Tropism

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

Retrieved on: 
Tuesday, April 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

Retrieved on: 
Monday, April 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

Retrieved on: 
Monday, April 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

Retrieved on: 
Tuesday, April 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

Retrieved on: 
Monday, April 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: 
Monday, April 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.

Fractyl Health Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Monday, April 1, 2024
Research, Glucose, Health, Congress, Obesity, Human, Drug discovery, Exercise, Glycated hemoglobin, DMR, ADAS, Partnership, Insulin, IDA, Patient, Weight loss, Michigan Medicine, Diabetes, Public expenditure, IDE, University, IPO, Tropism, Peptide, Transgene, Insulin resistance, Animal, NUB, Type 2 diabetes, Pancreas, Investigational New Drug, Therapy, Food, Marketing, FDA, Engineering, Annual general meeting, Pharmaceutical industry

BURLINGTON, Mass., April 01, 2024 (GLOBE NEWSWIRE) -- Fractyl Health (Nasdaq: GUTS), a metabolic therapeutics company focused on pioneering new approaches for the treatment of type 2 diabetes (T2D) and obesity, today reported its fourth quarter and full year 2023 financial results and provided a business update.

Key Points: 
  • “In addition, we expect to complete enrollment of our pivotal Revitalize-1 study and are excited to report topline data in the fourth quarter of 2024.
  • The Company expects to complete enrollment in the first half of 2024 and report topline data in the fourth quarter of 2024.
  • In December 2023, Fractyl presented preclinical findings in an oral presentation at the World Congress of Insulin Resistance Diabetes and Cardiovascular Disease 2023 Annual Meeting.
  • Cash Position: As of December 31, 2023, Fractyl had approximately $33.2 million in cash and cash equivalents.

Adicet Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Tuesday, March 19, 2024
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, ChromeOS, Administration, MCL, ASH, Protocol, Patient, Tumor microenvironment, Holocene, G&A, CD70, Cancer, Tropism, Renal cell carcinoma, NHL, IND, Drug, Mantle cell lymphoma, Research and development, Society, TGF, ATM, Safety, FDA, Lupus nephritis, Autoimmunity, Pharmaceutical industry

Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today reported financial results and operational highlights for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today reported financial results and operational highlights for the fourth quarter and year ended December 31, 2023.
  • In December 2023, the FDA cleared the Company’s IND application for ADI-001 in lupus nephritis.
  • Adicet plans to initiate a Phase 1 study to evaluate the safety and efficacy of ADI-001 in lupus nephritis during the second quarter of 2024.
  • Preliminary clinical data from the trial are expected in the fourth quarter of 2024 or first quarter of 2025, pending study site activation progression and patient enrollment.

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

Retrieved on: 
Wednesday, March 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.

Therapeutic mRNA Patent Monitoring Service 2024: Track Competitors' IP Activities and their Future Intentions

Retrieved on: 
Tuesday, April 2, 2024
PDF, Q&A, COVID-19, DSM-IV codes, Knowledge, RNA, Messenger, Tropism, IP, Cancer, Vaccination, Invention, Pharmacokinetics, Patent, Microsoft Excel, Therapy, Vaccine, Orientation, Directory

The directory includes new patent applications, new granted patents, patents expired/abandoned, patent transfers (re-assignment, licensing), and patent litigation/opposition.

Key Points: 
  • The directory includes new patent applications, new granted patents, patents expired/abandoned, patent transfers (re-assignment, licensing), and patent litigation/opposition.
  • The success of mRNA vaccines as a response to the COVID-19 pandemic has shone a light on the disruptive aspect of mRNA-based therapeutic technology.
  • With the help of the patent monitoring service, you will be aware of your competitors' current patenting activities, their IP dynamics, patent transfers including acquisitions and licenses, patent litigation, technology development, and R&D strategies.
  • Main patent applicants and their inventions, blocking patents, promising patents, and key newly expired or abandoned patents will be highlighted
    Featured image of Therapeutic mRNA patent monitor.