Glucocerebrosidase

Vanqua Bio Announces First Patient Dosed in Phase 1 Clinical Trial Evaluating VQ-101, its Small Molecule GCase Activator for GBA-Parkinson’s Disease and Related Disorders

Retrieved on: 
Tuesday, April 9, 2024
Cell, Recycling, PD, Patient, Glucocerebrosidase, Doctor of Philosophy, Alpha-synuclein, Trial of the century, Protein, Neuron, Safety, Pharmacokinetics, Lysosome

CHICAGO, April 09, 2024 (GLOBE NEWSWIRE) -- Vanqua Bio, a clinical-stage biopharmaceutical company dedicated to discovering and developing next-generation medicines for the treatment of neurodegenerative diseases, announced that the first patient has been dosed in a first-in-human Phase 1 clinical study evaluating VQ-101 in healthy individuals and patients with various forms of Parkinson’s disease (PD). VQ-101 is an orally administered brain-penetrant small molecule allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase).

Key Points: 
  • VQ-101 is an orally administered brain-penetrant small molecule allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase).
  • “VQ-101 demonstrated promising efficacy, safety, pharmacokinetics, and target engagement in preclinical studies.
  • Mutations in GBA1 are the most common genetic risk factor for PD, representing approximately 10% of patients with PD worldwide.
  • In developing VQ-101, Vanqua is adopting a precision-medicine approach that focuses initially on patients with GBA-PD, the largest genetically defined segment of PD.

Vanqua Bio to Present at the Stifel 2024 CNS Days Conference

Retrieved on: 
Wednesday, March 13, 2024
Conference, Glucocerebrosidase

CHICAGO, March 13, 2024 (GLOBE NEWSWIRE) -- Vanqua Bio, a biopharmaceutical company dedicated to discovering and developing next-generation medicines for the treatment of neurodegenerative diseases, announced today that the company will participate in the Stifel 2024 CNS Days Conference, which is being held virtually March 19-20.

Key Points: 
  • CHICAGO, March 13, 2024 (GLOBE NEWSWIRE) -- Vanqua Bio, a biopharmaceutical company dedicated to discovering and developing next-generation medicines for the treatment of neurodegenerative diseases, announced today that the company will participate in the Stifel 2024 CNS Days Conference, which is being held virtually March 19-20.
  • The company will present data on its lead program targeting glucocerebrosidase (GCase) as a potential treatment for Parkinson’s disease.

Gain Therapeutics Announces the Initiation of the Multiple Ascending Dose (MAD) Part of the Phase 1 Clinical Trial of GT-02287, a Novel GCase-targeting Small Molecule Therapy for GBA1 Parkinson’s Disease

Retrieved on: 
Tuesday, February 27, 2024
SAD, Glucocerebrosidase, Safety, Single, Therapy, AFL, Clinical trial, Plasma, GBA1, Multiple

Following the HREC approval, administration of GT-02287 in the MAD part of the study has been initiated.

Key Points: 
  • Following the HREC approval, administration of GT-02287 in the MAD part of the study has been initiated.
  • “Our Phase 1 clinical trial of GT-02287 remains on track and according to plan,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics.
  • Compelling preclinical data presented at WORLDSymposium™ earlier in February 2024 demonstrated that treatment with GT-02287 completely restored motor function and reduced plasma levels of the emerging neurodegeneration biomarker NfL back to normal levels.
  • Based on these data, GT-02287 may have the potential to slow or even stop the progression of Parkinson’s disease.

Vanqua Bio to Present Pre-Clinical Data on VQ-101, a Novel GCase Activator For The Treatment of GBA-Parkinson’s Disease

Retrieved on: 
Tuesday, February 27, 2024
Molecule, Glucocerebrosidase, DSM-IV codes, Neuroprotection, Doctor of Philosophy, Conference

CHICAGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- Vanqua Bio today announced that the company will participate in the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders, which will be held in Lisbon, Portugal and virtually from March 5-9.

Key Points: 
  • CHICAGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- Vanqua Bio today announced that the company will participate in the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders, which will be held in Lisbon, Portugal and virtually from March 5-9.
  • “We created Vanqua Bio with the aim of developing innovative therapies capable of effectively slowing or halting the progression of neurodegenerative disorders, including GBA-PD, which is the largest genetically defined subset of Parkinson’s disease,” said Jim Sullivan, PhD, Co-founder and Chief Executive Officer of Vanqua Bio.
  • “We look forward to discussing the pre-clinical characterization of our clinical stage program, VQ-101, a brain-penetrant small molecule allosteric activator of GCase.
  • Furthermore, we look forward to sharing data on the development of a live cell GCase activity assay, which has enabled measurement of GCase activation in our preclinical systems and will be used to assess GCase activation in our Phase 1 trial.”
    Title: VQ-101, A Small Molecule Allosteric Activator of Glucocerebrosidase, Demonstrates Neuroprotection in Models of GBA-Parkinson’s Disease and Robust In-Vivo Target Engagement

Gain Therapeutics’ GT-02287 Completely Restores Motor Function in Mouse Models of Parkinson’s Disease

Retrieved on: 
Tuesday, February 6, 2024
Neuroprotection, Poster, Pharmacokinetics, ALS, AFL, Clinical trial, Patient, Safety, Mutation, FDA, Animal, Food, MPS II, Disease, Therapy, MPS, Neurofilament, SOD1, Glucocerebrosidase, GBA1, Plasma, Pharmaceutical industry

The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.

Key Points: 
  • The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.
  • “We believe the data showing complete restoration of motor function in a therapeutic model are remarkable and further support the potential of GT-02287 to slow or stop the progression of Parkinson’s disease, a disease for which only symptomatic treatments are available to patients at this time,” said Matthias Alder, Gain Therapeutics’ Chief Executive Officer.
  • Further, animals in the most challenging treatment group – those that began treatment eight days following onset of the disease – showed motor improvement from day 14 to day 27, which suggests progressive reversal of neuronal deficit associated with continued treatment duration.
  • Further details of the study, including protocol and specific results can be found in the poster, which was presented today and can be accessed here .

Gain Therapeutics’ CEO Matthias Alder Issues Letter to Shareholders and Provides Operational Update

Retrieved on: 
Wednesday, January 31, 2024
Multiple sclerosis, Movement, Poster, GM1 gangliosidoses, Neuroprotection, Enamine, Workflow, SAD, AFL, GM1, ChromeOS, Clinical trial, Research, PLOS, Neuroinflammation, Biomarker, Patient, Safety, Ferdinand Magellan, FDA, Drug discovery, Organization, Neurodegenerative disease, AbbVie, Dopamine, Disease, Protein, Therapy, Acquisition, Merck, Letter, Glucocerebrosidase, GBA1, GLB1, University, Parkinson's disease, ALS, Plasma, Conference, Caraway, Pharmaceutical industry

In 2023, we advanced GT-02287 through preclinical development and initiated the company’s first clinical trial in September 2023 on time and on plan.

Key Points: 
  • In 2023, we advanced GT-02287 through preclinical development and initiated the company’s first clinical trial in September 2023 on time and on plan.
  • The dose escalation of the SAD phase is underway, and the MAD phase of the study is expected to begin in Q1 2024.
  • In 2023, we made several data presentations of results of our GBA1 program in preclinical models of Parkinson’s disease and Alzheimer’s disease.
  • Xavi is leaving the world of academia as a professor at the University of Barcelona and his part-time engagement with Gain to join a major pharmaceutical company.

Gain Therapeutics Announces Acceptance of Late-Breaking Abstract for Platform Presentation at the 20th Annual WORLDSymposium™ 2024

Retrieved on: 
Tuesday, January 2, 2024
Glucocerebrosidase, Neuroprotection, Therapy

BETHESDA, Md., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced that a late-breaking abstract with new data of GT-02287 in preclinical models of Parkinson’s disease has been accepted for a platform presentation at the 20 th Annual WORLDSymposium™ being held February 4-9, 2024 in San Diego, CA.

Key Points: 
  • BETHESDA, Md., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced that a late-breaking abstract with new data of GT-02287 in preclinical models of Parkinson’s disease has been accepted for a platform presentation at the 20 th Annual WORLDSymposium™ being held February 4-9, 2024 in San Diego, CA.
  • Details of the platform presentation are as follows:
    Title: GT-02287, a clinical stage GCase enhancer, displays neuroprotection and restores motor function in preclinical models of Parkinson’s disease following delayed administration

Vanqua Bio Advances Clinical Candidate for Parkinson’s Disease, Expands Leadership Team to Support Further Development

Retrieved on: 
Tuesday, December 12, 2023
Lysosome, Protein, Immune system, Élan, Recycling, Research, Hope, Bristol Myers Squibb, Health, Movement disorder, Doctor of Philosophy, Human, Alector, Glucocerebrosidase, PD, GLP, Glycogen storage disease, Retina, CNS, Biomarker, Regeneron Pharmaceuticals, Growth, Cell, MPH, Patient, Safety, Alpha-synuclein, Neuron, Pharmaceutical industry

Mutations in GBA1 are the most common genetic risk factor for PD, representing approximately 10% of patients with PD worldwide.

Key Points: 
  • Mutations in GBA1 are the most common genetic risk factor for PD, representing approximately 10% of patients with PD worldwide.
  • His research activities have encompassed efforts to develop novel clinical outcome measures and biomarkers, including digital health technologies for clinical trials.
  • As Chief Development Officer, Mr. Siddiqui will drive the progression of Vanqua Bio’s programs through clinical development.
  • Previously, he served as head of clinical development operations at Alector, a biotech company working on leveraging the immune system to discover cures for neurodegenerative diseases.

Freeline Presents Positive New Data from Phase 1/2 GALILEO-1 Trial of FLT201, Its Novel Gene Therapy Candidate, in Gaucher Disease at ESGCT 30th Annual Congress

Retrieved on: 
Wednesday, October 25, 2023
Bone pain, Research, Plasma, Clinical trial, AAV, DBS, Biomarker, AST, Congress, Liver, Cancer Treatment Centers of America, CEST, Cell, Enzyme replacement therapy, White blood cell, Tissue, Gene, Glucocerebrosidase, Blood, ESGCT, Gaucher's disease, Safety, European Society for Primary Immunodeficiencies, Lysosome, ALT, Pharmaceutical industry, Patient

LONDON, Oct. 25, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that new clinical data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, show a substantial reduction of glucosylsphingosine (lyso-Gb1) levels in the blood of the first patient treated with FLT201. Lyso-Gb1 is a well-established biomarker of clinical response in Gaucher disease, with reductions in lyso-Gb1 correlating with positive clinical outcomes. These data are being highlighted in an oral presentation at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress held in Brussels, Belgium.

Key Points: 
  • Lyso-Gb1 is a well-established biomarker of clinical response in Gaucher disease, with reductions in lyso-Gb1 correlating with positive clinical outcomes.
  • These data are being highlighted in an oral presentation at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress held in Brussels, Belgium.
  • Reduction in lyso-Gb1 in the blood is strongly correlated with improvement in clinical outcomes in Gaucher disease.
  • The presentation entitled “Results from GALILEO-1, a First-In-Human Clinical Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1” is part of a session taking place from 14:30-16:30 CEST (8:30-10:30 a.m.

Freeline to Present New Clinical Data for FLT201 in Gaucher Disease in Oral Presentation at Upcoming ESGCT 30th Annual Congress

Retrieved on: 
Monday, October 16, 2023
Congress, Cell, Glucocerebrosidase, Gene, Safety, CEST, Gaucher's disease, AAV, ESGCT, European Society of Gene and Cell Therapy, Patient, Pharmaceutical industry

The oral presentation will include data on safety, tolerability, GCase enzyme activity, substrate, and hemoglobin and platelet levels from the first two patients treated with FLT201.

Key Points: 
  • The oral presentation will include data on safety, tolerability, GCase enzyme activity, substrate, and hemoglobin and platelet levels from the first two patients treated with FLT201.
  • Details of the oral presentation are below:
    Date and Time: Wednesday, October 25 from 14:30-16:30 CEST (8:30-10:30 a.m.
  • ET)
    Additional details on the meeting can be found at the ESGCT 30th Annual Congress website .
  • The presentation materials will be available on the Investors section of Freeline’s website following presentation at the ESGCT Congress.