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.
Gene therapy could save and improve lives, but at $2 million per treatment, it’s out of reach for the most vulnerable of cases in the US. CVS Health is leveraging its 2018 acquisition of health insurance company Aetna to launch a new employer-sponsored insurance product by the end of March to absorb the financial risk of covering these therapies.
If approved, Valrox would be the most expensive drug in the world and the first gene therapy in the U.S. that targets inherited hemophilia, one of the most common types of hemophilia out there. Clinical results have shown significant improvements in patients, with the number of bleeding incidents dropping to zero for years after receiving a Valrox injection.
Nationwide Children's, a gene therapy leader, launches manufacturing spinout Nationwide Children's Hospital, a hot spot for gene therapy research, this week launched Andelyn Biosciences, a biotech spinout dedicated to produce gene therapy components for biotech and pharmaceutical companies running clinical trials.
Astellas has teamed up with Adaptimmune to develop allogeneic chimeric antigen receptor T-cell (CAR-T) and T-cell receptor (TCR) therapies. The agreement sees Astellas pay $50 million (€45 million) upfront and commit to many times as much in milestones to work with Adaptimmune to identify targets and develop cell therapies against them.
Adaptimmune will pocket $50 million and $7.5 million a year in research funding, plus development and sales milestones that could bring the total deal value up toward $900 million.
Astellas Pharma is the latest on a growing list of companies to see gene therapies in their future, as it has agreed to acquire Audentes Therapeutics for $3 billion. Audentes uses adeno-associated viruses (AAVs) to deliver DNA, primarily to treat genetic neuromuscular diseases, and it is set to file for approval of X-linked myotubular myopathy (XLMTM) gene therapy AT132 in the U.S. and Europe in mid-to-late 2020.
Zolgensma is Novartis’ lifesaving gene therapy treatment for infants with spinal muscular atrophy, a rare and devastating neurological disease. Zolgensma has been used to treat 100 patients since its launch and brought in $160 million last quarter, beating analysts’ expectations. Novartis expects revenue to grow in the future. The company is counting on approvals for Zolgensma in Europe and Japan next year, and it recently presented data demonstrating the gene therapy’s effects on older SMA patients.
Asklepios BioPharmaceutical, a fully integrated Adeno-Associated Virus gene therapy platform company focused on providing curative therapeutics for genetic disorders, has acquired the technology assets of RoverMed BioSciences. RoverMed developed nanotechnology cargo delivery of therapeutics into the nucleus of diseased cells without affecting healthy cells. Under the terms of the agreement, AskBio will integrate the company's technology and assume all assets of RoverMed
Castle Creek Pharmaceutical Holdings will acquire Fibrocell, which focuses on transformational autologous cell-based therapies for skin and connective tissue diseases, for $63.3M and the deal is slated to be finalized by the end of the year. Fibrocell currently has two product candidates: the FCX-007 and the FCX-013. FCX-007 is an investigational, late-stage stage gene therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa; FCX-013 product works toward the treatment of moderate to severe localized scleroderma, a chronic connective tissue disease that causes of hardening of the skin and tissue below
Grünenthal agrees deal with Mesoblast to develop and commercialize an allogeneic cell therapy for chronic lower back pain; Mesoblast’s drug candidate, MPC-06-ID, is currently in Phase III trials for the treatment of chronic lower back pain due to degenerative disc disease. Grünenthal will pay $150M upfront to gain access to the potential treatment, with over $1bn reserved in milestone payments for the late-stage candidate
Genenta Science, a italian gene therapy company, has received $14.4 million in round three funding from the Chinese firm Qianzhan Investment Management and Fidim, that will propel their unique gene therapy through Phase I/II trials for multiple myeloma and glioblastoma. This therapy, called Temferon, aims to prevent CAR-T relapses, that occurs in around 40% of cases, by rebuilding the patient’s immune system within the tumor sites themselves; the treatment involves injecting a lentivirus for a gene transfer in hematopoietic stem and progenitor cells, triggering interferon-α expression in the targeted areas, in such a way as to enable the patients to fighting off future relapses
Passage Bio, a genetic medicines company developing AAV-delivered gene therapies for the treatment of rare monogenic central nervous system diseases, has announced that it has licensed a sixth gene therapy development program under its research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program; the license is for the clinical development of a potential treatment for patients with Charcot-Marie-Tooth Neuropathy Type 2A. CMT2 patients experience progressive muscle atrophy of legs and arms, with no approved curative or symptomatic medications available
Syncona, a leading healthcare company focused on founding, building and funding global leaders in life sciences, announced that it has committed £48.0 million of Series B financing to Gyroscope Therapeutics; Gyroscope is a leading clinical-stage, retinal gene therapy company targeting the treatment of dry age-related macular degeneration, one of the leading cause of blindness and for which there is currently no therapy
Achilles Therapeutics, an UK-based biopharmaceutical company developing personalized immunotherapies for cancer has raised £100 million in Series B financing; Achilles will use the funds to begin this year two human proof-of-concept studies using its personalized T cell immunotherapy to treat non-small cell lung cancer and melanoma
Vertex Pharmaceuticals announced it has agreed to acquire Semma Therapeutics for $950 million cash in the fourth quarter of 2019, in a deal that would expand its pipeline of specialty treatment with Semma’s potentially curative cell therapy for type 1 diabetes; Semma said its type 1 diabetes therapy is designed to incorporate a pair of advances, that is the ability to produce large quantities of functional human pancreatic beta cells that restore insulin secretion and a novel device that encapsulates and protects these cells from the immune system, enabling durable implantation without the need for ongoing immunosuppressive therapy
Celgene will have opt-in rights to take responsibility for development, manufacturing and commercialization of T-cell receptor (TCR) therapies that Immatics develops using its in-house technology. The deal includes a $75 million investment in the company by Celgene; Immatics would develop TCRs against solid tumor cancers using its in-house technology
Esco Ventures announce the launch of Carmine Therapeutics and execution of an exclusive license agreement for the technology developed in the laboratories of Professors Minh Le and Jiahai Shi at the City University of Hong Kong. Carmine Therapeutics plans to establish a site in Cambridge, and it will be focused on pioneering red blood cell-derived extracellular vesicle-based gene therapies
Adaptimmune Therapeutics, a leader in T-cell therapy to treat cancer, and Noile-Immune Biotech, a biotechnology company focusing on the development of innovative cancer immunotherapies, announced that they will co-develop next-generation SPEAR T-cell products, incorporating Noile-Immune’s PRIME (proliferation inducing and migration enhancing) technology, based upon co-expression of IL-7 and CCL19. The PRIME technology, which is already being investigated for augmentation of CAR-T cell activity, will be investigated with Adaptimmune’s SPEAR T-cells
Pfizer announced a $500 millions additional investment in gene therapy, with a new modern facility in Sanford, North Carolina; this structure is expected to support Pfizer’s continued investment in gene therapy research and development, even with about 300 new jobs
The German-based drug giant has doubled down on one of its previous investments, announcing a deal to acquire the remainder of the stake in BlueRock Therapeutics for $600 million
Selecta Biosciences and Asklepios BioPharmaceutical AskBio), announced a strategic partnership to jointly develop, manufacture and commercialize targeted therapeutics for life-changing, next-generation adeno-associated virus (AAV) gene therapies in areas of high medical need. This partnership will leverage the unique proprietary technology platforms of both companies; Selecta is the first company with preclinical evidence to support the potential for re-dosing patients receiving gene therapy; AskBio’s gene therapy platform includes a robust pipeline of potentially curative gene therapies, an extensive capsid library, groundbreaking manufacturing process and several advanced AAV initiatives under development
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
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
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