Methylmalonyl-CoA mutase

Clinical Development Update from Portfolio Company LogicBio

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Monday, May 9, 2022
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As a listed company, we are able to bring this exciting growth phase of our industry to a broader range of investors.

Key Points: 
  • As a listed company, we are able to bring this exciting growth phase of our industry to a broader range of investors.
  • In its letter, the FDA acknowledged that the company satisfactorily addressed all clinical hold issues.
  • In connection with the lifting of the clinical hold, LogicBio amended the SUNRISE protocol in a manner that reflected its dialogue with the FDA.
  • LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood.

Arix Bioscience PLC: Clinical development update from portfolio company LogicBio

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Wednesday, February 2, 2022
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Arix Bioscience plc is a global venture capital company focused on investing in and building breakthrough biotech companies around cutting-edge advances in life sciences.

Key Points: 
  • Arix Bioscience plc is a global venture capital company focused on investing in and building breakthrough biotech companies around cutting-edge advances in life sciences.
  • ET
    LEXINGTON, Mass., Feb. 2, 2022 /PRNewswire/ -- LogicBio Therapeutics, Inc. (Nasdaq: LOGC), a clinical-stage genetic medicine company, today provided an update on the LB-001 clinical development program.
  • The U.S. Food and Drug Administration (FDA) has notified the company that its Phase 1/2 SUNRISE clinical trial of LB-001 in pediatric patients with methylmalonic acidemia (MMA) has been placed on clinical hold.
  • The company will host a conference call and webcast this morning to discuss this update.

LogicBio Therapeutics Provides Update on LB-001 Clinical Development Program

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Wednesday, February 2, 2022
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LEXINGTON, Mass., Feb. 2, 2022 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq: LOGC), a clinical-stage genetic medicine company, today provided an update on the LB-001 clinical development program.

Key Points: 
  • LEXINGTON, Mass., Feb. 2, 2022 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq: LOGC), a clinical-stage genetic medicine company, today provided an update on the LB-001 clinical development program.
  • The FDA subsequently notified the company that the SUNRISE trial has been placed on clinical hold.
  • LogicBio will be working closely with the FDA and the DSMB to determine the next steps for the SUNRISE trial and the LB-001 program.
  • LogicBio will host a conference call and webcast today,Wednesday, February 2, 2022, at8:30 a.m. ETto discuss the program update.

LogicBio Therapeutics Provides Business Updates

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Wednesday, December 22, 2021
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LEXINGTON, Mass., Dec. 22, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq: LOGC), a clinical-stage genetic medicine company, today provided updates around its SUNRISE Phase 1/2 clinical trial and development pipeline, and announced the appointment of Susan R. Kahn to its Board of Directors.

Key Points: 
  • LEXINGTON, Mass., Dec. 22, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq: LOGC), a clinical-stage genetic medicine company, today provided updates around its SUNRISE Phase 1/2 clinical trial and development pipeline, and announced the appointment of Susan R. Kahn to its Board of Directors.
  • In accordance with its previously announced guidance, LogicBio alsoannounced the nomination of a new development candidate, LB-401, for the treatment of hereditary tyrosinemia type 1 (HT1).
  • in applied mathematics-economics from Brown University and an M.B.A. from the Tuck School of Business at Dartmouth.
  • "I am excited to join the LogicBio Board at what I believe is a pivotal time for the company," said Ms. Kahn.

LogicBio Therapeutics Announces Early Clinical Trial Results Demonstrating First-Ever In Vivo Genome Editing in Children

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Monday, October 18, 2021
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www.arixbioscience.com

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  • www.arixbioscience.com
    - Based on safety data from first two patients, independent Data Safety Monitoring Board recommended continuation of trial, enabling enrollment of children as young as six months and dose escalation
    LEXINGTON, Mass., Oct. 18, 2021 -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company, today announced clinical trial results demonstrating the first-ever in vivo genome editing in children.
  • The SUNRISE trial is evaluating the safety, tolerability and preliminary efficacy of LB-001, the company's investigational, single-administration genome editing therapy, in pediatric patients with methylmalonic acidemia (MMA).
  • These results follow a recommendation from the independent Data Safety Monitoring Board (DSMB) overseeing the SUNRISE trial to continue the study without modification.
  • We look forward to continuing to progress the clinical study to better understand the biochemical and clinical effect of this genome editing therapy."

Moderna Announces First Patient Dosed in Phase 1/2 Study of mRNA-3705 for Methylmalonic Acidemia

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Monday, August 16, 2021
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We sincerely hope that it brings this brave patient, and many more like them, a brighter future free of the restrictions of this terrible disease.

Key Points: 
  • We sincerely hope that it brings this brave patient, and many more like them, a brighter future free of the restrictions of this terrible disease.
  • Methylmalonic acidemia is a rare, life-threatening, inherited metabolic disorder that is most commonly (approximately 60% of cases) caused by a deficiency in the mitochondrial enzyme methylmalonic-CoA mutase (MUT).
  • This deficiency can lead to metabolic crises due to a toxic buildup of acids in the body and progresses into multi-organ disease.
  • Moderna recognizes the impact of rare diseases on patients and their families, particularly when the disease lacks effective treatment options.