Industry update: business development

Written by Dusko ILIC

Latest business developments compiled from 1 April—31 May 2015

Latest developments in the field of stem cell research and regenerative
medicine compiled from publicly available information and press releases
from non-academic institutions 1 April—31 May 2015, scheduled
to be published in Volume 10 Issue 6 of Regenerative Medicine.

Collaborations, partnerships & alliances

Co-development agreement: Catapult and Asymptote

Cell Therapy Catapult (UK; and Asymptote (UK; are combing forces to advance the development of a novel point-of-care Thawing System for Cell-Based Therapies. This device is intended to solve a global barrier to commercialization of cell therapies, by simplifying the delivery of these therapies to patients by addressing significant challenges of delivery at the point-of-care.

Co-development agreement: L’Oreal and Organovo

L’Oreal (France; is teaming up with Organovo (NY, USA; to 3D-print human skin. The printed skin would be used in product tests.

Co-development agreement: Takeda and CiRA

Takeda (Japan, and Center for iPS Cell Research Application (CiRA) of Kyoto University (Japan, have signed a US$ 268-million (32 billion yen), 10-year agreement to accelerate multiple research projects using induced pluripotent stem cells (iPSC) as standalone treatments and in the discovery of new drugs. Under the deal, Takeda will provide research facilities at its Shonan Research Center and collaborative funding of US$168 million (about 20 billion yen) over a 10-year period, as well as more than 12 billion yen (about $100 million) worth of research support.

Collaboration agreement: Juno and Editas

Juno Therapeutics (WA, USA; and Editas Medicine (MA, USA; have entered in an exclusive collaboration focused on creating chimeric antigen receptor (CAR T) and high-affinity T cell receptor (TCR) therapies to treat cancer. The companies will pursue three research programs together utilizing Editas’ genome-editing technologies, including CRISPR/Cas9, with Juno’s CAR and TCR technologies. Under the terms of the agreement, Juno will pay Editas an upfront payment of US$ 25 million and up to US$ 22 million in research support over the next five years across the three programs in the alliance. Editas is also eligible to receive future research, regulatory, and commercial sales milestones in excess of US$ 230 million for each program. Following the approval of any products resulting from the alliance, Editas is also eligible to receive tiered royalties.

Collaboration agreement: Juno and Fate

Juno Therapeutics (WA, USA; and Fate Therapeutics (CA, USA; have executed a strategic research collaboration and license agreement to identify and utilize small molecules to modulate Juno’s genetically engineered T cell product candidates to improve their therapeutic potential for cancer patients. Through the four-year research and development collaboration, Fate will be responsible for screening and identifying small molecules that modulate the biological properties of engineered T cells. Juno will be responsible for the development and commercialization of engineered T cell immunotherapies incorporating Fate’s small molecule modulators. Juno has the option to extend the exclusive research term for two years through an additional payment and continued funding of collaboration activities.

Collaboration agreement: Lonza and Nikon

Lonza (Switzerland, and Nikon (Japan; have entered an exclusive collaboration in the field of cell and gene therapy manufacturing in Japan. Nikon will have access to Lonza’s quality and operating systems, facility design and ongoing consulting services for the establishment of a wholly owned Nikon cell and gene therapy contract manufacturing business.

Collaboration agreement: MaxCyte and John Hopkins

MaxCyte (MD, USA; and The Johns Hopkins Kimmel Cancer Center (MD, USA; have entered in strategic research collaboration to develop unique CAR T-cell therapies, which harness patients’ own immune systems to combat cancers. MaxCyte’s unique approach to CAR cell therapy allows targeting of solid tumor cancers by enabling control over the on-target, off-tumor toxicity, which limits other CAR therapies to hematological cancers. MaxCyte achieves this by introducing the CAR construct as a transiently expressing messenger RNA (mRNA), thus allowing control of the duration of expression and toxicity against target antigens in normal tissue. This unique approach also avoids the cell expansion step required for standard approaches, dramatically reducing manufacturing time and expense for CAR therapies from days or weeks to a matter of hours. No financial terms are disclosed.

Collaboration agreement: MedImmune and Juno

MedImmune (MD, USA;, research and development arm of AstraZeneca (UK;, and Juno Therapeutics (WA, USA; have entered into a new collaboration to conduct combination clinical trials in immuno-oncology with one of Juno’s investigational CD19-directed CAR T cell candidates and MedImmune’s investigational human monoclonal antibody directed against programmed cell death ligand 1 (PD-L1), immune checkpoint inhibitor, MEDI4736. Under the initial development plan, both companies will explore the safety, tolerability and preliminary efficacy of the combination therapy as a potential treatment for patients with non-Hodgkin lymphoma. Under the terms of the non-exclusive collaboration, MedImmune and Juno will jointly co-fund the initial Phase Ib study, which is expected to begin later in 2015. The companies will also explore the combination of MEDI4736 with a next-generation, Juno-developed fully human CD19-directed CAR T cell candidate.

License agreement: Argos and Lummy

Argos Therapeutics (NC, USA; and Hong Kong subsidiary of Chongqing Lummy Pharmaceutical (China; have entered into a license agreement. Under the terms of the agreement, Lummy will license from Argos the rights to manufacture, develop and commercialize AGS-003, Argos’ investigational immunotherapy for the treatment of cancer based on proprietary Arcelis® technology platform, in China, Hong Kong, Taiwan and Macau.

License agreement: Bellicum and LUMC

Bellicum Pharmaceuticals (TX, USA; has entered into a license agreement with Leiden University Medical Center (LUMC; the Netherlands; for worldwide rights to develop, manufacture and commercialize high-affinity TCR (T cell receptor) product candidates targeting solid tumors expressing the preferentially-expressed antigen in melanoma or PRAME. TCRs are engineered T cells that are activated when in the presence of target antigens in cancer cells. The lead TCRs under this license agreement have been shown to have a high affinity to PRAME, a cancer antigen that is preferentially expressed in melanomas, sarcomas, neuroblastomas and other solid tumors, but generally not expressed in normal tissue. Bellicum’s first TCR product candidate under this agreement, BPX-701, targets PRAME and is expected to enter Phase 1/2 clinical trials before the end of 2015. BPX-701 incorporates Bellicum’s proprietary safety mechanism, CaspaCIDe®, for improved control over the cells.

License agreement: BioTime and City of Hope

BioTime (CA, USA; announced a nonexclusive License Agreement between BioTime’s subsidiary ES Cell International (ESI; CA, USA; and Beckman Research Institute of the City of Hope (CA, USA; through which ESI’s clinical-grade human embryonic stem (hESC) will be manufactured and provided to clinical collaborators, including medical research organizations intent on using the hESC to develop and commercialize therapeutic products to treat human disease. The Agreement is a critical part of BioTime’s strategy to leverage third party funding and generate future revenues by placing its hES cells in programs developing a wide array of therapeutic applications made possible with pluripotent stem cell technology, products that are currently not being developed by BioTime or its subsidiaries. Successful medical research organizations will need to obtain separate licenses from BioTime in order to advance their products based on ESI’s hES cells into clinical trials and commercialization. Those licenses may entitle BioTime to receive additional revenues such as milestone payments related to the attainment of clinical trial and commercial milestones and royalties on product sales. BioTime and BioTime subsidiaries will retain the rights to manufacture their own stem cell-based products as well as to license rights to other third parties.

License agreement: STEMCELL Technologies and Salk Institute

STEMCELL Technologies (BC, Canada; has signed a license agreement with the Salk Institute for Biological Sciences (CA, USA; for the rights to commercialize BrainPhysâ„¢ Neuronal Medium [1]. Classical culture media used for neuronal culture applications were found to impair action potential generation and synaptic communication. In order to improve the physiological relevance of neuronal culture conditions, Salk scientists developed BrainPhys medium to better represent the environment experienced by neurons in the human brain. This formulation promotes optimal neuronal and synaptic activity while supporting long-term culture of both human pluripotent stem cell-derived and primary tissue-derived neurons.

Partnership agreement: REGiMMUNE, JDRF and Pfizer

REGiMMUNE (Japan; and JDRF (NY, USA; have entered in a partnership agreement, along with financial assistance and scientific expertise from Pfizer (NY, USA;, for a research collaboration to develop an antigen-specific immunotherapy utilizing REGiMMUNE’s proprietary αGalCer/liposome platform for immunological tolerance for the treatment of type 1 diabetes (T1D). Under the terms of the collaboration, REGiMMUNE will develop an antigen-specific therapeutic liposome that potentially prevents or delays the onset of T1D and induces immunologic tolerance. αGalCer/liposome platform, when utilized with T1D autoantigens, may be effective in inducing T1D-specific Tregs and specific immune tolerance for T1D patients without promoting broad immunosuppression associated side effects as typically seen with other therapeutics.

Launching new projects, products and services

Bone Therapeutics

Bone Therapeutics (Belgium; has established its US based subsidiary, Bone Therapeutics USA, in Boston, MA, in line with its strategy as outlined at the time of its recent initial public offering. The establishment of the US headquarters is the first milestone in a process that will lead to the initiation of Bone Therapeutics’ clinical trials for fracture repair and osteonecrosis in the US.


Celprogen (CA; USA; has developed STEMC5007 and STEM7007, synthetic molecules capable of stimulating and activating the internal stem cell population. These molecules can be administered by the following methods: intravenous, intra-peritoneal and intramuscular injections, and oral doses. The molecule STEMC5007 activates adult stem cell populations including progenitor stem cells, mesenchymal, skin, human hair follicle stem cells and adipose stem cells, whereas STEMC7007 activates adult pancreatic b- and a-cell populations. The STEMC7007 molecule establishes normal b-cell function in diabetic cell populations by producing and secreting insulin and glucagon in in-vitro cell culture system.


Sorrento Therapeutics (CA, USA; has formed a wholly owned subsidiary, TNK Therapeutics, Inc. This subsidiary will focus on developing Chimeric Antigen Receptor Tumor-attacking Neukoplast® (CAR.TNKs)-based therapies as well as other complementary cellular and immunotherapies targeting both solid tumors and hematological malignancies. In December 2014, Sorrento entered into a global exclusive partnership with Conkwest (CA, USA;, an immunotherapy company developing proprietary Neukoplast (NK-92), a Natural Killer (NK) cell-line based therapy, to jointly develop next-generation CAR.TNK. The CAR.TNK technology platform combines Conkwest’s Neukoplast cell line with Sorrento’s proprietary G-MABâ„¢ fully human antibody technology and CAR designs to further enhance the anti-cancer potency and tumor targeting of Neukoplast. Under the terms of this agreement, both companies will jointly select CAR.TNKs to be developed with the ‘lead company’ responsible for advancing the candidates into the clinic. Profit sharing from future sales and potential strategic collaborations with other pharmaceutical partners will be determined by the development stage of the drug candidates.



Kallistem (France; produced fully formed human spermatozoa in the laboratory setting, using patient testicular biopsies containing only immature germ cells, or spermatogonia.

Ocata and CHA

Ocata Therapeutics (MA, USA;, and CHA Biotech (Korea, have published a study that includes positive results in Asian patients who were treated with Ocata’s proprietary hESC-derived retinal pigment epithelial cells (RPE), manufactured using its DeltaCellâ„¢ Technology [2]. The study features twelve months of post-transplant follow-up data from four Asian patients; two with Stargardt’s macular degeneration and two with dry age-related macular degeneration. In this study there was no evidence of adverse proliferation, tumorigenic behavior, ectopic tissue formation, or other serious safety issues related to the transplanted cells. Although designed as a safety study, visual acuity improved 9—19 letters in three patients and remained stable (+1 letter) in one patient. Vision was measured using the widely accepted standard for visual acuity testing, the Early Treatment Diabetic Retinopathy Study visual acuity exam.


[1] Song WK, Park KM, Kim HJ et al. Treatment of macular degeneration using embryonic stem cell-derived retinal pigment epithelium: preliminary results in Asian patients. Stem Cell Reports 4, 860-72 (2015).

[2] Bardy C, van den Hurk M, Eames T et al. Neuronal medium that supports basic synaptic functions and activity of human neurons in vitro. Proc Natl Acad Sci USA 112, E2725-34 (2015).