History
Atamyo is the first spin-off of Genethon in Gene Therapy. A pioneer in the development of gene therapies, Genethon has been working to find treatments for LGMDs for over 25 years. Researchers of the Progressive Muscular Dystrophies Laboratory at Genethon, under the leadership of Pr. Isabelle Richard, identified both the genes and the mechanisms responsible for many of these diseases and are using their skills and expertise in gene therapy and LGMDs to develop a new generation of effective and safe gene therapies for neuromuscular diseases. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD).
Genethon has been a pioneer in gene therapy for the last 30 years. Genethon’s teams have originated multiple gene therapy successes that are now bringing tremendous benefits for patients. Dedicated to tackle rare diseases, Genethon has accumulated a world-class expertise in the identification of gene targets, product design and manufacturing as well as preclinical and clinical development of gene therapies. With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases and cure these pathologies. Genethon is a not-for-profit organisation, created in 1990 by the French Muscular Dystrophy Association (AFM-Telethon)
Atamyo’s inception and strategic collaboration with Genethon to develop and design a gene therapy pipeline for neuromuscular diseases
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
First proof of principle for the efficiency of systemic gene transfer of gamma-sarcoglycan
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
Proof of principle for the efficiency of systemic gene transfer of FKRP
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
First proof of principle for the efficiency of systemic gene transfer of dysferlin with dual AAV (LGMD-R2/2B)
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
First proof of principle for the efficiency of systemic gene transfer of alpha-sarcoglycan by Genethon (Isabelle Richard team)
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
First proof of principle for the efficiency of gene transfer of calpain 3 by Genethon (Isabelle Richard’s team)
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
Participation of Isabelle Richard in the discovery of the FKRP gene
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
Participation of Isabelle Richard in the discovery of the dysferlin gene
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
First-ever discovery of an LGMD gene by Isabelle Richard during her PhD thesis
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
Participation of Isabelle Richard in the evidence that there were several LGMDs
Isabelle Richard, Ph. D
Co-founder & Chief Scientific Advisor
Dr. Isabelle RICHARD is a research Director at the CNRS. She is heading the Muscular Dystrophy team within the INSERM unit U951 at Genethon, Evry, France. Isabelle Richard is an expert in neuromuscular diseases and a pioneer in the research on LGMDs and in the development of gene therapy targeting LGMDs. She has published more than 160 scientific papers on muscular dystrophies. Among the most important highlights: identification of calpain 3 as the first gene implicated in an LGMD, demonstration of the heterogeneity of this group of diseases, participation in the identification of the gene of all the frequent LGMDs, proof-of-principle of the efficiency of gene therapy for all the most frequent LGMDs.
Participation of Isabelle Richard in the first demonstration that an LGMD was associated with a gene