Drug Discovery World

JANUARY 17, 2023

They will lead research into using LNPs to deliver a gene editing tool that could delete bad genes in the photoreceptor cells and replace them with correctly functioning genes. . AAV has limited packaging capacity compared to LNPs and it can prompt an immune system response,” Sahay said.

Gene Editing 98

Gene Editing Gene Gene Therapy Genetics 98

Drug Discovery World

FEBRUARY 16, 2023

The company’s protein nanoparticle (PNP) genetic medicine delivery platform is based on the discovery of endogenous, human proteins derived from retroelements that can self-assemble to form capsid-like structures and which can package and transfer nucleic acid cargo.

Genetics 52

Genetics Medicine Gene Editing In-Vivo 52

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 52

Gene Editing Gene In-Vivo Genome 52

STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The clinical trial was to be the first time anyone got a gene editing therapy for muscular dystrophy.

Gene Editing 105

Gene Editing Gene Gene Therapy Clinical Trials 105

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. . The post Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug appeared first on.

In-Vivo 100

In-Vivo Gene Editing Trials Drugs 100

XTalks

AUGUST 2, 2023

Pfizer’s Top 5 Best-Selling Drugs of 2022: 1) Comirnaty Comirnaty is an mRNA-based vaccine indicated for the prevention of COVID-19. Comirnaty was first approved by the US Food and Drug Administration (FDA) in August 2021 for individuals over the age of 16. billion the drug generated in 2021. billion in 2022. billion in 2022.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

Drug Discovery World

MAY 2, 2023

Steve Wowk , VP, Business Unit and General Management of Integrated DNA Technologies, shared with DDW the value of CRISPR-Cas9 in drug discovery. 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.

Genome 52

Genome Genomics Gene DNA 52