Chronic hepatitis B affects around 250 million people in the world and its cure remains elusive. At the 2023 European Association for the Study of the Liver Congress in Vienna, Austria, Emily Harrison of Precision Biosciences Inc. presented the company’s work on using a naturally occurring endonuclease in the development of its ARCUS gene editing approach to eradicating the persistent viral infection.
Promising early data continue to roll out for Intellia Therapeutics Inc.’s hereditary angioedema (HAE) candidate, NTLA-2002, with one of the earliest treated patients in the phase I study remaining attack-free for more than a year. But it was the systemic CRISPR candidate’s potential as a one-time treatment that generated the most discussion on the company’s call as investors tried to assess its potential advantage in a crowded HAE market.
In its bid to become, as Chairman and CEO David Hallal said, the “world’s most indispensable cell and gene therapy technology company,” Elevatebio LLC disclosed a $401 million series D round with support from new and existing investors. At the same time, the company’s Life Edit Therapeutics Inc. affiliate inked a potential billion-dollar collaboration focused on gene editing therapies.
Scribe Therapeutics Inc. is selling exclusive rights to its CRISPR-based technology to Prevail Therapeutics Inc. for a figure that could top $1.6 billion as the firms team up to develop genetic therapies for serious neurological and neuromuscular diseases.
A research initiative led by Jennifer Doudna and Jill Banfield of the Innovative Genomics Institute at the University of California, Berkeley, has landed $70 million in funding from the Audacious Project to bring the power and precision of CRISPR-based genome editing to the gut microbiome of humans and animals, in an ambitious effort to engineer complex microbial communities to achieve outcomes that can benefit human health and the environment.
Seamless Therapeutics GmbH raised $12.5 million in seed financing to take forward a novel gene editing technology based on reprogramming recombinase enzymes.
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.
Chroma Medicine Inc. closed a $135 million series B round as momentum builds at one of the early movers in the emerging field of epigenetic editing. It’s little more a year since Cambridge, Mass.-based Chroma emerged from stealth by disclosing a $125 million series A round and a stellar line-up of company founders. “It’s not all that long ago, but we have made a huge amount of progress,” CEO Catherine Stehman-Breen told BioWorld.
