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News

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

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

Prevail Therapeutics, a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, announced that the FDA has granted Fast Track Designation for the Company’s lead gene therapy program, PR001, for the treatment of Parkinson’s disease patients with a GBA1 mutation. PR001 is a potentially disease-modifying, single-dose, AAV9-based gene therapy being developed for the treatment of Parkinson Disease-GBA and neuronopathic Gaucher disease

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

Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, presents preclinical data on its gene therapy development candidate for Gaucher Disease at the European Working group on Gaucher Disease in Auvergne; the Freeline program FLT200 is a liver-directed adeno-associated virus gene therapy  designed to increase levels of beta-glucocerebrosidase in macrophages of patients. The preclinical data shows that a single infusion of FLT200 results in sustained GCase expression in the liver and greater bioavailability in the bloodstream as compared to enzyme replacement therapy.

Date: Jul 5, 2019

Celyad, a clinical-stage biopharmaceutical company focused on the development of CAR-T cell therapies, announced that Professor Dr. Eric Van Cutsem from the University Hospital of Leuven presented preliminary interim data from the ongoing SHRINK and alloSHRINK Phase 1 trials. The studies were designed to assess safety and clinical activity of the NKG2D-based CAR-T therapies CYAD-01 and CYAD-101 for the treatment of metastatic colorectal cancer at the European Society for Medical Oncology 21st World Congress on Gastrointestinal Cancer

Date: Jul 5, 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

Century Therapeutics has emerged from stealth mode to develop a new generation of ‘off the shelf’ cancer cell therapies, with $250 million backing from investors including Bayer. Century’s technology is based around induced pluripotent stem cells that have unlimited self-renewing capacity; this enables it to create master cell banks of modified cells that can be expanded and differentiated into immune effector cells to supply cast amounts of ‘off the shelf’ allogeneic homogeneous therapeutics products

Date: Jul 3, 2019

US biotech Amicus Therapeutics is to work with drug development services company Catalent Biologics to develop its cell and gene therapy manufacturing capabilities; Amicus will work with Paragon Gene Therapy, which Catalent acquired earlier this year in a $1.2bn deal to boost its own presence in the fast-developing field. The collaboration aims to expedite the process of moving Amicus’ preclinical gene therapy programmes into the clinic

Date: Jul 3, 2019

A research group, coordinated by Temple University, has studied a new combined treatment consists of last generation antiretroviral and gene editing system CRISPR-Cas9, able to eliminate the virus’DNA from infected cells. The results of the study were published on Nature Medicine

Date: Jul 3, 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

Amicus Therapeutics has entered into a strategic manufacturing collaboration with Brammer Bio, a part of Thermo Fisher Scientific. Thermo Fisher, a best-in-class contract development and manufacturing organization, provides Amicus with immediate clinical and commercial manufacturing capabilities and capacity for the Amicus intrathecal adeno-associated virus Batten disease gene therapy programs

Date: Jul 1, 2019

Researchers at the São Paulo State Cancer Institute in Brazil have succeeded in using a genetically manipulated virus to destroy tumor cells upon injection into mice with prostate cancer. The virus also made tumor cells more sensitive to chemotherapy drugs, halting tumor progression and almost eliminating tumors in some cases; the data are described in an article in Gene Therapy, a journal published by Springer Nature

Date: Jul 1, 2019

Pfizer presented initial Phase 1b clinical data on PF-06939926, an investigational gene therapy to potentially treat Duchenne muscular dystrophy at the 25th Annual Parent Project Muscular Dystrophy Connect Conference in Orlando. The goal of the study is to assess the safety and tolerability of this investigational gene therapy; other objectives of the clinical study include measurement of dystrophin expression and distribution, as well as assessments of muscle strength, quality and function

Date: Jul 1, 2019

One-time dosing with potential lifetime benefit creates challenges for payers to adequately reward the manufacturers of such innovations with a sufficient return on their investment.It is important to characterize the challenges for traditional approaches to assessing the value of one-time gene replacement therapies for the creation of a health economic rationale for a higher value-based cost-effectiveness threshold. The Institute for Clinical and Economic Review in the United States has discussed a range of up to $500K per quality-adjusted life-year gained for ultrarare diseases, and the National Institute for Health and Care Excellence in the United Kingdom has described a variable threshold up to £300,000 per QALY depending on the magnitude of the health gains

Date: Jun 28, 2019

OncoSec Medical, a company developing late-stage intratumoral cancer immunotherapies, announced that it has entered into a collaboration with Dana-Farber Cancer Institute, a world-leading cancer research and treatment institution, and The Marasco Laboratory, a cutting-edge CAR T-cell research laboratory, to develop CAR T-cell therapies for triple-negative breast cancer and other solid tumor cancers

Date: Jun 27, 2019

The ongoing SB-525 Phase 1/2 trial positively shows that a single infusion leads to stable and clinically relevant improvements in clotting factor VIII and marked reductions in the need for replacement therapy in patients with hemophilia A. The new findings will be presented at the 2019 Congress of the International Society on Thrombosis and Haemostasis taking place in Melbourne on 6-10 July. SB-525 is a gene therapy candidate under development by Sangamo and Pfizer , and it consists of a small portion of genetic material coding for the production of a functional factor VIII. The therapy has been granted fast track and orphan drug designations by the FDA

Date: Jun 26, 2019

Seelos Therapeutics, a clinical-stage biopharmaceutical company, announced the exclusive worldwide licensing of a gene therapy program targeting the regulation of the SNCA gene, which encodes alpha-synuclein expression,  from Duke University; Seelos plans to study this approach, named SLS-004, initially in Parkinson’s Disease. SNCA gene has been implicated as a highly significant risk factor for Parkinson, and accumulating evidence suggests that elevated levels of wild-type alpha-synuclein are causative in the pathogenesis of the disease

Date: Jun 26, 2019

A team of researchers partly supported by EU funded StemHealth project, has made a study, published on Nature, that led to discover of human fetal intestinal cells’ potential to become stem cells. According with the study, and contrary with the previous assumptions, the immature intestinal cells’ development is not predetermined, but it is influenced by the environment.This informations could help the scientist to simplify the manipulation of stem cells for the development of innovative therapies

Date: Jun 26, 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
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