GLB1

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

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Wednesday, January 31, 2024
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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 Preclinical Data Showing Restoration of Enzymatic Function with Novel Allosteric Regulators in GM1 Gangliosidosis Model

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Friday, December 1, 2023
Quality of life, Protein, GLB1, GM1, Disorder, PLOS, Brain, Muscle weakness, Doctor of Philosophy, Research, Therapy, Glycogen storage disease, Life expectancy, DSM-IV codes, Paratriathlon, Dystonia, Protein folding, Patient, HaloTag, Pharmaceutical industry

BETHESDA, Md., Dec. 01, 2023 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc., (Nasdaq: GANX), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced the publication of preclinical data identifying a novel class of small molecule allosteric regulators that demonstrate therapeutic potential for galactosidase beta 1 (GLB1)-related lysosomal storage disorders (LSDs), including GM1 gangliosidosis. The study, “Validation of a highly sensitive HaloTag-based assay to evaluate the potency of a novel class of allosteric β-Galactosidase correctors,” was published in PLOS ONE.

Key Points: 
  • The study, “Validation of a highly sensitive HaloTag-based assay to evaluate the potency of a novel class of allosteric β-Galactosidase correctors,” was published in PLOS ONE.
  • GM1-gangliosidosis is an inherited, progressive disorder characterized by the degeneration of brain and spinal cord cells, leading to muscle weakness, skeletal abnormalities, dystonia, and vision problems.
  • Mutations in the GLB1 gene significantly reduce the activity and function of the lysosomal hydrolase enzyme β-galactosidase (β-Gal) due to protein misfolding.
  • Additionally, there are no available disease-modifying treatments for GM1 gangliosidosis, with available therapies only focused on reducing symptoms and improving quality of life.

Passage Bio Announces Promising Interim Clinical Data from First Eight Patients with GM1 Gangliosidosis in Imagine-1 Study

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Monday, August 7, 2023
GLB1, PASG, Central nervous system, Natural history, ICM, Cohort, Dose, Survival, GM1, Biomarker, CNS, Infant, Patient, CSF, Safety, Disease, Pharmaceutical industry

Imagine-1 is a Phase 1/2, global, open-label, dose-escalation study of the AAVhu68 gene therapy PBGM01 delivered by intra-cisterna magna (ICM) injection in six cohorts of pediatric subjects with early and late infantile GM1 Gangliosidosis (GM1).

Key Points: 
  • Imagine-1 is a Phase 1/2, global, open-label, dose-escalation study of the AAVhu68 gene therapy PBGM01 delivered by intra-cisterna magna (ICM) injection in six cohorts of pediatric subjects with early and late infantile GM1 Gangliosidosis (GM1).
  • GM1 is a rare, fatal lysosomal storage disease in which mutations in the GLB1 gene result in very low activity of the enzyme beta-galactosidase (β-Gal).
  • "We are highly encouraged by the compelling data emerging from our Imagine-1 study, with results underscoring the potential of PBGM01 to be a transformative therapy for GM1 patients,” said William Chou, M.D., president and chief executive officer of Passage Bio.
  • The amended study protocol has been approved at several clinical trial sites, including in Brazil, Canada, Turkey and the United States.

Passage Bio Presents Additional Interim Data from Imagine-1 Study for GM1 Gangliosidosis at 19th Annual WORLDSymposium™ 2023

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Friday, February 24, 2023
ICM, GM1, Safety, Central nervous system, Patient, Structural Damage, Basal ganglia, Cohort, CSF, Brain, GLB1, MRI, Toxicity, CNS, White matter, Natural history, Infant, PASG, Urine, DRG, Pharmaceutical industry, Medical imaging

Imagine-1 is a global, open-label, dose-escalation study of the AAVhu68 gene therapy PBGM01 delivered by intra-cisterna magna (ICM) injection in four cohorts of pediatric subjects with early and late infantile GM1 Gangliosidosis (GM1).

Key Points: 
  • Imagine-1 is a global, open-label, dose-escalation study of the AAVhu68 gene therapy PBGM01 delivered by intra-cisterna magna (ICM) injection in four cohorts of pediatric subjects with early and late infantile GM1 Gangliosidosis (GM1).
  • GM1 is a rare, fatal lysosomal storage disease in which mutations in the GLB1 gene result in very low activity of the enzyme beta-galactosidase (β-Gal).
  • The presentations at the WORLDSymposium™ include safety, biomarker and efficacy data from six treated patients in the first three cohorts of the study.
  • A copy of the data presentation will be available on the Investor Events and Presentations page of the Passage Bio corporate website following presentation of the materials.

Passage Bio Announces Positive Interim Clinical Data from First Six Patients with GM1 Gangliosidosis in Imagine-1 Study

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Wednesday, December 14, 2022
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Cohort 4 (early infantile, high dose) patients have been dosed and data is expected by mid-2023.

Key Points: 
  • Cohort 4 (early infantile, high dose) patients have been dosed and data is expected by mid-2023.
  • “We are excited to share interim data from this first six patients in our Imagine-1 study, which further reinforce our confidence in PBGM01 as a promising treatment option for GM1 gangliosidosis,” said William Chou, M.D., chief executive officer of Passage Bio.
  • The clinical program has treated a total of four cohorts of two patients each, with separate dose-escalation cohorts for late infantile GM1 and early infantile GM1.
  • The primary goal of the study is to first assess safety and tolerability and then efficacy of PBGM01 in patients.

Passage Bio Presents New Interim Clinical and Biomarker Data for Patients with GM1 Gangliosidosis in Imagine-1 Study at ASGCT 25th Annual Meeting

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Wednesday, May 18, 2022
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M.M.Sc., senior vice president, Clinical Development, Passage Bio, will present the data at 8:30 a.m.

Key Points: 
  • M.M.Sc., senior vice president, Clinical Development, Passage Bio, will present the data at 8:30 a.m.
  • ET during a late-breaker oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 25thAnnual Meeting, being held in Washington, D.C. and virtually.
  • We continue to be encouraged by the interim data from the first cohort in our Imagine-1 trial and the potential promise it offers for patients.
  • The primary goal of the study is to first assess safety and tolerability and then efficacy of PBGM01 in patients.

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

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Friday, February 11, 2022
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, CNS, Patient, GLB1, LYS, Lists of diseases, Biomarker, Nerve, Financial Services and Markets Authority (Belgium), Company, GM3, Neurodegeneration, Autism, Spinal cord, HIV disease progression rates, GM2, Brain, Forward-looking statement, Review, MPS, Serum, Data monitoring committee, Clinical trial, Safety, HS, Infant, Adaptive clinical trial, GM1, A-DNA, Glycogen storage disease, Fragile X syndrome, Central nervous system, Disease, Blood, CSF, Ganglioside, Choroid plexus, Pharmaceutical industry, Medical imaging, Lysogenic cycle

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).

Sio Gene Therapies to Present at the 18th Annual WORLDSymposium™ 2022

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Wednesday, February 2, 2022
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NEW YORK andDURHAM, N.C., Feb. 02, 2022 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc.(NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that it will present data in an oral platform presentation and two poster presentations at the 18th Annual WORLDSymposium 2022, to be held February 7-11, 2022.

Key Points: 
  • NEW YORK andDURHAM, N.C., Feb. 02, 2022 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc.(NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that it will present data in an oral platform presentation and two poster presentations at the 18th Annual WORLDSymposium 2022, to be held February 7-11, 2022.
  • The Company is also collaborating with Invitae, who will present a poster on the Detect LSD program.
  • The gene therapy is delivered intravenously, which has the potential to achieve a broad central and peripheral biodistribution.
  • Sio Gene Therapiescombines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Passage Bio Announces Positive Interim Safety and Biomarker Data and Advances Phase 1/2 Trial of PBGM01 in GM1 Gangliosidosis

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Friday, December 17, 2021
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The primary goal of the study is to first assess safety and tolerability and then efficacy of PBGM01.

Key Points: 
  • The primary goal of the study is to first assess safety and tolerability and then efficacy of PBGM01.
  • The IDMC recommendation followed positive interim safety and biomarker data from Cohort 1 (n=2) patients with late infantile GM1 who received a low dose of PBGM01.
  • This study measured both cerebrospinal fluid (CSF) and serum levels of beta-galactosidase in patients with juvenile and early infantile GM1.
  • We are extremely encouraged by the interim data in this first low dose cohort of patients with late infantile GM1.

Sio Gene Therapies Announces Granting of FDA Fast Track Designation for Investigational AXO-AAV-GM1 (AAV9-GLB1) Gene Therapy in Patients with GM1 Gangliosidosis

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Thursday, October 21, 2021
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Receiving Fast Track Designation is a critical step in our mission to develop the first potential treatment for all pediatric forms of this rare, terminal disease.

Key Points: 
  • Receiving Fast Track Designation is a critical step in our mission to develop the first potential treatment for all pediatric forms of this rare, terminal disease.
  • AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from theFDAand is the only gene therapy in clinical development for all pediatric forms of GM1 gangliosidosis.
  • In 2018, Sio licensed exclusive worldwide rights from UMass Chan Medical Schoolfor the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.
  • Sio Gene Therapiescombines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.