ARM Recaps 2019, Discusses Outlook for 2020 at the 10th Annual Cell & Gene Therapy State of the Industry Briefing
January 21, 2020 – (ARM) – ARM CEO Janet Lambert provided an industry overview, presenting updated metrics for the 987 regenerative medicine therapeutic developers active worldwide and discussing the outlook for the sector in 2020. Industry experts provided a deeper dive during panels on Emerging Cell Therapies for Cancer and Next Generation Gene and Cell Technologies. A recording of the event, as well as slides from the industry overview, are available online here.
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B-MoGen Biotechnologies and CytoSen Therapeutics Partner on Research Collaboration to Develop Next Generation Natural Killer Cells for Human Therapeutics
January 23, 2020 – (B-MoGen Biotechnologies) – B-MoGen will utilize its expertise in genetic design and its patented non-viral gene delivery platform in conjunction with CytoSen’s nanoparticle expansion platform to improve efficacy and reduce cost of NK cell therapeutics.
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The Center for Breakthrough Medicines is Building the World's Largest Cell and Gene Therapy Contract Development and Manufacturing Organization to Launch in King of Prussia, PA
January 23, 2020 – (The Discovery Labs) – The immense $1.1B facility will provide instant capacity as the largest known single source for accelerating the delivery and affordability of gene and cell therapies. The company expects to hire more than 2,000 team members within the next 30 months.
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Adaptimmune Therapeutics Prices Public Offering of American Depositary Shares
January 22, 2020 – (Adaptimmune) – The gross proceeds for this offering are expected to be approximately $84M. Net proceeds will be used to advance the development of Adaptimmune’s immunotherapies into and through clinical trials as well as for other general corporate purposes.
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Autolus Announces Pricing of Public Offering
January 22, 2020 – (Autolus) – Autolus Therapeutics announced the pricing of an underwritten public offering in the United States for total gross proceeds of approximately $80M.
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PDC*line Pharma Raises €20M ($22.2M) in Series B Financing
January 22, 2020 – (PDC*line) – In addition, the company will receive €6.1M ($6.8M) in loans and subsidies from the Walloon Region of Belgium, which has backed the company since it expanded its operations there in 2016.
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Bellicum Pharmaceuticals Enters Into Asset Purchase and Master Services Agreements With MD Anderson
January 21, 2020 – (Bellicum Pharmaceuticals) – Bellicum Pharmaceuticals announced it has entered into an asset purchase agreement under which the University of Texas MD Anderson Cancer Center will acquire Bellicum’s 60,000-square-foot Houston facility, including manufacturing, office, and laboratory space, for $15M.
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Ncardia and BlueRock Therapeutics Announce Collaboration Agreement and Licensing of Process Development Technologies for the Manufacture of iPSC-Derived Cardiomyocytes
January 21, 2020 – (Ncardia) – Under the terms of the agreement, Bluerock gains access to Ncardia’s large-scale production processes and intellectual property for the production of iPSC-derived cardiomyocytes for therapeutic use.
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Orgenesis Announces $9.2M Private Placement
January 21, 2020 – (Orgenesis) – The company intends to use the proceeds of this financing to expand its point-of-care cell-therapy platform, which is designed to collect, process, and supply therapeutic cells within the patient care setting for various treatments, as well as for general corporate purposes
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Three Industry Leaders Form New Venture to Create Scalable Manufacturing Platforms for Cell and Gene Therapies
January 21, 2020 – (Bio-Techne) – Bio-Techne, Fresenius Kabi, and Wilson Wolf have formed a new joint venture company to provide dedicated support to researchers and biopharmaceutical companies in the field of cell and gene therapy.
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CoImmune and Formula Pharmaceutical Agree to Merge in an All-Stock Transaction
January 15, 2020 – (CoImmune) – Upon completion of the merger, the company will continue to focus on running a Phase 2b trial for lead asset, CMN-001, in advanced renal cell carcinoma and a Phase 1 trial for CAR-CIK in acute lymphoblastic leukemia.The close of the deal will be accompanied by a $6M investment in the combined entity to fund the CAR-CIK program.
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AGTC Reports Encouraging Interim Six-Month Data from the Dose Escalation Cohorts of its Ongoing Phase 1/2 Clinical Trials in Achromatopsia
January 23, 2020 – (AGTC) – The data shows encouraging preliminary signs of biologic activity and a favorable safety profile. The company plans to report data from additional dose groups, age groups, and time-points in the second half of 2020.
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Aruvant Receives Orphan Drug Designation for ARU-1801 for the Treatment of Sickle Cell Disease
January 22, 2020 – (Aruvant) – Aruvant announced that the FDA has granted Orphan Drug designation to ARU-1801, Aruvant’s investigational therapy for the treatment of sickle cell disease. Aruvant announced that ARU-1801 received rare Pediatric Disease designation earlier this month.
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Celularity Announces FDA Clearance of Landmark IND for CYNK-001, an Allogeneic, Off-the-Shelf Cryopreserved NK Cell Therapy
January 22, 2020 – (Celularity) – The clinical investigation of CYNK-001 in patients with GBM is expected to be the first clinical trial in the US to investigate intratumoral administration of an allogeneic NK cell therapy.
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Genprex Receives FDA Fast Track Designation for Gene Therapy Targeting Lung Cancer
January 21, 2020 – (Genprex) – Genprex has treated more than 50 lung cancer patients with Oncoprex in Phase I and II clinical trials. The company believes the data from these trials are encouraging as to both safety and efficacy.
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Researchers Uncover Mechanism for How Common Gene Therapy Vectors Enter Cells
January 23, 2020 – (Massachusetts Eye and Ear) – In the study, researchers used a genome-wide CRISPR screening tool to look at 100,000 genes and determine which cells played a role in AAV targeting. GPR108 was shown to be critical for most AAVs that are currently being studied clinically, including AAVs used in the two FDA-approved gene therapies.
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Potential Way to Halt Blinding Macular Degeneration Identified
January 22, 2020 – (University of Virginia) – Researchers found that the absence of a particular enzyme could drive both forms of AMD. They were then able to restore the enzyme in mice by adapting a form of gene therapy already used to treat other eye diseases.
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Drug Profiling and Gene Scissors Open New Avenues in Immunotherapy
January 21, 2020 – (University of Helsinki) – By employing the CRISPR gene editing method, the researchers investigated which mechanisms impact the sensitivity of cancer cells to CAR-T cells. A process known as death receptor signalling and the FADD gene required to initiate this process were found to be vital for CAR T-cell function.
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Discovery of New T-Cell Raises Prospect of ‘Universal’ Cancer Therapy
January 20, 2020 – (Cardiff University) – Researchers have discovered T-cells equipped with a new type of receptor which recognises and kills most human cancer types, while ignoring healthy cells. Researchers injected T-cells able to recognise MR1 into mice bearing human cancer and with a human immune system, with encouraging cancer-clearing results.
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Tweaking How CAR-T Therapy Kills Tumors Could Stop a Dangerous Side Effect, Study Finds
January 17, 2020 – (STAT News) – Tweaking the receptor on CAR-T cells that recognize cancer cells seemed promising, but made the CAR-Ts less effective against cancer cells. A more effective approach, at least in mice, was to block the molecules that make tumor cells die via pyroptosis rather than less violently.
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Dorothy Schafer Lab Shows Gene Therapy Protection of Eyesight in Models of Multiple Sclerosis
January 15, 2020 – (University of Massachusetts) – Researchers used a gene therapy approach and adeno-associated virus to deliver Crry, an inhibitor of C3, specifically to synapses in the visual system while leaving the rest of the brain untouched, to see if synapses could be spared and vision preserved.
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Gene and Cell Therapies in Asia: Pricing, Manufacturing Stand-Out Issues in Supportive Japan Ecosystem
January 22, 2020 – (Pink Sheet) – The Japanese environment for cell and gene therapies and regenerative medicine in general continues to be highly supportive. But will pricing pressures under the NHI scheme move to the fore as more expensive products come to market?
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UK Report Provides Update on Life Sciences Industrial Strategy
January 13, 2020 – (gov.uk) – The report is organized into 5 key themes: NHS collaboration; business environment; reinforcing the UK science offer, including clinical research, data and genomics; skills; and advanced therapies, including developing advanced therapies and advanced therapies manufacturing.
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Director / VP, Finance and Operations
Alliance for Regenerative Medicine – Washington, DC
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