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Gilead signs a $1.5B deal for Genhouse Bio’s oral cancer drug & the FDA rejects Disc Medicine’s rare disease drug

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Joachim Eeckhout & Eduardo Longoria
February 17, 2026

 

👉 Explore the German Biotech Days 2026 by BIO Deutschland

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Vertex’s shares rose 6% after stronger-than-expected Q4 sales of Casgevy, its CRISPR-based therapy for sickle cell disease and beta thalassemia. The one-time treatment works by editing a patient’s own blood stem cells to produce fetal hemoglobin, with the potential to functionally cure the disease.

Yes, but: Commercialization has been slow by design. Each patient must undergo stem cell collection, months of manufacturing, and chemotherapy conditioning before reinfusion; a process that can stretch close to a year and carries risks like infertility. 

The bright side: The number of patients initiating treatment nearly tripled year over year, and 30 of 64 2025 infusions happened in Q4 alone. Analysts now see potential for $227 million in 2026 sales, with pediatric expansion and an FDA priority voucher adding growth levers.

Bottom line: Gene editing doesn’t scale like a pill, but with time, Casgevy might end up being a blockbuster.

—Joachim E.

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SNAPSHOT

Gilead spends $80M upfront on Genhouse Bio’s oral cancer drug in $1.5B-plus deal

去年五月去苏州拍的东方之门，那天天气真的好，拍出来非常漂亮，可惜飞机对的歪了点，希望疫情早日结束，以后有机会再去拍

Gilead is acquiring global rights to GH31, a MAT2A-targeted oral cancer drug from China-based Genhouse Bio, for $80 million upfront and up to $1.45 billion in milestone payments.

Why it matters: The deal bolsters Gilead’s oncology pipeline as cancer revenues slip and highlights the growing role of China-based biotechs in global drug development.

Backstory: Gilead’s oncology sales fell about 2% from 2024 to 2025, while HIV maintains itself as Gilead’s biggest unit. In recent years, Gilead has focused heavily on cell therapy and antibody-based cancer treatments via its subsidiary Kite Pharma, which struck a $120 million upfront deal with Pregene Biopharma last year and partnered with Biocytogen in 2024.

Big picture: Western drugmakers have increasingly turned to China for licensing deals, with this one marking the 17th China–West biotech deal this year, according to data from Endpoints News.

Zoom in: GH31 targets MAT2A, a metabolic regulator overexpressed in some tumors, using a synthetic lethality approach. The drug has cleared clinical trial approvals in both the U.S. and China, with a Phase 1 trial planned in China this year. Other players in MAT2A include BeOne Medicines and Ideaya; GSK exited a similar partnership with Ideaya in 2022.

What’s next: Gilead will advance GH31 through early-stage development as it continues expanding its oncology portfolio through partnerships. Genhouse will progress its broader pipeline, including SHP2 inhibitor GH21, slated for a Phase 3 trial in 2027. Genhouse recently raised 300 million RMB (~$43 million) in crossover financing and filed for a Hong Kong IPO.

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German Biotech Days 2026: Community of Innovators

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A comprehensive conference program, a structured partnering system and extensive networking opportunities make DBT 2026 a central meeting point for the life sciences community.

SNIPPETS

What’s happening in biotech today?

🚫FDA rejection: The FDA has issued a complete response letter rejecting Disc Medicine’s rare disease drug bitopertin for erythropoietic protoporphyria (EPP), despite the therapy receiving expedited review through the Commissioner’s National Priority Voucher program. Although the agency acknowledged that the phase 2 trial met its primary endpoint by reducing protoporphyrin IX (PPIX) levels, it questioned the reliance on a surrogate endpoint and noted that the reduction did not clearly correlate with improved sun tolerance, a key clinical outcome. Disc plans to address these concerns through its ongoing phase 3 Apollo trial.

🔬 R&D retrenchment: Moderna reduced its R&D spending by 31% in 2025 to $3.1 billion, down from $4.5 billion in 2024, primarily due to winding down large phase 3 respiratory trials and ongoing portfolio prioritization. Amid federal funding cuts, including the loss of a $590 million bird flu contract, and growing U.S. skepticism toward mRNA vaccines under new health leadership, the company has shifted focus away from new late-stage vaccine trials toward cancer programs and a phase 3 norovirus vaccine. Moderna also faced layoffs, pipeline cuts and an FDA refusal-to-file letter for its next-generation flu shot.

💰 Pharma prepper: Eli Lilly has built a $1.5 billion pre-launch inventory of its oral obesity drug orforglipron ahead of an FDA decision expected in April 2026, aiming to prevent the supply shortages that affected its injectable GLP-1 therapies Mounjaro and Zepbound in 2022. The stockpiling, supported by major U.S. manufacturing investments, reflects confidence in approval and preparation for strong demand in the growing oral weight loss market, where Novo Nordisk’s recently launched oral Wegovy has gained early traction. Analysts forecast orforglipron could generate $13 billion in sales by 2031.

 🦷 Tooth stem cells: Kidswell Bio and Treehill Partners have launched Kidswell USA, a new U.S.-based regenerative medicine company aimed at accelerating the development of SQ-SHED, a stem cell therapy derived from human exfoliated deciduous teeth for pediatric cerebral palsy (CP), a permanent, non-progressive neurodevelopmental disorder caused by brain damage close to birth. The partnership combines Kidswell’s scientific expertise with Treehill’s clinical development optimization model to advance the program efficiently toward commercialization, with a focus on the U.S. market, where CP affects 1 in 345 children and lacks disease-modifying treatments.

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