Modalities

Traditional chemically synthesized drugs that target proteins, enzymes, and receptors. Still the backbone of the pharmaceutical industry, now increasingly designed with AI-driven approaches for improved selectivity and ADMET properties.

Engineered proteins that bind specific targets with high affinity. Bispecific antibodies engage two targets simultaneously, enabling novel mechanisms like T-cell redirection. AI is accelerating antibody humanization, affinity maturation, and developability optimization.

Short chains of amino acids that can modulate biological targets with high specificity. The GLP-1 receptor agonist revolution (semaglutide, tirzepatide) has made peptides the fastest-growing therapeutic modality by revenue.

Targeted therapies combining antibody specificity with cytotoxic payloads via chemical linkers. The ADC field is experiencing explosive growth with next-generation linker-payload technologies and novel targets beyond oncology.

Small molecules that recruit the cell's protein disposal machinery to selectively destroy disease-causing proteins, including previously "undruggable" targets. PROTACs and molecular glues represent fundamentally new pharmacology.

Delivering functional copies of genes to correct genetic disorders using viral vectors like adeno-associated virus (AAV) or lentivirus. Promises one-time curative treatments for monogenic diseases.

Living medicines engineered from patient or donor cells to fight disease. CAR-T cells have transformed blood cancer treatment; next-generation approaches include allogeneic (off-the-shelf), CAR-NK, and tumor-infiltrating lymphocyte (TIL) therapies.

Using messenger RNA to instruct cells to produce therapeutic proteins. Proven at scale with COVID-19 vaccines, mRNA platforms are now being applied to oncology (personalized cancer vaccines), rare diseases, and autoimmune conditions.

Short nucleic acid sequences that modulate gene expression at the RNA level. Antisense oligonucleotides (ASOs), small interfering RNA (siRNA), and microRNA therapeutics can silence or modulate previously undruggable targets.

Precision tools for modifying DNA sequences in living cells. CRISPR-Cas9 achieved the first approved therapy (Casgevy); next-generation approaches like base editing and prime editing offer even greater precision without double-strand breaks.

Targeted drugs carrying radioactive isotopes to deliver radiation directly to cancer cells. Theranostics combines diagnostic imaging with targeted therapy. Massive investment in isotope supply chains (Ac-225, Lu-177) is enabling growth.

Therapies that modulate the human microbiome to treat disease. Includes live biotherapeutics, engineered bacteria, and microbiome-derived metabolites targeting conditions from C. diff infections to inflammatory bowel disease.

Transplanting organs or tissues from genetically modified animals (primarily pigs) into humans. Gene editing enables removal of pig antigens and insertion of human immune-compatible genes, potentially solving the organ shortage crisis.

Diagnostic tests co-developed with therapeutics to identify patients most likely to benefit from a specific treatment. Essential for precision medicine and increasingly required by regulators for targeted therapy approvals.

Blood-based tests that detect circulating tumor DNA (ctDNA), circulating tumor cells, or other biomarkers to diagnose cancer, guide treatment selection, and monitor response — without invasive tissue biopsies.

Machine learning applied to medical imaging and digital pathology for faster, more accurate diagnosis. Applications span radiology (CT, MRI), histopathology (whole slide imaging), and retinal screening.

Computational approaches using machine learning, deep learning, and foundation models to accelerate drug discovery — from target identification and molecular design to ADMET prediction and clinical trial optimization.

Engineering biological systems to create new functions — from designing novel enzymes and metabolic pathways to building cell factories for drug manufacturing. Enables production of complex molecules at scale.

Microfluidic devices that simulate human organ function for drug testing, reducing reliance on animal models. The FDA Modernization Act 2.0 has accelerated adoption of these alternative testing platforms.

Technologies for manufacturing biological drugs at scale, including continuous manufacturing, single-use bioreactors, and process analytical technology (PAT). Critical for making advanced therapies commercially viable.

AI-powered optimization of clinical trial design, patient recruitment, site selection, and endpoint analysis. Enables adaptive trials, synthetic control arms, and real-world evidence integration to reduce time and cost.