Krystal Biotech, Inc. (Nasdaq:KRYS), a gene therapy company, announced today the ground breaking of the second commercial gene therapy facility in Findlay Township, Pennsylvania. The Findlay-based Current Good Manufacturing Practice (cGMP) facility, named ASTRA, will have the capacity to produce commercial gene therapy medicines to treat patients suffering from debilitating rare diseases.
Chimeric antigen receptor (CAR)–T cells have been clinically effective in killing certain hematological malignancies, but achieving long-term patient responses for solid tumors remains a challenge. Reinhard et al. describe a two-part “CARVac” strategy to overcome poor CAR-T cell stimulation and responses in vivo. They introduce the tight junction protein claudin 6 (CLDN6) as a new CAR-T cell target and designed a nanoparticulate RNA vaccine encoding a chimeric receptor directed toward CLDN6. This lipoplex RNA vaccine promotes CLDN6 expression on the surface of dendritic cells, which in turn stimulates and enhances the efficacy of CLDN6-CAR-T cells for improved tumor therapy.
Anemocyte, an innovative Italian company working in the field of cell and gene therapies, with special focus on plasmid production and non-viral gene modification approaches, attends Phacilitate Leaders World (Miami, 21-24 January 2020), a leading event for companies, professionals and investors working in the Advanced Therapy sector. Anemocyte is a key player and the first ever Biotech Manufacturing Organization (BMO) operating in the Life Science sector: company helps CGT developers to articulate initial ideas, perform clinical trials and engage in commercial production. The BMO also develops technological platform strategies for innovative R&D, HQ and GMP processes.
The planarian flatworm is a simple animal with a mighty ability: it can regenerate itself from nearly every imaginable injury, including decapitation. Scientists have studied these worms for decades to better understand fundamental principles of natural regeneration and repair. Specifically, Petersen and Schad discovered that a gene called mob4 suppresses tissue growth in the animals. The gene, they found, works in a rather surprising way: by preventing the descendants of stem cells from producing a growth factor called Wnt, a protein released from cells to communicate across distances. The Wnt signaling pathway is known to play a role in cancer cell regeneration.
Aruvant, a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for the treatment of severe blood disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug designation to ARU-1801, Aruvant’s investigational therapy for the treatment of sickle cell disease.
According to a study published in the Blood journal, drug profiling and the CRISPR-Cas9 gene editing method have opened new avenues in the development of CAR T-cell therapy, used to treat leukaemia and lymphoma. The study, carried out collaboratively by the University of Helsinki and the Finnish Red Cross Blood Service, surveyed the effect of more than 500 cancer drugs on the function of CAR T cells. The drug profiling highlighted a class of drugs known as SMAC mimetics, which in laboratory tests sensitised cancer cells to CAR T cells. At the same time, drugs that inhibit the function of CAR T cells were found, which have potential in the treatment of adverse effects. By employing the CRISPR gene editing method, the researchers investigated which mechanisms impact the sensitivity of cancer cells to CAR T cells.
The Center for Breakthrough Medicines, a Contract Development and Manufacturing Organization (CDMO) and specialty investment company, to alleviate the critical lack of capacity that is preventing patients from accessing critically needed cell and gene therapies. The CDMO provides preclinical through commercial manufacturing of cell and gene therapies and component raw materials. It offers process development, plasmid DNA, viral vectors, cell banking, cell processing, and support testing capabilities all under one roof.
Chemical nerve agents are some of the most horrifying tools of war today. For years, researchers have been searching for antidotes or treatments that could save those afflicted by these deadly chemicals. In a paper out today in Science Translational Medicine, a team from the U.S. Army Medical Research Institute of Chemical Defense has announced a potential solution: a gene therapy that grants immunity to the effects of nerve agents like sarin.
A novel anti-CD19 CAR T-cell therapy attained a high response rate with minimal neurotoxicity in patients with advanced B-cell lymphoma, according to a preliminary clinical trial.
The results showed that 11 of 20 patients attained complete remissions (CRs) with the Hu19-CD828Z CAR T-cell construct, identical to the CR rate in a previous trial of the FMC63-28Z construct. However, only one of the 20 patients developed severe neurologic toxicity, as compared with half of the patients in the trial of FMC63-28Z, the construct used in axicabtagene ciloleucel (Yescarta).
New research by Dorothy P. Schafer, PhD, at the University of Massachusetts Medical School, reveals the molecular process in which synaptic connections in the brain are damaged in multiple sclerosis and how this contributes to neurodegenerative symptoms. The paper, published in Immunity, also shows how gene therapy may be used to preserve neural circuits and protect against vision loss in the disease.
In the new study, led by scientists at Cardiff University in the UK, researchers used CRISPR–Cas9 screening to discover a new kind of TCR in T-cells: a receptor molecule called MR1.
MR1 functions similarly to HLA in terms of scanning and recognising cancer cells, but one big difference is that, unlike HLA, it doesn't vary in the human population – which means it could potentially form the basis of a T-cell therapy that works for a much broader range of people (in theory, at least).
The activities of the three-year project on Car T therapies will start by January in 13 of 26 IRCSS of Alleanza Contro il Cancro, as announced in October by the Minister of Health, Roberto Speranza. In the program funded by the Ministry of Health (10 million euros granted over two years) other non-IRCCS entities such as CNR, Fondazione Tettamanti and MolMed are involved in different ways. The main objective of the project is the development of new Car T cell therapies designed to destroy tumours that are currently not covered by the pharmaceutical industry (including pancreas, rectum, breast and melanoma) and which cannot be effectively treated in other ways.
LabCorp has unveiled a new suite of cell and gene therapy development offerings from Covance as it looks to get in on this burgeoning new trend. Covance says it will tap its experience to help offer the life sciences industry a close-knit partner and help push on with current and next-gen capabilities in this area.
Genprex, Inc., a clinical-stage gene therapy company utilizing a unique, non-viral proprietary platform designed to deliver tumor suppressor genes to cancer cells, today announced that the U.S Food and Drug Administration (FDA) has granted Fast Track Designation for Genprex’s Oncoprex™ immunogene therapy in combination with EGFR inhibitor osimertinib for the treatment of non-small cell lung cancer (NSCLC) patients with EFGR mutations that progressed after treatment with osimertinib alone.
U.K.-based PhoreMost and Otsuka Pharmaceutical Co. entered into a multi-project collaboration to seek out disease-relevant pathways that can be exploited for the development of gene therapies to treat different illnesses. According to the structure of the agreement, PhoreMost, which has its headquarters in Cambridge, England, will focus its SIZTESEEKER next-generation phenotypic screening platform on those disease-relevant pathways nominated by Otsuka.
In UK, the Cell and Gene Therapy Catapult (CGT Catapult) and the University of Hertfordshire today announced the launch of a new course specifically addressing the foreseeable skills gap in the manufacture of cell and gene therapies as they progress towards manufacturing at scale.
With cell and gene therapies moving towards commercialisation and employment in this space predicted to reach over 6,000 jobs by 2024, this initiative aims to provide targeted training which will support this growth and address the accelerating demand for skills in the sector.
Gene therapy could save and improve lives, but at $2 million per treatment, it’s out of reach for the most vulnerable of cases in the US. CVS Health is leveraging its 2018 acquisition of health insurance company Aetna to launch a new employer-sponsored insurance product by the end of March to absorb the financial risk of covering these therapies.
If approved, Valrox would be the most expensive drug in the world and the first gene therapy in the U.S. that targets inherited hemophilia, one of the most common types of hemophilia out there. Clinical results have shown significant improvements in patients, with the number of bleeding incidents dropping to zero for years after receiving a Valrox injection.
The U.S. Food and Drug Administration (FDA) has approved Avrobio‘s application to expand to the U.S. its ongoing Phase 1/2 trial testing its gene therapy candidate AVR-RD-02 in people with type 1 Gaucher disease. The trial (NCT04145037), called GAU-201, is investigating the safety and efficacy of AVR-RD-02 in eight to 16 patients, ages 16 to 35.
PTC has submitted a Marketing Authorization Application (MAA) for the potential approval of a gene therapy treatment, PTC-AADC, for AADC deficiency with the European Medicines Agency (EMA). PTC expects the Committee for Medicinal Products for Human Use (CHMP) opinion in 2H 2020.
Nationwide Children's, a gene therapy leader, launches manufacturing spinout Nationwide Children's Hospital, a hot spot for gene therapy research, this week launched Andelyn Biosciences, a biotech spinout dedicated to produce gene therapy components for biotech and pharmaceutical companies running clinical trials.
Two $2m treatments launch this year but a reluctance to fork out could dim investor enthusiasm. The drive to cure deadly diseases is about to enter a new and demanding phase as the pharmaceutical industry prepares for the first time to test the appetite for hyper-expensive gene therapies in Europe. The question, however, is whether European health systems, particularly those with a well-established process for determining the cost-effectiveness of drugs, will be able to adapt their models to look beyond the immediate upfront cost and take into account longer-term savings
Astellas has teamed up with Adaptimmune to develop allogeneic chimeric antigen receptor T-cell (CAR-T) and T-cell receptor (TCR) therapies. The agreement sees Astellas pay $50 million (€45 million) upfront and commit to many times as much in milestones to work with Adaptimmune to identify targets and develop cell therapies against them.
Adaptimmune will pocket $50 million and $7.5 million a year in research funding, plus development and sales milestones that could bring the total deal value up toward $900 million.
Bluebird bio, Inc. announced the launch in Germany of ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) for the treatment of transfusion-dependent β-Thalassemia patients without the β0/β0 genotype that are over the age of 12; and has entered into value-based payment agreements with multiple statutory health insurances in Germany, based on the innovative model limited to five payments made in equal instalments. An initial payment is made at the time of infusion. The four additional annual payments are only made if no transfusions for TDT are required for the patient.
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