What’s New in Biotech in 2026? Breakthroughs and Research Trends

Regulatory momentum for human-relevant testing models

• What’s changing: The FDA has formally implemented its "Roadmap to Reducing Animal Testing," transitioning toward New Approach Methodologies (NAMs) like organ-on-a-chip and in silico models for preclinical safety.
• Why it matters: This shifts R&D toward human-relevant data, improving translational fidelity and reducing the high failure rate of drugs that pass animal trials but fail in humans.
• What to watch in 2026: A surge in INDs (Investigational New Drugs) incorporating NAM data and pilot programs that offer accelerated review for animal-free submissions.
• What it means for labs: Investment in organoid platforms and advanced in vitro systems is now a strategic necessity for regulatory alignment, especially as labs look to integrate new tools into existing workflows and systems such as ELNs, LIMS, and purchasing infrastructure. Learn how modern lab workflows support this kind of integration in ZAGENO’s procurement platform FAQ.

AI becomes part of regulated drug development

• What’s changing: Following the 2025 draft guidance on AI in the drug lifecycle, the FDA now requires specific "credibility assessment frameworks" for AI-generated data in regulatory packages.
  • Why it matters: AI has moved from a "black box" discovery tool to a transparent, validated component of formal submissions.
• What to watch in 2026: AI models cited in IND/BLA packages and the use of "digital twins" in early-phase trial design.
• What it means for labs: Data governance and reproducibility are the new gold standards. Labs must ensure AI-driven insights are auditable.

Read more: FDA draft guidance on advancing AI credibility for drug and biological submissions

Prime editing expands beyond early leads

• What’s changing: While initial leads like PM359 proved the concept, the focus has shifted toward high-impact targets like cystic fibrosis. Prime editing is now entering a broader "human validation" phase across multiple therapeutic areas.
• Why it matters: Its ability to perform "search-and-replace" genetic edits with fewer off-target effects than traditional CRISPR makes it a safer alternative for complex diseases.
• What to watch in 2026: Clinical data from new prime editing candidates targeting liver and lung diseases.
• What it means for labs: Labs should evaluate molecular biology workflows that support high-precision "search-and-replace" assay validation.

Read more: Prime Medicine corporate strategy and pipeline

In vivo base editing targets cardiovascular disease

• What’s changing: Following Eli Lilly’s 2025 acquisition of Verve Therapeutics, the base-editing candidate VERVE-102 has entered expanded Phase 2 trials for permanent LDL-C reduction.
• Why it matters: This represents a move toward "one-and-done" genetic treatments for common chronic conditions like hyperlipidemia.
• What to watch in 2026: Long-term safety data and the expansion of base editing into other metabolic disorders.
• What it means for labs: Preclinical and translational teams should prepare for more complex workflows spanning lipidomics, genetic analysis, and safety profiling, which places additional operational demands on lab managerscoordinating people, tools, and suppliers across programs

Read more: Verve Therapeutics pipeline progress

CAR-T therapies extend to autoimmune disease

• What’s changing: Early 2026 data confirms that CD19-targeted CAR-T therapies can achieve "durable remission" in patients with lupus and systemic sclerosis by "resetting" the immune system.
• Why it matters: CAR-T is no longer just a cancer treatment; it is a potential cure for autoimmune diseases that previously required lifelong immunosuppression.
• What to watch in 2026: Results from Phase 2 "Immune Reset" trials across multiple rheumatology indications.
• What it means for labs: High demand for T-cell engineering and immunophenotyping capabilities and scalable workflows.

Read more: Bristol Myers Squibb Phase 1 CAR-T autoimmune therapy data

Personalized mRNA cancer vaccines progress

• What’s changing: The Moderna/Merck phase 3 trial (INT-001) for personalized melanoma vaccines is a focal point for 2026, moving the needle on neoantigen-based therapy.
• Why it matters: This proves mRNA's versatility in training the immune system to recognize a patient’s specific tumor markers.
• What to watch in 2026: Late-stage trial readouts and biomarker correlations for solid tumors.
• What it means for labs: Increased reliance on rapid NGS (Next-Gen Sequencing) and bioinformatics pipelines.

Read more: Moderna mRNA cancer vaccine pipeline

Radioligand therapies move into earlier oncology use

• What’s changing: The FDA’s 2025 label expansion for agents like Pluvicto now allows for use in prostate cancer patients before they undergo chemotherapy.
• Why it matters: Targeted radiation is becoming a first-line consideration rather than a "last resort" treatment.
• What to watch in 2026: The emergence of new isotopes (like Actinium-225) that offer even higher precision.
• What it means for labs: Specialized infrastructure for handling radiolabeled compounds is becoming a major lab requirement.

Read more: FDA approval for expanded Pluvicto use

Spatial biology platforms scale commercially

• What’s changing: With the 2026 launch of Illumina’s whole-transcriptome spatial technology and their "Connected Multiomics" software, spatial biology is now accessible to the broader market.
• Why it matters: Researchers can now map gene expression directly onto tissue architecture at a commercial scale, rather than in niche studies.
• What to watch in 2026: The integration of spatial transcriptomics with proteomics in a single workflow.
• What it means for labs: A need for high-throughput spatial imaging platforms and significant data storage solutions.

Read more: Illumina spatial transcriptomics technology

Targeted protein degradation (TPD) nears the clinic

• What’s changing: The field has moved from complex PROTACs toward “Molecular Glues”—smaller, more orally bioavailable molecules that can degrade "undruggable" proteins.
• Why it matters: This modality creates access to 80% of the proteome that traditional inhibitors cannot reach.
• What to watch in 2026: First-in-human data for molecular glues targeting neurodegeneration and inflammation.
• What it means for labs: Assay strategies must pivot toward measuring protein turnover rather than just inhibition.

Read more: Targeted protein degradation

Agentic AI platforms reshape discovery partnerships

• What’s changing: In early 2026, major partnerships (like Variant Bio and Boehringer Ingelheim) have showcased "Agentic AI"—platforms that don't just find data, but actually design and refine their own experiments.
• Why it matters: This automates the hypothesis-testing loop, drastically shortening the time from target identification to lead optimization.
• What to watch in 2026: AI-led licensing deals where the "agent" is credited with the lead discovery.
• What it means for labs: Labs will increasingly prioritize data integration infrastructure and interoperable systems to leverage agentic AI insights.

Read more: Variant Bio launches AI-powered platform for drug discovery using genetic data