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Beam presented updated open-label Phase 1/2 data for BEAM-302, an LNP-delivered in vivo base-editing treatment for alpha-1 antitrypsin deficiency. Follow-up extended to 18 months after one dose, with increased functional AAT and reduced mutant Z-AAT and circulating polymers. Dosing is underway in a pivotal cohort. These biomarker findings do not yet establish controlled evidence of improved lung or liver clinical outcomes.
Intellia announced FDA acceptance of its biologics license application for lonvoguran ziclumeran, or lonvo-z, in hereditary angioedema, with Priority Review and a March 10, 2027 target action date. The investigational treatment uses LNP-delivered CRISPR components for in vivo editing. Application acceptance advances the regulatory process but is not marketing approval and does not guarantee a favorable decision.
International journal of biological macromolecules
An injectable hydrogel co-delivered hyaluronidase and light-responsive nanoparticles carrying IL-12 and GM-CSF mRNAs. The design combined stromal remodeling, local retention, and photoactivated intracellular delivery. In a postoperative mouse breast-cancer model, treatment reduced recurrence and altered local and systemic immune responses. The multiple-component system remains experimental, with light access, long-term safety, and clinical feasibility requiring further work.
Proceedings of the National Academy of Sciences of the United States of America
Investigators combined mRNAs encoding an EpCAM-directed T-cell engager, IL-12, and GM-CSF through intratumoral nanoparticle administration. In partially humanized mouse xenograft experiments, the combination altered immune activity in both injected and uninjected tumors and inhibited tumor growth. The study supports localized production of several immune proteins, while the model cannot establish safety or effectiveness across human cancers.
Typewriter Therapeutics emerged from stealth with $56 million in Series A financing led by AN Venture Partners and RA Capital. Its platform uses LNP-delivered RNA, including mRNA encoding an R2 protein, to pursue targeted insertion of therapeutic genes. Initial priorities include in vivo CAR T and genetic liver disease. The funding announcement does not establish human safety, durability, or clinical benefit.
ARPA-H selected five teams for its GIVE research program, with funding of up to $125 million for automated, distributed RNA medicine manufacturing. Selected approaches include RNA synthesis, lipid nanoparticle formulation, fill-finish, and integrated quality control. The agency plans engagement with FDA on regulatory frameworks. These are development objectives for technologies still being built, rather than established distributed GMP production capabilities.
The study develops charge detection mass spectrometry for intact mRNA constructs spanning 997–4,522 nucleotides. Optimized preparation and calibration enabled length determination with 0.5% uncertainty, comparisons of modified nucleotides and poly(A) tails, and analysis after extraction from lipid nanoparticles. The method adds intact-molecule information, while counterion effects and orthogonal identity and impurity measurements remain relevant.
D&D Pharmatech and QIC announced selection for a Korean national R&D program targeting oral α4β7 therapeutics, with a July 2026–June 2028 period and KRW1.358 billion total budget. QIC designs candidates while D&D applies ORALINK and evaluates activity. QIC identifies the program as an oral cyclic peptide in lead optimization, rather than a clinically validated medicine.
CREATE Medicines entered a research collaboration and license agreement with WestGene to combine immune-cell programming and RNA gene-writing technologies with targeted lipid nanoparticles. Initial work spans three in vivo CAR programs involving autoimmune disease, solid tumors, and durable gene expression. The partnership expands delivery access and development options, but the announcement does not demonstrate clinical efficacy of the combined approaches.
BioNTech and Genentech decided to terminate BNT122-01, a Phase 2 trial of autogene cevumeran monotherapy after surgery in ctDNA-positive, high-risk stage II or stage III colorectal cancer. An independent board cited a numerical overall-survival imbalance and the low likelihood that continuing would change the efficacy outcome. No new product safety signals were identified; a pancreatic-cancer combination trial remains unaffected.
Evonik announced a Vancouver expansion for GMP manufacturing of lipid-based drug products, involving more than C$150 million in investment and up to C$68 million in Canadian government support. The project includes production, quality control, and microbiology capabilities. Evonik expects the expansion to more than triple existing capacity; this is planned capacity rather than production already available to customers.
Pfizer and BioNTech announced FDA approval of the 2026–2027 XFG-adapted COMIRNATY formula for adults 65 and older and people ages 5–64 with at least one condition increasing the risk of severe COVID-19. The decision drew on existing clinical, nonclinical, and real-world platform evidence plus formulation-specific quality and nonclinical data. The label does not provide unrestricted use across all age groups.
