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News

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

Personalized Stem Cells, a human adipose-derived stem cell company, has received FDA approval for their Investigational New Drug application to use a person’s own adipose-derived stem cells to treat their osteoarthritis; the first clinical trial, expected to begin in August, uses stem cells as a treatment of osteoarthritis in the knee. PSC plans to conduct a series of FDA approved clinical trials starting with uses in orthopedics and expanding to other medical conditions in the future

Date: Jul 22, 2019

Apic Bio, an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, announced that the FDA has granted orphan drug designation to APB-102, a gene therapy soon to be in clinical development for the treatment of genetic SOD1 amyotrophic lateral sclerosis. The incidence of ALS is estimated to be 1.5 to 2.5 cases in 100.000 people in the United States and in Europe, of which 10% are thought to be inherited, and approximately 15% to 20% of these cases are caused by mutations in the gene that produces the Cu-Zn superoxide dismutase 1 enzyme (SOD1)

Date: Jul 22, 2019

Adaptimmune Therapeutics, a leader in T-cell therapy to treat cancer, has initiated a radiation sub-study of its ADP‑A2M4 trial in collaboration with the University of Texas MD Anderson Cancer Center in Houston; published data indicate that low-dose radiation has the potential to enhance T-cell responses in the context of solid tumors. The study is planned to enroll 10 patients across multiple solid tumor indications

 

Date: Jul 22, 2019

PACT Pharma, a leader in the fields of cancer immunology and cell therapy, presented new data demonstrating for the first time the ability to identify mutation targets unique to each person's cancer and verify the cancer specificity of multiple cloned T cell receptors. The company's approach is designed to select and confirm tumor-exclusive mutations to empower a patient's immune system to target their specific cancer

 

Date: Jul 21, 2019

Marker Therapeutics, a company specialized in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, announced interim data from an ongoing clinical trial evaluating its MultiTAA T cell therapy in patients with pancreatic adenocarcinoma; the data were reviewed at the American Association for Cancer Research's Immune Cell Therapies for Cancer conference in San Francisco. The data suggest that MultiTAA therapy may contribute to more durable responses without added toxicity when used in combination with standard-of-care chemotherapy, or as a second-line therapy for patients who are chemo-refractory

 

Date: Jul 20, 2019

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

Casebia Therapeutics’ two-stage gene therapy was successful in delivering the human gene coding for clotting factor VIII in a mouse model of hemophilia A, leading to stable and increasing levels of the factor; this approach is based on the administration of viral vector carrying the human FVIII gene and then the injection of the CRISPR-Cas9 gene editing tool. Hemophilia affects an estimated 400.000 people worldwide, with a majority having hemophilia A. The results were presented in the International Society on Thrombosis and Haemostasis 2019 Congress in Melbourne

 

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
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