Transthyretin depletion for treatment of hereditary systemic and senile cardiac amyloidosis (360G-Wellcome-082989_Z_07_D)

£831,143

Systemic transthyretin amyloidosis is a fatal late onset disease caused by tissue deposition of amyloid fibrils composed of variant and wild type transthyretin. The objective of the project is to construct compounds to trigger the accelerated clearance of plasma transthyretin molecules by the liver by synthesising palindromic ligand-linker-ligand compounds capable of cross-linking transthyretin molecules or oligosaccharide-ligand conjugates that direct hepatic clearance, which could be used as drugs for treating and preventing acquired and hereditary human systemic transthyretin amyloidosis. The aim is to optimise the design, synthesis, and properties of a transthyretin depleting drug and complete the comprehensive safety and efficacy evaluation required prior to administration of a validated candidate compound in humans.

Where is this data from?

This data was originally published by The Wellcome Trust. If you see something about your organisation or the funding it has received on this page that doesn't look right you can submit a grantee amendment request. You can hover over codes from standard codelists to see the user-friendly name provided by 360Giving.

Grant Details

Amount Awarded 831143
Applicant Surname Pepys
Approval Committee Seeding Drug Discovery Committee
Award Date 2010-02-15T00:00:00+00:00
Financial Year 2009/10
Grant Programme: Title Seeding Drug Discovery Award
Internal ID 082989/Z/07/D
Lead Applicant Prof Sir Mark Pepys
Partnership Value 831143
Planned Dates: End Date 2010-07-30T00:00:00+00:00
Planned Dates: Start Date 2009-07-31T00:00:00+00:00
Recipient Org: Country United Kingdom
Region Greater London