Homozygous familial hypercholesterolemia (HoFH) is a severe rare life-threatening condition where high blood levels (>500 mg/dL) of low-density lipoprotein cholesterol (LDL-C), triglycerides (TG), and premature and progressive atherosclerotic cardiovascular disease (ASCVD) are the main features.

The intended use of gene editing tools on pre-implantation human embryos would be to avoid the development of congenital diseases in the upcoming baby. But it may have its own risks. Those risks were illustrated in a publication in the March 7, 2023, issue of Nature Communications, where researchers from the Center for Embryonic Cell and Gene Therapy, Oregon Health & Science University (OHSU) showed that the method that is most frequently used for evaluating the effects of gene editing zygotes did not always result in an accurate picture of those edits.

The researcher who pioneered prenatal surgery to correct neural tube defects has turned her attention to using CRISPR-edited gene therapies to correct severe monogenic diseases in utero. The availability of prenatal genetic diagnosis and advances in treating fetuses, and also in gene therapy/gene editing, make it possible to repair almost any defect in the genetic code. At the same time, there is a clear rationale for intervening before birth, Tippi MacKenzie, professor of surgery at UCSF’s School of Medicine, told attendees of the third International Human Genome Editing Conference in London on March 7.

The U.S. FDA marked the 40^th anniversary of the Orphan Drug Act with Rare Disease Day 2023 as Robert Califf, the agency’s commissioner of food and drugs, opened the day by expressing his wonder and accompanying concern regarding gene editing and gene therapy.

By applying deep learning methods to a large database of zinc finger nucleases, researchers at the University of Toronto and New York University have developed an algorithm, Zfdesign, that was able to design custom zinc fingers for any given stretch of DNA. “I think this system levels the playing field for zinc fingers and CRISPR,” said Philip Kim, co-corresponding author of the team's paper published online in Nature Biotechnology on Jan. 26, 2023.

Programmable genome insertion of long DNA sequences, useful for both gene therapy and basic research, commonly relies on cellular responses to double-strand breaks (DSBs) using programmable nucleases, such as CRISPR-Cas9, for induction of repair pathways such as non-homologous end joining (NHEJ). To overcome the current limitations of gene integration approaches, scientists from the Massachusetts Institute of Technology and colleagues developed a new strategy based on advances in programmable CRISPR-based gene editing, such as prime editing, together with the application of precise site-specific integrases.

A lackluster efficacy signal has prompted Editas Medicine Inc. to pause enrollment in a phase I/II trial of its CRISPR/Cas9-based gene editing therapy, EDIT-101, which is in development for patients with a particular form of Leber congenital amaurosis type 10.

Verve Therapeutics Inc.’s heart disease candidate, VERVE-101, is the latest gene editing-based therapy to hit a snag at the FDA, which issued a clinical hold, delaying the start of phase I testing in the U.S. News of the hold, which followed preclinical presentations over the weekend at the American Heart Association 2022 meeting, sent shares of Verve (NASDAQ:VERV) falling 30.5% to close Nov. 7 at $21.75.

Iecure Inc. has announced the presentation of data by research collaborators...