Industry Update: Business Development

Written by Dusko ILIC

Latest business developments compiled from April 01 — May 31, 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 April 01 — May 31 2016., scheduled to be published in Volume 11 Issue 6 of Regenerative Medicine.

Collaborations, partnerships & alliances

Collaboration agreement: Neurologica and Alcyone

Nanologica (Sweden; has entered into a License and Collaboration Agreement with Alcyone Lifesciences (MA, USA; to develop a novel therapeutic platform named Abela; ‘to breathe’ in Hebrew. The partners want to combine Nanologica’s NLAB SilicaTM with Alcyone’s proprietary drug delivery platform technology. The goal is to develop and commercialize through partnership with biopharma a treatment of certain motor neuron disorders, including Amyotrophic Lateral Sclerosis, based on targeted and sustained delivery of trophic factors in combination with human embryonic stem cell (hESC)-derived cells. The role of trophic factors is to rescue degenerating neurons or to support survival of transplanted stem cells in patients with ALS or other motor neuron disorders. According to the agreement, Alcyone will operate the Abela program and will be responsible for further development and financing of the program through collaboration with biopharma.

Collaboration agreement: TxCell and OSR

TxCell (France; and Ospedale San Raffaele (OSR; Italy; have entered into a strategic R&D collaboration. The development part of the collaboration will focus on the non-clinical development of Chimeric-Antigen-Receptor (CAR) engineered regulatory T (Treg) cells for the treatment of Lupus Nephritis. Lupus Nephritis is one of the most serious complications of Lupus, a chronic autoimmune disease affecting over 5 million people worldwide. TxCell scientists identified a first relevant antigenic target for its CAR-Treg cellular therapy product. They successfully created a CAR-Treg product candidate by engineering forkhead box P3 (FOXP3)+ Treg cells with a CAR. After their isolation from the blood of patients, FoxP3+ Treg cells are genetically modified by transduction with CAR. The CAR introduced into FoxP3+ Treg cells is designed to allow FoxP3+ Treg cell activation and immuno-modulation through in vivo recognition of a protein present in inflamed areas in patients suffering from autoimmune and chronic inflammatory diseases. This CAR integrates the binding domain of a pathogenic antibody from patients suffering from Lupus Nephritis. As per the terms of the agreement, TxCell and OSR will conduct the nonclinical pharmacology and toxicology studies with CAR-Treg cells to prepare for a first-in-man study in Lupus Nephritis patients.

Commercialization agreement: Juno and Celgene

Celgene (NJ, USA; and Juno Therapeutics (WA, USA; announced that Celgene exercised its option to develop and commercialize the Juno CD19 program outside North America and China. With the exercise of this option, Celgene will pay Juno a fee of US$ 50 million and the companies will now share global development expenses for products in the CD19 program. Celgene has commercial rights outside of North America and China and will pay Juno a royalty at a percentage in the mid-teens on any future net sales of therapeutic products developed through the CD19 program in Celgene’s territories. Juno retains commercialization rights in North America and China. Juno currently has three CD19-directed product candidates in clinical development.

Licensing agreement: Cell Therapy and Daiichi Sankyo

Cell Therapy (UK; granted the Japan license for its innovative cardiac regeneration medicine, Heartcelâ„¢ (immuno-modulatory progenitor [iMP] cells) to Daiichi Sankyo (Japan; Daiichi Sankyo will undertake all development, regulatory and commercial activities for iMP cells in the territory of Japan only, while Cell Therapy retains its worldwide rights outside of Japan as well as global manufacturing responsibilities. Under the terms of the agreement, Cell Therapy receives a US$16.7 (£12.5) million upfront licensing fee and additional milestone payments and royalties.

Licensing agreement: Cynata and apceth

Cynata Therapeutics (Australia; has entered into a worldwide license option agreement with apceth (Germany; the use of Cynata’s unique Cymerusâ„¢ technology in combination with genetic modification technologies designed to open up prospective new therapeutic fields to mesenchymal stromal/stem cells (MSCs), particularly the treatment of cancer. The agreement provides for an immediate upfront cash payment to Cynata, followed by a series of success-based milestones, which could potentially total more than US$ 30 (A$ 40) millions. Royalties on product sales will be also payable to Cynata. Given the substantial unmet medical need s that this relationship seeks to address, the agreement has the potential for substantial revenues to Cynata. apceth is presently evaluating Cynata’s technology in its in-house cell culture and genetic modification systems as part of an initial collaboration, expected to conclude toward the end of 2016. Further development by apceth will be pursuant to a development plan to be discussed by both parties and incorporated into the definitive license agreement.

Licensing agreement: STEMCELL Technologies and IMBA

STEMCELL Technologies (BC, Canada; has entered into an exclusive licensing agreement with the Institute of Molecular Biotechnology (IMBA; of the Austrian Academy of Sciences for the rights to develop products for cerebral organoid culture to serve the international research community. Cerebral organoids, or brain organoids, are three-dimensional multicellular structures that mimic brain tissue in vitro and represent a groundbreaking new way to model neurological disease [1, 2]. Deriving cerebral organoids from patient-specific induced pluripotent stem cells (iPSC) enables unprecedented insight into mechanisms of human neural development and disease.

Manufacturing agreement: Juno and WuXi

Juno Therapeutics (WA, USA; and WuXi AppTec (China; have started a new company in China, JW Biotechnology, with a mission to build China’s leading cell therapy company by leveraging Juno’s world-class chimeric antigen receptor and T cell receptor (TCR) technologies together with WuXi AppTec’s R&D and manufacturing platform and local expertise to develop novel cell-based immunotherapies for patients with hematologic and solid organ cancers.

Partnership agreement: CDI and MJFF

Cellular Dynamics International (CDI; WI, USA;, a FUJIFILM company (Japan;, has entered in agreement with The Michael J. Fox Foundation for Parkinson’s Research (MJFF; NY, USA; to derive induced pluripotent stem cells (iPSCs) from 85 people in the MJFF-sponsored Parkinson’s Progression Markers Initiative (PPMI; The collaborators will provide the cells to the scientific community in concert with rich available clinical and imaging data on these study volunteers in service to their shared goal to provide resources to accelerate the development of new therapies.



Osiris Therapeutics (MD, USA; presented 15 clinical and scientific abstracts at the Symposium on Advanced Wound Care & Wound Healing Society Spring Conference, April 2016, in Atlanta, GA, USA. Six clinical studies demonstrated the positive outcomes of Osiris’ cryopreserved placental membrane, Grafix®, cryopreserved placental tissue, Stravixâ„¢, and cryopreserved skin allograft, TruSkinâ„¢. The nine scientific studies demonstrated the characterization and benefits of placental and skin allografts.


Plasticell (UK, has been awarded the 2016 Queen’s Award for Enterprise in Innovation. The award is the UK’s highest accolade for outstanding commercial success as a result of innovation in recognition of Plasticell’s contribution to the advancement of regenerative medicine, cell and gene therapy as well as other areas of biomedical research. Plasticell’s technology, CombiCult®, performs miniaturised screening of large numbers of variables in combination to determine optimal culture conditions for cells grown in the laboratory [3]. CombiCult® allows scientists to perform and compare 10,000 stem cell experiments in parallel, whereas the individual experiments would require around 200 years performed back-to-back. The technology thus greatly accelerates the process of drug discovery and has widespread applications in biomedical R&D, notably in stem cell research and further in industrial manufacture of antibody therapeutics, immunotherapies, cell therapies, gene therapies and research reagents.


[1] Lancaster MA, Renner M, Martin CA et al. Cerebral organoids model human brain development and microcephaly. Nature.501(7467):373-9 (2013)

[2 ]Lancaster MA, Knoblich JA. Generation of cerebral organoids from human pluripotent stem cells. Nat Protoc. 9(10):2329-40 (2014)

[3] Tarunina M, Hernandez D, Johnson CJ et al. Directed differentiation of embryonic stem cells using a bead-based combinatorial screening method. PLoS One. 9(9):e104301 (2014)