Foundation for ARID1B Research (FAR)

Fantastic news! We’re delighted to announce that Dr Omer Revah from the Hebrew University of Jerusalem has secured funding to develop a cutting-edge ARID1B-RD brain organoid model over the next two years. Find out more... arid1b.org/article/develo… #ARID1B #Raredisease

This project significantly enriches FAR's evolving portfolio of ARID1B-RD treatment methodologies currently under research. We are deeply thankful for @UpliftingAth and @julianhalmai and just can’t wait to start this exciting work!

We are excited to announce our collaboration with @UpliftingAth to award @julianhalmai from @ucdavisstemcell a $20k grant in support of their research on dual vector approach for treating ARID1B-RD!

FAR launches an exciting ARID1B-RD drug repurposing project with @aexbrown from @MRC_LMS. Utilizing @aexbrown’s novel high-throughput C. elegans system, we aim to identify promising candidates for treatments. Anticipating results by March. Stay tuned for updates!

Next up: testing on neurons & haploinsufficient models. Stay tuned for more updates! #ARID1B #ARID1B_RD

Exciting ARID1B-RD breakthrough at @ucl! As part of a @FAR_ARID1B-funded project, @LignaniLab_UCL & @jennacolette93 successfully upregulated the ARID1B gene in mouse & human cells. A pivotal step for potential ARID1B-RD treatments.

Big thank you to @ChrisDiasTweets from our scientific advisory board, who joined us and offered invaluable guidance. With several potential collaborations on the horizon, we're excited about what comes next.

Was a pleasure to meet and learn from our rare disease community friends @KabukiUK @PittHopkinsUK @NorrieDiseaseUK @4Lilyfoundation @MyMitoMission @TimothySyndrome @mndassoc @CureGrin @YBRP4hnrnph2 @CureDhdds @ReverseRett.

Recently, we attended the #AspireBiosciences Rare CNS partnering event where we, along with other patient advocacy organizations, met many life sciences companies to explore potential directions for collaboration.

We’re delighted to welcome @CKadoch from @DanaFarber and @harvardmed to our Scientific Advisory Board! Dr. Kadoch’s groundbreaking studies on chromatin & gene regulation show the way to therapeutics development for many diseases inc. NDDs. Looking forward to working together!

@FAR_ARID1B is thrilled to announce today a collaboration with @LignaniLab_UCL for the development and initial evaluation of ARID1B-RD gene therapy tools. There is no one better than @gabrilignani and his fantastic team to lead this effort. Stay tuned for updates!

Fascinating findings in @LabWester's study (funded by @SFARIorg ): ARID1B loss interferes mostly with inhibitory neurons, marking them as potential targets for therapeutics. Identifying damaged circuity caused by ARID1B mutations is key for successful intervention.

@EichlerLab @SPARKforAutism @LabNowakowski More about it on our LinkedIn>> linkedin.com/posts/far-foun… More about the findings>> pnas.org/doi/abs/10.107…

In the largest cohort and examined to date for de novo pathogenic variants, ARID1B was the most common cause of NDDs (@EichlerLab @SPARKforAutism @LabNowakowski )ARID1B variants were less prevalent in the autism sub-group. FAR is working to address this huge unmet medical need.

Nadav is in UCSF (and not UCSD), of course.

We’re thrilled to welcome Prof. @NadavAhituv from UCSD to our Scientific Advisory Board! Prof. Ahituv has been doing groundbreaking work on gene regulation therapy and will be guiding our cis-regulation strategy. Looking forward to working together!

Positive interim results in @StokeTx Dravet trial! An ASO for Dravet Syndrome, a genetically comparable condition to ARID1B-RD, showed very promising interim results in a stage 1-2 clinical trial. Delighted for the @curedravet community Read more>> bit.ly/3Up2gjD

FAR has been quiet lately, but we have been very busy behind the scenes. From now on, we’ll bring you a whole new regular set of updates about our progress in developing treatments for our ARID1B-RD family.

Huge news for the neuro #raredisease community. First gene therapy approved for andrenoleukodystrophy🧠🧬 Second FDA approved #GeneTherapy product from ⁦@bluebirdbio⁩ in a month. Have the flood gates opened? reuters.com/business/healt…

We are thrilled to launch the Foundation for ARID1B Research (FAR), an international nonprofit with the urgent mission to accelerate the development of disease-modifying treatments for ARID1B-RD. Learn more and get involved at arid1b.org