Metachromatic leukodystrophy

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

Retrieved on: 
Monday, March 18, 2024
Encephalitis, Locomotion, Brain, B cell, Bone marrow, Cell, Dietary supplement, Nervous system, Risk, Patient, Public, Urinary tract infection, Animal, Metachromasia, DSM-IV codes, Peripheral nervous system, Viral, Human services, Hope, Death, Quality of life, Atidarsagene autotemcel, Rash, Therapy, Metachromatic leukodystrophy, Natural history, Fever, MLD, Heated tobacco product, Safety, Disability studies, Cosmetics, Health, Vaccine, Food, Swelling, FDA, Liver, Multimedia, ARSA, Pharmaceutical industry

Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.

Key Points: 
  • Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system.
  • The stem cells are collected from the patient and modified by adding a functional copy of the ARSA gene.
  • In children with MLD, treatment with Lenmeldy significantly reduced the risk of severe motor impairment or death compared with untreated children.
  • Eighty five percent of the children treated had normal language and performance IQ scores, which has not been reported in untreated children.

Homology Medicines Announces First Data from IND-Enabling Studies with GTx-mAb Candidate HMI-104 for PNH, Which Demonstrated Sustained Expression of Functional C5mAb Levels, at the ASGCT Annual Meeting

Retrieved on: 
Tuesday, May 16, 2023
Inhibitor, Neutralizing antibody, Hemolysis, Gene, NOD, MLD, PAH, Serum, SCID, Phenylketonuria, Trabecular meshwork, Gene editing, Gene expression, FIX, Publication, Metachromatic leukodystrophy, Messenger, PKU, Future, Administration, Immunosuppression, Genome, Intravitreal administration, Homologous recombination, Treatment, Vivisection, Immune system, PNH, Cell, Retinal pigment epithelium, NHP, Poster, RPE, Detection, Identification, Virus, ARSA, Society, Vaccine, Pharmaceutical industry, Truffle, Mouse, IND, C5

BEDFORD, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today six presentations supporting its gene editing, gene therapy and GTx-mAb programs at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, including the first data from IND-enabling studies with HMI-104, the anti-C5 GTx-mAb development candidate for paroxysmal nocturnal hemoglobinuria (PNH). These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.

Key Points: 
  • These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.
  • Additionally, Homology highlighted methods to identify genomic sites with improved homologous-recombination (HR)-based gene editing integration, which could be used to enhance and streamline development of future product candidates.
  • These data support the immunosuppression regimen used in Homology’s ongoing gene editing and gene therapy clinical trials.
  • In the poster, “Gene Therapy Candidate for Metachromatic Leukodystrophy (MLD): Optimization of HMI-202 Leading to HMI-204 Nomination,” Homology highlighted preclinical data on its optimized HMI-204 gene therapy candidate for MLD.

Orchard Therapeutics Highlights Recent Progress Across HSC Gene Therapy Portfolio and Outlines Key 2023 Milestones

Retrieved on: 
Monday, January 9, 2023
IND, Disease, Agency, Therapy, Natural history, HSC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Orchard, MLD, FDA, HAE, Type, Australia New Zealand Therapeutic Products Authority, Burn, Partnership, NOD2, U.S. FDA, Growth, Food, NBS, BLA, Patient, File, Investigational New Drug, Workforce, POC, Hereditary angioedema, Society, Investment, MAA, EU, Pharmaceutical industry, Medical device, Metachromatic leukodystrophy, Lancet

To date, over 170 patients have been treated with Orchard’s current and former HSC gene therapy programs across seven different diseases, reflecting the broad clinical applicability of the company’s approach.

Key Points: 
  • To date, over 170 patients have been treated with Orchard’s current and former HSC gene therapy programs across seven different diseases, reflecting the broad clinical applicability of the company’s approach.
  • “Orchard’s accomplishments in 2022 showcase the depth and strength of our neurometabolic portfolio,” said Bobby Gaspar, M.D., Ph.D., chief executive officer.
  • “We right sized our workforce and narrowed the focus of our HSC gene therapy portfolio to concentrate on severe neurometabolic diseases and research programs where alternative treatment options are limited or do not exist.
  • Refined the company’s HSC gene therapy portfolio and workforce to focus on severe neurometabolic diseases and pre-clinical research programs.

Orchard Therapeutics Announces Swissmedic Validation of the Marketing Authorization Application for Libmeldy (atidarsagene autotemcel)

Retrieved on: 
Thursday, December 1, 2022
United Kingdom, Literature, Australia New Zealand Therapeutic Products Authority, Therapy, Brain, Social media, Agency, Medication package insert, Risk, Patient, GSK, HSC, Late, Union, Infant, Dementia, ARSA, Royal commission, DOI, SAN, Systematic review, Nervous system, Seizure, Spasticity, MAA, Bone marrow, EMA, Partnership, Mortality, European Commission, Disease, Gallbladder, Liver, Safety, Multiple birth, Spleen, Mutation, Gene, MLD, File, Pharmaceutical industry, Child, Metachromatic leukodystrophy, EU, Orchard

The Swiss filing was based on the European Union (EU) MAA for Libmeldy, which was approved by the European Commission in December 2020.

Key Points: 
  • The Swiss filing was based on the European Union (EU) MAA for Libmeldy, which was approved by the European Commission in December 2020.
  • Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures.
  • For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.
  • At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases.

Orchard Announces Multiple Presentations at 2022 ESGCT Annual Congress Showing the Potential of HSC Gene Therapy in Several Therapeutic Areas

Retrieved on: 
Monday, October 10, 2022
LinkedIn, Union, GSK, MLD, EMA, Hematology, Program, Securities Act of 1933, Bone marrow, European Society of Gene and Cell Therapy, Stem cell, 2021 Essex County Council election, Medication package insert, European Commission, ARSA, Poster, HSC, Therapy, Cell, Twitter, Dementia, Private Securities Litigation Reform Act, BST, GLOBE, ESGCT, U.S. Securities and Exchange Commission, Hospital, Review, Gene, Research, Risk, Disease, SEC, Royal commission, San Raffaele Hospital, Patient, Partnership, Leukemia, Social media, Safety, Infant, File, Product, Pharmaceutical industry, Vaccine, Metachromatic leukodystrophy, Orchard, SAN

BOSTON and LONDON, Oct. 10, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced multiple presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), taking place October 11-14, 2022, in Edinburgh.

Key Points: 
  • BOSTON and LONDON, Oct. 10, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced multiple presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), taking place October 11-14, 2022, in Edinburgh.
  • Together with our clinical and research partners, were proud of our presence at the upcoming ESGCT Annual Congress, said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics.
  • Alongside our extensive body of clinical data and real-world evidence in MLD, our programs are showing the transformative potential of HSC gene therapy across many different genetic diseases with high unmet medical need.
  • The presentations are listed below, and the full program is available online on the ESGCT website .

Orchard Therapeutics Announces Multiple Presentations at 2022 SSIEM Annual Symposium Highlighting Neurometabolic Disease Portfolio

Retrieved on: 
Monday, August 29, 2022
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BOSTON and LONDON, Aug. 29, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced seven presentations from across its neurometabolic portfolio will be featured at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, taking place from August 30 to September 2, 2022, in Freiburg, Germany.

Key Points: 
  • BOSTON and LONDON, Aug. 29, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced seven presentations from across its neurometabolic portfolio will be featured at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, taking place from August 30 to September 2, 2022, in Freiburg, Germany.
  • The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies.
  • During the clinical studies of Libmeldy, the safety profiles of these interventions were consistent with their known safety and tolerability.
  • At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases.

Passage Bio Receives FDA Clearance of IND Application for PBML04 for Treatment of Metachromatic Leukodystrophy

Retrieved on: 
Wednesday, June 8, 2022
Frontotemporal dementia, Metachromatic leukodystrophy, Lysosomal storage disease, Adult, Program, Food, Central nervous system, GM1, Partnership, Security (finance), Failure, U.S. Securities and Exchange Commission, Nasdaq, IND, AAV, University, Risk, FDA, GLOBE, Patient, Private Securities Litigation Reform Act, Glycogen storage disease, Incidence, SEC, Clinical trial, Peripheral nervous system, Rigidity, CMC, ARSA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PASG, MLD, Krabbe disease, CNS, ICM, Pharmaceutical industry

PHILADELPHIA, June 08, 2022 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for PBML04, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of Metachromatic Leukodystrophy (MLD). MLD is a rare, fatal, pediatric, lysosomal storage disease that currently has limited available treatment options.

Key Points: 
  • MLD is a rare, fatal, pediatric, lysosomal storage disease that currently has limited available treatment options.
  • We are thrilled to have received IND clearance for our program in metachromatic leukodystrophy, marking our fourth IND clearance as a company and our third pediatric lysosomal storage disorder program to reach clinical development, said Edgar (Chip) Cale, interim chief executive officer of Passage Bio.
  • MLD, GM1 gangliosidosis and Krabbe disease are similar diseases each caused by a single gene mutation leading to deficiency of a critical enzyme.
  • We are excited by the promise of PBML04 to offer a potentially transformative treatment for this devastating disease.

Orchard Therapeutics Announces Agreement Enabling Access and Reimbursement for Libmeldy for All Eligible MLD Patients in Germany

Retrieved on: 
Monday, May 2, 2022
Mutation, Medication package insert, MLD, Safety, Seizure, Caregiver, SEC, Risk, Royal commission, Brain, Mortality, Systematic review, Twitter, Securities Act of 1933, European Commission, Patient, Partnership, Neurodegeneration, Program, Spasticity, LinkedIn, Disease, Liver, GSK, Therapy, SAN, Lists of diseases, Private Securities Litigation Reform Act, Literature, EMA, Union, U.S. Securities and Exchange Commission, Bone marrow, Gallbladder, Gene, Product, Federal Joint Committee (Germany), ARSA, COVID-19, File, GLOBE, Nervous system, Dementia, HSC, Multiple birth, Spleen, Review, Infant, DOI, Late, Sibling, Social media, Health insurance, Pharmaceutical industry, Orchard, Child, Metachromatic leukodystrophy

Our agreement with GKV-SV for Libmeldy marks a significant milestone for children with MLD, their caregivers and the entire community, said Braden Parker, chief commercial officer.

Key Points: 
  • Our agreement with GKV-SV for Libmeldy marks a significant milestone for children with MLD, their caregivers and the entire community, said Braden Parker, chief commercial officer.
  • We are encouraged by the continued progress we are making across the region to help eligible patients gain access to this therapy.
  • As previously disclosed, one German patient has already received Libmeldy at Tbingen University Hospital through an interim reimbursed access arrangement.
  • Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

Orchard Therapeutics Announces Reimbursement Agreement Making Libmeldy Available for All Eligible MLD Patients in Italy

Retrieved on: 
Monday, April 11, 2022
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The news marks the second reimbursement agreement to cover all eligible MLD patients that Orchard has reached with a European market this year.

Key Points: 
  • The news marks the second reimbursement agreement to cover all eligible MLD patients that Orchard has reached with a European market this year.
  • Earlier this month, an MLD patient from the Middle East also received Libmeldy through reimbursed international treatment abroad at Ospedale San Raffaele, in Milan, Italy.
  • Patients with MLD gradually lose the ability to move, talk, swallow, eat and see.
  • Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

Orchard Therapeutics Announces Multiple Presentations at WORLDSymposium™ 2022

Retrieved on: 
Monday, February 7, 2022
Private Securities Litigation Reform Act, Securities Act of 1933, GLOBE, Forward-looking statement, GSK, File, U.S. Securities and Exchange Commission, MLD, Social media, ARSA, Gene, Medication package insert, COVID-19, Twitter, HSC, Hurler syndrome, Quality of life, Marketing, Union, Dementia, Risk, Patient, Infant, Safety, Review, SEC, European Commission, EMA, Goal, Failure, Product, Disease, PST, LinkedIn, Partnership, Bone marrow, Royal commission, Fashion, Pharmaceutical industry, Orchard, Metachromatic leukodystrophy, EU

BOSTONandLONDON, Feb. 07, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today outlined five upcoming presentations from across its neurodegenerative portfolio to be featured at the 18th Annual WORLDSymposium being held on February 7-11, 2022.

Key Points: 
  • BOSTONandLONDON, Feb. 07, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today outlined five upcoming presentations from across its neurodegenerative portfolio to be featured at the 18th Annual WORLDSymposium being held on February 7-11, 2022.
  • During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.
  • For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.
  • At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases.