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

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

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

Start-up aims to advance transformative genetic medicines

Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

Genetics

Genetics Medicine Gene Editing In-Vivo

Leading innovators in CRISPR nucleases for the pharmaceutical industry

Pharmaceutical Technology

FEBRUARY 15, 2023

Cas, also called CRISPR associated proteins, are a family of nucleases such as Cas9, Cas12a, Cas12b, and CasX that process these sequences. The eukaryotic genome can be cut at any desired position by introducing plasmids containing Cas genes and specifically constructed CRISPRs into eukaryotic cells.

In-Vivo

In-Vivo Genome Genomics Gene Editing

Vertex eyes controllable genetic drugs with $1.3bn Obsidian alliance

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

Gene Editing

Gene Editing Genetics Drugs Gene

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

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

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

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

Turning science into business: An optimised alternative to antibodies

Drug Discovery World

APRIL 3, 2023

As the industry’s awareness of alternatives to traditional antibodies continues to grow, there is increasing demand for these alternatives, which we are seeing particularly within the therapeutic sector, to enable targeted therapeutics and enable the emerging gene therapy market. To what do you attribute your success?

Antibody

Antibody Gene Therapy Gene In-Vitro

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

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

Medicine

Medicine Development Clinical Trials Clinical Trials

This week in drug discovery (13-17 November)

Drug Discovery World

NOVEMBER 17, 2023

The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing. However, there were a number of other breakthroughs announced in the cell and gene therapy field.

Gene Therapy

Gene Therapy Gene Drugs Gene Editing

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. These efforts, along with many others, are opening doors for new possibilities in translational medicine.

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

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In million deaths worldwide” 1.

Drugs

Drugs Gene Clinical Trials Clinical Trials

Clinical Research Informer

Gene editing: beyond the hype

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

Trending Sources

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

Start-up aims to advance transformative genetic medicines

Leading innovators in CRISPR nucleases for the pharmaceutical industry

Vertex eyes controllable genetic drugs with $1.3bn Obsidian alliance

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

Gene editing in organoids: accounting for complexity in drug discovery

Delivering on the promise of gene editing

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

Turning science into business: An optimised alternative to antibodies

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

This week in drug discovery (13-17 November)

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

Advances in neuroscience drug discovery

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