DEVELOPMENT OF ADVANCED THERAPY MEDICICAL PRODUCTS (ATMPs): EARLY CLINICAL TRIALS

Biomanufacturing

Gene therapy
Cell therapy
CAR-T cells

Clinical research (TRL 6-8)

The Clinical Investigation Center for Biotherapies provides additional expertise in the development of early-phase trials for advanced therapy medicinal products (ATMPs) aimed at treating malignant hematologic disorders.

Located across two sites (Institut Paoli-Calmettes & Hôpital de la Conception), the Clinical Investigation Center for Biotherapies provides clinical, technological, and logistical support for the manufacturing and clinical validation of cell and gene therapy medicinal products. The unit based at the Institut Paoli-Calmettes site (Cell Therapy Center) is primarily involved in the development of cellular immunotherapies, with applications in leukemia treatment.

Description

Scope

A clinical research infrastructure for early-phase trials supporting the development of cell and gene therapy medicinal products:

Collection of patient cells
Coordination of logistical flows for biological materials
Decentralized CAR-T cell manufacturing (i.e. near patients)

Project management

Steps:

Analysis of the R&D strategy
Design of the development plan for biomanufacturing and associated quality controls
Drafting of contracts and quality agreements
Preparation of regulatory submissions
Project implementation and technology transfer
Initiation of the clinical trial and batch manufacturing

Organization

Dr. Clémence Demerle, Biologist, responsible for quality control

Research infrastructure

Cytapheresis Unit
Multidisciplinary Cell Therapy Unit
CAR-T Cell Manufacturing Unit
ATMP Manufacturing: two manufacturing laboratories (Grade A/B)
Quality Control Laboratory: located adjacent to the manufacturing labs, enabling rapid sample transfer and real-time process analysis. The platform is equipped with multi-parameter flow cytometers and a dedicated culture room for phenotypic and functional qualification of products, during biomanufacturing.
Quality Management System: GMP, European Pharmacopoeia, ISO 15189

Quality assurance

JACIE Accreditation
ANSM Authorization: FR01302C-21-01
ANSM Authorization for ATMP-PP Manufacturing: ETI/19/O/001
COFRAC Accreditation (ISO 15189)
Approval for GMO Facility Operation: L2-0036

Specifications

The platform

A Cell Therapy Center dedicated to the manufacturing of advanced therapy medicinal products (ATMPs):

Pre-industrial development
Manufacturing of clinical batches (Phases I-II)
Regulatory support
Implementation of clinical trials

Supported projects

Investigational cell and gene therapy medicinal products for early clinical development (Phases I-II)
Industry-academic collaborations
Participation in the ATMP manufacturing value chain (e.g., cell sorting)

Examples of partnerships

Safety and efficacy of SMART101 in pediatric and adult Patients with hematological malignancies after T cell depleted allo-HSCT – This project aims to evaluate the safety and efficacy of injecting human T lymphoid progenitor cells to enhance post-allograft immune reconstitution. Our center performs the cell sorting required for the preparation of this ATMP, using a portion of the graft collected from the donor.

Partners: Smart Immune, MEARY