Transcytosis

Voyager Therapeutics Presents Data for Second-Generation, TRACER™-Generated Capsids and CNS Gene Therapy Programs Advancing Toward Clinical Trials at the ASGCT 27th Annual Meeting

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Mercoledì, Maggio 8, 2024

LEXINGTON, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the presentation of data related to its TRACER™ capsid discovery platform and TRACER-driven gene therapy programs at the American Society of Gene & Cell Therapy’s (ASGCT) 27th annual meeting.

Key Points: 
  • Voyager identified a highly conserved cell surface receptor that mediates enhanced brain tropism of the VCAP-101/102 engineered capsid class.
  • Voyager has advanced machine learning to predict production fitness of capsid variants with high accuracy, helping guide development of high-production-fit libraries.
  • Robust manufacturing processes are needed to remove empty and partially filled capsids from recombinant AAV (rAAV) drug product.
  • This cost-efficient cell line provides an AAV expression platform to produce drug candidates for neurological disorders.

Affinia Therapeutics to Present Data on Novel Cardiotropic and BBB-Penetrant AAV Capsids and Preclinical Efficacy and Safety in Genetic Cardiomyopathies and Sporadic ALS at the American Society of Gene & Cell Therapy 2024 Annual Meeting

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Lunedì, Aprile 22, 2024

Affinia's novel capsids have favorable manufacturing yields and levels of preexisting population immunity.

Key Points: 
  • Affinia's novel capsids have favorable manufacturing yields and levels of preexisting population immunity.
  • "We are excited to unveil data on our next-generation bespoke capsids that demonstrate robust cardiac transduction while detargeting the liver and DRG.
  • One oral presentation showcases data from Affinia's novel, BBB-penetrant AAV capsid with a potential therapeutic application to sporadic ALS.
  • The first poster showcases data that confirm the novel cardiotropic AAV capsid's safety and therapeutic efficacy in an animal model of cardiac dysfunction with a potential therapeutic application in genetic cardiomyopathies.

Novel Diagnostic Imaging Platform to Monitor the Progression and Treatment of Metastatic Cancers presented via Hidden GEMS Program from One Million Solutions in Health

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Giovedì, Giugno 24, 2021

Dr. Stephan T. Stern shares his work in this scientific presentation and licensing opportunity review which is spearheaded by One Million Solutions in Health.

Key Points: 
  • Dr. Stephan T. Stern shares his work in this scientific presentation and licensing opportunity review which is spearheaded by One Million Solutions in Health.
  • This licensing opportunity introduces a novel technology polymeric delivery platform that targets the lymphatic system through scavenger receptor A1 (SR-A1) mediated transcytosis.
  • In this next year through the Hidden GEMS Program, we will be reviewing 100's of new solutions and new scientific discoveries.
  • Through the Hidden GEMS Program, the Technology Evaluation Consortium from One Million Solutions in Health is dedicated to improving and accelerating life sciences R&D and health care outcomes.

Novel Diagnostic Imaging Platform to Monitor the Progression and Treatment of Metastatic Cancers presented via Hidden GEMS Program from One Million Solutions in Health

Retrieved on: 
Lunedì, Febbraio 1, 2021

Dr. Stephan T. Stern shares his work in this scientific presentation and licensing opportunity review which is spearheaded by One Million Solutions in Health .

Key Points: 
  • Dr. Stephan T. Stern shares his work in this scientific presentation and licensing opportunity review which is spearheaded by One Million Solutions in Health .
  • This licensing opportunity introduces a novel technology polymeric delivery platform that targets the lymphatic system through scavenger receptor A1 (SR-A1) mediated transcytosis.
  • In this next year through the Hidden GEMS Program , we will be reviewing 100's of new solutions and new scientific discoveries.
  • Through the Hidden GEMS Program , the Technology Evaluation Consortium from One Million Solutions in Health is dedicated to improving and accelerating life sciences R&D and health care outcomes.

Applied Molecular Transport Announces Publication of Preclinical Data Demonstrating Potential of Novel Oral IL-10 Biologic Therapeutic (AMT-101) for Inflammatory Diseases in The Journal of Immunology

Retrieved on: 
Martedì, Novembre 10, 2020

IL-10 is a potent immunomodulatory cytokine with significant therapeutic potential in intestinal inflammatory diseases as well as in those associated with systemic inflammation.

Key Points: 
  • IL-10 is a potent immunomodulatory cytokine with significant therapeutic potential in intestinal inflammatory diseases as well as in those associated with systemic inflammation.
  • However, clinical utilization of IL-10 to treat inflammation and immune-dysregulation has been limited due to side effects associated with systemic administration.
  • Our technology platform is based on native, active vesicular transcytosis mechanisms to rapidly and efficiently traverse intestinal epithelial cells.
  • Applied Molecular Transport Inc. is a clinical-stage biopharmaceutical company leveraging its proprietary technology platform to design and develop a pipeline of novel oral biologic product candidates to treat autoimmune, inflammatory, metabolic, and other diseases.

Ossianix to Present Preclinical Data Demonstrating Best-in-Class Blood Brain Barrier Delivery in Non-Human Primates by Single Domain VNAR Antibodies

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Lunedì, Novembre 9, 2020

PHILADELPHIA and STEVENAGE, United Kingdom, Nov. 9, 2020 /PRNewswire/ --Ossianix, an antibody engineering company, todayannounced the results of a non-human primate study demonstrating best-in-class blood-brain barrier (BBB) delivery by single domain antibodies.

Key Points: 
  • PHILADELPHIA and STEVENAGE, United Kingdom, Nov. 9, 2020 /PRNewswire/ --Ossianix, an antibody engineering company, todayannounced the results of a non-human primate study demonstrating best-in-class blood-brain barrier (BBB) delivery by single domain antibodies.
  • Poor brain delivery is a major barrier in the development of biological therapeutics for neurologic diseases because they are too large to cross the BBB.
  • The VNAR shuttle is a single domain antibody derived from sharks that is significantly smaller than monoclonal antibodies used today as therapeutics.
  • Ossianix is an antibody engineering companythat utilizes single domain antibodies (VNAR) from the shark to develop novel biopharmaceuticals for a number of therapeutic areas including CNS.

Unlocking the Blood-Brain-Barrier: A new simple and innovative way of delivering therapeutics to brain

Retrieved on: 
Martedì, Ottobre 15, 2019

Scientists have traditionally faced the problem that blood capillaries in the brain are not permeable to many drugs.

Key Points: 
  • Scientists have traditionally faced the problem that blood capillaries in the brain are not permeable to many drugs.
  • For example, currently, one form of delivery to the brain is to use modified viruses to bypass the BBB and deliver medication to the brain.
  • The new technological breakthrough for delivering genetic materials (as well as other therapeutics) to cells in the brain uses a modified peptide identified from a virus that targeted the brain capillaries.
  • The platform technology allows for minimally invasive and combination delivery of various drugs, peptides, and nucleic acid therapeutics to the brain and other organs.

BioArctic Strengthens its Blood-brain Barrier Technology Platform With World Leading Scientist Recruitment

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Giovedì, Maggio 23, 2019

Through the recruitment of Dr. Freskgrd, BioArctic further strengthens its capabilities in the neuroscience therapeutic area, antibody engineering and blood-brain barrier competence.

Key Points: 
  • Through the recruitment of Dr. Freskgrd, BioArctic further strengthens its capabilities in the neuroscience therapeutic area, antibody engineering and blood-brain barrier competence.
  • Today, Dr. Freskgrd is one of the world's leading experts on mechanisms that increase the passage of biologic drugs across the blood-brain barrier.
  • In collaboration with Uppsala University, BioArctic is developing novel technologies to increase the passage of antibodies over the blood-brain barrier.
  • "That Per-Ola Freskgrd, a world leading scientist on technologies to facilitate the passage of biologics across the blood-brain barrier, now has chosen to join BioArctic is a recognition of the high quality research at BioArctic.

Global Gene Therapy Companies, Technologies, & Markets, 2018-2019 & 2028: Focus on Genetic Disorders, Cancer, Neurological Disorders, Cardiovascular Disorders, & Viral Infections

Retrieved on: 
Martedì, Maggio 21, 2019

The delivery of drugs to central nervous system (CNS) is a challenge in the treatment of neurological disorders.

Key Points: 
  • The delivery of drugs to central nervous system (CNS) is a challenge in the treatment of neurological disorders.
  • The major challenge to CNS drug delivery is the blood-brain barrier (BBB), which limits the access of drugs to the brain substance.
  • Advances in understanding of the cell biology of the BBB have opened new avenues and possibilities for improved drug delivery to the CNS.
  • A number of cell and gene therapy companies with products in development for CNS disorders are included.