Daily Snap - 13. October 2025

Author

Joachim Eeckhout
October 13, 2025

Good morning! Researchers at the French non-profit organization Genethon successfully treated a patient with Crigler–Najjar syndrome who was previously immune to the adeno-associated viral (AAV) vector used in the therapy. Typically, AAV vectors serve as vehicles to deliver therapeutic genes into target cells, but pre-existing neutralizing antibodies can recognize the vector before it reaches its target, rendering the treatment ineffective. Genethon tackled this by using imlifidase, an enzyme from Hansa Biopharma, to neutralize those antibodies before delivering the gene therapy. The result? The patient no longer needs daily phototherapy, a first for someone with this condition. While more data are needed to confirm long-term success, this breakthrough could open the door for many patients currently ineligible for gene therapies due to pre-existing immunity.

Enjoy today’s read!

—Joachim E. 

SNIPPETS

What’s happening in biotech today?

💸Deal rumors: Johnson & Johnson is in discussions to acquire Protagonist Therapeutics, its long-time immunology partner, potentially valuing the biotech company well above its current $4 billion market cap, according to The Wall Street Journal. The two firms have collaborated since 2017 on icotrokinra, an oral IL-23 receptor blocker currently under regulatory review for plaque psoriasis and in trials for other inflammatory conditions. The drug recently outperformed a rival treatment from BMS and is seen as a potential blockbuster. A deal would also give J&J access to rusfertide, a blood disorder treatment licensed by Takeda, strengthening J&J’s immunology and hematology pipeline.

 ✂️Exit interview: Novo Nordisk is discontinuing its entire cell therapy division, including a program aimed at curing Type 1 diabetes, as part of new CEO Maziar Mike Doustdar’s cost-saving strategy to cut $1.3 billion annually by 2026. The move will eliminate nearly all 250 jobs in the unit and end R&D efforts in Parkinson’s disease and chronic heart failure. This decision follows Novo’s recent termination of a $598 million cardio cell therapy deal and is part of a broader restructuring involving 9,000 job cuts. The company plans to refocus resources on core areas like obesity and diabetes, while seeking partners to continue cell therapy research.

🧪 Beat the B cell: Cabaletta Bio’s CAR-T therapy, rese-cel, showed promising early results in a small phase 1/2 trial for pemphigus vulgaris, a rare autoimmune disease, by eliminating overactive B cells in two of three patients without the need for preconditioning chemotherapy. All three patients demonstrated significant clinical improvement and tolerated the treatment well, with only mild side effects reported. These findings suggest a potentially less toxic approach to autoimmune therapy, which could broaden patient access. Despite limitations such as a small sample size and short follow-up, analysts view the results as compelling early evidence for CAR-T use without preconditioning, prompting a rise in Cabaletta’s stock.

🤖 AI acquisition: Shuttle Pharmaceuticals has announced a planned $10 million acquisition of Molecule.ai, marking its entry into the AI-driven drug discovery space. Previously focused on cancer therapies and diagnostics, particularly a Phase 2 study of its radiosensitizer ropidoxuridine for glioblastoma, Shuttle now aims to tap into the $3.24 billion AI pharmaceutical market. Molecule.ai, founded by AI scientist ZT Zhang, applies machine learning and large language models to drug discovery processes. If finalized, the deal would expand Molecule.ai’s platform to include automated workflows for drug-target interaction and discovery, aligning with Shuttle’s vision to accelerate and transform therapeutic development through AI.

 📈Survival spike: OS Therapies’ cancer vaccine, OST-HER2, significantly improved overall survival in a Phase IIb trial for patients with recurrent, fully resected pulmonary metastatic osteosarcoma, with 75% of treated patients alive at two years compared to 40% in historical controls. The study also linked 12-month event-free survival to longer-term survival outcomes. Following these results, OS Therapies plans to seek regulatory approvals in the US, UK, and EU starting in late 2025, aiming to bring the first targeted therapy for metastatic osteosarcoma to market. The vaccine, acquired from Ayala Pharmaceuticals, has been shown to be safe and well-tolerated, addressing a high unmet need in this rare cancer.

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