Krabbe disease

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
Diamond, Community, Diagnosis, Civil service commission, Prevalence, Disease, Patient, Committee, EMA, IRIS, Krabbe disease, GALC, Agency, European, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

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.

Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation

Retrieved on: 
Tuesday, March 19, 2024
Biotechnology, Pharmaceutical, Genetics, Health, Infant, AAV, MAA, Disease, Patient, FBX, Innovation, HSCT, EMA, Krabbe disease, GMP, European Medicines Agency, MHRA, Education minister, Clinical trial, Safety, FDA, Neurodegenerative disease, Hematopoietic stem cell transplantation, Medication, Pharmaceutical industry

FBX-101 was created for the treatment of patients with Krabbe disease, a rare neurodegenerative disease that is usually fatal in untreated patients by age two.

Key Points: 
  • FBX-101 was created for the treatment of patients with Krabbe disease, a rare neurodegenerative disease that is usually fatal in untreated patients by age two.
  • FBX-101 has been granted Orphan Drug Designation and Priority Medicines (PRIME) designation by the European Medicines Agency (EMA), allowing Forge to advance and expedite the development of FBX-101 as the leading worldwide gene therapy for patients with Krabbe disease.
  • In the US, the FDA has granted FBX-101 Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation.
  • Through this designation Forge has expanded global regulatory experience to include interactions with the MHRA in the UK.

Passage Bio Reports Fourth Quarter and Full-Year 2023 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Monday, March 4, 2024
GRN, Granulin, Interim management, Cohort, Senior, ALS, Alzheimer's disease, GM1, PASG, Research and development, Frontotemporal dementia, FTD, Interim, Safety, Immunosuppression, Administration, Patient, Clinical trial, CSF, Krabbe disease, Pharmaceutical industry

"In 2023, we achieved a significant milestone by announcing encouraging data from three patients in Cohort 1 of our FTD-GRN program.

Key Points: 
  • "In 2023, we achieved a significant milestone by announcing encouraging data from three patients in Cohort 1 of our FTD-GRN program.
  • We eagerly anticipate a catalyst-rich 2024, supported by our robust balance sheet, bringing us closer to improving patient outcomes across neurodegenerative diseases."
  • Additionally, Dose 1 of PBFT02 continued to be generally well-tolerated in patients who received an enhanced steroid regimen for immunosuppression.
  • Cash Position: Cash, cash equivalents and marketable securities were $114.3 million as of December 31, 2023, as compared to $189.6 million as of December 31, 2022.

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.

Passage Bio Announces Promising Initial Data From Phase 1/2 Clinical Trial of PBFT02 in FTD-GRN and Updated Strategic Priorities

Retrieved on: 
Wednesday, December 20, 2023
CSF, Immunosuppression, Partnership, ALS, GM1, Aes, Krabbe disease, FTD, Loss, ICM, PASG, Clinical trial, Plasma, Inflammation, Methylprednisolone, AAV, Toxicity, GRN, Patient, Frontotemporal dementia, Safety, Cell biology, Hepatotoxicity, MLD, DRG, Pharmaceutical industry

Additionally, the company shared updated strategic priorities aimed at further optimizing its portfolio for the treatment of neurodegenerative conditions.

Key Points: 
  • Additionally, the company shared updated strategic priorities aimed at further optimizing its portfolio for the treatment of neurodegenerative conditions.
  • "We are proud to announce initial clinical data from our upliFT-D clinical trial, which showcases the ability of PBFT02 to elevate CSF progranulin to supraphysiologic levels at the lowest tested dose, Dose 1, up to six months post-treatment.
  • The upliFT-D clinical trial evaluates PBFT02 as a single dose delivered via intra-cisterna magna (ICM) injection.
  • These strategic priorities and clinical milestones underscore Passage Bio's dedication to advancing cutting-edge, one-time genetic medicines and protecting patients and families against loss in neurodegenerative conditions.

Forge Biologics Receives Qualified Person (QP) Declaration to Manufacture AAV Gene Therapies to Support European Clinical Programs

Retrieved on: 
Monday, April 24, 2023
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Senior, QMS, Krabbe disease, Quality management, AAV, Quality, QP, CDMO, Patient, Regulatory affairs, Medical device, EU, Forge, European

Having fulfilled Europe’s GMP requirements, Forge has reached another key regulatory milestone, expanding its CDMO services to support clients in the United States and Europe.

Key Points: 
  • Having fulfilled Europe’s GMP requirements, Forge has reached another key regulatory milestone, expanding its CDMO services to support clients in the United States and Europe.
  • Forge clients can rely on the Company’s QP audit as one less hurdle they need to overcome when their AAV gene therapy products are being manufactured for clinical trials and on the path to commercialization in European countries.
  • The QP declaration was received in support of Forge’s internal FBX-101 clinical gene therapy program for the treatment of patients with Krabbe disease.
  • The QP declaration adds to Forge's capabilities and makes the Company among one of the few U.S. AAV CDMO's able to support clients intending to export gene therapy products for clinical programs in Europe.

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, a Leading Manufacturer of Genetic Medicines, to Present at 41st Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Wednesday, January 4, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Bioreactor, Patient, Conference, AAV, Krabbe disease, Multimedia, Safety, Entrepreneurship, Q&A, Pharmaceutical industry

Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced today that Timothy J. Miller, Ph.D., CEO, President, and Co-Founder, will present at the 41st Annual J.P. Morgan Healthcare Conference on Tuesday, January 10, 2023, at 2:00 p.m. Pacific Time, followed by a live Q&A session.

Key Points: 
  • Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced today that Timothy J. Miller, Ph.D., CEO, President, and Co-Founder, will present at the 41st Annual J.P. Morgan Healthcare Conference on Tuesday, January 10, 2023, at 2:00 p.m. Pacific Time, followed by a live Q&A session.
  • This press release features multimedia.
  • View the full release here: https://www.businesswire.com/news/home/20230104005389/en/
    “As a leading manufacturer of genetic medicines, we are delighted to be presenting in San Francisco at the J.P. Morgan Healthcare Conference this year and look forward to highlighting our 2022 milestones and 2023 growth plans,” said Dr. Miller.
  • “It is our privilege to work with over 30 gene therapy clients and also advance our own AAV clinical stage program, all promising to bring hope to patients around the world.”
    Expanded our 200,000 sq.

Gain Therapeutics, Inc. Reports Third Quarter 2022 Financial Results and Business Update

Retrieved on: 
Thursday, November 10, 2022
Molecule, GLOBE, IND, Security, Neurodegeneration, CBE, Shaare Zedek Medical Center, Conduritol, Nasdaq, Gaucher's disease, Movement disorders, Mouse, Lysosome, Drug discovery, Krabbe disease, Neuron, Therapy, IP, Cash, Potency (pharmacology), Safety, Pharmacokinetics, Company, Movement, GBA1, Clinical trial, Research, Pathology, Poster, Dopaminergic, International Congress on Tuberculosis, Patient, GAAP, Animal, Industry, IPSC, Cryptocurrency, Pharmaceutical industry, Lease

BETHESDA, Md., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company transforming drug discovery with its proprietary computational discovery platform identifying novel allosteric binding sites and creating small molecule treatments, today announced its financial results for the third quarter ended September 30, 2022, and highlighted recent corporate progress.

Key Points: 
  • The latest preclinical data provide further validation of the potential utility of our approach and mechanism of action of GT-02287.
  • If these results are supported in our upcoming clinical trials, we believe that GT-02287 could become a potential first-in-class disease-modifying therapy for Parkinsons disease.
  • Presented new preclinical data for GT-02287, Gains lead compound for Parkinsons Disease at the International Congress of Parkinsons Disease and Movement Disorders 2022 Annual Meeting.
  • Cash, cash equivalents and marketable securities were $25.7 million as of September 30, 2022 compared to $36.8 million at December 31, 2021.