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.
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.
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.
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
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).
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.
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.
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.
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.
Rexgenero, a regenerative medicine company developing advanced cell therapies to treat chronic limb-threatening ischaemia (CLI), today announces the acquisition of all the key assets of aratinga.bio SAS Group, a preclinical-stage immunotherapy biotech company based in Paris, France.The acquisition includes all patent applications and related intellectual property, the exclusive license agreement for polymeric nanoparticle technology and the bioproduction facilities of aratinga.bio SAS Group, as well as the entire aratinga.bio team.
The landscape of startups expands with Empirica Therapeutics, created by a group of researchers from McMaster University and the University of Toronto, both in Canada. The researchers founded it on the encouraging results of a new Car-T therapy, which they developed for glioblastoma, an highly aggressive brain tumor. The study, published in Cell Stem Cell, reported that the drug helped to reduce the tumor burden and improved survival in mouse models. The Canadian startup now aims to bring Car-T therapy into clinical phase, on patients with recurrent glioblastoma, by 2022.
Seattle Children’s Research Institute, one of the top pediatric research institutions in the world, and global biotechnology leader CSL Behring announced a strategic alliance to develop stem cell gene therapies for primary immunodeficiency diseases. Initially, the alliance will focus on the development of treatment options for patients with two rare, life-threatening primary immunodeficiency diseases -- Wiskott-Aldrich Syndrome and X-linked Agammaglobulinemia. CSL Behring researchers, working with researchers from Seattle Children’s Research Institute, will investigate applying the proprietary platform technologies, Select+™ and Cytegrity™, to several pre-clinical gene therapy programs. These technologies, which have broad applications in ex vivo stem cell gene therapy, are designed to address some of the major challenges associated with the commercialization of stem cell therapy, including the ability to manufacture consistent, high-quality products, and to improve engraftment, efficacy and tolerability.
Novasep, a leading supplier of services and technologies for the life sciences industry, and Lysogene, a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced the signature of an agreement for the development and manufacturing of LYS-GM101, an AAVrh10-based gene therapy drug candidate for the treatment of GM1 Gangliosidosis, a rare neuronopathic lysosomal storage disorder
The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), one of the world’s leading gene therapy research institutes jointly managed by Fondazione Telethon and Ospedale San Raffaele and Genespire, a gene therapy company developing transformative therapies for genetic diseases, and spin-out of SR-Tiget, announced today their alliance on the research and development of candidate therapeutic products for people affected by primary immunodeficiencies and metabolic diseases based on novel gene editing and lentiviral vector technologies developed by SR-Tiget.
Santhera Pharmaceuticals announces the signing of two agreements with Rutgers, The State University of New Jersey as part of its program to advance gene therapy research for the treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2 MD or MDC1A).
BioMarin Pharmaceutical today announced that the company has entered into a preclinical collaboration and license agreement with DiNAQOR AG (DiNAQOR), a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. The license initially covers DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM).
Codexis, Inc., a leading protein engineering company and developer of novel biotherapeutics, announces the signing of a strategic collaboration and license agreement with Takeda Pharmaceutical Company Limited (Takeda) for the research and development of novel gene therapies for certain disease indications, including the treatment of lysosomal storage disorders and blood factor deficiencies. Under the terms of the agreement, Codexis will generate novel gene sequences encoding protein variants tailored to enhance efficacy as a result of increased activity, stability, and cellular uptake using its CodeEvolver® protein engineering platform. Takeda will combine these improved transgenes with its gene therapy capabilities to generate novel candidates for the treatment of rare genetic disorders.
Asklepios BioPharmaceutical, Inc. (AskBio), a leading clinical-stage adeno-associated virus (AAV) gene therapy company, today announced that it has entered into a research collaboration and licensing agreement with the University of North Carolina at Chapel Hill (UNC) for the development and commercialization of gene therapy for Angelman syndrome. Collaboration builds upon preclinical research that demonstrates potential for gene therapy to correct deficiency in UBE3A gene.
MolMed, a biotechnology company focused on research, development, production and clinical validation of gene and cell therapies for the treatment of cancer and rare diseases, announces the execution of a development and supply agreement with Autolus Therapeutics, a clinical-stage biopharmaceutical company developing next-generation programmed T celltherapies for the treatment of cancer. Under the multi-year agreement, MolMed will develop and supply viral vectors for the manufacture of certain Autolus’ CAR-T programs for use in clinical trials and potentially for commercial sale.
GE Healthcare Life Sciences and Children’s Medical Research Institute will jointly drive the development of new affinity ligands for the purification of adeno-associated viral (AAV) vectors used in gene therapies. The focus of the collaboration is to bring to market-specific ligands for multiple AAV types, enhancing the chromatographic separation of AAV-based vectors. This will improve the manufacturing efficiency and scalability of gene therapies, enabling the availability of viral vectors on a global scale.
Avacta Group PLC on Monday said it has signed a collaboration and licence agreement with its new joint venture AffyXell Therapeutics. Avacta and AffyXell will now work together to develop Affimer proteins against a range of targets which, when produced by mesenchymal stem cells (MSCs), are intended to inhibit inflammatory and autoimmune pathways and improve the overall efficacy of MSCs, creating a next generation of stem cell therapies.
Novartis’ bumper crop of new product launches in 2019 helped it post 9% sales growth in the fourth quarter, and showed that a high price hasn’t dampened take-up of new gene therapy Zolgensma. Overall, sales were $2.4 billion in the fourth quarter, coming in a little ahead of analyst consensus estimates, helping operating profit to climb 17% to $1.8 billion. Zolgensma launched in the US in May – added $186 million in the quarter and took its total tally for the year to $361 million. Novartis said the gains came from increased screening of newborns for SMA and improving reimbursement coverage among commercial health insurance plans as well as Medicaid, which provides cover for people on low incomes.
Krystal Biotech, Inc. (Nasdaq:KRYS), a gene therapy company, announced today the ground breaking of the second commercial gene therapy facility in Findlay Township, Pennsylvania. The Findlay-based Current Good Manufacturing Practice (cGMP) facility, named ASTRA, will have the capacity to produce commercial gene therapy medicines to treat patients suffering from debilitating rare diseases.
The Center for Breakthrough Medicines, a Contract Development and Manufacturing Organization (CDMO) and specialty investment company, to alleviate the critical lack of capacity that is preventing patients from accessing critically needed cell and gene therapies. The CDMO provides preclinical through commercial manufacturing of cell and gene therapies and component raw materials. It offers process development, plasmid DNA, viral vectors, cell banking, cell processing, and support testing capabilities all under one roof.
The activities of the three-year project on Car T therapies will start by January in 13 of 26 IRCSS of Alleanza Contro il Cancro, as announced in October by the Minister of Health, Roberto Speranza. In the program funded by the Ministry of Health (10 million euros granted over two years) other non-IRCCS entities such as CNR, Fondazione Tettamanti and MolMed are involved in different ways. The main objective of the project is the development of new Car T cell therapies designed to destroy tumours that are currently not covered by the pharmaceutical industry (including pancreas, rectum, breast and melanoma) and which cannot be effectively treated in other ways.
LabCorp has unveiled a new suite of cell and gene therapy development offerings from Covance as it looks to get in on this burgeoning new trend. Covance says it will tap its experience to help offer the life sciences industry a close-knit partner and help push on with current and next-gen capabilities in this area.
U.K.-based PhoreMost and Otsuka Pharmaceutical Co. entered into a multi-project collaboration to seek out disease-relevant pathways that can be exploited for the development of gene therapies to treat different illnesses. According to the structure of the agreement, PhoreMost, which has its headquarters in Cambridge, England, will focus its SIZTESEEKER next-generation phenotypic screening platform on those disease-relevant pathways nominated by Otsuka.
In UK, the Cell and Gene Therapy Catapult (CGT Catapult) and the University of Hertfordshire today announced the launch of a new course specifically addressing the foreseeable skills gap in the manufacture of cell and gene therapies as they progress towards manufacturing at scale.
With cell and gene therapies moving towards commercialisation and employment in this space predicted to reach over 6,000 jobs by 2024, this initiative aims to provide targeted training which will support this growth and address the accelerating demand for skills in the sector.