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San Rocco Therapeutics Scientific Founder Dr. Michel Sadelain is Recipient of American Cell and Gene Therapy’s Highest Honor

·3-min read

San Rocco Therapeutics (SRT), formerly Errant Gene Therapeutics, applauds Dr. Michel Sadelain, MD, PhD, Director of Memorial Sloan Kettering’s Center for Cell Engineering (MSK) and San Rocco’s Scientific Founder as the 2021 recipient of the Outstanding Achievement Award from the American Society of Gene and Cell Therapy (ASGCT) for discoveries to treat Sickle Cell Disease and Beta-Thalassemia.

This press release features multimedia. View the full release here:

Pictured above: (as presented in Dr. Michel Sadelain’s presentation at the May 12, 2021 ASGCT George Stamatoyannopoulos Memorial Lecture: Dr. Patricia Giardina, Dr. Richard J. O’Reilly, Dr. Michel Sadelain, Dr. Farid Boulad, Patrick Girondi from RAC June 20, 2007). (Photo: Business Wire)

San Rocco’s alliance with MSK led to the development of the world’s first commercial GMP batch of lenti-viral vector, the first lenti-viral vector to treat a US patient for Beta-Thalassemia, the first clinical trial which employed both myelosuppression and full myeloablative therapies and the first therapy to be evaluated by the FDA’s Recombinant Advisory Committee (RAC) in June 2007. At the request of the RAC committee regarding safety concerns, Dr. Isabelle Rivière at MSK crafted a first of its kind clinical study to demonstrate the utility and safety of employing G-CSF for mobilization. MSK also went on to discover and demonstrate that Plerixafor could be used in Sickle Cell Disease to condition patients. Since the IND approval, every adult Beta-Thalassemia trial employs the same mobilization protocol using G-CSF that MSK and SRT pioneered 10 years ago. The IND for our Beta-Thalassemia gene therapy using the SRT vector was approved in July 2012 and SRT was granted Orphan Drug Designation in both the US and Europe.

The FDA is presently evaluating lenti-virus based cell therapy trials in SCD and Beta-Thalassemia. SRT and MSK are exploring the possibility of non-myeloablative conditioning as well as next generation insulator constructs within the vector to achieve superior safety. Our original trial is ongoing and after 8 years of follow up there have been no safety issues and patients received clinical benefit, having a reduction in the number of required blood transfusions.

Dr. Sadelain’s data demonstrates that the new SNS23 vector is capable of 10X enhanced gene transfer relative to the original EGT/MSK vector, giving rise to the possibility of eliminating the use of transduction enhancers, increased safety, dramatically reducing therapy cost, allowing broader use of the curative therapy.

SRT would like to thank the critical support from our patient Foundations: Cooley’s Anemia International, FITHAD, Thalassemics India and TIF.

SRT commends Drs. Galanello, Persons, Sorrentino and Drs. Ballas, Locatelli, Luzzatto, Maggio, Moi, Rivella, Rivière, Tisdale, Wilber and Yannaki, for their passionate support of our patients.

The SRT mission is to provide safe and affordable gene therapy to patients.

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For further details and information regarding San Rocco’s development activities in Sickle Cell Disease and Beta-Thalassemia, please contact us via email:

Patrick Girondi, CEO
(312) 441-1800 x 11

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