Gene editing

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

Retrieved on: 
Thursday, May 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.

Regeneron and Mammoth Biosciences Collaborate to Pursue Next-Generation CRISPR-Based Gene Editing for Multiple Diseases

Retrieved on: 
Thursday, April 25, 2024
University, Cas9, Regeneron Pharmaceuticals, Tissue, Gene editing, Nobel Prize, Mammoth, University of California, Berkeley, Research, AAV, Nobel Prize in Chemistry, Vivisection, Senior, Liver, Patient, Genome, Mammoth Biosciences, Pharmaceutical industry

TARRYTOWN, N.Y., April 25, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) and Mammoth Biosciences, Inc., today announced a collaboration to research, develop and commercialize in vivo CRISPR-based gene editing therapies for multiple tissues and cell types. Regeneron is developing adeno-associated viral vectors (AAVs) using antibody-based targeting to enhance delivery of genetic medicine payloads to specific tissues and cell types. Mammoth is developing novel ultracompact nucleases and associated gene editing systems, with a variety of editing functionalities at a significantly smaller size than other CRISPR-based systems, including first generation Cas9 nucleases. By leveraging Regeneron’s expertise in AAV and antibody engineering and Mammoth’s expertise in ultracompact gene editing systems, the teams will endeavor to create disease-modifying medicines that can be delivered to tissues beyond the liver, to which most gene editing treatments are currently limited.

Key Points: 
  • TARRYTOWN, N.Y., April 25, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) and Mammoth Biosciences, Inc., today announced a collaboration to research, develop and commercialize in vivo CRISPR-based gene editing therapies for multiple tissues and cell types.
  • Mammoth is developing novel ultracompact nucleases and associated gene editing systems, with a variety of editing functionalities at a significantly smaller size than other CRISPR-based systems, including first generation Cas9 nucleases.
  • “We believe in the incredible power of gene editing, which we are utilizing in our diverse preclinical and clinical genetic medicines pipeline.
  • We look forward to working with Regeneron to enable all-in-one AAV delivery and unlock the true potential of in vivo gene editing,” said Trevor Martin, Ph.D., co-founder and Chief Executive Officer of Mammoth Biosciences.

Regeneron to Highlight Advances in Genetic Medicine Research at American Society of Gene and Cell Therapy (ASGCT)

Retrieved on: 
Monday, April 22, 2024
Hope, Hearing loss, Tissue, Regeneron Pharmaceuticals, Gene silencing, Abstract, Gene editing, Cell, Genetics, Senior, Liver, Patient, Gene, Society, Antibody, Clinical trial, Head, Pharmaceutical industry

TARRYTOWN, N.Y., April 22, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that new and updated data across its genetic medicines portfolio will be presented at the American Society of Gene and Cell Therapy (ASGCT) annual conference in Baltimore, Maryland, from May 7 to 11, 2024. Data from 10 abstracts, including six oral presentations, provide insight on Regeneron’s approach to overcoming obstacles to clinical implementation of genetic medicines, from pre-dosing to delivery to long-term sustained expression. The company will also present updated data from the Phase 1/2 CHORD trial investigating DB-OTO in children with profound genetic hearing loss due to mutations of the otoferlin gene.

Key Points: 
  • Data from 10 abstracts, including six oral presentations, provide insight on Regeneron’s approach to overcoming obstacles to clinical implementation of genetic medicines, from pre-dosing to delivery to long-term sustained expression.
  • The company will also present updated data from the Phase 1/2 CHORD trial investigating DB-OTO in children with profound genetic hearing loss due to mutations of the otoferlin gene.
  • “Regeneron continues to advance methods to overcome these obstacles through our proprietary delivery approaches utilizing next-generation viral vectors, particularly specific retargeting antibodies and innovative payloads.
  • The ASGCT presentation will build on promising early results in the first patient,” said Aris Baras, M.D., Senior Vice President, Co-Head of Regeneron Genetic Medicines and Head, Regeneron Genetics Center®.

Editas Medicine to Present Pre-clinical Data Demonstrating Progression of in vivo Medicines Pipeline at the American Society of Gene and Cell Therapy Annual Meeting

Retrieved on: 
Monday, April 22, 2024
Guide, LSID, Abstract, Research, Editas Medicine, Abstract (summary), Gene editing, RNA, Cell, Medicine, Gene, Society, Vaccine

The Company is presenting pre-clinical data to support its development of transformative in vivo gene editing medicines.

Key Points: 
  • The Company is presenting pre-clinical data to support its development of transformative in vivo gene editing medicines.
  • Editas Medicine presentations at ASGCT include:
    Pre-clinical data demonstrating AsCas12a gRNA modifications that enable high-potency gene editing in multiple cell types and improve gene editing outcomes in vivo, enabling the development of in vivo gene editing medicines.
  • Research on identifying potent large serine recombinases (LSRs) as a foundation to develop novel in vivo gene editing technologies for whole gene knock-in, expanding potential in vivo gene editing targets for developing medicines.
  • These in vivo data are an important step towards confirming in vivo proof of concept by the end of the year.”
    The complete list of Editas Medicine presentations is below.

Excision BioTherapeutics Announces Oral Presentation of Preclinical HSV-1 Keratitis Data at CRISPRMED24 Conference on April 24, 2024

Retrieved on: 
Monday, April 22, 2024
CRISPR, Hope, Gene editing, Conference, Research, Patient, Viral, SAN, Vaccine

Excision’s EBT-104 is a CRISPR-based gene therapy that is being developed as a potential cure for HSV-1 Keratitis.

Key Points: 
  • Excision’s EBT-104 is a CRISPR-based gene therapy that is being developed as a potential cure for HSV-1 Keratitis.
  • EBT-104 utilizes a CRISPR/Cas gene editing system to inactivate the latent HSV-1 virus.
  • “The exceptional in vivo efficacy demonstrated by our gene editing approach offers new hope for patients suffering from this debilitating condition.
  • We look forward to sharing these new data from our EBT-104 program at the first CRISPRMED24 Conference.”

SNIPR Biome receives funding from CARB-X to support advancement of CRISPR-medicine SNIPR001 into clinical trials in haematological cancer patients

Retrieved on: 
Monday, April 22, 2024
Bone marrow, FQR, CRISPR, Nature Biotechnology, Safety, Education, Pharmacokinetics, Infection, Neutropenia, Survival, AMR, Febrile neutropenia, Gene editing, Partnership, Bloodstream infections, Development, BMBF, Escherichia coli, Research and development, Patient, Quinolone antibiotic, Synthetic biology, HSCT, Pharmaceutical industry

Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.

Key Points: 
  • Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.
  • Despite the significant advances in hematologic cancer therapy over the past decade, infectious complications, and antimicrobial resistance (AMR) continue to pose significant threats to patients and clinical outcomes1.
  • Currently, there are no approved therapies for the prevention of bloodstream infections (BSIs) in hematological cancer patients.
  • SNIPR Biome is developing SNIPR001 to address this urgent unmet need to combat infections in hematological cancer patients.

SNIPR Biome receives funding from CARB-X to support advancement of CRISPR-medicine SNIPR001 into clinical trials in haematological cancer patients

Retrieved on: 
Monday, April 22, 2024
Bone marrow, FQR, CRISPR, Nature Biotechnology, Safety, Education, Pharmacokinetics, Infection, Neutropenia, Survival, AMR, Febrile neutropenia, Gene editing, Partnership, Bloodstream infections, Development, BMBF, Escherichia coli, Research and development, Patient, Quinolone antibiotic, Synthetic biology, HSCT, Pharmaceutical industry

Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.

Key Points: 
  • Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.
  • Despite the significant advances in hematologic cancer therapy over the past decade, infectious complications, and antimicrobial resistance (AMR) continue to pose significant threats to patients and clinical outcomes1.
  • Currently, there are no approved therapies for the prevention of bloodstream infections (BSIs) in hematological cancer patients.
  • SNIPR Biome is developing SNIPR001 to address this urgent unmet need to combat infections in hematological cancer patients.

Muscular Dystrophy Association and Friedreich’s Ataxia Research Alliance Announce Collaborative Research Grant Using Novel Gene Editing Technology to Address Root Cause of Friedreich’s Ataxia Disease

Retrieved on: 
Wednesday, April 17, 2024
Pillar, Enzyme, Friedreich's ataxia, Gene editing, CRISPR, GAA, Hope, Muscular dystrophy, Ataxia, FXN, MDA, Diabetes, FARA, Muscular Dystrophy Association, Column, RNA, Doctor of Philosophy, Research, Scoliosis, Parent, Schnucks, University of Massachusetts, Disease, Therapy, Biomedical Research, Nikolaus Friedreich, Fatigue, Movement, FA, Art, Genome, Organization, Pharmaceutical industry

This funding will further research into using novel genetic technologies to treat Friedreich’s ataxia (FA).

Key Points: 
  • This funding will further research into using novel genetic technologies to treat Friedreich’s ataxia (FA).
  • The grant, Paired Prime Editors to treat Friedreich’s Ataxia, involves prime editing (PE), a next-generation CRISPR gene editing tool that can precisely target the removal of the GAA expansions in the frataxin (FXN) gene.
  • “Our team of investigators is excited to bring multi-disciplinary expertise to the unique challenges of developing PE for FA.
  • This treatment method being targeted – prime editing – aims to directly address the cause of the disease, which is the GAA expansion in the FXN gene.

Nvelop Therapeutics Announces Members of Its Scientific Advisory Board

Retrieved on: 
Thursday, May 2, 2024
Health, Genetics, Other Science, Research, Science, Biotechnology, Harvard University, Danaher Corporation, Novartis, TIL, PACE, Howard Hughes Medical Institute, MIT, Gene therapy, Broad Institute, Gene editing, Duke University, Beam Therapeutics, Microbiology, Faculty, Harvard Medical School, Mammoth Biosciences, Therapy, Thomas Dudley Cabot, Aarhus University, Molecular genetics, Hospital, Arena, Biomedical engineering, RNA, Editas Medicine, Natural science, Bluebird bio, Kite Pharma, Torque, BioMarin Pharmaceutical, Patient, Gene, HSC, Massachusetts General Hospital, Growth, Pharmaceutical industry

Nvelop Therapeutics , a biotechnology company engineering programmable, non-viral vehicles for the in vivo delivery of therapeutic cargo, today announced the members of its scientific advisory board, bringing together a world-class group of experts from across the fields of gene therapy, gene editing and drug delivery.

Key Points: 
  • Nvelop Therapeutics , a biotechnology company engineering programmable, non-viral vehicles for the in vivo delivery of therapeutic cargo, today announced the members of its scientific advisory board, bringing together a world-class group of experts from across the fields of gene therapy, gene editing and drug delivery.
  • Keith Joung, M.D., Ph.D. : Dr. Joung is co-founder of Nvelop Therapeutics, and a pioneer in the development of targeted gene and epigenetic editing technologies.
  • He has co-founded multiple additional biotechnology companies, including Beam Therapeutics, Chroma Medicine, Editas Medicine, Pairwise Plants, SeQure Dx and Verve Therapeutics.
  • He is also the founder or co-founder of several biotechnology and therapeutics companies, including Beam Therapeutics, Prime Medicine, Editas Medicine, Pairwise Plants, Exo Therapeutics and Chroma Medicine.

Mekonos Enters Partnership Agreement with Accelerated Biosciences to Develop and Commercialize Engineered Human Trophoblast Stem Cells (hTSCs) for Biomanufacturing

Retrieved on: 
Thursday, May 2, 2024
Semiconductor, Health, Technology, Health Technology, Nanotechnology, Biotechnology, Immortalised cell line, Cell, GEMS, Gene editing, Partnership, Doctor of Philosophy, B cell, Cuprate superconductor, Cell engineering, Toxicology, Drug discovery, Medicine, Growth, Vaccine

Mekonos, Inc. , a biotech platform company building the future of cell engineering on a chip, today announced it has entered into a partnership agreement with Accelerated Biosciences Corp. (“Accelerated Bio”) to codevelop a powerful, new manufacturing cell line based on Accelerated Bio’s human Trophoblast Stem Cells (hTSCs) and Gene Engineered Multi-Sites (GEMS) technology.

Key Points: 
  • Mekonos, Inc. , a biotech platform company building the future of cell engineering on a chip, today announced it has entered into a partnership agreement with Accelerated Biosciences Corp. (“Accelerated Bio”) to codevelop a powerful, new manufacturing cell line based on Accelerated Bio’s human Trophoblast Stem Cells (hTSCs) and Gene Engineered Multi-Sites (GEMS) technology.
  • The hTSC platform is the earliest ethically sourced pluripotent stem cells that are naive, powerful, lively and have high plasticity.
  • These limitations compound and can cause cell functionality compromises and performance issues downstream, given the lack of consistency in engineered cells and inefficient scale-up.
  • Mekonos recognizes there is a better way, and we are rising to the challenge,” Narasimha added.