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

XTalks

MAY 12, 2021

Dubbed “CRISPRoff,” the technology targets the epigenome to silence genes involved in diseases, with applications in cancer, AML and other conditions with a heritable component. Subscribe to the Xtalks Life Science Podcast to never miss a new episode. New Study Looks at Effects of ADHD Drugs in Preschoolers.

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Life Science Gene Expression Gene DNA 52

Drug Discovery World

NOVEMBER 27, 2023

By Anis H Khimani , PhD, Senior Strategy Leader, Life Sciences strategy group, Revvity, Inc. 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.

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Production Engineer Gene Editing Gene 59

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. These companies are at various stages of research and clinical trials.

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Life Science Gene Editing Development Clinical Trials 119

XTalks

MARCH 28, 2024

Building Cancer Awareness and Empowering Métis Youth: Featuring Jordyn Playne, President, Métis Nation of Ontario Youth Council – Xtalks Life Science Podcast Ep. 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

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.

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XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. Given the system’s powerful ability to make cuts in genes in a precise manner, the system is being leveraged in gene therapies.

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Gene Therapy Gene Gene Editing In-Vivo 59

Drug Discovery World

OCTOBER 16, 2023

Maina Bhaman, Partner at Sofinnova Partners, says: “The UK is an attractive space for drug discovery companies and a prime environment for investment due to its talent, infrastructure, and genomics research. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

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Drugs Life Science Gene Therapy Genome 75

Delveinsight

JANUARY 14, 2021

Altitude Life Science Ventures, and the second SoftBank Vision Fund, with participation from the Qatari Investment Authority and other undisclosed investors. Verve Therapeutics has an aim to bring one-and-done gene editing treatments to heart disease. The round was led by Alaska Permanent Fund Corp.,

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Gene Editing Gene Genetics DNA 40

XTalks

NOVEMBER 30, 2023

In 2021, the FDA granted approval to San Francisco-based Excision BioTherapeutics to start trials assessing CRISPR gene editing as a potential treatment for HIV. The noteworthy candidate, EBT-101, is designed to eliminate HIV proviral DNA using CRISPR-Cas9 along with two guide RNAs (gRNAs).

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

The Pharma Data

JANUARY 12, 2021

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. Photo courtesy of Bayer. The platform is already bearing fruit.

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Gene Therapy Gene Gene Editing Licensing 52

XTalks

JULY 14, 2022

CRISPR-Cas9 gene editing was used to simultaneously perturb thousands of genes by targeting specific points of genetic code. The focus was on genes that create transcription factors , which are proteins that switch genes on or off and therefore control multiple other genes at once, including the three immune factors of interest.

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Gene Regulation Protein Gene Editing 98

Pharmaceutical Technology

FEBRUARY 13, 2023

This is particularly the case for pediatric forms of epilepsy where gene editing techniques like Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) are becoming more important, says Ferraro. In his work, Ferraro focuses on the links between gene variations and different responses to drugs.

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Research Gene Gene Therapy Genome 264

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.

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Gene Editing DNA Gene Genome 98

Drug Discovery World

JANUARY 26, 2023

Colin Terry, European Life Sciences R&D leader at Deloitte, explains: “Revitalising today’s clinical trials processes and operations will drastically improve trial efficiency, enhance science-based decisions, and expand collective health equity. “AI

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Development Drugs Clinical Trials Clinical Trials 98

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

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DNA Gene Gene Silencing Genome 98

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. While type II (Cas9) is mainly used for gene-editing applications, CRISPR detecting tools primarily rely on type V (Cas12) and type VI (Cas13) collateral activity.

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pharmaphorum

AUGUST 31, 2022

One area of the gene therapy space that could become a major treatment modality is CRISPR technology. The key to why this technology could become central to gene editing is due to the specificity with which it can edit DNA and that it is also a relatively cheap process, compared with existing technologies.

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Gene Therapy Gene Gene Editing DNA 98

XTalks

FEBRUARY 11, 2021

The Human Genome Project recently marked 20 years since the publication of the first full sets of human genomic sequences, an endeavor that spanned well over a decade. Today, new next-generation sequencing technologies allow for the sequencing of complex genomes within just a day or two.

Genetics 119

Genetics DNA Genome Genomics 119

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

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DNA Gene Gene Silencing Genome 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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Research Drugs Gene Therapy Clinical Trials 59

XTalks

DECEMBER 24, 2020

The pandemic propelled the life science and healthcare sectors onto center stage, and they rose to the occasion against the most unprecedented health challenge in recent times. While COVID-19 has undoubtedly been the biggest story in the life science industry in 2020, it was a busy and positive year in many other areas.

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Life Science Vaccination Vaccine Gene 111

XTalks

SEPTEMBER 21, 2020

This difference could be mitigated when the scientists used a gene editing technique to either reduce KDM6A protein levels in the female-derived neurons, or increase KDM6A levels in neurons from males. Increasing KDM6A expression in male mice led them to be more resilient to the effects of amyloid beta plaques.

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Gene Protein Gene Expression Scientist 89