There are several distinctive features that must be considered in the ATMP development:

1. ATMPs are mainly for the unmet medical needs = severe, rare, or chronic diseases with no adequate conventional treatments.
2. Risk-based approach = Dominant strategy of the ATMPs development.
3. ATMP = strong scientific data + consistent/robust/aseptic manufacturing process.
4. Early start with the potency assays development (mode of action/the intended effect).
5. Quality = starting materials, raw materials, supply chain, etc.
6. Preclinical safety assessment (immunogenicity, biocompatibility, viral or bacterial contamination, ectopic tissue formation, tumorigenicity, mutagenesis, tropism and tissue distribution, stability, biodistribution, shedding, etc.).
7. Difficulties with the reproducibility and high variability (donors, patients, batches, etc.).
8. Bidirectional Traceability system.
9. Designing In Process Control Tests (comparability tests) and Product Release Tests (identity, concentration, viability, recovery, impurities, etc.), including method/tool/equipment validations behind the tests.
10. Formulation, packaging, storage/cryopreservation, and transportation conditions do have a strong impact on the intended effect.

Have you experienced additional difficulties in your ATMP development? We would love to learn more about your development program and explore how we can impact your work.

Let us facilitate your ATMP advancement in the minimum of time and with the most efficient use of resources, while ensuring the high quality. It would be great to establish a cooperation between Venn Life Sciences and your company.

Discover how we can support and accelerate the ATMP development:

• Technical, scientific, and regulatory expertise.
• Provide CMC input into development strategies for your ATMP.
• Assessment of the regulatory requirements for your ATMP development.
• Clinical trial execution under GCP with specific protocol design and considerations for your ATMP.
• Advise on the specific ATMP logistic from the production site to the clinical site.
• QA and audits.
• Biostatistics and Medical Writing.
• Guide, coach, and train professionals in your company.

Blog written by Ion Tcacencu

Ion Tcacencu (MD, PhD) is an ATMP expert with over 17 years of experience as scientist within regenerative medicine and immunology, being responsible for projects developing new ATMPs.