Tropism

American Lung Association Funds $13.6 Million in Research for a Future Free of Lung Disease

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Giovedì, Ottobre 12, 2023
Havasupai Tribe v. the Arizona Board of Regents, Manning Innovation Awards, T helper cell, Immunotherapy, COVID-19, Omicron, Lung cancer, Tropism, Icahn School of Medicine at Mount Sinai, Asthma, University of Arizona, Natural killer T cell, Research, COPD, Boston University, Immunoglobulin E, University, Disease, Lung, Patient, Award, American Lung Association, CHI3L1, Canadian Lung Association, Immunology, Helminthiasis, Trustee, Research institute, Infection, SARS-CoV-2, RSV, TFH, Blood, Mount Sinai, Pharmaceutical industry, Vaccine, School

CHICAGO, Oct. 12, 2023 /PRNewswire/ -- Today, the American Lung Association Research Institute announced it awarded $13.6 million in research grants to fund 129 innovative projects to advance today's science to end the burden of lung disease tomorrow. Lung research is critical because one in 10 people in the U.S. are living with a lung disease and each year, millions of people are impacted by respiratory viruses like COVID-19 and influenza.

Key Points: 
  • Organization funds 129 promising research grants on lung cancer, asthma, COVID-19 and more
    CHICAGO, Oct. 12, 2023 /PRNewswire/ -- Today, the American Lung Association Research Institute announced it awarded $13.6 million in research grants to fund 129 innovative projects to advance today's science to end the burden of lung disease tomorrow.
  • Through the Awards and Grants Program, the Lung Association supports trailblazing research, novel ideas and innovative approaches.
  • The Lung Association's Awards and Grants Program promotes innovative research, collaboration, translation of discoveries and scientific exchange to transform today's science into tomorrow's solutions," said Harold Wimmer, President and CEO of the American Lung Association.
  • For more information about the new grant awardees and the entire American Lung Association research team, visit Lung.org/research-team .

Otsuka collaborates with ShapeTX to develop novel AAV gene therapies for ocular diseases

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Giovedì, Settembre 7, 2023
Drug discovery, Nephrology, Midjourney, Eye, Gene expression, Tropism, RNA, Antibody, Doctor of Philosophy, AAV, CNS, Patient, Machine learning, Aavida Maa Aavide, Ophthalmology, Engineering, Vivisection, Otsuka Pharmaceutical, AI, NHP, Vaccine

ShapeTX’s AI-driven AAVid platform combines massive throughput screening of billions of unique AAV variants and machine learning to identify novel AAV capsids for direct-to-NHP in vivo selection to maximize clinical translation.

Key Points: 
  • ShapeTX’s AI-driven AAVid platform combines massive throughput screening of billions of unique AAV variants and machine learning to identify novel AAV capsids for direct-to-NHP in vivo selection to maximize clinical translation.
  • As part of the collaboration, ShapeTX will also apply the company’s transgene engineering technology to optimize payloads provided by Otsuka for therapeutic levels of gene expression in targeted cell types.
  • Our recent research activities have led to identification of target molecules and antibodies for specific ocular diseases of interest with high unmet medical needs.
  • This holds the potential to become dramatically beneficial to patients who have suffered from specific, chronic ocular diseases.”

IconOVir Bio Announces First Patient Dosed in Phase 1 Clinical Trial of its Lead Product Candidate, ICVB-1042

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Martedì, Luglio 11, 2023
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Lung, Neoplasm, Cancer, Head, Breast, Safety, Patient, Tropism, Neck, Pharmaceutical industry, Medical imaging

IconOVir Bio, Inc. (IconOVir), a clinical-stage biotechnology company pioneering the next generation of oncolytic virus (OV) therapy to improve the treatment of patients with cancer, today announced that the first patient has been dosed in a Phase 1 dose escalation and expansion clinical trial evaluating intravenously (IV) administered ICVB-1042 for the treatment of advanced solid tumors.

Key Points: 
  • IconOVir Bio, Inc. (IconOVir), a clinical-stage biotechnology company pioneering the next generation of oncolytic virus (OV) therapy to improve the treatment of patients with cancer, today announced that the first patient has been dosed in a Phase 1 dose escalation and expansion clinical trial evaluating intravenously (IV) administered ICVB-1042 for the treatment of advanced solid tumors.
  • “We are delighted to begin clinical evaluation of ICVB-1042, our potentially best-in-class OV and the first product candidate to emerge from our portfolio,” said Mark McCamish, M.D., Ph.D., President and Chief Executive Officer of IconOVir.
  • “We founded IconOVir to pioneer the next generation of OVs to improve the care and treatment of people living with solid tumors, with the ultimate mission of curing cancer and restoring life to patients everywhere.
  • We look forward to reporting initial safety data later this year, with potential biological proof-of-concept in the first half of 2024 and we are pleased that our first patient tolerated ICVB-1042 therapy well.”

Voyager Therapeutics Presents Robust, Multi-Species Results from Preclinical Studies of IV-Delivered, TRACER™-Generated Novel Capsids at the ASGCT 26th Annual Meeting

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Giovedì, Maggio 18, 2023
Infection, Neuron, DNA, GBA1, Human, Central nervous system, Capsid, Spinal cord, TRACER, Traffic barrier, Brain, Research, Gene expression, Tropism, RNA, Antibody, AAV, CNS, Astrocyte, ESGCT, BBB, Transgene, Rita Raave, DRG, Mouse, Liver, Sf9 (cells), Disability studies, Therapy, Glucocerebrosidase, Neurology, Oligonucleotide, Family, Society, Vaccine, Substantia nigra, Putamen

These results are being presented at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, taking place May 16-20, 2023, in Los Angeles.

Key Points: 
  • These results are being presented at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, taking place May 16-20, 2023, in Los Angeles.
  • “These data demonstrate that our TRACER capsid discovery platform is able to generate brain-penetrant capsids in a highly reproducible manner,” said Todd Carter, Chief Scientific Officer of Voyager Therapeutics.
  • In these studies, VCAP-102 demonstrated de-targeting of the dorsal root ganglia (DRG) and liver.
  • Researchers identified a new capsid called VCAP-103, which demonstrated enhanced brain and spinal cord transduction in marmosets and African green monkeys.

Coave Therapeutics to Participate in Upcoming Conferences

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Mercoledì, Maggio 17, 2023
Central nervous system, Therapy, Eye, Tropism, AAV, CNS, Pharmaceutical industry

Paris, France, May 17, 2023 - Coave Therapeutics (‘Coave’), a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases, today announces that it will participate at the following conferences:

Key Points: 
  • Paris, France, May 17, 2023 - Coave Therapeutics (‘Coave’), a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases, today announces that it will participate at the following conferences:
    CEO Rodolphe Clerval will be available in Boston for one-to-one meetings with potential partners and investors.
  • Meetings can be scheduled via the partnering system .
  • 4th Annual Gene Therapy for Neurological Disorders Summit Europe, June 6-8
    Chief Operating Officer, Patricia Françon, will discuss the challenges of deep brain delivery and distribution of gene therapies and how AAV-ligand conjugates can be used to overcome these challenges and improve efficacy of gene therapies in the CNS.
  • Next Generation Gene Therapy Vectors Summit 2023, June 27-29
    CEO Rodolphe Clerval will review post-translational chemical modifications available for capsids, how these improve on-target tropism and discuss how to manage manufacturing issues with chemically modified capsids.

Capsida Biotherapeutics to Present New Data Showing Increased Capsid Expression in CNS at American Society of Gene & Cell Therapy 26th Annual Meeting

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Mercoledì, Maggio 10, 2023
Learning, Neuron, DNA, Central nervous system, Gene, Therapeutic index, Tropism, AAV, CNS, Thalamus, Lilly, AbbVie, Putamen, Host, Vivisection, Annual general meeting, Cell, Escape Room, Liver, Therapy, Society, Potential, Vaccine, Medical device

THOUSAND OAKS, Calif., May 10, 2023 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced it will present preliminary data on the company's next-generation engineered adeno-associated virus (AAV) capsids in non-human primates. The initial data showed that Capsida's engineered AAV capsids, when delivered intravenously at a clinically relevant dose, resulted in widespread transduction including protein production across multiple brain regions including cortex, thalamus, and putamen, in up to 68% of neurons. Importantly, liver transduction was reduced five-fold compared to wild-type AAVs.

Key Points: 
  • Capsida will present these data at an industry-sponsored symposium on Friday, May 19, 2023 at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20, 2023 in Los Angeles.
  • In addition, Capsida will present several posters that highlight optimization of process and analytical methods for engineered AAV capsids as part of the ASGCT meeting.
  • "This capsid performance represents the potential to achieve a significantly increased therapeutic index," said Susan Catalano, Chief Scientific Officer of Capsida.
  • Capsida will present three posters that highlight the optimization of process and analytical development methods for engineered AAV capsids.

Apertura Gene Therapy Announces Scientific Founder Presentations on AAV Capsid Engineering Platforms at ASGCT 2023 Annual Meeting

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Mercoledì, Maggio 3, 2023
Science, Biotechnology, Research, Pharmaceutical, Health, Engineering, Genetics, Manufacturing, Vector, Heart, Broad Institute, Capsid, Gene, MIT, Tropism, Harvard University, AAV, Doctor of Philosophy, Cross, Cell, Society, Apertura and Clausura, Gene therapy, Vaccine, Target

Dr. Deverman and members of the Vector Engineering Lab of the Broad Institute will present findings on the founding platforms on which Apertura is based.

Key Points: 
  • Dr. Deverman and members of the Vector Engineering Lab of the Broad Institute will present findings on the founding platforms on which Apertura is based.
  • Both the Fit4Function AAV (adeno-associated virus) capsid engineering platform and a platform approach to engineering AAV capsids targeting known receptors leverage machine learning to accelerate capsid identification and selection.
  • Members of the Vector Engineering Lab will deliver five oral presentations and two poster presentations on AAV capsid engineering optimization strategies to enhance genetic medicine tropism and delivery efficiency.
  • Details of the presentations are as follows:

Affinia Therapeutics to Present New Data at the American Society of Gene and Cell Therapy Annual Meeting

Retrieved on: 
Mercoledì, Maggio 3, 2023
Gene, Cardiac muscle, Tropism, Capsid, AAV, CNS, Cell, Annual general meeting, American Society of Gene and Cell Therapy, Therapy, Society, Vaccine

WALTHAM, Mass., May 3, 2023 /PRNewswire/ -- Affinia Therapeutics, an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and prevalent devastating diseases, today announced that new preclinical data will be presented in an oral and poster sessions at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20, 2023 in Los Angeles, CA.

Key Points: 
  • WALTHAM, Mass., May 3, 2023 /PRNewswire/ -- Affinia Therapeutics, an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and prevalent devastating diseases, today announced that new preclinical data will be presented in an oral and poster sessions at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20, 2023 in Los Angeles, CA.
  • Presentation details are as follows:
    Title: Structure-Activity Relationships Guided Engineering of AAV Capsid with Optimized Skeletal Muscle, Cardiac Muscle, and CNS Tropism

Solid Biosciences Outlines Strategy for Leadership in Precision Genetic Medicines with Focus on Neuromuscular and Cardiac Diseases

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Martedì, Gennaio 10, 2023
IND, Disease, Spinal cord, CFA, Tissue, Heart, AAV, Duchenne muscular dystrophy, FA, HSV, Solid, Conference, Capsid, Cerebellum, Tropism, NHP, Investigational New Drug, Duchenne, Research, Heart failure, Liver, FXN, Cardiac muscle, Investment, BAG3, Pharmaceutical industry, Management, Vaccine, Agriculture, Hospital

CHARLESTOWN, Mass., Jan. 10, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing genetic medicines for neuromuscular and cardiac diseases, today outlined its strategic priorities for 2023. These announcements are being made in advance of Solid’s presentation at the 41st Annual J.P. Morgan Healthcare Conference scheduled for January 12 at 9:00 am PT.

Key Points: 
  • “The Solid leadership team has deep expertise in precision genetic medicine, and our diversified pipeline targets diseases with high unmet need, strong scientific rationale, and significant commercial potential.
  • Solid remains focused on developing next-generation AAV capsid libraries with two strategies designed to enhance cardiac and skeletal muscle tropism.
  • Clear mechanistic rationale between genetic BAG3 insufficiency and myofilament damage, poor contraction and heart failure
    Solid’s construct candidates utilize the AAVrh74 capsid and cardiac specific promoter.
  • In early nonclinical studies, the specific capsid and cardiac-specific promoter combination increased cardiac expression while reducing expression in the liver.

Neurocrine Biosciences and Voyager Therapeutics Enter Strategic Collaboration for Development and Commercialization of Voyager’s GBA1 Program and Other Next-Generation Gene Therapies for Neurological Diseases

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Lunedì, Gennaio 9, 2023
Hart–Scott–Rodino Antitrust Improvements Act, Therapy, Doctor of Philosophy, AAV, CNS, Telephone, Partnership, GBA1, Patient, RNA, Neurocrine Biosciences, Neuropharmacology, SAN, Sale, Ataxia, Tropism, Vaccine, Management, Pharmaceutical industry, Health insurance, Voyager

SAN DIEGO and CAMBRIDGE, Mass., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Neurocrine Biosciences, Inc. (NASDAQ: NBIX) and Voyager Therapeutics, Inc. (Nasdaq: VYGR) today announced the formation of a new strategic collaboration to advance multiple gene therapies for the treatment of neurological diseases. The collaboration includes Voyager’s preclinical, intravenously administered GBA1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases, which combines a GBA1 gene replacement payload with novel capsids from Voyager’s TRACERTM (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) platform. In addition, Neurocrine Biosciences and Voyager have agreed to collaborate on three new gene therapy programs directed to rare CNS targets, each also leveraging Voyager’s novel TRACER capsids. The collaboration builds upon the long-standing strategic partnership between Neurocrine Biosciences and Voyager and continues to combine Voyager’s expertise in novel capsid discovery, payload design, and neuropharmacology with Neurocrine Biosciences’ expertise in neuroscience and the clinical and commercial development of therapies for patients suffering from serious neurological diseases.

Key Points: 
  • In addition, Neurocrine Biosciences and Voyager have agreed to collaborate on three new gene therapy programs directed to rare CNS targets, each also leveraging Voyager’s novel TRACER capsids.
  • The collaboration builds upon the long-standing strategic partnership between Neurocrine Biosciences and Voyager and continues to combine Voyager’s expertise in novel capsid discovery, payload design, and neuropharmacology with Neurocrine Biosciences’ expertise in neuroscience and the clinical and commercial development of therapies for patients suffering from serious neurological diseases.
  • Regarding the GBA1 gene therapy program, Neurocrine Biosciences has agreed to fund development through the completion of a first Phase 1 trial.
  • Neurocrine Biosciences and Voyager have agreed that, following the completion of the transaction, Jude Onyia, Ph.D., Chief Scientific Officer at Neurocrine Biosciences, will join Voyager’s Board of Directors.