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Regulatory and policy—EMA trial advice becomes permanent
The European Medicines Agency confirmed it will turn an EU-level trial advice pilot into a permanent option for sponsors, extending the mechanism that provides regulator input during development...
Company dealmaking—Pfizer partners with Enodia on Sec61 inhibitors
Pfizer agreed to a research collaboration and licensing deal with Enodia Therapeutics to discover and develop small-molecule inhibitors targeting Sec61, a translocon required for protein...
Oncology—Rapid manufacturing CAR-T in early clinical testing
A Phase I study at Ohio State reported early results for an engineered CAR-T platform designed for faster manufacturing—produced in about six days, infused fresh, and targeting three B-cell...
Psychedelics—Compass Pathways signals cautious rollout for potential FDA-era launch
Compass Pathways CEO Kabir Nath said the company plans to “go slow before we go fast” as it approaches regulatory submission and prepares for a potential psychedelics approval environment in the...
Clinical innovation—Potassium binder helps CKD heart-failure patients reach full RAAS inhibitor doses
A randomized double-blind trial found that sodium zirconium cyclosilicate helped chronic kidney disease patients with heart failure reach target RAAS inhibitor doses by enabling treatment...
Regulatory and evidence—Biofabrication of trial recruitment information across social platforms
A study published in Nature found that how people share clinical trial information differs sharply between Bluesky and X, with platform-specific communication patterns affecting the accessibility...
Virology and immunology—mRNA-delivered bispecific antibodies show broad SARS-CoV-2 neutralization
Researchers engineered mRNA-delivered bispecific antibodies derived from llama candidates to neutralize a range of SARS-CoV-2 variants. In mouse experiments, the approach protected against the...
Infectious disease—Drug-resistant fungus Candida auris surges in US surveillance data
US national surveillance shows Candida auris clinical cases more than doubled from 2022 to 2024, according to CDC reporting cited in a recent summary. The rise is concentrated among older adults...
Drug discovery—Arrowhead’s RNAi brain-targeting program lowers tau in early human data
Arrowhead Pharmaceuticals reported early results for ARO-MAPT, an investigational RNA interference therapy aimed at lowering tau levels by silencing a gene that drives tau expression in the...
R&D platform and funding—Soufflé Therapeutics raises $135M for multiple RNA medicines
Soufflé Therapeutics raised $135 million in venture capital to advance at least three experimental oligonucleotide medicines. The funding adds to momentum in RNA therapeutics that emphasize...
Regulatory: EMA permanent trial advice pilot
The European Medicines Agency will turn its EU-level trial advice pilot into a permanent option for sponsors, letting companies keep seeking regulator input as they plan studies. The change...
Rare disease financing: Ultragenyx sells priority review vouchers
Ultragenyx Pharmaceutical secured $413 million by selling two FDA priority review vouchers tied to gene-therapy approvals. The company says proceeds will strengthen its financial position for...
AI x clinical trials: platform effects on trial information sharing
A Nature study found that social-media platforms shape how clinical trial information is shared and discussed during recruitment—Bluesky posts were more cohesive and research-oriented, while...
Obesity investing: Zealand partners with Generate Biomedicines
Zealand Pharma agreed to use Generate Biomedicines’ AI technology to develop protein-based treatments for obesity and other metabolic diseases. The Danish company will spend $30 million upfront...
Alzheimer’s therapeutics: Arrowhead targets tau with brain-reaching RNAi
Arrowhead Pharmaceuticals reported early trial data showing its brain-targeting RNA interference approach can lower tau levels after subcutaneous dosing in healthy volunteers. In the company’s...
Gene therapy preclinical proof: AAV delivered at CSF source for hydrocephalus
A University of Cincinnati and UC Davis team reported proof of concept for an AAV gene therapy aimed at hydrocephalus at its source. In animal models, the therapy targeting...
Immunotherapy platform: MIT lipid nanoparticles reprogram NK cells in vivo
MIT engineers reported lipid nanoparticles designed to reprogram natural killer cells inside the body. In mouse models, the LNP approach shifted NK-cell behavior toward cancer and autoimmune...
M&A and platform licensing: Pfizer collaborates with Enodia on Sec61 inhibitors
Pfizer entered a research collaboration and licensing agreement with Enodia Therapeutics to discover Sec61 inhibitor candidates. Enodia will run discovery to screen, optimize, and develop...
Trial readout: potassium binder enables full RAAS inhibitor dosing in CKD+HF
A randomized double-blind trial reported that sodium zirconium cyclosilicate helped chronic kidney disease patients with heart failure reach target doses of RAAS inhibitors while reducing...
Trial financing and portfolio moves: Soufflé Therapeutics raises $135M for RNA medicines
Soufflé Therapeutics raised $135 million in VC funding to advance at least three experimental oligonucleotide medicines. The Bob Langer-affiliated startup said the capital is intended to move...