Intellia Therapeutics says its CRISPR-based treatment succeeds in pivotal trial

Intellia Therapeutics said its Crispr-based treatment for hereditary angioedema met its goals in a Phase 3 trial, marking a ...

Intellia Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for Lonvoguran Ziclumeran (lonvo-z) as a One-Time Treatment for Hereditary Angioedema

Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, today announced it has ...

Intellia Therapeutics Reports Positive Phase 3 Results in Hereditary Angioedema, Marking a Global First for In Vivo Gene Editing

Phase 3 HAELO trial of lonvoguran ziclumeran (lonvo-z) met primary and all key secondary endpoints; favorable safety and tolerability data ...
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” ...

CRISPR takes a bold leap toward silencing Down syndrome's extra chromosome

Scientists have taken an important step toward a gene therapy that could one day turn off the extra genetic material that ...
GEN

Center for Pediatric CRISPR Therapies Founded by IGI, CZI

The recent clinical success of treating “Baby KJ” Muldoon—an infant born with a rare metabolic disease—with the first-ever personalized gene-editing therapy brought much-needed enthusiasm to the ...
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 ...
The Business Journals

Here's how the Chan Zuckerberg Initiative and Nobel winner Jennifer Doudna plan to cure more kids

Nicole and Kyle Muldoon with their child, KJ, who received a CRISPR gene editing therapy at Children's Hospital of Philadelphia. From the San Francisco Business Times. Months after helping manufacture ...
GEN

Fyodor Urnov Discusses CRISPR Cures

In a keynote talk during GEN’s “The State of CRISPR & Genome Editing” virtual summit, originally broadcast on June 11, 2025, Fyodor Urnov, PhD, Director, Technology & Translation, Innovative Genomics ...
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

Novartis wins EU approval as Intellia rallies on CRISPR hopes

Novartis secured European Commission approval for Rhapsido, the first oral targeted therapy for chronic spontaneous urticaria, while Intellia shares surged ahead of historic Phase 3 data for an ...