Beta-galactosidase

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 Reports Second Quarter 2022 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Thursday, August 4, 2022
Cell, Private Securities Litigation Reform Act, Gene, Food, Nasdaq, IND, ARSA, American Society of Gene and Cell Therapy, CSF, Frontotemporal dementia, Central nervous system, Caregiver, Risk, Research and development, Glycogen storage disease, Investor, ICM, MLD, CNS, Safety, Passage, FDA, Krabbe disease, Adult, Investigational New Drug, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Infant, GLOBE, U.S. Securities and Exchange Commission, Cohort, Patient, GM1, Beta-galactosidase, Serum, Webcast, Administration, Cerebrospinal fluid, University, Clinical trial, Corporate governance, Failure, Security (finance), Company, PASG, Society, Research, R, SEC, Pharmaceutical industry

ET

Key Points: 
  • ET
    PHILADELPHIA, Aug. 04, 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 reported financial results for the second quarter ended June 30, 2022 and provided recent business highlights.
  • The team at Passage is driven to fulfill our vision of developing ground-breaking therapies that transform the lives of patients with CNS diseases.
  • Research and Development (R&D) Expenses: R&D expenses were $26.8 million for the second quarter ended June 30, 2022, compared to $33.1 million for the same quarter in 2021.
  • General and Administrative (G&A) Expenses:G&A expenses were $13.0 million for the second quarter ended June 30, 2022, compared to $15.4 million for the same quarter in 2021.

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.

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.

Passage Bio Reports Third Quarter 2021 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Thursday, November 4, 2021
Safety, COVID-19, Central nervous system disease, Research, Charcot–Marie–Tooth disease, University, Metachromasia, Failure, CNS, Data, Amyotrophy, Security (finance), U.S. Securities and Exchange Commission, Patient, Krabbe disease, Investor, Private Securities Litigation Reform Act, Infant, SEC, PASG, Frontotemporal dementia, Risk, Adult, Research and development, Company, Beta-galactosidase, Clinical trial, Webcast, GM1, Temporal lobe epilepsy, IND, Program, R, Nasdaq, Serum, Cerebrospinal fluid, Amyotrophic lateral sclerosis, Metachromatic leukodystrophy, GLOBE, Administration, Pharmaceutical industry

ET

Key Points: 
  • ET
    PHILADELPHIA, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system disorders, today reported financial results for the third quarter ended September 30, 2021 and provided recent business highlights.
  • As we close this year, we remain steadfastly focused on strong execution of our three clinical programs.
  • Appointed Maxine Gowen, Ph.D., as chairwoman of the board of directors in August 2021: This appointment followed the unexpected passing of Passage Bio co-founder and former board chair Dr. Tadataka Yamada.
  • General and Administrative (G&A) Expenses:G&A expenses were $15.0 million for the third quarter ended September 30, 2021, compared to $7.8 million for the same quarter in 2020.

Gain Therapeutics to Present at 2021 International Virtual GM1 Community Conference

Retrieved on: 
Thursday, September 9, 2021

GM1 Gangliosidosis is a hereditary, progressive disease mostly impacting neurons in the brain and spinal cord, caused by mutations in GLB1.

Key Points: 
  • GM1 Gangliosidosis is a hereditary, progressive disease mostly impacting neurons in the brain and spinal cord, caused by mutations in GLB1.
  • In both diseases, these mutations result in the misfolding and subsequent dysfunction of GLB, which leads to toxic substrate accumulation in organs and tissues.
  • Gain Therapeutics, Inc. is positioned at the confluence of technology and healthcare and focused on redefining drug discovery with its SEE-Tx target identification platform.
  • In July 2020, Gain Therapeutics, Inc. completed a share exchange with Gain Therapeutics, SA, a Swiss corporation, whereby GT Gain Therapeutics SA became a wholly owned subsidiary of Gain Therapeutics, Inc. For periods and at dates prior to the Corporate Reorganization, the consolidated financial statements were prepared based on the historical financial statements of GT Gain Therapeutics SA.

Passage Bio Announces First Patient Dosed in Imagine-1 Study of PBGM01 Gene Therapy for Infantile GM1 Gangliosidosis

Retrieved on: 
Thursday, April 1, 2021
Enzymes, Branches of biology, Catalysts, GLB1, Galactosides, Hydrolases, Lysosomal storage disease, Rare diseases, Beta-galactosidase, Lysosome, GM1 gangliosidoses

GM1, a rare monogenic lysosomal storage disease, is caused by mutations in the GLB1 gene, which encodes the lysosomal enzyme beta-galactosidase (-gal).

Key Points: 
  • GM1, a rare monogenic lysosomal storage disease, is caused by mutations in the GLB1 gene, which encodes the lysosomal enzyme beta-galactosidase (-gal).
  • We are extremely grateful to the families and patients for volunteering to support the effort to bring the PBGM01 gene therapy into clinical studies.
  • The clinical program will enroll a total of four cohorts of two patients each, with separate dose-escalation cohorts for late infantile GM1 and early infantile GM1.
  • As part of the global Imagine-1 study for PBGM01, Passage Bio plans to open 10 clinical study sites around the world, many of which are considered GM1 Centers of Excellence.

Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase 1/2 Trial of AXO-AAV-GM1 for GM1 Gangliosidosis

Retrieved on: 
Tuesday, December 15, 2020
Rare diseases, Lipid storage disorders, Enzymes, Catalysts, GLB1, Branches of biology, GM1 gangliosidoses, Gangliosidosis, GM1, Beta-galactosidase

Further, the safety profile observed in the low-dose cohort support moving forward with the high-dose cohort of AXO-AAV-GM1 in the ongoing Phase 1/2 study.

Key Points: 
  • Further, the safety profile observed in the low-dose cohort support moving forward with the high-dose cohort of AXO-AAV-GM1 in the ongoing Phase 1/2 study.
  • I am encouraged by the results-to-date where we have seen preservation of function and in a few measures even improvement.
  • All patients had documented biallelic mutations in GLB1 gene, deficiency of beta-galactosidase enzyme activity, and clinical phenotype consistent with GM1 gangliosidosis.
  • Additional data will be collected at the 12-month evaluation including several measures of the systemic manifestations of GM1 gangliosidosis.

Passage Bio Receives Rare Pediatric Disease Designation for PBGM01 for Patients with GM1 Gangliosidosis

Retrieved on: 
Thursday, May 21, 2020
Enzymes, Rare diseases, Lipid storage disorders, Branches of biology, Catalysts, GLB1, GM1 gangliosidoses, Lysosomal storage disease, GM1, Beta-galactosidase, Gangliosidosis, Lysosome

PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1 gangliosidosis (GM1) and has previously been granted Orphan Drug designation.

Key Points: 
  • PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1 gangliosidosis (GM1) and has previously been granted Orphan Drug designation.
  • We believe that PBGM01 has the potential to restore developmental progression, enabling patients to achieve additional milestones and improve quality of life.
  • Passage anticipates starting a Phase 1/2 trial for PBGM01 for the treatment infantile GM1 in the fourth quarter of 2020.
  • GM1 gangliosidosis (GM1) is a rare and often life-threatening monogenic recessive lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid beta-galactosidase (-gal).

FDA Grants Passage Bio Orphan Drug Designation for PBGM01 for Treatment of Infantile GM1 Gangliosidosis

Retrieved on: 
Tuesday, April 21, 2020
Rare diseases, Lipid storage disorders, Enzymes, Catalysts, Branches of biology, GM1 gangliosidoses, GLB1, Gangliosidosis, Lysosomal storage disease, GM1, Beta-galactosidase

PHILADELPHIA, April 21, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead product candidate, PBGM01, for the treatment of infantile GM1 gangliosidosis (GM1).

Key Points: 
  • PHILADELPHIA, April 21, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead product candidate, PBGM01, for the treatment of infantile GM1 gangliosidosis (GM1).
  • Infantile GM1 is an incredibly devastating disease that impacts one of our most vulnerable populations, said Bruce Goldsmith, Ph.D, chief executive officer of Passage Bio.
  • This designation represents an important recognition of the dire need for an effective treatment option for the children and their families impacted by GM1.
  • GM1 gangliosidosis (GM1) is a rare and often life-threatening monogenic recessive lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid beta-galactosidase (-gal).