Cytoplasm

Gennao Bio Debuts Preclinical Data for First-in-Class Antibody-Drug Conjugate from Gene Monoclonal Antibody Platform (GMAB ADC)

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Tuesday, April 9, 2024

Gennao Bio , a privately held genetic medicines company developing first-in-class, targeted nucleic acid therapeutics, today announced new preclinical results on the application of its non-viral, cell penetrating gene monoclonal antibody (GMAB) platform technology as an antibody-drug conjugate (ADC) for the treatment of solid tumors.

Key Points: 
  • Gennao Bio , a privately held genetic medicines company developing first-in-class, targeted nucleic acid therapeutics, today announced new preclinical results on the application of its non-viral, cell penetrating gene monoclonal antibody (GMAB) platform technology as an antibody-drug conjugate (ADC) for the treatment of solid tumors.
  • The data were presented in a poster at the American Association for Cancer Research (AACR) Annual Meeting 2024.
  • In preclinical studies, our GMAB technology demonstrated selective delivery of payloads into tumors by targeting ENT2, a nucleoside transporter that is highly overexpressed in many tumors.
  • “These preclinical results reinforce the versatility of the GMAB platform and its ability to deliver therapeutic payloads beyond genetic medicine to targeted tissue,” Chris Duke, chief executive officer of Gennao.

Making Long-Term Memories Requires Nerve-Cell Damage

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Wednesday, March 27, 2024

"But our findings suggest that inflammation in certain neurons in the brain's hippocampal region is essential for making long-lasting memories."

Key Points: 
  • "But our findings suggest that inflammation in certain neurons in the brain's hippocampal region is essential for making long-lasting memories."
  • But looking more closely, we found, to our surprise, that TLR9 was activated only in clusters of hippocampal cells that showed DNA damage."
  • But in this population of hippocampal neurons, the DNA damage appeared to be more substantial and sustained.
  • Importantly, the researchers found that blocking the TLR9 inflammatory pathway in hippocampal neurons not only prevented mice from forming long-term memories but also caused profound genomic instability, i.e, a high frequency of DNA damage in these neurons.

Altamira Therapeutics’ Peptide-Based Delivery Platform Shown to Enhance Potency of Commonly Used Gene Delivery Method as Published in Peer-Reviewed Journal

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Wednesday, February 7, 2024

HAMILTON, BERMUDA, Feb. 7, 2024 -- Altamira Therapeutics Ltd. (Nasdaq: CYTO) ("Altamira" or the "Company"), a company providing nanoparticle-based technology for efficient RNA delivery to extrahepatic targets, announced today the publication of a peer-reviewed article in the Journal of Integrative Medicine titled, "Melittin analog p5RHH enhances recombinant adeno-associated virus transduction efficiency". This work evaluates the use of various peptides to enhance adeno-associated virus (AAV) cell transduction and was conducted by an independent research group.1 Recombinant AAVs are commonly used as carriers to introduce nucleic acids in cells for gene therapy; several AAV-based gene therapy drugs have already been approved by the U.S. Food and Drug Administration (FDA).

Key Points: 
  • The study sought to find ways of increasing the endosomal release of AAV-based therapeutics by using peptides derived from melittin, a component of bee venom known for its ability to permeabilize biological membranes.
  • The research group evaluated 76 melittin derivatives, including p5RHH, the peptide underlying Altamira’s OligoPhore™ / SemaPhore™ nanoparticle platform for RNA delivery.
  • The scientists discovered that insertion of p5RHH into the AAV vector (p5RHH-rAAV) not only enhanced cell transduction, but also succeeded in transducing cell lines typically considered resistant to AAVs.
  • “Better transduction efficiency means that lower doses of AAVs may be used, which could lower the risk for deleterious immune responses and increase the safety of AAV-based vectors.

Windtree Renews Agreement with Chang Gung University for Scientific Collaboration to Further SERCA2a Research

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Thursday, February 1, 2024

The scientific collaboration includes the Company’s lead product candidate istaroxime and the next generation compounds called SERCA2a activators.

Key Points: 
  • The scientific collaboration includes the Company’s lead product candidate istaroxime and the next generation compounds called SERCA2a activators.
  • SERCA2a activity is decreased in heart failure and disordered calcium handling can play a role in cardiac arrythmias.
  • Windtree believes activation of SERCA2a could represent an important advancement in heart failure treatment for patients.
  • Outside of this scientific collaboration, istaroxime is being studied in the Phase 2 SEISMiC Extension Study in early cardiogenic shock.

ProMIS Neurosciences Announces Publication on Novel Target for ALS

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Wednesday, December 20, 2023

TORONTO, Ontario and CAMBRIDGE, Massachusetts, Dec. 20, 2023 (GLOBE NEWSWIRE) -- ProMIS Neurosciences Inc. (Nasdaq: PMN), a biotechnology company focused on the generation and development of antibody therapeutics targeting toxic misfolded proteins in neurodegenerative diseases such as Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA), today announced a publication identifying Receptor for Activated C-Kinase 1 (RACK1) as a novel misfolded protein target for ALS and frontotemporal lobar degeneration (FTLD). The article published in the online edition of Acta Neuropathologica Communications is titled, "Targeting RACK1 to alleviate TDP-43 and FUS proteinopathy-mediated suppression of protein translation and neurodegeneration."

Key Points: 
  • The article published in the online edition of Acta Neuropathologica Communications is titled, "Targeting RACK1 to alleviate TDP-43 and FUS proteinopathy-mediated suppression of protein translation and neurodegeneration."
  • Under normal conditions, TDP-43 and FUS are predominantly localized in the nucleus of cells, where they participate in the regulation of protein expression.
  • In disease, however, they typically become mislocalized in the cytoplasm of neurons where they form aggregates.
  • The study authors reported that pathological FUS and TDP-43 both co-aggregate with RACK1 resulting in suppression of protein synthesis.

ROME Publishes Landmark Nature Paper Revealing First High-Resolution Structure of LINE-1 Reverse Transcriptase (RT) for Drug Discovery

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Thursday, December 14, 2023

ROME Therapeutics , a biotechnology company harnessing the power of the dark genome to develop breakthrough medicines for serious diseases, today announced a landmark publication in Nature revealing the first high-resolution structure of the LINE-1 reverse transcriptase (RT).

Key Points: 
  • ROME Therapeutics , a biotechnology company harnessing the power of the dark genome to develop breakthrough medicines for serious diseases, today announced a landmark publication in Nature revealing the first high-resolution structure of the LINE-1 reverse transcriptase (RT).
  • The supporting research was co-led by ROME as part of a significant collaboration with more than a dozen leading academic groups and industry organizations.
  • View the full release here: https://www.businesswire.com/news/home/20231214251491/en/
    LINE-1 reverse transcriptase (orange) can produce RNA-DNA hybrids in the cytoplasm, a new study finds.
  • These aberrant products can trigger inflammation, and might explain how LINE-1 contributes to autoimmune disease and other disease biology.

ROME Therapeutics Presents First Data to Validate LINE-1 RT as a Novel Target in Autoimmune Diseases and the Therapeutic Potential of its First-in-Class LINE-1 RT Inhibitors

Retrieved on: 
Thursday, November 16, 2023

Data also demonstrated the therapeutic potential of the company’s first-in-class LINE-1 RT inhibitors to stop disease-driving inflammation.

Key Points: 
  • Data also demonstrated the therapeutic potential of the company’s first-in-class LINE-1 RT inhibitors to stop disease-driving inflammation.
  • Additionally, the poster highlighted the first preclinical data demonstrating the therapeutic potential of ROME’s first-in-class inhibitors of LINE-1 RT.
  • These data are the first to validate LINE-1 RT as a novel target for potential therapeutic intervention across a wide range of autoimmune diseases,” said Rosana Kapeller, M.D., Ph.D., President, CEO and Co-founder of ROME.
  • “We’re also pleased to share the first in vivo and in vitro data validating the therapeutic effect of our first-in-class LINE-1 RT inhibitors in the autoimmune disease setting.

Three Articles in the Journal of Pharmaceutical Analysis Uncover Previously Unknown Drug and Disease Mechanisms

Retrieved on: 
Friday, December 15, 2023

XI'AN, China, Dec. 15, 2023 /PRNewswire/ -- Long-term use of analgesics such as morphine leads to "tolerance" that reduces drug efficacy. In such cases, dose escalation may be required to achieve the same analgesic effects. The key molecular mechanism of morphine tolerance is its inefficiency in inducing mu-opioid receptor (MOR) endocytosis. None of the currently available strategies to counter morphine tolerance address this underlying molecular mechanism. Moreover, understanding how drugs work is crucial to determine their efficacy. There is also an unmet need for new diagnostic biomarkers that can help identify diseases at an early stage. The October issue of JPA features three articles that address these issues and systematically lay a foundation for future studies and treatment strategies.

Key Points: 
  • XI'AN, China, Dec. 15, 2023 /PRNewswire/ -- Long-term use of analgesics such as morphine leads to "tolerance" that reduces drug efficacy.
  • The October issue of JPA features three articles that address these issues and systematically lay a foundation for future studies and treatment strategies.
  • In the second study , researchers dug deeper into the anticancer mechanisms of the drug 'pracinostat,' which is a histone deacetylase inhibitor (HDACi).
  • Corresponding author Prof. Xinan Wu speculates by saying, "Our findings could aid in adjusting drug dosage or predicting renal injury via OAT channels."

SOLA Biosciences Presents Preclinical Proof-of-Concept Data for SOL-257, a Gene Therapy Targeting Misfolded TDP-43 in ALS, at Neuroscience 2023 and ALS One Symposium

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Thursday, November 9, 2023

As a pioneer in innovative chaperone technology, SOLA Biosciences Inc. will present its compelling preclinical proof-of-concept data for SOL-257, a targeted gene therapy for misfolded TDP-43 in Amyotrophic Lateral Sclerosis (ALS), at Neuroscience 2023 in Washington, D.C. on November 15 and the ALS One Symposium on November 17.

Key Points: 
  • As a pioneer in innovative chaperone technology, SOLA Biosciences Inc. will present its compelling preclinical proof-of-concept data for SOL-257, a targeted gene therapy for misfolded TDP-43 in Amyotrophic Lateral Sclerosis (ALS), at Neuroscience 2023 in Washington, D.C. on November 15 and the ALS One Symposium on November 17.
  • In vivo proof-of-concept studies have showcased the effectiveness of SOL-257 gene therapy in alleviating TDP-43-related toxicity in both the TDP-43-based mouse model and the C9orf72-based ALS mouse model.
  • “Our findings underscore SOL-257 as a highly promising translational therapy for ALS,” stated Dr. Akinori Hishiya, Chief Scientific Officer at SOLA.
  • Committed to excellence, we are advancing this promising therapy, striving to bring hope to the ALS community.”

Proteintech Genomics Launches First Commercially Available Solution for the Detection of Intracellular Proteins for Single-Cell RNAseq Experiments

Retrieved on: 
Monday, November 6, 2023

This cocktail contains 53 antibodies against intracellular and cell surface proteins, plus 5 isotype controls.

Key Points: 
  • This cocktail contains 53 antibodies against intracellular and cell surface proteins, plus 5 isotype controls.
  • The pre-titrated cocktail is demonstrated to be compatible with the 10x Genomics® Chromium® Single Cell Gene Expression Flex product.
  • We have been working diligently to provide researchers with the ability to delve beyond the cell surface and provide a high resolution view of intracellular proteins in addition to cell surface proteins and gene expression.
  • While several groups have subsequently obtained believable signal from intracellular proteins coupled with single cell gene expression, the resulting data was suboptimal.