GM1 gangliosidoses

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.

Passage Bio Reports First Quarter 2023 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Thursday, May 11, 2023
PASG, Central nervous system, GM1 gangliosidoses, Cohort, Dose, GRN, Clinical trial, G&A, GM1, CNS, Infant, Patient, Administration, Frontotemporal dementia, Research and development, R, Research, Catalent, FTD, Safety, Pharmaceutical industry

Dose first patient at higher dose (Dose 3) in Imagine-1 clinical trial for GM1 in the second half of 2023.

Key Points: 
  • Dose first patient at higher dose (Dose 3) in Imagine-1 clinical trial for GM1 in the second half of 2023.
  • Present initial safety and biomarker data for Cohort 1 in upliFT-D clinical trial for FTD in the second half of 2023.
  • Research and Development (R&D) Expenses: R&D expenses were $16.8 million for the quarter ended March 31, 2023, as compared to $26.2 million for the same quarter in 2022.
  • General and Administrative (G&A) Expenses: G&A expenses were $19.0 million for the quarter ended March 31, 2023, as compared to $15.1 million for the same quarter in 2022.

Passage Bio Reports Third Quarter 2022 Financial Results and Provides Business Updates

Retrieved on: 
Thursday, November 10, 2022
Investor, GLOBE, Private Securities Litigation Reform Act, PASG, IND, Adult, Investment, Risk, U.S. Securities and Exchange Commission, Workforce, Research and development, Amyotrophic lateral sclerosis, GM1, Partnership, Nasdaq, GM1 gangliosidoses, Webcast, Research, Cohort, Krabbe disease, Infant, R, SEC, Failure, Safety, Company, Program, Passage, Clinical trial, University, Patient, Security (finance), Administration, Central nervous system, CNS, Frontotemporal dementia, Pharmaceutical industry, Medicine

ET

Key Points: 
  • ET
    PHILADELPHIA, Nov. 10, 2022 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system (CNS) disorders,today provided business updates and reported financial results for the third quarter ended September 30, 2022.
  • Passage Bio has tremendous potential to transform the lives of patients with devastating CNS orders for which there is significant unmet clinical need, said William Chou, M.D., chief executive officer of Passage Bio.
  • Cash Position: Cash, cash equivalents and marketable securities were $213.8 million as of September 30, 2022, as compared to $239.3 millionas of June 30, 2022.
  • General and Administrative (G&A) Expenses: G&A expenses were $10.7 million for the third quarter ended September 30, 2022, compared to $15.0 million for the same quarter in 2021.

Passage Bio to Present Updated Imagine-1 Study Clinical and Biomarker Data at the American Society of Gene and Cell Therapy 25th Annual Meeting

Retrieved on: 
Monday, May 2, 2022
PASG, Efficacy, University, Infant, IND, GTP, NASDAQ, GLOBE, CNS, MLD, Cell, ICM, Food, GM1, Superstring theory, GM1 gangliosidoses, Poster, Patient, Gene, Cohort, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Very early onset inflammatory bowel disease, Late-onset hypogonadism, Society, Trial of the century, American Society of Gene and Cell Therapy, Annual general meeting, Central nervous system, Biomarker, Pharmaceutical industry, Safety, Tolerability

The data to be presented in the following poster supported Passage Bios Investigational New Drug submission for MLD to the U.S. Food and Drug Administration.

Key Points: 
  • The data to be presented in the following poster supported Passage Bios Investigational New Drug submission for MLD to the U.S. Food and Drug Administration.
  • Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work.
  • This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing.
  • As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies.

Lysogene Provides Medical Update

Retrieved on: 
Monday, February 7, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Adaptive clinical trial, Glycogen storage disease, Lists of diseases, Forward-looking statement, GM1, Fragile X syndrome, AP-HP, Competition and Markets Authority, Company, CNS, Neurology, MPS, Clinical trial, Neurodegeneration, Knowledge, Patient, Safety, LYS, Autism, Central nervous system, Cros, CEO, Pierre and Marie Curie University, GM1 gangliosidoses, A-DNA, Child, Pierre, Pharmaceutical industry

Lysogene (FR0013233475 LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today provides a medical update.

Key Points: 
  • Lysogene (FR0013233475 LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today provides a medical update.
  • First, Lysogene has dosed the first patient in France with LYS-GM101 investigational gene therapy at the Armand Trousseau Hospital (AP-HP).
  • Second, Lysogene has appointed a seasoned Chief Medical Officer, Dr. Marie Trad, effective February 1st, 2022.
  • Karen Aiach, Chairman and CEO of Lysogene will present preliminary safety data from the ongoing adaptive clinical study in children with GM1 gangliosidosis.

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

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Wednesday, February 2, 2022
Glycogen storage disease, GM1 gangliosidoses, GM1, Private Securities Litigation Reform Act, Patient, Infant, NASDAQ, Organization, Sensor, MD, COVID-19, Type 2, Sio, Sandhoff disease, GLB1, Genetic testing, National, Orphan, Invitae, GM2, U.S. Securities and Exchange Commission, University of Massachusetts, Company, Neurodegeneration, GLOBE, Tay–Sachs disease, NIH, Imagination, Doctor of Philosophy, Industry, Security (finance), National Human Genome Research Institute, National Institutes of Health, SIOX, AAV, Pharmaceutical industry, Medicine

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.

Lysogene Enters into a Loan Agreement of €15 million with the European Investment Bank (EIB)

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Friday, December 24, 2021
Biotechnology, Genetics, Health, Kepler, Sarepta Therapeutics, European Investment Bank, Fragile, Sale, Autism, Lists of diseases, MPS, Exercise, CNS, European Agricultural Guarantee Fund, Financial Services and Markets Authority (Belgium), Investment, BPI, Achievement, Forward-looking statement, COVID-19, Central nervous system, Fragile X syndrome, EIB, Union, History, Climate, Company, LYS, Therapy, Research, Adaptive clinical trial, GM1, Clinical trial, Parity, Vaccine, Partnership, Pharmaceutical industry, EU, GM1 gangliosidoses, the European Investment Bank (EIB), Lysogene, THE EUROPEAN INVESTMENT BANK (EIB), LYSOGENE

Lysogene (FR0013233475 LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, announced today that it has entered into a 15 million loan agreement with the European Investment Bank to accelerate the development of its gene therapy platform.

Key Points: 
  • Lysogene (FR0013233475 LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, announced today that it has entered into a 15 million loan agreement with the European Investment Bank to accelerate the development of its gene therapy platform.
  • Lysogene will receive the EIB loan in three tranches including a first tranche of 3 million that can be unconditionally drawn down and two other tranches of respectively 5 million and 7 million available upon completion of pre-defined milestones.
  • With the 4.3 million BPI non-dilutive announced in November, this brings total funding agreements to nearly 20 million.
  • The loan is supplemented by an agreement to issue warrants to the EIB, whose number varies according to the tranche and Lysogenes share price.

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

Retrieved on: 
Thursday, October 21, 2021
Safety, Type 2, Fast track (FDA), COVID-19, SIOX, Orphan drug, Disease, Industry, Neurodegeneration, GM2, Tay–Sachs disease, Ill, Organization, Fast Track, Security (finance), U.S. Securities and Exchange Commission, ESGCT, Patient, NASDAQ, Private Securities Litigation Reform Act, FDA, GLB1, Glycogen storage disease, Food, GM1 gangliosidoses, Review, University of Massachusetts, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Sandhoff disease, GM1, Biomarker, Sio, GLOBE, Imagination, AAV, 2012 Giro d'Italia, Stage 1 to Stage 11, Infant, Pharmaceutical industry

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.

Sio Gene Therapies Announces Positive Interim Safety and Biomarker Data from Ongoing Phase 1/2 Clinical Study of AXO-AAV-GM1 Gene Therapy in GM1 Gangliosidosis

Retrieved on: 
Thursday, October 21, 2021
Safety, Type 2, MRI, SIOX, Research, Disease, Neurodegeneration, Industry, CSF/serum glucose ratio, Patient, Platelet, ESGCT, NASDAQ, FDA, GLB1, Glycogen storage disease, Biomedicine, GM1 gangliosidoses, Data, Cell, University of Massachusetts, Review, Doctor of Philosophy, CSF, Conference, Natural history, NHGRI, National, Principal, GM1, Biomarker, EDT, Gene, Serum, Biomedical Research, Cerebrospinal fluid, GLOBE, European Society for Primary Immunodeficiencies, AAV, Pharmaceutical industry, Medical imaging, Medical device

Our team continues to lead the industry in the development of a new, potentially disease-modifying therapeutic option for GM1 gangliosidosis.

Key Points: 
  • Our team continues to lead the industry in the development of a new, potentially disease-modifying therapeutic option for GM1 gangliosidosis.
  • We observed dose-dependent responses in two key biomarkers, serum -galactosidase and cerebrospinal fluid (CSF) GM1 ganglioside, including normalization of both biomarkers in the high-dose cohort.
  • The biomarker dose response and favorable safety profile is a remarkable finding for the gene therapy field.
  • I am excited about the potential impact that AXO-AAV-GM1 may have on the lives of these children and their families.

UPDATE: Sio Gene Therapies to Present New Data at the European Society of Gene and Cell Therapy Virtual Congress 2021

Retrieved on: 
Monday, October 4, 2021
GM1 gangliosidoses, Food, Sandhoff disease, Neurodegeneration, Food and Drug Administration Amendments Act of 2007, HEXA, Security (finance), NASDAQ, Private Securities Litigation Reform Act, U.S. Securities and Exchange Commission, University of Massachusetts Medical School, COVID-19, Patient, European Society of Gene and Cell Therapy, GM2, Infant, Central nervous system, Gene, Type 2, Tay–Sachs disease, SIOX, Company, University, Disease, Dopamine, Cell, GM1, Brain, Organization, AAV, Imagination, GLB1, HEXB, Sio, Industry, Enzyme, GLOBE, ESGCT, Pharmaceutical industry, Vaccine

The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.

Key Points: 
  • The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.
  • The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well.
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.