Glucocerebrosidase

Freeline Presents Positive New Data from Phase 1/2 Trial of FLT201, Its Novel Gene Therapy Candidate for Gaucher Disease, in Late-Breaking Oral Presentation at ASGCT 27th Annual Meeting

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Giovedì, Maggio 9, 2024
FDA, Bone marrow, Mutation, Cell, Risk, Blood, News, Research, ERT, White blood cell, Glycogen storage disease, SRT, ALT, GBA1, White, European Medicines Agency, Brain, Enzyme replacement therapy, Regenerative Medicine Advanced Therapy, Therapy, Plasma, Patient, Mouse, Safety, Tissue, Glucocerebrosidase, Substantia nigra, Lysosome, Neuropsychological test, AAV, Dementia, PRIME, Fatigue, EMA, Drug development, Bone pain, Gaucher's disease, FACIT, Disease, Liver, Gene, Society, Standard of care, Annual general meeting, Pharmaceutical industry

LONDON, May 09, 2024 (GLOBE NEWSWIRE) --  Freeline Therapeutics today announced 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, showing substantial reductions in glucosylsphinogsine (lyso-Gb1), one of the best predictors of clinical response, in patients with persistently high levels despite years of treatment with currently approved therapies, as well as early signs of clinical improvements in bone marrow burden and fatigue. FLT201 continues to demonstrate a favorable safety and tolerability profile. These data are being showcased in a late-breaking oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, Maryland.

Key Points: 
  • These data are being showcased in a late-breaking oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, Maryland.
  • Reductions in lyso-Gb1 levels in the blood are highly correlated with substrate reduction in disease-affected tissues and positive clinical outcomes in Gaucher disease.
  • Currently approved treatments have made a significant difference for people with Gaucher disease, but there is not an existing cure.
  • Both the late-breaking oral presentation and the poster presentation are now available on the News & Events section of Freeline’s website.

Freeline to Present New Data on its Gaucher and Parkinson’s Disease Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

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Mercoledì, Aprile 24, 2024
Bone pain, Congress, Gaucher's disease, Glucocerebrosidase, News, Abstract, Cell, Fracture, Patient, Gene, Society, Annual general meeting, Therapy, Pharmaceutical industry

LONDON, April 24, 2024 (GLOBE NEWSWIRE) -- Freeline Therapeutics today announced the acceptance of multiple abstracts, including one for a late-breaking oral presentation on new clinical data from its Gaucher disease program, at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place May 7-11, 2024 in Baltimore, Maryland.

Key Points: 
  • LONDON, April 24, 2024 (GLOBE NEWSWIRE) -- Freeline Therapeutics today announced the acceptance of multiple abstracts, including one for a late-breaking oral presentation on new clinical data from its Gaucher disease program, at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place May 7-11, 2024 in Baltimore, Maryland.
  • “FLT201, our investigational gene therapy for Gaucher disease, has the potential to deliver better efficacy with a one-time treatment, and we are excited to present new clinical data from our ongoing Phase 1/2 trial in a late-breaking oral presentation at ASGCT.
  • We will also be sharing new data from our Parkinson’s disease research program, which builds on our work in Gaucher disease by leveraging the enhanced stability of our rationally engineered GCase enzyme.
  • The presentation materials will be available on the News & Events section of Freeline’s website following presentation at the ASGCT Annual Meeting.

Gain Therapeutics Announces Positive Results from the Single Ascending Dose (SAD) Part of the Phase 1 Clinical Trial of GT-02287, a Novel GCase-Targeting Small Molecule Therapy for GBA1 Parkinson’s Disease

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Mercoledì, Aprile 24, 2024
Safety, Pharmacokinetics, AFL, Glucocerebrosidase, Index, Disease, SAD, MTD, Patient, GBA1, Biomarker, PD, Therapy, Plasma, Pharmaceutical industry

GT-02287 was generally well tolerated up to and including the highest planned dose level, and there were no serious adverse events.

Key Points: 
  • GT-02287 was generally well tolerated up to and including the highest planned dose level, and there were no serious adverse events.
  • The good safety and tolerability profile and the appropriate range of plasma exposure levels achieved after oral administration further bolster GT-02287’s best and first-in-class potential.
  • The Phase 1 clinical trial is a single center, randomized, double-blind, placebo-controlled, single- and multiple ascending dose (SAD/MAD) study to evaluate the safety and tolerability of GT-02287 administered orally once daily in healthy adults.
  • Based on these data, GT-02287 may have the potential to slow the progression of Parkinson’s disease.

Capsida Biotherapeutics to Present New Data on its Wholly Owned Gene Therapy Programs in Genetic Epilepsy and Parkinson's Disease at the Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

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Lunedì, Aprile 22, 2024
Doctor of Philosophy, Brain, Parkinson's disease, Mouse, Glucocerebrosidase, Poster, Abstract (summary), MD, Cell, Associate professor, STXBP1, AAV, CNS, Genetics, Liver, Gene, Society, Central nervous system, GBA, NHP, Annual general meeting, Baylor University, Vaccine

THOUSAND OAKS, Calif., April 22, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida"), a leading fully integrated targeted gene therapy company, developing treatments for rare and common diseases across all ages, today announced eight presentations – three oral presentations and five poster presentations – at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting, taking place May 7-11, 2024 in Baltimore, MD and virtually.

Key Points: 
  • "We are excited to unveil our PD-GBA program and new data on our STXBP1 program as part of oral presentations at ASGCT," said Peter Anastasiou, Capsida's Chief Executive Officer.
  • "Both development candidates are IV-administered and achieve breakthrough levels of neuronal transduction throughout the brain, up to 70% in certain brain regions, while simultaneously detargeting the liver.
  • Data from the oral presentations are embargoed until 6:00 AM ET on the presentation day.
  • Presenter: Nicholas Flytzanis, Ph.D., Founder, Chief Research and Innovation Officer, Capsida

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

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Martedì, Aprile 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

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Mercoledì, Marzo 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

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Martedì, Febbraio 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

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Martedì, Febbraio 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

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Martedì, Febbraio 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

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Mercoledì, Gennaio 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.