San Rocco Therapeutics, LLC (“SRT”) Pushes Clinical Trial for a Curative Treatment for Beta-Thalassemia and Sickle Cell Disease

April 12, 2022 |

2 min read

Using the SRT vector and myelosuppressive preparation therapy, Memorial Sloan Kettering (“MSK”) reported encouraging results in Nature Medicine, [Boulad, F., Maggio, A., Wang, X. et al. Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial.

TAMPA, Fla.--(BUSINESS WIRE)-- Using the SRT vector and myelosuppressive preparation therapy, Memorial Sloan Kettering (“MSK”) reported encouraging results in Nature Medicine, [Boulad, F., Maggio, A., Wang, X. et al. Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial. Nat Med 28, 63–70 (2022). https://doi.org/10.1038/s41591-021-01554-9]. The clinical trial using the SRT vector demonstrates that two out of three patients had a reduction in transfusions by 43% for almost a decade.

Further, MSK published in Molecular Therapy that globin lentiviral vectors, harboring powerful LCR HS elements, such as Bluebird Bio’s Beti-Cel may expose patients to the risk of trans-activating cancer-related genes.

Bluebird Bio’s patients have had issues with Clonal Dominance, Myelodysplastic Syndrome and Leukemia in patients treated with its vector as reported in Endpoints News [Mast, Jason. “Bluebird Shares Tank after FDA Puts Another Hold on Its Long Delayed Sickle Cell Gene Therapy.” Endpoints News, Endpoints News, 20 Dec. 2021, https://endpts.com/bluebird-shares-tank-after-fda-puts-another-hold-on-its-long-delayed-sickle-cell-gene-therapy/.]

“Manufacturing procedures have greatly improved since we produced our vector over twelve years ago. Mild chemotherapy reduces short term toxicities, hospital time and is essential for patients with organ damage. Insulators set up barriers that prevent the activation of other genes. With modern production, including an insulator, the SRT vector will be the best choice for patients,” says Dhesh Govender, Special Consultant, of SRT.

Megan Euker, SRT’s Project Manager states: “For years San Rocco Therapeutics has warned about Bluebird Bio’s product. The NY Supreme Court case showed that Bluebird Bio’s management knew San Rocco Therapeutics’ vector was superior and safer for patients, but sabotaged and pushed on with their faulty product for financial motives. Patients’ well-being must be the goal of research funds, not an executive smorgasbord.”

About San Rocco Therapeutics:

SRT is a privately held biopharmaceutical company established in 2003 and headquartered in Tampa, Florida. The SRT team consists of Dr. Christopher Ballas, Dhesh Govender, Dr. Frank Park from the University of Tennessee and Dr. Andrew Wilber from Southern Illinois University.