology development .

In the Nature Communication paper by Lisowski et al. 2024, we have implicated a new gene in the progression of Huntington’s disease in a brain organoid model. The gene may contribute to brain abnormalities much earlier than previously thought. Moreover, our genome editing strategies, in particular the removal of the CAG repeat region in the Huntington gene, showed great promise in reversing some of observed developmental defects. This suggests a potential gene therapy approach.

More information can be found:
https://www.nature.com/articles/s41467-024-51216-w
https://www.sciencedaily.com/releases/2024/08/240822125933.htm
https://e3.eurekalert.org/news-releases/1055398
https://e3.eurekalert.org/multimedia/1038832
https://huntingtonsdiseasenews.com/news/proteins-loss-brain-may-cause-huntingtons-long-before-onset/