The Parc Taulí Research and Innovation Institute (I3PT) has obtained funding for develop two projects presented to the competitive call "Public-private collaboration projects" of the State Research Agency of Ministry of Science and Innovation for a value of $733.241.
The granting of this endowment, as emphasized by the head of the entity's Projects Unit, Anna Aguilar, "will serve to continue researching therapies that improve the quality of life of patients with rare diseases. Both projects address specific needs of conditions that often have little visibility and resources for their research”.
Principal Investigator: Assumpta Caixàs | €542.566
Goal: Advance in the clinical development of PBF-999 for the treatment of Prader-Willi Syndrome (SPW). A phase II clinical trial will be developed to evaluate the safety, tolerability and efficacy of this component in SPW. In addition, in this project new reserve compounds will be designed and synthesized, and their pharmacological properties will be evaluated in different in vitro and in vivo tests, and the optimal ones will be selected. An intensive Industrial Property activity will be carried out in order to protect the new compounds and methods derived from the biomarkers found.
Principal Investigator: Ana Roche | €190.675
Goal: Developing a new disruptive therapy for Fragile X syndrome (FSX). SXF is a cognitive-behavioural disorder of genetic origin caused by a deficiency in the protein FMRP (Fragile X Messenger Ribonucleoprotein 1). The main objective of the proposal is to investigate CTH120 as an effective therapy for the treatment of SXF in adult patients through a Phase IIa clinical trial, through an interdisciplinary public-private collaboration between the company and entities of genomic and clinical research.
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