Delving into the Latest Updates on DUOC-01 with Synapse

Overview

Basic Info

Drug Type

Stem cell therapy

Synonyms-

Target-

Action-

Mechanism

Cell replacements

Therapeutic Areas

Immune System DiseasesNervous System DiseasesCongenital Disorders+ [4]

Active Indication

Multiple Sclerosis, Primary ProgressiveAdrenoleukodystrophyAlpha-Mannosidosis+ [9]

Inactive Indication-

Originator Organization

Duke University

Active Organization

Duke University

Inactive Organization-

Drug Highest PhasePhase 1

First Approval Date-

Regulation-

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This study is a prospective Phase 1a open-label single- center trial. It will assess the safety of intrathecal administration of DUOC-01 cells to adults with Primary Progressive Multiple Sclerosis (PPMS). DUOC-01 is a population of cells expanded from donated human umbilical cord blood cells and is intended for treatment of neurodegenerative and demyelinating diseases. There will be approximately 20 participants enrolled. Exploratory objectives include changes in MS assessment scores, changes in brain MRI findings, and changes in blood biomarkers.

Start Date24 Jan 2021

Sponsor / Collaborator

Duke University

NCT02254863

/ RecruitingPhase 1IIT

Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases with Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.

Start Date01 Sep 2014

Sponsor / Collaborator

Duke University

100 Clinical Results associated with DUOC-01

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100 Translational Medicine associated with DUOC-01

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100 Patents (Medical) associated with DUOC-01

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6

Literatures (Medical) associated with DUOC-01

17 Jan 2025·Stem Cells Translational Medicine

Safety and feasibility of umbilical cord blood transplantation in children with neuronal ceroid lipofuscinosis: a retrospective study

Article

Author: Kurtzberg, Joanne ; Prasad, Vinod K ; Bauchat, Andrea ; Page, Kristin M ; Fabrizio, Vanessa A ; Mahadeo, Kris M ; Driscoll, Timothy A ; Martin, Paul L ; Brondon, Jennifer E ; Polishchuk, Veronika

AbstractCeroid lipofuscinosis neuronal (CLN) encompasses rare inherited neurodegenerative disorders that present in childhood with clinical features including epilepsy, psychomotor delay, progressive vision loss, and premature death. Published experience utilizing umbilical cord blood transplant (UCBT) for these disorders is limited. This retrospective analysis includes patients with CLN (2, 3, and 5) who underwent UCBT from 2012 to 2020. All subjects (n = 8) received standard-of-care myeloablative conditioning. Four also enrolled in clinical trial NCT02254863 and received intrathecal DUOC-01 cells posttransplant. Median age at UCBT was 5.9 years. All subjects achieved neutrophil engraftment with >95% donor chimerism at a median of 28.5 days. Sinusoidal obstructive syndrome was not observed. Severe acute graft-versus-host disease occurred in 12.5%. Other complications included autoimmune hemolytic anemia (25%) and viral reactivation/infection (62.5%). No transplant-related mortality was observed. Two CLN2 patients died, 1 from progressive disease and 1 from unknown cause at days +362 and +937, respectively. With median follow-up of 8 years, overall survival at 100 days and 24 months was 100% and 88%, respectively. Three of 4 CLN3 subjects stabilized Hamburg motor and language scores. While UCBT appears safe and feasible in these patients, given the variable expression and natural history, extended follow-up and further studies are needed to elucidate the potential impact of UCBT on clinical outcomes.

01 Apr 2023·Stem Cells and Development

Cellular Therapies: Yesterday, Today, and Tomorrow

Article

Author: Mayani, Hector

Cellular therapy (CT) can be defined as the transference into a person of healthy cells to correct defective functions. Yesterday (1950-2010), CT consisted mostly of hematopoietic transplants for the treatment of a variety of hematological disorders. Interestingly, during that period of time other cell types with therapeutic potential-including certain lymphoid populations and other nonhematopoietic cells-were discovered and characterized; thus, CT became a promising discipline for the treatment of a broader diversity of diseases. Today (2011-2023), CT has significantly grownup through preclinical studies and clinical trials, and it is currently progressing toward its consolidation as one of the pillars of medicine in the 21st century. Indeed, different types of stem cells (e.g., hematopoietic, mesenchymal, neural, and pluripotent), as well as different lymphoid and myeloid cell populations (e.g., TILs, CAR-Ts, CAR-NKs, and DUOC-01) are being used in clinical settings or are being tested in clinical trials. For the past decade, several CT modalities have been developed, and today, many of them are being used in the clinic. Tomorrow (2024-2040), already established CT modalities will surely be improved and applied more frequently, and novel therapies (that will include cell types such as iPSCs) will enter and expand within the clinical ground. It is noteworthy, however, that despite significant advancements and achievements, problems still need to be solved and obstacles need to be overcome. Technical, ethical, and economic issues persist and they need to be addressed. Undoubtedly, exciting times of challenges and opportunities are coming ahead in the CT arena.

01 Jun 2017·CytotherapyQ3 · MEDICINE

Gene products promoting remyelination are up-regulated in a cell therapy product manufactured from banked human cord blood

Q3 · MEDICINE

Article

Author: Noeldner, Pamela ; Saha, Arjun ; Kurtzberg, Joanne ; Buntz, Susan ; Balber, Andrew E ; Scotland, Paula ; Gentry, Tracy

BACKGROUND AIMSDUOC-01, a cell product being developed to treat demyelinating conditions, is composed of macrophages that arise from CD14⁺ monocytes in the mononuclear cell (MNC) population of banked cord blood (CB). This article demonstrates that expression of multiple gene products that promote remyelination is rapidly up-regulated during manufacturing of DUOC-01 from either MNC or purified CB CD14⁺ monocytes.METHODSCell cultures were initiated with MNC or with immunoselected CD14⁺ monocytes isolated from the same CB unit. Cell products present in these cultures after 2 and 3 weeks were compared by three methods. First, quantitative polymerase chain reaction was used to compare expression of 77 transcripts previously shown to be differentially expressed by freshly isolated, uncultured CB CD14⁺ monocytes and DUOC-01. Second, accumulation of 16 soluble proteins in the culture medium was measured by Bioplex methods. Third, whole transcriptomes of the cell products were compared by microarray analysis.RESULTSKey transcripts in multiple pathways that promote remyelination were up-regulated in DUOC-01, and substantial secretion of proteins corresponding to many of these transcripts was detected. Cell products manufactured from MNC or from CD14⁺ monocytes were similar with regard to all metrics. Upregulation of gene products characteristic of DUOC-01 was largely completed within 14 days of culture.CONCLUSIONWe demonstrate that expression of multiple gene products that promote remyelination is up-regulated during the first 2 weeks of manufacturing of DUOC-01. Measuring these mechanistically important transcripts and proteins will be useful in monitoring manufacturing, evaluating manufacturing changes, and developing mechanism-based product potency assays.

100 Deals associated with DUOC-01

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Adrenoleukodystrophy	Phase 1	United States	Duke University	01 Sep 2014
Alpha-Mannosidosis	Phase 1	United States	Duke University	01 Sep 2014
Leukodystrophy, Globoid Cell	Phase 1	United States	Duke University	01 Sep 2014
Leukodystrophy, Metachromatic	Phase 1	United States	Duke University	01 Sep 2014
Mucopolysaccharidosis II	Phase 1	United States	Duke University	01 Sep 2014
Mucopolysaccharidosis III	Phase 1	United States	Duke University	01 Sep 2014
Niemann-Pick Diseases	Phase 1	United States	Duke University	01 Sep 2014
Pelizaeus-Merzbacher Disease	Phase 1	United States	Duke University	01 Sep 2014
Sandhoff Disease	Phase 1	United States	Duke University	01 Sep 2014
Tay-Sachs Disease	Phase 1	United States	Duke University	01 Sep 2014