DNA CRISPR code on computer screen with hand. Biology and research.

A Toolbox for Keeping CRISPR in Check

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 Partnering with Catalent’s Paragon to Manufacture Gene Therapies

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 Boosting Pipeline, Manufacturing, Global Presence with $325M in Series B Financing

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.
Venus symbol pattern.

CRISPR System Ensures Female-Only Progeny in Mammals

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.

With VEGAS System, Faster Directed Evolution Could Be a Sure Thing

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.

Drones Land in the Cell and Gene Therapy Space

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.
HIV infection, artwork

CRISPR and LASER ART Eliminate HIV from Mice

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, Thermo Fisher’s Brammer Bio Partner on Gene Therapy Manufacturing

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.
Pair of scissors

Live CRISPR-Cas9 Loaded with Short RNA Deadens Transcription of Targeted Genes

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

CRISPR-Accelerated Gene Drives Pump the Brakes

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.

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