The Biotech Startup Resource Hub

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.

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