ARID1B-RD Gene Therapy is Advancing to Pre-Clinical Testing

FAR is thrilled to announce that the first-ever ARID1B-RD gene therapy is advancing to pre-clinical testing. This marks a significant milestone in our quest for treatments for ARID1B-RD. The exceptional Lignani Lab from UCL has successfully secured the necessary funding to test the potential genetic treatment they developed on ARID1B-RD mice and brain organoids.

‍

Just over a year ago, after receiving funding from FAR, Prof. Lignani and Dr. Carpenter began developing a gene regulation treatment for ARID1B-RD, leveraging their expertise from developing treatments for other neurological diseases. Throughout the year, the team, particularly Evie Goss-Sampson, has proven the viability of their innovative approach. The results they gathered enabled Prof. Lignani and Dr. Carpenter to obtain additional funding from the generous Great Ormond Street Hospital Children's Charity to continue this pivotal project.

‍

Currently, the team is establishing a colony of transgenic mice that carry a mutation in the ARID1B gene. In a few months, they will administer the treatment to these mice and assess their social and cognitive functions, hoping to demonstrate improvements to match those of their typical peers. Simultaneously, Prof. Lignani and FAR are collaborating with another team to explore the effects of the treatment on the most advanced ARID1B-RD brain organoids developed to date. We will share more information on this soon.

‍

This progress exemplifies FAR’s strategy of supporting early-stage proof-of-concept projects, with the expectation that demonstrating potential will lead to additional funding due to the significant and urgent clinical need.

‍

This is a historic moment for FAR and the ARID1B-RD community, as it is the first time a potential treatment has reached this stage of pre-clinical evaluation. We are eager to see the outcomes and hold profound gratitude and high hopes for Prof. Lignani and his team.

Stay tuned for further updates.

‍

‍