“Errant Gene Therapeutics, LLC (“EGT”) Pushing Clinical Trial of Potentially Curative Treatment for Beta-Thalassemia and Eventually Sickle Cell Disease”

Errant Gene Therapeutics, LLC (“EGT”) Pushing Clinical Trial of Potentially Curative Treatment for Beta-Thalassemia and Eventually Sickle Cell Disease

-The technology, developed by scientists at Memorial Sloan Kettering Cancer Center (“MSKCC”), headed by Dr. Michel Sadelain and Errant Gene Therapeutics, is a one-time treatment that inserts an encoded gene into a patient’s own bone marrow stem cells restoring the production of normal hemoglobin. This technology is known as Thalagen.

In June of 2020, an abstract was released to the European Hematology Association (EHA) noting the results of patients treated in a clinical trial at MSK with the EGT vector. The abstract is based on patients treated with the 2009 EGT-produced vector in the MSK Clinical Trial. The EHA abstract, submitted by Simona Raso, reports that 2 out of 3 Thalassemia patients treated with EGT’s vector have sustained dramatic reduction in blood transfusions after 8 and 5 years, respectively. These 3 patients are the only Thalassemic patients treated with Lentiglobin in the US for whom there is an 8-year follow-up. The abstract is publicly available on the European Hematology Association’s website. The reductions in transfusions for the patients reported in the EHA abstract means a marked reduction in risk and damage created by the chronic transfusions, transferred diseases and iron build-up. One of the patients with significant transfusion deduction used the EGT-produced vector with a mild chemotherapeutic prep-regimen. The abstract does not report any clonal dominance. EGT is the only company with experience in development of a non-myeloablative potential treatment for Thalassemia patients.

EGT produced the world’s first commercial batch of gene therapy vector in 2009. The EGT vector uses the wildtype beta globin gene, the most natural form of the gene.

EGT Founder, Patrick Girondi noted, “After some delay, we are happy to be moving forward once again, and the EHA abstract is incredible news for patients. Today, with modern production, enhancers, improved filtration and other prep regimen drugs, we believe that the vector EGT will produce in 2021, honed by 12 years of advancement in the field and using modern transduction enhancers will cure Thalassemia patients and that EGT will make quick headway towards curing Sickle Cell patients using the same therapy.”

View the rest of the article here

View the PDF