Tropism

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
TRACER, Brain, Traffic barrier, Cell, Mouse, Neuron, Tropism, Machine learning, RNA, B cell, AAV, Voyager, IND, CNS, Alkaline phosphatase, Crab-eating macaque, Human, DNA, Therapy, Astrocyte, Gene, Society, ALS, Spinal cord, BBB, DSM-IV codes, Central nervous system, Messenger, Capsid, Transcytosis, NHP, ALPL, HEK293, Centrifuge, SOD1, Vaccine

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.

Ring Therapeutics Presents New Data on Anellogy™ Platform at the 27th Annual American Society of Gene & Cell Therapy Conference

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Martedì, Maggio 7, 2024
Commensalism, AMD, Flagship Pioneering, Tropism, MD, Cell, AAV, Eye, Potential, Real-time, Human, DNA, Patient, Gene, Immune system, ANV, MBA, NHP, Therapy, Vaccine

CAMBRIDGE, Mass., May 07, 2024 (GLOBE NEWSWIRE) -- Ring Therapeutics, a life sciences company founded by Flagship Pioneering to revolutionize genetic medicines with its commensal virome platform, today announced new preclinical data demonstrating successful transduction and redosability of its Anellovector within non-human primates, as well as expanded payload capacity for the vector beyond the natural genome size of anelloviruses (ANV). The data, which will be presented as one oral presentation and two posters at the 27th Annual America Society of Gene & Cell Therapy (ASGCT) conference, showcases the viability of Anellovectors as the next generation of viral vectors.

Key Points: 
  • The data, which will be presented as one oral presentation and two posters at the 27th Annual America Society of Gene & Cell Therapy (ASGCT) conference, showcases the viability of Anellovectors as the next generation of viral vectors.
  • Additionally, the expanded payload capacity of Anellovectors widens the therapeutic potential of this novel vector class allowing even more patients to be potentially treated.
  • Unlike AAV, which appears to exhibit a strict packaging limit, Anellovectors allow for the packaging of genomes larger than the wild-type genome.
  • A combination of qPCR and next-generation sequencing was used to verify that the encapsidated expanded cargo DNA is intact, circular, and single-stranded.

Carbon Biosciences Announces Four Presentations at the American Society of Gene and Cell Therapy 27th Annual Meeting

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Giovedì, Maggio 2, 2024
Carbon, Parvoviridae, Analytic, Tropism, Poster, Heart, Cystic fibrosis, CFTR, Primate, Cell, Abstract, AAV, PAVE, Liver, Patient, Gene, Process, Society, Lung, Annual general meeting, Capsid, CBN, Knowledge, Vaccine

WALTHAM, Mass., May 2, 2024 /PRNewswire/ -- Carbon Biosciences, a preclinical stage biotechnology company developing genetic medicines for the treatment of pulmonary and cardiac diseases, announced that it has been selected for an oral presentation at the Presidential Symposium during the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place from May 7-11, 2024, in Baltimore, MD. The presentation will highlight the development of CGT-001, a potential gene therapy for the treatment of Cystic Fibrosis (CF), which utilizes the novel pulmonary targeting vector, CBN-1000, capable of delivering genes beyond the capacity of AAV. In addition, the Company will be featuring three poster presentations detailing new preclinical data, and manufacturing process development and analytical characterization for two of its novel viral gene therapy vectors.

Key Points: 
  • In addition, the Company will be featuring three poster presentations detailing new preclinical data, and manufacturing process development and analytical characterization for two of its novel viral gene therapy vectors.
  • Key insights from Carbon Biosciences' presentations at ASGCT:
    CBN-1000, a non-AAV parvovirus-based gene therapy vector, demonstrates excellent tolerability and transgene expression throughout the lung and airway in nonhuman primates after nebulization.
  • CBN-1100 vector demonstrates transgene packaging of up to 5.9 kb, representing >1 kb more capacity than AAV vectors.
  • The complete absence of expression in the liver suggests the potential for a differentiated specificity and safety profile compared to AAV-based vectors for cardiac gene therapy.

Tenaya Therapeutics to Highlight Growing Capabilities in Capsid Engineering, Gene Editing and Manufacturing at the American Society of Gene and Cell Therapy 27th Annual Meeting

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Giovedì, Maggio 2, 2024
Cardiomyopathy, Mutation, CRISPR, Good manufacturing practice, Safety, Risk, Abstract, Tropism, PLN, Gene editing, RNA, Cell, AAV, Disease, Therapy, Gene, Society, Fibrosis, HEK293, Annual general meeting, SAN, Vaccine

The company’s pipeline includes two clinical-stage gene therapies for cardiomyopathies, as well as earlier-stage research related to gene therapy, gene editing and cardiac cell regeneration, all using adeno-associated virus (AAV) as a delivery vehicle.

Key Points: 
  • The company’s pipeline includes two clinical-stage gene therapies for cardiomyopathies, as well as earlier-stage research related to gene therapy, gene editing and cardiac cell regeneration, all using adeno-associated virus (AAV) as a delivery vehicle.
  • Tenaya’s seven presentations at this year’s ASGCT will highlight new capsid engineering insights, the company’s emerging gene editing efforts, and manufacturing process optimizations intended to enhance the safety and efficacy profiles of AAV-based gene therapies.
  • Tenaya has developed a gene editing therapy utilizing a single AAV vector designed to deliver a proprietary self-inactivating CRISPR-Cas9 and PLN- R14del-mutation-specific single guide RNA.
  • At ASGCT, Tenaya researchers will present abstracts related to increasing yield and scalability associated with Sf9/rBV manufacturing processes.

Sania Therapeutics presents latest from AAV gene therapy platform at the American Society of Gene & Cell Therapy Annual Meeting

Retrieved on: 
Martedì, Aprile 23, 2024
Spasticity, Systemic disease, Date, Safety, Abstract (summary), Tropism, Abstract, Microfluidics, Outline, Cell, AAV, Oral medicine, Gene, Society, IPSC, DSM-IV codes, Brain cell, Neuron, Annual general meeting, Therapy, Vaccine

Andy Murray, Ph.D, CEO & Co-Founder of Sania Therapeutics, said: “Since our official launch last May, we have been pressing ahead with developing truly differentiated AAV gene therapies.

Key Points: 
  • Andy Murray, Ph.D, CEO & Co-Founder of Sania Therapeutics, said: “Since our official launch last May, we have been pressing ahead with developing truly differentiated AAV gene therapies.
  • Sania’s approach, using its R-Scan platform, is focused on generating novel, targeted vectors that can be successfully and rapidly translated into the clinic.
  • It combines human induced pluripotent stem cell derived cell types and microfluidics to perform directed AAV capsid evolution in an in vitro human system.
  • It combines an intramuscular injection of AAV gene therapy with oral medicine to selectively reduce excitability in targeted motor neurons.

Ring Therapeutics Announces Presentations at the 27th Annual American Society of Gene and Cell Therapy

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Lunedì, Aprile 22, 2024
Tropism, Therapy, Flagship Pioneering, Baltimore Convention Center, Gene, Society, Cell, Conference, Vaccine

CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Ring Therapeutics, a life sciences company founded by Flagship Pioneering to revolutionize genetic medicines with its commensal virome platform, today announced an oral presentation and two poster presentations at the 27th Annual American Society of Gene & Cell Therapy (ASGCT) Conference, to be held in Baltimore, Maryland from May 7 – 11, 2024. These presentations showcase the immense promise of a new class of viral vectors from Ring’s Anellogy™ platform to overcome some of the largest barriers associated with delivery of current genetic medicines including redosability and tissue tropism.

Key Points: 
  • CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Ring Therapeutics , a life sciences company founded by Flagship Pioneering to revolutionize genetic medicines with its commensal virome platform, today announced an oral presentation and two poster presentations at the 27th Annual American Society of Gene & Cell Therapy (ASGCT) Conference, to be held in Baltimore, Maryland from May 7 – 11, 2024.
  • These presentations showcase the immense promise of a new class of viral vectors from Ring’s Anellogy™ platform to overcome some of the largest barriers associated with delivery of current genetic medicines including redosability and tissue tropism.
  • Session Title and Location: Emerging Viral Vectors – Ballroom 4, Baltimore Convention Center

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
DRG, Heart, Traffic barrier, Computational science, Safety, Abstract (summary), Tropism, Cardiac muscle, MD, Structure, Diagram, Cell, BAG3, AAV, Skeletal muscle, CNS, Senior, Synthetic media, Liver, Gene, Society, ALS, Ataxin-2, BBB, Toxicity, Central nervous system, Messenger, Generative, Transcytosis, Annual general meeting, Therapy, Vaccine

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.

Avirmax Inc. to Make Scientific Presentations at ASGCT in Baltimore

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Martedì, Aprile 16, 2024
Rabbit, AMD, Mouse, PCV, Tropism, Cell, AAV, Sf9, Gene, Society, Sf9 (cells), Molecular Therapy, HEK293, CGMP, Vaccine

HAYWARD, Calif., April 15, 2024 /PRNewswire/ -- Avirmax Inc., will make two scientific presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) annual meeting in Baltimore between May 7th and 11th, 2024.

Key Points: 
  • HAYWARD, Calif., April 15, 2024 /PRNewswire/ -- Avirmax Inc., will make two scientific presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) annual meeting in Baltimore between May 7th and 11th, 2024.
  • In addition, Avirmax will have an exhibitor booth #818 to showcase the latest progress and data package.
  • ASGCT is the largest international society dedicated to understanding, development, and application of gene and cell therapy.
  • Avirmax Biopharma has completed cGMP manufacturing of ABI-110 using VSafTM rAAV Production Platform with Sf-rhabdovirus free Sf9 cells and presently prepares regulatory submission for clinical investigations.

Solid Biosciences Receives Rare Pediatric Disease Designation from the FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

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Lunedì, Aprile 1, 2024
Patient, DNA, Hope, Tropism, Duchenne muscular dystrophy, Duchenne, NOS1, Safety, FDA, DMD, Food, Pharmaceutical industry

CHARLESTOWN, Mass., April 01, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.

Key Points: 
  • “Solid’s receipt of Rare Pediatric Disease Designation for SGT-003 highlights the continuing need for transformational treatments for this devastating disease,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences.
  • “The key components of SGT-003 were rationally designed to improve on first generation gene therapies to provide skeletal muscle tropism, enhanced durability, and improved clinical outcomes.
  • nNOS is believed to play a crucial role in both muscular function and endurance,” said Dr. Gabriel Brooks, M.D., Chief Medical Officer at Solid Biosciences.
  • “We look forward to rapidly bringing SGT-003 to the clinic and hope to all Duchenne patients in need.”

Ring Therapeutics Publishes a Novel Gene Delivery Platform Based on the Commensal Human Anellovirus

Retrieved on: 
Lunedì, Aprile 1, 2024
Cell, Patient, Mouse, Flagship Pioneering, MD, Tropism, Biology, MBA, Iris (anatomy), Publication, Therapy, RPE, Immune system, Immune privilege, BioRxiv, Vaccine

CAMBRIDGE, Mass., April 01, 2024 (GLOBE NEWSWIRE) -- Ring Therapeutics, a life sciences company founded by Flagship Pioneering to revolutionize programmable medicines with its commensal virome platform, today announced a pre-print publication in bioRxiv featuring the first gene delivery vector system based on a human commensal virus, the anellovirus. These data support the immense promise of Ring’s AnellogyTM platform, harnessing the unique biology of commensal anelloviruses to engineer the next generation of durable and potentially redosable programmable viral vectors.

Key Points: 
  • – Publication describes the first gene delivery vector system based on a human commensal virus, the anellovirus –
    CAMBRIDGE, Mass., April 01, 2024 (GLOBE NEWSWIRE) -- Ring Therapeutics, a life sciences company founded by Flagship Pioneering to revolutionize programmable medicines with its commensal virome platform, today announced a pre-print publication in bioRxiv featuring the first gene delivery vector system based on a human commensal virus, the anellovirus.
  • These data support the immense promise of Ring’s AnellogyTM platform, harnessing the unique biology of commensal anelloviruses to engineer the next generation of durable and potentially redosable programmable viral vectors.
  • “We set out to develop an entirely new vector system based on human commensal viruses to address the many hurdles facing current genetic medicine delivery – and we’ve achieved that goal at remarkable speed,” said Tuyen Ong, MD, MBA, Chief Executive Officer of Ring and CEO-Partner at Flagship Pioneering.
  • This technology could enable the Anellogy platform to take advantage of the remarkable diversity of anelloviruses.