Ganglioside

Denali Therapeutics Announces New Interim Data from DNL310 Phase 1/2 Study for MPS II and DNL126 Preclinical Data for MPS IIIA at WORLDSymposium™

Retrieved on: 
Wednesday, February 22, 2023

DNL310 is an investigational brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II.

Key Points: 
  • DNL310 is an investigational brain-penetrant enzyme replacement therapy designed to address the behavioral, cognitive, and physical manifestations of MPS II.
  • The interim Phase 1/2 data are being presented at the 19th Annual WORLDSymposium™ in Orlando, Florida, February 22-26, 2023.
  • “As the study progresses and enrollment continues for the global Phase 2/3 COMPASS study, I am excited to learn more about the potential of DNL310 to offer meaningful benefit for the entire MPS II patient population."
  • The safety profile of DNL310 remains consistent with standard of care, now with data up to two years of treatment with DNL310.

Lysogene Reports Additional Positive Biomarker Data With LYS-SAF302 and Favorable Safety Data With LYS-GM101 Presented at the WORLDSymposium™ 2022

Retrieved on: 
Friday, February 11, 2022

Data were presented in oral presentations at the WORLDSymposium 2022, on February 10th, 2022.

Key Points: 
  • Data were presented in oral presentations at the WORLDSymposium 2022, on February 10th, 2022.
  • Following treatment with LYS-SAF302 in AAVance, mean serum NF-L concentrations increased, reaching about 2-fold above baseline at 6 months.
  • We look forward to confirming these results in additional patients and timepoints, said Dr. Ralph Laufer, Chief Scientific Officer of Lysogene.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).

Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis

Retrieved on: 
Tuesday, February 1, 2022

Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel oral small molecule, in GM2 gangliosidosis including both Sandhoff and Tay-Sachs diseases.

Key Points: 
  • Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel oral small molecule, in GM2 gangliosidosis including both Sandhoff and Tay-Sachs diseases.
  • The ODD for GM2 gangliosidosis has been granted based on efficacy demonstrated in a Sandhoff mouse model, including a clear effect on animal survival.
  • Orphan Drug Designation by the US FDA provides drug developers with special status and incentives to facilitate the development of therapeutics for rare diseases affecting fewer than 200,000 people in the US.
  • Azafaros completed a first-in-human clinical trial with AZ-3102 in healthy volunteers in 2021 and received Orphan Drug Designation in GM2 Gangliosidosis from the FDA in February 2022.

Polaryx Therapeutics Announces FDA Grants Orphan Drug Designation for PLX-200 in GM2 Gangliosidoses

Retrieved on: 
Monday, August 30, 2021

PARAMUS, N.J., Aug. 30, 2021 /PRNewswire/ --Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.

Key Points: 
  • PARAMUS, N.J., Aug. 30, 2021 /PRNewswire/ --Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.
  • "We are very pleased to be granted Orphan Drug Designation for PLX-200 from the FDA for the treatment of GM2 gangliosidoses.
  • Furthermore, this designation validates the rationale for clinical use of PLX-200 in GM2 gangliosidoses patients.
  • Polaryx Therapeutics, Inc. is developing drug candidates for lysosomal storage disorders, for which there are currently no safe and patient-friendly treatment options available.

Lysogene Announces FDA Fast Track Designation for LYS-GM101 Gene Therapy for the Treatment of GM1 Gangliosidosis

Retrieved on: 
Thursday, July 8, 2021

GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons resulting in progressive neurodegeneration.

Key Points: 
  • GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons resulting in progressive neurodegeneration.
  • A product that receives Fast Track designation is eligible for more frequent interactions with FDA, potential eligibility for accelerated approval, priority review, and rolling Biologics License Application (BLA) review.
  • This Fast Track designation demonstrates the regulators sustained interest in Lysogenes cutting edge gene therapy program.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).

Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis

Retrieved on: 
Wednesday, June 9, 2021

GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons resulting in progressive neurodegeneration.

Key Points: 
  • GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons resulting in progressive neurodegeneration.
  • "Dosing the first patient in this clinical study represents a significant milestone for Lysogene, as it marks the second gene therapy program from our portfolio to enter the clinic.
  • We are always looking for opportunities to accelerate the development of new gene therapy treatments to improve patients lives and are excited to bring this investigational therapy to patients with GM1 gangliosidosis.
  • Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS).

Passage Bio Announces Launch of Natural History Study to Evaluate Patients with GM1 Gangliosidosis

Retrieved on: 
Tuesday, August 13, 2019

The study is being conducted by the Orphan Disease Center (ODC) in the Perelman School of Medicine at the University of Pennsylvania.

Key Points: 
  • The study is being conducted by the Orphan Disease Center (ODC) in the Perelman School of Medicine at the University of Pennsylvania.
  • There is a limited understanding of GM1 gangliosidosis, particularly in pediatric patients, and thus its important that we sponsor this Natural History Study in order to gather information on the symptoms and progression of this incredibly devastating rare disease, said Stephen Squinto, co-founder and interim chief executive officer at Passage Bio.
  • Data from this foundational study will be used to help rationally design our clinical development programs with the goal of fully addressing the unique and clinically meaningful challenges of GM1.
  • GM1 gangliosidosis (GM1) is an autosomal recessive genetic disorder, caused by an inactivating mutation of the lysosomal enzyme -galactosidase (GLB1), which is required for the degradation of GM1 ganglioside and keratan sulfate.

Tay-Sachs Disease Forecast In 19 Major Markets 2018-2028: Risk Factors, Disease Diagnosis and Prognosis Along with Specific Variations by Geography and Ethnicity

Retrieved on: 
Friday, August 10, 2018

The "Tay-Sachs Disease Forecast In 19 Major Markets 2018-2028" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Tay-Sachs Disease Forecast In 19 Major Markets 2018-2028" report has been added to ResearchAndMarkets.com's offering.
  • Hexosaminidase A deficiency (HEX A deficiency) results in a group of neurodegenerative disorders caused by the dysfunctional activity of the specific glycosphingolipid GM2 ganglioside.
  • Along with the current prevalence, the report also contains a disease overview of the risk factors, disease diagnosis and prognosis along with specific variations by geography and ethnicity.
  • These sub-populations within the main disease are also included at a country level across the 10-year forecast snapshot.