San Rocco Therapeutics is focused on developing innovative gene therapies to address life-threatening rare diseases, particularly beta-thalassemia and sickle cell disease. One of their key technologies involves the use of Arginine Butyrate, an early gene therapy that stimulates the expression of the gamma globin gene, which is crucial for producing fetal hemoglobin. This approach aims to correct the genetic defects that lead to these blood disorders by utilizing gene transfer techniques. The company has a robust pipeline, including the TNS9 product, which involves collecting a patient's stem cells, correcting the genetic mutations, and reinfusing these modified cells to restore normal red blood cell production.

Additionally, San Rocco Therapeutics is exploring the CD47 signaling pathway, which plays a significant role in regulating immune responses and cellular stress responses. By targeting the TSP1-CD47 axis, the company aims to enhance the efficacy of cancer therapies while protecting normal cells from the adverse effects of radiation and chemotherapy. This dual approach not only seeks to improve treatment outcomes for patients with rare blood disorders but also has implications for broader applications in oncology and regenerative medicine.

United States

San Rocco Therapeutics is a biotechnology company with its primary focus on curative gene therapies for Sickle Cell Disease and Thalassemia

Cancer

Genetic Diseases

Hematologic Diseases

San Rocco Therapeutics

Sickle cell disease

Improved TNS9

Biologics

Gene Therapy

San Rocco Therapeutics Drug #1 (CD47/SRT001)

San Rocco Therapeutics Drug #2 (SRT002)

Tampa, Florida, United States of America 

Florida

Cell Therapies

Regenerative Medicine

Stem Cells

Therapeutic

San Rocco Therapeutics

Company Details