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Biotech strategy glossary

Plain-language definitions for the terms used across this resource hub and BridgeLine's capabilities.

Resources · Glossary · BridgeLine · v1.0 published 2026-04-29 · last reviewed 2026-04-2983 terms · A–Z

The biotech strategy and regulatory vocabulary used across the BridgeLine site. Definitions are written for a non-ICP reader - a career changer, an MBA, an MD-PhD without industry experience, a securities lawyer, a journalist - without sacrificing the precision an operator needs.

A

ADCAntibody-Drug Conjugate
A targeted therapy that links a small-molecule cytotoxic payload to a monoclonal antibody via a chemical linker. The antibody directs the payload to cells expressing a target antigen, ideally sparing healthy tissue. ADCs are a major modality in oncology and have meaningful CMC and manufacturing complexity.
ATMPAdvanced Therapy Medicinal Product
An EU regulatory category for gene therapies, somatic cell therapies, and tissue-engineered products. The classification carries specific manufacturing, quality, and clinical requirements set by EMA's Committee for Advanced Therapies.
AAVAdeno-Associated Virus
A small, non-pathogenic viral vector widely used to deliver gene therapies. Different AAV serotypes have different tissue tropisms, which lets developers target liver, eye, muscle, or central nervous system. Capsid engineering and manufacturing yield are central CMC concerns.

B

Bayh-Dole Act
35 USC §§200-212. The 1980 US law allowing universities, nonprofit research institutions, and small businesses to retain title to inventions made with federal funding (subject to election within statutory windows). Bayh-Dole is the foundational US academic-technology-transfer framework and creates obligations around disclosure (iEdison), preference for US manufacturing, and the federal government's reserved license and march-in rights.
Biosimilar
A biologic product highly similar to an already-approved reference biologic, with no clinically meaningful differences in safety or efficacy. Biosimilars follow an abbreviated regulatory pathway that relies on extensive analytical comparability rather than full duplication of the reference product's clinical program.
Bridging study
A clinical or nonclinical study designed to extrapolate data from one population, formulation, or region to another. Common uses include bridging from one manufacturing process to a new one, or from one ethnic population to another for global development.
Briefing book
The structured document a sponsor sends to a regulatory agency before a formal meeting (Pre-IND, Type B, Type C, or scientific advice). It contains background, specific questions, and the sponsor's proposed answers, and it sets the agenda for the meeting itself.
BLABiologics License Application
The FDA submission that requests approval to market a biologic product in the United States. The BLA contains the full chemistry, manufacturing, and controls package alongside nonclinical and clinical data, and approval triggers the right to commercial distribution.

C

CMCChemistry, Manufacturing, and Controls
The portion of a regulatory submission covering how a drug substance and drug product are made, characterized, tested, and released. CMC scales in cost and complexity through development and is a frequent diligence flashpoint for biologics and cell and gene therapy assets.
CDMOContract Development and Manufacturing Organization
A vendor that develops and manufactures drug substance or drug product on behalf of a sponsor. CDMOs differ from CROs by owning physical manufacturing assets (cell-culture suites, fill-finish lines) rather than primarily running studies.
CROContract Research Organization
A vendor that runs nonclinical or clinical studies on behalf of a sponsor. Scope ranges from a single GLP toxicology study to full clinical operations across multiple trials. CRO selection and oversight are recurring diligence topics in early-stage biotech.
CDxCompanion Diagnostic
An in vitro diagnostic device that provides information essential for the safe and effective use of a corresponding therapeutic product. CDx development typically runs in parallel with the therapeutic and is reviewed by FDA under both the drug pathway (CDER or CBER) and the device pathway (CDRH), with co-approval expected at therapeutic launch when the label requires the test.
Crossover (in trial)
A clinical trial design where each participant receives more than one treatment in sequence, separated by a washout period. Crossover designs improve statistical power per subject but only fit indications and endpoints that return to baseline between treatments.
CRLComplete Response Letter
An FDA letter stating that an application cannot be approved in its current form, with the deficiencies named. A CRL is not a final denial; the sponsor can address the issues and resubmit, but the letter typically delays approval by months to years.
CTAClinical Trial Application
The submission used to authorize a clinical trial in jurisdictions outside the United States, including the EU (under the Clinical Trials Regulation) and most other markets. Functionally analogous to the FDA's IND, with jurisdiction-specific content and review timelines.

D

Diligence-grade
BridgeLine usage: work product whose claims, sources, and assumptions are documented to a standard that survives scrutiny by an investor, partner, or acquirer. The opposite of pitch-deck-grade material that holds up only at a glance.
Drug Master FileDMF
A confidential filing submitted to a regulatory agency that contains detailed information about a manufacturing facility, process, or component. A sponsor can reference a third party's DMF in its own application without the third party disclosing proprietary information directly to the sponsor.
Dose escalation
The Phase 1 design pattern in which sequential cohorts receive increasing doses of an investigational product to identify the maximum tolerated dose, recommended Phase 2 dose, or pharmacologically active range. Common designs include 3+3, accelerated titration, and Bayesian model-based approaches.

E

eCTDElectronic Common Technical Document
The standardized electronic submission format used by FDA, EMA, PMDA, and other ICH regulators. The eCTD organizes a regulatory submission into five modules covering administrative information, summaries, quality, nonclinical, and clinical content.
EOP2End-of-Phase 2 meeting
An FDA meeting held after Phase 2 data are available to align on Phase 3 design, endpoints, statistical plan, and any additional nonclinical or CMC requirements before pivotal trials begin. Type B meeting in FDA nomenclature.
Equivalent doseHED
A dose adjusted across species using allometric scaling so that nonclinical species exposures can be related to a human dose. See HED.

F

F&AFacilities and Administrative costs
The indirect cost recovery a university charges on top of direct costs in a sponsored research agreement or federal grant. Federally negotiated F&A rates at major US research universities are typically 50-70% on top of direct costs, which can dramatically inflate the true budget for company-funded research at an academic founder's lab. Industrial F&A rates are usually negotiable and lower than the federal rate.
Fast Track
An FDA designation that facilitates development and expedites review of drugs treating serious conditions where the drug demonstrates the potential to address an unmet medical need. Benefits include more frequent agency interaction and eligibility for rolling review.
FIHFirst-in-Human
The first administration of an investigational product to a human subject, typically in a Phase 1 study. FIH design considers starting dose (often informed by NOAEL and HED), dose escalation rules, and a careful safety monitoring plan.
FDA Form 483
The form an FDA investigator issues at the conclusion of a facility inspection to list observed conditions that may violate the FD&C Act and related regulations. A 483 is not a final agency action but signals issues the sponsor or facility must address.

G

GMPGood Manufacturing Practice
The quality system that governs how drug substance and drug product are manufactured, tested, and released. GMP applies most stringently to material intended for human use; nonclinical material may be made under reduced quality systems with appropriate justification.
GLPGood Laboratory Practice
The quality system governing nonclinical safety studies that will be submitted to regulators. GLP covers protocol, study conduct, data integrity, archiving, and quality assurance, and is required for the pivotal toxicology and safety pharmacology studies supporting an IND or CTA.
GCPGood Clinical Practice
The international quality standard that governs how clinical trials are designed, conducted, recorded, and reported. ICH E6 is the operative reference. GCP compliance is required for data to be accepted by regulators in ICH jurisdictions.
Gene therapy
A therapy that introduces, removes, or modifies genetic material in a patient's cells to treat or prevent disease. Delivery vehicles include AAV, lentivirus, lipid nanoparticles, and ex vivo modification of patient cells. Subject to specific FDA and EMA frameworks for ATMPs and gene therapies.
GRASGenerally Recognized as Safe
A US FDA pathway under section 201(s) of the FD&C Act for food substances exempt from premarket approval requirements applicable to food additives. A substance can be GRAS through scientific procedures or, for substances used before 1958, through experience based on common use in food. The pathway is administered by CFSAN and does not apply to drug or biologic products.

H

HEDHuman Equivalent Dose
A dose in humans that is calculated to produce exposure comparable to a dose given in a nonclinical species, typically using body-surface-area scaling. HED is one input to first-in-human starting-dose selection and is a recurring topic in Pre-IND interactions.
HKEX 18AHong Kong Stock Exchange Chapter 18A
Hong Kong Stock Exchange's 2018 listing rule allowing pre-revenue biotech IPOs. Requires HK$1.5B minimum market capitalization at listing and a "Senior Clinical Trial" product (at least one Phase II or III asset with regulatory confirmation of significant clinical progress). Cornerstone investors face a 6-month post-IPO lockup; Weighted Voting Rights for biotech founders carry sunset provisions. A primary alternative IPO route to Nasdaq for China and Asia biotech.

I

iEdison
NIH's online system (Interagency Edison) for federal funding recipients to disclose subject inventions and report utilization. Required compliance for Bayh-Dole subject inventions; submissions cover invention disclosure, election of title, patent prosecution updates, and annual utilization reports. iEdison filings are an active, ongoing obligation - distinct from the (theoretical) march-in rights of the funding agency.
IIAInter-Institutional Agreement
A contract between two or more universities or research institutions that share rights to a jointly-invented technology. The IIA names the lead institution for IP prosecution and licensing, allocates revenue, and sets the framework for downstream company licensing. Without an IIA, joint inventions can produce blocked title that surfaces during company diligence years later.
INDInvestigational New Drug
The FDA submission a sponsor files to begin clinical trials in the United States. The IND contains nonclinical pharmacology and toxicology, CMC, prior human experience, and the proposed clinical protocol. The clinical hold clock starts at submission and runs for 30 days unless extended.
IND-enabling
The nonclinical work - toxicology, safety pharmacology, ADME, and CMC - required to support an IND submission. Often used as a programmatic label for the late-preclinical phase of biotech development.
INTERACTInitial Targeted Engagement for Regulatory Advice on CBER ProducTs
INTERACT (Initial Targeted Engagement for Regulatory Advice on CBER ProducTs) is an FDA mechanism for early scientific dialog with CBER, occurring BEFORE a Pre-IND meeting. Used by gene therapy, cell therapy, and biologic sponsors to discuss specific scientific or regulatory issues that may inform later Pre-IND submissions. INTERACT is not a substitute for Pre-IND; it is upstream of it.
IMPDInvestigational Medicinal Product Dossier
The EU equivalent of the IND content, submitted as part of a Clinical Trial Application. The IMPD documents the quality, manufacture, and nonclinical and clinical data supporting investigational use of the product in a trial.
IP / FTOIntellectual Property / Freedom to Operate
Two related but distinct concepts. IP refers to the patents, trade secrets, and other rights a company owns. FTO asks whether the company can practice its planned product or process without infringing third-party patents. Both are diligence-critical and are typically assessed independently.
IVDRIn Vitro Diagnostic Regulation
EU Regulation 2017/746 governing in vitro diagnostic medical devices placed on the EU market. IVDR replaced the prior IVD Directive with a substantially stricter classification scheme, conformity assessment requirements, and clinical evidence standards. It applies to companion diagnostics co-developed with therapeutics in EU regulatory pathways.

J

J-NDAJapanese New Drug Application
Japan's new drug application dossier filed to PMDA - distinct from the US NDA; ICH M4 implementation differs in dossier expectations and Japanese-language requirements; review clocks 12 months standard / 9 months priority. Historically required Japan-specific clinical data; under ICH E5 and E17, sponsors increasingly use bridging strategies and global multi-regional trials. Review is conducted by PMDA with consultation from MHLW's Pharmaceutical Affairs and Food Sanitation Council.

K

kakushinteki iyakuhin shitei seidoInnovative Drug Designation (Japan)
Japan's Innovative Drug Designation under the Pharmaceutical Affairs Law - a parallel PMDA pathway to Sakigake providing prioritized consultation, faster review timelines, and concierge-style regulatory support for innovative medicines addressing serious diseases with high unmet need. Conferred designation triggers specific PMDA support mechanisms; functionally analogous in spirit to FDA Breakthrough or EMA PRIME.
KOLKey Opinion Leader
A clinician, scientist, or researcher whose expertise and reputation influence how a therapeutic area thinks about disease, endpoints, or specific products. KOL engagement supports protocol design, advisory boards, and commercial preparation, and is subject to disclosure and compliance rules.

L

LCMLifecycle Management
The portfolio strategy a sponsor applies after initial approval: new indications, new formulations, combination products, geographic expansion, pediatric extensions, and patent strategy. Strong LCM extends the commercial value of an asset; weak LCM compresses it.
Lei do BemBrazil R&D Tax Incentives (Law 11.196/2005)
Brazilian federal R&D tax incentive framework under Law 11.196/2005 (the "Good Law"). Provides automatic income tax deductions, IPI tax reductions on R&D equipment, and accelerated depreciation for companies engaged in technological research and innovation in Brazil, including biotech and pharma R&D. A primary fiscal incentive supporting Brazilian biopharma R&D investment.
LNPLipid Nanoparticle
A non-viral delivery vehicle composed of ionizable lipids, helper lipids, cholesterol, and PEG-lipids, used to encapsulate nucleic acid payloads such as mRNA and siRNA. LNPs are the delivery system behind several mRNA vaccines and a growing class of nucleic-acid therapeutics.

M

March-in rights
A theoretical Bayh-Dole authority allowing a federal funding agency to require a university or its licensee to grant additional licenses on a subject invention when practical application has not been achieved or health and safety needs are not being met. NIH has never exercised march-in despite 50+ years of authority and dozens of petitions, including under the 2023 NIST RFI on the framework. March-in is a federal-funding-agency right, not a routine commercial risk; do not conflate it with active iEdison reporting obligations.
MTAMaterial Transfer Agreement
A contract governing the transfer of biological materials, reagents, cell lines, plasmids, antibodies, or other tangible research property between institutions. MTAs allocate intellectual property rights in any modifications or derived inventions, restrict use to defined purposes, and set publication and confidentiality terms. UBMTA (Uniform Biological MTA) is the standardized template used between many US academic institutions.
MABELMinimum Anticipated Biological Effect Level
A dose-selection methodology for first-in-human studies of biotech-derived products, especially high-risk biologics. MABEL is calculated from in vitro receptor occupancy, cell-based potency, and PK/PD modeling rather than NOAEL alone, and is the EMA-preferred approach for agonist antibodies, T-cell engagers, and other immune-activating modalities where NOAEL-based starting doses can be unsafe.
MDREU Medical Device Regulation 2017/745
EU Regulation 2017/745 governing medical devices placed on the EU market, in force since May 2021. MDR replaced the prior Medical Device Directive with stricter classification rules, more demanding clinical evidence requirements, mandatory unique device identification (UDI), and an expanded role for notified bodies. Drug-device combination products and companion-device aspects of therapeutic programs may invoke MDR pathways alongside the medicinal-product framework.
Modality
The class of therapeutic platform a program is built on: small molecule, monoclonal antibody, ADC, cell therapy, gene therapy, mRNA, protein, peptide, oligonucleotide, and so on. Modality determines the regulatory framework, manufacturing approach, and diligence questions an asset will face.
MoAMechanism of Action
The biochemical or cellular pathway through which a therapeutic produces its effect - receptor binding, enzyme inhibition, gene knockdown, immune cell engagement, and so on. A clear, testable MoA underpins target selection, biomarker strategy, and translational design.
mRNA therapeutic
A therapy in which messenger RNA encoding a protein of interest is delivered to a patient's cells, where the cells then transiently produce that protein. Delivery is most often via LNPs. The category covers prophylactic vaccines, oncology vaccines, and protein-replacement therapies.

N

NADA / ANADANew Animal Drug Application / Abbreviated NADA
FDA Center for Veterinary Medicine (CVM) pathways for veterinary drug approval. NADA is the application for a new animal drug, with full safety and effectiveness data. ANADA is the abbreviated route for a generic equivalent of a previously approved animal drug, relying on bioequivalence rather than independent effectiveness studies. Both sit outside the human-therapeutics frame this hub uses.
NDANew Drug Application
The FDA submission requesting approval to market a small-molecule drug in the United States. The NDA contains the full nonclinical, clinical, CMC, and labeling package and is reviewed under PDUFA timelines.
NOAELNo Observed Adverse Effect Level
The highest dose of a test article in a nonclinical study at which no adverse effects are observed in the test species. NOAEL informs first-in-human starting-dose selection in combination with allometric scaling and pharmacokinetic considerations.
Nonclinical
All testing conducted outside of human subjects: in vitro assays, in vivo studies in animals, and computational models. Distinguished from preclinical, which is sometimes used loosely; in regulatory writing, nonclinical is the precise term for the data submitted to support clinical trials.
Notified body
An organization designated by an EU member state to assess the conformity of certain products before they are placed on the EU market. For medical devices and IVDs (under MDR and IVDR), notified bodies perform conformity assessment, technical-file review, and quality-system audits, and issue the CE certificates that allow CE marking. Notified-body capacity has been a multi-year bottleneck for MDR/IVDR transitions and is a planning input for any sponsor whose program touches device or diagnostic regulation.
Novel food
An EU regulatory category (Regulation (EU) 2015/2283) for foods or food ingredients that were not consumed to a significant degree in the EU before May 1997. Examples include cell-cultured proteins, insect-based ingredients, and certain biotech-derived food products. Pre-market authorization by the European Commission, supported by an EFSA safety assessment, is required before a novel food can be placed on the EU market. Outside the human-therapeutics scope of this hub but a recurring touchpoint for biotech founders working on agtech, food-tech, or cellular agriculture adjacencies.

O

Option agreement
A contract giving one party the right but not the obligation to license intellectual property within a defined window, typically in exchange for an option fee and a commitment to advance evaluation work. TTOs use option agreements to give startups time to evaluate whether a technology is worth a full exclusive license without the institution losing the ability to talk to other potential licensees if the option expires unexercised.
Operator-to-acquirer (BridgeLine usage)
BridgeLine's positioning, not industry vocabulary. Means strategy work is done with both the company-builder's perspective and the eventual acquirer's scientific-diligence perspective in mind: decisions made in early development are pressure-tested against the questions a strategic acquirer or large-pharma diligence team will ask years later. Listed here because the phrase appears across BridgeLine's own pages; readers should not treat it as a standardized industry term.

P

PANDRHPan-American Network for Drug Regulatory Harmonization
Voluntary technical alignment network across Pan-American national drug regulators, hosted by PAHO. Provides harmonized technical guidance documents and convening across LATAM jurisdictions but does not confer auto-recognition or mutual recognition of approvals - a Brazilian (ANVISA) protocol approval is not auto-recognized by INVIMA (Colombia), ANMAT (Argentina), ISP (Chile), or DIGEMID (Peru).
PCCPPredetermined Change Control Plan
An FDA mechanism for AI/ML-enabled Software as a Medical Device that allows specified, planned algorithm modifications to occur after authorization without a new submission. The sponsor pre-specifies the modifications, the methods used to implement them, and the assessment plan. PCCP is central to the FDA approach for adaptive, learning-based digital health products.
Pre-IND
A formal FDA meeting held before an IND submission to align on nonclinical and CMC requirements, proposed clinical design, and any program-specific issues. Type B meeting in FDA nomenclature; the agency provides written responses and a meeting if requested.
PROTACProteolysis-Targeting Chimera
A bifunctional small molecule that brings a target protein into proximity with an E3 ubiquitin ligase, marking the target for degradation by the proteasome. The mechanism allows degradation rather than inhibition and can address proteins long considered undruggable.
PIPPaediatric Investigation Plan
An EMA-mandated plan describing the studies and measures a sponsor will undertake to develop a product for use in the pediatric population. A PIP (or a waiver) is generally required to support a marketing authorization application in the EU.
PRIMEPRIority MEdicines
An EMA scheme providing enhanced support to developers of medicines targeting unmet medical needs. PRIME-designated programs receive earlier and more frequent scientific advice and are eligible for accelerated assessment.

Q

QbDQuality by Design
A systematic approach to pharmaceutical development that defines target product quality at the outset and uses science and risk management to design processes that consistently deliver it. ICH Q8, Q9, Q10, and Q11 codify the framework. QbD elements increasingly appear in regulatory expectations for CMC packages.

R

R&D Tax Incentive (Australia)
Australian Government program providing a 43.5% refundable tax offset for entities with aggregated turnover under AUD 20M; a 38.5% non-refundable tax offset applies above the AUD 20M threshold. A major Australian biotech advantage and a primary reason early biotechs run first-in-human trials in Australia under the TGA CTN/CTA notification scheme.
RMATRegenerative Medicine Advanced Therapy
An FDA designation for cell therapies, therapeutic tissue-engineering products, and certain gene therapies intended to treat serious conditions where preliminary clinical evidence indicates the product has the potential to address an unmet medical need. RMAT carries benefits similar to Breakthrough Therapy with regenerative-specific provisions.
RWD/RWEReal-World Data / Real-World Evidence
RWD is data relating to patient health status or care delivery from sources outside traditional randomized trials (claims, electronic health records, registries, wearables). RWE is the clinical evidence derived from analyzing RWD. FDA, EMA, and other regulators have published frameworks for RWE in regulatory decision-making.

S

Sakigake
Japan's PMDA designation system that gives priority review and consultation to innovative medical products targeting serious diseases with high medical need, where development in Japan occurs ahead of or concurrent with other countries. Functionally analogous in spirit to FDA Breakthrough Therapy.
SaMDSoftware as a Medical Device
Software intended for one or more medical purposes that performs those purposes without being part of a hardware medical device. SaMD includes diagnostic algorithms, decision-support tools, and digital therapeutics. It is regulated under CDRH using a risk-based framework aligned to the IMDRF SaMD categorization, distinct from the drug pathway.
SBIR/STTRSmall Business Innovation Research / Small Business Technology Transfer
Two US federal non-dilutive funding programs that support early-stage research at small businesses (SBIR) and at small businesses partnering with research institutions (STTR). NIH is the largest funder for biotech; awards are structured in phases tied to milestones.
SRASponsored Research Agreement
A contract between an industry sponsor (often a startup) and a university or research institution under which the sponsor funds research at the institution's lab against specific deliverables and IP terms. Distinct from an unrestricted gift or a grant; SRAs typically negotiate the F&A rate, the IP-ownership default for new inventions, publication rights, and the scope-of-work milestones. Common vehicle for academic founders to fund continued research at their former lab.
Subject invention
Bayh-Dole term (35 USC §201(e)) for an invention that is conceived or first actually reduced to practice in the performance of work under a federal funding agreement. Recognition of a subject invention triggers the institution's obligation to disclose to the funding agency through iEdison, elect or decline title within statutory windows, and file appropriate patent protection. Misclassification of a subject invention as a non-federally-funded invention is a recurring source of chain-of-title problems at Series A diligence.
Survive diligence (BridgeLine usage)
BridgeLine's tagline language, not standardized industry vocabulary. To survive diligence means the strategy and the supporting evidence package will withstand scrutiny by an investor, partner, or strategic acquirer without surfacing material gaps. Listed here for clarity because the phrase appears across BridgeLine's own pages; readers comparing advisory firms should not treat it as a defined industry term.

T

TPPTarget Product Profile
A structured document that captures the desired characteristics of a drug at launch - indication, patient population, efficacy bar, safety bar, dosing, label claims - and is used to align the development program around what the asset must demonstrate to be commercially viable.
Translational
The body of work that connects basic biological findings to human therapeutics: target validation, biomarker development, model selection, dose translation, and early clinical proof-of-mechanism. Translational quality is a primary determinant of whether a program clears proof-of-concept.
Type B/C meeting
FDA meeting types categorized by purpose. Type B includes Pre-IND, End-of-Phase 1, End-of-Phase 2, and Pre-NDA/Pre-BLA meetings. Type C covers other meetings about product development. Each type carries specific procedural and timing rules in FDA guidance.

V

Vector
In gene therapy and cell engineering, the delivery vehicle that carries genetic material into a target cell. Vectors include viral systems (AAV, lentivirus, adenovirus) and non-viral systems (LNPs, electroporation, transposons). Vector choice drives manufacturing, regulatory, and clinical trade-offs.

Disclaimer

Definitions are educational summaries, not regulatory, legal, scientific, or investment advice. Terms drawn from FDA, ICH, EMA, and other agency frameworks evolve over time; consult the primary issuing source before acting on a definition for a regulatory or commercial decision.

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