BlueRock Therapeutics Receives FDA Fast Track Designation for DA01 in the Treatment of Advanced Parkinson’s Disease

BlueRock Therapeutics LP announced that the US FDA has granted Fast Track designation for DA01 for advanced Parkinson’s disease. DA01, BlueRock’s PSC-derived dopaminergic neuron therapy, is under evaluation in a Phase I study.
[BlueRock Therapeutics LP]
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BlueRock Therapeutics Receives FDA Fast Track Designation for DA01 in the Treatment of Advanced Parkinson’s Disease

BlueRock Therapeutics LP announced that the US FDA has granted Fast Track designation for DA01 for advanced Parkinson’s disease. DA01, BlueRock’s PSC-derived dopaminergic neuron therapy, is under evaluation in a Phase I study.
[BlueRock Therapeutics LP]
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Analysis of Extracellular Vesicles as a Potential Index for Monitoring Differentiation of Neural Lineage Cells from Induced Pluripotent Stem Cells

Researchers evaluated changes in extracellular vesicles morphology, concentration, and miRNA and protein expression in culture supernatants during the differentiation of iPSCs into neural lineage cells, for application in regenerative medicine for Parkinson’s disease.
[Journal of Bioscience and Bioengineering]
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Identification of Bioactive Metabolites in Human iPSC-Derived Dopaminergic Neurons with PARK2 Mutation: Altered Mitochondrial and Energy Metabolism

Scientists used isogenic human induced pluripotent stem cells (iPSCs) with and without PARK2 knockout to investigate the effect of parkin loss of function by comparative metabolomics supplemented with ultrastructural and functional analyses.
[Stem Cell Reports]
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First in the Nation, FDA-Approved Phase II Mesenchymal Stem Cell Therapy for Parkinson’s Disease Begins

A Phase II clinical trial to assess mesenchymal adult stem cells as a disease-modifying therapy for Parkinson’s disease has been launched at The University of Texas Health Science Center at Houston
[The University of Texas Health Science Center at Houston (Newswise, Inc.)]
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MedRhythms Launches Clinical Trial of Digital Therapeutic in Parkinson’s Disease and Expands Pipeline to Aging

MedRhythms has announced the initiation of a multi-site clinical trial, in collaboration with Boston University, of its pipeline asset MR-005 in Parkinson’s disease as well as the expansion of the company’s pipeline into aging with the development of its Aging Scientific Advisory Board.
[MedRhythms (PR Newswire, LLC)]
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Sex Differences in the Blood–Brain Barrier and Neurodegenerative Diseases

The authors describe sex-related differences in neurodegenerative diseases and the blood–brain barrier, whose dysfunction is linked to neurodegenerative disease development and progression.
[APL Bioengineering]
Weber, C. M., & Clyne, A. M. (2021). Sex differences in the blood–brain barrier and neurodegenerative diseases. APL Bioengineering, 5(1), 011509. https://doi.org/10.1063/5.0035610 Cite
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Monitoring the Neurotransmitter Release of Human Midbrain Organoids Using a Redox Cycling Microsensor as a Novel Tool for Personalized Parkinson’s Disease Modelling and Drug Screening†

Scientists developed a novel electrochemical sensing approach capable of detecting dopamine, the main biomarker in Parkinson’s disease, within the highly complex cell culture matrix of human midbrain organoids in a non-invasive and label-free manner.
[Analyst]
Zanetti, C., Spitz, S., Berger, E., Bolognin, S., Smits, L. M., Crepaz, P., Rothbauer, M., Rosser, J. M., Marchetti-Deschmann, M., Schwamborn, J. C., & Ertl, P. (2021). Monitoring the neurotransmitter release of human midbrain organoids using a redox cycling microsensor as a novel tool for personalized Parkinson’s disease modelling and drug screening. Analyst. https://doi.org/10.1039/D0AN02206C Cite
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Alterity Announces Funding from Michael J. Fox Foundation for ATH434 Dose Optimization for Parkinson’s Disease Clinical Trials

Alterity Therapeutics, Ltd. announced the award of a grant from The Michael J. Fox Foundation for Parkinson’s Research to determine optimal dosing of its lead drug candidate ATH434 for Parkinson’s disease based on imaging of brain iron.
[Alterity Therapeutics, Ltd.]
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BlueRock Therapeutics in Collaboration with Memorial Sloan Kettering Cancer Center Receives IND Clearance for DA01 in Parkinson’s Disease

BlueRock Therapeutics, in collaboration with Memorial Sloan Kettering Cancer Center, announced that the US FDA has cleared their Investigational New Drug application to proceed with a Phase I study in patients with advanced Parkinson’s disease (PD). This is the first trial in the US to study pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease.
[BlueRock Therapeutics (PR Newswire, Inc.)]

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Identification of Potential Transcription Factors that Enhance Human iPSC Generation

Using an RNA-seq platform, scientists identified and validated the differential gene expression of five transcription factors that were associated with a remarkable increase in the number of iPSC colonies generated from a patient with Parkinson’s disease.
[Scientific Reports]

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