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

Mesoblast Limited, global leader in cellular medicines for inflammatory diseases, announced that the FDA has granted the company’s product candidate, rexlemestrocel-L, Orphan Drug Designation for prevention of post-implantation mucosal bleeding in end-stage chronic heart failure patients who require a left ventricular assist device. The therapy is also being evaluated in patients with New York Heart Association Class II/III moderate to advanced heart failure across multiple sites in North America. Mesoblast’s investigational cell therapy consists of 150 million allogeneic mesenchymal precursor cells and is being developed for injection into heart muscle of patients

Date: Jun 24, 2019

Krystal Biotech, a Pittsburgh-based clinical-stage gene therapy company, is developing a topical gene therapy, KB103, for the most severe form of epidermolysis bullosa, a disease caused by mutations in the COL7A1 gene usually diagnosed in infancy; the patients  affected by the disease will have widespread blistering and areas where there is no skin, which can lead to vision loss and difficulty eating due to blistering and scarring in the mouth and esophagus. Krystal’s treatment uses a viral vector to deliver working copies of the COL7A1 gene directly to the patient’s skin cells and heal wounds without injection, caractherisitc of the competitor gene therapy developed by Fibrocell

Date: Jun 24, 2019

Minerva Biotechnologies, a biopharmaceutical company focused on developing immunotherapies for cancer and cellular therapies in regenerative medicine, announced that the FDA has approved their Investigational New Drug application to conduct clinical trials with huMNC2-CAR44, an autologous CAR T cell therapy for solid tumors

Date: Jun 24, 2019

The University Hospital of Modena will become a national reference centre for the developement of oncology cell therapies, by the estabilishment of the “Cell therapies and immuno-oncology” program, in May; the aim of the program is the creation of a network between Region/Hospital and cell therapies practitioners. The focus is currently the treatment of pancreatic cancer, and a 1/2 clinical trial is expected for 2020. This therapy is based on enginereed adipocytes, able to produce anti-cancer molecules, like a high-performance version of TNF-related apoptosis-inducing ligand also known as TRAIL

Date: Jun 22, 2019

Francesca Pasinelli, general manager of Telethon, exlplains the achievements on treatment of genetic rare diseases, especially spinal muscular atrophy, without treatment until recently and often debilitating or lethal for the children. Zolgensma approval reveals new perspectives to children with SMA, but early diagnosis remains central; for that, in Italy (Lazio and Tuscany) have started a 2 years pilot study for newborn screening aiming to involve 140.000 babies, to promptly diagnose the SMA

Date: Jun 20, 2019

Axovant Gene Therapies, a clinical-stage company developing innovative gene therapies, announces it has signed a strategic partnership with Yposkesi, the largest European Contract Development and Manufacturing Organization for cGMP grade viral vector production. Under this strategic collaboration, Yposkesi will provide expertise in process development, technology transfer, manufacturing scale-up, quality control and quality assurance. The partnership is expected to provide Axovant with sufficient manufacturing capacity to deliver the company gene therapies to patients, a key component for the continued development of its gene therapy pipeline

Date: Jun 20, 2019

Gracell Biotechnologies, a clinical-stage cellular immunotherapy company, announced preliminary results of a multi-center pilot study designed for evaluating the safety and efficacy of Gracell's patented FasT CAR-19 investigational cell gene therapy. Currently approved anti-CD19 CAR-T bioprocessing takes on average two weeks to manufacture and seven days to pass quality test, with Gracell's FasT CAR solution, preparation time can be cut to 24 hours, significantly reducing production cost and waiting time. In addition, FasT CAR can be administered within seven days after leukapheresis, providing substantial meaning and significant benefit to physicians and patients. With a manufacturing success rate of 100% without patient loss, FasT CAR-19 cells are considered much more potent and durable in comparison to currently available alternatives

Date: Jun 20, 2019

Date: Jun 19, 2019

Source: http://www.biopharminternational.com

The companies have signed a strategic multi-year manufacturing agreement for the future commercial production a late-phase investigational cell therapy omidubicel. Under the agreement, Lonza will construct and dedicate production suites at Geleen (Netherlands) for the commercial launch. The agreement also enables Gamida Cell to increase the number of dedicated production suites over time to ensure commercial supply and gives the company the option of expanding into Lonza's global cell and gene

Incysus Therapeutics, a biopharmaceutical company focused on delivering an innovative gamma-delta T cell immunotherapy for the treatment of cancers, has announced that the United States Patent and Trademark Office has issued U.S. Patent titled “Drug Resistant Immunotherapy for Treatment of a Cancer.” The patent covers the treatment of brain cancer with the combination of certain chemotherapies and a gamma-delta T cell genetically modified to confer chemotherapy resistance; this combination reduces the immunosuppression of solid tumor cancers while maintaining γδ T cell functionality

Date: Jun 19, 2019

Rocket Pharmaceuticals has started dosing patients in a Phase I clinical trial of its adeno-associated viral vector based gene therapy, RP-A501, to treat Danon disease, a rapidly progressive cardiomyopathy; current standards of care for it, including heart transplant, are not curative and are associated with considerable morbidity and mortality. The trial has been designed to evaluate the safety and tolerability of a single infusion of the treatment

Date: Jun 19, 2019

Triumvira Immunologics, a clinical-stage biopharmaceutical company developing a novel platform for engineering T cells to attack cancers, announced that both the FDA and Health Canada have cleared Triumvira’s Investigational New Drug and Clinical Trial Applications for its novel T cell therapy product, TAC01-CD19, in patients with CD19-postive B-cell malignancies. The Phase 1/2 study is expected to be initiated in the third quarter of 2019. TAC01-CD19 comprises patient-derived T cells that have been genetically engineered to express the CD19 T cell Antigen Coupler; this novel approach has been developed to deal with the problem of CAR-T toxicities and limited tumor types where CAR-T are effective

Date: Jun 18, 2019

Abeona Therapeutics, leader company in gene and cell therapy, announced that the FDA has granted Fast Track designation to its ABO-202, a one-time gene therapy for CLN1 disease designed to deliver a functional copy of the PPT1 gene to the central nervous system and peripheral organs. Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need like CLN1 disease, also known as Infantile Neuronal Ceroid Lipofuscinosis, a rapidly-progressing rare lysosomal storage disease with no approved treatment

Date: Jun 18, 2019

Torque, a immuno-oncology specialized company, is developing Deep-Primed T Cell Therapeutics to direct immune power deep within the tumor microenvironment. The company has announced today that the FDA granted Fast Track designation for its first Deep-Primed T cell immunotherapy program, TRQ-1501. This Fast Track designation is for the treatment of relapsed or refractory solid tumors and lymphomas that express any of five tumor-associated antigens. Torque is currently conducting a Phase 1/2 clinical trial of TRQ-1501 for these indications

Date: Jun 18, 2019

Innovent Biologics announced that the clinical results of CT103A, the potential best-in-class therapy of fully-human BCMA CAR-T co-developed by Innovent and Nanjing IASO Biotherapeutics, was presented at two of the most prestigious clinical meetings in the worlds of hematology and oncology, the 24th Congress of the European Hematological Society and the American Society of Clinical Oncology Annual Meeting 2019. The data of CT103A show an impressive efficacy results, persistence and safety profile and an objective response rate (ORR) of 100% and they are especially encouraging for patients who relapsed from a prior CAR-T treatment

Date: Jun 17, 2019

CASI Pharmaceuticals, a U.S. biopharmaceutical company that is focusing on developing and accelerating the launch of innovative therapeutics and pharmaceutical products, announces the signing of a license agreement for exclusive worldwide license and commercialization rights to CNCT19, an autologous anti-CD19 T-cell therapy product from Juventas Cell Therapy. CD19 is a B-cell surface protein widely expressed during all phases of B-cell development and a validated target for B-cell driven hematological malignancies. CASI will be responsible for payment of certain future development milestones and sales royalties

Date: Jun 17, 2019

Voyager Therapeutics, a clinical-stage gene therapy company focused on developing treatments for severe neurological diseases, announced a restructuring of its gene therapy relationship with Sanofi Genzyme. Under the terms of the agreement, Voyager gains worldwide rights to the VY-HTT01 Huntington’s disease program and ex-U.S. rights to the VY-FXN01 Friedreich’s ataxia program, then transferred to Neurocrine Biosciences. Voyager has agreed to make a $10 million upfront payment to Sanofi Genzyme, partially offset by a $5 million payment from Neurocrine Biosciences to Voyager. An additional $10 million payment is due to Sanofi Genzyme from Voyager upon filing of an investigational new drug (IND) application for VY-HTT01

Date: Jun 17, 2019

The biotechnology company is working as it weighs options including a sale or partnerships, which giving to the purchaser the access to pipeline of experimental treatments for hemophilia, Huntington’s disease and other disorders; major pharmaceutical firms have been pursuing gene-therapy companies that promise to treat rare, debilitating diseases by correcting DNA flaws. UniQure’s shares have risen more than 151% in New York this year, giving it a market value of about $2.7 billion

Date: Jun 16, 2019

After the obtaining of Marketing Authorisation by EMA in the early days of June, Bluebird Bio annouces a price for Zynteglo, its gene therapy for the treatment of  transfusion-dependent β-thalassemia in patients without donors for hematopoietic stem cell transplantation, of €1.575 millions; payers only pay if Zynteglo works, with a €315.000 advance payment and 5 annual instalments. According to the company,  ability to avoid transfusions and to improve patient’s quality of life justifies the price

Date: Jun 14, 2019

The phase 2-3 clinical trial, funded by the European Union, was designed to evaluate the efficacy of MesoPher, an antitumoral vaccine that combines patient’s dendritic cells and a specific cell line engineered to recognize the cancer cells. Mesothelioma is a rare form of tumor, also related to asbestos exposure, with a mean survival of 9-12 months after diagnosis; the chemotherapy increases life expectancy by 3 months

Date: Jun 13, 2019

The phase 1 clinical trial, developed by researchers of Memorial Sloan Kettering Cancer Center, explores the combination of CAR-T and anti PD-1 drugs for the treatment of malignant mesothelioma; among the 18 mesothelioma patients in the study, 14 received a combination of two different immunotherapy treatments, and 11 of this group experienced a complete response, a partial response or stable disease. The rationale for the combination includes the anti PD-1 ability to limit cancer to block immune cells from attacking it, bolstering the immune system to recognize and kill cancer

Date: Jun 13, 2019
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