Running First-in-Human Gene Therapy Trials in Latin America: Regulatory Pathways and Country Options

A comprehensive regulatory guide for gene therapy and advanced biologic startups evaluating Latin America for first-in-human clinical trials — country options, pathway analysis, and practical considerations.

April 1, 2026

15

min read

By

Julio G. Martinez-Clark, CEO, bioaccess®

gene therapy

first-in-human

clinical trials

Latin America

Panama

El Salvador

Brazil

regulatory

advanced biologics

nonviral

dermatology

ATMP

**Key Takeaways**

• • Panama, El Salvador, and Brazil offer viable pathways for first-in-human gene therapy trials
• • Full GLP toxicology is not universally required — ethics committees evaluate the totality of preclinical evidence
• • Regulatory classification ambiguity (biologic vs. device vs. combination) can actually benefit FIH sponsors in EC-driven jurisdictions
• • Nonviral gene therapy approaches may face fewer regulatory hurdles than viral vector approaches
• • Latin American FIH data is accepted by FDA under 21 CFR 312.120 for IND submissions

**Who Is This For?**

This guide is for gene therapy CEOs, CSOs, VP Clinical/Regulatory Affairs, and founders developing novel gene therapies, cell therapies, ATMPs, or advanced biologics — particularly nonviral approaches for dermatology, oncology, rare diseases, or regenerative medicine — who need first-in-human clinical data but face prohibitive timelines and costs in the US/EU.

The Next Frontier: Gene Therapy FIH Trials in Latin America

Gene therapy and advanced biologics — ATMPs, cell therapies, nonviral gene therapies, mRNA therapeutics — represent the next frontier of medical innovation. For startups developing these therapies, the path to first-in-human testing is uniquely challenging.

Unlike conventional small-molecule drugs or even traditional biologics, gene therapy products present regulatory challenges that are fundamentally different: full GLP toxicology packages may not be required or even possible for novel modalities, the regulatory classification (drug vs. device vs. combination product) may be uncertain, and the preclinical-to-clinical translation pathway may not fit neatly into established regulatory frameworks.

These challenges are compounded by the reality that FDA and EMA gene therapy review processes can take 18-36 months from pre-IND meeting to first patient dosing — timelines that can exhaust startup runway and investor patience.

Latin America offers an alternative: ethics-committee-driven pathways that evaluate gene therapy investigations based on their risk-benefit narrative and scientific merit, rather than requiring definitive product classification or exhaustive preclinical packages designed for conventional therapeutics.

Section 1: Why Gene Therapy Startups Are Looking at Latin America

The convergence of several factors is driving gene therapy startups to evaluate Latin American FIH pathways:

• Speed to first patient: 3-6 months in LATAM vs. 18-36 months through FDA IND or EMA CTA for gene therapy products
• Ability to proceed with less extensive toxicology data than FDA/EMA would require for conventional IND filing
• Ethics-committee-driven pathways in select countries that evaluate risk-benefit narratives rather than applying rigid product classification checklists
• GCP-compliant clinical data accepted by FDA for IND submissions under 21 CFR 312.120
• Lower costs: $200K-$500K for a 5-15 patient FIH study vs. $1M-$3M+ in the US
• Regulatory flexibility for novel therapeutic modalities that don't fit established classification frameworks

**The Gene Therapy Regulatory Paradox**

Gene therapy startups face a paradox: FDA and EMA require extensive preclinical data before authorizing FIH studies, but the preclinical models available for many novel gene therapy modalities are inherently limited in their predictive value for human outcomes. Latin American EC-driven pathways acknowledge this reality — they evaluate whether the available preclinical evidence supports a controlled, closely-monitored FIH study in a small number of patients, rather than demanding preclinical data packages designed for conventional therapeutics.

Section 2: Country-by-Country Analysis for Gene Therapy FIH

Panama: Fast, EC-Driven, Direct FIH Experience

Panama offers the most streamlined pathway for gene therapy FIH studies among the recommended Latin American jurisdictions. The EC-driven pathway through the CNBI (Comité Nacional de Bioética de Investigación) enables 3-5 month timelines from submission to first patient.

Panama's advantages for gene therapy FIH:

• No separate national authority approval required — EC-driven pathway handles gene therapy investigations
• Regulatory framework evaluates risk-benefit narratives rather than requiring definitive product classification
• Direct FIH experience with novel technologies (bioaccess® has conducted brain-computer interface FIH in Panama)
• US-trained specialists available for gene therapy-specific clinical procedures
• Proximity to US (3-hour flight from Miami, same time zone) — critical for biological material logistics
• Bilingual medical community reduces communication barriers

El Salvador: Flexible, Cost-Effective

El Salvador provides a flexible EC-driven pathway with 3-5 month timelines and competitive operational costs. The country has demonstrated capability for novel device FIH studies, and its regulatory flexibility extends to advanced biologic and gene therapy investigations.

El Salvador's advantages for gene therapy startups include lower overall study costs, a streamlined regulatory environment, and ethics committees that evaluate scientific merit and patient protection rather than applying product-specific classification requirements.

Brazil: Larger Scale, More Complex

Brazil's CEP-only pathway under Law 14.874/24 and RDC 837/2023 enables gene therapy FIH studies without ANVISA or CONEP approval for studies not intended for local market clearance. The 30-business-day ethics review cap provides timeline predictability.

Brazil offers advantages for gene therapy programs that may need larger patient populations or specific disease demographics. However, the pathway is more complex than Panama or El Salvador, and sponsors should budget 4-6 months for the full start-up process.

Colombia: Slower for Novel Biologics

**Colombia: Not Recommended for Gene Therapy FIH**

Colombia's INVIMA pathway routes novel biological products through a national authority review that can take 8-14 months and involves unpredictable technical questions — particularly for products that don't fit established classification frameworks. For gene therapy FIH studies where speed and regulatory flexibility are priorities, Panama, El Salvador, or Brazil are preferable first choices.

Section 3: Key Regulatory Considerations for Gene Therapy FIH

Classification Challenges: Biologic vs. Device vs. Combination

Gene therapy products present unique classification challenges in any regulatory jurisdiction. A nonviral skin gene therapy delivered via topical application might be classified as a biological product, a drug, or — if delivered via a device applicator — a combination product. In the US, this classification determination (the "Request for Designation" process with FDA's Office of Combination Products) can itself take months.

In EC-driven Latin American jurisdictions, this classification ambiguity is less of an obstacle. Ethics committees evaluate the investigation's risk-benefit profile, not the product's regulatory classification. This means a gene therapy startup can proceed to FIH testing without resolving the classification question that might otherwise delay the program by 6-12 months in the US or EU.

Preclinical Data Requirements

For gene therapy FIH studies in the recommended Latin American jurisdictions, ethics committees typically evaluate:

• Mechanism of action characterization: Clear scientific rationale for the therapeutic approach
• In vitro efficacy and safety data: Cell-based studies demonstrating the intended biological effect and absence of cytotoxicity
• Animal model data: Relevant animal studies (which may be non-GLP) demonstrating safety and preliminary efficacy signals
• Product characterization: Identity, purity, potency testing of the gene therapy product
• Risk analysis: Comprehensive risk-benefit assessment addressing known and theoretical risks (e.g., off-target effects, immunogenicity, insertional mutagenesis for viral vectors)
• Manufacturing and quality: Evidence of a controlled manufacturing process (not necessarily GMP-certified for FIH) with batch-level quality testing

Section 4: Practical Considerations

Gene therapy FIH studies in Latin America require attention to several practical challenges:

Cold Chain and Supply Chain: Biological materials (viral vectors, plasmid DNA, mRNA, lipid nanoparticles) often require controlled temperature storage and transport. Panama and Brazil have established cold chain infrastructure through their pharmaceutical distribution networks, but sponsors should verify specific temperature requirements (e.g., -80°C ultra-cold storage) are available at the proposed clinical site.

Specialized Site Infrastructure: Depending on the indication, gene therapy FIH studies may require specialized clinical facilities — dermatology clinics with controlled application environments, oncology centers with infusion capabilities, or surgical suites for direct tissue administration. bioaccess®'s site search process identifies facilities with the specific capabilities required for each gene therapy protocol.

Clinical Trial Insurance: Clinical trial insurance for gene therapy studies may carry higher premiums than conventional device studies due to the theoretical risks associated with genetic modification. Premiums typically range from $10,000-$25,000 depending on the therapy type and risk profile.

Investigator Training: Gene therapy-specific training for principal investigators and study staff is essential, covering: product handling and administration procedures, gene therapy-specific adverse event monitoring (including delayed-onset events), informed consent requirements for genetic modification studies, and long-term follow-up protocols.

Section 5: Case Perspective — Nonviral Skin Gene Therapy

**Case Perspective: Nonviral Gene Therapy FIH in LATAM**

A US-based nonviral gene therapy company developing a topical skin gene therapy chose to conduct its Phase 0/FIH study in Latin America because regulators in EC-driven jurisdictions focus on safety and the innovation's risk-benefit narrative rather than requiring extensive preclinical data packages designed for conventional drug development. Countries like Panama and El Salvador were prioritized for their fast EC-driven pathways, regulatory flexibility for novel therapeutic modalities, and availability of qualified dermatology investigators.

The company's decision reflected a pragmatic assessment: generating initial human safety data through a controlled, GCP-compliant LATAM study would strengthen their subsequent FDA IND submission — providing real-world clinical evidence that supplements their preclinical package, rather than waiting 18-24 months for FDA to authorize FIH testing based on preclinical data alone.

Frequently Asked Questions

JM

Julio G. Martinez-Clark

CEO & Founder, bioaccess® · 15+ years leading clinical trials across Latin America · Experience with novel therapeutic modalities including neurotechnology, advanced biologics, and gene therapy FIH program planning across Panama, El Salvador, and Brazil.

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Latin America offers viable, operationally proven pathways for first-in-human gene therapy trials that accommodate the unique challenges of early-stage therapeutic development — from regulatory classification ambiguity to limited preclinical data availability.

bioaccess® has direct experience navigating novel therapeutic modality FIH studies in the region and can provide country-specific regulatory assessments, site identification, and operational planning for your gene therapy program.

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Related resources:

• Panama FIH Clinical Trials — /blog/panama-first-in-human-clinical-trials-medical-devices
• El Salvador FIH Trials — /blog/el-salvador-first-in-human-medical-device-clinical-trials
• Early Feasibility Device Trials in LATAM — /blog/early-feasibility-device-trials-latin-america-glp-gmp-sterility-requirements
• Services Overview — /services
• First-in-Human CRO — /first-in-human-cro