Newco Teitur Trophics ApS has raised €28 million (US$30.1 million) in a series A with which it will lay out a new route to targeting sortilin in the treatment of neurodegenerative diseases. The company is targeting the sortilin-related Vps10p domain containing receptor, which plays a role in regulating a number of pathways involved in the control of neuronal viability and function.

Somatic human genome editing has made huge strides in the past five years, but the likely extremely high prices will be unsustainable. A global commitment to affordable, equitable access is urgently needed because the costs and infrastructure needs of this form of treatment are not manageable for either patients or health care systems.

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. Recover program, set up in July 2022 to identify the causes of long COVID, find biomarkers of disease and discover new therapeutic targets, is now preparing to move to its next phase and begin testing potential treatments in a multi-arm, randomized, placebo-controlled trial. But with 200 different symptoms, and limited understanding of relevant system-level pathological targets, there are significant hurdles to be overcome.

Newco Relation Therapeutics Ltd. is showing its colors after raising $25 million in a seed round to work on integrating single cell transcriptomics, functional genomics and machine learning – and cut through previously undecipherable combinatorial space – to find and validate drug targets in the non-coding genome.

The U.S. Recover program, set up in July 2022 to identify the causes of long COVID, find biomarkers of disease and discover new therapeutic targets, is now preparing to move to its next phase and begin testing potential treatments in a multi-arm, randomized, placebo-controlled trial. But with 200 different symptoms, and limited understanding of relevant system-level pathological targets, there are significant hurdles to be overcome.

In a striking demonstration of how the success of mRNA vaccines against COVID-19 has opened up the potential of the technology in cancer, mice that cleared a tumor after a single administration of an mRNA vaccine, also cleared a second tumor without receiving further immunization. This display of potent immunogenicity formed part of a head-to-head study of three different mRNA technology platforms in human papillomavirus (HPV)-16 associated tumors.

First the stick and now the carrots. The pharma industry in the U.K. on March 1 published its proposal for a new pricing scheme, under which it is offering to pay a fixed rebate of 6.88% across all eligible drug sales, an offer it said will deliver more than £1 billion (US$1.2 billion) per annum back to the National Health Service.