The Rett Syndrome Research Trust (RSRT) is a strong US-based patient organisation supporting global research to develop a cure for Rett Syndrome and related MECP2 disorders. Through our relationship with RSRT VICO has gained access to Rett patient cell cultures and associated animal models. This is essential for the discovery and development of antisense oligonucleotides that are based on our RNA-editing technology platform and correct mutations causing this serious lifelong neurological disorder.
UCL is one of the world’s leading multi-disciplinary universities, and the number one London university for Research Strength, recognised for its academic excellence and global impact.
VICO has a research collaboration with the lab of Prof. Paola Giunti at UCL, on the application of antisense oligonucleotides in diverse spinocerebellar ataxias (SCAs) caused by polyQ expansions. Prof. Giunti is a neurologist and recognised ataxia expert and member of VICO’s scientific advisory board.
VICO participates as an industrial partner in the Netherlands SCA1 consortium, which is supported by a grant from The Netherlands Organisation for Health Research and Development (ZonMW).
Aim of the consortium is the development and clinical evaluation of an RNA-targeting therapy for Spinocerebellar Ataxia type 1, based on VICO’s antisense oligonucleotide for polyQ disorders. The consortium encompasses a SCA1 natural history study, biomarker discovery and design and execution of a phase 1/2a clinical trial.
We are pleased to announce that the SCA1 website aimed at providing information to SCA1 patients and families is now live see link below.
NAF Drug Development Collaborative
The Collaborative provides a centralized source for access to resources needed to support research and development of Ataxia therapies. Members will benefit from the integration of the patient experience with sound Ataxia scientific and clinical expertise.
VICO is one of the early members of the Collaborative
CRITICAL PATH TO ATAXIA THERAPEUTICS CONSORTIUM MEMBERSHIP
The CPAT mission is to establish a broad-based consortium which will advance ataxia disease specific data standards and drug development tools, as well as define regulatory pathways to speed the approval of ataxia therapeutics.
VICO is one of the early members of C-Path for Ataxia.