Genome Editing

Scientists at Harvard University have engineered virus-like particles that overcome three bottlenecks to protein delivery that reduce the efficacy of VLP-mediated protein delivery: cargo release, cargo packaging and localization, and VLP component stoichiometry. The study showed the new mode of DNA- and virus-free delivery efficiently transports base editor proteins and guide RNAs to target cells in multiple organs with minimal off-target editing in live newborn and adult mice.

A preclinical study provides proof-of-concept evidence for a new gene therapy approach that offers a potential long-term treatment for limb-girdle muscular dystrophy 2B. Researchers at Children’s National have developed the first non-muscle targeted gene therapy that enhances diseased muscle fiber repair and improves muscle function through a single low dose of an AAV vector that produces human acid sphingomyelinase, released by muscle cells immediately following muscle injury.

Vascular endothelial cells serve as a protective barrier in blood-vessel walls and serve as an active source for the synthesis, metabolism, uptake, storage, and degradation of a number of vasoactive substances. Endothelial cell damage can also be a factor in diseases. Now, a new mouse study demonstrates how the vascular endothelial cells could be reached through genome editing. 

Gene editing history will have a lot to say about 2021. CRISPR-Cas9 systems are attaining greater power and precision, and alternatives such as homing nuclease and “gene writer” systems are becoming more capable, too.

Technological developments and therapeutic applications of third-generation AAV vectors.

Besides Omicron, the industry will focus on RNA-based drugs, AI programs, gene therapy trials, financing deals, regulatory issues, and synthetic biology challenges.

In this GEN webinar, sponsored by Twist, our distinguished speaker, Dr. Manuel Kaulich, CSO of Vivlion and group leader at Goethe University, will show us how CRISPR gene editing and functional genetic screening can be used to identify co-dependencies and functional genetic maps, focusing on the key technological advancements that make human combinatorics possible.

Foresite Labs, the entrepreneurial innovation hub of Foresite Capital, focuses on launching companies that apply data science tools to solve unmet medical needs, has incubated five companies: Esker Therapeutics, Interline Therapeutics, Sestina Bio, and two companies in stealth mode: A precision platform therapeutics company leveraging novel genomic biology across multiple disease areas; and a developer of cardiometabolic therapeutics based on data science and human genomics.

A new study from researchers at Harvard Medical School shows that the extracellular matrix can provide the driving force needed for tissue morphogenesis, challenging earlier studies that proposed motor proteins within cells drive tissue-structuring processes. The authors showed that hyaluronate pressure in combination with proteins that connect cells and direct the pressure generated through the swelling of hyaluronate in water, powers morphological change. The findings provide new insights for bioengineering, regenerative medicine, and basic biology.

The patient participated in the non-ambulatory cohort of the trial, a first-in-human/first-in-patient, multi-center, non-randomized, ascending dose, safety and tolerability study. The study was designed to assess a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with DMD. Pfizer said screening and dosing in the trial has been paused while the company reviews data with the study’s independent External Data Monitoring Committee.