The $12.5 billion acquisition of Avidity Biosciences Inc. by Novartis AG strengthens the company’s neuroscience pipeline and marks the second biggest deal that’s been announced this year. San Diego-based Avidity brings Novartis three RNA-powered neuromuscular disease treatments that are in late stages of development. The antibody oligonucleotide conjugates are a fit for Novartis’ goal of delivering RNA directly to the muscle for a company that has a strategy to fulfill its plan to treat genetic diseases. The late-stage Avidity drugs are designed to treat myotonic dystrophy type 1, facioscapulohumeral muscular dystrophy and Duchenne muscular dystrophy.

‘Home run scenario’ for Bridgebio’s BBP-418 in limb-girdle phase III

Ahead of the interim phase III readout for Bridgebio Pharma Inc.’s BBP-418 in limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), the company had anticipated possibly filing for accelerated approval, with full approval hinging on final outcomes of the pivotal study. But the better-than-expected data, particularly the statistically significant findings on the functional endpoints, could open up a pathway to full approval. Either way, Bridgebio is meeting with the U.S. FDA shortly and anticipates submitting an NDA for what could be the first LGMD2I/R9 therapy in the first half of 2026. Company shares (NASDAQ:BBIO) were trading up more than 17% at midday.

Q3 funding figures point to ‘a fragile moment’ for U.K. biotech

U.K. biotech companies raised 46% less in the third quarter (Q3) of 2025 than in the previous quarter, at £187 million (US$249.3 million). The Q3 2025 figure was also in the shade compared to Q1 2025, when biotechs raised £881 million, and 73% less than in Q3 2024. The U.K. downturn in Q3 was mirrored elsewhere in Europe, where financing declined to £832 million, down from £1.21 billion in Q2 2025. In the U.S. meanwhile, there were some standout large deals and venture capital funding reached £4.63 billion in Q3, up from £3.16 billion in Q2, according to figures compiled by the UK Bioindustry Association from Beauhurst, H2radnor, the London Stock Exchange and Pitchbook data.

Intellia plunges on phase III pause in CRISPR nex-z dosing

Shares of Intellia Therapeutics Inc. (NASDAQ:NTLA) were trading $14.72, down $10.88, or 42%, on word that the Cambridge, Mass.-based firm has temporarily paused patient dosing and screening for its Magnitude and Magnitude-2 phase III trials with nexiguran ziclumeran, also known as nex-z, for patients with transthyretin amyloidosis with cardiomyopathy and polyneuropathy, respectively. The action comes as a result of the Oct. 24 news that a patient in his early 80s who was dosed Sept. 30 in the Magnitude experiment developed grade 4 liver transaminases and increased total bilirubin. Intellia has begun investigating the matter and said the studies will resume “as soon as appropriate.” Nex-z is an in vivo CRISPR-based gene editing drug.

Argenica’s stroke drug shows positive trends in functional outcomes

Although Argenica Therapeutics Ltd.’s stroke drug, ARG-007, saw mixed results in top-line phase II data, new data in functional outcomes studies showed signs the drug helped patients think more clearly, regain independence, and enjoy a better quality of life after stroke. As previously reported by BioWorld, top-line results for the phase II Seancon trial in acute ischemic stroke in patients undergoing endovascular revascularization, or thrombectomy, showed ARG-007 met the primary endpoint of safety and was well-tolerated, but the compound did not show a treatment effect compared to placebo. The functional outcomes measures were part of the same study, but they were exploratory outcomes, rather than primary or secondary endpoints, Argenica CEO Liz Dallimore told BioWorld.

Metis plans China NDA submission for orally dissolving PBA drug

Based on positive phase III study results, Metis Techbio is planning to file an NDA for its AI-derived orally disintegrating tablet drug candidate for pseudobulbar affect (PBA), MTS-004, in China next year. MTS-004 is the first drug candidate in China to be designed by AI-driven formulation technologies and the first and only therapy for PBA in the country to complete phase III development, according to Cambridge, Mass.- and Beijing-based Metis. 

New modalities redefining druggability in genomically unstable cancers

At this year’s AACR-NCI-EORTC conference, several presentations brought to light new ways to tackle the treatment of genomically unstable cancers. Genomically unstable cancers can be treated by exploiting their repair dependencies, inducing catastrophic DNA damage, or harnessing immune responses to instability. The next frontier combines DDR pathway inhibition, targeted protein degradation, and immune modulation to selectively eliminate these unstable cells.

At AACR-NCI-EORTC 2025, new strategies reshape the field

During the first poster session of the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, held in Boston, several presentations highlighted novel strategies that move beyond traditional antibody-drug conjugate payloads and targets. These included the use of dual payloads, novel antigen targets, next-generation linkers and modular platforms that allow for safer and more effective therapeutic combinations.

Also in the news

Aprea, Arcutis, Armata, Ascletis, Biossil, Chugai, Coya, Dicot, Elevara, Eli Lilly, Entera, George Medicines, Grace, GSK, Hanchorbio, Harbour, Incyte, Inhibrx, Johnson & Johnson, Journey Medical, Maruho, Medivir, Metis, Mitocarex, Moderna, Nanobiotix, Nielsen, Partner, Rani, Renalys, Revolution, Ribocure, Sagimet, Scenic, Scisparc, Secarna, Silver Creek, Spinogenix, Sydnexi, Teijin, Ventyx, Verastem, X4, Zai Lab, Zymeworks