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.
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.
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.
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.
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.
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
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
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
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.
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
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
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
The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader regenerative medicine sector, released a Therapeutic Developers’ Statement of Principles, setting forth a bioethical framework for the use of gene editing in therapeutic applications; the statement, developed by ARM’s Gene Editing Task Force and signed by 13 therapeutic developers using gene editing technologies, specifies five key principles for the ethical use of gene editing and genetic modification
DiscGenics, a biotechnology company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine, announced that the FDA has granted Fast Track designation for its investigational cell therapy, IDCT, currently being evaluated in regulator-allowed clinical trials in the USA and Japan for the reduction in pain and disability associated with degenerative disc disease. IDCT is a homologous, allogeneic, injectable cell therapy that utilizes biomedically engineered progenitor cells, known as discogenic cells, that have been derived from intervertebral disc tissue to offer a non-invasive, potentially regenerative solution for the treatment of mild to moderate degenerative disc disease
Ziopharm Oncology, announced the EMA Committee for Orphan Medicinal Products adopted a positive opinion recommending Ad-RTS-hIL-12 plus veledimex for designation as an orphan medicinal product for the treatment of glioma. The medicinal product is an investigational gene therapy designed to induce and control the production of human interleukin 12, a master-regulator of the immune system
Con l’approvazione da parte del Consiglio di amministrazione di AIFA alla rimborsabilità della prima terapia a base di cellule CAR-T disponibile in Italia, si è concluso l’iter procedurale per “garantire l’accesso a queste nuove terapie salvavita. Kymriah è indicato per pazienti adulti con linfoma diffuso a grandi cellule B resistenti alle altre terapie o nei quali la malattia sia ricomparsa dopo una risposta ai trattamenti standard, e per pazienti fino a 25 anni di età con leucemia linfoblastica acuta a cellule B. Kymriah è stato approvato attraverso un innovativo modello di rimborso usato per la prima volta in Italia, il payment at results
Mustang Bio, a biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, announced that the FDA has approved the Company’s Investigational New Drug application to initiate a multi-center Phase 1/2 clinical trial of MB-102 in acute myeloid leukemia, blastic plasmacytoid dendritic cell neoplasm and high-risk myelodysplastic syndrome
AIFA and Novartis reached the agreement for Kymriah, CAR-T designed for the treatment of acute lymphoblastic leukemia and diffuse large B-cell lymphoma; based on payment by result model, this agreement can be a point of reference in Europe. The total price of therapy will be around €300.000 per patient, but the real innovation is that the National Health Services will pay on effective results; the scheme involves the payment for the first infusion bag, while the further tranches (6 and 12 months) will be payed only if there is the remission of disease
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
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
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
Personalized Stem Cells, a human adipose-derived stem cell company, has received FDA approval for their Investigational New Drug application to use a person’s own adipose-derived stem cells to treat their osteoarthritis; the first clinical trial, expected to begin in August, uses stem cells as a treatment of osteoarthritis in the knee. PSC plans to conduct a series of FDA approved clinical trials starting with uses in orthopedics and expanding to other medical conditions in the future
Apic Bio, an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, announced that the FDA has granted orphan drug designation to APB-102, a gene therapy soon to be in clinical development for the treatment of genetic SOD1 amyotrophic lateral sclerosis. The incidence of ALS is estimated to be 1.5 to 2.5 cases in 100.000 people in the United States and in Europe, of which 10% are thought to be inherited, and approximately 15% to 20% of these cases are caused by mutations in the gene that produces the Cu-Zn superoxide dismutase 1 enzyme (SOD1)
Caladrius Biosciences, a late-stage therapeutics development biopharmaceutical company pioneering advancements of cell therapies in select cardiovascular and autoimmune diseases, announced that the EMA has granted Advanced Therapy Medicinal Product classification to the Company’s CD34+ cell therapy product, CLBS12, for the treatment of Critical Limb Ischemia. CLI is a result of severe obstruction of the arteries that markedly reduces blood flow to the extremities and can lead to pain, skin ulcers and dermal sores and even amputation
BioMarin Pharmaceutical plans on submitting its hemophilia A gene therapy to U.S. and European regulators sometime in the fourth quarter, which could bring approval decisions as early as mid-2020. In support of the submissions for valrox are an ongoing Phase 1/2 study as well as an interim analysis of a Phase 3 investigation, and the Company said the results represent a 96% reduction in patients' mean annualized bleed rates
Prevail Therapeutics, a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, announced that the FDA has granted Fast Track Designation for the Company’s lead gene therapy program, PR001, for the treatment of Parkinson’s disease patients with a GBA1 mutation. PR001 is a potentially disease-modifying, single-dose, AAV9-based gene therapy being developed for the treatment of Parkinson Disease-GBA and neuronopathic Gaucher disease
NantKwest, a clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer cells to treat cancer, infectious diseases and other diseases, announced that the company’s PD-L1 t-haNK investigational new drug application has cleared FDA review and the program has now transitioned to a first-in-human clinical trial in patients with locally advanced or metastatic solid cancers. Programmed death-ligand 1 (PD-L1) is a transmembrane protein that plays a major role in suppressing the immune system in many cancer patients
Mesoblast Limited, global leader in cellular medicines for inflammatory diseases, announced that the FDA has granted the company’s product candidate, rexlemestrocel-L, Orphan Drug Designation for prevention of post-implantation mucosal bleeding in end-stage chronic heart failure patients who require a left ventricular assist device. The therapy is also being evaluated in patients with New York Heart Association Class II/III moderate to advanced heart failure across multiple sites in North America. Mesoblast’s investigational cell therapy consists of 150 million allogeneic mesenchymal precursor cells and is being developed for injection into heart muscle of patients
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Mar 31- Apr 2 , 2020