Biotech

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    Vertex CRISPR therapy hits early goal in children with blood disorders

    Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label expansion and stronger commercial uptake.

    By • Dec. 6, 2025
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    Vaccines

    RFK Jr.’s hand-picked panel questions childhood vaccine schedule

    After weakening an endorsement for the hepatitis B vaccine, ACIP began debating the merits of the protocol used to inoculate U.S. children against many infectious diseases.

    By • Dec. 5, 2025
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    Trendline

    Emerging biotech

    New biotechs continue to emerge despite a challenging market environment that has forced venture firms to build their drug startups more cautiously.

    By BioPharma Dive staff
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    News roundup

    Praxis soars higher; Bristol Myers’ CAR-T therapy wins broader use

    Praxis shares climbed another 40% on a pair of positive updates on two different drugs. Elsewhere, Bristol Myers’ Breyanzi was cleared for its fifth cancer indication and AstraZeneca expanded a rare disease deal.

    By BioPharma Dive staff • Dec. 5, 2025
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    Emerging biotech

    An Arch-backed biotech raises $53M to fight neurodegeneration

    SciNeuro Pharmaceuticals represents the latest in a string of Arch investments directed at the nervous system, a notoriously challenging area of drug development.

    By • Dec. 4, 2025
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    UniQure slides further on outlook for Huntington’s gene therapy

    Finalized minutes from a late-October meeting with the FDA confirm the agency doesn't see the data collected so far as enough to support an approval filing.

    By • Dec. 4, 2025
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    Capricor soars on positive results for Duchenne cell therapy

    Shares quadrupled now that the company appears on track to reverse an earlier FDA rejection of what could be the first treatment for heart-related complications of Duchenne.

    By Kristin Jensen • Dec. 3, 2025
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    Pharvaris’ pill succeeds in late-stage trial for rare swelling disorder

    Phase 3 data could make the Dutch biotech's drug competitive in the increasingly crowded market of hereditary angioedema treatments.

    By • Dec. 3, 2025
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    News roundup

    Janux sinks on ‘overreaction’ to prostate cancer data; RFK Jr. replaces ACIP chair

    Janux’s “masked” T cell engager had a lower response rate than reported last year. Elsewhere, Lilly dropped Zepbound’s price and ACIP named a new chairman days before a key vaccine meeting.

    By BioPharma Dive staff • Dec. 2, 2025
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    Belite continues ascent as Stargardt drug hits mark in late-stage trial

    The results position Belite, one of the sector’s top-performing companies since 2022, to seek approval of what could be the first marketed medicine for the rare eye condition.

    By • Dec. 1, 2025
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    Emerging biotech

    With $130M, Protego pushes forward a new type of amyloidosis drug

    The startup’s lead prospect stabilizes abnormal proteins in patients with amyloid “light chain” amyloidosis, an approach that could represent a step forward compared to current therapies. 

    By • Dec. 1, 2025
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    Sponsored by AgriNovus Indiana

    From feedstocks to infrastructure, Indiana’s bioeconomy is on the move

    Indiana shows high potential for the state to become the premiere destination to research, commercialize, scale biotechnology processes, platforms and products.  

    Dec. 1, 2025
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    Otsuka gains approval for first-of-its-kind treatment against rare kidney disease

    In a rapidly heating market, Otsuka is the first to secure FDA clearance for an "anti-APRIL" therapy targeting IgA nephropathy.

    By • Nov. 26, 2025
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    Emerging biotech

    Biogen strikes deal with Versant-backed biotech to expand immune portfolio

    Newly launched Dayra Therapeutics has agreed to identify for Biogen "oral macrocycle candidates" that go after "high-priority immunological targets."

    By • Nov. 24, 2025
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    News roundup

    FDA tests new program to speed drugmaker talks; Bezos-linked AI startup raises $106M

    The FDA will now allow biotechs to ask agency staff for “quick clarifications” via email. Elsewhere, Novartis hiked guidance for two top drugs and a startup nearing “unicorn” status secured a megaround.

    By BioPharma Dive staff • Nov. 21, 2025
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    Moderna secures $1.5B lifeline as it looks to break even in 2028

    At an analyst day event, Moderna said it wants to invest revenue from a hopefully expanded seasonal vaccine franchise into oncology and rare disease programs, to “set the stage for additional growth in 2027 and 2028.”

    By • Nov. 20, 2025
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    Brain drug revival

    Aspen raises $115M for a Parkinson’s stem cell treatment

    The cell therapy maker said Thursday the raise will support an ongoing trial of its experimental treatment for the neurodegenerative condition, as well as plans for building its manufacturing capabilities.

    By • Nov. 20, 2025
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    Agios shares fall on mixed sickle cell results for blood disease drug

    Pyrukynd, which Agios sells for a rare type of anemia, missed one of its main objectives in a study seen as crucial for its commercial outlook and the company’s reinvention as a rare disease specialist.

    By • Nov. 19, 2025
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    News roundup

    Alkermes escalates bidding war; US biotech risks ‘falling behind’ China

    A bipartisan report warned Congress about China’s growing control over the pharmaceutical supply chain. Elsewhere, Alkermes sweetened its bid for Avadel and Moderna is investing $140 million in U.S. drug production.

    By BioPharma Dive staff • Nov. 19, 2025
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    RNAi biotech Arrowhead wins first FDA approval

    The clearance of Redemplo for a rare genetic disorder marks Arrowhead’s transition, after two decades, into a commercial-stage company and sets up a turf war with Ionis Pharmaceuticals.

    By • Updated Nov. 18, 2025
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    Zymeworks to change course, pursue ‘royalty-driven’ biotech model

    On the heels of a data readout suggesting one of its cancer drugs could become a future blockbuster, Zymeworks is adopting a new strategy its CEO says will create “more consistent value for shareholders.”

    By • Nov. 18, 2025
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    J&J adds to pharma’s M&A spree with $3B buyout of startup Halda

    The deal gives J&J access to a pipeline of drugs designed to “hold and kill” tumors, led by a prostate cancer medicine that’s shown promise in early-stage testing. 

    By • Nov. 17, 2025
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    News roundup

    FDA limits Elevidys use; Nxera to lay off staff

    Elevidys now has a “boxed” warning and is no longer available for Duchenne patients who can’t walk. Elsewhere, Nuvalent and Zymeworks unveiled data for targeted cancer drugs.

    By • Nov. 17, 2025
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    Startup launches

    Solve lines up $120M to push ADCs into further testing

    The startup, which is backed by a dozen investors including Merck & Co., claims its novel “linkers” can help address some of the issues limiting the potential of earlier ADCs. 

    By • Nov. 17, 2025
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    Emerging biotech

    A ‘DNA damage repair’ drugmaker raises $115M for cancer treatments

    Artios Pharma, which looking for newer targets that, like PARP inhibitors, affect the way tumors fix themselves, said its Series D round will help advance ongoing mid-stage studies.

    By • Nov. 17, 2025
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    Emerging biotech

    Sofinnova Partners raises another $750M to back biotech, medtech startups

    The new fund will mostly support biotechs making new drugs, with investments spanning from initial financings to follow-on rounds. 

    By • Nov. 16, 2025