23-29 Sep, 2019
23-26 Oct, 2019
May 12-15, 2021
bluebird bio announced its plan to separate its severe genetic disease and oncology businesses into differentiated and independent publicly traded companies. bluebird bio will retain focus on severe genetic disease and will launch its oncology business as a new entity. bluebird bio said that once the split has been finalized by year-end 2021, it will focus on therapies for beta thalassemia, cerebral adrenoleukodystrophy and sickle cell disease in the US and Europe, including expanding access and reimbursement for Zynteglo (betibeglogene autotemcel) in Europe. Moreover, bluebird will increase addressable patient populations through geographic expansion, label expansions, and product profile enhancement. Oncology newco will support commercial success of investigational BCMA CAR T cell therapy, idecabtagene vicleucel (ide-cel) in multiple myeloma and continued development of investigational bb21217 product candidate. Moreover, the newco will develop other cellular therapies with a focus on non-Hodgkin’s lymphoma, acute myeloid leukemia, next-generation multiple myeloma and solid tumors.
Luxturna, Novartis & Spark Therapeutics gene therapy, obtains reimbursability as published in the Italian Gazzetta Ufficiale n.6 09-01-2021 following AIFA decision. Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
The European Commission granted full (standard) market authorization for the world's first gene therapy for the treatment of a rare and very serious neurodegenerative disease metachromatic leukodystrophy. Libmeldy is the result of the strategic alliance between the Telethon Foundation, IRCCS San Raffaele Hospital and the biotech company Orchard Therapeutics, through more than 15 years of studies supported by Fondazione Telethon and Ospedale San Raffaele. MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk and most die before adolescence. Libmeldy is designed as a one-time therapy in children with late infantile or early juvenile forms, without clinical manifestations of the disease, or the early juvenile form, with early clinical manifestations of the disease, who still can walk independently and before the onset of cognitive decline. Libmeldy will also be available in the cryo-preserved formulation to increase access to therapy. This procedure involves freezing lentiviral vector engineered patient's cells.
European Commission has now granted conditional marketing authorization for CAR-T Tecartus, drug developed by Kite, now a subsidiary of Gilead Sciences committed to cell therapy for cancer treatment. Tevartus is first cell therapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma, following at least two lines of systemic therapy including a BTK inhibitor. Conditional authorization is granted in the interest of public health where the benefit of immediate availability outweighs the risk of less comprehensive data available. Multinational, single-arm, Phase II open-label ZUMA-2 pivotal trial demonstrated an overall response rate (complete or partial) of 93%, with 67% of patients achieving a complete response, following a single infusion of Tecartus.
Bayer and Atara Biotherapeutics announced an exclusive worldwide license agreement and research, development, and manufacturing collaboration for mesothelin-directed CAR T-cell therapies for the treatment of solid tumours, such as malignant pleural mesothelioma and non-small-cell lung cancer. The agreement includes the ongoing autologous CAR-T ATA2271 phase 1 study and its off-the-shelf allogenic version, ATA3271, which offers major flexibility for patients and lower manufacturing cost.
The University of Sheffield and Pfizer have launched a new, five-year, consortium: Accelerating Research and Innovation for Advanced Therapies. ARDAT is supported by the Innovative Medicines Initiative (IMI) and brings together the leading expertise of 34 academic, nonprofit and private organisations from across Europe and the US. Consortium will help to fill the knowledge gaps in how advanced therapies could potentially work, and to develop appropriate standards to aid researchers, developers and regulators in accelerating effective and safe gene and cell therapies to benefit patients. The field of ATMP research is expected to grow exponentially in the coming years, with potentially up to 10-20 new drug applications submitted per year to the FDA by 2025.
AIFA included Zolgensma in the list of drugs that can be dispensed entirely by NHS in full under Law 648/96- Zolgesma is indicated for the treatment within the first six months of life of patients with genetic diagnosis (bi-allele mutation in the SMN1 gene and up to 2 copies of the SMN2 gene) or clinical diagnosis of spinal muscular atrophy (SMA) type 1.
Zolgensma (onasemnogene abeparvovec) is the first gene therapy for SMA and it represents a turning point for the treatment of the disease. Onasemnogene abeparvovec is, in fact, designed to address the genetic cause of the disease; it replaces the function of the missing or non-functioning gene SMN1 and it is intravenously administered only once in the patient's life.
Funded by the Lombardy Region with three million €, the project "Plagencell - A network for cell and gene therapies for devastating diseases" was born for the creation of a network of five cell factories specialized in gene and cell therapies promoted by Fondazione Tettamanti, San Gerardo Hospital in Monza, San Raffaele Hospital in Milan, Policlinico San Matteo in Pavia, Policlinico di Milano , Papa Giovanni XXIII Hospital in Bergamo. Four working groups are each dedicated to a specific therapeutical area (oncohematology, neurology and neurodegenerative diseases, nephrology and organ transplantation, and oncology) and one to the development of technologies.
The aim of the oncoematological research group is the development of genetically reprogrammed T-cell banks from cells obtained from umbilical cord blood and ready to be used when needed.
EMA CHMP recommended the gene therapy Libmeldy for the treatment of metachromatic leukodystrophy (MLD), a rare hereditary metabolic disease. Nowadays there is no therapy available for MLD, which affects the nervous system, and it is caused by a fault in the ARSA gene. Most children affected by the disease lose the ability to walk or to talk within a few months of the first symptoms onset, and more than 75% of them die within five years. Libmeldy is a gene therapy; autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells are transduced ex vivo into the patient, who is able to produce the enzyme ARSA.
GenSight Biologics, a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that it has submitted the Marketing Authorisation Application (MAA) for its lead product LUMEVOQ® (Lenadogene nolparvovec) to the European Medicines Agency (EMA), seeking approval for the treatment of patients with vision loss due to Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. Lenadogene nolparvovec is a recombinant adeno-associated viral vector, serotype 2 (rAAV2/2), containing a cDNA encoding the human wild-type mitochondrial NADH dehydrogenase 4 protein (ND4), which has been specifically developed for the treatment of LHON associated with mutation in the ND4 gene. It received orphan drug designation status for the treatment of LHON from the EMA in 2011 and from the U.S. Food and Drug Administration (FDA) in 2013.
Axovant Gene Therapies , a clinical-stage company developing innovative gene therapies for neurological diseases, today announced that it has signed a strategic partnership with Viralgen, a leading Contract Development and Manufacturing Organization (CDMO). Leveraging the technology platforms of AskBio, Viralgen is able to support all aspects of manufacturing for Axovant’s AAV programs, including large-scale manufacturing, fill-finish, and quality control in a GMP-certified environment custom-designed to bring therapies to market as quickly as possible. Under the terms of the partnership, Axovant will have access to manufacturing resources for Axovant’s AAV-based gene therapy programs, AXO-AAV-GM1 for GM1 gangliosidosis and AXO-AAV-GM2 for GM2 gangliosidosis (also known as Tay-Sachs/Sandhoff disease) with sufficient capacity to support ongoing development and eventual commercialization.
The change to Novartis Gene Therapies is the natural evolution as the company scales up to deliver Zolgensma globally and expand its reach via a robust pipeline of AAV-based gene therapies for rare genetic diseases including investigational treatments for Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Instead of alternating between the AveXis and Novartis brands by market, the company comes together under one banner as a unified entity.
Triumvira Immunologics successfully completed a USD $55 million Series A financing round. The financing was co-led by Leaps by Bayer, the impact investment unit of Bayer AG, and Northpond Ventures. Triumvira’s foundational technology is the T-cell Antigen Coupler (TAC). TAC is a hybrid molecule comprising multiple protein domains to combine tumor targeting abilities with the T-cell’s own activation machinery, leveraging the potential for the development of superior therapies for a broader range of patients with solid or liquid malignancies. Triumvira’s preclinical data with autologous and allogeneic programs demonstrate unique biological differences between TAC-engineered T-cells and second-generation CAR-T cells, with TAC-T cells showing absence of tonic signaling, strong tumor penetration, and long-term persistence. These functional properties help TAC T-cells produce strong anti-tumor activity and no evidence of toxicity, particularly in models of solid tumors
4D Molecular Therapeutics (4DMT) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to 4D-310 for Fabry disease, a debilitating lysosomal storage disorder caused by a deficiency of alpha-galactosidase A (AGA) enzyme activity. 4D-310 is a gene medicine engineered with a proprietary optimized AAV capsid discovered by 4DMT through its proprietary Therapeutic Vector Evolution platform.
Neurogene announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to adeno-associated virus vector with engineered transgene encoding the human CLN7 gene for patients with CLN7, a form of Batten disease. Batten disease, a common name for a rare class of diseases called neuronal ceroid lipofuscinoses (NCLs), affects an estimated 2-4 out of every 100,000 children in the United States. In July, the company announced it had received Orphan Drug Designation from the FDA for its gene therapy for the treatment of CLN5 Batten disease.
Orchard Therapeutics, a global gene therapy leader, today announced that the company has entered into two worldwide royalty-bearing license agreements with GlaxoSmithKline plc (GSK) for use of their proprietary lentiviral stable cell line technology (LV-SCLT) for Orchard’s investigational hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome (OTL-103 for WAS) and transfusion-dependent beta thalassemia (OTL-300 for TDT).
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).
The Italian government has decided to exercise Golden power over Molmed, the Italian biotech company whose main shareholder is the Berlusconi family's Fininvest, thus putting at risk the Opa (public tender offer) - which is on the market until July 24 - by the Japanese company Agc and its Italian subsidiary. As a result of the executive's desire to exercise special powers over transactions with the Japanese, the Italian biotech company lost about 5.4% of its shares on the stock exchange. However, the Japanese company could still accept the conditions and still proceed with the acquisition.
AVROBIO, a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the first patient has been dosed in the company’s GuardOne clinical trial, a Phase 1/2 investigational study evaluating AVR-RD-02 for Gaucher disease type 1. The company also announced that the second patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.
Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases, today announced an agreement with Hansa Biopharma, the leader in immunomodulatory enzyme technology for rare Immunoglobulin G (IgG) mediated diseases, for imlifidase. Under the terms of the agreement, Sarepta obtains an exclusive, worldwide license to develop and promote imlifidase as a pre-treatment to enable Sarepta gene therapy administration in Duchenne muscular dystrophy (DMD) and Limb-girdle muscular dystrophy (LGMD), for patients who may otherwise not be eligible for treatment.
The first clinical trial of a regulatory T-cell therapy enhanced with a chimeric antigen receptor is planned for 2020. Preclinical studies are promising and potential applications wide, but there is still a long way to go (although an off-the-shelf version is already being considered).
Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases, and Codiak BioSciences, a company at the forefront of advancing engineered exosomes as a new class of biologic medicines, today announced a global research and option agreement to design and develop engineered exosome therapeutics to deliver gene therapy, gene editing and RNA technologies for neuromuscular diseases.
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.
CSL Behring announced today that it has agreed to acquire exclusive global license rights to commercialize an adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), for the treatment of hemophilia B from uniQure (NASDAQ: QURE), a leading gene therapy company. The AMT 061 program, currently in Phase 3 clinical trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B.