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

Tiziana Life Sciences, a biotechnology company focused on innovative therapeutics for oncology, inflammation and infectious diseases, is pleased to announce that it has submitted a patent application on potential use of Foralumab, a fully human anti-CD3 monoclonal antibody (mAb), to improve success of CAR-T therapy for cancer and other human diseases. The patent application conveys inventions related to improving CAR-T expansion and/or survival using anti-CD-3 mAbs administered either alone or in combination with other co-stimulatory molecules, such as an anti-IL-6 receptor monoclonal antibody, an anti-CD28 monoclonal antibody or specific inhibitors of signaling pathways of phosphatidylinositol 3-kinase (PI3K), protein kinase B (AKT), or mammalian target of rapamycin (mTOR).

Date: Jul 16, 2020

Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 euros, and says it hopes to reach a “smart deal” allowing UK patients access by the end of the year.

Date: Jun 24, 2020

Kite, a Gilead Company, today announced it has received approval to implement a variation to the Yescarta® (axicabtagene ciloleucel) Marketing Authorization from the European Medicine Agency (EMA) for end-to-end manufacturing. The new European facility sits next to one of Europe’s largest airports, Amsterdam Airport Schiphol. This central location, with its transport links to the region, will reduce the delivery time to and from treatment centers. The facility has the capacity to produce therapy for up to 4,000 patients per year.

Date: Jun 12, 2020

AveXis, a Novartis company, today announced the European Commission (EC) granted conditional approval for Zolgensma® (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a biallelic mutation in the SMN1 gene and up to three copies of the SMN2 gene. The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.

Date: May 21, 2020

Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announces that the European Commission (EC) has granted orphan drug designation for FLT190 for the treatment of Fabry Disease, based on a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA).

Date: Mar 27, 2020

AVROBIO, a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s investigational gene therapy, AVR-RD-04, for the treatment of cystinosis. The gene therapy consists of the patient’s own hematopoietic stem cells, which are genetically modified to express cystinosin, the protein that is functionally deficient in people with cystinosis.

Date: Mar 9, 2020

MeiraGTx, clinical stage gene therapy company, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Advanced Therapy Medicinal Product (ATMP) designations to AAV-RPGR, investigational gene therapy jointly developed by MeiraGTx and Janssen Pharmaceuticals for the treatment of x-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. PRIME designation was granted based on clinical data from MeiraGTx and Janssen’s ongoing Phase 1/2 trial of AAV-RPGR in patients with XLRP (NCT03252847). AAV-RPGR is the only gene therapy in development for the treatment of XLRP to receive PRIME designation.

Date: Mar 2, 2020

Tessa Therapeutics (Tessa), a clinical-stage cell therapy company developing next-generation cancer treatments, today announced that the Company’s investigational CD30-directed autologous chimeric antigen receptor T cell (CD30 CAR-T) therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with relapsed or refractory CD30- positive classical Hodgkin lymphoma (cHL). Tessa expects to initiate its pivotal Phase II multi-site trial in the fourth quarter of 2020.

Date: Feb 27, 2020

Lysogene, a pioneering Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its LYS-SAF302 program for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA). LYS-SAF302, a second-generation gene therapy, is designed to deliver a functional copy of the SGSH (N-sulfoglucosamine sulfohydrolase) gene to the brain through a one-time direct-to-CNS administration, and is being investigated in the international Phase 2/3 clinical trial AAVance (NCT03612869).

Date: Feb 25, 2020

BMS announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review its Biologics License Application (BLA) for lisocabtagene maraleucel (liso-cel), the company’s autologous anti-CD19 chimeric antigen receptor (CAR) T-cell immunotherapy with a defined composition of purified CD8+ and CD4+ CAR T cells for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma after at least two prior therapies. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of August 17, 2020. The BLA, submitted by Juno Therapeutics, a wholly owned subsidiary of Bristol-Myers Squibb Company, is based on the safety and efficacy results from the TRANSCEND NHL 001 trial, evaluating liso-cel in 268 patients with R/R large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL), high-grade lymphoma, primary mediastinal B-cell lymphoma and Grade 3B follicular lymphoma

Date: Feb 17, 2020

Calibr, the drug discovery and development division of Scripps Research, today announced that the U.S. Food and Drug Administration has given clearance to the Investigational New Drug (IND) application for Calibr's "switchable" CAR-T cell therapy, which is being evaluated for the treatment of certain cancers, including relapsed/refractory B-cell malignancies such as non-Hodgkin lymphoma and chronic lymphocytic leukemia. Having achieved this regulatory milestone, Calibr can begin clinical trials for its novel cell therapy candidate, CLBR001 + SWI019. The therapy leverages a patient's own immune cells to treat cancer, putting them under the control of a novel molecular "switch" that seeks to eliminate life-threatening side effects that have hampered the use of cell therapies to date.

Date: Feb 3, 2020

The EMA has given the therapy priority medicine designation for KTE-X19, second CAR-T from Gilead Sciences’ Kite Pharma subsidiary, allowing for an expedited review of the dossier and setting up a possible approval before year-end. Kite has filed for approval of the new CAR-T in mantle cell lymphoma (MCL) on the strength of impressive data from the ZUMA-2 trial – reported at last year’s American Society of Haematology (ASH) meeting – patients in relapsed or refractory MCL. If approved, KTE-X19 will become the first CAR-T therapy for this indication, according to Kite, which also submitted it to the FDA last month. The therapy has also been granted breakthrough status by the US agency, allowing for a potential six-month review of data.

Date: Jan 28, 2020

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.

Date: Jan 22, 2020

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.

Date: Jan 21, 2020

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.

Date: Jan 16, 2020

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.

Date: Jan 16, 2020

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.

Date: Jan 13, 2020

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

Date: Sep 18, 2019

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

Date: Sep 16, 2019

Adaptimmune Therapeutics, a leader in T-cell therapy to treat cancer, announced that the US FDA has granted Orphan Drug Designation to SPEAR T-cells targeting MAGE-A4 (Adaptimmune’s ADP-A2M4 program) for the treatment of soft tissue sarcomas. Orphan Designation by FDA was created to encourage the development of drugs for rare diseases, such as sarcomas

Date: Sep 9, 2019

Orchard Therapeutics said it planned to file data from its OTL-200, gene therapy for the life-threatening neurodegenerative rare disease metachromatic leukodystrophy, with regulators early next year in Europe after results from a late-stage trial showed clinical benefit on cognitive and motor function; OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy, purchased by Orchard from GlaxoSmithKline in 2018; it originated from a collaboration between GSK and San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

Date: Sep 4, 2019

The FDA has granted Orphan Drug Designation to CARsgen Therapeutics’ investigational CAR-T cell therapy fully human anti-BCMA (B Cell Maturation Antigen) autologous CAR-T cells, CT053, for the treatment of multiple myeloma; under the Orphan Drug Act, the CT053 anti-BCMA product would be eligible for certain benefits including seven years of market exclusivity in the United States following marketing approval by the FDA. CT053 has demonstrated outstanding potency in an exploratory Phase I clinical study; a total of 19 of 24 patients with relapsed and refractory multiple myeloma showed complete response, without severe adverse event, like grade 3 or higher Cytokine Release Syndrome

Date: Sep 2, 2019

Tedros Adhanom Ghebreyesus, Director General of World Health Organization, announced the estabilishment of a global registry that keep track of all gene editing research in the world; the Director also highlighted that the various Countries shoud not allow clinical trial based on gene editing of human germ cells, til technical and ethical implications will not be properly considered

Date: Aug 30, 2019

The FDA has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A; the treatment candidate consists of human cells modified to incorporate large amounts of synthetic DNA-encoding therapeutic proteins into cells, specifically human FVIII. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application fees

Date: Aug 28, 2019
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