triplex-forming oligonucleotide (TFO) technology may cure thalassemia

  • 0 Replies
Helix Therapeutics Inc Secures Additional Funding to Advance Cutting-Edge Stem Cell Gene Correction Therapy for Genetic Diseases
 Press Release Source: Helix Therapeutics Inc On Tuesday November 23, 2010, 8:00 am EST
NEW HAVEN, Conn., Nov. 23, 2010 /PRNewswire/ -- Helix Therapeutics, a biopharmaceutical company pursuing therapies for genetic disorders such as sickle cell anemia, B-Thalassemia and lysosomal storage disorders, announced today that Canaan Partners and Connecticut Innovations (CI) have made an investment of $2.5 million in the company. The funds will add to earlier seed-stage funds that the company has used to further its proprietary targeted gene modification (TGM) technology platform, which allows correction of certain genetic mutations in blood stem cells of patients with rare genetic diseases.

Joseph J. Catino, Ph.D., president and CEO of Helix Therapeutics Inc, said, "We welcome the addition of Canaan Partners as an investor in Helix and a team with synergistic drug development expertise. We have made great progress in moving this potentially game-changing technology toward clinical studies in patients, and will use these additional funds to further support those efforts."

The company's technology, which was developed at the Yale University School of Medicine and licensed exclusively on a worldwide basis to Helix, uses patented oligonucleotides that bind to the human genome resulting in permanent, targeted gene modification. Helix's proprietary TGM technology platform is empowered by the company's triplex-forming oligonucleotide technology, and corrects a genetic defect in a patient's stem cells by stimulating the cell's own DNA repair machinery to permanently correct them. The corrected cells are then returned to the patient who is then able to make the proteins capable of stopping the course of their genetic disease. Genetic diseases such as sickle cell anemia, B-Thalassemia, severe immune disorders and lysosomal storage disorders are among those that could be treated with therapies from Helix.

Timothy Shannon, M.D., a venture partner at Canaan, will join the Helix Board. Dr. Shannon said, "The Helix technology fits well into the growing interest in personalized medicine and rare diseases within the NIH, FDA and pharmaceutical industry. Canaan sees the potential in a technology like Helix's that takes advantage of the convergence of years of research in gene correction approaches, stem cell therapy and rare diseases. Helix has the opportunity to be at the forefront of an important leap in medical care."

"Wise investments like this help to grow our tech sector and fuel the job creation engine that is so vital to the health of Connecticut's economy," Connecticut Governor M. Jodi Rell stated.

CI Managing Director of Investments Russell Tweeddale will continue to represent CI on Helix's board of directors. He said, "We are a part of an important collaboration with the Helix team, Canaan Partners and the Yale founders to develop an approach that has the potential to fundamentally change the way many genetic diseases are treated. Therapies for diseases targeted by Helix represent a tremendous market opportunity, on the order of $500-800 million."

The Helix board also includes Peter Glazer, M.D., Ph.D., a founder of Helix, inventor of the company's technology platform, and professor and chair of the Department of Therapeutic Radiology at the Yale University School of Medicine; and Kevin Rakin, a Helix founder and Chairman and CEO of


SMF spam blocked by CleanTalk