ACPE UAN: 0475-0000-22-009-L04-P. A knowledge-based activity.
ACPE logo The European Association of Hospital Pharmacists (EAHP) is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education.
More information: https://www.eahp.eu/congresses/acpe
Self-assessment questions:

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* 1. Are gene therapy products only used for the treatment of inherited diseases?

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* 2. Do pharmacists need specific skills, training and extra resources to perform ATMP activity in a hospital setting?

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* 3. mRNA vaccines against infectious diseases can be classified as advanced therapy medicinal products

PC1 - Opportunities and threats for hospital pharmacy production in gene and cell therapy scenarios

Linked to EAHP Statements
Section 3 - Production and Compounding: Statement 3.1, 3.2, 3.4, 3.5, 3.6
Section 6 - Education and Research: Statement 6.2, 6.4, 6.5

ACPE UAN: 0475-0000-22-009-L04-P. A knowledge-based activity.

Abstract
New technologies are about to modify the way hospital pharmacists work. To be ready, further skills have to be acquired in terms of getting familiar and expert for upcoming opportunities and threats. These new technologies comprise pharmacogenomics, gene editing, CRISPR-Cas, transfection of modified genetic materials into cells or into nanoparticles. In this way, it will become a reality to really personalize treatments. As such medicines do not reach blockbuster figures, it might be important for hospital pharmacists to participate in these treatments on a middle-scale production.

Single nucleotide polymorphisms (SNPs) and their inherited interrelation with non-communicable diseases have been detected in the past years by genome-wide association studies (GWAS). The "common variant - common disease" hypothesis led to the identification of the mutated loci on the chromosomes and thus to treatment options for Inherited diseases with high penetrance such as Huntington, Spinal Atrophy, or Cystic Fibrosis. A further step from actual GWAS associations of mutated genes to diseases will lead to exam sequencing and in a short future to whole genome sequencing. Cell therapies become more and more available due to technologies as CRISPR/CAS but remain costly. A response to medicines depends on normal functions of enzymes on the pharmacokinetic pathway and on normal structures of the receptors on a pharmacodynamic level.

Whereas diagnostics (sequencing high numbers of genetic specimen and biotechniques) will be a business for specialized molecular biology and chemistry laboratories, medical professions will be challenged by the interpretation of data from genetic profiling. Bioinformatics will have to prepare a set of interpretable data for physicians and pharmacists. Basing on these data, responders to a novel treatment can be distinguished from non-responders. Repaired genes after editing and application of molecular genetical techniques, such as CRISPR-Cas, will have to be packed in a suitable carrier box, either a cell or a nanoparticle, which is to fit perfectly to its target in the body. These new medicines will certainly require new types of handling drugs and prepare ready-to-use gene and cell medicines. It will obviously be more requiring to equipment, investment, training of hospital pharmacy staff. Cooperation with other professionals is likely in a way we know today from transplant teams.

The main focus of the seminar will set on personalised and precision medicine and the inherent development of novel biological compounds. Differences between CAR T-cell treatments and nanoparticle-based ways of bringing the repaired genes to their targets will be presented as well.

Learning objectives
After the session, participants should be able to:
  • Identify the limits of hospital pharmacies if the technology upgrade is not done;
  • Assess typical analytical challenges of the omics and cell biology techniques and how hospital pharmacies can cope with the new requirements;
  • Recognise the novelty and impact of gene editing and CAR T - cell treatments on the way of quality assurance in hospital pharmacies;
  • Understand the principles of gene editing and therapeutic options to the therapy of diseases.
Educational need addressed
Personalised medicine is currently doing the next step from tailored small molecules treatments and dose adaption to treatments arising from

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