GALC

Orphan designation: Adeno-associated virus serotype rh10 containing the human GALC gene Treatment of Krabbe disease, 15/10/2021 Positive

Retrieved on: 
Tuesday, April 9, 2024
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Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of Krabbe disease in the European Union on 15 October 2021.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
  • For more information, see:
    Key facts
    - Active substance
    - Adeno-associated virus serotype rh10 containing the human GALC gene
    - Intended use
    - Treatment of Krabbe disease
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/21/2511
    - Date of designation
    - Sponsor
    Forge Biologics Europe S.L.

Jaya Biosciences Presents Promising Preclinical Data in Alzheimer’s Disease at the 20th Annual WORLDSymposium™ 2024

Retrieved on: 
Monday, February 12, 2024
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SOUTH SAN FRANCISCO, Calif., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Jaya Biosciences, Inc. (“JayaBio” or “the Company”), a privately held early-stage life-sciences company developing CNS-directed gene therapies to address unmet needs in genetically defined neurodegeneration, reported non-clinical data at the 20th Annual WORLDSymposium™, a leading research conference on lysosomal diseases. During a late-breaking news platform presentation, Jaya Biosciences’ scientific founder, Prof. Mark Sands, reported preliminary human genetic analysis suggesting that heterozygous loss-of-function mutations in lysosomal enzyme genes are enriched in Alzheimer’s patients, as well as preclinical efficacy results in the animal model of Alzheimer’s disease (AD) for JB111, the Company’s lead therapy.

Key Points: 
  • “We are thrilled to showcase a new paradigm for the treatment of genetically defined neurodegeneration at the 20th WORLDSymposium™,” said Pawel Krysiak, President and CEO of JayaBio.
  • “The preclinical data we presented demonstrate a tremendous promise of targeting PPT1 haploinsufficiency in Alzheimer’s disease.
  • Heterozygosity of five different lysosomal enzyme genes (PPT1, NAGLU, GALC, IDUA, GUSB) significantly affects amyloid precursor protein (APP) processing and favors pro-amyloidogenic pathway.
  • CNS-directed, AAV-mediated gene therapy significantly increases the life span and improves cognitive function of 5xFAD/PPT1+/- mice.

Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote

Retrieved on: 
Monday, January 29, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Mortality, Death, HSCT, Patient, Legislation, GALC, Advisory Committee, AAV, Psychosine, ESGCT, Data, Gene, DSM-IV codes, Neurodegenerative disease, Disease, Safety, Central nervous system, Sphingolipid, Physician, Krabbe disease, Cell, Human services, Parent, Nervous system, Clinical trial, Forge, Peripheral nervous system, Hematopoietic stem cell transplantation, Brain, UCBT, Pharmaceutical industry

Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.

Key Points: 
  • Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.
  • Previously published data have demonstrated that patients with Krabbe treated with HSCT demonstrate increased lifespan and stabilization of neurodegenerative disease in the central nervous system.
  • FBX-101, an investigational adeno-associated viral (AAV) gene therapy, has been designed to address the peripheral nerve disease not corrected by HSCT.
  • As a result, after a year of patient and foundation advocacy, Krabbe disease is again being voted on for potential inclusion on the RUSP.

Forge Biologics Reports Positive FBX-101 Clinical Updates for Patients with Krabbe Disease at WORLDSymposium

Retrieved on: 
Thursday, February 23, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Cerebrospinal fluid, Psychosine, Safety, Central nervous system, University, HSCT, Improvement, GALC, Risk, CSF, Krabbe disease, Hematopoietic stem cell transplantation, Standard of care, Intravenous therapy, CNS, PNS, Inflammation, Plasma, Infant, Human, AAV, Peripheral nervous system, Multimedia, Pharmaceutical industry, Patient, Forge

Forge has also dosed the first FBX-101 subject in the REKLAIM Phase 1b clinical trial at the University of Michigan Medical Center.

Key Points: 
  • Forge has also dosed the first FBX-101 subject in the REKLAIM Phase 1b clinical trial at the University of Michigan Medical Center.
  • View the full release here: https://www.businesswire.com/news/home/20230223005450/en/
    Patients with infantile Krabbe disease have mutations in the gene encoding the lysosomal enzyme galactocerebrosidase (GALC), which is essential for normal metabolism of myelin components.
  • This results in progressive motor disease and often early death of patients by two years of age.
  • Notably, some of the patients were identified by newborn screening, which enabled early disease intervention,” stated Dr. Escolar.

Forge Biologics Receives Priority Medicines (PRIME) Designation from the European Medicines Agency (EMA) for Novel Gene Therapy FBX-101 for the Treatment of Patients with Krabbe Disease

Retrieved on: 
Tuesday, January 17, 2023
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Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced that the European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge’s lead adeno-associated virus (AAV) drug candidate and novel gene therapy for treating patients with Krabbe disease.

Key Points: 
  • Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced that the European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to FBX-101, Forge’s lead adeno-associated virus (AAV) drug candidate and novel gene therapy for treating patients with Krabbe disease.
  • The goal is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address and treat patients that have an unmet medical need.
  • The results demonstrated that systemically delivered FBX-101 administered after hematopoetic stem cell transplant (HSCT) is safe and well-tolerated in Krabbe patients.
  • Findings also support this novel approach for extending the delivery of gene replacement strategies to target metabolic diseases amenable to HSCT.

Forge Biologics Announces Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial for Patients with Krabbe Disease

Retrieved on: 
Tuesday, October 11, 2022
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Clinical data support preclinical observations that this gene therapy approach after HSCT may lessen many of the immune challenges previously observed with systemic AAV gene delivery and may create a safer environment for gene replacement.

Key Points: 
  • Clinical data support preclinical observations that this gene therapy approach after HSCT may lessen many of the immune challenges previously observed with systemic AAV gene delivery and may create a safer environment for gene replacement.
  • Findings also support this novel approach for extending the delivery of gene replacement strategies to target metabolic diseases amenable to HSCT.
  • Krabbe disease is characterized by mutations in the GALC gene which lead to loss of motor function.
  • RESKUE a Phase 1/2 clinical trial to investigate the safety and efficacy of FBX-101 in patients with Infantile Krabbe disease.

Forge Biologics Reports Positive Clinical Data on Brain Development and Motor Function from the RESKUE Novel Phase 1/2 Gene Therapy Trial in Patients with Krabbe Disease at the SSIEM Annual Symposium

Retrieved on: 
Tuesday, August 30, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, GALC, Muscle weakness, Locomotion, Hematopoietic stem cell transplantation, Lipid, Peripheral neuropathy, Lists of diseases, Central nervous system, Krabbe disease, Brain, Entrepreneurship, Disease, Death, Peripheral nervous system, CGMP, Cell, Multimedia, Neurodegeneration, CET, Irritability, Hearing loss, Psychosine, UCBT, Clinical trial, Environment, Society, AAV, Patient, Pharmaceutical industry, Vaccine, Krabbe, Forge, Hearth, Metabolism

The data will be presented on August 31, 2022, at the annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Freiburg, Germany.

Key Points: 
  • The data will be presented on August 31, 2022, at the annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Freiburg, Germany.
  • The first patients data from the RESKUE clinical trial demonstrate that intravenous FBX-101 following UCBT has been safe and well tolerated through Day 180.
  • Notably, the data demonstrate an absence of humoral immune response against the vector and significantly increased GALC enzyme activity.
  • Through Day 180, the patient exhibited improved motor activity and normal brain development compared to previously reported transplanted patients with Krabbe disease.

Passage Bio Doses First Patient in Global Clinical Trial for Infantile Krabbe Disease, A Rare Fatal Pediatric Condition

Retrieved on: 
Thursday, March 10, 2022
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It is gratifying to dose the first patient in our GALax-C trial, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio.

Key Points: 
  • It is gratifying to dose the first patient in our GALax-C trial, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio.
  • Krabbe disease is a rare pediatric lysosomal storage disorder caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine.
  • We also are grateful to the children, families, and clinical trial investigators who have chosen to participate in our studies.
  • The U.S. Food and Drug Administration (FDA) has granted PBKR03 Fast Track, Orphan Drug, and Rare Pediatric Disease designations.

Forge Biologics Announces Regulatory Updates from FDA and EMA, Accelerating Manufacturing and Clinical Trial Momentum

Retrieved on: 
Monday, September 20, 2021
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FBX-101 previously received Orphan Drug and Rare Pediatric Disease Designations from the FDA earlier this year.

Key Points: 
  • FBX-101 previously received Orphan Drug and Rare Pediatric Disease Designations from the FDA earlier this year.
  • Forge presented information to the FDA on its proprietary HEK293 suspension cell line, Ignition Cells, and adenovirus helper plasmid, pEMBR, and received FDA alignment that these technologies are suitable for cGMP manufacturing of clinical drug products.
  • The Hearth is a custom-designed cGMP facility dedicated to AAV vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.
  • By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

Polaryx Therapeutics Receives FDA Orphan Drug Designation for PLX-200 to Treat Krabbe Disease

Retrieved on: 
Thursday, September 2, 2021
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PARAMUS, N.J., Sept. 02, 2021 (GLOBE NEWSWIRE) -- PolaryxTherapeutics, Inc. ("Polaryx"), abiotechcompany developing small molecule therapeutics forlysosomalstorage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat Krabbe disease.

Key Points: 
  • PARAMUS, N.J., Sept. 02, 2021 (GLOBE NEWSWIRE) -- PolaryxTherapeutics, Inc. ("Polaryx"), abiotechcompany developing small molecule therapeutics forlysosomalstorage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat Krabbe disease.
  • Krabbe disease is a rare, genetic disorder caused by the deficiency of lysosomal enzyme, galactocerebrosidase (GALC).
  • Under the U.S. Orphan Drug Act, the FDA's Office of Orphan Products Development provides sponsors with special status and incentives to facilitate the drug development for rare disease affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides seven years of market exclusivity if the drug candidate receives regulatory approval together with tax credits for qualified clinical trial cost, exemptions from certain FDA application fees, and assistance in clinical trial design.
  • "Granting by the FDA of Orphan Drug Designation for PLX-200 in Krabbe disease supports the use of PLX-200 to treat key lysosomal storage disorders with unmet medical needs.