FKRP

First Clinical Results of ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), Presented at ESGCT

Retrieved on: 
Friday, October 27, 2023
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Patient, FDA, United Kingdom, DSM-IV codes, IND, FKRP, International, Facial muscles, Conference, Cardiomyopathy, Weakness, Circulatory system, Data monitoring committee, Cell, Clinical trial, CNRS, Gene, ESGCT, Part, Rigshospitalet, Safety, European Society of Gene and Cell Therapy, Therapy, Muscular dystrophy, DSMB, Pharmaceutical industry, Vaccine, MD

ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).

Key Points: 
  • ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).
  • Atamyo also announced today that Data Safety Monitoring Board (DSMB) authorized the enrollment of the second dose cohort of the ATA-001 clinical trial.
  • ATA-100 is being evaluated in a multi-center phase 1b/2b in Denmark, France, and the United Kingdom, and has received IND clearance by FDA.
  • ATA-100, a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins.

ATA-200, Atamyo Therapeutics’ Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C/R5, Reaches Key Milestones with the Filing of a Clinical Trial Application in Europe and a Non-Dilutive Financing from France 2030 Program

Retrieved on: 
Tuesday, September 19, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Muscular dystrophy, Clinical trial, Pathology, Hope, CTA, Biomarker, AAV, Weakness, Patient, SGCG, Liver, Entrepreneurship, Therapy, Environment, Disease, DSM-IV codes, Safety, LGMD, Pharmaceutical industry, Vaccine, Dietary supplement, FKRP

No curative treatment exists for this disease,” said Dr Sophie Olivier, Chief Medical Officer of Atamyo.

Key Points: 
  • No curative treatment exists for this disease,” said Dr Sophie Olivier, Chief Medical Officer of Atamyo.
  • “ATA-200 incorporates a new promoter that enhances the liver and cardiac safety of gene therapy,” said Isabelle Richard, Ph.D., Co-founder and Chief Scientific Officer of Atamyo.
  • ATA-200, the gene therapy for LGMD2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan.
  • In addition to its LGMD2C/R5 gene therapy, Atamyo is developing a clinical trial with ATA-100 gene therapy for LGMD2I/R9, related to deficiencies in FKRP; and is in IND-enabling studies for LGMD2A/R1, related to deficiencies in calpain protein.

IND for ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), cleared to proceed by FDA

Retrieved on: 
Wednesday, September 6, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, FDA, Clinical Trials, Cardiomyopathy, Muscular dystrophy, FKRP, CNRS, Weakness, Patient, Therapy, IND, DSM-IV codes, Pharmaceutical industry, Vaccine

ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).

Key Points: 
  • ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).
  • ATA-100 (formerly known as GNT0006) is being evaluated in a multi-center phase 1b/2b in Denmark, France, and United-Kingdom.
  • ATA-100, a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins.
  • The therapy is based on the research of Atamyo Chief Scientific Officer Isabelle Richard, Ph.D., Research Director at CNRS who heads the Progressive Muscular Dystrophies Laboratory at Genethon.

AskBio Announces First Patient Dosed in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

Retrieved on: 
Thursday, August 3, 2023
Muscular dystrophy, Heart, Huntington's disease, Arm, Hearing, Bayer, Quality of life, Weakness, B cell, Multiple system atrophy, Glycogen storage disease type II, Leg, Department of Neurology, Xuanwu hospital, Doctor of Philosophy, RESEARCH, FKRP, Wasting, Parkinson's disease, MD, Patient, European, Knowledge, Entrepreneurship, Research, FDA, VCU School of Medicine, Safety, PARK, LGMD, Disease, Pharmaceutical industry

Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9.

Key Points: 
  • Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9.
  • "Hearing that the first patient has been dosed in this study evaluating AB-1003 is an exciting moment for the limb-girdle muscular dystrophy community and individuals living with this debilitating disease," said Kelly Brazzo, Co-Founder & CEO, CureLGMD2i.
  • "Given the current lack of disease modifying treatments for LGMD, many in the community know of the potential of gene therapy.
  • The initiation of this trial offers hope that patients with this condition may, in the future, have a significantly improved quality of life."

BridgeBio Pharma Announces Opportunity for Accelerated Approval Pathway in Limb-girdle Muscular Dystrophy Type 2I (LGMD2I/R9) Based on Glycosylated Alpha-dystroglycan (⍺DG) Levels and Announces First Patient Dosed in FORTIFY Phase 3 Study

Retrieved on: 
Monday, July 31, 2023
WMS, Creatine kinase, Myopathy, University, PALO, CDKL5 deficiency disorder, Fear, Pathology, Quality of life, Clinical trial, B cell, University of California, ClinicalTrials.gov, Accelerated approval (FDA), FKRP, Patient, Love, Cancer, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, Neurology, Food, NSAD, LGMD, Disease, Pharmaceutical industry, Medical imaging, Agency

BridgeBio also announced that the first patient with LGMD2I/R9 has been dosed in its Phase 3 FORTIFY clinical trial of BBP-418.

Key Points: 
  • BridgeBio also announced that the first patient with LGMD2I/R9 has been dosed in its Phase 3 FORTIFY clinical trial of BBP-418.
  • The Phase 3 FORTIFY registrational study is a randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of BBP-418.
  • FORTIFY has a planned interim analysis at 12 months focused on assessing glycosylated αDG as a surrogate endpoint to potentially support an accelerated approval.
  • “Currently, our Phase 2 data suggest that glycosylated αDG levels are improved and sustained over time following treatment with BBP-418.

AskBio Announces 11 Presentations at the American Society of Gene and Cell Therapy 26th Annual Meeting in 2023

Retrieved on: 
Thursday, May 11, 2023
Muscular dystrophy, Huntington's disease, Associate Director of National Intelligence and Chief Information Officer, Gene, CC, Heart, Bayer, Assay, Genetic diversity, Multiple system atrophy, Glycogen storage disease type II, Research, AAV, RESEARCH, Innate resistance to HIV, FKRP, Parkinson's disease, Poster, Patient, Annual general meeting, Cell, ISCB Senior Scientist Award, Mouse, R, American Society of Gene and Cell Therapy, Society, BB, Library, AA, PARK, Kinetics, Vaccine, Pharmaceutical industry, Fine chemical

AskBio presentations cover pre-clinical research into immune responses to AAV, data on methods to improve the efficacy of AAV-gene therapy and research into improved analytics and bioassays.

Key Points: 
  • AskBio presentations cover pre-clinical research into immune responses to AAV, data on methods to improve the efficacy of AAV-gene therapy and research into improved analytics and bioassays.
  • By targeting these therapy areas, AskBio aims to deliver breakthrough treatments that could benefit more than 35 million patients worldwide.1–7
    AskBio's presentations at ASGCT include:
    A Platform Approach for Adeno-Associated Virus Manufacturing to Support Gene Therapy Products.
  • Presented by Audry Fernandez, R&D, Asklepios BioPharmaceutical, Inc. [Oral presentation: 16:00 May 18 (Room 411)]
    Impact of Genetic Diversity on Gene Therapy Efficacy.
  • Presented by Sandro Alves, Head preclinical (France), Asklepios BioPharmaceutical, Inc. [Poster presentation: 12:00 May 19 (Poster #1261)]

Third International Limb Girdle Muscular Dystrophy Conference to Be Held in Washington, D.C. On October 27-29, 2023

Retrieved on: 
Wednesday, April 5, 2023
Linda, International, Drug development, University, Nationwide Children's Hospital, ALL, COVID-19, Clinical trial, Center for Biologics Evaluation and Research, Grand Hyatt Washington, FKRP, Sarepta Therapeutics, Patient, Physician, Virginia Commonwealth University, IT University of Copenhagen, Diagnosis, Hospital, FDA, MDA, Therapy, Conference, NIH, Caregiver, LGMD, Medical device, Research

LANCASTER, Pa., April 5, 2023 /PRNewswire/ -- On October 27, 2023, the third International Limb Girdle Muscular Dystrophy (LGMD) Conference will be hosted by The Speak Foundation at the Grand Hyatt Washington, DC.

Key Points: 
  • LANCASTER, Pa., April 5, 2023 /PRNewswire/ -- On October 27, 2023, the third International Limb Girdle Muscular Dystrophy (LGMD) Conference will be hosted by The Speak Foundation at the Grand Hyatt Washington, DC.
  • The second International LGMD conference was in 2021 and was offered in a virtual format due to COVID-19.
  • The conference will be back in person this year and will be offering live-streamed sessions for those unable to attend.
  • There will be a number of "breakout sessions" at the Conference geared toward providing practical advice to the patient community.

BridgeBio Pharma Shares Preliminary Findings on Novel Bioassay Measuring Glycosylated Alpha-dystroglycan (⍺DG) in Patients with Limb-girdle Muscular Dystrophy Type 2I (LGMD2I)

Retrieved on: 
Tuesday, March 21, 2023
Linda, Creatine kinase, Myopathy, Loma Linda University School of Public Health, Department of Nutrition, Natural history, Ribitol, PALO, Calendar, Loma Linda University, B cell, Month, FKRP, Muscle weakness, Patient, Cancer, Webcast, MDA, Glycosylation, Neurology, NSAD, Disease, Pharmaceutical industry, Medical imaging, Fine chemical

Additionally, BridgeBio shared updated results at Month 15 for its Phase 2 clinical trial and the design for its pivotal Phase 3 study for patients with LGMD2I.

Key Points: 
  • Additionally, BridgeBio shared updated results at Month 15 for its Phase 2 clinical trial and the design for its pivotal Phase 3 study for patients with LGMD2I.
  • The novel bioassay was developed by BridgeBio to measure the amount of glycosylated ⍺DG, and to evaluate the impact of BBP-418 treatment.
  • The bioassay allowed the Company to evaluate natural history and Phase 2 data to inform the Phase 3 study design.
  • If approved, BridgeBio believes BBP-418 could be the first approved orally administered therapy for the treatment of patients with LGMD2I.

BridgeBio Pharma Presents 12-month Results from Phase 2 Study of BBP-418 in Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i)

Retrieved on: 
Friday, October 14, 2022
LinkedIn, Lists of diseases, Department of Neurology, Xuanwu hospital, Cardiomyopathy, Time, Congress, Drug Quality and Security Act, Disease, SEC, LVEF, Securities Exchange Act of 1934, Science, VCU, WMS, Solution, Annual report, Patient, Human, Creatine kinase, Genotype, Twitter, Virginia Commonwealth University, CK, Nasdaq, NSAD, Securities Act of 1933, Biomarker, Company, Mutation, Cardiac muscle, Poster, Neurology, FKRP, Safety, Glycosylation, Environment, GLOBE, Myopathy, Pathology, Cancer, Muscular dystrophy, Risk, Medical imaging, Pharmaceutical industry, Medical device

BridgeBio and ML Bio Solutions continue to engage with regulatory agencies and intend to initiate a Phase 3 clinical trial in 2023.

Key Points: 
  • BridgeBio and ML Bio Solutions continue to engage with regulatory agencies and intend to initiate a Phase 3 clinical trial in 2023.
  • Given impaired glycosylation of DG directly leads to the development of muscular dystrophy in LGMD2ipatients, this novel assay was designed to assess the core pathology of the disease.
  • The Phase 2 trial enrolled 14 participants, including both ambulatory and non-ambulatory patients with LGMD2i.
  • Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time.

Atamyo Therapeutics Announces First Patient Dosed with ATA-100 Gene Therapy in LGMD-R9 Clinical Trial

Retrieved on: 
Monday, September 26, 2022
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Safety, Trial of the century, FR, EudraCT, UMR, AAV, Food, European Medicines Agency, Weakness, Hope, Muscular dystrophy, National Hospital Organization Nagara Medical Center, Lists of diseases, Life, Therapy, Clinical trial, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Patient, FKRP, LGMD, Degenerative disease, Rigshospitalet, Research, Motivation, Pharmaceutical industry, Vaccine

John Vissing, Director of the Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet, in Copenhagen, where the first patient was dosed, and principal investigator of this trial.

Key Points: 
  • John Vissing, Director of the Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet, in Copenhagen, where the first patient was dosed, and principal investigator of this trial.
  • Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases.
  • A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon.
  • Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments.