Advanced Cell Technology, Inc. is applying stem cell technology in the field of regenerative medicine to bring effective, patient-specific therapies to the bedside. The Company’s Myoblast program is an autologous adult stem cell therapy for the treatment of heart disease. The Myoblast program has successfully completed four Phase I clinical trials and has clearance from the FDA to begin Phase II trials shortly. The company is also rapidly moving towards human clinical trials for its embryonic stem cell therapies including its RPE and HG programs. ACT published positive data from animal studies for its RPE (retinal pigment epithelial) cell program for the treatment of retinal degenerative disorders completed in collaboration with the Casey Eye Institute at Oregon Health and Science University. The company also has GLP Safety Studies in process for its RPE Program. ACT published positive data in the journal NATURE Methods for its HG (hemangioblast) cell program for the treatment of blood and cardiovascular diseases. The company expects to file INDs for its RPE and HG programs in 2008. In August 2006, ACT announced a novel technique to generate embryonic stem cell lines without destroying embryos, a breakthrough in the ethical debate surrounding the industry. Since then, the company has announced on multiple occassions the creation of human embryonic stem cell lines without destroying the developmental potential of the embryos. ACT owns or licenses over 380 patents and patent applications.
ACTC is currently focused on three product areas.
1) The myoblast program, which could yield novel therapies for cardiac diseases such as heart failure, successfully completed Phase I human clinical trials. The program has clearance from the FDA to begin Phase II human clinical trials and ACTC expects to commence those trials shortly.
2) The retinal pigment epithelium (RPE) program, which ACTC believes could yield new therapies for treating indications such as age-related macular degeneration. The Company announced a collaboration to conduct preclinical trials, completed pre-IND meetings with the FDA, and expect to file an IND in 2008.
3) The hemangioblast (HG) cell program for which ACTC are currently conducting preclinical research programs investigating the possibility of using the cells to treat cardiovascular disease, stroke, and cancer. The Company''s research, published in Nature Methods, demonstrated success in treating vascular indications such as heart attack and limb loss due to vascular damage.
The company is led by an experienced management team and scientific experts in the field, including CEO William Caldwell, Chief Scientific Officer Robert Lanza M.D., and SVP, Regulatory and Clinical Jonathan Dinsmore, Ph.D. The company operates GMP capable laboratories in Massachusetts and California.
The company is actively pursuing strategic collaborations in the United States (e.g., in California, where in 2004, the state passed Proposition 71, often referred to as the “Stem Cell Initiative,” which will provide $3.0 billion of funding over the next ten years for stem cell research) and around the world with members of academia, industry and foundations to further accelerate the pace of our research efforts.
- Completed acquisition of Mytogen and its Myoblast program, an autologous adult stem cell therapy for treating cardiac disease. Myoblast program successfully completed Phase I clinical trials. Phase II trials to begin shortly.
- Published positive results from HG program demonstrating hemangioblasts’ ability to repair vascular damage in animals. Therapy could be effective for treatment of blood and cardiovascular diseases. Expects to file IND in 2008.
- Made progress in macular degeneration indication by reporting positive data for RPE cell animal study. Entered into collaboration with Casey Eye Institute for preclinical trials. Completed Pre-IND meeting with FDA. Expects to file IND in 2008.
- Developed and demonstrated technique to generate embryonic stems that maintain developmental potential of the embryo and therefore has potential to shift the ethical debate. Announced hESC lines created without destroying an embryo in multiple studies.
- Owns or licenses over 380 patents and patent applications related to stem cell therapy.
- Large Target Market: Regenerative Medicine has potential to treat diseases of the eye, blood disorders, heart disease, neurodegenerative diseases, liver disease, diabetes, and others.
- Led by experienced management team and scientific experts including CEO William Caldwell, Chief Scientific Officer Robert Lanza M.D., and SVP, Regulatory and Clinical Jonathan Dinsmore, Ph.D.
Advanced Cell’s technologies could provide therapies for a broad range of diseases, including myoblasts for treating heart failure; hemangioblasts for treating blood disorders, cardiovascular disease, and cancer; retinal pigment epithelial cells for treating macular degeneration and other retinal degenerative disorders; skin cells for dermatological conditions; neuronal cells for spinal cord injury; and liver cells for hepatitis and cirrhosis. Most of the body’s specialized cells cannot be replaced by natural processes if they are seriously damaged or diseased. Stem cells can be used to generate healthy and functioning specialized cells, which can then replace diseased or dysfunctional cells. Replacing diseased cells with healthy cells, called cell therapy, is similar to the process of organ transplantation. Some conditions or injuries can be treated through the transplantation of entire healthy organs, but there is an acute shortage of donors. Stem cells can serve as an alternate and renewable source for specialized cells. Currently, researchers are investigating the use of adult, fetal and embryonic stem cells as a resource for various specialized cell types such as nerve cells, muscle cells, blood cells and skin cells. Adult stem cells do not require the manipulation or destruction of an embryo thus they lack the controversy surrounding embryonic stem cell research, but the cells are limited in their ability to differentiate into different cell types. Embryonic stem cells, while limited in use because of the current political and ethical debate, can develop into one of over 200 cell types in the body when given the necessary stimulation (i.e., they are totipotent). ACT is using a combination of embryonic and adult stem cell technologies to develop novel therapies for indications with few or no alternatives.
The ability to produce embryonic stem cells that are immunologically compatible with the patient is one of the strengths of ACTC's embryonic stem cell technology platform. The Company believes its technology platform will enable the transformation of a patient’s cells into an embryonic state where those cells can be differentiated into specific therapeutically relevant cell types that are genetically identical to the patient. ACTC believes its technology may also enable the production of stem cell lines, from sources external to the patient, that have a sufficiently high level of histocompatibility to be useful in making cell therapies readily accessible to a large segment of the patient population, without the need for exact genetic matching of tissues. In August 2001, The President of the United States set guidelines for federal funding of research on embryonic stem cells from human embryos created by in-vitro fertilization (IVF). IVF-ES cells have the drawback that they are not genetically matched to the recipient patient. These ES cells are allogeneic. The word allogeneic literally means “other DNA type.” Therapies using allogeneic cell lines can result in immune system incompatibilities where the host immune system attacks and rejects the transplanted cells or the transplanted cells attack the host. These incompatibilities may be partially suppressed with powerful immunosuppressive drugs, but the side effects can be severe and result in life-threatening complications. As a result, these incompatibilities will generate significant inefficiencies in the application of cell therapies.
ACT is also strongly focused on solving the problem of immune rejection and graft-versus-host disease by creating stem-cell therapeutics using donor tissues that are compatible, or that the immunologically active antigens on the donor cells are the same as those of the recipient so that they will not activate an immune response leading to rejection. This ability to produce tissue cells for transplantation that have the same genetic make-up as the patient may constitute a competitive advantage for Advanced Cell Technology.