Abstract
Cambridge Healthtech Institute’s 16th International Molecular Medicine Tri-Conference held on 25–27 February, 2009, in San Francisco is one of the most comprehensive drug discovery and development events worldwide. 11 concurrent tracks had covered areas from molecular diagnostics to translational medicine including The Fourth Annual Stem Cell Congress. Stem-cell niche and control of stem-cell differentiation were the focus of the first day, whereas cardiac regeneration and harnessing cells for regenerative healing were dominant topics of the second day. The morning of the last day was dedicated to induced pluripotent stem cells and personalized medicine. The last session of the meeting called ‘Venture Capital and Emerging Company forum‘ was actually something very new and unique. A panel of venture-capital experts presented an overview of their venturecapital firm and gave helpful tips in building a cell-based technology into a viable business.
At the introductory forum Beth Seidenberg from Kleiner Perkins Caufield & Byers pointed out that the field of regenerative medicine lost too much time on finding ‘the right source‘ of stem cells and that the focus of future investment would be turned rather toward translational medicine business models. On the other hand, Kleiner Perkins Caufield & Byers are funding the latest rage in the stem-cell field: induced pluripotent stem cell-based company iZumi from Mountain View, CA, USA (www.izumibio.com). Other members of the panel were Gregory Bonfiglio from Proteus Venture Partners, Michael Goldberg from Mohr Davidow Ventures and Ken Aldrich from Tech Coast Angels and Convergent Ventures, who recently had to step in as CEO to salvage their investment in the International Stem Cell Corporation. The venture-capital (VC) forum was followed with presentations from four emerging companies. Each presenter had limit of five slides to present their business model within 5 min. Presentations were then discussed and commented by VC panel members. The forum, as designed, was extremely useful, not only for presenting companies but also for entrepreneurs in the audience. The four presenting companies were:
AuxoCell Laboratories, Inc. from Amesbury, MA, USA (www.auxocell.com), which bases its business on banking cells isolated from Wharton jelly. Wharton-jelly stem cells are characterized as fetal mesenchymal stem cells and can be induced to form adipose tissue, bone, cartilage, skeletal muscle cells, cardiomyocyte-like cells and neural cells. These cells are CD105+CD73+CD90+CD34-CD45-Oct4+Sox2+nanog+ and amenable for tissue engineering, gene therapy and regenerative medicine.
California Stem Cells from Irvine CA, USA (www.californiastemcell.com) presented a dual business model: 1) high-purity populations of specific cell types differentiated from human embryonic stem cells (hESCs) available for high-throughput screening; and 2) predictive toxicology applications. hESC-derived neural progenitor cells, neurons and cardiac myocytes were sold in the 96-well and 384-well plates as ready-to-use in manual or automated systems for US$1000–1200 per plate and therapy programs targeting coronary heart diseases, amyotrophic lateral sclerosis, spinal cord injury and type 1 infantile spinal muscular atrophy, for which the company plans to file an Investigation New Drug (IND) application with the US FDA in the second quarter of 2009.
Capricor from Los Angeles, CA, USA (www.capricor.com) built its therapeutic program around proprietary and novel-proprietary cardiac-derived cells (CDCs) technology based upon the work of its principal founder, Eduardo Marbán, Director of the Heart Institute at Cedars Sinai Medical Center, Los Angeles, CA, USA (www.csmc.edu). Capricor has exclusively licensed definitive technology to harvest these cells from human heart biopsies and clinically manufacture an autologous heart stem-cell therapy. Biopsies are obtained with a safe and routine 15-min catheter-based outpatient procedure. The patient‘s cardiac stem cells are then extracted out of the biopsied tissue, expanded in number and reintroduced into the same patient‘s injured heart. The targeted population is patients who have suffered cardiac infarctions and the biopsy will be performed after healing occurred. Initial CDC therapy costs US$25,000 and each multiple-dose administration is US$12,000. Banking of personal CDCs will cost patients US$7600 per year. Preclinical studies performed by the Johns Hopkins School of Medicine, Baltimore, MD, USA (www.hopkinsmedicine.org) and the University of Rome, Rome, Italy (www.uniroma1.it) demonstrate that Capricor‘s cardiac stem cells, when reimplanted into injured animal hearts, improve heart function and grow new, healthy heart tissue. An FDA-approved IND application for a Phase I/II human clinical trial will be conducted at Cedars Sinai (CA, USA) and John Hopkins.
Girus Inc. from San Jose, CA, USA (www.girusinc.com) was formed in 2005 after all rights and title to the company‘s Directed Cell Trafficking™ (DCT) Technology were acquired from the University of California San Francisco, USA. DCT is the technology that facilitates the use of fewer cells in hematopoietic stem cell transplants. It is being developed to improve current procedures and to enable routine use of cord-blood stem cells in transplants for adults. A long-term objective of the company is for DCT to become the standard of care in stem-cell transplantation.
Financial & competing interests disclosure
The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.
No writing assistance was utilized in the production of this manuscript.