Capsid

Comparability of Five Alternative AAV9 Downstream Processes, from Ultracentrifugation to Chromatography, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Donnerstag, Juni 13, 2024
Research and development, Bioreactor, Therapy, UC, Red fluorescent protein, Indian stock exchange, Chromatography, AAV, Centrifuge, Senior, CEX, DNA, Capsid, Outline, Development, AEX, Vaccine

TORONTO, June 13, 2024 /PRNewswire-PRWeb/ -- Improved analytical methods are helping improve the understanding of capsid heterogeneity in adeno-associated virus (AAV) therapeutic preparations. This understanding allows the development of manufacturing processes that produce AAV therapeutics with fewer non-functional capsids.

Key Points: 
  • In this webinar, take a deep dive into the downstream manufacturing processes for producing functional adeno-associated virus (AAV) capsids.
  • Attendees will explore the different parameters that set apart ultracentrifugation from chromatography, including drug potency, vector yields, plasmid and host-cell deoxyribonucleic acid (DNA) levels and more.
  • TORONTO, June 13, 2024 /PRNewswire-PRWeb/ -- Improved analytical methods are helping improve the understanding of capsid heterogeneity in adeno-associated virus (AAV) therapeutic preparations.
  • In this webinar, the speakers will present the results of a five-arm AAV9 comparability protocol that includes four alternative two-column downstream processes at 50-L scale and an ultracentrifugation (UC) process at 200-L scale.

Affinia Therapeutics Presents Preclinical Data for Genetic Cardiomyopathy and Sporadic ALS Programs with Novel Cardiotropic and BBB-Penetrant AAV Capsids at the American Society of Gene & Cell Therapy 2024 Annual Meeting

Retrieved on: 
Freitag, Mai 10, 2024
Traffic barrier, Patient, Cell, Abstract (summary), Transcytosis, Toxicity, Liver, Cardiac muscle, Capsid, AAV, NHP, BAG3, Therapy, Safety, ALS, Heart, Ballroom, MBA, Senior, MD, Ataxin-2, Spinal cord, Research, Gene, HEK293, Neuron, CNS, BBB, DRG, Brain cell, Computational science, Brain, Society, Science, Cardiomyopathy, Tropism, Skeletal muscle, Central nervous system, Annual general meeting, Vaccine

WALTHAM, Mass., May 10, 2024 /PRNewswire/ -- Affinia Therapeutics ("Affinia"), 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 the presentation of new preclinical data on its novel AAV capsids for genetic cardiomyopathies and diseases of the central nervous system (CNS) such as amyotrophic lateral sclerosis (ALS), as well as the Company's high-yield manufacturing process. This research will be presented in oral sessions today at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting, being held May 7-11, 2024 in Baltimore, MD and virtually.

Key Points: 
  • This research will be presented in oral sessions today at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting, being held May 7-11, 2024 in Baltimore, MD and virtually.
  • Preclinical efficacy and safety results with a cardiotropic vector encoding for the BAG3 protein provide proof-of-concept data to support the expansion to multiple cardiomyopathies.
  • "Compared with AAV9, our next-generation bespoke capsids demonstrate superior cardiac transduction while detargeting the liver and DRG in single-clone NHP studies.
  • Affinia has also been invited to present on the rational design of cardiotropic capsids that detarget the liver and DRG.

Sangamo Therapeutics Reports Recent Business Highlights and First Quarter 2024 Financial Results

Retrieved on: 
Donnerstag, Mai 9, 2024
Biotechnology, Neurology, Health, Genetics, Pharmaceutical, STAC, FDA, CTA, Haemophilia, Enzyme replacement therapy, Kite, Investment, Nav1.7, Fabry disease, Poster, Disease, ERT, Sigma-Aldrich, STAAR, Health care, MAA, Partnership, Sangamo Therapeutics, MINT, AAV, Ligand Pharmaceuticals, IND, Tauopathy, Novartis, DNA, Patient, Pivotal, DSM-IV codes, EMA, Prion, BBB, Genome, BLA, MHRA, Manuscript, BioRxiv, Capsid, Modularity, Biogen, Therapy, Food and Drug Administration, European Medicines Agency, Gene, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.
  • “We are pleased with the progress being made in business development discussions across our portfolio, including our Fabry disease program.
  • An IND submission is expected in the fourth quarter of 2024.
  • Revenues for the first quarter ended March 31, 2024 were $0.5 million, compared to $158.0 million for the same period in 2023.

Kate Therapeutics Unveils Platform and Pipeline Progress at American Society of Gene & Cell Therapy 2024 Annual Meeting

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Donnerstag, Mai 9, 2024
Mortality, FDA, University, Facioscapulohumeral muscular dystrophy, Mouse, RNA interference, Safety, Tissue, Heart, Animal, Death, MD, Primate, Cell, Duchenne, Science, Disease, Rockefeller University, Patient, Liver, Society, Gene, X-linked myotubular myopathy, Pediatrics, Gene expression, Judgement, Duchenne muscular dystrophy, Capsid, Annual general meeting, SAN, Muscular dystrophy, Vaccine

SAN DIEGO, May 9, 2024 /PRNewswire/ -- Kate Therapeutics Inc. ("KateTx"), a next-generation gene therapy company, will present detailed preclinical efficacy and safety results on its pipeline and platform at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting being held May 7-11, 2024, in Baltimore, MD and virtually. The results support the best-in-class potential of KateTx's newest generation of skeletal muscle- and heart-targeted, liver de-targeted engineered MyoAAV capsids and gene regulation technology to treat Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), and other devastating genetic skeletal muscle and heart diseases.

Key Points: 
  • KateTx remains on track to select development candidates for additional muscle and cardiac programs in the near term.
  • "KateTx has made significant advances over the past 12 months," said Kevin Forrest, Ph.D., co-founder, president and CEO of KateTx.
  • KateTx's results show the potential to give a one-time gene therapy that potently suppresses DUX4 whenever it is expressed in skeletal muscles.
  • In vitro, the company's MyoAAV-LD mediated RNAi gene therapy candidate showed potent knockdown of DUX4 in FSHD patient myotubes with no off-target effects.

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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Mittwoch, Mai 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.

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

Retrieved on: 
Donnerstag, Mai 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.

Sangamo Therapeutics to Present Neurology-Focused Pre-Clinical Data From Its Epigenetic Regulation, Capsid Delivery and Genome Engineering Platforms at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Retrieved on: 
Montag, April 22, 2024
Research, Infectious Diseases, Neurology, Stem Cells, Biotechnology, Health, Pharmaceutical, General Health, Science, STAC, Neuron, Central nervous system disease, Dravet syndrome, Central nervous system, MD, Sangamo Therapeutics, Cell, AAV, Tauopathy, CNS, Engineering, DNA, Charcot–Marie–Tooth disease, Patient, Gene, Society, DSM-IV codes, Prion, Angelman syndrome, ALS, Genome, Capsid, Therapy, Powder metallurgy

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for presentation at the 27th ASGCT Annual Meeting being held May 7-11, 2024, in-person in Baltimore, MD and in a virtual format.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for presentation at the 27th ASGCT Annual Meeting being held May 7-11, 2024, in-person in Baltimore, MD and in a virtual format.
  • Presentations will focus on the progression of Sangamo’s neurology-focused pre-clinical pipeline, including data supporting innovations in zinc finger epigenetic regulation, advances in AAV capsid engineering, and discovery of next-generation integrase technology.
  • These data will showcase a novel AAV capsid, STAC-BBB, that exhibited robust and widespread central nervous system transduction in cynomolgus macaques after intravenous delivery, as well as another novel AAV capsid, STAC-150, engineered to accelerate the discovery of potent and highly specific epigenetic regulators.
  • Sangamo will also present additional data from its AAV capsid engineering platform SIFTER and its efforts for integrase evolution and utilization.

GeoVax Presents Data on GEO-CM04S1, a Next Generation Covid-19 Vaccine

Retrieved on: 
Donnerstag, April 4, 2024
Biotechnology, Georgia State University, Messenger, Death, Doctor of Philosophy, SARS-CoV-2, Capsid, ACE2, Combat, Infection, Cancer, Omicron, Vaccine, Antigen

ATLANTA, GA, April 04, 2024 (GLOBE NEWSWIRE) -- via NewMediaWire -- GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced that its Chief Scientific Officer, Mark Newman, PhD, presented data on GEO-CM04S1, the Company’s next-generation Covid-19 vaccine candidate, during the 24th Annual World Vaccine Congress in Washington, DC.

Key Points: 
  • During his presentation, titled “Vaccine Induction of Broadly-Specific Antibody and T Cell Responses to Combat SARS-CoV-2 Variation”, Dr. Newman described GeoVax’s development program for GEO-CM04S1.
  • The data presented were generated in collaboration with scientists at Georgia State University using the human ACE2 transgenic mice, one of the “gold standard” small animal models used for studying Covid vaccines.
  • GEO-CM04S1 incorporates both the spike (S) and nucleocapsid (N) antigens of SARS-CoV-2 and is specifically designed to induce broadly specific antibody and T cell responses.
  • I anticipate similar findings will be observed in our Phase 2 clinical trials as the studies are unblinded later this year.”

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Retrieved on: 
Dienstag, April 9, 2024
Community, Eurostat, Civil service commission, Diagnosis, U1 spliceosomal RNA, Capsid, Gene, B cell, Cell, Disease, Agonal respiration, Patient, Committee, Knowledge, Regulation, EMA, IRIS, Protein, Human, Duchenne muscular dystrophy, Dystrophin, European, COMP, Heart, European Commission, Safety, Muscle weakness, Blood, DMD, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Key Points: 


Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Sangamo Therapeutics Announces Data From Novel Proprietary Neurotropic AAV Capsid Demonstrating Industry-leading Blood-brain Barrier Penetration and Brain Transduction in NHPs

Retrieved on: 
Mittwoch, März 13, 2024
Research, Neurology, Genetics, Clinical Trials, Health, Pharmaceutical, General Health, Other Science, Science, STAC, AAV, Sangamo Therapeutics, Gene, Brain, Capsid, Gene expression, ALS, BBB, Central nervous system, Novartis, CNS, CTA, DRG, Traffic barrier, Prion, NHP, Tropism, IND, Nav1.7, Neuron, Biogen, Tauopathy, Excipient, Therapy, DSM-IV codes, Spinal cord, Liver, Animal, Vaccine

Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.

Key Points: 
  • Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.
  • In NHP studies when administered intravenously at clinically relevant doses, STAC-BBB demonstrated its potential to be a leading neurotropic capsid.
  • Exhibited 700-fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published neurotropic capsid variants evaluated in the study.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.