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

AlloVir, a clinical stage allogeneic T-cell therapy company, announced that it has received the Regenerative Medicine Advanced Therapy designation from the FDA for Viralym-M, its allogeneic multi-virus specific T-cell therapy, for the treatment of hemorrhagic cystitis caused by BK virus in adults and children following allogeneic hematopoietic stem cell transplantation. The FDA may grant RMAT designation to drug candidates with preliminary clinical evidence indicating the potential to address unmet medical needs in patients with serious or life-threatening conditions. Viralym-M is specifically designed to restore natural T-cell immunity in immunocompromised patients

Date: Jun 11, 2019

Researchers at Helmholtz Zentrum München and the Technical University of Munich, working in collaboration with researchers at the University Medical Center Hamburg- Eppendorf and the University Hospital Heidelberg, have for the first time succeeded in conquering a chronic infection with the hepatitis B virus in a mouse model. According to the study, published in the Journal of Clinical Investigation, the new T-cell therapy was specifically developed as an approach to fighting HBV infection and HBV-associated liver cancer. According to the World Health Organisation, more than 260 million people worldwide are chronically infected with the virus

Date: Jun 11, 2019

Lysogene, a pioneering biopharmaceutical company specializing in gene therapy targeting central nervous system diseases, announced the first European patient has been dosed in AAVance, a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA. The clinical trial aims to evaluate the safety and effectiveness of the therapy, based on adeno-associated viral vector to deliver a functional copy of SGSH gene. MPS IIIA is a lethal neurological disease caused by mutations in the SGSH gene, which is involved in producing an enzyme necessary for the breakdown and disposal of long chain sugar molecules

Date: Jun 11, 2019

Proteona has announced the launch of its latest ESCAPE assay designed specifically for CAR-T cell characterization. This assay is able to identify CAR-positive cells, evaluate changes in cytokine expression, measure the expression of 25 key cell surface proteins and discover new gene expression patterns; The procedure, based on the Enhanced Single Cell Analysis with Protein Expression RNA sequencing technology, is the first commercial service that uses DNA-barcoded antibodies to capture proteomic and gene expression data simultaneously at the single cell level

Date: Jun 11, 2019

Prevail Therapeutics announced that PR001, potential gene therapy for Parkinson’s disease associated with mutations in the GBA1 gene, will move into clinical testing in patients after the FDA accepted an application for the therapy, with the aim to evaluate its tolerability and safety. People with mutations in the GBA1 gene have a higher risk of developing Parkinson’s disease, indeed it is estimated that 7-10% of all Parkinson’s cases are related to GBA1 mutations. PR001 is intended to be a disease-modifying single-dose gene therapy, based on adeno-associated viral vectors to deliver a fully working copy of the GBA1 gene to nerve cells

Date: Jun 11, 2019

Homology Medicines, a genetic medicines company, announced today that it has commenced enrollment of the Phase 1/2 pheNIX trial for HMI-102, a one-time gene therapy development candidate for the treatment of adults with phenylketonuria. The study is designed to evaluate the safety and efficacy of the gene therapy in up to 21 patients ages 18-55 years old who have been diagnosed with PKU. The Company expects to report initial clinical data from the pheNIX trial by the end of 2019

Date: Jun 10, 2019

Autologous anti-CD19 CAR-T therapy lisocabtagene maraleucel showed clinical activity and acceptable toxicity in patients with relapsed-refractory chronic lymphocytic leukemia or small lymphocytic lymphoma, according to data from the ongoing phase 1/2 TRANSCEND CLL 004 trial. The best overall response rate was 81.8%, of which nearly half of patients had a complete response. A health care resource utilization analysis of patients with relapsed/refractory non-Hodgkin lymphoma who received liso-cel on the TRANSCEND NHL 001 trial showed that costs of Cytokine Release Syndrome and Neurological Events were primarily due to the cost of hospitalization, ranging from $177 for a grade 1 NE to $263,743 for concurrent CRS and NE of grade 3 or higher

Date: Jun 10, 2019

Axovant has reported positive results from the ongoing phase 2 clinical trial on its investigational gene therapy for the treatment of Parkinson disease. The therapy is based on a viral vector that delivers some genes coding for a set of enzyme necessary for dopamine synthesis. The gene therapy was generally well-tolerated, without any serious adverse events associated with the product or the procedure, in addition to a condition improvement from baseline

Date: Jun 10, 2019

Roche and Spark Therapeutics today announced that they have each received a request for additional information and documentary material from the U.S. Federal Trade Commission in connection with the FTC’s review of Roche’s pending acquisition of Spark. Issuance of the Second Request is part of the regulatory review process. The effect of the Second Request is to extend the waiting period until 10 days, but the parties remain committed to the transaction and are working cooperatively and expeditiously with the FTC in connection with its review

Date: Jun 10, 2019

Biogen has announced that it has completed its acquisition of Nightstar Therapeutics, a gene therapy company focused on adeno-associated virus treatments for inherited retinal disorders, for $800 millions. The takeover includes NSR-REP1 for the treatment of choroideremia, a rare and degenerative X-linked inherited retinal disorder, and NSR-RPGR, gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP), which is also a rare inherited retinal disease primarily affecting males with no approved treatments

Date: Jun 9, 2019

Enzyvant, biopharmaceutical company focused on developing and commercializing transformative therapies for patients with rare conditions, has announced that FDA has accepted for filing its Biologics License Application and to have obtained the Priority Review Status for RVT-802, novel investigational tissue-based regenerative therapy for the treatment of pediatric congenital athymia. The children affected by this rare condition are born without thymus, resulting in a severe immunodeficiency that leading to death during the first two years of life. The BLA includes clinical data that demonstrated long-term durability of treatment with this therapy, the Priority Review Status accelerates the FDA assessment

Date: Jun 9, 2019

The combination between rituximab, anti CD-20 monoclonal antibody directed against cancer cells, and IFN-DC, patient’s monocytes cultivated in presence of interferon alpha, leads to an antitumor immune response with disease remission in patients affected by relapsed or refractory lymphoma. These are the results of a phase 1 clinical study conducted by a group of researchers of National Institute of Health, Sant’Andrea Hospital of Rome and Oncology Referral Center of Aviano. The cells show strong antigen presentation capacity, with consequent T-lymphocytes activation against tumor cells; to use this capacity the IFN-DC are administrated after treatment with rituximab, monoclonal antibody that kills cancer cells causing antigens release

Date: Jun 7, 2019

Vertex Pharmaceuticals has announced its wish to acquire Exonics Therapeutics and to expand its collaboration with Crispr Therapeutics. The Companies are focused on development of gene therapies for the treatment of severe genetic neuromuscular diseases. Targets of Vertex are the treatments for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1

Date: Jun 7, 2019

Following the success of preclinical studies about gene therapy FTL190, Freeline Therapeutics announces the beginning of a phase 1/2 clinical trial called MARVEL1. FTL190 is a gene therapy that uses an adeno-associated virus as a vehicle to deliver a healthy copy of the GLA gene that could induce the production of normal alpha-GAL A. The study will take place in multiple centers in Europe and in the USA, and it will test a single dose of FLT190 in both untreated patients and those previously treated with either pharmacological chaperone therapy or enzyme replacement therapy, with the aim of evaluating  drug’s safety profile

Date: Jun 7, 2019

Enlivex Therapeutics, a clinical-stage immunotherapy company, has provided a clinical update on the positive safety profile of its off-the-shelf cell therapy Allocetra in the first three severe sepsis patients, as part of its ongoing Phase 1b clinical trial. All three patients tolerated the infusion without serious adverse events. The first such indication Enlivex is currently targeting is prevention of cytokine storms and organ dysfunction associated with sepsis

Date: Jun 6, 2019

Kiadis Pharma, a dutch biopharmaceutical company, announces that it has closed the acquisition of CytoSen Therapeutics; the transaction creates a leader in cell-based cancer immunotherapy with proprietary and synergistic NK-cell and T-cell therapy platforms that have the potential to revolutionize hematopoietic stem cell transplant and create a pipeline with novel cancer treatments. Kiadis has a complementary development pipeline focused on improving outcomes for patients undergoing hematopoietic stem cell transplants with a T-cell therapy (ATIR101; in EU registration and a global Phase 3 clinical trial) and a NK-cell therapy (CSTD002; expected to enter the clinic in the US in 2020)

Date: Jun 6, 2019

Flexion Therapeutics has announced that the U.S. Patent and Trademark Office has issued patent for FX201, gene therapy product for the treatment of osteoarthritis. The patent covers the composition of matter and method of use of FX201 in the treatment of the disease. The gene therapy product is designed to stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist, with the aim of suppressing inflammation to both reduce pain and modify the disease

Date: Jun 5, 2019

Allogene Therapeutics, biotechnology company pioneering the development of allogeneic CAR-T therapies for cancer, has announced that the FDA has cleared Investigational New Drug application for ALLO-715, allogeneic CAR-T therapy against B-Cell Maturation Antigene in patients with relapsed/refractory multiple myeloma. Multiple myeloma is the second most common hematological tumor in the USA, with over 30.000 new cases and over 10.000 deaths estimated in 2019

Date: Jun 4, 2019

The European commission has granted the conditional market authorisation for Zynteglo, the first transfusion-dependent β-thalassemia gene therapy. The drug, manufactured by bluebird bio, is indicated for the treatment of patients 12 years and older with transfusion dependent β-thalassaemia who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen matched related HSC donor is not available. The therapy has also obtained from the European commission the orphan designation for β-thalassemia intermedia and major

Date: Jun 4, 2019

A novel gene therapy clinical trial at University Hospitals Seidman Cancer Center and the Case Comprehensive Cancer Center shows promising results in terms of mean survival in patients with glioblastoma, the deadliest form of brain cancer and among the most difficult cancer to treat. The gene therapy was notably done with a new safety-modified lentiviral vector similar to those used for CAR T cell therapy. Results of the Phase 1 clinical trial will be shared on June 2 at the American Society of Clinical Oncology's annual meeting

Date: Jun 3, 2019
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