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.

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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., LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery.

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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. . Limitations of AAV gene therapy . We’re hoping to use what we’ve learned so far about LNPs to develop an improved gene editor delivery system.” .

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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).

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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. .

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Gene Editing RNA Gene Drugs 52

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. .

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Drug Discovery World

DECEMBER 21, 2023

DDW’s top 10 most popular articles for 2023 reflect the key subjects in drug discovery and development. In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. This well-read article featured in the DDW Winter 22/23 issue’s Cell & Gene Therapy Guide.

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Gene Therapy Gene Antibody FDA Approval 59

Drug Discovery World

OCTOBER 25, 2023

bit.bio has launched the first offering from its new product range ioCRISPR-Ready Cells, which allow research and drug discovery scientists to knockout any gene of interest in glutamatergic neurons. The post New CRISPR-ready neurons could democratise genomics appeared first on Drug Discovery World (DDW).

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Genomics Genome Gene Scientist 52

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. Food and Drug Administration. SATURDAY, Dec.

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XTalks

NOVEMBER 30, 2023

In 2022, the US Food and Drug Administration (FDA) granted approval to the medication Sunlenca (lenacapavir) for adults living with multi-drug resistant HIV-1 infection. Developed by Gilead, Sunlenca is the first drug in a novel class known as capsid inhibitors to receive FDA approval for treating HIV-1.

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Drug Discovery World

JUNE 27, 2024

Scientists at the University of Sydney have developed a gene-editing tool with greater accuracy and flexibility than CRISPR. SeekRNA uses a programmable ribonucleic acid (RNA) strand that can directly identify sites for insertion in genetic sequences, simplifying the editing process and reducing errors.

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Gene Editing Gene DNA Genetic Engineering 59

Drug Discovery World

OCTOBER 16, 2023

The United Kingdom has established itself as a hub for drug discovery expertise worldwide. 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. billion in funding.

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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. Vor Biopharma’s AML drug secures Fast Track Designation.

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RNA Clinical Trials Clinical Trials Drugs 67

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. 14, 2020 10:00 UTC.

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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.

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Gene Editing Gene In-Vivo Genome 52

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. Many of these genes are related to inherited forms of cancer and heart conditions. . References .

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Genome Genomics Development Genetics 98

Delveinsight

FEBRUARY 25, 2021

Beam adds to drug delivery stable with USD 120 Million GuideTx buy. 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. GRAIL and Quest collaborate for the cancer blood test.

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DNA In-Vivo Genetics Medicine 52

Bioengineer

JULY 23, 2021

“We are creating a platform technology using the organ-on-a-chip approach where tumor biology can be advanced, and new drugs can be identified by recreating the platelet-tumor and platelet-tumor-drug interactions under the influence of flow, supporting blood vessels and the extracellular matrix,” Jain said. ” ###.

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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.

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Gene Editing Gene Engineer RNA 52

XTalks

SEPTEMBER 4, 2024

Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.

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Trials RNA Protein Gene Editing 52

Worldwide Clinical Trials

MAY 30, 2024

This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. The shared placebo design is another approach to accelerating drug development in ALS research.

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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.

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Gene Editing Gene DNA Engineer 98

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.

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Gene Editing Trials Drugs Gene 98

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. In three more patients treated at 0.1mg/kg levels fell by 52%.

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In-Vivo Gene Editing Trials Drugs 104

Drug Discovery World

MAY 2, 2023

Steve Wowk , VP, Business Unit and General Management of Integrated DNA Technologies, shared with DDW the value of CRISPR-Cas9 in drug discovery. The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system.

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Genome Genomics Gene DNA 52

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?

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Life Science RNA Vaccination Vaccine 98

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. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

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.

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Drug Discovery World

MAY 30, 2023

From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. Immunopheresis is a ‘subtractive therapy,’ in contrast to alternative drugs that constitute ‘additive’ therapies.

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Research Drugs Protein RNA 52

Drug Discovery World

JULY 18, 2023

Since its discovery in 2012, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has revolutionised the biomedicine and cell and gene therapy fields, providing a versatile tool for precise and efficient genome editing. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg.

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DNA Genome Genomics RNA 98

XTalks

AUGUST 2, 2023

Pfizer’s Top 5 Best-Selling Drugs of 2022: 1) Comirnaty Comirnaty is an mRNA-based vaccine indicated for the prevention of COVID-19. Comirnaty was first approved by the US Food and Drug Administration (FDA) in August 2021 for individuals over the age of 16. billion the drug generated in 2021. billion in 2022. billion, a 26.55

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Sales Manufacturing FDA Approval Life Science 98

Drug Discovery World

NOVEMBER 30, 2023

Next-generation immunotherapies In the session focused on cell-based immunotherapies, Steven Quistad, Senior Applications Scientist, DNA Script, presented on how to rapidly assemble genes in a laboratory using automated enzymatic oligo synthesis. To demonstrate the utility of the company’s SYNTAX system in gene assembly, the 1.7

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Drug Discovery World

FEBRUARY 22, 2024

Lu Rahman looks at neuroscience drug discovery – advances and challenges – and how breakthroughs in this field are helping address areas of unmet need. Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research.

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XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. Drugs, Testing and Vaccines. Drug Approvals.

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Pharmaceutical Technology

FEBRUARY 20, 2023

On the other hand, a lack of diversity in clinical trial populations, particularly when the disease population is significantly underrepresented, greatly diminishes the quality of data obtained for drug safety and efficacy profiles. This may sound obvious, but it is still a major hurdle in drug development.

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