Moderna inks another gene editing deal
Bio Pharma Dive
FEBRUARY 22, 2023
The messenger RNA specialist said Wednesday it is teaming up with Life Edit Therapeutics to develop therapies that can modify genes “in vivo.”
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Bio Pharma Dive
FEBRUARY 22, 2023
The messenger RNA specialist said Wednesday it is teaming up with Life Edit Therapeutics to develop therapies that can modify genes “in vivo.”
BioSpace
JUNE 12, 2022
Shape Therapeutics is evolving programmable RNA medicines that push the limits of current gene editing capabilities. BioSpace spoke with CSO David Huss.
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STAT News
NOVEMBER 14, 2022
Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA. On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.
Pharmaceutical Technology
FEBRUARY 23, 2023
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The post Moderna partners with Life Edit for mRNA gene editing therapies appeared first on Pharmaceutical Technology.
Pharmaceutical Technology
MAY 23, 2023
ReNAgade Therapeutics has burst onto the RNA technology scene by announcing a $300m financing round. With its delivery system, Cambridge, Massachusetts-based biotech says it aims to “address major limitations in RNA therapeutics by enabling the delivery of RNA medicines to previously inaccessible tissues and cells in the body.”
Bio Pharma Dive
FEBRUARY 28, 2023
An alliance with the startup brings Vertex an RNA-based medicine for the disease, adding to a portfolio that already includes a gene editing treatment.
Bio Pharma Dive
NOVEMBER 14, 2022
The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.
Bio Pharma Dive
AUGUST 18, 2022
The company plans to soon seek approval of a once-monthly preventive drug for hereditary angioedema, a disease other drugmakers are targeting with gene editing and RNA medicines.
BioPharma Reporter
FEBRUARY 23, 2023
The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.
pharmaphorum
NOVEMBER 1, 2022
14th Annual RNA Therapeutics. Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases. Date: 8 – 9 February 2023.
Pharmaceutical Technology
MARCH 28, 2023
Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.
Drug Discovery World
DECEMBER 13, 2022
Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene.
Pharmaceutical Technology
MAY 25, 2023
ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.
Drug Discovery World
OCTOBER 14, 2022
Meanwhile, Ochre Bio successfully raised $30 million to advance its pipeline of RNA therapies for liver disease, and researchers found that combining CRISPR gene editing with Spherical Nucleic Acids could expand the potential of the technology. . 30 million financing for RNA therapies for chronic liver diseases .
The Pharma Data
JUNE 7, 2023
Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells. Source link: [link]
Drug Discovery World
OCTOBER 17, 2022
Meanwhile, Ochre Bio successfully raised $30 million to advance its pipeline of RNA therapies for liver disease, and researchers found that combining CRISPR gene editing with Spherical Nucleic Acids could expand the potential of the technology. .
Camargo
JULY 27, 2021
Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.
Drug Discovery World
JANUARY 17, 2023
Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . The research aims to develop solutions for the limitations associated with adeno-associated virus (AAV), the current primary means of delivery for gene editing. .
Drug Discovery World
OCTOBER 17, 2022
Meanwhile, Ochre Bio successfully raised $30 million to advance its pipeline of RNA therapies for liver disease, and researchers found that combining CRISPR gene editing with Spherical Nucleic Acids could expand the potential of the technology. .
Drug Discovery World
JUNE 22, 2023
“This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”. The post Evox Therapeutics acquires exosome AAV technology from Codiak appeared first on Drug Discovery World (DDW).
XTalks
DECEMBER 14, 2022
Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.
Delveinsight
SEPTEMBER 14, 2021
ADARx bags USD 75 Million to advance its RNA tech pipeline. ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. SR One Capital Management and OrbiMed Advisors co-led Series B. Simeon George, M.D.,
XTalks
NOVEMBER 30, 2023
Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV. percent in the placebo group.
Drug Discovery World
DECEMBER 15, 2022
As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.
The Pharma Data
DECEMBER 13, 2020
“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO
Drug Discovery World
DECEMBER 21, 2023
In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article: Where are the hottest drug discovery hubs in the US?
Delveinsight
JANUARY 14, 2021
Last year, the company disclosed its gene-writing service, which combined various gene editing, manufacturing, and synthesizing technologies to provide more tailored therapeutic instructions to genetic code. Verve Therapeutics has an aim to bring one-and-done gene editing treatments to heart disease.
Drug Discovery World
OCTOBER 25, 2023
The neurons arrive ready-to-use and can enable researchers to achieve functional experimental readouts in days and without prior expertise in iPSC differentiation or guide RNA delivery optimisation.
The Pharma Data
MARCH 7, 2022
Cas9 is the protein used in the gene editing process called CRISPR. Cas9 binds and uses RNA as an address-tag. The system allows scientists, by synthesizing a specific “address-tag” RNA, to bring Cas9 to a precise location in genome and therefore cut and splice genes at specific sites. .”
Pharma Marketing Network
DECEMBER 21, 2020
those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. greater safety, transient and reversible effect on gene expression).
Drug Discovery World
MARCH 11, 2024
Researchers from Japan have used CRISPR gene editing to enhance the effectiveness of rejuvenated cytotoxic T lymphocytes (rejTs) for targeted immunotherapy. To overcome this issue, the team used CRISPR-Cas9 two-step ‘scarless’ gene editing on iPSCs derived from an HPV-specific cytotoxic T lymphocyte clone.
The Pharma Data
DECEMBER 5, 2020
The first of the new therapies, developed at Harvard Medical School by a team led by Dr. David Williams, uses a virus to introduce RNA into the extracted bone marrow that turns off BCL11A. The CRISPR gene editing method won the 2020 Nobel Prize in Chemistry, and uses chemicals to open up and directly edit DNA sequences, Hsu said.
Bioengineer
JULY 23, 2021
“By applying high-resolution imaging, advanced cell and molecular readouts and RNA sequencing methods leveraging the OTME-Chip, we discovered the actual genetic signaling pathways behind the blood cell triggered metastasis of ovarian cancer and a new drug strategy to stop this process.” ” ###.
Drug Discovery World
NOVEMBER 7, 2022
7 The collaboration will see Children’s Mercy Kansas City use PacBio’s Sequel IIe system in the study of genetic disease and the research will apply direct methylation detection and Iso-Seq full-length RNA sequencing.
XTalks
DECEMBER 22, 2021
The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?
Delveinsight
FEBRUARY 25, 2021
The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.
pharmaphorum
FEBRUARY 3, 2021
Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.
pharmaphorum
JANUARY 10, 2022
Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The biotech is focused on mRNA but has developed a self-amplifying mRNA (sa-RNA) platform in an attempt to develop the potential of the technology further.
Drug Discovery World
OCTOBER 16, 2023
A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. Cambridge is a focal point for many pharmaceutical companies working in this therapeutic area.
pharmaphorum
JULY 13, 2022
Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.
XTalks
AUGUST 29, 2022
Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.
The Pharma Data
JANUARY 19, 2021
A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.
Drug Discovery World
JANUARY 23, 2023
Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.
pharmaphorum
JUNE 28, 2021
The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.
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