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News

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

The FDA has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A; the treatment candidate consists of human cells modified to incorporate large amounts of synthetic DNA-encoding therapeutic proteins into cells, specifically human FVIII. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application fees

Date: Aug 28, 2019

Celgene will have opt-in rights to take responsibility for development, manufacturing and commercialization of T-cell receptor (TCR) therapies that Immatics develops using its in-house technology. The deal includes a $75 million investment in the company by Celgene; Immatics would develop TCRs against solid tumor cancers using its in-house technology

Date: Aug 28, 2019

Esco Ventures announce the launch of Carmine Therapeutics and execution of an exclusive license agreement for the technology developed in the laboratories of Professors Minh Le and Jiahai Shi at the City University of Hong Kong. Carmine Therapeutics plans to establish a site in Cambridge, and it will be focused on pioneering red blood cell-derived extracellular vesicle-based gene therapies

Date: Aug 28, 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

The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader regenerative medicine sector, released a Therapeutic Developers’ Statement of Principles, setting forth a bioethical framework for the use of gene editing in therapeutic applications; the statement, developed by ARM’s Gene Editing Task Force and signed by 13 therapeutic developers using gene editing technologies, specifies five key principles for the ethical use of gene editing and genetic modification

Date: Aug 27, 2019

 Adaptimmune Therapeutics, a leader in T-cell therapy to treat cancer, and Noile-Immune Biotech, a biotechnology company focusing on the development of innovative cancer immunotherapies, announced that they will co-develop next-generation SPEAR T-cell products, incorporating Noile-Immune’s PRIME (proliferation inducing and migration enhancing) technology, based upon co-expression of IL-7 and CCL19. The PRIME technology, which is already being investigated for augmentation of CAR-T cell activity, will be investigated with Adaptimmune’s SPEAR T-cells

Date: Aug 27, 2019

Scientists may have found a way to pull down the protective wall that surrounds tumours, potentially re-exposing them to the killing power of the immune system and immunotherapy treatments, according to a study part funded by Cancer Research UK and published in EBioMedicine. The presence of myeloid-derived suppressor cells (MDSCs) causes a worse outlook for tumor patients, and their cancer can become resistant to treatment and is more likely to spread to other parts of the body; this approach made it possible to eliminate the tumor barrier, potentially boosting the immunotherapy activity

Date: Aug 26, 2019

DiscGenics, a biotechnology company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine, announced that the FDA has granted Fast Track designation for its investigational cell therapy, IDCT, currently being evaluated in regulator-allowed clinical trials in the USA and Japan for the reduction in pain and disability associated with degenerative disc disease. IDCT is a homologous, allogeneic, injectable cell therapy that utilizes biomedically engineered progenitor cells, known as discogenic cells, that have been derived from intervertebral disc tissue to offer a non-invasive, potentially regenerative solution for the treatment of mild to moderate degenerative disc disease

Date: Aug 26, 2019

Pfizer announced a $500 millions additional investment in gene therapy, with a new modern facility in Sanford, North Carolina; this structure is expected to support Pfizer’s continued investment in gene therapy research and development, even with about 300 new jobs

Date: Aug 21, 2019

A combination of humanized anti-CD19 and anti-B-cell maturation antigen CAR-T cell therapy resulted in a 95% response rate among patients with relapsed/refractory multiple myeloma , according to a study published in The Lancet Haematology; a total of 21 patients received the CAR-T treatment, 20 of which had an overall response at a median follow-up of 179 days. 9 out of 20 responders showed complete response and 5 a very good partial response

Date: Aug 21, 2019

The study is designed to assess KITE-439 and it is now enrolling patients at City of Hope, one of USA best hospitals in cancer treatment. Kite’s TCR therapy targets HPV-16, a viral strain associated with 70% of all cervical cancers worldwide as well as oropharyngeal, anal, penile and vaginal cancers. This personalized immunotherapy thus activates the immune system’s ability to recognize and target specifically the tumor cells

Date: Aug 16, 2019

The German-based drug giant has doubled down on one of its previous investments, announcing a deal to acquire the remainder of the stake in BlueRock Therapeutics for $600 million

Date: Aug 8, 2019

Ziopharm Oncology, announced the EMA Committee for Orphan Medicinal Products adopted a positive opinion recommending Ad-RTS-hIL-12 plus veledimex for designation as an orphan medicinal product for the treatment of glioma. The medicinal product is an investigational gene therapy designed to induce and control the production of human interleukin 12, a master-regulator of the immune system

Date: Aug 8, 2019

Selecta Biosciences and Asklepios BioPharmaceutical AskBio), announced a strategic partnership to jointly develop, manufacture and commercialize targeted therapeutics for life-changing, next-generation adeno-associated virus (AAV) gene therapies in areas of high medical need. This partnership will leverage the unique proprietary technology platforms of both companies; Selecta is the first company with preclinical evidence to support the potential for re-dosing patients receiving gene therapy; AskBio’s gene therapy platform includes a robust pipeline of potentially curative gene therapies, an extensive capsid library, groundbreaking manufacturing process and several advanced AAV initiatives under development

Date: Aug 7, 2019

 A research team of the Cibio Department of the University of Trento was able to prove the efficacy of CRISPR-Cas, in solving once and for all the problem that causes cystic fibrosis. The disease is caused by a mutation of the gene that produces cystic fibrosis transmembrane conductance regulator or CFTR, whose malfunctioning affects multiple organs, especially the lungs; the team used CRISPR to permanently edit at least two types of the mutation that cause cystic fibrosis, fixing the gene and restoring the protein production mechanism at the same time

Date: Aug 7, 2019

Con l’approvazione da parte del Consiglio di amministrazione di AIFA alla rimborsabilità della prima terapia a base di cellule CAR-T disponibile in Italia, si è concluso l’iter procedurale per “garantire l’accesso a queste nuove terapie salvavita. Kymriah è indicato per pazienti adulti con linfoma diffuso a grandi cellule B resistenti alle altre terapie o nei quali la malattia sia ricomparsa dopo una risposta ai trattamenti standard, e per pazienti fino a 25 anni di età con leucemia linfoblastica acuta a cellule B. Kymriah è stato approvato attraverso un innovativo modello di rimborso usato per la prima volta in Italia, il payment at results

Date: Aug 7, 2019

Mustang Bio, a biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, announced that the FDA has approved the Company’s Investigational New Drug application to initiate a multi-center Phase 1/2 clinical trial of MB-102 in acute myeloid leukemia, blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic syndrome

Date: Aug 5, 2019

Eureka Therapeutics, a biopharmaceutical company developing novel T-cell therapies, announced initiation of Phase 1/2 clinical trial of ET140202 ARTEMIS T-Cell therapy in advanced hepatocellular carcinoma at City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, based in California. The therapy targets an alpha fetoprotein-peptide/HLA-A2 complex on HCC cancer cells, and demonstrated a favorable safety profile with no observed cytokine release syndrome or drug-related neurotoxicity

Date: Aug 5, 2019

Mount Sinai researchers have developed a way to use immunotherapy drugs against treatment-resistant non-Hodgkin's lymphomas for the first time by combining them with stem cell transplantation, an approach that also dramatically increased the success of the drugs in melanoma and lung cancer, according to a study published on Cancer Discovery

Date: Aug 5, 2019

Precigen, a biopharmaceutical company specializing in the development of innovative gene and cellular therapies, announced that the first patient has been dosed with Precigen's PRGN-3005, a first-in-class investigational therapy using Precigen's UltraCAR-T therapeutic platform. PRGN-3005 UltraCAR-T is an autologous chimeric antigen receptor T cell therapy manufactured using non-viral gene delivery and is under investigation for the treatment of patients with advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer

Date: Aug 5, 2019
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