Industry update: business development

Latest developments compiled from 01 — 31 January 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 01 — 31 January 2015, scheduled to be published in Volume 10 Issue 4 of Regenerative Medicine.

Collaborations, partnerships & alliances

Codevelopment agreement: Intellia and Novartis

Intellia Therapeutics (MA, USA; http://intelliatx.com) announced a five-year research and development collaboration with Novartis (Switzerland; www.novartis.com) to accelerate the ex vivo development of new clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9-based therapies using chimeric antigen receptor (CAR) T cells and hematopoetic stem cells (HSCs). This collaboration comes only three months after Intellia was launched. Under the terms of the agreement, Novartis receives exclusive rights to develop all collaboration programs focused on engineered CAR T cells. Within HSCs, Novartis and Intellia will jointly advance multiple programs, and have agreed to a process for assigning development and ownership rights, which will enable Intellia to develop its own proprietary internal HSC pipeline. In addition to increasing its equity holding in Intellia, Novartis is making an upfront payment, and providing technology access fees and funding for research and development (R&D) programs during the five-year term of the collaboration. Intellia is also eligible to receive downstream success-based milestones and royalties. Intellia will gain access to certain Novartis intellectual property and technology for the development of its own product pipeline. Intellia also reserves the right to pursue additional enabling partnerships in other areas of therapeutic interest.

Collaboration agreement: Cord Blood Registry and Cellular Dynamics International

Cord Blood Registry (CBR; CA, USA; www.cordblood.com) and Cellular Dynamics International (CDI; WI, USA; www.cellulardynamics.com) have entered into a research collaboration to reprogram newborn stem cells from both umbilical cord blood and umbilical cord tissue collected, processed and cryopreserved under CBR’s protocols into induced pluripotent stem cells (iPSCs) using CDI’s proprietary methods. Through this research collaboration, CBR will provide to CDI multiple de-identified, research-donated and cryopreserved umbilical cord blood units as well as multiple de-identified units of mesenchymal stem cells (MSCs) isolated from previously cryopreserved cord tissue. CDI will reprogram the cells into iPSCs and confirm their pluripotent nature.

Collaboration agreement: Cord Blood Registry and China Cord Blood Corporation

Cord Blood Registry (CBR; CA, USA; www.cordblood.com) and China Cord Blood Corporation (CCBC; China; www.chinacordbloodcorp.com), entered into a memorandum of understanding regarding their strategic collaboration in China and the US. The two prominent cord blood banks will share data on cord blood collection, processing and storage to advance international standards. The companies will also work together under the memorandum, to develop a family disease registry for CCBC’s clients in China and jointly support newborn stem cell-related clinical trials in the US and China.

Collaboration and license agreement: Amgen and Kite

Amgen (CA, USA; www.amgen.com) and Kite Pharma (CA, USA; www.kitepharma.com) have entered into a strategic research collaboration and license agreement to develop and commercialize the next generation of novel CAR T cell immunotherapies based on Kite’s engineered autologous cell therapy (eACTâ„¢) platform and Amgen’s extensive array of cancer targets. The collaboration brings together Amgen’s commitment to and capabilities in advancing new approaches in immuno-oncology and Kite’s industry-leading presence in CAR T cell therapy. Under the terms of the agreement, Amgen will contribute cancer targets, and Kite will leverage its proprietary CAR platform, R&D and manufacturing capabilities, and expertise. Kite will be responsible for conducting all preclinical research and cell manufacturing and processing through Investigational New Drug (IND) filing. Each company will then be responsible for clinical development and commercialization of their respective CAR therapeutic candidates, including all related expenses. Kite will receive from Amgen an upfront payment of US$ 60 million, as well as funding for R&D costs through IND filing. Kite will be eligible to receive up to US$ 525 million in milestone payments per Amgen program based on the successful completion of regulatory and commercialization milestones, plus tiered high single- to double-digit royalties for sales and the license of Kite’s intellectual property for CAR T cell products. Amgen is eligible to receive up to US$ 525 million in milestone payments per Kite program, plus tiered single-digit sales royalties. Further terms of the agreement are not being disclosed.

Cooperative research agreement: MitoGenome, BoyaLife and Sooam

Shoukhrat Mitalipov of the Oregon Health & Science University (OR, USA; www.ohsu.edu) founder of MitoGenome Therapeutics (no website), the first company aimed to commercialize mitochondrial DNA (mtDNA) replacement in oocytes to treat inherited mitochondrial diseases, have agreed to jointly work on the mechanisms of cloning with BoyaLife Group (China; www.boyalifegroup.com/english/index.aspx) and Sooam Biotech Research Foundation (Korea; http://en.sooam.com/main.html), dog cloning company led by Woo Suk Hwang, the scientist who once fraudulently claimed to have created human embryonic stem cells (hESC) matched to human donors. Boyalife, which has a number of stem cell— and regenerative medicine—related research and commercial efforts, will put up about US$ 93 million. They say that their initial work will focus on animal cloning but eventually move on to work with human materials. The laboratory will be placed in China to avoid Korea’s strict bioethics regulations.

License agreement: Catapult and iPS Academia

The Cell Therapy Catapult (UK; https://ct.catapult.org.uk), which is focused on the development of the UK cell therapy industry to increase the nation’s health and wealth, has signed a patent license agreement with iPS Academia Japan (www.ips-cell.net/e), which represents a pivotal step in the development of the UK’s Good Manufacturing Practices (GMP) iPSC bank. Under the agreement, the Cell Therapy Catapult is permitted to sub-license, manufacture and commercialize iPSC lines for use in early-stage research and clinical trials. With iPSC gaining in importance as a source of new therapies and the first products in clinical trials, the cell bank is of strategic importance to the UK cell therapy community. Under the terms of the agreement with iPS Academia Japan, which is worldwide and non-exclusive, the Cell Therapy Catapult is able to create, sell and distribute iPSC lines for research and clinical use (including its own). Any organization undertaking clinical research or commercial use based on the cell lines will need an additional sub-license, for which not-for-profit organizations, charities and universities will not be required to pay fees as far as their activities are for non-commercial purposes. Establishment of the cell bank was announced in 2013 with an initial investment of US$ 3 (£ 2) million.

Launching new projects, products and services

ESI BIO

ESI BIO (CA, USA; www.esibio.com), the stem cell products division of BioTime (CA, USA; www.biotimeinc.com) announced that its cGMP and research grade stem cell lines, reagents and cell matrix products are now available in the UK and Ireland through 2BScientific (UK; www.2bscientific.com).