23-29 Sep, 2019
23-26 Oct, 2019
May 12-15, 2020
Oct 20-23, 2020
May 12-15, 2021
AVROBIO, a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the first patient has been dosed in the company’s GuardOne clinical trial, a Phase 1/2 investigational study evaluating AVR-RD-02 for Gaucher disease type 1. The company also announced that the second patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.
The first clinical trial of a regulatory T-cell therapy enhanced with a chimeric antigen receptor is planned for 2020. Preclinical studies are promising and potential applications wide, but there is still a long way to go (although an off-the-shelf version is already being considered).
Prof. Dr. Alessandro Aiuti, a physician and research scientist of San Raffaele Telethon Institute for Gene Therapy, based in Milan is the winner of the Else Kröner Fresenius Prize for Medical Research 2020, which is awarded by the EKFS in recognition of Aiuti's significant scientific contributions to the development of gene therapies. Together with his team, Professor Alessandro Aiuti pushed forward the development of successful gene therapy approaches for diseases such as Wiskott-Aldrich Syndrome or Metachromatic Leukodystrophy. He achieved a widely recognized success as a key figure in the development of a gene therapy for ADA-SCID (Adenosine Deaminase Severe Combined Immuno Deficiency). This gene therapy and the achievement of its market approval in Europe is considered to be one of the most important results in the development of gene therapies worldwide.
Presented during the Digital Week in Milan, WelCARe is a real "virtual ambulatory" based on the digital platform developed by Novartis, a pioneer in the development of CAR-T therapies, together with the Milan start-up Soluzioni Salute Informatica. WelCARe is a digital tool that will link telemedicine systems with artificial intelligence (AI), which meets two important needs related to the care and treatment of a patient treated with CAR-T therapy: from one side it improves access to therapies even for those who are not located close to an authorized center for their administration, from the other side it helps patients to maintain contact and be monitored by the centre where the therapy was administered.
"Alleanza Contro il Cancro" (ACC), an important Italian cancer research organization, has published an expression of interest that aims, through the harmonization and automation of processes, to reduce costs related to the CAR-T cell manufacturing and to implement production capabilities, building a national network open to the participation of other pharmaceutical and industrial companies.
With the Regional Deliberation of the Regional Government 07.02.2020, n. 6-982, the Piedmont Region identified the regional centres prescribing the CAR-T therapy, according to the minimum criteria indicated by the AIFA Technical Scientific Committee (CTS).
The identified regional centres prescribing CAR-T therapies are:
After decades of clinical research targeting development of more “personalised” medicines, the biotechnology sector is now starting to deliver with the first generation of promising new cell and gene therapies. To better understand deal-making trends related to cell and gene therapies, Lev Gerlovin and Pascale Diesel conducted an analysis of recent activity in the sector.
The review shows that overall the pace of deal-making in cell and gene therapy is faster and unlike deals made decades ago related to mAbs, larger pharmaceutical companies are not waiting for the cell and gene therapy sector to become well established before pursuing partnerships or M&A deals.
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
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
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
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
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
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
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
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.
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
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
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
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
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
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
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
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
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
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
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
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
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