Developing a Treatment

We are currently working on these specific pre-clinical development tasks with funds from this GoFundMe/GiveButter Campaigns and HOPE for Harvey Foundation Fundraising Efforts:  

• Induced Pluripotent Stem Cells (iPSC) derived neurons ( 3 lines including Harvey's dominant/negative mutation, 1 haploinsufficient line, and 1 control-$33,750 total)-all lines completed
• Commercial iPSC Line for Quality Control (3 lines at $33,750)-completed
• Neuronal Differentiation ($28,000)
• Assay Development and Mass Spec for Harvey's mutation to examine allele expression differences ($15,000)
• DNA Long Read Sequencing for treatment attack spots/SNPs ($11,500)-completed
• Testing drug library of 7,000 small molecules for improvement in Harvey’s neurons, worm, and protein functioning ($75,000)-working on developing assay
• Small Molecule assay development and basic science research on dominant/negative mutation ($50,000)
• Mouse Models (currently have two grant applications accepted at Jax and UAB respectively for a Harvey dominant/negative mouse and a nonsense, haploinsufficient rat model-both in progress-no cost (grants)
• Worm Model of Harvey's mutation for possible small molecule screens (grant-no cost)
• ASO exploration ($8,000)-completed
• Project Management/Research Consultation on Projects ($100,000)
• AAV development at Hebrew University-HOPE for Harvey's responsibility ($20,000)
• Base editor (design completed by MIT researcher team); Cost of work will be completed at later date
• Dual Modality AAV design (grant program and work to be completed in progress)
• FDA Approval for Clinical Trial (will apply for grant assistance once we have quality pre-clinical data)