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

At the recent Huntington’s Disease Society of America annual convention in Boston, UniQure announced crucial details of its clinical trial, planned by the end of 2019, for its experimental therapy AMT-130; this gene therapy allows to express a Huntingtin-lowering molecule instead of trying to delete the HD mutation. The drug will be administrated with a brain injection after the creation of some small holes in the pateints’ skulls

Date: Jul 19, 2019

Apellis Pharmaceuticals, a biopharmaceutical company focused on the development of therapeutic compounds to treat disease through the control of the complement system, announced its plans to develop APL-9 for the prevention of complement immune system activation coincident with adeno-associated virus vector administration for gene therapies. APL-9, an investigational drug, is a cyclic peptide designed to modulate the complement cascade

Date: Jul 18, 2019

Kite Pharma, part of Gilead Sciences, has announced that it will build a 6.000 square meters facility at its Oceanside, California, biologics site just for developing viral vectors, the tools needed to deliver genetic material into cells. So far, Kite has been buying from contractors the viral vector, material needed for its CAR-T treatment Yescarta and for the novel cell therapies, like KTE-X19, whose dossier will be submitted to the Regulatory Authorities within this year

Date: Jul 18, 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

Scientists at Boston Children's Hospital are developing gene therapy that they hope proves effective in treating an inherited form of heart arrhythmia and other more common cardiac diseases; in two studies, published in the journal Circulation, scientists built what could be the first human tissue model of Catecholaminergic Polymorphic Ventricular Tachycardia, a potentially lethal form of arrhythmia. Using that model, they developed a gene therapy procedure that aims to treat CPVT by restoring calcium regulation, pathologically modified by RYR2 gene mutation

Date: Jul 17, 2019

Pluristem Therapeutics, a regenerative medicine company developing novel placenta-based cell therapy products, announced that results from a series of studies of the company’s PLX-R18 cell therapy product conducted by the U.S. Department of Defense’s Armed Forces Radiobiology Research Institute, will be jointly presented at the upcoming 2019 Radiation Injury Treatment Network Workshop on July 31st; this cell therapy could be a potential prophylactic countermeasure against Acute Radiation Syndrome administered prior to radiation exposure. PLX-R18 was granted an FDA orphan drug designation and an IND for the treatment of ARS

Date: Jul 17, 2019

After four years of collaboration, Kyoto University was transferring the first novel induced pluripotent stem cell-derived CAR-T (iCAR-T) therapy to Takeda; under the terms of the deal, Takeda has global rights to develop and commercialize the product and Kyoto University will receive various milestone payments. The first-generation autologous CAR-T products are very individualized, which is both slow and expensive; the hope with this novel generation of CAR-T is that this can be both less expensive and less time-consuming, as it is very scalable

Date: Jul 16, 2019

Developing a CAR-T therapy platform for T-cell cancers comes with its own unique set of challenges. The main hurdle for using CAR-T therapy for T-cell cancers is that the treatment is also the target; all T-cells express the same antigens, regardless if they are healthy or cancerous. To develop a CAR-T therapy against a T-cell cancer, the CAR-T cells would be reprogramed to target one of these T-cell specific antigens. Wugen, a St. Louis-based biotech company, is bridging this treatment gap by developing the first allogeneic CAR-T therapy for T-cell cancers, by gene-editing to remove the T-cell specific target antigen, to reduce the self-killing ability of CAR-T, and the T-cell receptor, reducing the risk of Graft-versus-Host Disease

Date: Jul 16, 2019

Avalon GloboCare, a leading global developer of cell-based technologies and therapeutics, announced an update on its four clinical programs in cellular therapy, including AVA-001 and AVA-101, that leverage CAR-T cell therapy for immuno-oncology, as well as AVA-201 and AVA-202 exploring novel development with stem cell derived exosomes

Date: Jul 15, 2019

Caladrius Biosciences, a late-stage therapeutics development biopharmaceutical company pioneering advancements of cell therapies in select cardiovascular and autoimmune diseases, announced that the EMA has granted Advanced Therapy Medicinal Product classification to the Company’s CD34+ cell therapy product, CLBS12, for the treatment of Critical Limb Ischemia. CLI is a result of severe obstruction of the arteries that markedly reduces blood flow to the extremities and  can lead to pain, skin ulcers and dermal sores and even amputation

Date: Jul 15, 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

Chimeric antigen receptor–T cell therapy has been effective in the treatment of hematologic malignancies, but it has shown limited efficacy against solid tumors. A study, published by researchers of MIT on Science, shows an approach to enhancing CAR-T function in solid tumors by the administration of vaccine, containing ligands for chimeric receptor, that stimulates CAR-T expansion and antitumor efficacy

Date: Jul 12, 2019

After the experience of the Tumors National Institute in Milan with CAR-T therapy, the Seràgnoli Ematological Institute in Bologna is ready to begin. At the moment the compassionate use is the only mean of access to this therapy, but the experts hope that the negotiation procedure in AIFA will be completed by the end of the year

Date: Jul 12, 2019

Cryoport and EVERSANA have announced a strategic alliance to create a fully integrated regenerative medicine supply chain solution. The alliance will be designed to leverage EVERSANA's position as the leading provider of commercial services addressing access, affordability and adherence challenges for cell and gene therapies and Cryoport's complete suite of temperature-controlled logistics solutions to ensure a safe and effective supply chain solution for innovative therapeutic advancements

Date: Jul 11, 2019

A new Italian study conducted  by Irccs Neuromed, Irccs MultiMedica and the University of Salerno has reduced cardiovascular disease in mice by expressing an anti-aging gene often seen in centenarians, that often express a specific variant of a gene called BPIFB4 that lowers blood pressure and improves cardiovascular health. This could one day lead to a gene therapy treating the same condition in humans. Published in the European Heart Journal, the study found that the mice given the gene therapy had healthier blood vessels, with fewer blood clots and less inflammation.

Date: Jul 10, 2019

Results from an ongoing phase 1 clinical trial using CAR-T in pleural cancers were presented at the 2019 American Society of Clinical Oncology Annual Meeting; the study featured preliminary results from 27 patients, 25 of whom had mesothelioma, and the other 2 had metastatic solid tumors of the lung or breast that had spread to the pleura. All of the patients on the trial had a minimum of 3 previous treatment cycles with conventional therapies. The CAR-T therapy directly administred in chest cavity has shown low toxicity and high efficacy

Date: Jul 9, 2019

Seelos Therapeutics has acquired the rights for a gene therapy program targeting the regulation of the SNCA gene, which provides instructions to make alpha-synuclein, a key player in the development of Parkinson’s disease. The accumulation of abnormal alpha-synuclein protein can result in toxic aggregates that lead to the death of dopaminergic neurons. These toxic aggregates are the main component of Lewy bodies found in the brains of Parkinson’s patients

Date: Jul 8, 2019

BioMarin Pharmaceutical plans on submitting its hemophilia A gene therapy to U.S. and European regulators sometime in the fourth quarter, which could bring approval decisions as early as mid-2020. In support of the submissions for valrox are an ongoing Phase 1/2 study as well as an interim analysis of a Phase 3 investigation, and the Company said the results represent a 96% reduction in patients' mean annualized bleed rates

Date: Jul 8, 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

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
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