San Rocco Therapeutics Announces Major Advances Toward Clinical Development of MiNiRoLu™ Gene Therapy at ESGCT 2026

New manufacturing and therapeutic protein expression data support advancement of an investigational gene therapy for sickle cell disease and beta-thalassemia

TAMPA, FL, UNITED STATES, November 3, 2026 /EINPresswire.com/ -- San Rocco Therapeutics, a biotechnology company developing innovative gene therapies for inherited blood disorders, today announced significant progress toward clinical development of its lead investigational gene therapy, SRT8 (also known as MiNiRoLu), following new data presented at the 2026 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) in Hamburg, Germany.

In a presentation titled “A GMP Manufacturing Protocol for a T87Q β-Globin Drug Product to Treat β-Hemoglobinopathies,” Dr. Frank Park of the University of Tennessee Health Science Center presented findings on behalf of the research team and San Rocco Therapeutics, highlighting advances in manufacturing, vector stability, gene transfer efficiency, and therapeutic gene and protein expression. These achievements represent an important milestone toward the production of genetically modified, autologous hematopoietic stem cells for therapeutic application.

Key Findings
SRT8 is designed to address the underlying genetic cause of sickle cell disease and β-thalassemia by introducing a therapeutic β-globin gene into a patient’s own blood-forming stem cells. The research demonstrated:
• Production of a high-titer, GMP-grade self-inactivating lentiviral vector (LVV) with demonstrated stability >18 months
• Efficient LVV genetic modification of primary human hematopoietic stem cells at relatively low vector doses (MOI 25-75) resulting in T87Q β-globin protein
expression levels exceeding those previously reported for Bluebird bio's BB305 platform.
• Quantification of T87Q β-globin mRNA using a novel variant-specific RT-PCR assay

“Our mission has always been to develop treatments that can fundamentally change the lives of people suffering from sickle cell disease and β-thalassemia,” said Frank Park, who is also the acting Chief Scientific Officer of San Rocco Therapeutics’ European subsidiary. “The results presented at ESGCT represent another important step toward bringing SRT8 from the laboratory to patients and ultimately making potentially curative treatments more affordable and accessible to the patients who need them.”

Clinical Development Planned in the United States and Europe
San Rocco Therapeutics is pursuing a phased clinical strategy with initial trials planned at the National Institutes of Health (NIH), followed by European testing and development in Italy. MiNiRoLu remains an investigational gene therapy with clinical validation studies intended to assess its safety and efficacy in patients with either sickle cell disease or βeta-thalassemia.

“The scientific and manufacturing progress presented at ESGCT strengthens the foundation for our next stage of development,” said Megan Euker, Vice President of San Rocco Therapeutics. “By combining rigorous scientific research, international collaboration, and a long-term commitment to affordable access for these one-time gene therapy drug products, we aim to deliver this transformative therapy to patients on both sides of the Atlantic.”
About San Rocco Therapeutics

San Rocco Therapeutics is an emerging biotechnology company in the USA and Europe focused on developing innovative gene therapies for serious inherited diseases. For more information, visit www.sanroccotherapeutics.com.

Authors of poster presented in Hamburg: Park, Frank; Song, Hannah; Highfill, Steven; Mirto, Catherine; Tisdale, John; Wilber, Andrew

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