Fierce Biotech

Intellia races in vivo CRISPR therapy to FDA after phase 3 data paint ‘compelling’ picture

A phase 3 trial of Intellia Therapeutics’ in vivo gene-editing therapy lonvoguran ziclumeran (lonvo-z) has hit its primary ...
BioSpace

Gene therapy leaders aim to help more Baby KJs with novel regulatory models

As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” ...
Labiotech.eu

Modified CRISPR tool targets Down syndrome mutation

Researchers have used modified CRISPR to silence the extra chromosome 21 in Down syndrome cells, showing promising ...
Hosted on MSN

CRISPR breakthrough and new drug wins reshape pharma landscape

Intellia’s in vivo CRISPR gene therapy for hereditary angioedema cut attack rates by 87% in a Phase III trial, prompting a rolling FDA submission. Novartis secured EU approval for the first oral ...
Duke Pratt School of Engineering

Beyond the Cut: A New Era for CRISPR Medicine

Charles Gersbach and his colleagues are pursuing promising CRISPR technologies focused on controlling gene activity rather ...
BioSpace

Intellia heads to FDA after notching first late-stage win for in vivo gene editor

With Phase 3 data in hand, Intellia Therapeutics is seeking approval for its in vivo CRISPR gene editing therapy for ...
Labroots

CRISPR Screening Made Easy with the Revvity Dharmacon™ All-in-One Lentiviral Platform

Revvity's Dharmacon All-in-one lentiviral platform has expanded to include whole-genome library options for CRISPR knockout (CRISPRko), CRISPR interference (CRISPRi), and CRISPR activation ...
Healio

Advanced gene editing ‘promising’ for sickle cell disease, but access challenges remain

Two papers published in The New England Journal of Medicine highlight the potential of gene editing for treatment of sickle ...
Hosted on MSN

CRISPR reveals key five-gene protein role in plant growth

Rice University scientists used advanced CRISPR techniques to study PEX11, a protein coded by five genes in Arabidopsis plants, revealing its role in controlling peroxisome growth during the seed-to ...