Psychosine

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
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Congress, Gene, EMA, PRIME, European Medicines Agency, Quality, Psychosine, AAV, Society, HSCT, Krabbe disease, Senior, ESGCT, Regulatory affairs, Patient, GALC, Medicine, Report of the 1st Annual Symposium on Relaxed Improvisation, Cell, Pharmaceutical industry, Forge, Krabbe, Metabolism

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
Children, Biotechnology, Manufacturing, Health, Pharmaceutical, Research, Other Manufacturing, Genetics, Consumer, Science, Clinical Trials, CA, HSCT, Congress, ESGCT, CSF, Hope, Diagnosis, Locomotion, Krabbe disease, Lists of diseases, Peripheral nervous system, Cell, CGMP, Patient, Gene, Mesa, Sphingolipid, Dementia, Peripheral neuropathy, Multimedia, White matter, Clinical trial, European Society of Gene and Cell Therapy, Nerve, Psychosine, PNS, Hearing loss, AAV, Cerebrospinal fluid, Degenerative disease, Irritability, GALC, CEO, Hematopoietic stem cell transplantation, Incidence, Death, Safety, Natural history, CNS, M.D, Neurodegeneration, Brain, Plasma, ARM, Infant, Central nervous system, Nervous system, Pharmaceutical industry, Vaccine, Hearth, Krabbe, Forge

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
Peripheral nervous system, University, Risk, Cisterna magna, U.S. Securities and Exchange Commission, Survival, Glycogen storage disease, Private Securities Litigation Reform Act, Brain, Central nervous system, AAV, Disease, GM1, Safety, Biomarker, Peripheral neuropathy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, Failure, CNS, Seizure, Severe cognitive impairment, GLOBE, Patient, European Commission, Death, SEC, Clinical trial, Apnea, IND, GALC, Deafness, Galactosylceramidase, Security (finance), ICM, Central nervous system disease, Doctor of Philosophy, Â, Life expectancy, Genetics, Nasdaq, Psychosine, PASG, Department, Profile, Cohort, Royal commission, Krabbe disease, Genetic testing, Population, Food, Pharmaceutical industry, Vaccine, Krabbe

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.

Gain Therapeutics Progresses Krabbe Disease Program and Provides Scientific Update

Retrieved on: 
Tuesday, December 7, 2021
European Union, GBA, Forward-looking statement, GLOBE, Technology, Degenerative disease, Lists of diseases, Multiple sclerosis, Drug discovery, Krabbe disease, Neurodegeneration, Psychosine, Brain, Patient, Therapy, Glycogen storage disease, Champaign County, Illinois, College, University, The Michael J. Fox Foundation, Program, Galactosylceramidase, Nasdaq, MJFF, Pharmaceutical industry, Medical device

BETHESDA, Md., Dec. 07, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (Gain, or the Company), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced a scientific update on the companys Krabbe disease program.

Key Points: 
  • BETHESDA, Md., Dec. 07, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (Gain, or the Company), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced a scientific update on the companys Krabbe disease program.
  • Gain is developing allosteric regulators to stabilize the galactosylceramidase (GALC) enzyme and reduce psychosine disease causing toxic substrate, potentially providing the first treatment option to patients with this devastating disease.
  • These data provide additional validation of our approach in treating lysosomal storage diseases, said Eric Richman, Chief Executive Officer of Gain.
  • Krabbe disease, also known as globoid cell leukodystrophy, is a genetic disease that affects the central and peripheral nervous systems.

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

Retrieved on: 
Monday, September 20, 2021
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Ignition, COMP, Muscle weakness, Hearing loss, The Hearth, Peripheral neuropathy, Therapy, European Medicines Agency, CGMP, FDA, Psychosine, Multimedia, Patient, Regulatory affairs, Hematopoietic stem cell transplantation, Irritability, Quality, Death, GALC, European Union, Krabbe disease, Disease, Technology, HEK293, Peripheral nervous system, Union, Research, Brain, Cell, AAV, Lipid, EMA, Galactosylceramidase, Pharmaceutical industry, Vaccine, Krabbe Disease, FBX-101, Orphan Designation, Forge Biologics, KRABBE DISEASE, FBX-101, ORPHAN DESIGNATION, FORGE BIOLOGICS

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
Glycogen storage disease, Drug development, Phenotype, Therapy, Lists of diseases, Genetic disorder, Life, Gene, Trial of the century, Krabbe disease, Metabolic disorder, GLOBE, GALC, EB, Food, Disease, Myelin, Death, KD, Lipid storage disorder, LLM, Inflammation, Degenerative disease, Board, Enzyme, CEO, Psychosine, Apoptosis, TFEB, FDA, Neurodegeneration, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry

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.

Passage Bio Announces Presentation of Data from Animal Models of Krabbe Disease at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting

Retrieved on: 
Tuesday, May 12, 2020
Branches of biology, Neurological disorders, Rare diseases, Enzymes, Lipid storage disorders, Krabbe disease, Leukodystrophies, Galactosylceramidase, Psychosine, Krabbe, Lysosomal storage disease, Gene therapy

This data was presented today in an virtual oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting by Juliette Hordeaux, D.V.M., Ph.D., senior director of translational research at the University of Pennsylvanias Gene Therapy Program.

Key Points: 
  • This data was presented today in an virtual oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting by Juliette Hordeaux, D.V.M., Ph.D., senior director of translational research at the University of Pennsylvanias Gene Therapy Program.
  • We are extremely excited about this data for our Krabbe program.
  • Krabbe disease is a rare and often life-threatening lysosomal storage disease caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine.
  • PBKR03 thus has the potential to treat both the central nervous system and peripheral nerve manifestations observed in Krabbe disease patients.