San Rocco Therapeutics announces their Next-Generation Gene Therapy for Hemoglobin Disorders

To be presented at the 2026 ASGCT conference in Boston, MA

TAMPA, FL, UNITED STATES — San Rocco Therapeutics (SRT) today announced two abstracts at the upcoming 2026 ASGCT Annual Meeting highlighting its gene therapy platform, MiNiRoLu (SRT8-T87Q), and a companion

diagnostic for quantifying therapeutic gene expression. These advances address key limitations of current gene therapies for hemoglobin disorders, including high cost, manufacturing complexity, and challenging drug monitoring.

Key Highlights

-Higher vector production yields with reduced cost of goods and improved scalability

-Enhanced Biological Performance: Improved gene delivery in human hematopoietic stem cells and increased therapeutic hemoglobin expression in red blood cells

-Targeted Expression & Safety Profile: Red blood cell–specific activity and clinically validated vector backbone supporting favorable safety profile

-Companion Diagnostic: First-in-class assay to directly quantify therapeutic T87Q hemoglobin expression for improved monitoring and dose optimization

-Cost Disruption Potential: Designed to enable substantially lower cost gene therapy with expanded global access

Scientific Highlights

SRT’s gene therapy works by inserting a functional T87Q hemoglobin gene into a patient’s own hematopoietic stem cells. In laboratory studies, the company’s optimized delivery system showed:

-Improved efficiency of therapeutic gene delivery into hematopoietic stem cells

-Higher production of anti-sickling hemoglobin in red blood cells

-Precise activation only in red blood cells

Info:

Abstract ID: 3507

A lentiviral vector with enhanced titer and T87Q β-globin expression for treatment of severe β-hemoglobin disorders

Poster Presentation: Thursday, May 14, 2026 Poster Set-Up: 9:00 AM – 10:30 AM

Poster Reception: 5:00 PM – 6:30 PM

In parallel, SRT developed a diagnostic test capable of measuring therapeutic T87Q hemoglobin levels addressing a current gap in gene therapy monitoring.

Info:

Abstract ID: 465

A RT-qPCR assay for precise detection and absolute quantification of the T87Q β-globin variant in erythroid progeny of lentivirus transduced human CD34+ cells

Oral Scientific Session: Analytics and assay development: Potency evaluation Date and Time: Friday, May 15, 2026 10:15 AM – 12:00 PM

Room: MCEC Room 257AB (Level 2) Presentation Time: 10:30 AM – 10:45 AM

Path to the Clinic

SRT has completed GMP manufacturing of MiNiRoLu vector and is advancing to a Phase I/II clinical trial in β-globin disorders with academic and NIH collaborators in USA and Europe.

Founder’s Statement

“We believe gene therapy should be both curative and accessible,” said Patrick Girondi, Founder of San Rocco Therapeutics. “Our platform is designed to improve outcomes while significantly lowering cost and giving clinicians better tools to measure success.”

About San Rocco Therapeutics

SRT is a biotechnology company focused on developing curative and affordable gene therapies for sickle cell disease and β-thalassemia. The company’s mission is to expand access to life-changing treatments by scientific innovation with cost-focused scalable manufacturing.

Forward-Looking Statements

This press release contains forward-looking statements regarding development timelines, regulatory strategy, manufacturing, pricing, and potential therapeutic performance. Actual results may differ materially due to risks and uncertainties, including clinical outcomes and regulatory review. Preclinical results may not predict clinical success.

Megan Euker – Vice President, San Rocco Therapeutics San Rocco Therapeutics