Drug Discovery Today

OCTOBER 27, 2022

27, Vilnius will host the EFIB’2022 event which will bring the European life science sector together. In the field of life sciences, Lithuania is best known for the discovery of gene scissors by Prof. Lab, focused on research and development of novel genome editing tools. Virginijus Šikšnys.

Genomics 40

Genomics Genome Gene Editing Life Science 40

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.

Life Science 40

Life Science Gene Expression Gene DNA 40

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 Genomics 98

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.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

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.

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

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.

Gene 98

Gene Regulation Protein Gene Editing 98

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.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

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 Gene 111

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.

DNA 52

DNA Gene Gene Silencing Genomics 52

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.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

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.

Gene Therapy 52

Gene Therapy Gene Gene Editing Business Development 52

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.,

Gene Editing 40

Gene Editing Gene Genetics DNA 40

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.

Research 59

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.

Life Science 111

Life Science Vaccination Vaccine Gene 111

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 Genomics Genome 119

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.

Gene 89

Gene Protein Gene Expression Scientist 89

XTalks

JANUARY 6, 2025

Gene Editing CRISPR-Cas9 and related gene-editing technologies continue to advance, and today they are widely used to study and develop therapeutic approaches for a broad range of human diseases. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111