Autophagy

Coave Therapeutics to Collaborate with World-Renowned Institute of Neurodegenerative Diseases of Bordeaux to Develop Gene Therapy Programs Targeting Protein Degradation in Neurodegenerative Disorders

Retrieved on: 
Wednesday, September 14, 2022

Overexpression of TFEB via gene therapy demonstrates potential to reduce and prevent the accumulation of toxic protein aggregates1 and to consequently prevent neurodegeneration.

Key Points: 
  • Overexpression of TFEB via gene therapy demonstrates potential to reduce and prevent the accumulation of toxic protein aggregates1 and to consequently prevent neurodegeneration.
  • We look forward to leveraging our collective strengths to best develop gene therapy programs for neurodegenerative diseases with the potential to improve patient outcomes."
  • "We are delighted to be collaborating with IMN to develop coAAVs carrying TFEB and explore these gene therapy constructs for the treatment of neurodegenerative diseases.
  • Coave Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases.

FDA Grants GENFIT’s GNS561 Orphan Drug Designation for the Treatment of Cholangiocarcinoma

Retrieved on: 
Tuesday, September 13, 2022

GNS561 has completed pre-clinical studies and a Phase 1b trial confirming the rationale for targeting cholangiocarcinoma.

Key Points: 
  • GNS561 has completed pre-clinical studies and a Phase 1b trial confirming the rationale for targeting cholangiocarcinoma.
  • Dr Mark Yarchoan, Associate Professor of Oncology at John Hopkins Medicine (Baltimore, MD) commented: Cholangiocarcinoma is a rare cancer with a high mortality rate.
  • This is why new therapies are urgently needed and is one of the reasons that GNS561 was granted Orphan Drug Designation by the FDA.
  • There is a real path forward for new options for second line treatment in cholangiocarcinoma, and GNS561 represents a strong second-line therapy candidate and hope to patients.

BioMed Valley Discoveries Announces First Patient Dosed in Phase II Combination Trial with Ulixertinib (BVD-523), its First-in-Class and Best-in-Class ERK Inhibitor, in Combination with Hydroxychloroquine

Retrieved on: 
Monday, September 12, 2022

KANSAS CITY, Mo., Sept. 12, 2022 /PRNewswire/ -- BioMed Valley Discoveries (BVD) announced that the first patient has been dosed in a phase II clinical trial of ulixertinib (BVD-523) in combination with hydroxychloroquine (HCQ).

Key Points: 
  • KANSAS CITY, Mo., Sept. 12, 2022 /PRNewswire/ -- BioMed Valley Discoveries (BVD) announced that the first patient has been dosed in a phase II clinical trial of ulixertinib (BVD-523) in combination with hydroxychloroquine (HCQ).
  • Ulixertinib is a first-in-class and best-in-class ERK inhibitor, with this clinical trial focusing on patients with advanced gastrointestinal malignancies and mutations in the MAPK pathway.
  • Previous efforts have also established a recommended phase 2 dose in combination with palbociclib, with additional combination efforts ongoing.
  • About BioMed Valley Discoveries (BVD): BioMed Valley Discoveries is a clinical stage biotechnology company focused on addressing unmet medical needs in a variety of therapeutic and diagnostic areas.

Deciphera Pharmaceuticals, Inc. Presents Initial Phase 1 Single Agent Dose Escalation Data for First-in-Class ULK Inhibitor of Autophagy, DCC-3116, at the European Society for Medical Oncology (ESMO) Congress 2022

Retrieved on: 
Saturday, September 10, 2022

As the first ULK1/2 inhibitor to enter clinical development, these positive initial results represent a significant milestone as we prepare to initiate combination dose escalation later this year.

Key Points: 
  • As the first ULK1/2 inhibitor to enter clinical development, these positive initial results represent a significant milestone as we prepare to initiate combination dose escalation later this year.
  • The preliminary data show DCC-3116 to be a very well-tolerated agent that has demonstrated strong target inhibition of ULK 1/2 from even the lowest tested dose.
  • I look forward to the selection of the combination starting dose and advancing the program into the first combination studies with MEK and KRASG12C inhibitors.
  • Deciphera, the Deciphera logo, QINLOCK, and the QINLOCK logo are registered trademarks of Deciphera Pharmaceuticals, LLC.

Juntendo University research: Itchy business: Investigating a cure for eczema

Retrieved on: 
Wednesday, September 7, 2022

Now, a team of researchers from Juntendo University, led by Franois Niyonsaba, has revealed a candidate called human--defensin-3 (hBD-3) with potential in this regard.

Key Points: 
  • Now, a team of researchers from Juntendo University, led by Franois Niyonsaba, has revealed a candidate called human--defensin-3 (hBD-3) with potential in this regard.
  • One of the characteristics of AD is the disruption of the outermost layer of the skin: the epidermal barrier.
  • Inside the cells that line this barrier, a mechanism called autophagy is involved in their differentiation and antimicrobial activity.
  • Using the skin of human patients and mice with AD, the state of autophagy in affected cells was first analyzed.

Juntendo University research: Itchy business: Investigating a cure for eczema

Retrieved on: 
Wednesday, September 7, 2022

Now, a team of researchers from Juntendo University, led by Franois Niyonsaba, has revealed a candidate called human--defensin-3 (hBD-3) with potential in this regard.

Key Points: 
  • Now, a team of researchers from Juntendo University, led by Franois Niyonsaba, has revealed a candidate called human--defensin-3 (hBD-3) with potential in this regard.
  • One of the characteristics of AD is the disruption of the outermost layer of the skin: the epidermal barrier.
  • Inside the cells that line this barrier, a mechanism called autophagy is involved in their differentiation and antimicrobial activity.
  • Using the skin of human patients and mice with AD, the state of autophagy in affected cells was first analyzed.

Anavex Life Sciences Reports New Publication in Medical Journal Highlighting the Relevance of SIGMAR1 Activation to Compensate for Early Alzheimer's Disease

Retrieved on: 
Tuesday, August 23, 2022

NEW YORK, Aug. 23, 2022 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer's disease, Parkinson's disease, Rett syndrome, and other central nervous system (CNS) diseases, today reported a relevant new peer-reviewed publication in the journal Science Translational Medicine, titled "Widespread cell stress and mitochondrial dysfunction occur in patients with early Alzheimer's disease."1

Key Points: 
  • "1
    One of the featured observations was an increase in SIGMAR1 expression in the early stages of AD.
  • Combinatorial therapy has been suggested as a treatment strategy; however, the existence of drug-drug interaction is a concern.
  • Hence, there is a need to develop drug molecules that can target multiple pathways to halt disease progression and improve memory function.
  • Anavex Life Sciences' precision medicine platform includes small molecule drug lead candidate ANAVEX2-73 for the treatment of Alzheimer's disease, Parkinson's disease, and Rett syndrome and ANAVEX3-71 for schizophrenia, frontotemporal dementia, and Alzheimer's disease.

Publication in Nature Molecular Psychiatry Supports Genuv’s Alternate Theory for Treatment of Alzheimer’s Disease

Retrieved on: 
Thursday, August 18, 2022

The article presents findings from Genuvs preclinical experiments treating a mouse model of Alzheimers with SNR1611, a commercially available MEK 1/2 inhibitor.

Key Points: 
  • The article presents findings from Genuvs preclinical experiments treating a mouse model of Alzheimers with SNR1611, a commercially available MEK 1/2 inhibitor.
  • All research relevant to the publication was conducted in Genuvs laboratories or under Genuvs supervision.
  • The new publication in Molecular Psychiatry comes just weeks after an investigation in the journal Science uncovered evidence of potential data fabrication in a paper on Alzheimers disease published in 2006.
  • The publication has intensified interest in alternative approaches to treating Alzheimers disease, including Genuvs novel neuroprotection/neurogenesis hypothesis.

Cognition Therapeutics Presents New Proteomic Data on Effect of CT1812 Treatment on Normalization of Disrupted Alzheimer’s Disease Processes

Retrieved on: 
Thursday, August 4, 2022

The analytic results support the proposed synaptoprotective mechanism of action of CT1812 and role in normalizing cellular processes known to be adversely disrupted in Alzheimer's disease .

Key Points: 
  • The analytic results support the proposed synaptoprotective mechanism of action of CT1812 and role in normalizing cellular processes known to be adversely disrupted in Alzheimer's disease .
  • The analyses demonstrated the effect of CT1812 on multiple priority Alzheimers biomarkers, including YKL-40, a biomarker of inflammation, which is upregulated in Alzheimers disease and is subject of intense focus by the field.
  • Participants treated with CT1812 exhibited a downward shift in YKL-40 towards levels observed in healthy, non-demented individuals, supporting a positive impact of CT1812 on disease biology.
  • This damage to sensitive synapses can progress to a loss of synaptic function, which manifests as cognitive impairment and Alzheimers disease progression.

Novel Carbohydrate Antiviral Drug Candidate Acts Through Galectin Inhibition to Block SARS-CoV-2 Coronavirus

Retrieved on: 
Monday, August 1, 2022

BOSTON, MASSACHUSETTS, Aug. 01, 2022 (GLOBE NEWSWIRE) -- BIOXYTRAN, INC. (Symbol: BIXT) (the “Company”), a clinical stage biotechnology company developing oral drugs to treat COVID-19 and other viral causing diseases announced today that the journal of International Journal of Health Sciences1 released a peer-reviewed article, “Carbohydrate ProLectin-M, a Galectin-3 Antagonist, Blocks SARS-CoV-2 Activity”, that supports ProLectin-M’s in vitro Mode of Action and the initial clinical data results reported in a previous article, referred to below. ProLectin-M is the Company’s leading drug molecule in its pipeline to treat viral infections.

Key Points: 
  • ProLectin-M is the Companys leading drug molecule in its pipeline to treat viral infections.
  • The journal article begins to outline and further define the mechanism of action (MOA) behind the oral galectin inhibitor Prolectin-M.
  • The leading drug candidate, Prolectin-M, is a new class of antiviral drug designed to antagonize galectins implicated in inflammatory, fibrotic, and malignant diseases and bind to the conserved region of the spike protein commonly known as the galectin fold.
  • ProLectin-M is an orally administered experimental new drug candidate that targets the Carbohydrate Binding Domain portion on the SARS-CoV-2, coronavirus.