XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

JUNE 22, 2023

Evox Therapeutics has purchased Codiak Biosciences engEx-AAV technology platform, including all intellectual property rights, and exclusive access to certain engineering and manufacturing rights.

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Gene Therapy Gene Editing Drug Delivery Gene 52

Drug Discovery World

NOVEMBER 27, 2023

Upstream cell line development has seen notable progress with recent advances in cell line engineering, particularly in enhancing gene of interest integration specificity, clone selection, and production efficiency. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.

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Production Engineer Gene Editing Gene 59

Drug Discovery World

OCTOBER 10, 2023

The National Institutes of Health has awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome.

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Gene Editing Development Drugs Clinical Trials 52

Drug Discovery World

DECEMBER 12, 2022

Dr Ramakrishnan, who holds a Bachelor of Biotechnology and Biochemical Engineering from the University of Kerala in India, presented a proposal examining microbiome-based therapeutics for asthma. . The study advances our understanding of ADHD by showing how the condition is tied to changes in how certain genes are expressed in the brain.

Drugs 52

Drugs Gene Editing Genome Genomics 52

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

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Life Science Gene Editing Development Clinical Trials 119

XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

Drug Discovery World

DECEMBER 15, 2022

Vor Bio, a clinical-stage cell and genome engineering company, has announced initial clinical data from VBP101, its Phase I/IIa multicentre, open-label, first-in-human study of tremtelectogene empogeditemcel or “trem-cel” (formerly VOR33) in patients with acute myeloid leukemia (AML). .

Drugs 52

Drugs Genome Genomics Engineer 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genome 98

Cloudbyz

MARCH 6, 2024

Personalized Medicine: Biologics offer the potential for personalized treatment approaches by taking into account individual variations in genetics, immune responses, and disease characteristics. Looking ahead, the future of pharmaceutical therapies is undeniably intertwined with the continued advancement of biologics.

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Gene Immune Response Manufacturing Gene Therapy 72

XTalks

JULY 14, 2022

A downstream approach has traditionally been used by scientists to map networks, wherein a gene for a protein is removed one at a time and the impact on the immune cell’s function is observed. CRISPR-Cas9 gene editing was used to simultaneously perturb thousands of genes by targeting specific points of genetic code.

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Gene Regulation Protein Gene Editing 98

Drug Discovery World

JANUARY 12, 2023

The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. Altering one base pair can drastically change how the gene’s protein product functions, while mutations can lead to the development of a range of genetic disorders. . Limitations of organoids.

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Gene Editing Gene Drugs In-Vivo 98

The Pharma Data

FEBRUARY 22, 2021

Gene editing investments advance agriculture with improved food quality, higher yield, and improved crop nutrition. Gene editing is the key to better solutions and greater choices for farmers and consumers around the world. This enhances sustainability and helps us reduce the environmental impact of agriculture.”.

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Gene Editing Genome Genomics Protein 40

The Pharma Data

SEPTEMBER 1, 2021

Louis have genetically engineered cells that, when implanted in mice, will deliver a biologic drug in response to inflammation. The engineered cells reduced inflammation and prevented a type of damage to bone, known as bone erosion, in a mouse model of rheumatoid arthritis. ” The new findings are published online Sept.

Drugs 52

Drugs Engineer Drug Delivery Gene 52

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.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Epigenome Editing with CRISPR.

DNA 52

DNA Gene Gene Silencing Genome 52

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize. The illness was later named coronavirus disease 2019 (COVID-19).

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Life Science Vaccination Vaccine Gene 111

XTalks

NOVEMBER 30, 2023

More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease. In addition, Vir Biotechnology, Inc.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Delveinsight

FEBRUARY 25, 2021

Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. 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.

DNA 52

DNA In-Vivo Genetics Medicine 52

Drug Discovery World

DECEMBER 12, 2022

The meeting will focus on the application of genome engineering in identification of novel drug targets through large-scale CRISPR based functional genomics studies, developing advanced cellular and in vivo disease models for target validation, and its applications in therapeutic genome editing. Register here.

Genome 52

Genome Genomics Engineer In-Vivo 52

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 96

Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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Research Gene Gene Therapy Genome 264

Drug Discovery World

OCTOBER 16, 2023

From the Beatson Institute for Cancer Research in Glasgow, to Edinburgh Drug Discovery in the University of Edinburgh’s Institute for Genetics and Cancer (IGC). The UK’s infrastructure is bolstered by initiatives such as the Cell and Gene Therapy Catapult, supporting the research and manufacturing of cutting-edge therapies.

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Drugs Life Science Gene Therapy Genome 75

Roots Analysis

FEBRUARY 15, 2022

Presently, there are several companies and universities, which are exploring the potential of different gene editing technologies beyond CRISPR for basic research, and the development of gene editing solutions. Genome Editing is a way of making changes in the DNA.

Genome 52

Genome Genomics Gene Editing DNA 52

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 52

Advarra

NOVEMBER 29, 2022

Bottlenecks in the manufacturing process, supply chain issues such as accessibility of good manufacturing practice (GMP)-grade reagents, and shortages of qualified scientists and engineers have caused many therapies to fail at critical stages of the clinical development pipeline.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

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.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

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. In bacteria with unaltered tracr-L, levels of CRISPR-related genes were low. The authors found that tracr-L redirects Cas9 in S.

Bacteria 52

Bacteria Gene Editing Research RNA 52

Drug Discovery World

JANUARY 26, 2023

At the height of the pandemic, DCTs were seen as a viable alternative to keeping the engines running on clinical trials when non-critical in-person care was paused. Opportunities will progress for use cases such as cancer diagnoses based on liquid biopsies, predicting cancer progression and improving gene editing tools potential (e.g.

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Development Drugs Clinical Trials Clinical Trials 98

Drug Discovery World

MAY 30, 2023

However, current TME remodelling therapies such as engineered cytokines or immune cell agonists usually come with strong sides effects, which reduces therapeutic index. One of the most exciting recent developments is the use of gene editing tools to create cell and gene therapies.

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Research Drugs Protein RNA 52