Industry Update: Business Development
Latest business developments compiled from October 01 — November 30 2016
Latest business developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions from October 01 — November 30 2016, scheduled to be published in Volume 12 Issue 2 of Regenerative Medicine.
Collaborations, partnerships & alliances
Co-development agreement: Boehringer and SEU
Boehringer Ingelheim (Germany; www.boehringer-ingelheim.com) and China Southeast University Institute of Life Sciences (www.seu.edu.cn/english/232/list.htm) started a joint research project to develop new treatment approaches for hearing loss through regeneration of hair cells from inner ear stem cells.
Collaboration agreement: Axiogenesis and Metrion
Axiogenesis (Germany; www.axiogenesis.com) and Metrion Biosciences (UK; www.metrionbiosciences.com) have signed a collaboration agreement to validate, optimize and commercialize induced pluripotent stem cell (iPSC)-derived cell based assays to facilitate drug discovery and more accurately predict cardiac arrhythmia and neuronal risk. Under the terms of the agreement, Axiogenesis will share newly developed iPSC-derived cardiomyocytes and neurons with Metrion for detailed profiling and validation on its screening platforms, and Metrion will use commercial Axiogenesis cell products in its optimized ion channel screening, cardiac safety and translational phenotypic assays. The two companies are active participants in the FDA’s Comprehensive in vitro Proarrhythmia Assay (CiPA) initiative, contributing by improving cardiac safety assays using iPSC-derived cardiomyocytes.
Collaboration agreement: Belicum and OPBG
Bellicum Pharmaceuticals (TX,USA; www.bellicum.com) and Ospedale Pediatrico Bambino Gesù (OPBG, Italy, www.ospedalebambinogesu.it) announced an expanded collaboration focused on preclinical and clinical development of CD19 and other Chimeric Antigen Receptor (CAR) T and T cell receptor (TCR) therapeutics engineered with Bellicum’s CaspaCIDe® molecular safety switch technology, allowing the reduction or elimination of cells that become toxic or are no longer needed. CaspaCIDe® consists in a Chemical Induction of Dimerization (CID) safety switch technology designated to eliminate cells in the event of toxicity, where the CID-binding domain is coupled to the signaling domain of caspase-9, an enzyme that is part of the apoptotic pathway. Infusion of rimiducid is designed to trigger activation of this domain of caspase-9 (iCasp9), which in turn leads to selective apoptosis of the CaspaCIDe-containing cells. Under terms of this agreement, Bellicum agreed to provide financial support to the research collaboration in exchange for exclusive worldwide rights to commercialize cell therapies that are developed, while OPBG maintains rights for research purposes. OPBG will conduct research and clinical studies of CAR T and TCR therapeutics in pediatric patients, with initial CD19 CAR T and GD2 CAR T clinical trials in pediatric acute lymphoblastic leukemia and neuroblastoma patients, respectively, expected to start in 2017. In addition, OPBG agreed to manufacture European clinical trial supplies for the investigational programs, as well as Bellicum’s PRAME-targeted TCR, BPX-701, in its GMP facility.
Collaboration agreement: Emulate and Covance
Emulate (MA, USA; www.emulatebio.com) has announced a strategic collaboration with Laboratory Corporation of America Holdings (LabCorp; NC, USA; www.labcorp.com ) that seeks to integrate Emulate’s Organs-on-Chips technology into preclinical drug evaluation and testing services. Emulate’s proprietary technology attempts to recreate the natural physiology of human tissues and organs, and is designed to provide a predictive model of human response to diseases, medicines, chemicals and foods with greater precision and detail than other preclinical options. The collaboration will combine the advanced technology capabilities of Emulate and the end-to-end drug development expertise of Covance Drug Development (www.covance.com), with the goal of qualifying and commercializing the Organs-on-Chips technology as a new platform to enhance preclinical drug development for pharmaceutical and biotechnology companies.
Collaboration agreement: Emulate and USC
Emulate (MA, USA; www.emulatebio.com) has announced a strategic collaboration with the Lawrence J. Ellison Institute for Transformative Medicine of University of Southern California (CA, USA; https://ellison.usc.edu) to use Emulate’s Organs-on-Chips technology to conduct leading-edge translational R&D that can advance cancer treatment and patient well-being. Emulate and the Ellison Institute will collaborate on innovative approaches for modeling the human physiology of cancer and predicting human response to treatments, expanding the functionality and applications of using the Organs-on-Chips technology in cancer research. This R&D initiative is announced as part of Emulate’s growing network of collaborators and is designed to foster translational research to expand the range of academic, commercial and clinical applications for Emulate’s Human Emulation Systemâ„¢.
Collaboration agreement: Sernova and CTI
Sernova (ON, Canada; www.sernova.com) and CTI Clinical Trial and Consulting Services (OH, USA; www.ctifacts.com) have announced their collaboration on regulatory matters respecting Sernova’s Cell Pouch Systemâ„¢, a novel implantable and scalable medical device that when combined with therapeutic cells, may provide a convenient, safe and effective long-term therapeutic option for patients with chronic diseases such as insulin-dependent diabetes who seek to improve their quality of life.
Collaboration agreement: TxCell and UBC
TxCell (France; www.txcell.com/index.php/en) signed a strategic Research and Development collaboration agreement with the University of British Columbia (UBC; BC, Canada; www.ubc.ca) to develop a Chimeric Antigen Receptor (CAR) – Treg-based cellular immunotherapy for the prevention of graft rejection in the context of Solid Organ Transplantation. Non-clinical pharmacology studies with CAR-Treg cells will be conducted in the UBC laboratories UBC with the aim of initiating a first-in-man study in transplantation patients as soon as possible. The UBC team demonstrated that, in a preclinical xenogeneic Graft-vs-Host Disease (GvHD) model, human CAR-engineered Treg cells that are specific for the molecule HLA-A2 were more effective than polyclonal Treg cells in reducing GvHD-related inflammation. Whilst the UBC team is focused on product development activities, it will in parallel perform research activities in the CAR-Treg field with the aim of optimizing and broadening the new product platform for transplantation. TxCell has an exclusive option on programs and products developed under this agreement. Financial terms of the collaboration have not been disclosed.
Distribution agreement: HemaCare and AccuResearch
HemaCare (CA, USA; www.hemacare.com) has expanded its global capabilities through a strategic distribution agreement with AccuResearch (Korea; http://accuresearch.en.ec21.com). This partnership ensures researchers in South Korea can access HemaCare’s human healthy and disease state fresh and cryopreserved hematopoietic cell products for their basic scientific and cell therapy research and development needs in a timely manner and with local regional support.
Licensing and development agreement: TissueGene and Mitsubishi
TissueGene (MA, USA; www.tissuegene.com) announced an exclusive licensing and development agreement between Mitsubishi Tanabe Pharma (Japan, www.mt-pharma.co.jp/e/) and TissueGene’s Asia licensee, Kolon Life Science of Korea (Korea, www.kolonls.co.kr/eng/). The agreement between the two companies focuses on the development and commercialization of Invossaâ„¢, an allogeneic, cell-mediated gene therapy for degenerative osteoarthritis of the knee for the Japanese market. Clinical trials completed in Korea and on-going trials in the US have demonstrated pain relief, increased mobility, and improvements in joint structure. TissueGene has completed U.S. Phase II trials of Invossa and received a Special Protocol Assessment (SPA) designation for Phase III trials scheduled to begin in the second quarter of 2017. The US Phase III will aim for approval from the US FDA as the first disease-modifying osteoarthritis drug. Under the terms of the agreement, Mitsubishi Tanabe will pay an upfront payment of approximately US$ 24 Million plus additional payments of up to approximately US$ 410 Million upon achievement of development, regulatory and commercial milestones, as well as a double-digit sales royalty. The deal amount announced today represents the largest single territory deal on record for Korea.
Supplier agreement: Regenicin and Pure Med Farma
Regenicin (NJ, USA, www.regenicin.com) announced a supplier agreement with Pure Med Farma (NJ, USA; www.puremedfarma.com) to provide closed herd collagen for production of its proprietary collagen scaffolds for the manufacturing of NovaDermâ„¢. The agreement with Pure Med Farma states Regenicin will receive priority pricing and guaranteed inventory availability. This is a key milestone, as guidance documents from the U.S. FDA clearly state that any product requesting approval to enter clinical trials will be denied until the applicant presents evidence that components derived from animal sources were obtained from closed herds.
Launching new projects, products & services
CBMT and AmnionStem
Creative Medical Technology (AZ, USA; www.creativemedicaltechnology.com) announced the formation of a wholly-owned subsidiary, AmnioStem, focused on the development of therapeutic products derived from amniotic fluid stem cells. Creative Medical Technology has signed an exclusive license agreement with the University of California San Diego (CA, USA; www.ucsd.edu) to commercialize and develop a novel type of amniotic fluid derived stem cell derived from amniotic fluid of pregnant women, which is obtained during routine amniocentesis.
Cook and Asymptote
Cook Regentec (IN, USA; www.cookregentec.com) and Asymptote (UK; www.asymptote.co.uk) have launched the CellSeal® Automated Thawing System. The new system, which has been developed as part of a collaboration between the two companies, provides users with thorough control in the thawing of cryogenically frozen cells, a critical step in the delivery of cell and gene therapies.
Coriell
The Allen Institute for Cell Science (WA, USA; www.alleninstitute.org/what-we-do/cell-science/) has released the Allen Cell Collection: the first publicly available collection of gene edited, fluorescently tagged human iPSC that target key cellular structures with unprecedented clarity. Distributed through the Coriell Institute for Medical Research (NJ, USA; www.coriell.org), these powerful tools are a crucial first step toward visualizing the dynamic organization of cells to better understand what makes human cells healthy and what goes wrong in disease. This first collection of five cell lines targets a set of major cellular structures that help to orient the cell. These include the nucleus (tagged by the protein lamin B1), mitochondria (Tom20), microtubules (alpha-tubulin), cell-to-cell junctions (desmoplakin) and adhesion (paxillin). Subsequent collections will be released throughout 2017.
Elixirgen
Elixirgen (MD, USA; www.elixirgen.com) has announced on October 03, 2016 launching of Quick-Muscleâ„¢ 1.0, a groundbreaking biological reagent that develops skeletal muscle cells from human stem cells in just a few days – a cutting-edge advancement for a laboratory process that previously took a month or longer. Pre-orders were also being taken for Quick-Neuronâ„¢ 1.0, the fastest and most efficient neuron differentiation kit ever available. Quick-Neuron 1.0 yields an abundance of neurons in less than one week. However, the information about kits were not found any more on the company website by end of December 2016.
Matritek
MatriTek (NY, USA: www.matritek.com) has launched commercial production of novel extracellular matrix (ECM) biomaterials for biomedical research. TissueSpecTM Hydrogel Kits are acellular matrix products derived from a variety of porcine organs and tissues. Versatile in format, hydrogel matrix kits offer the matrix components present in natural tissues and recreates a physiological substrate for 3D cell culture. TissueSpec Hydrogel Kits have been shown to enhance cell attachment, growth, and function in stem cell research. These kits are designed for easy application in cell culture and provide structural, mechanical, and compositional cues of the native tissue microenvironment.
Achievements
CardioCell
CardioCell (CA, USA; www.cardiocell.com) published detailed results from the completed Phase IIa clinical trial (https://clinicaltrials.gov; ID: NCT02467387) using intravenous administration of MSCs for chronic heart failure indications [1]
Celprogen
Celprogen (CA, USA; www.celprogen.com) have successfully finished printing a 3D Pancreas from flexible Poly Lactic Acid (PLA) material scaffold that was populated with adult human pancreatic stem cells. These 3D printed human pancreases were coated and seeded with human adult pancreatic stem cells. The 3D pancreas scaffold was populated with three T225 human Pancreatic Stem Cells 36097-24-T225, and with human adult pancreatic cells 3002-04-T225; the scaffold was coated with extracellular matrix proteins prior to seeding the cells. This flexible PLA scaffold allows the seeded pancreatic stem cells to potentially differentiate into an adult functional pancreas. The 3D print was reduced from an adult 18 year old pancreas to approximately 1/5 its original size. The present invention relates to 3D organ printing programs at Celprogen that identify potential future use in transplants of major organ systems.
Emulate
Emulate (MA, USA; www.emulatebio.com) has announced that its human Lung-Chip system has expanded functionality to now also model conditions in which inhaled toxins enter the lung, including cigarette smoke, e-cigarettes constituents, and potentially environmental airborne contaminants. The new applications are based on results from the Wyss Institute for Biologically Inspired Engineering at Harvard University (MA, USA; https://wyss.harvard.edu), the academic source of Emulate’s founding team and fundamental technology [2]. Emulate has exclusively licensed the technology used in the study from the Wyss Institute for translation into commercial products, including the company’s Human Emulation System. The expanded functionality of the Lung-Chip system is based on the addition of a life-like breathing ‘inhalation and exhalation’ component, enabling new research and insights into how non-smokers or patients with a chronic lung disease — such as chronic obstructive pulmonary disease — may respond to smoke or environmental pollutants. As demonstrated in the published study, using the Lung-Chip to compare human cells before and after exposure to an inhaled agent enables the effect of the agent to be isolated in a human-relevant system, with higher precision than conventional cell culture systems and better fidelity than animal models.
ORIG3N
ORIG3N (CA, USA; www.orig3n.com) has identified human leukocyte antigen compatible matches within its crowdsourced blood cell repository LifeCapsule for 90 percent of the U.S. population.
Vertera
Vertera Spine (GA, USA; www.verteraspine.com) announced that the COHERE® Cervical Interbody Fusion Device has been recognized by Orthopedics This Week (https://ryortho.com) as one of the Top Ten Best Spine Technologies for 2016. COHERE features Vertera Spine’s patented porous polyetheretherketone (PEEK) Scoria® biomaterial technology, a porous architecture that seamlessly integrates with solid PEEK to mimic the cancellous to cortical transition of native bone. Every year, Orthopedics This Week recognizes the engineering teams and inventors who develop and successfully translate innovative products into clinical use to treat back pain.
References
[1] Butler J, Epstein SE, Greene SJ et al. Intravenous Allogeneic Mesenchymal Stem Cells for Non-Ischemic Cardiomyopathy: Safety and Efficacy Results of a Phase II-A Randomized Trial. Circ. Res. doi.org/10.1161/CIRCRESAHA.116.309717 (2016) (Epub ahead of print).
[2] Benam KH, Novak R, Nawroth J et al. Matched-Comparative Modeling of Normal and Diseased Human Airway Responses Using a Microengineered Breathing Lung Chip. Cell Syst. 3(5), 456-466.e4 (2016).