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What’s in Today’s Brief? (February 11th Preview)
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FDA refuses to review Moderna mRNA flu vaccine — CBER flags trial design
The FDA declined to start a review of Moderna’s experimental mRNA influenza vaccine, citing deficiencies in the trial’s control arm and overall study design in a letter signed by Vinay Prasad, director of the Center for Biologics Evaluation and Research. Moderna says the decision was procedural and not driven by safety or efficacy concerns; the company has requested a meeting with FDA leadership to clarify next steps. Moderna’s mRNA influenza program completed phase 3 and had reported positive trial results last year; the agency’s refusal to file introduces regulatory delay and raises questions about trial comparators and evidentiary standards for next‑generation vaccine platforms. The public dispute also unfolds amid broader policy shifts at HHS and renewed scrutiny of vaccine rules under the current administration.
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FDA issues complete response letter to Regenxbio — Hunter syndrome approval paused
The FDA issued a complete response letter to Regenxbio for RGX‑121, a proposed gene therapy for mucopolysaccharidosis type II (Hunter syndrome), saying the submission did not adequately support efficacy using the surrogate endpoints and historical comparisons the company provided. Regenxbio had previously faced clinical holds on related programs after a safety event in a separate trial; the agency flagged unresolved issues with the evidentiary package and recommended potential paths including new trials or extended follow‑up. Regenxbio plans to request a meeting with FDA to discuss the CRL and next steps, but the regulator’s decision adds further delays to a gene‑therapy field already coping with safety and evidentiary scrutiny. Investors and rare‑disease advocates are watching closely because the ruling affects approval pathways for one‑time therapies in ultra‑rare indications.
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Capsida confirms cerebral edema in AAV trial fatality — probe continues
Capsida Biotherapeutics reported that an autopsy confirmed cerebral edema as the cause of death in a patient treated in its SYNRGY AAV gene‑therapy trial for STXBP1, but the company said the autopsy did not establish the underlying trigger for the swelling. Capsida maintains the trial was conducted per protocol and that product manufacturing and handling were appropriate, while external experts have called for broader data transparency. The fatality led to a trial suspension in late 2025; Capsida says it will continue internal investigations and seeks access to autopsy samples for further analysis. The case highlights persisting safety and public‑trust challenges for systemic AAV programs and may influence monitoring and risk‑mitigation requirements for future gene‑therapy studies.
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ILiAD raises $115M Series B to launch pivotal pertussis program
ILiAD Biotechnologies closed an oversubscribed $115 million Series B led by RA Capital Management, with participation from Janus Henderson and BNP Paribas Asset Management Alts, to advance its live attenuated intranasal pertussis vaccine BPZE1 toward Phase III development. The company plans a pivotal human challenge study in 2026 with initial data expected in 2027 and aims to demonstrate durable mucosal and systemic immunity that could curb transmission as well as disease. BPZE1 was developed at Institut Pasteur de Lille and has clinical data from six prior studies including a Lancet Microbe challenge trial; the financing is explicitly targeted at pivotal trials and global development. Investors framed the round as backing a needle‑free vaccine approach that could address persistent pertussis epidemics despite high global vaccine coverage.
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Madrigal snaps up six China‑sourced siRNAs: $60M now, $4.4B possible
Madrigal Pharmaceuticals struck a series of deals with Suzhou Ribo Life Science and Ribocure Pharmaceuticals to license six preclinical siRNA programs aimed at metabolic dysfunction‑associated steatohepatitis (MASH), paying $60 million upfront and agreeing to as much as $4.4 billion in downstream milestones plus royalties. The transactions are part of Madrigal’s strategy to combine liver‑targeted gene‑silencing approaches with its marketed THR‑beta agonist Rezdiffra to create multi‑modality regimens for MASH. Madrigal described the acquisitions as pipeline acceleration after Rezdiffra’s market success and follows other recent deals, including a DGAT2 inhibitor from Pfizer. The company expects the siRNAs to enable genetically targeted combinations that could address complementary drivers of fatty‑liver disease.
...and 5 more selected Biotech stories in today’s full edition — or archive.
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