Cisterna magna

CereVasc's Minimally Invasive Central Nervous System Therapy Delivery System Receives Outstanding Presentation Award at American Society of Gene & Cell Therapy Annual Meeting

Retrieved on: 
Wednesday, June 14, 2023
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BOSTON, June 14, 2023 /PRNewswire/ -- CereVasc, Inc., a privately held, clinical-stage, medical device company developing novel treatments for neurological diseases, announced today that a presentation of study results for its minimally invasive Central Nervous System (CNS) Delivery System was awarded an Outstanding Poster Presentation Award by the American Society of Gene and Cell Therapy as part of their 2023 Annual Meeting.

Key Points: 
  • BOSTON, June 14, 2023 /PRNewswire/ -- CereVasc, Inc., a privately held, clinical-stage, medical device company developing novel treatments for neurological diseases, announced today that a presentation of study results for its minimally invasive Central Nervous System (CNS) Delivery System was awarded an Outstanding Poster Presentation Award by the American Society of Gene and Cell Therapy as part of their 2023 Annual Meeting.
  • "We are honored that ASGCT recognized data on our minimally invasive CNS Delivery System with an Outstanding Poster Presentation Award," said Dan Levangie, Chairman and CEO of CereVasc.
  • "We appreciate the challenges faced by CNS therapeutic researchers and industry to identify an effective and minimally invasive route of administration for their therapeutic assets.
  • We believe our data highlighted with this Award demonstrates the potential of our novel alternative to more invasive techniques employed to deliver therapies to the CNS.

Passage Bio Reports Fourth Quarter and Full-Year 2022 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Monday, March 6, 2023
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ET

Key Points: 
  • ET
    PHILADELPHIA, March 06, 2023 (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 fourth quarter and full-year ended December 31, 2022 and provided recent business highlights.
  • 2023 is an exciting year for Passage as we focus on operational execution and generation of clinical data across our two lead programs.
  • A live audio webcast of the event will be available on the Investors & News section of Passage Bio’s website at investors.passagebio.com .
  • The archived webcast will be available on Passage Bio's website approximately two hours after the completion of the event and for 30 days following the call.

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

Retrieved on: 
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 Doses First Patient in Global Clinical Trial of PBFT02 Gene Therapy for Frontotemporal Dementia with Granulin Mutations

Retrieved on: 
Thursday, August 11, 2022
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We are grateful for the support from the families and clinical trial investigators who have chosen to participate in our studies.

Key Points: 
  • We are grateful for the support from the families and clinical trial investigators who have chosen to participate in our studies.
  • This gene therapy uses an AAV1 viral vector to deliver a functional copy of the GRN gene encoding progranulin (PGRN) to a patient's cells.
  • If you are interested in a referral to a clinical trial site, please contact Passage Bio here to learn more.
  • PBFT02 is supported by extensive preclinical studies conducted by Passage Bios collaborator, the University of Pennsylvanias Gene Therapy Program.

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

Retrieved on: 
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.

$7.6 Million Worldwide Alpha Mannosidosis Industry to 2030 - Identify Growth Segments for Investment - ResearchAndMarkets.com

Retrieved on: 
Thursday, March 31, 2022
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The "Alpha Mannosidosis Global Market Opportunities And Strategies To 2030, By Therapy Type, Indication, End-User" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Alpha Mannosidosis Global Market Opportunities And Strategies To 2030, By Therapy Type, Indication, End-User" report has been added to ResearchAndMarkets.com's offering.
  • The global alpha mannosidosis market reached a value of nearly $7.60 million in 2020 to $70.06 million in 2025 at a rate of 55.9%.
  • The hospitals market was the largest segment of the alpha mannosidosis market segmented by end-user, accounting for 81.5% of the total in 2020.
  • The alpha mannosidosis market size will gain the most in the USA at $30.7 million.

Passage Bio Doses First Patient in Global Clinical Trial for Infantile Krabbe Disease, A Rare Fatal Pediatric Condition

Retrieved on: 
Thursday, March 10, 2022
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It is gratifying to dose the first patient in our GALax-C trial, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio.

Key Points: 
  • It is gratifying to dose the first patient in our GALax-C trial, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio.
  • Krabbe disease is a rare pediatric lysosomal storage disorder caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine.
  • We also are grateful to the children, families, and clinical trial investigators who have chosen to participate in our studies.
  • The U.S. Food and Drug Administration (FDA) has granted PBKR03 Fast Track, Orphan Drug, and Rare Pediatric Disease designations.

Passage Bio to Showcase GM1 Gangliosidosis and Krabbe Clinical Programs at 2022 WORLDSymposium, February 7 – 11

Retrieved on: 
Monday, January 31, 2022
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Passage Bio will also deliver two platform presentations on the designs of its clinical trials for Imagine-1 for GM1 gangliosidosis and GALax-C for Krabbe disease on Wednesday, February 9.

Key Points: 
  • Passage Bio will also deliver two platform presentations on the designs of its clinical trials for Imagine-1 for GM1 gangliosidosis and GALax-C for Krabbe disease on Wednesday, February 9.
  • On Friday, February 11, as part of the conferences late-breaking science session, the company will present safety and clinical development milestone data for the two patients in Cohort 1 of the Imagine-1 clinical trial.
  • As part of our Imagine-1 late-breaker presentation, we will be excited to share new clinical data from Cohort 1 in the ongoing Imagine-1 trial, said Bruce Goldsmith, Ph.D., president and chief executive officer, Passage Bio.
  • Presenter: David Weinstein, M.D., M.M.Sc., Passage Bio, Philadelphia, PA
    Passage Bio is planning to host a virtual investor webinar on Friday, February 11 at 1 p.m.

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

Retrieved on: 
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.