December 2022 – Fratagene Therapeutics terminated its activities. The main goal of the company, i.e. finding potential therapeutics for Friedreich ataxia, has been achieved, with five clinical trials completed, prompted by Fratagene’s original and proprietary discoveries. We are grateful to all individuals, scientists and patients, that contrubuted their time, effort, resources and enthusiasm, to this endehavour.
October 2020. The first Phase 2 clinical trial aimed at evaluating the “Safety and efficacy of Etravirine in Friedreich ataxia patients”, conducted at the IRCCS Medea Scientific Institute, Conegliano, Italy, has completed patients recruitment (ClinicalTrials.gov Identifier: NCT04273165).
June 2020. AtaxiaUK, a major European patients association, will co-fund Fratagene's effort at investigating Etravirine as a potential therapeutic for Friedreich ataxia, particularly focusing on elucidating the molecular mechanism through which etravirine, and its analogs, increases frataxin in frataxin-defective cells.
January 2020. Results from the Phase 2 clinical trial (ClinicalTrials.gov Id: NCT03888664), conducted at the Medea Scientific Institute, Conegliano, Italy, in collaboration with Fratagene Therapeutics, the Department of Biomedicine and Prevention of the University of Rome "Tor Vergata" and Harvard Medical School, Boston, aimed at assessing the safety and efficacy of interferon gamma (Imukin) in FA patients, indicate that the drug is generally well tolerated and that it could completely stop disease progression during treatment. Data have been published in Movement Disorders.
November 2019. Fratagene Therapeutics to present exciting new data at the International Ataxia Research Conference, in Washington DC, November 14-16.
October 2019. The USPTO granted Fratagene Therapeutics US patent 10,426,775, on the use of Etravirine as a therapeutic for Friedreich ataxia and US patent 10,442,779, on novel small molecules that increase Frataxin by preventing its ubiquitination.
September 2019. The CureFA Foundation, Rye, NY, USA, has joined the ownership of Fratagene Therapeutics. The CureFA foundation funds scientific projects and industry partnership that leverage new technologies for the treatment and cure of Friedreich ataxia. Its assets in Fratagene will help support the first clinical trial aimed at assessing the safety and efficacy of etravirine in FA patients.
January 2019. Researchers at Fratagene Therapeutics, in collaboration with researchers at the Department of Biomedicine and Prevention of the University of Rome “Tor Vergata” and at Harvard Medical School, have identified etravirine, a well known anti-retroviral drug in use for the treatment of HIV infection, as a drug capable to elevate frataxin levels in frataxin-defective cells. Because of its excellent safety profile, etravirine may therefore represent a promising therapeutic for Friedreich ataxia. The results of this work have been published in Movement Disorders.
April 2018. The United States Patent and Trademark Office granted Fratagene Therapeutics the US Patent 9,944,906 "Frataxin mutants", on the possible therapeutic use of long-lasting frataxin.
January 2018. Fratagene Therapeutics to present new drug discovery strategies for Friedreich ataxia at the Keystone Symposia "Ubiquitin Signaling", Tahoe City, CA, USA, January 28 - February 1.
November 2017. The European Research Council invites Fratagene Therapeutics to present its discovery programs at the Slush, the world’s leading venturing event for startups to be held in Helsinki, November 29 - December 1, during the Slush y Science vertical.
September 2017. Fratagene Therapeutics to present exciting new findings at the International Ataxia Research Conference to be held in Pisa, Italy, on September 27-30.
February 2017. Researchers at Fratagene Therapeutics, in collaboration with researchers at the Department of Biomedicine and Prevention of the University of Rome “Tor Vergata”, have identified the enzyme RNF126 as a new therapeutic target for Friedreich ataxia. The investigators found that RNF126 is the ubiquitin E3 ligase responsible for the degradation of frataxin. Molecular strategies aimed at the inhibition of RNF126 would pave the way to the development of a new class of drugs able to prevent frataxin degradation, thus restoring frataxin levels in patients and providing hope of cure. The results of this work have been published in Cell Reports.
January 2017. Fratagene Therapeutics to fully support the salary of one investigator, that will establish in vitro cultures of sensory neurons, differentiated form iPSC derived from Freidreich ataxia patients, as a cellular disease model.
November 2016. Fratagene Therapeutics to fully support the salary of one investigator, that will be involved in the analysis of frataxin modifications, at the Department of Biomedicine and Prevention, University of Rome “Tor Vergata”
October 2016. Dr. Mark Pook, Brunel University, London UK and Dr. Javier Santos, IQUIFIB, University of Buenos Aires, Argentina, have joined the Scientific Advisory Board. Dr. Pook developed some of the best animal models for Friedreich ataxia currently available. Dr. Santos is among the top experts in frataxin structure and dynamics.
August 2016. Fratagene Therapeutics to fully support the salary of two investigators, that will be involved in research projects concerning the modulation of frataxin ubiquitination and phosphorylation as possible therapeutic strategies for Friedreich ataxia, at the Department of Biomedicine and Prevention, University of Rome “Tor Vergata”.
July 2016. Fratagene Therapeutics to fund, with 0.5 M euros, research programs combining both drug discovery and drug repositioning approaches, that may lead to new therapies for Friedreich ataxia, within the premises of the Department of Biomedicine and Prevention, University of Rome “Tor Vergata”.
June 2016. Fratagene Therapeutics and the University of Rome “Tor Vergata” enter a framework agreement aimed at supporting the discovery and development of new therapies for Friedreich ataxia.
April 2016. Fratagene Therapeutics and Colosseum Combinatorial Chemistry Centre for Technology, Rome, team to develop small molecules that could act as inhibitors of frataxin ubiquitination and degradation, as potential new drugs for Friedreich ataxia.
March 2016. Fratagene Therapeutics and SARomics Biostructures, Lund, Sweden, will collaborate to elucidate structure-function relationships of the E3 ligase responsible for frataxin ubiquitination, a potential therapeutic target for Friedreich ataxia.
February 2016. Fratagene Therapeutics and Horizon Pharma, Dublin, Ireland, enter a license agreement on the possible use of interferon gamma as a therapeutic agent for Friedreich ataxia.
November 2015. The European Research Council (ERC) awards the 2015 Proof-of-Concept Grant to Prof. Roberto Testi, as a continuation of a previously awarded ERC Advanced Grant, to help develop a cure for Friedreich ataxia and bring it to market. Fratagene Therapeutics srl is created in Rome.