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Clinical

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

bluebird bio announced that new data from its ongoing Phase 1/2 HGB-206 study of investigational LentiGlobin™ gene therapy for adult and adolescent patients with sickle cell disease (SCD) show a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS). These data are being presented at the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.

Date: Jun 22, 2020

Pfizer and Sangamo Therapeutics, today announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec (SB-525, or PF-07055480), an investigational gene therapy for patients with severe hemophilia A. All five patients with severe hemophilia A who received the 3e13 vg/kg dose showed sustained factor VIII (FVIII) activity levels, with a median of 64.2% via chromogenic assay . No patients experienced bleeding events or required FVIII infusions.

Date: Jun 18, 2020

A team of researchers from the University of California, San Diego, produced Nk cells from induced pluripotent stem cells (iPSC), derived from somatic skin cells or blood cells, which were restored to embryonic stage and then transformed into Nk cells, but without a gene called Cish.  In this way they proved successful in treating leukemia in experimental trials conducted on mice and reported in the journal Cell Stem Cell. Cish is relevant because it regulates the production of a protein that inhibits the signalling of cytokines, molecules used by the immune system to suppress infections and external pathogens. If applied in the clinic it would be a standardised and not personalised therapy with Car-T.

Date: Jun 16, 2020

Today Lysogene announced that, following discussions with the U.S. Food and Drug Administration (FDA), a clinical hold was issued for the clinical trial AAVance (NCT03612869), a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A). The clinical hold follows observations in some patients of localized findings on MRI images at the intracerebral injection sites. To date, no clinical symptoms have been observed that could be directly attributed to the observed MRI findings and all findings have been reported to competent authorities, ethics committees and the trial’s DSMB (Data Safety Monitoring Board). All trial participants continue to be closely monitored by their care teams.

Date: Jun 5, 2020

The Janssen Pharmaceutical Companies of Johnson & Johnson announced today updated results from the Phase 1b/2 CARTITUDE-1 study (NCT03548207) evaluating the efficacy and safety of JNJ-4528, an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy in the treatment of patients with relapsed or refractory multiple myeloma. Longer-term follow-up results from the Phase 1b portion of the study (n=29), to be shared in an oral presentation at the American Society of Clinical Oncology (ASCO) Virtual Scientific Program (Abstract #8505), show that all patients responded to treatment and that the responses were deep and durable with 86 percent of patients achieving stringent complete response at a median follow-up of 11.5 months and 86 percent of patients being alive and progression free at 9 months

Date: May 13, 2020

Allogene Therapeutics, a biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) therapies for cancer, in collaboration with its development partner Servier, an independent international pharmaceutical company, announced the release of the abstract related to an upcoming oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting. The ASCO abstract includes preliminary data on the first nine patients treated with escalating doses of ALLO-501 and lower dose (39mg) ALLO-647. In this limited dataset with a small number of patients, the overall response rate (ORR) was 78% (95% exact CI: 40%, 97%) with three complete responses (CR) and four partial responses (PR).

Date: May 13, 2020

Researchers at two Harvard-affiliated hospitals are adapting a proven form of gene therapy to develop a coronavirus vaccine, which they expect to test in people later this year, they announced on Monday. A rendering of the outer shell of an adeno-associated virus with the exterior partially removed. The shell is used as a Trojan horse to deliver a genetic component of the coronavirus to raise an immune response. The technique aims to make a person’s cells churn out proteins that will stimulate the body to fight the coronavirus.

Date: May 12, 2020

In an attempt to limit cytokine release syndrome, side effect of CAR-T therapy, without affecting the effectiveness of gene therapy, in recent months the clinicians of the Red Area Paediatric Intensive Care Unit and those of the Paediatric Oncohaematology of the Bambino Gesù Hospital in Rome have successfully experimented with a new approach: rapidly and selectively purify the blood. In the study published in the journal Critical Care Explorations, Dr. Gabriella Bottari and colleagues report the first case of a fourteen-year-old boy suffering from a severe form of acute lymphoblastic leukemia treated with CAR-T in combination with extracorporeal hemoperfusion, after the onset of a serious form of cytokinic release syndrome. The young patient, admitted to paediatric intensive care, developed a severe inflammatory respiratory failure one week after the infusion of CAR-T and was saved and discharged after 15 days from paediatric intensive care

Date: Feb 17, 2020

Hoth Therapeutics Inc., a biopharmaceutical company focused on unique targeted therapeutics for patients suffering from dermatological indications ranging from atopic dermatitis, psoriasis and acne along with gene therapy treatment for asthmatics, is pleased to announce the initiation of a preclinical study for the treatment of asthma and allergic inflammation in collaboration with North Carolina State University (NC State). 

Date: Feb 4, 2020

Asklepios BioPharmaceutical (AskBio), a clinical-stage adeno-associated virus (AAV) gene therapy company, and its NanoCor Therapeutics subsidiary today announced that the first patient has been dosed in a Phase 1 clinical trial of NAN-101. NAN-101 is a gene therapy that aims to activate protein phosphatase inhibitor 1 (I-1c) to inhibit the activity of protein phosphatase 1 (PP1), a substance that plays an important role in the development of heart failure

Date: Feb 4, 2020

After official approval by AIFA, the first clinical trial with CAR-T CD44v6 therapy for acute myeloid leukemia and multiple myeloma at the IRCCS San Raffaele Hospital in Milan started.The first patient, suffering from multiple myeloma, was infused a few weeks ago and the treatment did not cause any adverse event, as stated during the European CAR T Cell Meeting in Barcelona Fabio Ciceri, Professor of Hematology at the University Vita-Salute San Raffaele and Head of the Bone Marrow Hematology and Transplantation Unit, as Principal Investigator of the international phase I/II multicenter study.

Date: Feb 4, 2020

A Ludwig Cancer Research study has devised a new type of chimeric antigen-receptor (CAR) T cell—a family of promising immunotherapies for cancer—that can be switched on and off on demand. The study, led by Melita Irving of the Lausanne Branch of the Ludwig Institute for Cancer Research, George Coukos, director of the Branch, and their colleague Bruno Correia of the École Polytechnique Fédérale de Lausanne (EPFL), addresses a central conundrum of CAR-T therapies: their tendency to provoke potentially deadly runaway immune responses against healthy tissues in patients. Their report appears in the current issue of Nature Biotechnology.

Date: Feb 3, 2020

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.

Date: Jan 24, 2020

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.

Date: Jan 22, 2020

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.

Date: Jan 22, 2020

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.

Date: Jan 22, 2020

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.

Date: Jan 22, 2020

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).

Date: Jan 21, 2020

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.

Date: Jan 21, 2020

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).

Date: Jan 21, 2020

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.

Date: Dec 16, 2019

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.

Date: Dec 9, 2019

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.

Date: Dec 9, 2019

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 ".

Date: Dec 9, 2019
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