The Therapeutic Areas that Stood Out in BioSpace’s NextGen Class of 2024

2024-01-08

基因疗法

Pictured: Map of NextGen Class of 2024/BioSpace Much of a scientist’s work involves uncovering patterns, and I’m finding the same is true for science journalists. While putting together the list for BioSpace’s NextGen Class of 2024, our team noticed that the therapeutic focus of many of the top companies mirrored that of the deals currently making headlines. While many people enjoyed holiday celebrations and recovered from food fatigue, Bristol Myers Squibb was busy locking up radiopharmaceutical company RayzeBio for $4.1 billion. In the deal, BMS picked up RayzeBio’s lead asset, RYZ101, which is targeting gastroenteropancreatic neuroendocrine tumors and extensive stage small cell lung cancer. Radiopharmaceuticals are also well-represented in the NextGen Class of ’24. The space has quietly gained popularity but has also been hampered by adverse effects. West Coast–based Abdera Therapeutics, No. 8 on this year’s list, is developing antibody-based radiopharmaceuticals with tunable PK properties, an approach it says can maximize therapeutic effect while mitigating systemic toxicities. And No. 13 Convergent Therapeutics, based in Cambridge, Mass., is targeting prostate cancer with CONV01-α using radionuclide 225Ac, which Convergent claims will reduce exposure of healthy tissues to the toxic radiation intended for cancer cells. Meanwhile, a host of new players are searching for novel therapeutic targets for intractable neurological diseases. Number one–ranked Rapport Therapeutics is leveraging receptor-associated proteins (RAPs), which enable “unprecedented precision” in targeting receptors in the specific neuroanatomical regions underlying neurological disorders, according to the bicoastal startup. Massachusetts-based Nido Biosciences, No. 5, is using tailored screens to identify novel therapeutic targets for neuromuscular and neurodegenerative diseases, and Seattle’s Cajal Neuroscience, No. 14, is throwing the kitchen sink at target discovery with human genetics and multi-omics, whole brain imaging and functional genomics. The appetite for deals focused on new neuroscience targets and promising clinical assets has not abated. December alone saw BMS paying $14 billion for Karuna Therapeutics and its potentially groundbreaking schizophrenia drug KarXT, while AbbVie bolstered its neuro portfolio with the acquisition of Cerevel Therapeutics and its broad portfolio of clinical and preclinical programs targeting Parkinson’s disease, epilepsy, schizophrenia and more. Gene therapy also featured prominently on our NextGen list. This technology converged with neuroscience in one of the first big partnerships of 2024 when Novartis and Voyager Therapeutics inked a potential $1.2 billion pact to develop gene therapies for Huntington’s disease and spinal muscular atrophy. The partners will leverage Voyager’s TRACER Capsid Discovery Platform, which uses an RNA-based screening approach to discover adeno-associated virus (AAV) capsids with robust central nervous system tropism that the company claims can penetrate the blood-brain barrier. Coming up behind Voyager are Aera Therapeutics (No. 4), SonoThera (No. 9) and Orbital Therapeutics (No. 7), which are each taking a unique approach to gene therapy delivery. Finally, what up-and-comers list would be complete without a company working on a Glucagon-like peptide-1 (GLP-1) drug, a class that surged to prominence last year with the continued success of Novo Nordisk’s Wegovy and approval of Eli Lilly’s Zepbound. Cambridge–based i2o Therapeutics, No. 26 in BioSpace’s 2024 NextGen class, is developing ITCA 650, an investigational, twice-yearly implantable GLP-1 for type 2 diabetes. The company’s ionic liquids platform aims to overcome the barriers to oral administration of biologics and peptides by protecting them from the harsh conditions in the gastrointestinal tract and assisting large molecules in crossing the epithelial lining. With all the action in these therapeutic spaces, I wouldn’t be surprised to soon see one of these new companies snapped up in an acquisition or united in a big-name partnership. Heather McKenzie is a senior editor at BioSpace. You can reach her at heather.mckenzie@biospace.com. Also follow her on LinkedIn.