Advanced therapy medicinal products: Gene therapy

2015 ◽  
Vol 17 (1,2) ◽  
pp. 253-264
Author(s):  
Antonio Ayala ◽  
Mario Muñoz ◽  
Sandro Argüelles ◽  
Francisco Zurita ◽  
Mercedes Cano
Pharmaceutics ◽  
2021 ◽  
Vol 13 (8) ◽  
pp. 1140
Author(s):  
Yolanda Diebold ◽  
Laura García-Posadas

The conjunctiva is a complex ocular tissue that provides mechanical, sensory, and immune protection for the ocular surface. It is affected by many diseases through different pathological mechanisms. If a disease is not treated and conjunctival function is not fully restored, the whole ocular surface and, therefore, sight is at risk. Different therapeutic approaches have been proposed, but there are still unsolved conjunctival alterations that require more sophisticated therapeutic options. Advanced therapy medicinal products (ATMPs) comprise a wide range of products that includes cell therapy, tissue engineering, and gene therapy. To the best of our knowledge, there is no commercialized ATMP specifically for conjunctival treatment yet. However, the conjunctiva can be a potential target for ATMPs for different reasons. In this review, we provide an overview of the advances in experimental phases of potential ATMPs that primarily target the conjunctiva. Important advances have been achieved through the techniques of cell therapy and tissue engineering, whereas the use of gene therapy in the conjunctiva is still marginal. Undoubtedly, future research in this field will lead to achieving commercially available ATMPs for the conjunctiva, which may provide better treatments for patients.


2020 ◽  
Vol 21 (14) ◽  
pp. 4982 ◽  
Author(s):  
Pietro Gentile ◽  
Aris Sterodimas ◽  
Jacopo Pizzicannella ◽  
Laura Dionisi ◽  
Domenico De Fazio ◽  
...  

Stromal vascular fraction (SVF) containing adipose stem cells (ASCs) has been used for many years in regenerative plastic surgery for autologous applications, without any focus on their potential allogenic role. Allogenic SVF transplants could be based on the possibility to use decellularized extracellular matrix (ECM) as a scaffold from a donor then re-cellularized by ASCs of the recipient, in order to develop the advanced therapy medicinal products (ATMP) in fully personalized clinical approaches. A systematic review of this field has been realized in accordance with the Preferred Reporting for Items for Systematic Reviews and Meta-Analyses-Protocols (PRISMA-P) guidelines. Multistep research of the PubMed, Embase, MEDLINE, Pre-MEDLINE, PsycINFO, CINAHL, Clinicaltrials.gov, Scopus database, and Cochrane databases has been conducted to identify articles and investigations on human allogenic ASCs transplant for clinical use. Of the 341 articles identified, 313 were initially assessed for eligibility on the basis of the abstract. Of these, only 29 met all the predetermined criteria for inclusion according to the PICOS (patients, intervention, comparator, outcomes, and study design) approach, and 19 have been included in quantitative synthesis (meta-analysis). Ninety-one percent of the studies previously screened (284 papers) were focused on the in vitro results and pre-clinical experiments. The allogenic use regarded the treatment of perianal fistulas, diabetic foot ulcers, knee osteoarthritis, acute respiratory distress syndrome, refractory rheumatoid arthritis, pediatrics disease, fecal incontinence, ischemic heart disease, autoimmune encephalomyelitis, lateral epicondylitis, and soft tissue defects. The information analyzed suggested the safety and efficacy of allogenic ASCs and ECM transplants without major side effects.


2020 ◽  
pp. 1-7

Advanced Therapy Medicinal Products (ATMPs) embody innovative therapies that have created great hope for patients suffering from previously untreatable diseases. Unfortunately, the pharaonic cost to produce and authorise ATMPs is a challenge for both patients and public health care systems, ultimately reducing patients’ access to treatment. Over the last 11 years, only 15 ATMP marketing authorisation applications received a positive draft opinion from the European Medicines Agency’s (EMA’s) Committee for Advanced Therapies (CAT). Moreover, due to poor return on investment, several ATMPs have already been removed from the market. In addition to the centralised procedure to obtain a marketing authorisation, the legislator foresees an alternative route for authorising ATMPs, the so-called “ATMP Hospital Exemption”. However, such ATMPs must be produced on a limited scale, on a non-routine basis. As a result, valuable ATMP therapies that have been used for years in hospitals may disappear. To avoid this, we propose, in this paper, an additional possibility to regularise ATMPs: the “Magistral Preparation of ATMPs”. It is a feasible pathway, which was already proposed for bacteriophage therapy, and which is particularly suitable for personalised therapies and considerably decreases the cost of the final products. We also discuss the practical impact of the ATMP regulation for (for-profit) industries and for (non-profit) hospitals. Two practical examples, the cultured human chondrocytes and the cultured human keratinocytes, are discussed.


2012 ◽  
Vol 3 (3) ◽  
pp. 421-424
Author(s):  
Audrey Chenesseau ◽  
Anna Pavlou

The present Flash News Report sets outs recent EU regulatory law developments (as of 25 June 2012) which pharmaceutical companies should be aware of in the areas of Pharmacovigilance, Information on Medicines, Pricing and Reimbursement, EU Data Protection and Advanced Therapy Medicinal Products.


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