Gene targeting

Cytosurge's CellEDIT Workflow, based on its Proprietary FluidFM® Technology, Receives Validation in Peer-Reviewed Publication

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Mercredi, mai 29, 2024
KO, FUT8, Genetic engineering, Cas9, Doctor of Philosophy, Paper, Paramount, Organization, CHO, Lead, University, MD, Research, DHFR, Drug discovery, Gene, BAX, Manuscript, Workflow, Hospital, CRISPR, Gene targeting, Chinese hamster ovary cell, RNP, Biotechnology Journal, Cell engineering, Gene editing, Mobile phone

Control of the amount and stoichiometry of RNP complexes could boost efficiency, particularly when targeting multiple loci simultaneously.

Key Points: 
  • Control of the amount and stoichiometry of RNP complexes could boost efficiency, particularly when targeting multiple loci simultaneously.
  • Demonstrated preservation of general cell characteristics and functionality post-editing in both single and multiple KO clones in CHO-K1 cells.
  • The study focuses on Chinese hamster ovary (CHO) cells, widely used in biopharmaceutical production, particularly for monoclonal antibody manufacturing.
  • Our CellEDIT technology, validated in this peer-reviewed publication, offers meticulous control over gene targeting,” stated Tobias A. Beyer, PhD, Chief Scientific Officer at Cytosurge and co-author of the publication.

Cytosurge's CellEDIT Workflow, based on its Proprietary FluidFM® Technology, Receives Validation in Peer-Reviewed Publication

Retrieved on: 
Mercredi, mai 29, 2024
KO, FUT8, Genetic engineering, Cas9, Doctor of Philosophy, Paper, Paramount, Organization, CHO, Lead, University, MD, Research, DHFR, Drug discovery, Gene, BAX, Manuscript, Workflow, Hospital, CRISPR, Gene targeting, Chinese hamster ovary cell, RNP, Biotechnology Journal, Cell engineering, Gene editing, Mobile phone

Control of the amount and stoichiometry of RNP complexes could boost efficiency, particularly when targeting multiple loci simultaneously.

Key Points: 
  • Control of the amount and stoichiometry of RNP complexes could boost efficiency, particularly when targeting multiple loci simultaneously.
  • Demonstrated preservation of general cell characteristics and functionality post-editing in both single and multiple KO clones in CHO-K1 cells.
  • The study focuses on Chinese hamster ovary (CHO) cells, widely used in biopharmaceutical production, particularly for monoclonal antibody manufacturing.
  • Our CellEDIT technology, validated in this peer-reviewed publication, offers meticulous control over gene targeting,” stated Tobias A. Beyer, PhD, Chief Scientific Officer at Cytosurge and co-author of the publication.

Seek Labs Announces Successful Completion of African Swine Fever Virus In Vivo Trial Utilizing CRISPR-based Treatments

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Lundi, mai 6, 2024
Research, Genetics, Clinical Trials, Other Health, Biotechnology, Health, Agriculture, Natural Resources, Science, Other Science, Mortality, African swine fever virus, Viral disease, CRISPR, Gene targeting, Hope, White, Chicken, Synthetic control method, Blood, ASF, Classical swine fever, Cas, ELISA, Animal, Survival, Litopenaeus, Infection, Doctor of Philosophy, Disease, ASFV, Real-time, Food security, Viral load, Food, Seek, H5N1, Genome, Candidate, Viral, Pharmacy, Injection, WSSV, Medication, Vaccine

Seek Labs, a healthcare innovations company developing next-generation point-of-care molecular diagnostic systems and novel CRISPR-based gene therapies, announced today the successful completion of a therapeutic trial using innovative CRISPR-based technologies to target the African Swine Fever Virus (ASFV) in pigs.

Key Points: 
  • Seek Labs, a healthcare innovations company developing next-generation point-of-care molecular diagnostic systems and novel CRISPR-based gene therapies, announced today the successful completion of a therapeutic trial using innovative CRISPR-based technologies to target the African Swine Fever Virus (ASFV) in pigs.
  • The trial, which aimed to replicate and expand an earlier trial, demonstrated pigs infected with ASFV and treated with Seek Labs’ CRISPR-based systems survived longer than untreated infected pigs.
  • With no available treatments for African Swine Fever, this is a breakthrough discovery.
  • Seek Labs’ work represents a beacon of hope for the swine industry and underscores the transformative potential of CRISPR-based technology in global healthcare.

Factor Bioscience to Deliver Seven Presentations at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting

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Mardi, avril 23, 2024
Ballroom, Safety, Transgene, Express, Lipid, Monocyte, Chromatin, Research fellow, IPS, MD, Cell, Gene targeting, Development, Macrophage, DNA, Gene, Society, Messenger, Annual general meeting, Dicarboxylic acid, Vaccine

CAMBRIDGE, Mass., April 23, 2024 /PRNewswire/ -- Factor Bioscience Inc., a Cambridge-based biotechnology company focused on developing mRNA and cell-engineering technologies, announced its participation in the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting to be held in Baltimore, MD from May 7-11, 2024. Factor will deliver seven presentations, representing the company's most expansive presentation of data to date.

Key Points: 
  • CAMBRIDGE, Mass., April 23, 2024 /PRNewswire/ -- Factor Bioscience Inc., a Cambridge-based biotechnology company focused on developing mRNA and cell-engineering technologies, announced its participation in the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting to be held in Baltimore, MD from May 7-11, 2024.
  • Factor will deliver seven presentations, representing the company's most expansive presentation of data to date.
  • "Factor will deliver seven presentations, representing the company's most expansive presentation of data to date."
  • For more information about the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, visit annualmeeting.asgct.org .

Beam Therapeutics Reports Pipeline Updates and First Quarter 2023 Financial Results

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Mercredi, mai 10, 2023
Well, GMP, Tissue, ESCAPE, Research and development, Drug development, Stargardt disease, Hematology, Gene targeting, Biotechnology, SCD, Clinical trial, Bone marrow, Cancer, G&A, Patient, Eye, Science, AATD, Investment, Security (finance), Administration, DSM-IV codes, Sickle cell disease, Liver, R, LNP, LNP Media Group, Services General Administration and Coordination Department, Government of Sindh, Society, Vaccine, Pharmaceutical industry, Medical imaging, Stargardt, Beam

CAMBRIDGE, Mass., May 10, 2023 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported first quarter 2023 financial results and provided an update on its BEACON clinical trial and pipeline progress.

Key Points: 
  • Beam expects to fully enroll the sentinel cohort in 2023.
  • Beam expects to initiate current good manufacturing practice compliant operations at its North Carolina manufacturing facility in late 2023.
  • Research & Development (R&D) Expenses: R&D expenses were $99.6 million for the first quarter of 2023, compared to $65.4 million for the first quarter of 2022.
  • General & Administrative (G&A) Expenses: G&A expenses were $23.5 million for the first quarter of 2023, compared to $19.2 million for the first quarter of 2022.

Ascidian Therapeutics to Present New Data From Its Lead Program Targeting ABCA4 Retinopathies at the ASGCT 2023 Annual Meeting

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Mardi, mai 9, 2023
Immortalised cell line, Bell, Trans-splicing, Gene, Macular degeneration, File, Stargardt disease, Gene targeting, Gene editing, PDT, RNA, Publication, AAV, CNS, Animal, Patient, Retinopathy, Cell, Ascidian mitochondrial code, Therapy, Society, Los Angeles Convention Center, Pharmaceutical industry, Vaccine, Stargardt, ABCA4, Ascidiacea, IND

BOSTON, May 9, 2023 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company focused on treating human diseases by rewriting RNA, announced today that Head of Research Robert Bell, Ph.D., will present new data from its lead ABCA4 program at the Twenty-Sixth Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).

Key Points: 
  • The lead program targets ABCA4-related retinopathies, including Stargardt disease, and is currently in IND-enabling studies advancing towards clinical development.
  • The oral presentation, taking place at 9:00 a.m. PDT on May 20, will highlight Ascidian's groundbreaking RNA exon editing platform.
  • Specifically, Dr. Bell will present new six-month data from Ascidian's lead ABCA4 program that demonstrate the production of full-length ABCA4 protein following a one-time treatment with a single AAV-delivered RNA exon editing development candidate in the non-human primate retina.
  • These data are the first to quantify therapeutically relevant levels of ABCA4 protein and represent the most efficient and durable RNA exon editing via trans-splicing ever demonstrated in large animals.

Epic Bio to Present Preclinical Data at American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting

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Jeudi, mai 4, 2023
Gene targeting, Biotechnology, DCAS, Gene, Society, Gene expression, SAN, Cell, Vaccine

SOUTH SAN FRANCISCO, Calif., May 04, 2023 (GLOBE NEWSWIRE) -- Epic BIO , a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced the acceptance of an abstract for oral presentation at the upcoming Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 16-20, 2023, in Los Angeles, Calif.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., May 04, 2023 (GLOBE NEWSWIRE) -- Epic BIO , a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced the acceptance of an abstract for oral presentation at the upcoming Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 16-20, 2023, in Los Angeles, Calif.
    Full details of the presentation are as follows:
    Session: Gene Targeting and Gene Correction: Hemoglobin, Muscle, and Eye

Tessera Therapeutics to Present New Data Showcasing the Broad Potential of its Gene Writing™ and Delivery Platforms at the American Society of Gene and Cell Therapy 26th Annual Meeting

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Mardi, mai 2, 2023
Gene, Gene targeting, Biotechnology, RNA, Patient, American Society of Gene and Cell Therapy, Cell, Annual general meeting, Small RNA, Therapy, Emerging technologies, Society, Vaccine, Antibiotics

SOMERVILLE, Mass., May 02, 2023 (GLOBE NEWSWIRE) -- Tessera Therapeutics, the biotechnology company pioneering a new approach in genetic medicine known as Gene Writing™, today announced that it will present new preclinical data at the upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place in Los Angeles, California, May 16 – 20, 2023.

Key Points: 
  • SOMERVILLE, Mass., May 02, 2023 (GLOBE NEWSWIRE) -- Tessera Therapeutics, the biotechnology company pioneering a new approach in genetic medicine known as Gene Writing™, today announced that it will present new preclinical data at the upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place in Los Angeles, California, May 16 – 20, 2023.
  • Details of the Company’s ASGCT 26th Annual Meeting presentations are as follows:
    Session: Gene Targeting and Gene Correction: Liver
    Session: Gene Targeting and Gene Correction: New Technologies
    Title: Highly Efficient Correction of the Sickle Cell Disease Mutation in Patient HSC Using an RNA Gene Writing System, an RNA-based, Nuclease-Free Approach to Genome Editing

EdiGene to Unveil Promising Preclinical POC Data for LEAPER™ 2.0-based in vivo RNA Editing Therapies in NHP Model at the 26th Annual Meeting of ASGCT via Oral Presentation

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Mardi, mai 2, 2023
Research, Genetics, Clinical Trials, Biotechnology, General Health, Pharmaceutical, Health, Science, Oncology, Other Science, Gene, Gene targeting, Algorithm, RNA, CNS, Human germline engineering, Patient, ADAR, Cell, NHP, POC, Society, Vaccine

LEAPERTM 2.0 is our proprietary exogenous protein-free RNA base editing technology that uses engineered circular ADAR-recruiting RNAs.

Key Points: 
  • LEAPERTM 2.0 is our proprietary exogenous protein-free RNA base editing technology that uses engineered circular ADAR-recruiting RNAs.
  • Data from specific NHP disease model have demonstrated the preclinical safety and efficacy of LEAPERTM 2.0-based in vivo therapeutic approach with outstanding editing efficiency.
  • With endogenous ADAR, LEAPERTM has achieved precise and highly efficient RNA base editing using only one guide RNA (arRNA).
  • Additionally, the exogenous protein-free feature significantly reduces delivery difficulty and minimizes the potential for immune response induced by foreign proteins.

Chroma Medicine to Present First Data Demonstrating Promise of Its Epigenetic Editing Platform at 26th ASGCT Annual Meeting

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Mardi, mai 2, 2023
DNA, Therapy, Gene, CSO, Gene targeting, Liver, Deletion, Gene expression, Human, Hypercholesterolemia, Chromosomal translocation, Data, LDL, Annual general meeting, Cell, DNA methylation, Society, Medicine, Antibiotics, Vaccine, Chroma

BOSTON, May 2, 2023 /PRNewswire/ -- Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced two oral data presentations that showcase the potential of its epigenetic editing platform at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16-20, 2023 in Los Angeles, California. The presentations include the first in vivo proof-of-concept data for Chroma's epigenetic editing platform along with data demonstrating the key advantages of epigenetic editing for multiplex gene regulation. Chroma plans to release data from additional pipeline programs at upcoming scientific conferences.  

Key Points: 
  • In vivo data provide compelling proof-of-concept for Chroma's epigenetic editing platform to enable highly efficient, specific, and durable gene regulation
    BOSTON, May 2, 2023 /PRNewswire/ -- Chroma Medicine , Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced two oral data presentations that showcase the potential of its epigenetic editing platform at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16-20, 2023 in Los Angeles, California.
  • The presentations include the first in vivo proof-of-concept data for Chroma's epigenetic editing platform along with data demonstrating the key advantages of epigenetic editing for multiplex gene regulation.
  • Chroma plans to release data from additional pipeline programs at upcoming scientific conferences.
  • Data presented at ASGCT demonstrate the ability of epigenetic editors to facilitate genotoxicity-free multiplexed editing in healthy donor-derived T cells, as compared to Cas9-mediated editing.