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Beta-Thalassemia Treatment Approved For FDA's Clinical Phase I Trials

EGT welcomes an important milestone towards curative therapy for beta-thalassemia major

Submitted by: Errant Gene Therapeutics, LLC

Categories: Health & Wellness, Health & Wellness

Posted: Nov 13, 2012 – 05:00 AM EST


CHICAGO, Nov. 13 /CSRwire/ - Beta-thalassemia, also called Cooley’s Anemia, is a blood disease that effects thousands of Americans. In a major step toward a curative therapy, rare disease drug development firm Errant Gene Therapeutics, LLC (“EGT”) applauds the FDA approval of the Phase I Clinical Trial of a potentially curative treatment for beta-thalassemia.

This trial represents an important milestone towards the prospect for a cure for a chronic condition, which would otherwise subject the patient to early death without incurring life-long regular blood transfusions and chelation therapy.

The technology, developed by Memorial Sloan Kettering Cancer Center ("MSKCC") and Errant Gene Therapeutics is a one-time therapeutic treatment that inserts an encoded gene into a patient's own bone marrow stem cells restoring the production of normal hemoglobin. By using a highly targeted genetic therapy to permanently restore normal hematological functionality, chronic carriers will benefit from this breakthrough curative treatment without major side-effects.  This technology is known as Thalagen.

EGT is particularly proud and remains committed to its long-standing collaboration with Dr. Michel Sadelain of Sloan Kettering, who invented the vector technology used in the treatment and has been dedicated to finding a cure for beta-Thalassemia for decades.

The clinical trial is sponsored by Memorial Sloan Kettering Cancer Center in New York City and is currently recruiting participants. You can find more information on the trial at, under identifier NCT01639690. For more information about this study and to inquire about eligibility, please visit:

Errant Gene Therapeutics founder and CEO, Pat Girondi, stated that “the palpable hope shared by patients of all ages in all parts of the world, and by researchers in key centers in the US, EU and Asia, reinforces our dedication to ensuring that this journey only ends with a cure."

Sam Salman, Errant Gene Therapeutics' president, added that, "the promise of delivering a cure derived from a patient's own bone marrow stem cells, provides the basis of a new standard of care for chronic hemoglobinopathies, many of which are life-threatening. Errant Gene Therapeutics is proud to be advancing the current state- of-the-art therapies targeting Rare Diseases."

Michael Chambers, CEO of Aldevron, a world-class service organization specializing in plasmid DNA and protein production technologies, antibody technologies, commented that “Beta Thalassemia is a very challenging disorder to treat. This pioneering work by Errant Gene Therapeutics and Memorial Sloan Kettering promises to rise to this challenge. Thalagen gene therapy treatment has the potential to restore normal haemoglobin production and overcome this disease. Furthermore, this extremely innovative technology will help to advance the entire gene therapy field.”

Lucio Luzzatto, MD, Scientific Director of the Tuscan Cancer Institute noted “For an inherited disease gene therapy is the most rational form of treatment. By inserting a normal gene in the patient’s own cells the disease can be naturally corrected. This goal has been achieved thus far only in very few cases, because one needs to understand the disease in depth: the nature of thalassemia has been worked out in fine detail. Michel Sadelain’s team has been the first to correct beta-thalassemia in pre-clinical models. It is very encouraging that a clinical trial is now starting in patients, and there is every reason to expect that it will succeed.”

Former Chicago Mayor Richard M. Daley noted,  “I was Mayor for 22 years and few events gave me the satisfaction of the announcement of this gene therapy trial. EGT is a Chicago company and I want people to know that Chicago is playing an important role in gene therapy research.”

Ronald Capano of Cooley’s Anemia International, Inc. added, “This clinical trial means so much to so many and represents the work and dedication of our organization and that of the family and friends of all thalassemic and sickle cell anemia patients.”

Martin Tenniswood, Ph.D., Director, Cancer Research Center, University at Albany, commented that, “Bringing Thalagen to the clinic has been an arduous undertaking, and is a testament to the dedication of the clinicians at Memorial Sloan Kettering and the unwavering commitment of Errant Gene Therapeutics to find a cure for this devastating disease.”

“The Cooley’s Anemia Foundation applauds the efforts of the many scientists whose efforts have led us here and especially those directly involved in this trial; we look forward to the wealth of knowledge that this trial will add to the search for a cure; and we thank those thalassemia patients who are willing to participate in order to move that search forward in such a meaningful way,” says Anthony J. Viola, National President of the Cooley’s Anemia Foundation.

Special thanks also to Cooley's Anemia International, the Italian Thalassemia Foundation, Italian Telethon, Thalassemia International Federation of India, and the Association for Research Piera Cutino Onlus.

About Errant Gene Therapeutics, LLC

Errant Gene Therapeutics (also known as EGT) is a privately held biopharmaceutical company established in 2003. In addition to its ongoing support of gene therapy for the cure of beta-thalassemia and sickle cell anemia, EGT is a pioneer in the emerging field of epigenetics. Their research is focused on the treatment of the deadly inflammatory breast cancer and hormone refractory cancers using its patented portfolio of small molecule histone deacetylase inhibitors, which change the way cells express their genetic material. EGT’s lead compound, CG-1521, targets inflammatory breast cancer and hormone refractory prostate cancer. CG-1521 results have been published in scientific venues and EGT is currently preparing a pre-IND filing. 

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For more information, please contact:

Phone: 312.441.1800 x11
Phone 2: 312.498.0025
Fax: 312.893.2200
Sam Salman President
Phone: 312.441.1800 x12
Fax: 312.893.2200


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