In this section, is reported the full ATMP news database, sorted by area of interest

Two $2m treatments launch this year but a reluctance to fork out could dim investor enthusiasm. The drive to cure deadly diseases is about to enter a new and demanding phase as the pharmaceutical industry prepares for the first time to test the appetite for hyper-expensive gene therapies in Europe. The question, however, is whether European health systems, particularly those with a well-established process for determining the cost-effectiveness of drugs, will be able to adapt their models to look beyond the immediate upfront cost and take into account longer-term savings

Date: Jan 15, 2020


10 centers in Italy are ready for the treatment of cancer with the first CAR-T therapy approved in august by AIFA. It’s estimated that the number of treatable patients for each center will be 1-2 per month, so as to safeguard their safety and to ensure a uniform treatment coverage on the territory; 7 Regions have decided to date which centers will be dedicated to CAR-T therapy: Liguria, Lombardy, Lazio, Tuscany, Abruzzo, Umbria and Emilia-Romagna

Date: Sep 3, 2019

Riccardo Palmisano, president of Assobiotec-Federchimica, summaries the main points of the advanced therapies’ postion paper, designed to highlight the priority measures to keep up the italian lead postion, to sustain the involved companies and to ensure appropriate and prompt responses to therapies from many patients

Date: Aug 29, 2019

After the AIFA green light for the reimbursement of CAR-T therapy, a Tuscany council decision showing the facilities authorised for the administration, that is four University Hospital: Careggi and Meyer in Florence and the AOU of Pisa and Siena; the authorised centers shall to identify specific and internal pathways shared by all the AOU

Date: Aug 28, 2019

Francesca Pasinelli, General Manager of Telethon Foundation, explains the advanced therapies state-of-art in Europe, within the recent report of Alliance for Regenerative Medicine, group including research centers, scientific societies, funding bodies, pharmaceutical and biotech Companies; among the ARM recommendations to improve the ATMP access process efficiency, are highlighted some useful methodologies already involved in Italy, such as the risk sharing agreements

Date: Jul 30, 2019


The researchers of Children’s Hospital of Philadelphia report a more sensitive method for capturing the footprint of adeno-associated virus, used as viral vector in gene therapy, improve the DNA transfer site detection capability. The new AAV screening method uses sensitive editing-reporter transgenic mice that are marked; in side-by-side comparisons with conventional screening methods, this method radically redefines the true extent of AAV-mediated gene transfer

Date: Jul 30, 2019


Scientists at the University of Eastern Finland and the University of Oxford have shown that small RNA molecules occurring naturally in cells, and they are also abundant in cell nuclei. The results of the study, published on Scientific Reports, suggest that microRNAs play a role in the expression of genes in the cell nucleus, and this observation is crucial for the development of novel gene therapy

Date: Jul 17, 2019

Researchers from Tufts University and the Chinese Academy of Sciences have developed a novel method, based on biodegradable synthetic lipid nanoparticles, to effectively deliver the CRISPR/Cas9 gene editing tools into the cells; the study was published on Advanced Materials.

Date: Jul 15, 2019

Cellectis, a biopharmaceutical company focused on developing immunotherapies based on allogeneic CAR T-cells, announced the publication of a study in BMC Biotechnology, describing and evaluating the development of the SWIFF-CAR, a CAR construct with an embedded on/off-switch, which enables tight control of the CAR surface presentation and subsequent cytolytic functions using a small molecule drug; this method represents a promising approach to further mitigate the potential toxicities that are associated with CAR T-cell administration

Date: Jul 8, 2019

The International Society on Thrombosis and Haemostasis has announced the official launch of “Gene Therapy in Hemophilia: An ISTH Education Initiative”. This landmark launch, the first of its kind in hemophilia, will take place during the ISTH XXVII Congress held in Melbourne on 6-10 July. As gene therapy emerges as a potential new treatment for patients with hemophilia, the ISTH recognizes an immediate need to educate clinicians, scientists and other interested healthcare professionals in the global hemophilia healthcare community

Date: Jul 8, 2019

Fondazione Telethon has provided €11 millions for scientific research exellence with the aim to identify and to develop new therapies for the treatment of rare genetic diseases. The fund will support 35 projects, that involve 50 research groups on the entire italian territory

Date: Jul 8, 2019

Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy and pioneer of the development and applications of lentiviral vectors for gene therapy and of advance targeted genome editing in cell and gene therapy, shows the results of advanced therapies and the prospects of Genenta, an italian gene therapy company focused on developing therapies against tumors and of which he is founder, in the treatment of oncological diseases

Date: Jul 4, 2019

Luigi Naldini, gene therapy pioneer, gives a sum up of the advanced therapy scenario: on one side it represents a new era of medicine, able to provide novel possibilities for rare diseases without therapies until now, on the other side it is a challenge for the world of scientific research and for the National Health Service, given the complexity and the high price of the therapies

Date: Jul 2, 2019

Professor Locatelli, President of the Italian Senior Health Council and director of the Pediatrics oncology/hematology, cell and gene therapy department of the Bambino Gesù Pediatric Hospital in Rome, talks about CAR-T, the most advanced immunotherapy against tumors available today, based on immune system cells of the patients genetically modified to attack the tumors by the addition of a gene sequence coding for the chimeric antigen receptor

Date: Jun 25, 2019

Approximately 800 adult patients and 40 children with blood tumors will soon be eligible for CAR-T therapy, the novel treatment for leukemia and lymphoma in patients not responding to conventional care, with a 50% of complete response rate; This is the state-of-the-art of CAR-T illustrated by Paolo Corradini, President of Italian Society of Hematology, director of the Hematology division at IRCCS Foundation National Cancer Institute in Milan and Hematology Professor at University of Milan, and by Franco Locatelli, President of the Italian Senior Health Council and director of the Pediatrics oncology/hematology, cell and gene therapy department of the Bambino Gesù Pediatric Hospital in Rome

Date: Jun 25, 2019

Francesca Pasinelli, general manager of Telethon, exlplains the achievements on treatment of genetic rare diseases, especially spinal muscular atrophy, without treatment until recently and often debilitating or lethal for the children. Zolgensma approval reveals new perspectives to children with SMA, but early diagnosis remains central; for that, in Italy (Lazio and Tuscany) have started a 2 years pilot study for newborn screening aiming to involve 140.000 babies, to promptly diagnose the SMA

Date: Jun 20, 2019

Alessandra Renieri, Professor in the Medical Biotechnology Department at the University of Siena, has been nominated member of EMA Committee for Advanced Therapies and she will alternate with Alessandro Aiuti, from San Raffaele Telethon Institute for Gene Therapy, as clinical expert member

Date: Jun 12, 2019

The study, published on Nature Communications, showed that the number of peripheral dendritic cells and the levels of serum FLT3L were significantly decreased in Systemic Lupus Erythematosus patients compared with healthy controls. Allogeneic umbilical cord-derived mesenchymal stem cells transplant showed an increase in tolerogenic dendritic cells number and of the serum-level of FLT3L, implying that these could have an active role in stem cell therapy positive effect in Lupus patients

Date: Jun 12, 2019

Proteona has announced the launch of its latest ESCAPE assay designed specifically for CAR-T cell characterization. This assay is able to identify CAR-positive cells, evaluate changes in cytokine expression, measure the expression of 25 key cell surface proteins and discover new gene expression patterns; The procedure, based on the Enhanced Single Cell Analysis with Protein Expression RNA sequencing technology, is the first commercial service that uses DNA-barcoded antibodies to capture proteomic and gene expression data simultaneously at the single cell level

Date: Jun 11, 2019

Francesca Pasinelli, general manager of Telethon, talks about rare diseases and their therapies; FDA approval of Zolgensma increase the focus on high cost therapies, triggering controversies on their real need, regardless of the huge impact on patients’ life

Date: Jun 3, 2019

A study of the Fred Hutchinson Cancer Research Center, published on Nature Materials, shows positive results about the use of gold nanoparticles like a CRISPR gene editing system’s carrier into the blood stem cells. The gene therapy technologies are inaccessible to millions of people, and the development of simpler systems for the gene editing key components’ delivery has therefore proved necessary. The gold nanoparticles were engineered to quickly cross the cell membrane, dodge cell organelles that seek to destroy them and go right to the cell nucleus to edit genes

Date: May 30, 2019

Starts in Padua Road map CAR-T, a series of regional conferences in order to create a communication link between medical system, politicians and stakeholders, to enable different Italian Regional Health Services to make the best operative decisions which will make the system efficient and sustainable, and at the same time to ensure a rapid patient access to the treatment. The CAR-T therapies should be read as complex and expensive treatment pathways, that require a careful services system organization, necessary to avoid the system’s crisis

Date: May 30, 2019

Published on the Official Journal the AIFA 2018 notice for independent researches carried out on specific themes, identified by the Agency, i.e. rare diseases, CAR-T and controlled comparative clinical studies. AIFA considers support for independent research an investment to build knowledge on effectiveness, safety and proper use of drugs, especially in areas that are out of the interest of for profit research.
Each project can collect a financing up to  €1,5 million for interventional studies, €500.000 for observational studies and €75.000 for meta-analysis

Date: May 28, 2019

The European Patent Office (EPO) grants the protection until 2035 for an innovative component, a “spacing structure” of CAR protein, already used in company’s CAR-T in development for different tumors. This “spacer” derives from the Low-affinity Nerve Growth Factor Receptor (LNGFR) allows to select the Car-expressing cells without the marker-gene inclusion, in-vivo cell tracking and to block the recognition by the innate immune system of Car, with an increased survival compared to Car with other spacers

Date: May 24, 2019
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