Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC) is a clinical stage biotechnology company focused on the development and commercialization of new therapies in the field of regenerative medicine. Regenerative medicine is defined as the process of replacing or regenerating human cells, tissues or organs to restore or establish normal function and has been called the “next evolution of medical treatments” and “the vanguard of 21st century healthcare” by the U.S. Department of Health and Human Services.
The company’s principal R&D and commercial efforts relate to its Regenerative Ophthalmology programs and involve development of therapies for the treatment of eye diseases and disorders. Beyond the eye, our other research areas include projects focusing on developing many different cell therapies that may be used to treat a range of diseases across several therapeutic categories. ACT’s principal laboratory, GMP facility and corporate offices are in Marlborough, Massachusetts. ACT is led by an experienced management team and a world-class scientific team helmed, respectively, by Chief Executive Officer Dr. Paul K. Wotton, Ph.D. and Chief Scientific Officer Dr. Robert Lanza, M.D.
Regenerative Ophthalmology Platform
The mission behind our Regenerative Ophthalmology efforts is clear. It is our goal to bring innovative new therapies to the market to treat diseases of the eye that otherwise may cause significant loss of vision in patients. Our emerging cell therapies focus on structural repair and functional restoration and have the potential to make a significant difference in the lives of people with ocular disorders.
We are at the forefront of developing new therapies to address some of the most significant unmet medical needs in eye diseases. These include potential treatments for diseases affecting the retina, such as age-related macular degeneration – as well as other forms of macular degeneration, diabetic retinopathy and retinitis pigmentosa, as well as inflammatory diseases such as uveitis, and vision loss from photoreceptor and other neurosensory retinal damage due to glaucoma.
- Our lead clinical program involves RPE Cell Therapy for treating macular degeneration. Critical to the development and the rate of progression of macular degeneration is the health status of the retinal pigment epithelium, or RPE, a single layer of cells whose presence and integrity in the retina are required for normal vision. The company is currently conducting clinical trials in the U.S. and U.K. for treating Stargardt’s macular degeneration with the RPE Cell Therapy, and in the U.S. for treating patients with dry age-related macular degeneration.
- We are developing a Photoreceptor Progenitor Cell Therapy which we intend to be used for treating a wide variety of retinal degenerations in diseases where photoreceptors malfunction and/or die.
- Our Ganglion Progenitor Cell Therapy is a preclinical candidate program. In animal models of glaucoma, the injection of these cells protects against damage of existing nerve cells as well as forms new ganglion nerve cells.
- The company is also developing therapeutic platforms using Corneal Endothelial Cells for use in treating corneal blindness.
Other Regenerative Medicine Programs
In addition to our regenerative ophthalmology programs we have invested in other programs where we feel that we can leverage our expertise in cellular and developmental biology to generate allogeneic therapies that have the potential to improve health care in other prevalent degenerative diseases and diseases of aging. At the core of our pipeline planning are approaches intended to address large unmet medical needs with allogeneic stem cell-derived therapeutics.
- Our Mesenchymal Stem Cells (MSCs) represent an “off-the-shelf” cellular therapy ready for treatment of autoimmune and inflammatory diseases in both acute and chronic settings. MSCs regulate immune and inflammatory responses, providing therapeutic potential for treating diseases characterized by the presence of an inflammatory component, which makes them an attractive tool for the cellular treatment of autoimmunity and inflammation. We believe we have succeeded at creating a differentiated MSC product by producing the cells in culture from a pluripotent stem cell source.
- We have discovered Neuroprotective Biologics that are secreted by certain of our neurosensory retina progenitor cell populations. We anticipate that the neuroprotective agent(s) that we may ultimately develop as drug candidates may be useful not only in retinal diseases and dystrophies, but may have broader applications in central nervous system and peripheral nervous system diseases and disorders, including diseases causing cognitive function impairment, movement disorders such as Parkinson’s Disease, and ischemic events such as caused by stroke.
- Through our Blood Components Programs, we have been able to generate Red Blood Cells, Platelets, Dendritic cells and other cells relevant immune response or hemostasis.
*Regenerative Ophthalmology is a trademark of Advanced Cell Therapeutics, Inc.