IEDAT Final Webinar
The main objective of the IEDAT project is to provide a treatment of the neurological symptoms of patients with Ataxia Telangiectasia (AT), a rare progressively disabling and life-shortening genetic disease for which no therapy is currently available. To achieve this, the IEDAT Project has developped solutions, which will be presented during this Final Event, to conclude the project.
EryDex, an innovative product developed by EryDel, used to administer dexamethasone sodium phosphate by ex-vivo encapsulation in autologous erythrocytes, which are infused into the patient. EryDex provides long-term delivery of low doses of dexamethasone without the typical steroid side effects and has reached a successful Phase II trial conducted in AT patients. The phase III trial has been an international, multi-center, 1 year, randomized, prospective, double-blind, placebo-controlled, designed to assess the effect of 2 dose ranges of EryDex, administered monthly by IV infusion, on neurological symptoms of AT patients. The protocol of the trial and the regulatory path to registration has already been agreed upon with EMA and FDA.
An international patient registry has also been set with the aim of establishing and maintaining a comprehensive clinical database of patients with AT and closely related conditions, enabling the monitoring of AT epidemiology, the development of an evidence-based natural history of the condition, identification of biomarkers as well as development of clinical guidelines.
The AT NEST, the first scale to assess symptoms specific to AT patients, coordinated by the AT centre at the John’s Hopkins University, has been tested in the study and represents the 1st scale assessing chief areas of impairment specific to AT.
In parallel to the clinical trial, investigations into the molecular mechanisms of action of EryDex has been performed with the objective to provide the validation of a new biomarker predictive of treatment efficacy.
The final webinar will take place on August 4th, at 2:30pm.
To register, click here.
Center for Targeted Therapeutics and Translational Nanomedicine (CT³N), a seminar entitled “Synthetic Lipid Nanoparticle for Gene Therapy and Gene Editing” with Qiaobing Xu, PhD.
2020 Meeting of the European Red Cell Society, PAvia online meeting, 11 September 2020.
SIB 2019 60th CONGRESS
SIB 2019 60th CONGRESS - Italian Society of Biochemistry and Molecular Biology Lecce, September 18 – 20, 2019
Interregional mapping of SIB Congress
The interregional mapping of SIB (Italian Society of Biochemistry and Molecular Biology) issues between Tuscany, Umbria and Marche took place at Ancona, Italy. The university of Urbino representatives were present at the congress.
18th Annual International Ataxia-Telangiectasia Workshop
The University of Urbino participated in the International Ataxia-Telangiectasia Workshop, which took place on May 1-4,2019 at the Houston Methodist Research Institute (Texas Medical Center).
Ataxia Telangiectasia Clinical Research Conference
Representatives of the IEDAT project participated in the Ataxia Telangiectasia Clinical Research Conference which took place from November 28 to December 1, 2018 in Naples.
Third General Assembly
Before atteding the A-T Clinical Research Conferences in Naples, the consortium meet to discuss the last achievements of the on-going Clincial Study.