Drug Discovery and Development

Drug discovery and development is the multistage process by which researchers identify a disease-relevant target, find compounds or biologics that modulate it, and characterize their efficacy, safety, and manufacturability before clinical testing. Only about 10% of clinical programs eventually receive approval,^1,2 and an analysis of clinical trial data from 2010 to 2017 attributed 40% to 50% of failures to lack of clinical efficacy and about 30% to unmanageable toxicity.¹ This brief guide highlights the general workflow, methods, and product categories that support each stage, from target discovery through process development, including tools for cell and gene therapy and outsourced services.

Drug discovery typically begins with target identification and validation, in which genomic, proteomic, and functional studies establish that modulating a gene or protein is likely to alter disease biology. Validated targets are then screened against compound or biologic libraries, often at high throughput, and confirmed hits are characterized in biochemical and cell-based assays that quantify potency, selectivity, and mechanism. Leads that pass these filters move into preclinical testing, which typically progresses from three-dimensional (3D) cell models to animal studies that assess efficacy, pharmacokinetics, and toxicity, followed by process development to establish scalable expression, purification, analytical, and formulation methods. Together, these tiered stages narrow a large candidate pool to a small number of well-characterized leads, and specialized tools for cell and gene therapies or contracted services can fill gaps in equipment, expertise, or capacity at any step.

Target Discovery and Validation

Target discovery and validation identifies the genes, proteins, or pathways implicated in disease and confirms their functional relevance, typically by combining genomic, functional, and spatial approaches to build a weight-of-evidence case before resources are committed to lead development. Human genetic evidence is one measure of that relevance, as the probability of success for drug mechanisms with genetic support is estimated to be 2.6 times greater than for those without.²

Next generation sequencing (NGS) kits, including library preparation, RNA sequencing (RNA-Seq), and single cell sequencing kits, support genome-wide identification of disease-associated variants and expression patterns.
Epigenetics tools, such as chromatin immunoprecipitation sequencing (ChIP-Seq) and bisulfite sequencing (Methyl-Seq) kits, characterize regulatory mechanisms like histone modification and DNA methylation.
CRISPR products, including CRISPR-Cas9 and guide RNA reagents, Cas9 nuclease, and knockout kits, are widely used to validate candidates by perturbing gene expression in cellular models, with transfection kits and gene editing services supporting delivery of the editing components.
Expression analysis tools, such as quantitative PCR (qPCR), reverse transcription PCR (RT-PCR), and microarrays, quantify transcript abundance across conditions, whereas proteomics tools, such as mass spectrometry and protein arrays, provide unbiased confirmation at the protein level.
Spatial biology platforms, along with immunohistochemistry (IHC) and in situ hybridization, confirm target expression within native tissue architecture, which matters because expression in bulk assays may not reflect the cellular context relevant to disease pathology.

Lead Discovery and Screening

Lead discovery and screening tests candidate compounds or biologics against a validated target to identify hits with a desired activity, such as enzyme inhibition, receptor modulation, or selective cytotoxicity. Primary screens are often run at high throughput across thousands to millions of candidates, and an assay with a Z'-factor between 0.5 and 1.0 is considered excellent for screening.³

Enzyme assay kits, including binding, inhibitor screening, and time-resolved fluorescence resonance energy transfer (TR-FRET) formats, measure catalytic activity or ligand-target interaction in microplates, and the homogeneous, wash-free TR-FRET format supports higher throughput than traditional colorimetric or radiometric alternatives.
Enzyme-linked immunosorbent assay (ELISA) kits, including multiplex formats, quantify protein targets or biomarkers, whereas cell-based assays such as viability, cytotoxicity, apoptosis, G protein-coupled receptor (GPCR), and parallel artificial membrane permeability assay (PAMPA) kits connect a compound's phenotypic effect to a mechanism, which complements biochemical formats that do not always capture mechanistic detail.
Cell analysis equipment, including high content screening and real-time cell analysis systems, provides image- or impedance-based readouts across large compound sets using primary cells and cell lines, and the choice between them typically balances physiological relevance against assay-to-assay reproducibility.
Enzyme inhibitors are used as comparators and tool compounds during hit validation, and high-throughput screening (HTS) services offer an outsourced route for laboratories that lack the automation or library access needed for large primary screens.

Preclinical and In Vivo Research Tools

Preclinical research bridges cell-based screening and clinical testing, as compounds that perform well in monolayer or biochemical assays often fail in more physiologically complex systems. The U.S. Food and Drug Administration (FDA) Modernization Act 2.0, signed in December 2022, allows sponsors to use cell-based assays, microphysiological systems, and bioprinted or computer models as nonclinical tests, although it does not eliminate animal testing.⁴

3D cell culture models, including spheroid and organoid culture media, hydrogels, basement membrane extracts, and ultra-low attachment plates, recapitulate tissue architecture more closely than monolayer culture, albeit at lower throughput and higher per-study expense, and 3D bioprinters can build multi-cell-type constructs.
Histology and IHC tools, including kits, antibodies, stains, and histology services, localize target expression and assess tissue-level effects in fixed sections from in vitro and in vivo studies.
Animal models remain the standard system for evaluating efficacy, pharmacokinetics, and toxicity, as no in vitro system fully captures the interactions among organs, immune function, and metabolism that govern drug response, and animal behavior and monitoring equipment, such as blood pressure monitoring systems, catheters, and syringe infusion pumps, supports standardized measurement of physiological endpoints.
In vivo imaging systems allow longitudinal, non-invasive tracking of tumor growth or biodistribution in the same animal, which can reduce the number of animals needed relative to endpoint-only designs, while neuroscience and electrophysiology equipment supports characterization of neural activity in central nervous system programs.

Cell and Gene Therapy

Cell and gene therapies are a growing class of biologics, and the FDA list of approved cellular and gene therapy products, last updated in September 2026, now includes more than 50 licensed products.⁵ Cell therapies use cells from the patient (autologous) or from donors (allogeneic), whereas gene therapies deliver transgenes through vectors such as adeno-associated virus (AAV), adenoviral, herpes simplex virus (HSV), or lentiviral vectors.

Cell sorters and cell isolation products are used to obtain the desired populations of human cells, with cell culture media formulated for stem and primary cells supporting expansion and viability.
CRISPR products enable editing of target genes, and transfection reagents support delivery of the editing components and transgenes.
Virus vectors and particles, including AAV, adenoviral, lentiviral, and retroviral formats, deliver transgenes, and although integrating vectors can provide long-term expression, they may alter cellular gene expression and contribute to tumorigenicity, which makes longer-term follow-up studies prudent.
Flow cytometers are commonly used to characterize cell identity and to assess viability, purity, and strength throughout manufacturing, and product-specific assays developed early in development help ensure a successful final product.

Drug Discovery and Development Services

Drug discovery and development services provide access to the expertise, instrumentation, and capacity that a research team may lack, and many providers accommodate customized project requests. Prospective buyers typically weigh the savings in capital expenditure and time from outsourcing against reduced direct control over methods and data, and should confirm how a provider handles customization, data transfer, and quality documentation.

HTS services and assay development services support the early stages, in which assays are designed and incorporated into workflows that allow thousands to millions of compounds to be tested without in-house automation.
Chemical synthesis services and in silico analysis services support lead identification and optimization, as computational methods are often used to prioritize candidates before synthesis and testing.
In vivo testing services provide animal studies of efficacy, pharmacokinetics, and toxicity, which can remove the need to maintain animal facilities in-house.
AAV services and lentivirus services supply viral vectors for gene delivery programs, and because viral vector manufacturing presents recurring quality control challenges, including confirming purity and potency, controlling contamination risk, and scaling production without loss of yield, a contract development and manufacturing organization (CDMO) is often a practical route for smaller developers without established in-house expertise.
Formulation development services and pharmaceutical manufacturing services address downstream needs such as drug formulation and Good Manufacturing Practice (GMP) manufacturing.

Process Development and Formulation

Process development and formulation translate a candidate from a laboratory-scale asset into a reproducible, scalable product suitable for manufacturing and clinical use, and the work covers expression, purification, analytical characterization, and formulation. Processes that are not adequately characterized at small scale often encounter yield, purity, or stability problems after scale-up, so analytical characterization is typically performed at each scale-up step to detect process-related problems before they affect product quality.

Cell culture equipment and bioreactors, including single-use and stirred-tank systems, together with automated cell culture systems, establish the upstream conditions that set how much protein or biomass is available for every downstream step.
Chromatography equipment, including high performance liquid chromatography (HPLC) and gas chromatography (GC) systems with their columns and media, and protein purification tools separate the target molecule from host cell proteins, nucleic acids, and other process-related impurities.
Protein analysis and characterization tools, such as surface plasmon resonance (SPR), mass spectrometry, amino acid analyzers, and protein crystallization systems, confirm identity, purity, and structure, while analytical instrumentation, including particle size analyzers and thermal analysis instruments, supports quantitative decisions such as buffer pH adjustment and particle size specifications for the formulated product.
Automated liquid handling systems, including nanoparticle and lipid nanoparticle systems, reduce the variability of manual pipetting and plate handling and increase the throughput needed during scale-up, and antibody production and development services supply antibodies used as analytical reagents for process monitoring or as therapeutic candidates.

Prospective buyers should consider how well each tool or service scales across stages, as methods that suit one stage do not always transfer to the next, and how its outputs feed the assays and decisions that follow. Selecting complementary methods at each stage and confirming findings with an orthogonal approach is generally the most reliable way to build confidence in a candidate before it advances toward the clinic.

References

Sun D, Gao W, Hu H, Zhou S. Why 90% of clinical drug development fails and how to improve it? Acta Pharm Sin B. 2022;12(7):3049-3062. doi:10.1016/j.apsb.2022.02.002
Minikel EV, Painter JL, Dong CC, Nelson MR. Refining the impact of genetic evidence on clinical success. Nature. 2024;629(8012):624-629. doi:10.1038/s41586-024-07316-0
Zhang JH, Chung TD, Oldenburg KR. A simple statistical parameter for use in evaluation and validation of high throughput screening assays. J Biomol Screen. 1999;4(2):67-73. doi:10.1177/108705719900400206
Consolidated Appropriations Act, 2023, Pub L No. 117-328, §3209 (FDA Modernization Act 2.0). Signed December 29, 2022.
U.S. Food and Drug Administration. Approved Cellular and Gene Therapy Products. Content current as of September 17, 2026. Accessed October 13, 2026. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products