Anavex Life Sciences Announces Notice of Allowance for US Patent Application with Expanding Claims Covering ANAVEX®2-73 (blarcamesine) for the Treatment of Rett Syndrome and other Neurodevelopmental Disorders

Anavex Life Sciences Corp. announced it received a Notice of Allowance from the United States Patent and Trademark Office for its patent application number 16/717,921 expanding coverage of treatment methods for ANAVEX®2-73 and related sigma-1 receptor agonist drug candidates, for treating a range neurodevelopmental disorders including Rett syndrome, autism spectrum disorder, Angelman syndrome, cerebral palsy and multiple sclerosis, among other indications.
[Anavex Life Sciences Corp.]
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Athira Pharma Announces Initiation of Patient Dosing for ACT-AD Clinical Trial of ATH-1017, Small Molecule HGF/MET Activator, for Treatment of Mild-to-Moderate Alzheimer’s Disease

Athira Pharma, Inc. announced that patient dosing has begun in ACT-AD, a Phase II randomized, placebo-controlled study of ATH-1017 in patients with mild-to-moderate Alzheimer’s disease. ATH-1017 is a small molecule therapeutic designed to enhance the activity of Hepatocyte Growth Factor (HGF) and its receptor, MET, which are expressed in the central nervous system, in order to promote brain health and function.
[Athira Pharma, Inc.]
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Istari Oncology Announces First Patient Dosed in the LUMINOS-101 Phase II Clinical Trial of PVSRIPO in Combination with Pembrolizumab for Patients with Recurrent Glioblastoma

Istari Oncology, Inc. announced the first patient was dosed in the LUMINOS-101 Phase II clinical trial, assessing the safety and efficacy of PVSRIPO in combination with the immune checkpoint inhibitor pembrolizumab (Keytruda®) in patients with recurrent glioblastoma multiforme.
[Istari Oncology, Inc.]
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18F-Meta-Fluorobenzylguanidine (18F-mFBG) to Monitor Changes in Norepinephrine Transporter Expression in Response to Therapeutic Intervention in Neuroblastoma Models

Researchers investigated the potential of 18F-mFBG, a positron emission tomography analogue of the 123I-mIBG radiotracer, to quantify norepinephrine transporter expression levels in mouse models of neuroblastoma following treatment with AZD2014, a dual mTOR inhibitor.
[Scientific Reports]
Turnock, S., Turton, D. R., Martins, C. D., Chesler, L., Wilson, T. C., Gouverneur, V., Smith, G., & Kramer-Marek, G. (2020). 18 F-meta-fluorobenzylguanidine ( 18 F-mFBG) to monitor changes in norepinephrine transporter expression in response to therapeutic intervention in neuroblastoma models. Scientific Reports, 10(1), 20918. https://doi.org/10.1038/s41598-020-77788-3 Cite
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Autism-Associated miR-873 Regulates ARID1B, SHANK3 and NRXN2 Involved in Neurodevelopment

A dual-luciferase reporter assay confirmed miR-873 variants have a 20-30% inhibition/dysregulation effect on candidate autism risk genes ARID1B, SHANK3 and NRXN2 and also confirmed the affected expression with qPCR.
[Translational Psychiatry]
Lu, J., Zhu, Y., Williams, S., Watts, M., Tonta, M. A., Coleman, H. A., Parkington, H. C., & Claudianos, C. (2020). Autism-associated miR-873 regulates ARID1B, SHANK3 and NRXN2 involved in neurodevelopment. Translational Psychiatry, 10(1), 1–12. https://doi.org/10.1038/s41398-020-01106-8 Cite
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Rare Variant Burden Analysis within Enhancers Identifies CAV1 as an ALS Risk Gene

Investigators discovered enhancer mutations reduced CAV1/CAV2 expression and disrupted membrane lipid rafts in patient-derived cells, and CRISPR-Cas9 perturbation proximate to a patient mutation was sufficient to reduce CAV1/CAV2 expression in neurons.
[Cell Reports]
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Gain Therapeutics and University of Maryland School of Medicine Announce Research Collaboration

Gain Therapeutics, Inc. announced a research collaboration with the University of Maryland School of Medicine (UMSOM), to investigate Gain’s structurally targeted allosteric regulators (STARs) in cellular models of neuronopathic Gaucher disease and Parkinson’s disease. STARs are proprietary small molecules targeting novel allosteric binding sites on enzymes.
[Gain Therapeutics, Inc. (Globe Newswire, Inc.)]
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A Comprehensive Library of Human Transcription Factors for Cell Fate Engineering

Scientists demonstrated orthogonal programming by including oligodendrocyte-inducible hPSCs with unmodified hPSCs to generate cerebral organoids, which expedited in situ myelination.
[Nature Biotechnology]
Ng, A. H. M., Khoshakhlagh, P., Rojo Arias, J. E., Pasquini, G., Wang, K., Swiersy, A., Shipman, S. L., Appleton, E., Kiaee, K., Kohman, R. E., Vernet, A., Dysart, M., Leeper, K., Saylor, W., Huang, J. Y., Graveline, A., Taipale, J., Hill, D. E., Vidal, M., … Church, G. M. (2020). A comprehensive library of human transcription factors for cell fate engineering. Nature Biotechnology, 1–10. https://doi.org/10.1038/s41587-020-0742-6 Cite
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Single Cell RNA Sequencing of Human Microglia Uncovers a Subset Associated with Alzheimer’s Disease

Scientists investigated the population structure of live microglia purified from human cerebral cortex samples obtained at autopsy and during neurosurgical procedures.
[Nature Communications]
Olah, M., Menon, V., Habib, N., Taga, M. F., Ma, Y., Yung, C. J., Cimpean, M., Khairallah, A., Coronas-Samano, G., Sankowski, R., Grün, D., Kroshilina, A. A., Dionne, D., Sarkis, R. A., Cosgrove, G. R., Helgager, J., Golden, J. A., Pennell, P. B., Prinz, M., … De Jager, P. L. (2020). Single cell RNA sequencing of human microglia uncovers a subset associated with Alzheimer’s disease. Nature Communications, 11(1), 6129. https://doi.org/10.1038/s41467-020-19737-2 Cite
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Chronic Neuronal Activation Increases Dynamic Microtubules to Enhance Functional Axon Regeneration after Dorsal Root Crush Injury

Researchers found that chemogenetic activation of adult dorsal root ganglion neurons improves axon growth on an in vitro model of the inhibitory environment after injury.
[Nature Communications]
Wu, D., Jin, Y., Shapiro, T. M., Hinduja, A., Baas, P. W., & Tom, V. J. (2020). Chronic neuronal activation increases dynamic microtubules to enhance functional axon regeneration after dorsal root crush injury. Nature Communications, 11(1), 6131. https://doi.org/10.1038/s41467-020-19914-3 Cite
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Treatment Strategies for Breast Cancer Brain Metastases

The authors provide an overview of the therapeutic option for brain metastases from breast cancer that is currently available and outlines potential new approaches for tackling these deadly secondary tumors.
[British Journal of Cancer]
Olah, M., Menon, V., Habib, N., Taga, M. F., Ma, Y., Yung, C. J., Cimpean, M., Khairallah, A., Coronas-Samano, G., Sankowski, R., Grün, D., Kroshilina, A. A., Dionne, D., Sarkis, R. A., Cosgrove, G. R., Helgager, J., Golden, J. A., Pennell, P. B., Prinz, M., … De Jager, P. L. (2020). Single cell RNA sequencing of human microglia uncovers a subset associated with Alzheimer’s disease. Nature Communications, 11(1), 6129. https://doi.org/10.1038/s41467-020-19737-2 Cite
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