Industry Update: Business Development

Latest business developments compiled from 01 — 30 June 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 01 — 30 June 2016, scheduled to be published in Volume 11 Issue7 of Regenerative Medicine.

Collaborations, partnerships & alliances

Co-branding agreement: BI and Corning

Biological Industries (CT, USA; www.bioind.com) announced a co-branding agreement with Corning’s subsidiary, Mediatech (NY, USA; www.corning.com and www.cellgro.com), which will enable cell therapy, research, and manufacturing organizations around the world to purchase the xeno-free NutriStem® human pluripotent stem cell (hPSC) medium in conjunction with Corning’s existing portfolio of stem cell-focused technologies. Before the end of this year, Corning and BI will launch a jointly-branded NutriStem hPSC XF Medium, which will continue to be manufactured by BI, but marketed, distributed and supported worldwide by Corning’s global commercial team.

Co-development agreement CDI and NEI

Cellular Dynamics International (CDI; WI, USA; www.cellulardynamics.com), a FUJIFILM company (Japan; www.fujifilmholdings.com) has entered into a Cooperative Research and Development Agreement with the National Eye Institute (NEI; www.nei.nih.gov), a division of the National Institutes of Health (NIH), to conduct research to advance a cell transplantation therapy for the treatment of retinal degenerative disease using retinal pigment epithelium derived from induced pluripotent stem cells (iPSC-RPE). The collaboration includes the joint development of an optimized biocompatible biodegradable scaffold that is likely to be a critical component for a potential future therapy.

Collaboration agreement: Orig3n and Kangstem

ORIG3N (MA, USA; www.orig3n.com) and Kangstem Biotech (Korea; www.kangstem.com) announced the beginning of a scientific collaboration to study the utility of induced neural stem cells (iNSC) in disease modelling. As part of the collaboration, Kangstem Biotech will provide patient‘specific iNSCs differentiated directly from skin fibroblasts for ORIG3N to use in its disease modelling platform.

Licensing agreement: Bone Biologics and UCLA

Bone Biologics (MA, USA; http://bonebiologics.com) signed an option agreement with the University of California Los Angeles for an opportunity to exclusively license the use of the bone growth factor Nell-1 in the treatment of osteoporosis. Currently, Bone Biologics is funding the development and formulation of a Nell-1 based product for use as a bone graft substitute on bone regeneration in spinal fusion procedures. The new technology would consist of NELL-1, Bone Biologics’ proprietary skeletal specific growth factor, used in combination with DBX®, a proprietary demineralized bone matrix from Musculoskeletal Transplant Foundation (www.mtf.org/index.html).

Licensing agreement: Emmaus and Cell Seed

A licensing agreement between Emmaus Medical (CA, USA; www.emmausmedical.com) and CellSeed (Japan, www.cellseed.com/index-e.html) has been signed to commercialize Epithelial Cell Sheet Corneal Regeneration technology for Limbal Stem Cell Deficiency (LSCD) in the United States. LSCD has been recognized as a major cause, either primary or secondary, of significant visual loss and blindness in many common corneal disorders. The new agreement extends Emmaus’ existing relationship with CellSeed, allowing the company to continue to conduct research and development activities on the epithelial cell sheet for the treatment of LSCD. Emmaus will pay a royalty to CellSeed upon commercialization.

Licensing agreement: TxCell and LIED

TxCell SA (France, www.txcell.com/index.php/en/) signed a strategic R&D collaboration agreement with the Lübeck Institute of Experimental Dermatology (LIED; Germany; www.lied.uni-luebeck.de/home.html). This specific collaboration agreement covers the development of a CAR-Treg-based cellular immunotherapy for bullous pemphigoid, a rare and potentially fatal autoimmune disease. TxCell scientists have already identified a relevant antigenic target for the development of a CAR-Treg product in bullous pemphigoid patients. The CAR construct will be designed to ensure the activation of CAR-Treg cells specifically in the inflammatory skin lesions. TxCell and LIED will conduct non-clinical pharmacology studies with CAR-Treg cells to prepare for a first in-man study in bullous pemphigoid patients. TxCell retains all rights on existing and future programs and products developed under this agreement. Financial terms of the collaboration have not been disclosed.

Licensing agreement: TxCell and Yeda

The European Patent Office has granted Yeda Research and Development. (Israel; www.yedarnd.com) the patent covering all redirected, genetically engineered T regulatory cells (CAR-Tregs) and their use in the suppression of autoimmune and inflammatory diseases (patent identification number: EP 2126054). TxCell has signed an exclusive worldwide licensing agreement with Yeda Research and Development, owner of the patent, to develop and commercialize CAR-Treg products for the treatment of autoimmune and inflammatory diseases, as covered by the patent family.

Licensing and collaboration agreement: CRISPR and Anagenesis

CRISPR Therapeutics (Switzerland; http://crisprtx.com) and Anagenesis Biotechnologies (France, http://anagenesis-biotech.com) announced a strategic in-licensing and collaboration agreement to develop Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 based cell therapies for Duchenne Muscular Dystrophy. Anagenesis Biotechnologies’ proprietary Paraxial Mesoderm Multipotent Cells (P2MCs) technology, which allows differentiation of pluripotent cells, iPSC or embryonic stem cells (ESC), into satellite cells, will support the advancement of CRISPR-based cellular therapies for the treatment of musculoskeletal diseases.

Manufacturing agreement: Amarantus and Lonza

Amarantus BioScience Holdings (CA, USA; www.amarantus.com) announced that the current Good Manufacturing Practices (cGMP) manufacturing process for its Engineered Skin Substitute (ESS) program is now qualified at Lonza Walkersville (MD, USA; www.lonza.com/custom-manufacturing/capabilities-overview/our-sites/walkersville-md-usa.aspx). ESS is a combination of cultured epithelium with a collagen-fibroblast implant that produces a skin substitute that contains both epidermal and dermal components made from a patient’s own skin and expanded to provide permanent wound coverage of large body surface areas. A non-GMP version ESS has already been used in investigator-initiated and compassionate-use clinical settings in over 150 patients, primarily pediatrics, for the treatment of severe burns up to 95% of total body surface area, and also in the treatment of two patients with Giant Congenital Melanocytic Nevi (GCMN). Amarantus can now commence its 10-patient, randomized, placebo-controlled, open-label phase II clinical trial (clinicaltrials.gov identifier: NCT01655407) for the treatment of adult patients (ages 18-40 years old) with deep-partial and full-thickness thermal burns covering ≥50% of total body surface area. The study will compare the safety and efficacy of the autologous ESS with meshed, split-thickness autograft (AG).

Marketing agreement: StemBioSys and Funakoshi

StemBioSys (TX, USA; www.stembiosys.com) has signed a three-year marketing and distribution deal with Funakoshi (Japan; www.funakoshi.co.jp/exports) to bring its stem cell production products to Japan. StemBioSys develops an extracelluar matrix from bone marrow stem cells. The matrix is made of natural proteins that are created by stem cells to act like a home, helping the cells divide, expand, and replicate.

Partnering agreement: Cell Medica and Baylor

Cell Medica (UK; https://cellmedica.co.uk) and Baylor College of Medicine (TX, USA; www.bcm.edu) will partner to develop a pipeline of next-generation cellular immunotherapy products designed to fight cancer by attacking solid tumors. Baylor has provided Cell Medica with an exclusive license to its proprietary natural killer T cell immunotherapy platform, five product candidates to be developed by Baylor research teams specializing in genetically engineered immune cells, and an option to license future product candidates. The partnership includes a co-development component designed to combine Baylor’s expertise in the creation of modified immune cell technologies with Cell Medica’s know-how in manufacturing and commercializing cell therapy products.

Research agreement: Bellicum and LUMC

Bellicum Pharmaceuticals (TX, USA; www.bellicum.com) announced a research agreement with Leiden University Medical Center (LUMC; the Netherlands; www.lumc.nl) to discover and validate natural high-affinity T cell receptor (TCR) product candidates targeting several cancers. The new collaboration builds on an earlier agreement with LUMC that gave Bellicum worldwide rights to TCR product candidates targeting solid tumors that express preferentially-expressed antigen in melanoma (PRAME) and other antigens. Under terms of the new collaboration, Bellicum will provide financial support to LUMC over a 3-year term in exchange for the right to exclusively license any high-affinity TCRs discovered under the agreement. Bellicum’s first TCR product candidate, BPX701 targeting PRAME, is expected to enter Phase 1/2 clinical trials in mid-2016 to treat refractory acute myeloid leukemia and myelodysplastic syndromes. BPX701 was licensed from Leiden in 2015 and incorporates Bellicum’s proprietary safety mechanism, CaspaCIDe, for improved control over the cells.

Launching new projects, products & services

Allo

AlloSource (CO, USA; www.allosource.org) launched two new innovative demineralized cortical fiber allografts, AlloFuse® Cortical Fibers and the AlloFuse® Fiber Boat, for use in a variety of spine and orthopedic procedures. The demineralized bone fiber (DBFâ„¢) technology used in these products is patented by TheraCell (MA, www.theracellinc.com/index.php), partner of AlloSource. These new DBF allografts consist of 100 percent demineralized cortical bone and offer a less costly alternative to synthetic bone void fillers. The size and geometry of the fibers have been optimized to provide exceptional handling characteristics for bone fusion procedures, primarily in spine and orthopedics.

DiscoveryBioMed

DiscoveryBioMed (DBM; AL, USA; www.discoverybiomed.com) was awarded a new 3-year Lab to Marketplace Small Business Innovation Research (SBIR) grant of $1.2 million to fund the establishment of novel diseased and normal renal cell platforms. The award comes from the Office of the Director at the National Institutes of Health. DBM is seeking to establish single cyst-derived primary cultures from diseased kidney but also primary human cell cultures from normal kidneys in order to immortalize cultures for expansion for larger initiatives such as therapeutics discovery. This would benefit therapies for polycystic kidney disease and, in particular, for the more common autosomal dominant genetic form.