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News

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

Gracell Biotechnologies, a immune cell therapy company, has announced a positive continued clinical readout of a multi-center pilot study designed to evaluate the safety and efficacy of Gracell's first-in-class FasT CAR-19 (GC007F) investigational cell gene therapy; the results were announced during the CAR-TCR Summit held from 10-13 September in Boston. At present, anti-CD19 CAR-T bioprocessing takes on average two weeks to manufacture and seven days to pass quality testings. However, with Gracell's FasT CAR solution, customized treatments which genetically modifies patient's T-cells to express CD19-specific CAR, preparation time can be cut to one day, with a manufacturing success rate of 100% without patient loss. The treatment efficacy was assessed in 24 treated patients over 28 days of follow-up, of which 23 achieved complete remission and 21 achieved undetectable minimal residual disease

Date: Sep 11, 2019

new gene therapy turns glial cells, abundant support cells in the brain, into neurons, repairing damage that results from stroke and significantly improving motor function in mice. A paper describing the new therapy, which uses the NeuroD1 gene, appears online in the journal Molecular Therapy. Once further developed, this NeuroD1-based gene therapy could potentially be used to treat stroke, which is a leading cause of disability in the USA, with 800.000 new stroke patients every year

Date: Sep 11, 2019

In a new study published in Nature, Penn Medicine's Abramson Cancer Center’s researchers have explored the potential of cell therapy for removing activated fibroblasts that contribute to the development of heart disease. Cardiac fibrosis is a contributing factor to the many different types of heart disease, caused by a proliferation of cardiac fibroblasts; when these cells are over-activated, often after cardiac injury, they deposit excess extracellular matrix in the cardiac muscle, causing thickening of the muscle and a loss of flexibility. The researchers built on cell immunotherapy work targeting cancer cells to try and target pathologic cardiac fibroblasts utilizing a mouse model; they endeavoured to identify a protein that was specifically expressed by activated fibroblasts, like fibroblast activation protein (FAP), with the promising initial results

Date: Sep 11, 2019

Researchers, coordinated by Krystof S. Bankiewicz, neurosurgen of Ohio State University College of Medicine, developed a novel gene therapy consists of adeno-associated viral vector able to carry a copy of gene coding for lysosomal enzyme acid sphingomyelinase, deficient in Niemann-Pick disease. The results, observed in animal models, showed an important decrease in sphingomyelinase level without serious immune and inflammatory reactions. These results were published on Science Translational Medicine

Date: Sep 11, 2019

Genenta Science, a italian gene therapy company, has received $14.4 million in round three funding from the Chinese firm Qianzhan Investment Management and Fidim, that will propel their unique gene therapy through Phase I/II trials for multiple myeloma and glioblastoma. This therapy, called Temferon, aims to prevent CAR-T relapses, that occurs in around 40% of cases, by rebuilding the patient’s immune system within the tumor sites themselves; the treatment involves injecting a lentivirus for a gene transfer in hematopoietic stem and progenitor cells, triggering interferon-α expression in the targeted areas, in such a way as to enable the patients to fighting off future relapses

Date: Sep 10, 2019

Adaptimmune Therapeutics, a leader in T-cell therapy to treat cancer, announced that the US FDA has granted Orphan Drug Designation to SPEAR T-cells targeting MAGE-A4 (Adaptimmune’s ADP-A2M4 program) for the treatment of soft tissue sarcomas. Orphan Designation by FDA was created to encourage the development of drugs for rare diseases, such as sarcomas

Date: Sep 9, 2019

Iovance Therapeutics, a Philadelphia based company, is working on new T-cell therapy to treat advanced metastatic cancers, this new immunotherapy uses specific lymphocytes from the patient’s immune system called tumor-infiltrating lymphocytes (TIL). The recent update has revealed remarkable results of the study on cervical cancer and metastatic melanoma and the researchers are hopeful that success in these clinical trials will pave the way for the treatment of other malignant tumors as well. Using positive results of these clinical trials, Iovance will seek FDA approval later in 2020

Date: Sep 9, 2019

Passage Bio, a genetic medicines company developing AAV-delivered gene therapies for the treatment of rare monogenic central nervous system diseases, has announced that it has licensed a sixth gene therapy development program under its research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program; the license is for the clinical development of a potential treatment for patients with Charcot-Marie-Tooth Neuropathy Type 2A. CMT2 patients experience progressive muscle atrophy of legs and arms, with no approved curative or symptomatic medications available

Date: Sep 9, 2019

Syncona, a leading healthcare company focused on founding, building and funding global leaders in life sciences, announced that it has committed £48.0 million of Series B financing to Gyroscope Therapeutics; Gyroscope is a leading clinical-stage, retinal gene therapy company targeting the treatment of dry age-related macular degeneration, one of the leading cause of blindness and for which there is currently no therapy

Date: Sep 9, 2019

Celyad expects to treat the first patients with its autologous CAR-T therapy CYAD-01 using the updated manufacturing process by the end of September. CYAD-01 is an autologous CAR-T-cell therapy developed for hematological malignancies and solid tumors, and it has the ability to bind to eight different ligands that are naturally expressed by cancer cells; the updated manifacturing process includes an eight day culture period, which is two days shorter than the previous manufacturing process, the incorporation of an NKG2D blocking antibody, and the inclusion of a selective PI3 kinase inhibitor

Date: Sep 9, 2019

Scientist of Helmholtz-Zentrum Dresden-Rossendorf have developed a strategy to make conventional CAR-T cells more potent against solid tumors. the newly developed CAR combines conventional T cells with a switchable universal CAR containing a CD3ζ (UniCAR ζ) signaling domain with co-stimulatory CD28 domain (UniCAR 28/ζ), making it escape Treg-mediated immunosuppression. Findings from this study published in OncoImmunology demonstrate that arming conventional CAR-T cells with a UniCAR 28/ζ conveys enhanced capability to escape Treg control and, therefore, this construct could prove to be more suited for treatment of solid tumors

Date: Sep 6, 2019

Krystal Biotech, a gene therapy company developing medicines to treat rare skin diseases, announced the initiation of the Phase 1-2 study of KB105 in transglutaminase-1 (TGM1) deficient Autosomal Recessive Congenital Ichthyosis. The study, termed GEM-3 study, will examine KB105, an HSV-1 based gene therapy engineered to deliver a human TGM1 gene, in patients with ARCI. TGM1-deficient ARCI is a debilitating rare skin disease characterized by excessive, thick scaling of the skin, causing multiple chronic health conditions; there are approximately 23,000 cases of TGM1-deficient ARCI worldwide and about 400 new cases per year globally

Date: Sep 5, 2019

UniQure has achieved its target patient enrollment goal ahead of schedule for its ongoing Phase 3 HOPE-B trial; the trial is assessing the safety and efficacy of AMT-061, the company’s investigational gene therapy for the treatment of patients with moderate and severe hemophilia B. AMT-061 is a gene therapy that uses the AAV5 viral delivery vector, that has received the designation of Breakthrough Therapy from the U.S. FDA, and priority medicines (PRIME) designation from the EMA

Date: Sep 4, 2019

Ultragenyx Pharmaceutical, a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, announced positive data from the second dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus based gene therapy for the treatment of glycogen storage disease type IA. All three patients in Cohort 2 have shown a clinical response with improvements in glucose control and other metabolic parameters compared to baseline; patients in the first, lower dose cohort continue to show longer-term durability in response

Date: Sep 4, 2019

Orchard Therapeutics said it planned to file data from its OTL-200, gene therapy for the life-threatening neurodegenerative rare disease metachromatic leukodystrophy, with regulators early next year in Europe after results from a late-stage trial showed clinical benefit on cognitive and motor function; OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy, purchased by Orchard from GlaxoSmithKline in 2018; it originated from a collaboration between GSK and San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

Date: Sep 4, 2019

Achilles Therapeutics, an UK-based biopharmaceutical company developing personalized immunotherapies for cancer has raised £100 million in Series B financing; Achilles will use the funds to begin this year two human proof-of-concept studies using its personalized T cell immunotherapy to treat non-small cell lung cancer and melanoma

Date: Sep 3, 2019

Autolus Therapeutics, a biopharmaceutical company developing next-generation programmed T cell therapies, announced that the journal Nature Medicine has published both pre-clinical results and clinical data from the ongoing Phase I CARPALL trial of AUTO1, demonstrating the potential of the company’s novel CAR T therapy targeting CD19 in development for the treatment of pediatric acute lymphoblastic leukemia; in the Phase I clinical trial, 86% of patients with acute lymphoblastic leukemia, with a median duration of remission of 7.4 months, showed complete response without severe adverse event, like grade 3 or higher Cytokine Release Syndrome

Date: Sep 3, 2019

Vertex Pharmaceuticals announced it has agreed to acquire Semma Therapeutics for $950 million cash in the fourth quarter of 2019, in a deal that would expand its pipeline of specialty treatment with Semma’s potentially curative cell therapy for type 1 diabetes; Semma said its type 1 diabetes therapy is designed to incorporate a pair of advances, that is the ability to produce large quantities of functional human pancreatic beta cells that restore insulin secretion and a novel device that encapsulates and protects these cells from the immune system, enabling durable implantation without the need for ongoing immunosuppressive therapy

Date: Sep 3, 2019

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

Date: Sep 3, 2019

The FDA has granted Orphan Drug Designation to CARsgen Therapeutics’ investigational CAR-T cell therapy fully human anti-BCMA (B Cell Maturation Antigen) autologous CAR-T cells, CT053, for the treatment of multiple myeloma; under the Orphan Drug Act, the CT053 anti-BCMA product would be eligible for certain benefits including seven years of market exclusivity in the United States following marketing approval by the FDA. CT053 has demonstrated outstanding potency in an exploratory Phase I clinical study; a total of 19 of 24 patients with relapsed and refractory multiple myeloma showed complete response, without severe adverse event, like grade 3 or higher Cytokine Release Syndrome

Date: Sep 2, 2019
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