Biotech industry links

Foundational guidance on first-in-human dose selection using NOAEL, human equivalent dose (HED) conversion, and safety factors.

FDA guidance on the content, format, and structure of original IND applications for Phase 1 studies of drugs and biologics.

Guidance on requesting, preparing for, and conducting Pre-IND, End-of-Phase 2, and Pre-NDA/BLA meetings.

Requirements for reporting safety information from clinical and nonclinical studies during the IND phase.

FDA recommendations on preclinical program design, study endpoints, and species selection for cell and gene therapies.

Guidance addressing nonclinical and clinical considerations specific to genome editing therapeutics.

FDA recommendations on long-term follow-up observation for gene therapy and gene-modified cell therapy products.

CMC guidance specific to gene therapy vectors, including characterization, manufacturing, and testing.

Overview of FDA expedited programs that can accelerate development timelines for serious conditions with unmet medical need.

Guidance on orphan drug designation, rare pediatric disease designation, and associated incentives (tax credits, market exclusivity).

Complete listing of all CBER guidance documents specific to cellular and gene therapy products.

Early-stage engagement with CBER before formal pre-IND meetings for cell and gene therapy product development.

Searchable database of all FDA-licensed biological products, including biosimilar and interchangeability evaluations.

FDA resources, webinars, and direct regulatory guidance assistance specifically for small pharmaceutical and biotech companies.

Free on-demand library of recorded presentations, webinars, online courses, and podcasts on drug development and regulation.

First dedicated FDA guidance (2024) on nonclinical safety evaluation for ASOs, siRNAs, and other oligonucleotide therapeutics, covering species selection, off-target effects, and class-specific toxicities.

Streamlined nonclinical requirements for individualized (n-of-1) antisense oligonucleotide drugs from well-characterized chemical classes for ultra-rare diseases.

Comprehensive rare disease guidance (2023). FDA will exercise broadest flexibility in applying nonclinical requirements when limited patient/animal models exist.

Guidance on designing natural history studies that inform preclinical target validation, biomarker selection, clinical endpoint design, and external control arms.

How FDA determines whether two gene therapy products are the 'same' for orphan drug exclusivity. Critical for competitive landscape and orphan strategy.

Guidance on assessing immunotoxicity risk including cytokine release, immunosuppression, hypersensitivity, and autoimmunity in nonclinical studies.

Nonclinical approaches for fixed-dose combinations, co-packaged products, and adjunctive therapies including study design and toxicity attribution.

New FDA program (2024) allowing platform technology designations that can streamline development for subsequent products using the same platform.

Preclinical and clinical considerations for gene therapy targeting retinal disorders, including animal model selection and bilateral dosing strategy.

Preclinical and clinical guidance for gene therapy products for hemophilia A and B, including nonclinical dose selection for AAV-based programs.

Nonclinical program design for gene therapy in rare diseases, addressing limited study populations and bioactivity endpoint challenges.

Nonclinical considerations for CNS-directed gene therapy including biodistribution in brain/CSF, animal model selection, and long-term follow-up.

RCR/RCL testing requirements for gammaretroviral and lentiviral vector products. Critical for ex vivo CAR-T and gene-modified cell therapies.

Recommendations on conducting shedding studies during preclinical and clinical development to assess transmission potential.

Phase 1 and early Phase 2 trial design for cell and gene therapy, including dose selection informed by nonclinical data.

Guidance for studying multiple vector constructs or product versions in a single early-phase trial to identify optimal candidates.

Recommendations for developing potency assays measuring biological activity of CGT products, including incremental assay development.

Manufacturing, nonclinical, and clinical considerations for CAR-T products including product characterization and nonclinical assessment of engineered T cells.

Foundational guidance covering the regulatory framework, preclinical testing, and manufacturing for somatic cell therapy and gene therapy products.

Nonclinical and clinical guidance for bispecific antibodies, including species selection, tissue cross-reactivity, and PK/PD of dual-binding components.

Risk-based approach to evaluating immunogenicity of therapeutic proteins (mAbs, fusion proteins, enzymes), including anti-drug antibody assay strategy.

PK/PD of ADCs and their constituents (antibody, linker, payload), immunogenicity assessment, and first-in-human dose selection approaches.

Nonclinical program design for ERT products, including species selection, FIH dose selection, and biodistribution to target and non-target tissues.

Framework for biosimilar development including analytical, nonclinical, and clinical data requirements, and when nonclinical studies may be waived.

Guidance on DDI evaluation for therapeutic proteins (mAbs, cytokines, enzymes), including immune-mediated DDI mechanisms and CYP interactions.

PK/PD, immunogenicity, and DDI evaluation for ASOs, siRNAs, and other oligonucleotides. Addresses unique PK characteristics of the oligonucleotide class.

Nonclinical requirements for nanomaterial-containing products including LNP-formulated mRNA and siRNA therapeutics. Covers quality, PK/tox, and risk-based approaches.

Streamlined nonclinical requirements for severe hematologic disorders: 1-month tox for FIH, 3-month for Phase 3. Relevant to gene therapies for sickle cell and hemophilia.

Reproductive toxicity evaluation for oncology products including biologics. Covers when EFD studies are needed and weight-of-evidence approaches.

FDA guidance on juvenile animal studies for pediatric drug development, covering organ systems at highest risk and timing of studies.

Searchable database of all current and draft FDA guidance documents across CDER, CBER, and other centers.