Gangliosidosis

Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium™

Retrieved on: 
Tuesday, January 23, 2024
RAINBOW, NPC, Glycogen storage disease, GM2, Patient, Research, GM1, Niemann–Pick disease, Gait, Science, Sleep disorder, Molecular genetics, Gangliosidosis, Safety, Central nervous system, Congress, Biomarker, Pharmacokinetics, Metabolism, Pharmaceutical industry

As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.

Key Points: 
  • As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - patients’ and caregivers’ assessments.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO)- baseline clinical data.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - evaluation of different assessments.

Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Patients

Retrieved on: 
Thursday, June 8, 2023
Pharmaceutical, Health, Clinical Trials, RAINBOW, Lead, NP-C, GM2, Clinical trial, Enzyme, Pharmacokinetics, GM1, Gangliosidosis, DSM-IV codes, Metabolism, Patient, Niemann–Pick disease, NP, Diagnosis, Hospital, Glycosphingolipid, Safety, Pharmaceutical industry

Azafaros B.V. today announced that the first patient has been enrolled into its Phase 2 RAINBOW study ( NCT05758922 ).

Key Points: 
  • Azafaros B.V. today announced that the first patient has been enrolled into its Phase 2 RAINBOW study ( NCT05758922 ).
  • Azafaros is developing the compound as a potentially disease-modifying treatment in severe metabolic disorders including GM1 and GM2 gangliosidoses and NP-C. Enrollment of the first patient in the study is an important milestone for Azafaros in its mission to bring new treatment options to these patients and their families.
  • The Phase 2 RAINBOW study is a randomized, double-blind, placebo-controlled, multicenter, 12-week trial assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of AZ-3102 in patients with GM2 gangliosidosis and NP-C.
  • Patients who complete the 12-week study period will be offered a double-blind extension if approved by the country’s health authorities.

Azafaros Receives FDA's IND Clearance and Fast Track Designation for Lead Asset AZ-3102

Retrieved on: 
Thursday, January 5, 2023
Managed Care, Research, Other Health, Pharmaceutical, Medical Devices, Genetics, Clinical Trials, Science, Biotechnology, Alternative Medicine, FDA, Health, Other Science, Investigational New Drug, Pharmacokinetics, Medical genetics, Child, Niemann–Pick disease, MD, NP-C, Biomarker, Gangliosidosis, Disease, GM1, Patient, Pediatrics, Safety, Mayo Clinic, FDA, IND, NP, GM2, Clinical professor, Pharmaceutical industry, Neurology

The study is being conducted in the U.S. as well as in other countries.

Key Points: 
  • The study is being conducted in the U.S. as well as in other countries.
  • The pharmacodynamics and the impact of AZ-3102 on potential disease biomarkers will also be measured during the trial.
  • AZ-3102 has unique characteristics that confer the potential to be an effective therapeutic innovation for these patients,” said Professor Patterson.
  • Stefano Portolano, MD, Chief Executive Officer of Azafaros, added: “The IND clearance and Fast Track Designation highlight AZ-3102’s potential and demonstrate the ability of the Azafaros team to achieve our ambitious goals.

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

Retrieved on: 
Tuesday, February 1, 2022
FDA, Pharmaceutical, Health, Clinical Trials, GCS, GM1, Patient, Food, Seizure, Lists of diseases, Clinical trial, ODD, Brain, Speech, Leiden University, Death, US Food Sovereignty Alliance, GM2, Disease, Frasier, AB, Dysphagia, Enzyme, Tay–Sachs disease, Courage, GBA2, Gangliosidosis, Survival, GM2 gangliosidoses, FDA, Therapy, Lipid, Neurogenetics, Ganglioside, CA, Pharmaceutical industry, Sandhoff disease, AZ-3102, Azafaros, AZ-3102, AZAFAROS

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
Hearing, Ganglioside, Niemann–Pick disease, Glycogen storage disease, Tay–Sachs disease, RXR, Drug development, Therapy, Lists of diseases, GM2, Gene, Lysosome, Trial of the century, Sandhoff disease, HEXB, Seizure, EB, Food, Disease, Neuron, LLM, Inflammation, Lysosomal storage disease, Board, Neurodegeneration, HEXA, Enzyme, Patient, CEO, Hexosaminidase, Apoptosis, TFEB, PPAR, FDA, Gangliosidosis, GM2 gangliosidoses, Nervous system, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry, Vaccine

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.

GC Pharma and Tottori University Enter Research Collaboration and License Agreement for Development of GM1 Gangliosidosis Chaperone Therapy

Retrieved on: 
Monday, August 2, 2021
Biotechnology, Hospitals, Pharmaceutical, Health, Lipid storage disorders, Rare diseases, GM1, Gangliosidosis, Tottori, Pharma, Branches of biology, Health, GM1 gangliosidosis, GC Pharma, Tottori University, GM1 GANGLIOSIDOSIS, GC PHARMA, TOTTORI UNIVERSITY

GC Pharma (006280.KS) and Tottori University today announced a worldwide licensing and collaboration agreement for the orally administrable chaperone drug of GM1 gangliosidosis (GM1).

Key Points: 
  • GC Pharma (006280.KS) and Tottori University today announced a worldwide licensing and collaboration agreement for the orally administrable chaperone drug of GM1 gangliosidosis (GM1).
  • Under the terms of the agreement, GC Pharma has obtained the exclusive right to develop and commercialize worldwide.
  • Tottori University is dedicated to contribute the international research collaboration with GC Pharma to develop and deliver an innovative chaperone therapy for patients with this devastating neurodegenerative disease.
  • Katsumi Higaki, Ph.D., head of GM1 research at Tottori University, has been working on the chaperone therapy related to orphan disorders for more than 20 years.

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

Retrieved on: 
Thursday, July 8, 2021
Stem cells, Biotechnology, Pharmaceutical, Health, FDA, Genetics, Clinical trials, Other Health, Lipid storage disorders, Rare diseases, Branches of biology, Life sciences, Biology, Glycolipids, Gangliosidosis, GM1 gangliosidoses, GLB1, Ganglioside, GM1, Fast track

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).

Azafaros Appoints Stefano Portolano, M.D., as Chief Executive Officer

Retrieved on: 
Wednesday, June 30, 2021
Health, Genetics, Other Science, Research, Science, Pharmaceutical, Biotechnology, Lipid storage disorders, Rare diseases, Glycolipids, Gangliosidosis, GM2 gangliosidoses, Branches of biology, GM2, Lluís Companys, GM1, Health, Spanish politicians, Dr. Stefano Portolano, Azafaros, DR. STEFANO PORTOLANO, AZAFAROS

Azafaros B.V. today announced that Stefano Portolano, M.D., has joined Azafaros as Chief Executive Officer (CEO) and has concurrently been appointed to the Board of Directors of the company.

Key Points: 
  • Azafaros B.V. today announced that Stefano Portolano, M.D., has joined Azafaros as Chief Executive Officer (CEO) and has concurrently been appointed to the Board of Directors of the company.
  • We are very happy to welcome Stefano to Azafaros as the company progresses further into clinical development.
  • "Stefano joins an outstanding team that is rapidly advancing lead candidate AZ-3102, the companys proprietary orally-available azasugar molecule for the treatment of GM1 and GM2 gangliosidoses.
  • Since the companys inception in 2018, Azafaros has made remarkable progress by building a strong and committed organization that has reached the clinic in less than three years, said Stefano Portolano, M.D., Azafaros' Chief Executive Officer.

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

Retrieved on: 
Wednesday, June 9, 2021
Other Health, Health, Genetics, Stem cells, Clinical trials, Branches of biology, Lipid storage disorders, Rare diseases, Biology, Health sciences, Gangliosidosis, GM1 gangliosidoses, GLB1, Ganglioside, GM1, Neurodegeneration, Gene therapy

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).

Sanofi provides update on venglustat clinical program

Retrieved on: 
Tuesday, June 1, 2021
Rare diseases, Medical specialties, Lipid storage disorders, Clinical medicine, Kidney diseases, Health, Autosomal dominant polycystic kidney disease, Channelopathies, Polycystic kidney disease, Gangliosidosis, GM2 gangliosidoses, Sanofi

PARIS JUNE 1, 2021 A pivotal Phase 2/3 study of venglustat in autosomal dominant polycystic kidney disease (ADPKD) did not meet futility criteria, and the company has halted the clinical program in ADPKD.

Key Points: 
  • PARIS JUNE 1, 2021 A pivotal Phase 2/3 study of venglustat in autosomal dominant polycystic kidney disease (ADPKD) did not meet futility criteria, and the company has halted the clinical program in ADPKD.
  • Sanofi has both completed and active studies evaluating venglustat in Gaucher diseasetype 3, Fabry disease and GM2 Gangliosidosis.
  • Venglustat is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
  • With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.