Nature Biotechnology editors pick their favorite research articles from 2023.

Nature Biotechnology’s annual survey highlights academic start ups that are, among other things, correcting misfolded or disordered proteins, creating second-generation GPCR agonists, building a new gene delivery platform and mining cancer genomes for novel targets.

Our ranking is out, and it has a few surprises. In pole position — predictably — is Wegovy, the weight-slashing GLP-1 agonist, with AI-hallucinated proteins a close second. These are followed by drugging options for RNA. The dark horses are virus-free gene therapies and a sweet protein.

The go-ahead for Vertex’s gene editing therapy in sickle cell disease and β-thalassemia is a historic milestone, but this one-time treatment is costly.

Cancer therapy with chimeric antigen receptor (CAR)-T cells has achieved impressive remissions in certain hematologic malignancies, and extending the approach to other types of cancer is a key goal of immunotherapy research. Solid tumors have proved particularly challenging because of immunosuppressive mechanisms in the tumor microenvironment. Writing in Science, Danino and colleagues outline a new

Central nervous system (CNS) tumors are a leading cause of cancer-related deaths, particularly among children, and the primary treatment is neurosurgical resection. Precisely identifying the tumor type is crucial for developing an effective surgical plan. However, the current methods of histological assessment and molecular analysis face challenges in providing a clear diagnosis during surgery. In

Recent patents relating to the manufacture and design of engineered immunoglobulin polypeptides.

Nkarta is charting a course for its allogeneic chimeric antigen receptor (CAR) natural killer cell (NK) therapy in autoimmune lupus nephritis after receiving approval from the US Food and Drug Administration (FDA) to test NKX019. The cell therapy specialist becomes the second to get FDA go-ahead for an NK product in a chronic autoimmune disease, following Artiva Biotherapeutics’ AlloNK. In August 2023

In October, the Chan Zuckerberg Initiative (CZI) launched two new biohubs: one in New York and another in Chicago. The biohubs will bring together academic researchers at leading institutions to solve scientific challenges that could result in better prevention, detection and treatment of disease. The CZ Biohub NY received, in addition to undisclosed CZI support, $10 million each from the State of

A new malaria vaccine for children is the first to meet the World Health Organization’s (WHO) 75% protection target. The vaccine, called R21 and developed and produced by Oxford University and the Serum Institute of India, is the second recommended by the WHO. The first was Mosquirix (RTS,S/AS01) made by London-based GlaxoSmithKline (GSK), which is 50% protective. At $3.90 per dose, R21 is cheaper

Despite the longstanding goal of reestablishing walking after spinal cord injury (SCI), existing strategies for axonal regeneration do not fully restore motor function. Now, as published in Science, Squair et al. use a targeted approach to promote axonal growth, leading to functional recovery of complete spinal cord damage in mice and restoring walking ability.

After growing up collecting fossils and reading science books, this group leader’s research now lies at the interface of biology, artificial intelligence and genomics.

Recent moves of note in and around the biotech and pharma industries.

By the end of the quarter, the US Food and Drug Administration (FDA) had racked up 39 innovative drugs approvals, already exceeding last year’s total of 37. Among them are two bispecific antibodies redirecting T cells via the CD3 antigen to overexpressed tumor markers in multiple myeloma that received accelerated approvals in August: Talvey (talquetamab) from Johnson & Johnson links CD3 to GPCRC5D

Policy changes are needed to ensure that Cooperative Research and Development Agreements and patent applications filed by the US National Institutes of Health are aligned with the interests of the American public.

ChatGPT holds transformative potential for medicine across clinical, research and educational domains.

Seven US biotech IPOs got away in the quarter, but only early-stage Apogee Therapeutics (Waltham, MA) remained steady in the aftermarket. With precious little IPO interest for biotech, US venture capitalists (VCs) focused on making sure their existing portfolio companies could last out the drought. Six of the top-ten venture rounds in the quarter were series C rounds. Generate:Biomedicines (Somerville

Quantum dots won the Nobel Prize, but proved to be a miss in the biomedical space.

Armed with gene technologies and CAR-Ts, scientists are attempting to eliminate viruses that escape immune detection and lurk in tissues for years.

Biopharma companies debate whether to patent their algorithms for finding medicines.

AI startups have been at the center of a wave of deals driven by companies looking to enrich their drug discovery pipelines. In August, Exelixis paid $80 million to license a small-molecule inhibitor of USP1 from Insilico Medicine, an AI biotech headquartered in Hong Kong. The deal grants Exelixis the right to develop and commercialize lead molecule ISM3091, aimed at a synthetic lethal target implicated

A new company, Arialys Therapeutics, has raised $58 million in seed funding to develop a therapy for a rare brain inflammation caused by autoimmune antibodies. If successful, the strategy could be applicable to other, more prevalent neuropsychiatric disorders. Although the received wisdom is that the central nervous system is shielded from immune responses, recent findings have identified B lymphocytes

Chemically induced dimerization tools are used for actively perturbing signal transduction in live cells, but most existing methods are not able to be repeatedly reversed. In a study published in Nature Methods, Bottone et al. introduce a system called CATCHFIRE (chemically assisted tethering of chimera by fluorogenic-induced recognition), which allows the monitoring of reversibly induced protein proximity

Kidney transplant rejection is the main cause of graft failure, but detection of early-stage rejection is challenging. Current approaches are either invasive or can have false-positive or false-negative predictions owing to alteration of known rejection biomarkers due to diet, infections or medications unrelated to rejection. In a study published in Science, Madhavpathy and colleagues develop an implantable

Nurix Therapeutics and biotech Seagen are combining their expertise to work on an emerging class of anticancer therapeutics known as degrader–antibody conjugates (DACs).

Missense variants are DNA nucleotide alterations that result in a protein amino acid change. Of the 71 million possible missense variants in the human genome, only around 0.1% have been clinically classified as pathogenic or benign. In a publication in Science, Cheng and colleagues from Google DeepMind introduce their artificial intelligence (AI) model AlphaMissense, which aims to predict the pathogenicity

Encouraging innovation will allow physicians and their academic medical centers to pursue the development of technologies and healthcare practices that will improve patient care.

Recent patents relating to the production of antimicrobial agents and materials.

Recent moves of note in and around the biotech and pharma industries.

The Weissman lab is taking another swing at ‘don’t eat me’ signaling. They think they have a new and better target.

Bifunctional molecules that link kinase enzymes with new targets could rewire disease-related cellular processes

Two start-ups are taking a deep dive into cancer genomes to unearth new therapeutic targets.

Decentralized autonomous organizations are growing as alternative research funding models, but are also strong scientific communities. We should get on board.