Containment, Gene Editing and Genome - Clinical Research Informer

Study in mice shows potential for gene-editing to tackle mitochondrial disorders

Scienmag

FEBRUARY 8, 2022

Defective mitochondria – the ‘batteries’ that power the cells of our bodies – could in future be repaired using gene-editing techniques. Scientists at the University of Cambridge have shown that it is possible to modify the mitochondrial genome in live mice, paving the way for new treatments for incurable mitochondrial disorders.

Gene Editing

Gene Editing Gene Genome Genomics

New CRISPR-ready neurons could democratise genomics

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. With ioCRISPR-Ready Cells, we’re putting the future of genomics in the hands of every scientist.

Genome

Genome Genomics Gene Scientist

Leading innovators in CRISPR nucleases for the pharmaceutical industry

Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry CRISPR nuclease is a key innovation area in pharmaceutical development CRISPR, which refers to clustered regularly interspaced short palindromic repeats, are bacteriophage-derived DNA sequences that had previously infected the prokaryote and are found in the genomes of bacteria and archaea.

In-Vivo

In-Vivo Genome Genomics Gene Editing

Eterna acquires Exacis’ allogeneic immuno-oncology platform

Drug Discovery World

MAY 5, 2023

The technology underlying Exacis’ platform uses mRNA cell reprogramming and mRNA gene editing to create engineered iPSC-derived cells for use in the development of cancer therapies that can target nearly any cancer antigen.

Gene Editing

Gene Editing Engineer DNA Life Science

How Potential Changes to the NIH Guidelines Could Impact IBC Review

WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

Genome

Genome Genomics Gene Editing Genetic Engineering

New research begins to write the rules on CRISPR activation

Drug Discovery World

MARCH 21, 2023

CRISPR activation (CRISPRa) is a type of CRISPR gene editing that is used to overexpress certain genes. Although this technique is broadly used, predicting its efficiency when aimed at certain points in the genome can be challenging, making it hard to reliably overexpress certain genes.

Research

Research Gene Gene Editing Genome

Delivering on the promise of gene editing

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

Gene Editing Gene In-Vivo Genome

The regulatory round-up: Eight key FDA decisions

Drug Discovery World

APRIL 4, 2023

The approval is for endometrial cancer that has progressed on or following a prior platinum-containing regimen in any setting and are not candidates for curative surgery or radiation.

FDA Approval

FDA Approval In-Vivo Gene Editing Drugs

LogicBio Therapeutics Receives FDA Fast Track Designation for LB-001 for the Treatment of Methylmalonic Acidemia (MMA)

The Pharma Data

NOVEMBER 3, 2020

LogicBio’s proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio is headquartered in Lexington, Mass. For more information, please visit www.logicbio.com.

Clinical Trials

Clinical Trials Clinical Trials Gene Editing Genome

The doors CRISPR libraries have and will open in phenotypic drug screening

Drug Discovery World

MAY 2, 2023

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. In an effort to defend themselves against viral infection, bacterial cells capture and copy DNA fragments of bacteriophages into their genome.

Genome

Genome Genomics Gene DNA

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

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. Epigenetic Editing with CRISPR. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

DNA

DNA Gene Gene Silencing Genome

Editas Medicine Announces the FDA has Cleared Initiation of the EDIT-301 Clinical TrialEDIT-301 is in development as a best-in-class, durable medicine for people living with sickle cell disease

The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. Enrolled patients will receive a single administration of EDIT-301.

Medicine

Medicine Development Clinical Trials Clinical Trials

Women in Science Who Have Paved the Way Forward in Genetics

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

Genetics DNA Genome Genomics

BioSpace Global Roundup: Ipsen Wins Fast Track Designation for Cancer Drug and More

The Pharma Data

DECEMBER 2, 2020

ERS Genomics – Ireland’s ERS Genomics Limited, and Germany’s Vivlion GmbH, announced a non-exclusive license agreement granting Vivlion access to ERS Genomics’ CRISPR/Cas9 patent portfolio, to enhance Vivlion’s gene editing reagents and screening services. Financial details of the agreement were not disclosed.

Drugs

Drugs Gene Editing Genome Genomics

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

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. Epigenome Editing with CRISPR. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

DNA

DNA Gene Gene Silencing Genome

Clinical Catch-Up: January 18-22 | BioSpace

The Pharma Data

JANUARY 24, 2021

CYAD-101 is the company’s allogeneic NKG2D-receptor and T cell receptor inhibitory molecule-based, non-gene edited CAR-T candidate. VP-102 is a proprietary drug-device combination that contains a GMP-controlled formulation of cantharidim delivered by way of a single-use applicator. BT-001 is based on the patented Invir.IO

Trials

Trials Allergies In-Vitro Antibody

Necessity Is the Mother of Innovation: Key Trends and Predictions from Biotech Showcase Digital After an Unprecedented Year That Drove Momentous Changes in Healthcare

Pharma Marketing Network

JANUARY 20, 2021

Assuming these findings are replicated in clinical trials, this vaccine, with its more-appealing route of administration and convenient (refrigerator) storage requirements could make an important contribution to containing the disease.

Vaccination

Vaccination Vaccine In-Vivo Genetics

Clinical Research Informer

Study in mice shows potential for gene-editing to tackle mitochondrial disorders

New CRISPR-ready neurons could democratise genomics

Trending Sources

Leading innovators in CRISPR nucleases for the pharmaceutical industry

Eterna acquires Exacis’ allogeneic immuno-oncology platform

How Potential Changes to the NIH Guidelines Could Impact IBC Review

New research begins to write the rules on CRISPR activation

Delivering on the promise of gene editing

The regulatory round-up: Eight key FDA decisions

LogicBio Therapeutics Receives FDA Fast Track Designation for LB-001 for the Treatment of Methylmalonic Acidemia (MMA)

The doors CRISPR libraries have and will open in phenotypic drug screening

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

Editas Medicine Announces the FDA has Cleared Initiation of the EDIT-301 Clinical TrialEDIT-301 is in development as a best-in-class, durable medicine for people living with sickle cell disease

Women in Science Who Have Paved the Way Forward in Genetics

BioSpace Global Roundup: Ipsen Wins Fast Track Designation for Cancer Drug and More

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

Clinical Catch-Up: January 18-22 | BioSpace

Necessity Is the Mother of Innovation: Key Trends and Predictions from Biotech Showcase Digital After an Unprecedented Year That Drove Momentous Changes in Healthcare

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