Adeno-associated virus, illustration

Several Age-Related Diseases, One Gene Therapy Combo, Multiple Improvements

The Wyss Institute reports that a single administration of an adeno-associated virus–based gene therapy delivering combinations of three longevity-associated genes to mice dramatically improved or completely reversed multiple age-related diseases, suggesting that a systems-level approach to treating such diseases could improve overall health and lifespan.

A Breath of Fresh CRISPR

If airway epithelia could be more easily modified by therapeutic proteins, including genome editing proteins, progress against various respiratory diseases would be enhanced. But such proteins lack effective delivery mechanisms, particularly since airway epithelia pose significant barriers to viral and nonviral transduction platforms. An alternative platform incorporates engineered amphiphilic peptides. It has shown promise delivering CRISPR proteins to cultured human epithelial cells and mouse airway epithelia.
CytoSeek Protein and Peptide

Synthetic Biology Seizes New Ground in Healthcare

Don’t fight the last war. Start field-testing synbio apps such as programmable biopolymers, therapeutics derived from synthetic DNA, and “bugs as drugs”—living platforms for drug and vaccine delivery.

Agrigenomics Yields a Next-Gen Cornucopia

Besides harvesting a new generation of modified plants, genome engineers are cultivating a new image. Rather than be thought of as GMO brutes, they’re using gene editing to show good breeding.

Improve your Gene Editing Success with CRISPR-SNIPER Technology

Sponsored content brought to you by Is your CRISPR workflow labor intensive and prolonged? Inefficient for your gene editing requirements? The new CRISPR-SNIPER technology and...

CRISPR is Editing its Way Toward Cell and Gene Therapy

In this GEN webinar, sponsored by Lonza, we will hear about a high-throughput platform for CRISPR-Cas9 gene editing in primary human cells for the robust functional interrogation of complex biological processes and disease states. We also learn about the clinically relevant scale of gene-editing, platforms, and approaches, process development considerations for bringing these gene-edited therapies to the clinic.
Firm had previously rejected Astellas’ unsolicited bid of roughly $3.5 billion. [© Yuri Arcurs -]

Forces Unite to Move Molecular Dx Out of the Gates

Sherlock Biosciences has announced that they are joining forces with two partners — the Bill & Melinda Gates Foundation and the lateral flow company Mologic — to help make their goals a reality as quickly as possible. The partnership, they hope, will lead to the to development of a simple, cost effective, rapid and highly sensitive test for infectious diseases.

FDA Places Partial Clinical Hold on Some Trials of Novartis Gene Therapy

The FDA has imposed a partial hold on clinical trials for intrathecal administration of the Novartis gene therapy AVXS-101, which won the FDA's first approval for treating some forms of spinal muscular atrophy (SMA) in May under the name Zolgensma® (onasemnogene abeparvovec-xioi).
Firms will also work together to develop diagnostics over the next two and a half years. [© AlienForce -]

On Higher Ground: The Dilemma on Gene Therapy Pricing

Katherine High, MD, the co-founder, president, and CSO of Spark Therapeutics speaks about the commercial success and renaissance of gene therapy. High is the developer of Luxturna, the first approved gene therapy for a hereditary form of blindness called Leber’s congenital amaurosis.
ESGCT 2019

Promising Results Reported in Tay-Sachs Gene Therapy Trial

Terence R. Flotte, MD, executive deputy chancellor, provost, and dean of the University of Massachusetts School of Medicine, presented the first signs of clinical benefit in an early-stage gene therapy trial for Tay-Sachs disease on two infants. Flotte said there are early signs that the therapy, which in 2018 was licensed to Axovant Gene Therapies, has the potential to modify the rate of disease progression.

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