Tropism

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.

Adicet Provides Corporate Update and Highlights Strategic Priorities for 2024

Retrieved on: 
Thursday, January 4, 2024
Oncology, Health, Infectious Diseases, Clinical Trials, Pharmaceutical, Biotechnology, Telephone, Initiate, Myositis, Tumor microenvironment, Risk, Probability, IND, Autoimmunity, FDA, Cytokine release syndrome, CD20, Renal cell carcinoma, Scleroderma, Tissue, Cmax, Cancer, TGF, Tropism, NHL, Patient, CD19, AUC, Safety, Lupus nephritis, CD70, MCL, Pharmaceutical industry

Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for cancer and autoimmune diseases, today provided corporate updates and highlighted upcoming priorities for its pipeline programs in 2024.

Key Points: 
  • Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for cancer and autoimmune diseases, today provided corporate updates and highlighted upcoming priorities for its pipeline programs in 2024.
  • “In 2024, we aim to make significant strides across our pipeline of differentiated gamma delta T cell therapies through our strategic and disciplined approach,” said Chen Schor, President and Chief Executive Officer at Adicet Bio.
  • Initiate Phase 1 clinical trial of ADI-001 for the treatment of lupus nephritis in the second quarter of 2024.
  • The Company remains on track to provide an ADI-001 clinical update in the second half of 2024.

Vesigen Receives National Institutes of Health Award in Targeted Genome Editor Delivery Challenge

Retrieved on: 
Tuesday, December 19, 2023
Biotechnology, Genetics, Health, SCGE, RNA, Gene editing, Cell, Therapy, Tropism, Genome, Bangladesh Technical Education Board, NIH, Vaccine

Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, was selected as a Phase 1 winner of the National Institutes of Health TARGETED (Targeted Genome Editor Delivery) Challenge .

Key Points: 
  • Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, was selected as a Phase 1 winner of the National Institutes of Health TARGETED (Targeted Genome Editor Delivery) Challenge .
  • The Company’s proposal, “Engineered ARMMs: Promising Human-Derived Vectors for Cell Type-Specific Delivery of Genome Editors”, builds on recent data demonstrating directed tropism of the Company’s non-viral ARMM (ARrestin-domain 1 Mediated Microvesicles) delivery platform and was among five proposals selected as “Winning Solutions” for programmable delivery systems for gene editing.
  • We look forward to advancing our platform toward the clinic and unlocking the full potential of genetic medicines.”
    The NIH launched the TARGETED Challenge to advance genome editing technology by sourcing innovative solutions for delivering genome editing components safely and effectively.
  • The three-phase challenge supports the NIH's Somatic Cell Genome Editing (SCGE) commitment to developing targeted delivery systems for delivering genome editors to somatic cells in the body.

Adicet Reports Third Quarter 2023 Financial Results and Provides Business Updates

Retrieved on: 
Wednesday, November 8, 2023
Science, Stem Cells, Biotechnology, Research, Pharmaceutical, Oncology, Health, Clinical Trials, CAR, Trial of the century, IND, Food, Bio, CD70, Renal cell carcinoma, International Conference on Afghanistan, London (2010), CD20, Society for Immunotherapy of Cancer, Tropism, TGF, Conference, CDMO, Regulation of tobacco by the U.S. Food and Drug Administration, Prostate cancer, R, Engineering, Poster, Goodwill, SITC, Immunotherapy, MCL, Research and development, Annual general meeting, Probability, ACET, Tumor microenvironment, Epitope, NHL, Society, Cancer, PSMA, Patient, Administration, Prognosis, Prostate, CD27, Pharmaceutical industry, Cryptocurrency

Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for cancer, today reported financial results and operational highlights for the third quarter ended September 30, 2023.

Key Points: 
  • Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for cancer, today reported financial results and operational highlights for the third quarter ended September 30, 2023.
  • In October 2023, Adicet presented new preclinical data building on the potential of Adicet’s allogeneic gamma delta platform as a promising approach to target prostate cancer.
  • Financial Results for Third Quarter 2023:
    Research and Development (R&D) Expenses: R&D expenses were $26.2 million for the three months ended September 30, 2023, compared to $16.6 million during the same period in 2022.
  • Cash Position: Cash and cash equivalents were $183.3 million as of September 30, 2023, compared to $257.7 million as of December 31, 2022.

Ring Therapeutics Presents New Data on Anellogy™ Platform at the 30th Annual European Society of Gene & Cell Therapy Congress

Retrieved on: 
Tuesday, October 24, 2023
RNA, Tropism, Flagship Pioneering, Transduction, MD, MBA, Tissue, Cell, Gene, ESGCT, European Society of Gene and Cell Therapy, Genome, Therapy, DNA, Vaccine

CAMBRIDGE, Mass., Oct. 24, 2023 (GLOBE NEWSWIRE) -- Ring Therapeutics, a life sciences company founded by Flagship Pioneering to revolutionize gene therapy with its commensal virome platform, today announced new preclinical data demonstrating a functional anellovirus-based viral vector and the innovative manufacturing technology enabling its production at scale. The data, which will be presented in two posters at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), validates the Anellogy platform’s potential to overcome some of the largest barriers facing gene therapy today.

Key Points: 
  • The data, which will be presented in two posters at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), validates the Anellogy platform’s potential to overcome some of the largest barriers facing gene therapy today.
  • "I am extremely proud of the team at Ring as we reach this critical milestone showcasing Anellovectors as a viable viral vector for genetic medicines,” said Tuyen Ong, MD, MBA, Chief Executive Officer of Ring Therapeutics and CEO-Partner at Flagship Pioneering.
  • “Building on the foundational work characterizing anelloviruses, we have achieved in vivo transduction of Anellovectors, supported by our modular AnelloBricks manufacturing technology, to produce them at scale.
  • DNA, RNA)
    AnelloBricks offers payload versatility while reducing manufacturing complexity, resulting in a readily scalable production system