Gene Therapy Company
- Company typePrivate
- Founded1992
- HeadquartersChicago, United States
- Headcount11–50
- GTM typeB2B
- OfferingServices
What Gene Therapy Company does
San Rocco Therapeutics is a privately held clinical-stage biotechnology company developing lentiviral vector-based gene therapies for rare, life-threatening hemoglobin disorders, principally sickle cell disease and transfusion-dependent beta thalassemia. The company's core product is MiNiRoLu (SRT8-T87Q), a next-generation lentiviral vector engineered from its earlier TNS9.3.55 platform that delivers a functional T87Q variant of adult human β-globin into autologous hematopoietic stem cells. Reported preclinical improvements include approximately 30% higher functional titer, 35% greater transduction efficiency, and roughly 40% higher β-globin expression compared with the TNS9.3.55 predecessor. The firm also develops a T87Q variant-specific RT-qPCR companion diagnostic for quantitative detection of the vector-encoded gene product, addressing a known manufacturing and quality control gap in β-globin gene therapies. A pipeline candidate targeting CD47 is also listed alongside the hemoglobin programs.
The company is founder-led by Patrick Girondi, whose son's 1992 thalassemia diagnosis initiated the mission, and operates with 11-50 employees from a US headquarters in Tampa, Florida with a European subsidiary in Altamura, Italy at the site of the former Centro Medico San Rocco thalassemia treatment center. Its business model is preclinical-to-early-clinical development conducted through research collaborations with academic institutions including Memorial Sloan Kettering (Dr. Michel Sadelain since 2000), Boston Children's Hospital, University of Milan, University of Minnesota, UTHSC, and Southern Illinois University School of Medicine. The company holds historical worldwide licensing rights to Thalagen for sickle cell disease and beta thalassemia (since 2005) and has actively defended related intellectual property in court, including a 2020 New York Appeals Court victory. Revenue generation has not yet commenced; pricing has not been disclosed, though the company explicitly positions its mission as bringing affordable gene therapy to patients in contrast to the ~$2.8 million price point of the FDA-approved beta-thalassemia therapy Zynteglo.
Gene Therapy Company firmographics
Firmographics- Name
- Gene Therapy Company
- Legal name
- San Rocco Therapeutics LLC
- Website
- https://errantgene.com
- Company type
- Private
- Founded year
- 1992
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Ownership category
- akta.pro rank
Gene Therapy Company industry classification
Industry- Product category
- Gene Therapy / Rare Disease Biotech
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414), Scientific Research and Development Services (5417)
- SIC
- Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Lentiviral Vector Gene Therapy Developers (HLAAACAB)
- akta.pro secondary industries
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN), Gene Therapy Analytical Testing & QC (potency, vg/ddPCR, infectivity, RCL/RCA) (HLAAACAK)
Keywords
Where Gene Therapy Company is headquartered
LocationHeadquarters
- HQ city
- Chicago
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
Gene Therapy Company business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure
Revenue model
- Gene Therapy Development: Pre-commercialization biotech developing gene therapies for rare diseases. Revenue generation not yet initiated as products are in clinical development phase.
Go-to-market motion1 record
Marketing channels3 records
Gene Therapy Company product offering
Product offeringCore offering
San Rocco Therapeutics is a clinical-stage biotechnology company developing lentiviral vector-based gene therapies for rare, life-threatening hemoglobin disorders, primarily Sickle Cell Disease and Beta Thalassemia. Its lead candidate, MiNiRoLu (SRT8-T87Q), is a next-generation lentiviral vector designed to insert a functional T87Q β-globin gene into autologous hematopoietic stem cells, with the goal of eliminating or reducing dependence on blood transfusions. The company also develops companion diagnostics, such as a T87Q-specific RT-qPCR assay, and pursues additional pipeline programs including CD47 research.
Product overview
San Rocco Therapeutics is a biotech company developing gene therapies for rare diseases, with primary focus on hemoglobin disorders including Sickle Cell Disease and Beta Thalassemia. The company's core product is MiNiRoLu (SRT8-T87Q), a next-generation lentiviral vector-based gene therapy that delivers a functional T87Q β-globin gene into patient-derived hematopoietic stem cells. This therapy is built upon the earlier TNS9.3.55 platform, which was validated in a Phase 1 clinical trial. The company also develops companion diagnostics for therapeutic gene quantification. Additional pipeline programs include CD47 research.
Differentiator
Problem solved
Functional benefit
Quantifiable outcome
- 30% higher functional titer
- +2 more outcomes
Companies that use Gene Therapy Company
Customer profileSegments2 records
Ideal customer profiles1 record
Gene Therapy Company technology and API
TechnologyAPI detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Gene Therapy Company partnerships and signals
Strategic signalPartnerships
Nine partnerships are on record, tiered core and major.
- Memorial Sloan Kettering / Dr. Michel SadelaincoreDr. Michel Sadelain of Memorial Sloan Kettering has been collaborating with SRT founder Patrick Girondi since 2000 on gene therapy projects for Sickle Cell Disease and Thalassemia. SRT supports Sadelain's vector development.
- Dr. Susan PerrinecoreDr. Perrine developed Arginine Butyrate, one of the first gene therapies that stimulated gamma globin gene expression. Her work with Patrick Girondi from 1992 led to founding Emerging Pharmaceutical Technologies.
- University of MilanmajorCollaborator in 38-patient clinical trial in 1998 with Arginine Butyrate and Isobutyramide.
- Boston Children's HospitalmajorResearch collaborator in the global team advancing Thalagen project for sickle cell disease and thalassemia.
- University of MinnesotamajorResearch collaborator in the global team advancing Thalagen project for sickle cell disease and thalassemia.
- University of Tennessee Health Science Center (UTHSC)majorDr. Frank Park, Associate Professor at UTHSC, received the 2025 Orphan Dream Award for work on MiNiRoLu gene therapy.
- Southern Illinois University School of MedicinemajorDr. Andrew Wilber, Associate Professor, received the 2025 Orphan Dream Award for groundbreaking work on MiNiRoLu.
- Children's Hospital of Oakland Research InstitutemajorResearch collaborator in the global team advancing Thalagen project.
- Sick Children's Hospital of TorontomajorInternational research collaborator in the global team.
Scale indicators2 records
Recent moves11 records
Expansion highlights4 records
Gene Therapy Company competitors and assessment
Company assessmentEmerging players
- Sangamo Therapeutics: Zinc finger nuclease gene-editing developer with prior hemoglobin disorder programs (ST-400) and ex vivo cell therapy manufacturing capabilities overlapping with SRT's lentiviral vector approach.
- Verve Therapeutics: In vivo gene editing company for cardiovascular disease using similar base editing and lipid nanoparticle delivery concepts relevant to SRT's lentiviral vector development skill set.
- REGENXBIO: Gene therapy platform company developing AAV-based therapies including hemoglobin disorder programs, providing a comparable regulatory and CMC pathway in rare disease gene therapy.
- Agios Pharmaceuticals: Rare disease biotech with commercial rare blood disorder franchise, comparable go-to-market structure for orphan genetic disease therapeutics addressing similar small patient populations.
- Beam Therapeutics: Base editing company developing BEAM-101 for sickle cell disease, representing a next-generation modality targeting the same patient population with a potentially curative one-time intervention.
- Editas Medicine: Clinical-stage gene editing company with hemoglobin disorder programs (reni-cel for sickle cell), pursuing the same autologous ex vivo editing approach that competes with SRT's lentiviral platform.
Direct peers
- Vertex Pharmaceuticals: Commercial partner on Casgevy and owner of the sickle cell disease gene therapy franchise, representing the incumbent large-cap player in SRT's chosen indication.
- Bluebird Bio: Direct competitor developing lentiviral vector gene therapies for sickle cell disease and beta thalassemia (Lyfgenia/Zynteglo), serving the same patient populations with the same modality that SRT is pursuing.
- CRISPR Therapeutics: Co-developer (with Vertex) of Casgevy, the first FDA-approved gene-edited therapy for sickle cell disease, directly addressing SRT's primary target indication with a competing ex vivo modality.
Broad incumbents
- Acceleron Pharma: Acquired by Merck for $11.5B based on rare blood disorder pipeline (luspatercept for beta-thalassemia), representing the orphan hematology commercial benchmark that defines the market SRT aims to enter.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat3 records
Key risks6 records
Key highlights7 records
Customer concentration
Gene Therapy Company social profiles
Digital presenceGene Therapy Company financial estimates
Financial estimateRevenue estimate
Valuation estimate
Gene Therapy Company leadership team
Management profileNumber of profiles
Gene Therapy Company funding detail
Funding detailFunding overview
Funding rounds
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Gene Therapy Company M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Gene Therapy Company
What does Gene Therapy Company do?
San Rocco Therapeutics is a clinical-stage biotechnology company developing lentiviral vector-based gene therapies for rare, life-threatening hemoglobin disorders, primarily Sickle Cell Disease and Beta Thalassemia. Its lead candidate, MiNiRoLu (SRT8-T87Q), is a next-generation lentiviral vector designed to insert a functional T87Q β-globin gene into autologous hematopoietic stem cells, with the goal of eliminating or reducing dependence on blood transfusions. The company also develops companion diagnostics, such as a T87Q-specific RT-qPCR assay, and pursues additional pipeline programs including CD47 research.
Is Gene Therapy Company a public or private company?
Gene Therapy Company is a private company. It is classified as founder individual operated bootstrapped and is currently operating.
When was Gene Therapy Company founded?
Gene Therapy Company was founded in 1992. It employs 11 to 50 people.
Where is Gene Therapy Company based?
Gene Therapy Company is headquartered in Chicago, United States, in the North America region.
How does Gene Therapy Company make money?
One revenue line is on record: gene Therapy Development.
Who are Gene Therapy Company's main competitors?
Emerging players on record are Sangamo Therapeutics, Verve Therapeutics, REGENXBIO, Agios Pharmaceuticals, Beam Therapeutics and Editas Medicine. Direct peers are Vertex Pharmaceuticals, Bluebird Bio and CRISPR Therapeutics. Acceleron Pharma is listed as a broad incumbent.
Does Gene Therapy Company have an API?
No public API is recorded for Gene Therapy Company.
What industry is Gene Therapy Company in?
Gene Therapy Company's product category is Gene Therapy / Rare Disease Biotech. Its primary akta.pro industry code is HLAAACAB, Lentiviral Vector Gene Therapy Developers, with a secondary code of HLAAACAE, Gene Therapy for Rare/Monogenic Diseases. Its NAICS code is 541714 and its SIC code is 2834.