Acelerando la traslación clínica de terapias avanzadas.

Somos una consultora científica y regulatoria especializada en terapias avanzadas, bioterapias y medicina regenerativa.

Entorno regulatorio competitivo - Parte 1

Regulatory environment aligned with regional reference standards

Colombia’s national regulatory authority, the Instituto Nacional de Vigilancia de Medicamentos
y Alimentos (INVIMA), has been recognized by the Pan American Health Organization (PAHO)
as a National Regulatory Authority of Regional Reference (NRAr) – Level IV, the highest level of
regulatory maturity within the PAHO/WHO framework for the Americas. This designation
indicates that the agency has demonstrated strong institutional capacity and consistent
performance across the core regulatory functions required to ensure the quality, safety, and
efficacy of health products.
To achieve NRAr Level IV status, regulatory authorities must demonstrate strong performance
across several core regulatory functions, including:

- Marketing authorization and scientific evaluation: rigorous assessment of quality, safety, and efficacy for medicines and biological products.
- Authorization and oversight of clinical trials: evaluation of clinical research protocols and compliance with Good Clinical Practice standards.
- Inspection of Good Manufacturing Practices (GMP): oversight of pharmaceutical manufacturing facilities and supply chains.
- Post-market surveillance and pharmacovigilance: systems for monitoring safety and quality once products are in use.
- Regulatory transparency and governance: documented procedures, technical independence, and evidence-based decision-making.
- Laboratory and quality control capacity: technical capabilities to verify product quality and support regulatory decisions.

Only a limited number of regulatory agencies in Latin America have achieved this level of regulatory maturity, positioning Colombia among the region’s most robust regulatory environments for pharmaceutical development and clinical research. Sources: PAHO/WHO – National Regulatory Authorities of Regional Reference framework.

Entorno regulatorio competitivo - Parte 2

Clinical research in Colombia is primarily governed by Resolution 8430 of 1993, which establishes the ethical and scientific standards for biomedical research involving human participants. The regulation mandates that all research must respect participant dignity and protect their rights, safety, and well-being, and requires prior review and approval by institutional ethics committees. Additional regulatory oversight is provided by INVIMA, which evaluates and authorizes clinical trials involving investigational medicinal products and ensures compliance with international standards such as Good Clinical Practice. The regulatory framework for biological products was further strengthened through Decree 1782 of 2014, which introduced specific pathways for biologics and biosimilars. While Colombia continues to strengthen its regulatory capacity for emerging technologies such as advanced cell and gene therapies, academic institutions and regulatory stakeholders have proactively formed collaborative initiatives to establish interim technical and ethical standards. These efforts help address existing regulatory gaps while supporting a safe and efficient pathway for the clinical development of advanced therapy medicinal products (ATMPs). By leveraging scientific and academic expertise, real-world clinical evidence, and GMP-compliant infrastructure, these initiatives contribute to the creation of pragmatic frameworks that safeguard patient safety while enabling sustained innovation in regenerative and advanced therapies.

Experience in advanced therapies research

Over the past two decades, Colombia has established a track record in executing international
and national clinical trials, with a notable shift in recent years towards complex interventions,
particularly immunotherapies and stem cell therapies.
Colombia’s clinical readiness for ATMPs is demonstrated by significant recent milestones.

Terapia Génica:

Enfermedades Huerfanas (2023): The country successfully authorized its first in vivo gene therapy clinical trial using viral vectors for Spinal Muscular Atrophy (SMA), proving the capacity of local clinical centers,ethics committees, and regulatory bodies to manage protocols requiring high biosafety levels.
Enfermedades Infecciosas (2019 – 2021): Los centros colombianos lideran estudios sofisticados de fase I, incluyendo ensayos de vacunas terapéuticas basadas en vectores virales (NCT05208125) y terapias celulares/génicas autólogas para pacientes con VIH (NCT04561258),

Terapia Celular y Medicina Regenerativa:

Liderazgo en terapias CAR-T. Se consolidó la Alianza Colaborativa para la Implementación de Terapias Avanzadas en Colombia (ACITAC), una coalicion publico-privada liderada por el IDCBIS y el Instituto Nacional de Cancerología (INC). Actualmente ejecutan el protocolo ACITAC-001, el primer estudio híbridoen el país de implementación de terapia celular CAR-T anti-CD19. Este estudio de transferencia tecnológica evalúa el producto lentiviral ARI-0001 bajo un modelo de manufactura point-of-care, buscando ofrecer estas terapias a pacientes con leucemias y linfomas refractarios. A este esfuerzo se suma el grupo de investigación LiliCAR-T que lideran el camino en Colombia para la manufactura local (point-of-care) y la futura aplicación clínica de terapias de células T con receptores quiméricos de antígenos (CAR-T) para cánceres hematológicos.
Osteoartritis (2021-2024): El INVIMA aprobó ensayos clínicos fase I/II y aleatorizados utilizando células madre mesenquimales. Destacan el estudio con Cellistem-OA (aprobado en 2021) y el reciente ensayo del IDCBIS y la Universidad Nacional de Colombia (UNAL) con células derivadas de cordón umbilical para la recuperación del cartílago articular (aprobado en julio de 2024).
Respuesta al COVID19 (2020). Aprobación del ensayo clínico (BIOXSOMCOV001) patrocinado por la Clínica Somer, para evaluar la eficacia y seguridad de la aplicación venosa de células madre mesenquimales derivadas de la gelatina de Wharton en pacientes hospitalizados con diagnóstico grave de COVID-19.

Infraestructura de Investigación Clínica: Centros GMP y Hospitales de Alta Complejidad

Infraestructura GMP de vanguardia (2021). Inauguración de la “Sala Blanca” del IDCBIS en Bogotá, la primera infraestructura pública en latinoamérica con estándares GMP/BPM, diseñada específicamente para la manufactura de terapias celulares (como CAR-T y MSC) y de ingeniería de tejidos. Esta infraestructura se une a la capacidad ya disponible de salas blancas con certificación GMP del biobanco de
la Universidad de Antioquia (UdeA) y la Fundación Oftalmológica de Santander (FOSCAL).
Mas de 160 instituciones hospitalarias cuentan actualmente con el certificado vigente en Buenas Prácticas Clínicas (BPC) otorgado por el INVIMA. Más de 70 Comités de Ética en Investigación consolidados e independientes, garantizando la rigurosidad ética y científica.
Colombia alberga 5 de los hospitales clasificados entre los mejores de toda América Latina (según la revista América Economía), los cuales participan activamente en investigación clínica, incluyendo Fundación Valle del Lili, La Cardio, el Hospital Pablo Tobón Uribe, entre otros.

The presence of both INVIMA-certified research centers and institutional ethics committees enables parallel regulatory and ethical review processes, contributing to efficient clinical trial activation timelines in comparison with other countries in the region.

Clinical Research Infrastructure: GMP Facilities and High-Complexity Hospitals

The inauguration of the IDCBIS “Clean Room” in Bogota marked the establishment of the first public GMP-compliant facility in Latin America specifically designed for the manufacturing of cellular therapies (such as CAR-T and mesenchymal stromal cells, MSC) and tissue engineering products. This infrastructure complements the existing GMP-certified clean room capabilities available at the Biobank of the Universityof Antioquia (UdeA) and the Fundación Oftalmológica de Santander (FOSCAL).
More than 160 hospital institutions currently hold valid Good Clinical Practice (GCP) certification granted by INVIMA, demonstrating the country’s capacity to conduct regulated clinical research. In addition, over 70 independent and well-established Research Ethics Committees ensure rigorous ethical and scientific oversight of biomedical studies.
Colombia is also home to five hospitals ranked among the best in Latin America, according to América Economía magazine. Several of these institutions actively participate in clinical research, including Fundacion Valle del Lili, Fundacion Cardioinfantil – LaCardio, and Hospital Pablo Tobon Uribe, among others.

Población diversa y acceso a pacientes complejo

Colombia ofrece acceso a una población diversa y a cohortes clínicas bien caracterizadas, lo que facilita el desarrollo de estudios en terapias avanzadas (ATMP) y medicina regenerativa. La combinación de registros clínicos especializados, centros de referencia nacional y fundaciones de pacientes permite identificar poblaciones con enfermedades complejas o de baja prevalencia. Factores clave del ecosistema clínico:

Población de más de 50 millones de habitantes con alta diversidad genética y étnica, resultado de la mezcla de poblaciones indígenas, europeas y afrodescendientes, lo que favorece la representatividad en investigación biomédica.

Registros clínicos y redes especializadas de pacientes en áreas de alto interés para terapias avanzadas, incluyendo:
- Enfermedades hematológicas y programas de trasplante de progenitores hematopoyéticos
- Enfermedades raras y huérfanas registradas en el sistema nacional de vigilancia epidemiológica
- Cáncer y hemato-oncología, con múltiples centros de referencia oncológica
- Enfermedades autoinmunes e inflamatorias crónicas
- Patologías neurodegenerativas y neuromusculares

Fundaciones de pacientes y asociaciones científicas activas en áreas como enfermedades raras, cáncer, enfermedades autoinmunes y trastornos genéticos, que facilitan la identificación de cohortes y el reclutamiento para investigación clínica.

Centros hospitalarios de alta complejidad con programas de referencia nacional en oncología, trasplantes, neurología y enfermedades raras, lo que permite concentrar pacientes con condiciones complejas susceptibles de terapias innovadoras.

En conjunto, estos factores posicionan a Colombia como un entorno favorable para estudios clínicos en terapias celulares, terapia génica y medicina regenerativa, permitiendo acceder a poblaciones con necesidades médicas no cubiertas y alto valor para la investigación traslacional.

Clinical Areas of Opportunity for ATMP Development in Colombia

Colombia presents several therapeutic areas where advanced therapy medicinal products (ATMP)-including cell therapies, gene therapies, and regenerative medicine approaches-may address significant unmet medical needs. The presence of specialized clinical centers, national disease registries, and patient foundations facilitates the identification and recruitment of eligible patient populations. Key therapeutic areas include:

Hemato-oncology
Colombia hosts multiple hematopoietic stem cell transplant programs and specialized oncology centers, creating a favorable environment for the development of CAR-T and other cellular immunotherapies targeting leukemias, lymphomas, and multiple myeloma.

Rare diseases and genetic disorders
The national rare disease registry (SISPRO) has identified thousands of patients across more than 2,000 rare conditions, providing an important foundation for the development of gene and cell therapies.

Autoimmune and inflammatory diseases
Conditions such as systemic lupus erythematosus, rheumatoid arthritis, and Crohn’s disease are managed through specialized clinical networks, making them potential targets for mesenchymal stromal cell (MSC) therapies and other immunomodulatory approaches.

Neurological and neurodegenerative disorder
Referral centers treat patients with conditions such as multiple sclerosis, Parkinson’s disease, and spinal cord injury, areas where regenerative medicine strategies are actively being explored.

Orthopedic and musculoskeletal conditions
High prevalence of osteoarthritis, cartilage injury, and musculoskeletal degeneration supports clinical research in cell-based regenerative therapies and tissue engineering approaches.

Ophthalmologic diseases
Colombia hosts specialized ophthalmology centers involved in research on retinal degenerative diseases
and corneal regeneration, areas where cell-based therapies are under active development.

Together, these clinical areas highlight the potential for Colombia to support translational and clinical development programs in advanced therapies, combining access to diverse patient populations with experienced clinical investigators and research institutions.