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This Week in Cell and Gene Therapy: 8 New Developments to Know (August 22, 2022)

There have been a number of recent developments in cell and gene therapy, as detailed in our bimonthly newsletter, Cell and Gene Therapy Business Outlook.

  • Prescient Therapeutics, a clinical stage oncology company based in Melbourne, Australia, has signed a five-year manufacturing agreement with Q-Gen Cell Therapeutics, a specialist cell therapy manufacturer based at the QIMR Berghofer Medical Research Institute in Brisbane, to produce Prescient’s OmniCAR cell lines for use in clinical trials.  The OmniCAR platform is a modular chimeric antigen receptor (CAR) system: T cells are transduced to express a SpyCatcher universal immune receptor, which can then bind to any desired targeting ligand equipped with a SpyTag motif.  The system enables tremendous control over T cell activity after infusion into the patient, including the ability to target multiple antigens, switch targets at will, and ramp T cell activity up or down.  The contract with Q-Gen Cell covers lentiviral transduction of T cells with the SpyCatcher vector, the arming of OmniCAR transduced T cells with SpyTagged binders, and the use of Prescient’s CellPryme-M platform, a high-performance manufacturing process.  Prescient has also signed an agreement Waltham, MA-based Thermo Fisher Scientific to develop and commercialize a scalable version of the OmniCAR platform, evaluating the potential use of automated, closed manufacturing systems and non-viral methods for large-scale applications.
  • National Resilience, a biomanufacturing company based in San Diego, CA, has announced a collaboration with Mayo’s Center for Regenerative Medicine in Rochester, Minnesota, to establish embedded process development, analytical development, and quality control labs at the Rochester site, and to jointly develop cell therapies and other advanced therapeutics derived from biologics, with the goal of bringing these therapies into the clinic.  The collaboration hopes to create a business incubator and attract third-party biotech companies to collaborate on process and analytical development and to sponsor clinical trials for new therapies.  Resilience has worked to establish a broad network of facilities across North America, occupying over 1 million square feet of manufacturing space with more than 1,600 employees.
  • RoosterBio, a supplier of human mesenchymal stem/stromal cells (hMSC) and extracellular vesicle (EV) products based in Frederick, MD, and AGC Biologics, a global biopharmaceutical contract development and manufacturing organization (CDMO) based in Seattle, WA, have announced a strategic partnership to develop hMSC and exosome therapeutics.  RoosterBio will provide expertise in genetic engineering of cells and exosomes, upstream processing, downstream purification, and analytical characterization of potential therapies.  AGC will provide full process development, cGMP manufacturing, quality control, and regulatory services up through Phase I/II clinical trials, with the potential to scale to Phase III trials and even commercial production.
  • EV Biologics, a nanoparticle-based  therapeutics (nanotherapeutics) company based in Cheyenne, WY, has announced that it has launched Excyte, a wholly owned subsidiary with a $100 million valuation.  EV Biologics is focused on developing the therapeutic potential of extracellular vesicles (EVs), particularly exosomes produced by mesenchymal stem cells (MSCs). Excyte will develop biomanufacturing, bioengineering, bioanalytic, and machine learning technologies to build a development platform for nanotherapeutic treatments targeting any indication.  The new company plans to raise $2.5 million in initial capital, and has already achieved 20% of that goal.  The funding will be used to evaluate small-scale automated bioreactor platforms and develop scalable biomanufacturing technologies for stem cell-derived nanotherapeutics. Excyte will also analyze secretome products and nanoparticle fractions produced by EV Biologics’ mesenchymal stem/stromal cell banks.  EV Biologics’ CEO, Daniel Mckinney, says the company’s ambition is to restore health, combat aging, and provide a longer health span.
  • WuXi Advanced Therapies, a contract testing, development, and manufacturing organization (CTDMO) based in Philadelphia, PA, has announced a licensing agreement with Janssen Biotech, a subsidiary of Johnson & Johnson based in Horsham, PA, for WuXi’s high-performance TESSA technology platform.  The agreement will also grant Janssen access to WuXi’s proprietary HEK293 suspension cell line.   WuXi says the TESSA manufacturing platform can produce 10 times more adeno-associated virus (AAV) than traditional AAV manufacturing systems, and at significantly higher quality.
  • Sarepta Therapeutics, a genetic medicine company based in Cambridge, MA, has announced that it has exclusively licensed the MyoAAV platform from Broad Institute of MIT and Harvard for Duchenne muscular dystrophy (DMD) and four other neuromuscular and cardiac indications.  The MyoAAV platform encompasses a novel group of adeno-associated viruses (AAV) with capsids optimized for muscle delivery.  Compared to natural AAV serotypes, MyoAAV capsids have demonstrated improved gene expression in multiple skeletal muscles (25-50 fold) and cardiac muscle (10-15 fold), with 50% lower delivery to the liver. Under the agreement, Sarepta will receive worldwide commercial rights for DMD and the other four neuromuscular and cardiac indications, with exclusive options for additional targets. In exchange, Broad Institute will receive an upfront payment in addition to future royalties and milestone payments.  Further financial details of the agreement were not disclosed.
  • RoslinCT, a cell and gene therapy contract development and manufacturing organization (CDMO) based in Edinburgh, UK, and Lykan Bioscience, a cell therapy CDMO based in Hopkinton, MA, have announced that they have entered into a business combination agreement to form a global advanced therapies CDMO.  Lykan is equipped with a 64,000 square-foot cell therapy manufacturing facility housing 16 cGMP processing suites at its Hopkinton site, which is expected to be fully operational by the end of 2022. RoslinCT plans to expand capacity at its 40,000 square-foot manufacturing facilities in Edinburgh with 8 cGMP suites.  Earlier this year, Global Healthcare Opportunities (GHO Capital Partners) announced an investment in RoslinCT, and will now make a majority investment in Lykan and support the combined entity. WindRose Health Investors, prior majority owner of Lykan, have also reinvested in the combined entity with Lykan Management.
  • ElevateBio, a cell and gene therapy technology company based in Cambridge, MA, and Boston Children’s Hospital have announced a new spinoff company to develop stem-cell-based, allogeneic chimeric antigen receptor T (CAR-T) cell therapies.  The yet to be named company is based on research from the laboratory of George Daley, MD, PhD, head of stem cell transplantation at Boston Children’s Hospital, and is the first company to spin out from a five-year collaboration between Boston Children’s Hospital and ElevateBio aimed at the development of cell and gene therapies.  Typically, induced pluripotent stem cells (iPSCs) tend to give rise to immature, embryonic blood cell types, but Daley’s team was able to derive mature αβ T cells from iPSCs by repressing the histone methyltransferase enzyme EZH1.  The resulting T cells, which they call EZ-T cells, show enhanced cytotoxicity, elevated cytokine production, superior persistence, and more efficient tumor clearance in preclinical studies when compared to conventionally produced iPSC-derived T cells.  A paper describing this new technology was published in the Aug 4th, 2022, issue of Cell Stem Cell.