Gene Editing, Genomics, In-Vivo and Protein - Clinical Research Informer

FDA clears in vivo gene editing therapy for trials in US

Drug Discovery World

MARCH 9, 2023

This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures. The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW).

In-Vivo

In-Vivo Gene Editing Gene Trials

Gene editing: beyond the hype

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

Gene Editing

Gene Editing Gene Gene Therapy In-Vivo

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

Single-course in vivo base editing therapy proven to lower cholesterol

Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol.

In-Vivo

In-Vivo Gene Editing Gene Genome

Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

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

In-Vivo Gene Editing Trials Gene

Gene editing in organoids: accounting for complexity in drug discovery

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. . What are organoids? .

Gene Editing

Gene Editing Gene Drugs In-Vivo

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

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. . — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo

In-Vivo Gene Editing Trials Drugs

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

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

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., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

Gene Therapy

Gene Therapy Gene Gene Editing In-Vivo

Start-up aims to advance transformative genetic medicines

Drug Discovery World

FEBRUARY 16, 2023

Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes. The post Start-up aims to advance transformative genetic medicines appeared first on Drug Discovery World (DDW).

Genetics

Genetics Medicine Gene Editing In-Vivo

CRISPR breakthroughs: New solutions for common diseases

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. Years of development and fast-paced research have continued to unlock its potential, expanding how CRISPR can be used to treat, detect, and prevent common diseases such as cancer and Covid-19.

DNA

DNA Genome Genomics RNA

CRISPR Cas-9 System Doubles Life Expectancy in Mice with Aggressive Cancers

The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. Photo courtesy of Science Advances.

Gene Editing

Gene Editing DNA Genome Genomics

The use of base editing in stem-cell based therapies

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

Gene Editing Gene Engineer DNA

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. CAMBRIDGE, Mass., 11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc.

Medicine

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Clinical Research Informer

FDA clears in vivo gene editing therapy for trials in US

Gene editing: beyond the hype

Trending Sources

Delivering on the promise of gene editing

Single-course in vivo base editing therapy proven to lower cholesterol

Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

Gene editing in organoids: accounting for complexity in drug discovery

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

Leading innovators in CRISPR nucleases for the pharmaceutical industry

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

Start-up aims to advance transformative genetic medicines

CRISPR breakthroughs: New solutions for common diseases

CRISPR Cas-9 System Doubles Life Expectancy in Mice with Aggressive Cancers

The use of base editing in stem-cell based therapies

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

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