Industry Update: Business Development

Written by Dusko ILIC

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 April 01 — May 31, 2017.

Latest business developments compiled from April 01 — May 31, 2017, scheduled to be published in Volume 12 Issue 6 of Regenerative Medicine.

Collaborations, partnerships & alliances 

Co-development agreement: 3D Systems and United Therapeutics

3D Systems (CA, USA; www.3dsystems.com) have announced a bioprinting agreement with United Therapeutics (MD, USA; www.unither.com), which will see the two companies develop solid-organ scaffolds for human transplants with 3D printing. The agreement will focus on the development of 3D printing systems for solid-organ lung scaffolds for a United Therapeutics subsidiary, Lung Biotechnology PBC (MD, USA; www.lungbiotechnology.com). The printing system will target collagen, and other building block proteins, as scaffold raw materials. Lung Biotechnology PBC will cellularize the scaffolds with patient-specific biological material, including re-differentiated stem cells.
 

Co-development agreement: CBMG and GE Healthcare

Cellular Biomedicine Group (CBMG; CA, USA; www.cellbiomedgroup.com) and GE Healthcare Life Sciences China (China; www.gelifesciences.com) have established a strategic research collaboration to co-develop state-of-the-art automated Chimeric Antigen Receptor T-cell (CAR-T) and stem cell manufacturing capabilities that build upon the accreditation of CBMG’s Good Manufacturing Practice (GMP) facilities in Shanghai, Wuxi and Beijing.  The co-development activity will aim to standardize the delivery of cell manufacturing to potentially improve throughput, alleviate cost burdens and to minimize variability in cell production, which may increase the availability of engineered cells upon commercialization. This partnership combines CBMG’s scientific expertise in the manufacturing of CAR-T and stem cell production in China and GE Healthcare’s renowned expertise in the design and development of innovative manufacturing technologies for the biopharmaceutical industry.
 

Co-development agreement: Evotec and Sanofi

Evotec (Germany; www.evotec.com) have announced that its strategic alliance with Sanofi (France; www.sanofi.com) in the field of diabetes has reached an important milestone, triggering a payment of EUR 3.0 million to Evotec, for achieving pre-clinical proof-of-concept. This milestone was achieved under the agreement between Evotec and Sanofi signed in August 2015. The goal of this collaboration is to develop a beta cell replacement therapy based on functional beta cells derived from human stem cells. In addition, Sanofi and Evotec also aim to use human beta cells for high-throughput drug screening to identify small molecules or biologics beneficial for beta cell activity.
 

Co-development agreement: Upside and AMEDD

Upside Biotechnologies (New Zealand; www.upside.nz) and the US Army Medical Research and Materiel Command (MD, USA; http://mrmc.amedd.army.mil/) have entered into a collaboration agreement on both the scientific and regulatory aspects of Upside’s engineered skin product development. Upside has been developing a skin replacement treatment for patients suffering major burns, and the US Army Medical Research and Materiel Command has an unpararelled depth of experience in the development and treatment of major burns.
 

Collaboration agreement: Be The Match and Magenta

Be The Match BioTherapies (MN, USA: www.bethematchbiotherapies.com) has entered into a strategic partnership with Magenta Therapeutics (MA, USA; www.magentatx.com) to improve transplant outcomes and expand the application of stem cell transplantation into disease indications that include autoimmunity, serious inherited immune and metabolic disorders, blood defects and blood cancers. Be The Match is also participating in Magenta’s Series B financing round, its first equity investment as an organization. Under the terms of the collaboration agreement, Be The Match and Magenta will explore opportunities to work together across Magenta’s discovery, clinical development and product delivery efforts. The collaboration leverages a wide range of Be The Match research assets and services, including the National Marrow Donor Program® (NMDP)/Be The Match® marrow registry, the largest in the world with nearly 16 million volunteer marrow donors. Magenta may also collaborate with Be The Match in the design of clinical trials and of its cell therapy delivery platform and services.
 

Collaboration agreement: CDI and Harvard

Cellular Dynamics International (CDI; WI, USA; www.cellulardynamics.com) has entered into a collaboration agreement with the Harvard University Stem Cell Institute (HSCI; MA, USA; www.hsci.harvard.edu) extending to its affiliated hospitals and partners in the biomedical industry. The objective of this partnership is to increase the availability of induced pluripotent stem cells (iPSC) and services. The goal is to make iPSCs and applications technology more accessible so that researchers across disciplines and the various institutions affiliated with HSCI can more successfully, rapidly and cost-effectively pursue the promise of stem cell science and regenerative medicine. Under the terms of the agreement, CDI will collaborate with HSCI’s iPSC Core Facility by providing iPSC projects seed funding, iPSC science and technology training and educational programs and the opportunity to bring iPSC research materials to full scale production at CDI production facilities.
 

Distribution agreement: CDI and STEMCELL Technologies

Cellular Dynamics International (CDI; WI, USA; www.cellulardynamics.com) has signed a distribution agreement with STEMCELL Technologies (BC, Canada; www.stemcell.com) to make CDI’s iPSC technology widely available to researchers worldwide. Under the terms of the agreement, STEMCELL Technologies will distribute CDI’s iCell® catalog of products in North America, Europe, and Singapore, with other countries under consideration. CDI’s iCell products are differentiated human iPSC-derived cells, which include cardiomyocytes, hepatocytes and others, totaling up to 12 cell types.

Manufacturing agreement: PolarityTE and University of Utah

PolarityTEâ„¢ (UT, USA; www.polarityte.com) has signed a manufacturing agreement with Cell Therapy and Regenerative Medicine Center at the University of Utah School of Medicine (UT, USA; www.medicine.utah.edu) for launch of the product SkinTEâ„¢. PolarityTE proprietary technology employs a patient’s own cells for the healing of full-thickness functionally polarized tissues.
 

Partnership agreement: Mundipharma and Kolon Life Science

Mundipharma (UK; www.mundipharma.com) has entered into a strategic partnership with Kolon Life Science (Korea; www.kolonls.co.kr) to market Invossa, a cell and gene therapy for the treatment of osteoarthritis that contains non-transformed chondrocytes and transduced chondrocytes. The product is expected to be approved by the South Korean Ministry of Food and Drug Safety in the second quarter of 2017. Kolon Life Sciences is the official Asia licensee for US regenerative medicine company TissueGene (MD, USA; www.tissuegene.com), which developed Invossa. Under the terms of the partnership, Mundipharma will be responsible for the marketing and distribution of Invossa to general and semi-private hospitals, while Kolon Pharma will take responsibility for the general practitioner market. Invossa is exported globally by Mitsubishi Tanabe.

Achievements
 

BioCardia

BioCardia (CA, USA; www.biocardia.com) published a preclinical study showing that their proprietary Helixâ„¢ transendocardial delivery system results in superior cell delivery and greater myocardial retention of stem cell therapy [1]. Use of the Helix system resulted in 18 times higher retention of injected cells in the myocardium than intra-coronary artery infusion, and three times higher than direct injection to the heart using a straight needle. The Helixâ„¢ system is currently being used in the company’s investigational CardiAMP® therapy, designed to deliver a high dose of a patient’s own bone marrow cells directly to the point of cardiac dysfunction with the goal to stimulate the body’s natural healing mechanism after a heart attack. This therapeutic approach is being evaluated in the CardiAMP Heart Failure Trial, a phase III, multi-center, randomized, double-blinded, sham-controlled study enrolling up to 260 patients at up to 40 centers worldwide.
 

Gamida Cell

Gamida Cell (Israel; www.gamida-cell.com) have published positive clinical outcomes from an investigator-initiated study of NiCord®-transplanted patients, highlighting data underscoring key benefits associated with NiCord® over standard umbilical cord blood (UCB) transplantation, including shorter time to engraftment, fewer infections and shorter hospital stays [2]. The study compared 18 consecutive NiCord-transplanted patients and 86 consecutive standard umbilical cord blood (UCB) transplanted patients with all patients receiving TBI-based myeloablative conditioning.
 

RenovaCare

RenovaCare (NY, USA; www.renovacareinc.com) highlighted an analysis of treatment results on a variety of wide-area and severe burn injuries [3]. The treatment method, which involved isolating and spraying the patient’s own skin stem cells on the burn wounds, is the technology underlying RenovaCare’s patented CellMistâ„¢ and SkinGunâ„¢. The published analyses are encouraging as patients were successfully treated with the technology regardless of the source of the burn.

Launching new projects, products & services
 

BioTime

BioTime (CA, USA; www.biotimeinc.com) has announced the formation of AgeX Therapeutics, Inc. to consolidate certain BioTime subsidiaries and programs in the field of interventional gerontology. The formation of AgeX continues the implementation of BioTime’s strategy to simplify its corporate structure and operations as well as focus resources on the continued clinical development of its two lead programs; Renevia®, a proprietary cell delivery matrix designed to facilitate the stable engraftment of transplanted cells that is currently in a pivotal clinical trial, and OpRegen®, a pluripotent cell-derived cell therapy that is in a Phase 1/2a clinical trial for the dry form of age related macular degeneration (dry-AMD). The formation of AgeX provides the new BioTime subsidiary greater flexibility to explore external financing alternatives as well as strategic options to grow its technology platform.
 

Irvine Scientifics

Irvine Scientific (CA, USA; www.irvinesci.com) has introduced PRIME-XV® Hematopoietic Cell Basal Medium. This new formulation follows other media in the PRIME-XV line as it is serum-free, xeno-free basal medium that specifically supports robust expansion of hematopoietic progenitors while maintaining their multipotency, or functional properties needed for cell-based therapies.
 

MedCision

MedCision (CA, USA; www.medcision.com), developer of standardization and automation for cell therapy product handling, have announced a ThawSTAR Early Adopter Program. Effective immediately, MedCision will offer up to 30 companies and institutions that wish to standardize and de-risk their clinical thawing effort free use of a limited number of ThawSTAR instruments for the duration of an early clinical trial phase. This special offer also includes full training and technical support. To ensure optimal clinical outcome, every ThawSTAR platform in the Early Adopter Program will be programmed with a customized algorithm, specific to each unique cell therapy product. The algorithm technology takes into account an understanding that cell size, volume and choice of cryopreservative affect the thawing rate needed to preserve optimal function in a given cell type.
 

NovaBone

NovaBone Products (FL, USA; www.novabone.com) has announced the addition of NovaBone IRM and NovaBone IRM MacroPOR to its portfolio of biologically active bone graft substitutes. NovaBone IRM and NovaBone IRM MacroPOR are among the most advanced bioactive synthetic bone graft substitutes available to the orthopedic community today. Both formulations optimize osteogenesis, uniquely signal and stimulate osteoblastic activity, and offer angiogenic potential. By design, both formulations are highly irrigation resistant with excellent cohesion and handling properties.
 

Progenitor Life Sciences

Progenitor Life Sciences (UT, USA; www.progenitorlifesciences.com) has introduced Stealthâ„¢ platform enabling sourcing of cells from a wide range of healthy donors, without donor-recipient HLA-matching. Stealthâ„¢-engineered cells also reduce immune-mediated adverse events caused by unintended activation of the recipient’s immune system following infusion of engineered cell therapies. In off-the-shelf therapies, allogeneic cells sourced from healthy donors undergo industrial-scale engineering and production in a centralized facility, producing a ready-to-ship product that requires minimal preparation before infusion, making broad distribution possible. A barrier to off-the-shelf allogeneic therapies has been the cost of HLA-matching and cross-typing between donor cells and recipients. HLA-incompatible cells trigger MHC-I-mediated destruction by recipient cytotoxic T lymphocytes (CTLs). Progenitor’s Stealthâ„¢ technology disrupts MHC-I via a non-viral method, while maintaining cell-surface MHC-I expression. Consequently, Stealthâ„¢-engineered cells evade both recipient CTL responses and NKC-mediated “missing-self’ responses triggered by absence of cell-surface MHC-I.
 

Provia

Provia Laboratories (MA, USA; www.provialabs.com) have launched a comprehensive library of stem cell material available for researchers. The library includes ready to ship mesenchymal stem cells (MSCs) from dental pulp (DPSC) found in human third molars or human exfoliated deciduous teeth, as well as from follicular tissue, adipose tissue, placental tissue, and umbilical cord tissue. Provia is currently developing inventory of MSCs from bone marrow, chorionic villi, and amniotic fluid. 

References

[1]           Mitsutake Y, Pyun WB, Rouy D et al. Improvement of Local Cell Delivery Using Helix Transendocardial Delivery Catheter in a Porcine Heart. Int. Heart J. 58(3), 435-440 (2017).

[2]           Anand S, Thomas S, Hyslop T et al. Transplantation of Ex Vivo Expanded Umbilical Cord Blood (NiCord) Decreases Early Infection and Hospitalization. Biol. Blood Marrow Transplant. 23(7), 1151-1157 (2017).

[3]           Esteban-Vives R, Choi MS, Young MT et al. Second-degree burns with six etiologies treated with autologous noncultured cell-spray grafting. Burns 42(7), e99-e106 (2016).