Boehringer backs Envisagenics in $1B RNA splicing deal

Why it matters: Envisagenics searches alternative RNA splicing for tumor-specific protein variants that conventional gene-centered approaches can miss. The multiyear collaboration combines Envisagenics’ AI-based SpliceCore platform with Boehringer’s oncology drug development capabilities.

• Boehringer will have options to exclusively license selected targets after the research work. The potential $1 billion-plus package includes an upfront payment, research funding, option fees and development, regulatory and commercial milestones, plus royalties. Individual amounts were not disclosed.

How it works: One gene does not necessarily produce just one protein. During RNA splicing, cells can assemble RNA transcripts in different ways, producing multiple protein variants (isoforms) from the same gene.

• In cancer, that process is often disrupted. The resulting isoforms can be more specific to tumor cells than the normal version of the protein, potentially providing drug developers with targets that spare healthy tissue. Such altered splicing has also been linked to mechanisms cancer cells use to develop drug resistance.

Zoom in: Envisagenics has built SpliceCore around more than 14 million RNA splicing events, according to the company. Its platform combines AI and machine learning with transcriptomic data and experimental validation to identify changes that are disease-specific, occur in enough patients and could realistically be targeted with a drug.

• That shifts the role of AI further upstream: rather than using algorithms primarily to optimize or characterize known targets, Envisagenics is using them to find new ones.
• Boehringer plans to assess the resulting targets across several drug formats, including antibody-drug conjugates, T-cell engagers and multispecific antibodies.

The context: The deal fits Boehringer’s broader oncology strategy of combining new biological targets with different therapeutic modalities.

• In April 2025, the German pharma partnered with Tessellate Bio to explore synthetic lethality in tumors using alternative lengthening of telomeres. Six months later, it teamed up with South Korea’s AimedBio to develop an ADC against a tumor-specific marker.
• Its internal pipeline is also becoming increasingly biomarker-driven. In June, Boehringer started three Phase 3 oncology trials: two testing DLL3-targeted T-cell engager obrixtamig and another testing HER2 inhibitor zongertinib.

Backstory: Envisagenics was founded in 2014 as a Cold Spring Harbor Laboratory spinout and was an early adopter of the combination of RNA sequencing, alternative splicing and AI. The company initially operated mainly as a bioinformatics business before moving computational findings into its own laboratory for experimental validation four years after its launch.

• It has previously worked with Johnson & Johnson, Biogen and Bristol Myers Squibb, which partnered with Envisagenics in 2022 to identify splicing-derived oncology targets. A 2021 Series A was led by Red Cell Partners, with investors including Microsoft’s M12 and Madrona Venture Group. Bristol Myers Squibb joined its Series B as a strategic investor in 2024. Envisagenics did not disclose the size of that round, although external databases put it at about $25 million.
• The company has since kept a relatively low profile while continuing to develop SpliceCore and build an internal pipeline. Its lead program, ENV-375, is a preclinical antisense therapy for ALS, while its oncology work includes targets for ADCs, T-cell engagers, multispecific antibodies and radioligands.

What to watch: The Boehringer partnership is now a test of whether Envisagenics can turn its large computational search space into targets that survive biological validation.

• That distinction matters. AI can narrow down candidates, but a target still needs to be sufficiently tumor-specific, biologically relevant and accessible with an appropriate drug format. Boehringer provides that second half of the equation, bringing experience across ADCs, bispecific and multispecific antibodies and other oncology modalities.
• If Envisagenics can repeatedly identify splicing-derived targets that meet those criteria, RNA isoforms could provide pharma companies with a new pool of oncology targets beyond the increasingly crowded set of conventionally identified proteins.