Industry Update: Business Development

Latest developments compiled from 1 October until 30 November 2014

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 October until 30 November 2014, scheduled to be published in Volume 10 Issue 2 of Regenerative Medicine.

Collaborations, partnerships & alliances

Co-development agreement: Fibrocell and Interexon

Living Cell Technologies (New Zealand; www.lctglobal.com) has formed collaboration with the Centre for Brain Research at the University of Auckland (www.fmhs.auckland.ac.nz/en/faculty/cbr.html). The research collaboration will identify additional neurodegenerative disease targets for clinical studies of Living Cell Technologies’s lead product NTCELL®. NTCELL is an alginate-coated capsule containing clusters of neonatal porcine choroid plexus cells. After transplantation NTCELL functions as a biological factory producing nerve growth factors to promote new central nervous system growth and repair disease induced nerve degeneration.

Collaborative and licensing agreement: Transposagen and Janssen

Transposagen Biopharmaceuticals (KY, USA; www.transposagenbio.com) have entered into a research collaboration and worldwide license agreement with Janssen Biotech (PA, USA; www.janssenbiotech.com) to develop allogeneic Chimeric Antigen Receptor T-cells (CAR-T). To date, CAR-T therapies have shown promise in early human clinical trials for the treatment of blood cancers and allogeneic CAR-Ts have the potential for use as off-the shelf cancer treatments without the need of matching donor with recipient. Transposagen will be using its proprietary genome editing technologies, including the piggybackâ„¢ Footprint-Freeâ„¢ Gene Editing System, to create the allogeneic CAR-T therapies. Under the agreement, Janssen has exclusive rights to any allogeneic CAR-T therapy that is jointly developed by Transposagen and Janssen. In addition, Janssen has received a non-exclusive research license to utilize Transposagen’s proprietary gene editing technologies for gene and cell therapy solutions for treating diseases with significant unmet medical need. Transposagen will retain the rights to develop autologous CAR-T therapies and CAR-T therapies using Natural Killer (NK) cells or NK-like cells. Janssen will pay Transposagen up to US$ 292 million per CAR-T therapeutic, which includes an up-front fee and potential development, regulatory, and commercial milestone payments. Transposagen will also receive tiered royalties on net sales of any allogeneic CAR-T products that are commercialized by Janssen. Transposagen will enter into 3-year research collaboration with Janssen whereby both companies will work together on preclinical research. Janssen will be responsible for manufacturing and commercialization of allogeneic CAR-T therapies.

Licensing and distribution agreement: Histogen and JPGH

Histogen Aesthetics (CA, USA; www.cellceuticalskincare.com), formed in 2008 as a subsidiary of Histogen (CA, USA; www.histogen.com), has entered a license and distribution agreement with JPGH (no website found), to bring CellCeuticals Regenerative Skin Treatments to Greater Asia. Fibroblasts are grown under unique conditions, producing a complex of naturally-secreted proteins and synergistic bio-products known to stimulate skin cells to regenerate and rejuvenate tissues.

Launching new projects, products and services

Baker

The Baker Company (ME, USA; www.bakerco.com) launched Cultivoâ„¢, a new CO₂ incubator designed to prevent contamination and deliver precise, stable and user-defined control over three variables critical for optimal cell growth – temperature, CO₂ concentration and relative humidity – all without noticeable condensation during normal operation. The precise, stable and user-defined environmental conditions Cultivo provides are possible through the use of InteliCELLâ„¢, a sophisticated proportional-integral-derivative (P.I.D.) control algorithm. By accounting for factors that other technologies leave as unknown, InteliCELL consistently maintains user-defined set points, giving users ultimate control with minimal effort.

Cellular Dynamics International

Cellular Dynamics International (CDI; WI, USA; www.cellulardynamics.com) is getting a US$ 1.2 million contract from the National Eye Institute, part of the NIH, in an effort to fight macular degeneration, a condition that leads to loss of vision. By reprogramming skin and blood samples from patients with age-related macular degeneration, CDI will create induced pluripotent stem cells (iPSC) and will turn them into human retina cells. The cells will be put back into the patient’s eyes to treat the disorder. The National Eye Institute has chosen ten patients for a pilot study. This will be the first study in the US using a patient’s own reprogrammed cells.

Global Stem Cells Group

Global Stem Cells Group (FL, USA; www.stemcellsgroup.com) has announced the launch of the official Regenestem Network website (www.regenestemnetwork.com), dedicated to promoting the world’s largest and most recognizable membership organization of regenerative medicine practitioners. The Regenestem Network was developed to promote the Global Stem Cells Group’s expansion program and establish affiliate representatives able to bring stem cell treatments, therapies, training courses and conferences to communities worldwide. In order to become a part of the Regenestem Network, medical practitioners are required to have more than five years experience in the health care industry that includes experience in regenerative medicine.

NYSCF

The New York Stem Cell Foundation (NYSCF; NY, USA; www.nyscf.org) Research Institute, through the launch of its repository in 2015, will provide for the first time the largest-ever number of stem cell lines available to the scientific research community. Initially, over 600 iPSC lines and 1,000 cultured fibroblasts from over 1,000 unique human subjects will be made available, with an increasing number available in the first year. To collect these samples, NYSCF set up a rigorous human subjects system that protects patients and allows for the safe and anonymous collection of samples from people interested in participating in research. A pilot of over 200 of NYSCF’s iPSC lines is already searchable on an online database: www.nyscf.org/repository.

TEL

Tokyo Electron Limited (TEL; Japan; www.tel.com) opened, through its European subsidiary Tokyo Electron Europe Limited (TEE), the company’s open innovation center at Stevenage Bioscience Catalyst (SBC) in the UK. The goal is to develop technologies to quantify and standardize cell quality with the National Institute for Biological Standards and Control (NIBSC; www.nibsc.org), as well as stem cell researchers in UK academia. TEL has unique experience of automation and quality management frameworks from decades of innovation and development in the semiconductor industry; it is therefore well placed to lead on the transfer of these techniques into life sciences. Smart Cell Processing Technologies will comprise hardware solutions in the form of equipment for culture and inspection of cells, as well as software algorithms and characterization methods. TEL installed in SBC a fully automated cell culture and inspection system and will orchestrate the technology eco-system partners – fifteen companies, which will jointly develop Smart Cell Processing Technologies. This is expected to lead to the generation of a novel system with a shared database and quality management framework to track production processes and ensure cell quality. TEL ultimately aims to develop a vital platform for the UK cell therapy industry, working with ecosystem partners, such as the Cell Therapy Catapult (https://ct.catapult.org.uk) and Roslin Cells (http://roslincells.com). The platform will be evaluated for clinical applications in multiple manufacturing sites.

Achievements

Ocata (former Advanced Cell Technology)

Ocata Therapeutics (MA, USA; www.ocata.com), formerly known as Advanced Cell Technology (MA, USA; www.advancedcell.com), reported that its proprietary iPSC Platelet technology is potentially capable of producing large-scale quantities of universal donor platelets for transfusion in almost any patient [1].

Pluristem

Pluristem (Israel; www.pluristem.com) has completed development of its second major product line, and can now begin manufacturing the cells on a large-scale at its state-of-the art facility in order to meet demand for anticipated studies in a range of hematologic conditions. This second cell product candidate, called Placental eXpanded (PLX)-RAD, was created using Pluristem’s proprietary three-dimensional cell-expansion technology platform [2]. The Company’s first product, PLX-PAD, is already in clinical trials for the treatment of peripheral artery disease, muscle injury and pulmonary arterial hypertension. Pluristem’s development plan for the PLX-RAD cells considers numerous potential clinical indications such as: 1) enhancement of engraftment of transplanted hematopoietic stem cells for the treatment of bone marrow deficiency, which can result from immune system disorders, genetic diseases, and treatment of leukemia and other blood cancers; 2) treatment of bone marrow deficiency in patients who have undergone chemotherapy; 3) treatment of acute radiation syndrome in conjunction with the US National Institutes of Health’s National Institute of Allergy and Infectious Diseases.

Case Western Reserve University (OH, USA; www.case.edu) will conduct a preclinical study of the Company’s PLX-RAD cells. The study will evaluate whether PLX-RAD cells increase the success rate of human umbilical cord blood transplantation.

References:

[1]Feng Q, Shabrani N, Thon JN et al. Scalable generation of universal platelets from human induced pluripotent stem cells. Stem Cell Reports. 3(5), 817-831 (2014).

[2]Gaberman E, Pinzur L, Levdansky L et al. Mitigation of Lethal Radiation Syndrome in Mice by Intramuscular Injection of 3D Cultured Adherent Human Placental Stromal Cells. PLoS One. 8(6), e66549 (2013).