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News

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

 GenSight is focused on finding treatments for patients with neurodegenerative diseases and is developing products that are a combination of gene therapy, its mitochondrial targeting sequence technology, and optogenetics. The therapies being developed by GenSight are gene-therapy based and designed to be administered to the eye via intravitreal injection; patients are expected to receive a long-lasting functional cure, possibly lasting the rest of their lives, and one such treatment is for Leber Hereditary Optic Neuropathy. The LHON causes the sudden loss of central vision in one eye, and is caused by a genetic mutation, inherited at birth, which impacts retinal ganglion cells

Date: Aug 1, 2019

Source: https://www.clinicalleader.com

Allogene Therapeutics is launching the first clinical trial testing ALLO-715, its CAR T-cell therapy, for people with multiple myeloma who failed prior therapies; this CAR T-cell product meant to recognize and eliminate cancer cells containing the B-cell mature antigen (BCMA)

Date: Jul 31, 2019

Source: https://myelomaresearchnews.com

In a paper published in the July 31 issue of Science Translational Medicine, researchers at Fred Hutchinson Cancer Research Center used CRISPR-Cas9 to edit blood stem cells to reverse the clinical symptoms observed with several blood disorders, including sickle cell disease and beta-thalassemia; the study suggests that efficient modification of targeted stem cells could reduce the costs of gene-editing treatments for blood disorders and other diseases while decreasing the risks of unwanted effects

Date: Jul 31, 2019

Source: https://www.sciencedaily.com

RegenxBio, a leader Company in adeno-associated virus gene therapy based in Rockville, announced it entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia; under the terms of the deal, RegenxBio granted Pfizer a non-exclusive global license RegenxBio’s NAV AAV9 vector, and in return, Pfizer is paying RegenxBio an upfront payment, various milestones and royalties on net sales. Patients with Friedreich’s ataxia have a genetic mutation in the FXN gene, and this limits the frataxin protein expression, which leads to various debilitating symptoms including coordination and balance problems, muscle weakness, impaired vision, hearing and speech, scoliosis, diabetes, and cardiomyopathy

Date: Jul 31, 2019

Source: https://www.biospace.com

Celgene plans to file for FDA approval of bb2121 (idecabtagene vicleucel) in multiple myeloma in the first half of 2020, and lisocabtagene maraleucel in lymphoma in the fourth quarter of this year. Notably, the high efficacy rates of liso-cel came without as much of the toxicity that has characterized CAR-T therapies: Cytokine Release Syndrome occurred in only 30% of patients, with only one case of life-threatening CRS, and the neurotoxicity occurred in 20% of patients, with 14% experiencing serious or life-threatening events

Date: Jul 30, 2019

Source: https://medcitynews.com

Francesca Pasinelli, General Manager of Telethon Foundation, explains the advanced therapies state-of-art in Europe, within the recent report of Alliance for Regenerative Medicine, group including research centers, scientific societies, funding bodies, pharmaceutical and biotech Companies; among the ARM recommendations to improve the ATMP access process efficiency, are highlighted some useful methodologies already involved in Italy, such as the risk sharing agreements

Date: Jul 30, 2019

Source: https://www.agi.it

The researchers of Children’s Hospital of Philadelphia report a more sensitive method for capturing the footprint of adeno-associated virus, used as viral vector in gene therapy, improve the DNA transfer site detection capability. The new AAV screening method uses sensitive editing-reporter transgenic mice that are marked; in side-by-side comparisons with conventional screening methods, this method radically redefines the true extent of AAV-mediated gene transfer

Date: Jul 30, 2019

Source: https://www.sciencedaily.com

Despite the success of CAR-T therapies for B-cell cancers and multiple myeloma, developing this therapies for solid tumors has posed more of a challenge; failure of the CAR-T cells to multiply to high enough levels, the tumor microenvironment being immunosuppressive, and tumor cells ‘escaping’ the therapy by losing target antigen expression are just some of the obstacles solid tumors pose. The new frontier is the possibility to use small-molecules approved by competent authorities to enhance the CAR-T activity against tumors, and several Companies are engaged for this purpose

Date: Jul 29, 2019

Source: https://www.biospace.com

BioMarin Pharmaceuticals is planning to submit marketing authorization applications to the FDA and the EMA seeking approval of valoctocogene roxaparvovec, its investigational gene therapy for the treatment of adults with hemophilia A, before the end of the year; the planned marketing authorization submissions are based on the latest findings from a Phase 1/2 trial, and on data from an interim analysis of the ongoing Phase 3 trial. The investigational treatment has received breakthrough therapy designation from the FDA, and priority medicines, or PRIME designation from the EMA; it also has orphan drug status from both regulatory agencies

Date: Jul 29, 2019

Source: https://hemophilianewstoday.com

Researchers at King's College London have used single cell RNA sequencing to identify a type of stem cell that may be able to regenerate liver tissue, treating liver failure without the need for transplants. This cell, called hepatobiliary hybrid progenitor or HHyP, persist in small quantities in adults and they can grow into the two main cell types of the adult liver (hepatocytes and cholangiocytes); the study was published on Nature Communications

Date: Jul 29, 2019

Source: https://sciencecue.it

Orchard Therapeutics, a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, announced that it has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for OTL-103, Orchard’s autologous hematopoietic stem cell-based gene therapy for the treatment of Wiskott-Aldrich Syndrome, developed at the San Raffaele-Telethon Institute for Gene Therapy in Milan; RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene therapies

Date: Jul 29, 2019

Source: https://finance.yahoo.com

An experimental cell therapy where a patient’s own skin cells are engineered in the lab to carry a healthy COL7A1 gene and then injected back into the patient is safe and potentially efficacious for treatment of Recessive Dystrophic Epidermolysis Bullosa, a new study shows; RDEB is one of the most severe forms of inherited skin fragility disease and is characterized by painful blisters and erosions, leading to skin infection and scarring during early adulthood, as well as other complications, and it is caused by mutations in the COL7A1 gene, which provides instructions to make type VII collagen. The study was published on JCI Insight

Date: Jul 26, 2019

Source: https://epidermolysisbullosanews.com

A Phase 1 clinical trial has been launched to assess the effects of NantKwest’s investigational PD-L1 t-haNK cell therapy in patients with locally advanced or metastatic solid cancers; PD-L1 t-haNK is a new type of immuno-oncology therapy based on the use of Natural Killer immune cells that have been genetically modified to recognize and destroy cancer cells from solid tumors

Date: Jul 26, 2019

Source: https://immuno-oncologynews.com

People with a rare form of inherited blindness are being enrolled for the world’s first in vivo study of a CRISPR-based therapy; the therapy, developed by Editas Medicine and Allergan, removes a deleterious mutation of CEP290 gene from cells in patients’ retinas, and will be tested in 18 adults and children later this year. This particular mutation leads to abnormal splicing and dysfunctional production of the CEP290 protein, which plays an important role in the photoreceptors in the eye

Date: Jul 26, 2019

A study of Massachusetts General Hospital Cancer Center, published in the journal Nature Biotechnology, shows a novel CAR-T approach for the treatment of glioblastoma; the selected target for the thearpy is epidermal growth factor receptor variant III, a mutated cancer-causing protein that is present on the surface of many but not all glioblastomas; to boost the effectiveness of CAR T cells, the researchers decided to target a second antigen, the wild type of EGFR, present in many type of cells, with high possibility of severe adverse effects. To overcome this problem, they crafted a CAR T cell that can be delivered into the cerebrospinal fluid, able to secrete a second type of immunotherapy, the bi-specific T-cell engager or BiTE, that direct T-cells to the target. The BiTE is too large to cross the blood-brain barrier, decreasing the possibility of toxic effects against organs other than brain

Date: Jul 25, 2019

Gene therapy trials are underway to improve the vision of patients with achromatopsia, a rare inherited retinal disease that affects approximately one in 30.000 individuals; it is a stable cone dystrophy causing severe hemeralopia, or day blindness, a condition in which any bright light reduces patients’ ability to see

Date: Jul 25, 2019

Abeona Therapeutics, a gene and cell therapy leader Company, announced positive data from its ongoing Phase 1/2 clinical trial evaluating ABO-102, the Company’s investigational one-time, adeno-associated virus gene therapy for Mucopolysaccharidosis type IIIA; these new results showed that treatment of the youngest patients with ABO-102 resulted in preservation of neurocognitive development 12-18 months post treatment. Furthermore long-term safety profile remained favorable eight months to two years after administration

Categoria Clinical

Date: Jul 25, 2019

Researchers of China’s Central South University have produced induced pluripotent stem cells (iPS) from patient’s somatic cells, and then induced the cellular differentiation to obtain retinal pigment epithelium cells, the specialized cells composing the retina, in the back of the eye; the study, published on Acta Biomaterialia, suggests that the iPS could form an ultra-thin layer with a similar structure to the retina

Date: Jul 25, 2019

A phase I clinical trial from Mayo Clinic, published on The Journal of Thoracic and Cardiovascular Surgery, is the first research monitored by the FDA that demonstrates the potential of regenerative therapy for Hypoplastic Left Heart Syndrome with infant's own stem cells directly administered into the heart at the time of surgery; the stem cells originate from the cord blood, then this blood was sent to Mayo Clinic for processing and storage and then again to the hospital for re-injection. This syndrome affects approximately 1.000 infants in the USA each year

Date: Jul 24, 2019

Mustang Bio, a biopharmaceutical company focused on developing cell and gene therapies for the treatment of hematologic cancers, solid tumors and rare genetic diseases, announced that the FDA has granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of acute myeloid leukemia. The FDA also previously granted Orphan Drug Designation to the drug for the treatment of blastic plasmacytoid dendritic cell neoplasm

Date: Jul 24, 2019
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