For the first time in Italy, two children with hereditary retinal dystrophy, of 8- and 9-years of age, have been treated with gene therapy at the Eye Clinic of the University of Campania "Luigi Vanvitelli"of Naples in collaboration with Novartis. The innovative therapy for hereditary retinal dystrophy, devised in Naples 12 years ago in collaboration with the Telethon Foundation and the Children's Hospital of Philadelphia, is approved at European level and is now awaiting AIFA approval. This gene therapy, which consists in correcting the genetic defect underlying the disease, is administered through surgery by injecting the drug directly into the retina, and has allowed the two treated children, that had severely impaired vision from birth due to the pathology, to regain their vision.
Patients with relapsed or refractory mantle cell lymphoma from prior therapies may benefit greatly from treatment with anti-CD19 KTE-X19 CAR T cells. The results of an interim analysis of the phase 2 ZUMA-2 study, presented in Orlando at the 61st Congress of the American Society of Hematology (ASH) demonstrated that after a single infusion of these CAR Ts, 93% of patients responded to treatment and 67% received a complete response.
Janssen, a Johnson & Johnson pharmaceutical company, presented the Phase 1b/II Cartitude-1 study, conducted in 29 patients with relapsed or refractory multiple myeloma to evaluate the efficacy of a Car-T therapy targeting B-cell maturation antigen (BCMA). The 'enhanced' immunotherapy that exploits the reworked T cells to better target the tumor allows a therapeutic response to be achieved in 100% of cases, with a very good or better response in 86% of patients and partial in 14%. The anti-Bcma Car-T therapy allowed 27 of 29 patients to be free from disease progression at the 6-month follow-up.
An Italian algorithm, presented at the 61st Congress of the American Society of Hematology (Ash) in Orlando, Florida, will help guide the choice of which beta-thalassemic transfusion-dependent patients could mostly benefit from gene therapy - a cure that corrects inside the DNA the defect underlying the blood disorder. The algorithm has been developed by an Italian group of super-experts with the support of Site (Italian society thalassemia and hemoglobinopathies). Gene therapy will not be a therapy for everyone, in fact, of the 5-6 thousand Italian beta-thalassemic patients, the forecast is about 800 candidates; the algorithm will help to divide the possible candidates into three categories, indicating: " Patient with high priority; patient, to be assessed, but with ongoing therapy readjustment and patient excluded ".
Four initial phase studies were presented at the 61st Congress of the American Society of Hematology (Ash) in Orlando, Florida as the forerunners of a second-generation anti-cancer cellular immunotherapy, which strives to overcome the limitations of products already available. The innovations include double-target Car-T, capable of hitting cancer cells by attacking them in two points instead of one and Car-Nk, obtained starting from induced pluripotent stem cells. These innovations bring substantial advantages: improving the efficacy of Car T cells therapy, designing products capable of attacking multiple targets; extending cellular immunotherapy to other blood cancers such as multiple myeloma and the possibility of thinking of a 'Car' cellular immunotherapy not produced each time tailored to each patient, but 'prêt-à-porter', standardized and ready to use, reducing production time and costs.
The first gene therapy treatment in Italy to treat a patient with severe hemophilia A has just ended at the Milan Polyclinic. Haemophilia A is a rare genetic disease that affects 5,000 people in Italy, and it is due to a deficit of one of the proteins involved in coagulation, leading to bleeding that can also be fatal. The administration of this gene therapy will allow the haemophiliac patient to avoid the frequent infusions (administered regularly: even 3 times a week for life) for several years, and to have a blood coagulation equal to that of anyone else, with a huge impact on its quality of life.
Astellas Pharma is the latest on a growing list of companies to see gene therapies in their future, as it has agreed to acquire Audentes Therapeutics for $3 billion. Audentes uses adeno-associated viruses (AAVs) to deliver DNA, primarily to treat genetic neuromuscular diseases, and it is set to file for approval of X-linked myotubular myopathy (XLMTM) gene therapy AT132 in the U.S. and Europe in mid-to-late 2020.
Zolgensma is Novartis’ lifesaving gene therapy treatment for infants with spinal muscular atrophy, a rare and devastating neurological disease. Zolgensma has been used to treat 100 patients since its launch and brought in $160 million last quarter, beating analysts’ expectations. Novartis expects revenue to grow in the future. The company is counting on approvals for Zolgensma in Europe and Japan next year, and it recently presented data demonstrating the gene therapy’s effects on older SMA patients.
Ocugen, a biopharmaceutical company focused on discovering, developing and commercializing of innovative therapies that address rare and underserved eye diseases, has announced the U.S. FDA granted the second orphan drug designation for OCU400, Ocugen’s novel gene therapy, for the treatment of CEP290 mutation associated retinal diseases; mutations in CEP290 have been associated with different diseases including Leber Congenital Amaurosis, Bardet-Biedl syndrome, Joubert syndrome, Senior-Loken syndrome and Meckel-Grüber syndrome
Biomedical engineers at Duke University have developed a method to address failures in a promising anti-cancer drug, bringing together tools from genome engineering, protein engineering and biomaterials science to improve the efficacy, accuracy and longevity of certain cancer therapies; using a combination of CRISPR-based targeting, a protein depot that allows for sustained release of the drug and a highly potent binding system, the team showed that this new strategy could overcome three critical problems that limit the efficacy of many cancer drugs, that is the limited potency, their quick elimination from the body, and the ability of cancer cells to develop resistance to the drug. The research was published on Science Advances
Asklepios BioPharmaceutical, a fully integrated Adeno-Associated Virus gene therapy platform company focused on providing curative therapeutics for genetic disorders, has acquired the technology assets of RoverMed BioSciences. RoverMed developed nanotechnology cargo delivery of therapeutics into the nucleus of diseased cells without affecting healthy cells. Under the terms of the agreement, AskBio will integrate the company's technology and assume all assets of RoverMed
Researchers at the University of Missouri School of Medicine have shown in a mouse study that the powerful gene editing technique known as CRISPR may provide the means for lifelong correction of the genetic mutation, that causes a deficiency of dystrophin, responsible for the disorder. With more study, the researchers hope this cell-targeted CRISPR approach may one day lead to longlasting therapies for children with DMD
In experiments with mice, researchers have developed a way to successfully transplant certain protective brain cells without the need for lifelong anti-rejection drugs; the idea behind the experiments, developed by Johns Hopkins Medicine, was to exploit the natural tendencies of costimulatory signals to train the immune system to accept transplanted cells as self-permanently. To do that, they used two antibodies, CTLA4-Ig and anti-CD154, which keep T cells from beginning an attack when encountering foreign particles by binding to the T-cell surface. The study was published in the journal Brain
A new study, presented at the American Society of Radiation Oncology Annual Meeting, shows that patients who received radiation therapy before chimeric antigen receptor T-cell therapy for relapsed or refractory non-Hodgkin lymphoma were less likely to have severe treatment-related toxicities; none of the patients who received induction radiation therapy less than 30 days before CAR T-cell infusion experienced high-grade cytokine release syndrome or neurotoxicity, two of the common treatment-related toxicities associated with CAR T-cell therapy
Benitec Biopharma, a gene therapy-focused biotechnology company developing novel genetic medicines derived from the proprietary DNA-directed RNA interference platform, announced their plans to complete three non-clinical studies that will facilitate the filing of an Investigational New Drug application and the formal initiation of a Phase I clinical trial in patients suffering from Oculopharyngeal Muscular Dystrophy. BB-301 is an internally optimized, AAV-based gene therapy agent that can both silence the expression of mutated, disease-causing genes and replace the mutant genes with normal, "wild type" genes. This approach to disease management is called "silence and replace" and this biological mechanism offers the potential to restore the underlying physiology of the treated tissues and, in the process, improve treatment outcomes for patients suffering from the chronic and, potentially, fatal effects of the disease
Castle Creek Pharmaceutical Holdings will acquire Fibrocell, which focuses on transformational autologous cell-based therapies for skin and connective tissue diseases, for $63.3M and the deal is slated to be finalized by the end of the year. Fibrocell currently has two product candidates: the FCX-007 and the FCX-013. FCX-007 is an investigational, late-stage stage gene therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa; FCX-013 product works toward the treatment of moderate to severe localized scleroderma, a chronic connective tissue disease that causes of hardening of the skin and tissue below
CytoSorbents Corporation, a critical care immunotherapy leader commercializing its CytoSorb blood purification technology to treat deadly inflammation, announced that Hannover Medical School in Germany will begin the first clinical study called CYTORELEASE, evaluating the use of CytoSorb in treating CRS and inflammation of the brain called CAR-related Encephalopathy Syndrome, following CAR-T cell immunotherapy. The trial is a randomized, controlled pilot study in 34 cancer patients who have received CAR-T cell immunotherapy and who have developed either severe CRS or CRES for a duration less than 6 hours
Precision BioSciences, a genome editing company, has announced the FDA has accepted its Investigational New Drug application for PBCAR20A, the Company’s second off-the-shelf CAR-T cell therapy program. Wholly owned by Precision, PBCAR20A is an allogeneic anti-CD20 CAR T therapy candidate in development for the treatment of non-Hodgkin lymphoma, chronic lymphocytic leukemia and small lymphocytic lymphoma. The company plans to initiate a Phase 1/2a clinical trial in the fourth quarter of 2019
Atara Biotherapeutics, a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, has announced the presentation of initial efficacy data as well as updated safety results from its ongoing Phase 1 study of ATA188 for the treatment of progressive forms of multiple sclerosis. The data are featured in a late-breaking poster presentation at the 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), held in Stockholm, September 11-13, 2019. At approximately 6 months from initial dose, 4 of 6 patients in cohort 1 demonstrated clinical decline which was maintained at 12 months; safety results showed that across the 4 planned dose cohorts, ATA188 was well tolerated in patients with progressive forms of MS with no evidence of cytokine release syndrome, graft versus host disease or dose-limiting toxicities
Biotherapeutics company Momotaro-Gene has announced the beginning of a Phase 2 clinical trial on their innovative treatment for malignant mesothelioma. The study will analyze the efficacy of the company’s novel gene therapy called MTG201 combined with the PD-1 inhibitor Opdivo on patients whose cancer has returned. MTG201 works in two ways: it first delivers the REIC/Dkk-3 gene into cancer cells via the company’s proprietary adenoidal vector technology platform, raising the gene expression in cells in which it has been reduced and triggering cell death, while at the same time increasing the gene’s expression in healthy cells within the tumor. This activates natural killer cells within the body’s immune system. The two actions together work particularly well when combined with the checkpoint inhibitor nivolumab
Data from the first two patients treated with FLT180a, Freeline’s single-infusion gene therapy for patients with hemophilia B, showed a rise in levels of clotting factor IX to 40%, with levels remaining stable for over a year. The experimental therapy uses a harmless adeno-associated virus capsid, or protein shell, to deliver a functional version of human clotting factor IX, whose low levels are the cause of hemophilia B
Freeline, a biotech company focused on innovative gene therapies, has announced the dosing of the first patient in its MARVEL1 study, a multi-centre Phase I/II clinical trial of its AAV gene therapy for Fabry Disease; this disease is a type of lysosomal storage disorder due to deficiency of α-galactosidase A enzyme resulting in an accumulation of lipids throughout the body. The study aimed to deliver a replacement copy of the missing gene to the liver, which will then produce continuous high levels of αGLA, offering the potential for therapy with a single treatment. It is estimated that Fabry Disease affects one in every 40,000 people
Grünenthal agrees deal with Mesoblast to develop and commercialize an allogeneic cell therapy for chronic lower back pain; Mesoblast’s drug candidate, MPC-06-ID, is currently in Phase III trials for the treatment of chronic lower back pain due to degenerative disc disease. Grünenthal will pay $150M upfront to gain access to the potential treatment, with over $1bn reserved in milestone payments for the late-stage candidate
Adverum Biotechnologies, a gene therapy company targeting unmet medical needs in ocular and rare diseases, has announced positive 24-week clinical data from the first cohort of patients treated with a one-time intravitreal dose of ADVM-022 in the OPTIC phase 1 clinical trial in wet age-related macular degeneration; patients treated in this cohort achieved vision maintenance and improvements in retinal anatomy, with zero anti-VEGF rescue injections required, after a one-time intravitreal dose of ADVM-022, through week 24. These patients previously required frequent anti-VEGF injections to control their wet AMD and to maintain functional vision. ADVM-022 was safe and well tolerated
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