Industry Update: Business Development
Latest business developments compiled from October 01 — November 30 2015
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 October 01 — November 30 2015, scheduled to be published in Volume 11 Issue 2 of Regenerative Medicine.
Collaborations, partnerships & alliances
Co-development agreement: Asterias and Catapult
Asterias Biotherapeutics (CA, USA; www.asteriasbiotherapeutics.com) has signed an agreement with Cell Therapy Catapult (UK; www.ct.catapult.org.uk) to advance development of large scale manufacturing processes for AST-VAC2, Asterias’ allogeneic human embryonic stem cell (hESC)-derived dendritic cell immunotherapy. Under the agreement, the Cell Therapy Catapult will streamline and scale manufacturing processes for AST-VAC2 to support advanced clinical trials and eventual commercialization of AST-VAC2.
Co-development agreement: ViaCyte and Takra
ViaCyte (CA, USA; www.viacyte.com) has entered into a non-exclusive licensing agreement with Takara Bio (Japan; www.takara-bio.com). Takara will utilize certain portions of ViaCyte’s cell differentiation technology to expand its stem cell product line, marketed under the Cellartis® brand. Takara will make the technology available for research purposes only, not for clinical or commercial application. The licensing agreement includes ViaCyte technology for a robust method of making definitive endoderm cells from induced pluripotent stem cells (iPSC), a necessary step in the production of a number of cell types. Takara will incorporate the technology into the kits it markets to researchers, including the Cellartis® iPS Cell to Hepatocyte Differentiation System.
Collaboration agreement: CDI and Roche
Cellular Dynamics International (CDI; WI, USA; https://cellulardynamics.com), a FUJIFILM (Japan; www.fujifilm.com) company, made an agreement with Roche (Switzerland; www.roche.com) to supply CDI’s iPSC-derived iCell products to contribute to the identification of novel drug candidates at the early stages of drug discovery. Under the terms of the supply and license agreement, Roche will have exclusive access to specific iCell products to be employed in High Throughput Screening and lead optimization for drug discovery. The partnership is dependent on certain development milestones and may amount to US$ 83 million in total.
Collaboration agreement: CEVEC and BioLamina
CEVEC Pharmaceuticals (Germany; www.cevec.com) and BioLamina (Sweden; www.biolamina.com) have entered in a strategic collaboration for the commercial production of research grade and clinical grade laminins for ex vivo cell culture applications using CEVEC’s proprietary CAP®Go technology. Financial details of this agreement were not disclosed. Under the terms of the agreement, CEVEC will provide its expertise in process development as well as its competence in Good Manufacturing Practice (GMP) production, in collaboration with its joint venture partner GenIbet (Portugal; www.genibet.com), of selected laminins based on the Company’s proprietary CAPGo technology. CEVEC retains the rights to develop laminins for therapeutic use and grants BioLamina an exclusive license for the commercialization of CAPGo based laminins patented by BioLamina for ex vivo cell culture therapies. The first laminin, Laminin521, which aims to support proliferation of stem cells without causing genomic abnormalities, will be launched in 2016.
Collaboration agreement: Cynata and MGH
Cynata Therapeutics (Australia; www.cynata.com) has commenced a program to develop modified mesenchymal stem cells (MSCs) to treat cancer, using its proprietary Cymerusâ„¢ platform technology. This new program will be complementary to ongoing commercial partnership discussions and the development of the Company’s lead product, CYP-001, a mesenchymal stem cell product using the cell’s ability to modulate the immune response and inflammation to treat Graft versus Host Disease. As part of this new development program, the company has entered into a collaborative agreement with Massachusetts General Hospital (MGH; MA, USA; www.massgeneral.org) to access their cell-modification technology.
Collaboration agreement: CytomX and MD Anderson Cancer Center
CytomX Therapeutics (CA, USA; http://cytomx.com) entered into a collaboration with The University of Texas MD Anderson Cancer Center (TX, USA; www.mdanderson.org) to research Probodyâ„¢-enabled chimeric antigen receptor natural killer (CAR-NK) cell therapies, to be known as ProCAR-NK cell therapies. MD Anderson will leverage its expertise in developing allogeneic umbilical cord blood and peripheral blood derived NK-cell therapies and combine it with CytomX’s Probody technology to address new targets for this novel modality in cancer immunotherapy. Designed for more precise binding to tumors and reduced binding to healthy tissue, ProCAR-NK cell therapies will be created against targets for which safety and toxicity have traditionally been limiting factors for CAR cell therapies. Under the collaboration, CytomX and MD Anderson will develop ProCAR-NK cell therapies against multiple targets, and CytomX will have the option to license therapeutics that demonstrate preclinical proof of concept for clinical and commercial development.
Collaboration agreement: RenovaCare and BCRT
RenovaCare, Inc. (NY, USA; www.renovacareinc.com) announced a partnership to validate the science behind its technology for treatments of wounds, burns and other skin defects. The company’s research partner is Berlin-Brandenburg Center for Regenerative Therapies (BCRT; http://bcrt.charite.de), a translational research center at Charité — Universitätsmedizin Berlin. The BCRT focuses on enhancing endogenous regeneration by cells, biomaterials, and factors, which can be used to develop and implement innovative therapies and products. RenovaCare, Inc. is the developer of the patented CellMistâ„¢ and SkinGunâ„¢ technologies for isolating and spraying a patient’s own stem cells onto burns and wounds for rapid self-healing. The objective the partnership is to evaluate protocols, methods, and outcomes related to the use of the RenovaCare proprietary SkinGun. This includes better understanding the ability of delicate stem cells to survive and grow after being gently sprayed through SkinGun on to targeted body sites. Scientists at BCRT will also study various additional features and behaviors related to skin stem cells and their ability to replicate and grow, for wound healing.
Collaboration and distribution agreement: Theradome and Nanjing XJS
Theradome (CA, USA; www.theradome.com) named Nanjing XJS (China; www.xiaojingsha.com) as its master distributor of the Theradomeâ„¢ laser hair regrowth helmet in the Chinese market. The Theradome helmet uses 678 nm laser light to stimulate the mitochondria in stem cells at the base of the hair follicle. The Chinese hair care market boasts a market value of RMB 33.6 billion (US$ 5.5 billion), in which sales of Western hair loss treatments grew 90% from 2007 to 2012.
Collaboration and production agreement: CBR and NYSCF
Cord Blood Registry (CBR; CA, USA; www.cordblood.com) will collaborate with The New York Stem Cell Foundation (NYSCF; NY, USA; www.nyscf.org) to customize the creation of high-quality stem cell lines. NYSCF will create iPSC lines from umbilical cord tissue collected after birth from healthy newborns provided by CBR. NYSCF will employ its proprietary robotic technology, the NYSCF Global Stem Cell ArrayTM, to efficiently and consistently create the pluripotent stem cells central to this partnership. NYSCF’s team of scientists will receive the material from CBR and identify a subset of samples for reprogramming to generate iPSC. NYSCF will then characterize the resulting stem cells, ensure quality, and cryopreserve the cells for research and future use.
Licensing agreement
Kite Pharma, Inc. (CA, USA; www.kitepharma.com) has entered into an exclusive, worldwide license with the NIH for intellectual property related to T Cell Receptor (TCR)-based product candidates directed against melanoma antigen family (MAGE) A3 and A3/A6 antigens for the treatment of tumors expressing MAGE, which include lung, pancreatic, gastric, and breast cancers, among others. The National Cancer Institute (NCI) is currently conducting two Phase 1-2a clinical trials of TCR-based product candidates targeting the MAGE antigens under a Cooperative Research and Development Agreement (CRADA) between Kite and the NCI. Pursuant to the terms of the license agreement, NIH will receive from Kite an upfront payment and certain clinical, regulatory, and sales milestone payments, as well as royalties on net sales of products covered by the license.
Licensing agreement: Lion and NIH
Lion Biotechnologies (CA, USA; www.lbio.com), a biotechnology company developing novel cancer immunotherapies based on tumor-infiltrating lymphocytes (TIL), has obtained an exclusive, worldwide license from the NIH to develop and commercialize TIL therapy in four additional tumor indications: bladder, lung, breast and HPV-associated cancers, including cervical and head and neck. The agreement was executed as an amendment to Lion’s existing exclusive licensing agreement with the NIH for the development and commercialization of TIL in the treatment of metastatic melanoma.
Launching new projects, products and services
Allele
Allele Biotechnology & Pharmaceuticals (CA, USA; www.allelebiotech.com) plans to form a commercial business for the banking of human iPSC by private individuals. In addition to serving its customers, Allele’s iPSC bank will be a resource for biomedical research. Proper consent and privacy guardianship will allow thousands of iPSC lines with accompanying sequence database and health information to be made available from the bank to scientists and clinicians. Towards the establishment of this business effort, Allele has recently purchased a 1700 m2 facility, located near its headquarters. This new cGMP facility will use Allele’s proprietary synthetic mRNA platform for reprogramming.
Ascendance Biotechnology
BioTime, Inc. (CA, USA; www.biotimeinc.com) and Hepregen Corporation (MA, USA; www.hepregen.com) jointly formed Ascendance Biotechnology. Ascendance combines Hepregen’s application directed, cellular micro-patterning drug and chemical screening technologies with BioTime’s ESI BIO research products and proprietary stem cell technologies. This asset combination aims to broaden Ascendance’s portfolio of current and new stem cell derived assays and other products and services to Hepregen’s major pharmaceutical and chemical company customers. Ascendance will continue to market Hepregen’s HepatoPac® and HepatoMuneâ„¢ micro-patterned liver products, and plans to develop new and unique cell-based and micro-patterned products using BioTime’s stem cell technologies and broad intellectual property and product portfolio, including BioTime’s PureStem® embryonic progenitors and ESI embryonic stem cells.
Betalin
Betalin Therapeutics (Israel; www.betalintherapeutics.com) introduced the Engineered Micro Pancreas (EMP), a novel technology that provides glucose-regulated insulin secretion over extended periods of time. Attempts to treat diabetes by transplants of pancreatic beta cells have up until now been complicated by the fact that the vast majority of transplanted cells die within two days of transplantation, and therefore about 50% of the patients are still insulin-dependent one year after transplantation, while only about 10% remain insulin-independent five years after transplantation. EMP is based on the premise that in order for beta cells to properly function, it is necessary to provide an appropriate connective tissue scaffold that ensures the long term survival of the cells. The proprietary platform technology is a method to prepare acellular organ-derived microscaffolds that preserve the architecture and the basic composition of organ connective tissue and ensure that no seeded cell will be more than 150 µm from a source of nutrients and gases.
Celprogen
Celprogen (CA, USA; www.celprogen.com) announced that they have adult adipose stem cell derived conditioned media (AASCDCM) containing stem cells growth components for the skin care regenerative products. These stem cell derived conditioned media have been utilized in skin care products for a decade. The lyophilized adult adipose stem cell derived conditioned media is approximately 90% as an active ingredient in regenerative skin care topical products. Celprogen manufactures these lyophilized adult adipose and mesenchymal derived stem cell conditioned media which is serum free and devoid of all animal components in mg to kg batch sizes for the cosmetic industry. This product is also available in the liquid format with 2000L to 10,000L batch sizes. The advantage of lyophilized material is that its shelf life is 2 years at 2-8°C, whereas the liquid product has 1 year at -20°C; the other advantage of the lyophilized product is the ease of shipment to any part of the world.
ISCT
The International Society for Cellular Therapy (ISCT; www.celltherapysociety.org) launched of its publication and reference guide on the Use of Unproven Cellular Therapies [1]. The documentation has been authored by the ISCT Presidential Task Force, comprising 23 leading figures in the field of cell therapy. The publication, constructed over two and a half years, is the most recent and comprehensive initiative by ISCT to combat and raise awareness about unethical and unproven cellular therapies and medical tourism. The project hopes to enable all concerned to make more informed decisions about treatments in this growing field of medical care.
Nexxt Spine
Nexxt Spine (IN, USA; www.nexxtspine.com) developed NanoMatrixx®, a porous bioactive titanium material designed to actively participate in the intervertebral fusion process. NanoMatrixx is manufactured to exacting specifications utilizing modern 3D printing technology to replicate the cellular structure of cancellous bone. This process makes it possible to create any three dimensional complex structure or geometry with a desired modulus of elasticity that cannot be created by traditional orthopedic manufacturing processes (https://vimeo.com/142053256). Following the manufacturing process, the material undergoes a series of proprietary treatments to produce a micro and nanosurface topography which stimulates mesenchymal stem cells to differentiate into bone forming osteoblast cells that produce bone growth onto and throughout the 3D printed material.
Osiris
Osiris Therapeutics, Inc. (MD, USA; www.osiris.com) launched TruSkinâ„¢, a cryopreserved, living skin allograft. TruSkin is a split-thickness, cryopreserved skin allograft that is offered by Osiris to address unmet medical needs of chronic wounds. TruSkin retains the extracellular matrix, growth factors, and endogenous living skin cells of native tissue, making it an alternative to fresh skin allograft. TruSkin will be available in multiple sizes and have a 5-year shelf life when stored at or below -40°C.
Osiris also announced the launch of Stravixâ„¢, a cryopreserved human placental tissue designed to address surgical needs for soft tissue repair. Stravix is a cryopreserved human placental tissue comprised of amniotic and connective layers of umbilical tissue that has been developed as a cover specifically for surgical procedures. It retains native components of the tissue including collagen and hyaluronic acid rich extracellular matrix, cytokines, fibroblasts, growth factors, viable endogenous mesenchymal stem cells and epithelial cells. Stravix conforms to the site of injury, requires minimal preparation prior to use and is stored at -75°C to -85°C.
Stem Med
Stem Med (Singapore; http://stem-med.sg) is the first private stem cell bank in Singapore to offer the processing, cryopreservation and storage of Bone Marrow and Adipose (fat) tissues MSC for immediate or future therapeutic use. It is a new joint venture between public-listed TalkMed Group (Sigapore; www.talkmed.com.sg) and StemCord (Singapore; www.stemcord.com). Its new wholly owned Stem Med Cellular Laboratory is equipped with ISO Class 5, Class 6 and Class 8 clean rooms to perform cell processing and culturing.
Achievements
Emulate
Emulate (MA, USA; www.emulatebio.com) announced that data on its Brain-on-Chip were presented by its collaborators at Cedars Sinai’s Board of Governors Regenerative Medicine Institute, who led the research and provided iPSC from patient samples to enable this new approach to model the human brain microenvironment. In the study, Emulate’s Brain-on-Chip demonstrated physiologically-relevant modeling of the human brain microenvironment, including successful co-culture of human brain microvascular endothelial cells, neurons and astrocytes. The findings from this collaborative research open up new applications for using Emulate’s Brain-on-Chip, in combination with patient-derived iPSC from Cedars-Sinai, as a system to advance drug R&D in the field of neurodegenerative diseases and improve understanding of key mechanisms driving disease development. The data were presented at the Society for Neuroscience (www.sfn.org), 45th annual meeting in Chicago.
KAIMRC
King Abdullah International Medical Research Center (Saudi Arabia; http://kaimrc.med.sa) cord blood bank received internationally recognized accreditation by the Foundation for the Accreditation of Cellular Therapy (FACT; NE, USA; www.factwebsite.org). The program at King Abdullah International Medical Research Center is the first and only one in Saudi Arabia to be recognized by FACT and is accredited for banking cord blood for both public and private family use.
References