Advanced Cell Technology, Inc. (ACTC)
Advanced Cell Technology, Inc.
P.O. Box 1700
Santa Monica, CA 90406
Tel: (310) 576 0611
Fax: (310) 576 0662
Massachusetts Laboratory Facility
33 Locke Dr.
Marlborough, MA 01752
Tel: (508) 756-1212
Fax: (508) 229-2333
Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC) is a biotechnology company developing cellular therapies for the treatment of diseases that impact millions of people worldwide. The company is currently conducting the only ongoing, FDA-cleared human embryonic stem cell (hESC)-based human clinical trials. The two trials, initiated in July 2011 at UCLA's Jules Stein Eye Institute, use retinal pigment epithelial (RPE) cells derived from hESCs to treat forms of macular degeneration. ACT also recently received approval from the UK Medicines & Healthcare Products Regulatory Agency (MHRA) to conduct the first hESC trial in Europe. The company is also developing its Hemangioblast (HG) platform for the treatment of blood and cardiovascular diseases, its Phase II-cleared Myoblast stem cell therapy for the treatment of chronic heart failure and other cardiac conditions, and other programs. PATENTS & INTELLECTUAL PROPERTY (IP):
ACT's principal laboratory and GMP facility is in Marlborough, Massachusetts, and its corporate offices are in Santa Monica, California. ACT is led by an experienced management team and a world-class scientific team helmed, respectively, by interim chairman and CEO Gary Rabin and chief scientific officer Robert Lanza, M.D.
ACT owns or licenses more than 150 patents and patent applications related to stem cell therapy and regenerative medicine, and is positioning itself to have a dominant patent position around its stem cell-derived RPE program in leading markets around the world.
In 2010 ACT secured three far-reaching patents related to its RPE program, and more recently was issued the first patent for generating hemangioblast cells to treat a broad spectrum of vascular and hematopoietic disorders. THERAPEUTIC PLATFORMS:
The company's RPE and HG programs are hESC-based, and its Myoblast program for cardiac disease is an adult autologous stem cell therapy. As documented in NATURE
and Cell Stem Cell
, ACT developed the "single-cell blastomere" technique
, the first-ever proven alternative method for successful hESC generation without harm to the embryo
, for which it was recently issued a broad patent RPE Program -
ACT has developed a fully-differentiated RPE cell derived from human embryonic stem cells, which can be used as a cellular therapy to treat retinal degenerative diseases. In July 2011 ACT initiated two Phase I/II clinical trials
to test the safety of the therapy for Stargardt's Macular Dystrophy (SMD), and for Dry Age-Related Macular Degeneration (Dry AMD). Dry AMD represents a $25-30 Billion market in the US and Europe alone,
and there are no approved therapies currently available for either condition. Two patients have thus far been treated at UCLA, Institutional Review Board (IRB) approval for the SMD clinical trial has also been issued by Oregon Health & Science University (OHSU) and European regulatory clearance has been granted to initiate the first hESC trial in Europe.
Other recent milestones in ACT's RPE program include: the NIH proposed expanding its definition of hESCs for funding purposes, in part to accommodate ACT's lines derived using its patented "embryo-safe" single-cell blastomere technique; and both the FDA and the European Medicines Agency (EMA) granting ACT's RPE cells "Orphan" status for treatment of Stargardt's Disease. Hemangioblast Program -
ACT's Hemangioblast program is for the treatment of blood and cardiovascular diseases. A paper published in NATURE Methods
revealed the company's successful generation of functional Hemangioblast cells from human embryonic stem cells, and a paper published earlier this year in Cell Research indicated that hESCs could be a potentially unlimited source of platelets for transfusion. Myoblast Program -
ACT's Myoblast treatment may prove particularly beneficial for patients who have experienced a serious heart attack and are at risk for heart failure. ACT has secured FDA approval to commence with Phase II clinical trials. The Myoblast program has several advantages over currently-available approaches, including symptom management and disease modification.