| Il progetto di terapia genica per la LGMD2E |
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2012: INIZIA IL PROGETTO DI TERAPIA GENICA FINANZIATO DA GFB Nel 2012 inizia il progetto di trasferimento del gene Beta-sarcoglicano per il trattamento della distrofia muscolare di tipo 2E all'Istituto di Ricerca Nationwide Children's Hospital di Columbus Ohio.
I Dr. JR Mendell e Dr.ssa L. Rodino Klapac iniziano il loro lavoro, che si basa sulle loro esperienze acquisite completando uno studio simile per il trattamento, attraverso una terapia di trasferimento genico, della Distrofia muscolare dei cingoli di tipo 2D.
2015: CONCLUSA LA FASE 2
Terminata la seconda fase del progetto. Nell'agosto 2015 sono stati pubblicati la prima e la seconda fase del progetto sulla rivista Gene Theraphy. Beta-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice .
2016: VERSO IL TRIAL CLINICO
Il 17 Marzo il GFB Onlus partecipa al congresso Myology 2016 a Lione, dove incontra il Dr. Mendell. Sono presenti Beatrice Vola (presidente GFB), Marco Perlini (Vicepresidente), Dr. Francesca De Santis (ospedale Policlinico di Milano) e una interprete.
SEPTEMBER 2016: Un altro grande sforzo per l'associazione che ha sede a Talamona in provincia di Sondrio. Venerdì 9 settembre GFB ONLUS ha inviato un altro contributo a Columbus Ohio, di 202.500 $. Dal 2012 ad oggi le famiglie del GFB hanno supportato finanziariamente i laboratori americani per un totale di 1.322.500 $. GFB è attualmente l'unico finanziatore del progetto.
MAGGIO 2017 : nasce la company Myonexus Therapeutics - il viaggio verso la sperimentazione clinica. L'obiettivo della company Myonexus Therapeutics, nata a Columbus nel maggio 2017, è quello di sviluppare la terapia genica per la distrofia muscolare dei cingoli (LGMD) di tipo 2B, 2C, 2D, 2E e 2L (entro l'anno 2027). GFB Onlus è membro di Myonexus. La nostra associazione, che rappresenta uno dei sostenitori della company, ha inviato il 15 giugno 2017 un importo totale di 225.000 $. Insieme a GFB Onlus, anche la Fondazione LGMD2D e la Fondazione KURT + PETER hanno contribuito a questo progetto. In particolare, per quanto riguarda LGMD2E, sono previsti due diversi studi clinici, rispettivamente per gli anni 2017 e 2020; l'ultimo (del 2020) avrà luogo anche a Milano.
APRILE 2018: Myonexus Therapeutics riceve dalla FDA la designazione di farmaco orfano per il trattamento della distrofia dei cingoli di tipo 2E.
MAGGIO 2018: GFB Onlus annuncia che Myonexus, una società di biotecnologie in cui ha investito, ha firmato una partnership esclusiva con Sarepta Therapeutics, per l'avanzamento di molteplici programmi di terapia genica, finalizzati al trattamento di forme distinte di distrofie muscolari dei cingoli.
2018: IN OTTOBRE VENGONO TRATTATI I PRIMI 3 PAZIENTI
Myonexus Therapeutics ha annunciato l'avvio di uno studio clinico di terapia genica sistemica per la beta-sarcoglicanopatia o LGMD2E. Il primo paziente della sperimentazione è stato curato nell'ottobre 2018 e tutti i pazienti del primo gruppo saranno trattati nel 2018, i primi risultati saranno annunciati all'inizio del 2019. Michael Triplett, presidente di Myonexus, ha dichiarato che Myo-101 può essere la prima terapia completa per i pazienti LGMD2E. Myo-101 è il culmine di anni di duro lavoro ed esperimenti, ha aggiunto il dott. Jerry Mendell.
FEBBRAIO 2019: RESULTATI PRELIMINARI SUL TRIAL CLINICO PER LA LGMD2E E ACQUISIZIONE DI MYONEXUS
Il 27 febbraio Sarepta ha pubblicato i risultati preliminari sul primo studio di terapia genica condotto su pazienti con LGMD2E a Columbus, Ohio, USA. Programma in cui i primi tre pazienti trattati hanno dimostrato l'espressione robusta e diffusa della proteina beta-sarcoglicano. A seguito di questi promettenti risultati, Sarepta ha annunciato che acquisirà Myonexus Therapeutics.
OTTOBRE 2019: La terapia genica SRP-9003 mostra risultati molto incoraggianti a 9 mesi nelle LGMD, comunicato di Sarepta
Nuovi dati a nove mesi di trattamento nel trial di fase 1 mostrano che una singola somministrazione del terapia genica SRP-9003 (precedentemente Myo-101) a basso dosaggio migliora significativamente le misure funzionali e abbassa i livelli di un biomarcatore di danno muscolare in tre bambini con distrofia muscolare dei cingoli (LGMD) tipo 2E.
FEBBRAIO 2020: SAREPTA ANNUNCIA: "abbiamo trattato altri tre pazienti ora nel gruppo ad alto dosaggio per la LGMD2E. I risultati di espressione e sicurezza saranno disponibili nel secondo trimestre e prevediamo di annunciare tali dati in occasione di un meeting medico nel secondo trimestre".
GIUGNO 2020: Sarepta annuncia positivi dati funzionali e di espressione per il trial di terapia genica SRP-9003 per trattare la lgmd2e. Nel comunicato sono riportati dati positivi a 3 mesi di trattamento ad alto dosaggio. Inoltre nei pazienti trattati a basso dosaggio ci sono ancora miglioramenti anche dopo i 9 mesi di trattamento.
If you or anybody else is interested in this project and wishes to get more information about it, please contact us to: info@beta-sacrcoglicanopatie.it
FUND FOR RESEARCH In 2015 the GFB ONLUS had to change its accounting system so to be able to to finance, for the first time, the American projects directly from the association’s bank account. For this purpose a special FUND FOR RESEARCH was created on its bank account of Banca Prossima. All donations on the account will be used to finance theAmerican projects and the scientific researches on the LGMD2E. HEADING FOR RESEARCH FUND: Gruppo Familiari Beta-sarcoglicanopatie Onlus IBAN: IT33X0335901600100000076500 BIC /SWIFT code BCITITMX
The GFB ONLUS is the only association in the world existing for LGMD2E, this project for the gene therapy is the only ongoing project in this moment for this pathology and the only hope for these patients.
AGENDA OF THE PROJECT:
November 8th-11th 2011: Participation of Beatrice Vola in the TREAT-NMD INTERNATIONAL CONFERENCE in GENEVA; 8-11 NOVEMBER 2011 meeting: Beatrice Vola and Prof. Jerry Mendell, Columbus USA. May 16th 2012: first conference call with Dr. Mendell and Dr. Rodino, participation of Dr. Cerletti and Bonetti, Fam. Gonella Andrea, Beatrice Vola and Perlini Marco. July 24th 2012: second conference call with Dr. Mendell and Dr. Rodino, participation of Dr. Cerletti and Bonetti, Fam. Gonella Andrea, Beatrice Vola and Perlini Marco. Start of the first phase with a funding of $ 172,500 November 1st-2nd 2012: Meeting: Dr. Massimiliano Cerletti in Columbus OHIO USA and Dr Jerry Mendell for discussing the state of gene therapy on sarcoglycan and the ongoing project on the beta. April 19th 2013: First National Conference GFB Milan, meeting: the GFB, Dr. Mendell and Dr. Rodino.
First National Congress GFB, april 19th 2013, Milan Italy. From left Gonnella A., Dr.ssa Rodino Klapac L., Prof. Mendell J., Vola B., Dr.ssa Bonetti P., Perlini M.
October 28th 2013: Third Conference Call of the GFB, Dr. Mendell and Dr. Rodino, participation of Dr. Cerletti and Bonetti, Fam. Gonella Andrea, Beatrice Vola and Perlini Marco for discussing on the report of the first phase. Start of the second phase with the second contribution of $ 373,750. September 2014: Fourth conference call with Dr. Mendell (participating Dr. Rodino, Dr. Cerletti, Bonetti, Vola, Perlini, Gonnella) for an update on the progress of the second phase. An additional contribution of $ 373,750. January 15th 2015: meeting "Gene Therapy and clinical trials for MD" with Dr. Alessandra Govoni, Policlinico of Milan (Participants Gonella, Dr. Bonetti, Perlini, Vola). Dr. Alessandra Govoni worked in 2014 in Columbus with Prof. Mendell. august 20th 2015: the article "Beta-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice" of Prof. Mendell and Rodino was pubblished on the scientific magazine Gene Therapy. November 20th 2015: fifth conference call with Prof. Mendell (partecipating Dr.ssa Rodino, Dr. Pozgay, Cerletti, Bonetti, Torrente, Vola, Perlini, Gonnella) for discuss the start of the clinical trial for LGMD2E. Start of the first clinical trial for LGMD2E with the fourth contribution of $ 200,000. March 17th 2016: Myology 2016 Congress, Lyon (Partecipating Prof. Mendell, Perlini, Vola, De Santis Francesca for Yvan Torrente), for discussing the systemic clinical trial for LGMD2E.
From left Dr.ssa De Santis F., Vola B., Prof. Mendell J., Perlini M.
March 24th 2016: sexth conference call with Prof. Mendell (partecipating Prof Mendell, Dr.ssa Rodino, Torrente, Bonetti, Vola, Perlini, Gonella), for discussing the systemic clinical trial for LGMD2E. May 5th 2016: GFB ONLUS Scientific-medical Comittee meeting at Policlinico of Milan (partecipating Prof Torrente, Dr. Bonetti, Vola, Perlini, Gonella, Rigoni), for discussing the fifth report of Columbus. GFB ONLUS families and sponsors meeting (partecipating families Vola, Perlini, Gonnella, Rigoni, Ferrari, Gazzola, Adamczyk, Vinci for Walter Vinci Onlus association), for discussing the american gene therapy projects. September 8th 2016: seventh conference call with Dr. M. Triplett (partecipating Dr. Bruce Halpryn, Vola, Perlini, Gonnella) for discussing the Myonexus Therapeutics Company. An additional contribution of 202.500 $. April 4th 2017: the article "Systemic AAV-Mediated Beta-sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorated Histological and Funcional Deficits in LGMD2E Mice" of Prof. Mendell and Rodino was pubblished on the scientific magazine Gene & Cell Therapy. April 20th 2017: eight conference call with Dr. M. Triplett (partecipating Rev1 Ventures, Dr. Bruce Halpryn, prof. Torrente, Vola, Perlini, Gonnella) for discussing the Myonexus Therapeutics Company. May 15th 2017: The Myonexus Therapeutics Company was born. June 15th 2017: GFB ONLUS enter into the Myonexus Therapeutics Company with 225.000 $. April 30th 2018: ninth conference call with Dr. M. Triplett (partecipating Vola, Perlini, Gonnella) for discussing the agreement between GFB Onlus - Myonexus - Sarepta for Myo-101 develop. May 2th 2018: GFB Onlus participates in the signing of the Myonexus - Sarepta agreement. May 17th 2018: first meeting of SAB (Scientific Advisor Board) in Chicago, the GFB is represented by Yvan Torrente, it is the first meeting of the scientific medical committee of Myonexus. June 13th 2018: tenth telephone call with the PAB (Patient Advisor Board), (participates Vola Perlini), it is the first meeting of the Myonexus patient committee. The role of patient associations is discussed. October 2018: the first gene therapy treatments on LGMD2E patients start in Columbus. The project for the creation of an Italian patient registry for LGMD2E begins at the Milan Polyclinic. November 30th 2018: eleventh telephone call for the patient register (Vola, Perlini, Dr. Torrente Yvan, Marchetti Giulia, Bruce Halpryn, Chris Jones participate). Coordination for the creation of registries for LGMD2E and other LGMD2D-2C dystrophies starts. February 27th 2019: twelfth telephone call (Vola, Perlini, Michael Triplett participate). The preliminary results of the clinical trial for LGMD2E and the early acquisition of Myonexus in Sarepta are discussed. March 11th 2019: webinar of Sarepta with members of the PAB Myonexus Patients Advisor Board. May 1st 2019: ASCGT congress in Washington, Dr. Torrente meet Sarepta Staff and prof. Mendell. May 21st-23th 2019: webinar (Tamara Wyzansky, other Sarepta Members, other american patient foundations, Beatrice Vola, Perlini Marco, Marchetti Giulia). Patients role and clinical trials structure are discussed. July 31th 2019: fourteenth telephone call (Tamara Wyzansky-Sarepta, Beatrice Vola, Marco Perlini). The creation of a mosaic with patients photographs with Lgmd and their cargivers was discussed. October 4th 2019: Sarepta Reports - Gene Therapy SRP-9003 Showing Very Encouraging Results at 9 Months in LGMD2E.
COMUNICATI STAMPA
2020 SRP-9003 Gene Therapy for LGMD2E Shows Benefit after 1 year - Muscular Dystrophy News Today - June 9 2020 New data gives hope for Limb Girdle Muscular Dystrophy gene therapy - Patient Worthy 17 giugno 2020
2019 Clinical Update: Myo-101 For LGMD Type 2E Business update: Myonexus Acquisition - Sarepta - february 27 2019 Distrofia dei cingoli, Sarepta decide di acquisire Myonexus - Osservatorio Malattie Rare - march 12 2019 Distrofia dei cingoli 2E, primi risultati clinici favorevoli per la terapia genica - Osservatorio Malattie Rare OMAR - April 12 2019 How an Ohio Kids' hospital Quitly Became Groung Zero for Gene Therapy - Xconomy - april 15 2019 Gene Delivery for Limb Girdle Muscular Dystrophy Type 2D (LGMD2D) by Isolated Limb Infusion - PubMed.gov - July 2019 La terapia genica SRP-9003 mostra risultati "molto incoraggianti" a 9 mesi nella distrofia dei cingoli - Sarepta Reports - october 9 2019 Distrofia dei cingoli 2E, nuovi risultati positivi per la terapia genica SRP-9003 - Osservatorio Malattie Rare OMAR - october 22 2019
2018 Myonexus Therapeutics recieves orphan drug designation for Myo-102 - Myonexus Clinical Update: Micro-dystrophin Study-101 - Sarepta
Myonexus Therapeutics announced the initiation of a trial for the first-ever Gene Therapy for Beta-Sarcoglycanopathy MDA Funds Creation of Limb-Girdle Muscular Dystrophy Clinical Research Network to Speed Therapy Development
Atrofia Muscolare Spinale: a luglio avvio in Italia sperimentazione di terapia genica. http://www.dire.it/21-07-2018/222201-atrofia-muscolare-spinale-avvio-sperimentazione-terapia-genica/
Positive preliminary results from the first three children dosed in phase 1/2A gene Therapy micro-dystrophin trial
Our vision for the future of Precision Genetic Medicine - Sarepta Therapeutics 2018 R&D Day
Myonexus Therapeutics receives FDA Rare Pediatric Disease Designation https://www.eurekalert.org/pub_releases/2018-05/c-mtr051618.php
Sarepta Therapeutics Announces Partnership with Myonexus Therapeutics for the Advancement of Multiple Gene Therapy Programs Aimed at Treating Distinct Forms of Limb-Girdle Muscular Dystrophies
Myonexus Therapeutics receives FDA orphan drug designation for LGMD type 2E treatment Pioneering, corrective gene therapies have potential to transform quality of life https://www.eurekalert.org/pub_releases/2018-04/c-mtr041918.php
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study https://clinicaltrials.gov/ct2/show/results/NCT03492346?view=results
MDA Clinical Conference Keynote Adress by Dr. Jerry Mendell - March 2018 https://www.youtube.com/watch?v=ScVLvXfBwYM
2018 Clinical Research Forum Top Ten Clinical Research Achivement Awards https://www.youtube.com/watch?v=fubjZ-YQEdo
How Myonexus Therapeutics is developing the first treatment for Limb Girdle Muscular Dystrophies
Young Boy Becomes First DMD Patient to Receive Investigational Systemic Microdystrophin Gene Therapy
First Duchenne Patient Dosed in Microdystrophin Gene Therapy! http://community.parentprojectmd.org/m/blogpost?id=1187424%3ABlogPost%3A245554
Myonexus Therapeutics Secures $ 2,5 Million Seed Financing to Clinically Advance Limb-Girdle Muscular Dystrophy (LGMD) Gene Theraphies https://www.businesswire.com/news/home/20171213005559/en
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy http://www.nejm.org/doi/full/10.1056/NEJMoa1706198#.WfsctKM_52d.facebook
Clinical Trials Bring Hope to Kids with Spinal Muscular Atrophy
FDA Clears IND Application for Micro-dystrophin Gene Therapy Program to Treat Duchenne
Sarepta & Nationwide Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne
Gene Therapy Continues to Show Promise for Limb-Girdle Muscular Dystrophy
Myonexus Therapeutics
Myonexus Therapeutics, Inc. Launches to Develop Pioneering, Clinical-Stage Gene Therapies Targeting Limb-Girdle Muscular Dystrophies
Myonexus Launches Development of Gene Therapies Targeting Limb-Girdle MD
AveXis Receives FDA Breakthrough Therapy Designation for AVXS-101 (Gene Therapy) for SMA http://www.curesma.org/news/avexis-receives-breakthrough.html?referrer=https://www.facebook.com/ New Gene Therapy Results Announced at World Muscle Congress http://www.curesma.org/news/gene-therapy-world-muscle-congress.html?referrer=https://www.facebook.com/Good news from the second patients treated in Columbus with the gen therapy into the legs. https://www.facebook.com/photo.php?fbid=716718035100280&set=a.131275193644570.2 1134.100002862707671&type=1&theater
Clinical trial for Max http://www.gofundme.com/oj0ono Dr. Jerry Mendell discusses Gene Therapy. Dr. Jerry Mendell shares his experiences from a decade of research and the challenges in developing gene therapy for muscular dystrophy at the Jain Foundation USA patient conference. https://www.youtube.com/watch?v=tm4G4hZdJlE
Human α7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient Mice http://on line.liebertpub.com/doi/10.1089/hum.2015.062
Pioneering gene therapy may save babies’ lives http://www.dispatch.com/content/stories/local/2015/06/17/reaching-for-hope-and-a-cure.ht
With Kinect, Kids May Clinch Clinical Trials (Essay) https://www.facebook.com/NationwideChildrensHospital/posts/10153166764440135
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models http://onlinelibrary.wiley.com/doi/10.1002/acn3.172/abstract;jsessionid=C126B52A2B26047B |
