Genome Editing

A pair of small molecules identified that could efficiently penetrate cells and efficiently and reversibly inhibit Cas9’s ability to cut genomic DNA without noticeable toxic effects. This work now gives investigators an effective workflow for not only identifying Cas9-modulating compounds, but also for exploring the mechanisms by which those compounds work.

Passage Bio, a developer of gene therapies for rare monogenic CNS diseases launched five months ago, will partner with Catalent’s Paragon Gene Therapy unit to develop a dedicated manufacturing suite at Paragon’s Harmans, MD, facility.

BioNTech's $325 million Series B financing proceeds will be used not only toward advancing its pipeline of immuno-oncology candidates, but adding to its manufacturing capacity and exploring an expanded global footprint as well.

Paternal and maternal mouse cell lines have been engineered to carry genome-editing components such that in crossed lines, all the components needed to target vital genes are present in male embryos, ensuring their elimination. Alternatively, the components could be distributed to ensure the targeting of female embryos.

A directed evolution system, VEGAS, works in mammalian cells and can yield useful new molecules within days, providing scientist with a powerful new research tool and a potential route to better therapeutics.

In collaboration with RPS Aerospace, the Italy-based biotech company Anemocyte developed a remotely piloted drone system to securely transport final cell and gene therapy products from manufacturing sites to clinical centers.

Although antiretroviral therapy has turned HIV into a chronic disease, a cure has eluded virologists. New research shows that a combination of long-acting slow-effective release antiviral therapy (LASER ART) and CRISPR-Cas9 successfully eliminated HIV from infected humanized mice. Although an interesting step forward, the road to a cure for HIV in humans is a long one with much more work to be done.

Amicus Therapeutics will address clinical and commercial supply needs for its intrathecal AAV Batten disease gene therapy programs by partnering with Thermo Fisher Scientific’s recently-acquired Brammer Bio.

In bacteria, catalytically active CRISPR-Cas9 programmed by short guide RNA can repress transcription of targeted genes without cutting DNA.

With CRISPR purring under the hood, gene drive is ready to shift into high gear. It also boasts superior handling thanks to new features: like split drives, daisy chains, and small-molecule control.