Realizing The Promise: How Personalized Gene Editing Can Treat Ultra-Rare Diseases

With a shared purpose, we can realize the true promise of CRISPR and improve healthcare, providing hope to more patients with ...

Gene-editing therapy proves effective for Leber's hereditary optic neuropathy

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...
Good News Network

Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’

Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
New Atlas

Gene editing takes a major step toward ending daily cholesterol pills

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
Technology Networks

Gene Editing Could Soon Be Applied Like A Skin Cream

A new skin-applied gene therapy uses lipid nanoparticles to deliver gene editing into skin stem cells, correcting ...

Bionano Announces Publication from Sanford Burnham Prebys Medical Discovery Institute Describing use of OGM to Detect Genomic Alterations Introduced by Gene Editing Technologies

OGM was selected as a genome-wide, unbiased method to detect large genomic rearrangements and structural variants (SVs) with sensitivity to variant allele fractions (VAFs) as low as 5% to analyze ...
STAT

FDA’s new ‘plausible mechanism pathway’ for personalized gene editing raises concerns

In November, six months after KJ’s debut, top Food and Drug Administration officials Marty Makary and Vinay Prasad published ...
Fierce Biotech

Touching base: Beam CEO on gene editing biotech's 'big push' for in vivo delivery

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...
Fierce Biotech

Lilly pens $1.1B pact with German biotech to work on gene editing med for hearing loss

Eli Lilly is continuing a run of dealmaking to strengthen its genetic medicine offering by penning a pact with gene editing ...
Drug Target Review

New CRISPR-based gene-editing treatment targets root cause of kidney disease

Explore groundbreaking gene-editing therapy for kidney disease, offering hope to those with polycystic kidney disease.
BioPharma Dive

GSK walks away from pioneering Wave RNA editing drug

Wave claimed GSK is handing back rights to the frontrunning program not because of questions about its potential, but because ...
EurekAlert!

Breakthrough in Gene-Editing Therapy for Leber’s Hereditary Optic Neuropathy

A research team led by Professor Hyunji Lee of the Department of Convergence Medicine at Korea University College of Medicine (co-first authors: PhD candidates Sanghun Kim, Ji Eun Kim, and Sungjin Ju; ...