Genome Editing

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.

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.

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.

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.

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Sponsor: LonzaIn 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.

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.

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).

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.

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.