Roche Extends Offer for Spark Therapeutics a Second Time, as Shareholders Balk at Tender...

Roche said today it will extend for a second time its tender offer for shares of Spark Therapeutics, the gene therapy developer it wants...
Doctor examines DNA molecules.

Gene Therapy Leaders Urge Global Moratorium on Germline Editing

On the eve of DNA Day, leaders in the gene therapy community have co-signed a letter to Alex Azar, the Secretary of the Department...

Takeda Inks Licensing Option Agreement with Gene Editing Platform Developer Emendo

Takeda Pharmaceutical has advanced further into gene editing, inking a licensing option agreement with and, through its venture capital arm, investing in Emendo Biotherapeutics,...

CRISPR-Cas9 Goes Immuno-Incognito

Before CRISPR-Cas9 can be used with confidence in the human body, it must demonstrate that it is safe—and not just with respect to off-target...

University of California, U. Vienna, Charpentier Awarded Fourth U.S. CRISPR-Cas9 Patent

A fourth U.S. patent covering CRISPR-Cas9 claims was granted to the Regents of the University of California, the University of Vienna, and CRISPR pioneer Emmanuelle Charpentier, PhD, director and scientific member at the Max Planck Institute of Infection Biology. The patent focuses on systems and methods for using CRISPR/Cas9 technology in a single guide format, including uses to target and edit or modulate genes.
girl with cancer

Personalized, Combination Therapies Improve Cancer Patient Outcomes

Researchers at the University of California, San Diego (UCSD) School of Medicine report that treating patients with personalized, combination therapies improved outcomes in patients...
CRISPR

DISCOVER-Seq Detects Off-Target CRISPR Effects

A concern that has plagued CRISPR-Cas9 technology from the beginning is the possibility, and unknown effect, of off-target effects—DNA mutations that result from CRISPR-Cas9...

Gene Circuits Wired into Cells Elicit Switch-Like Behavioral Output

Electronic gizmos and living cells alike can process signals, turning inputs into outputs—even recognizing when gray-area signaling is dark enough, or light enough, to...

Gene Therapy Cures Babies with “Bubble Boy” Disease

In a first for patients born with the deadly X-SCID immunodeficiency disease, in which immune cells do not develop or function normally, a safe and effective gene therapy has provided a cure. The eight babies involved in the study have functioning immune systems, normal growth, and are busy toddlers able to explore their surroundings.

CRISPR Base Editors Are Often Off Base with RNA

CRISPR base editors are supposed to be parsimonious. They avoid inflicting anything so extreme as a double-strand break, and they confine their genomic activities...

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