Minatx
Founded 2008 · 27 employees on LinkedIn · 1 known investors
MiNA Therapeutics develops small activating RNA (RNAa) therapeutics, a class of medicines that boost or restore normal gene function to treat disease. The company advances its own pipeline with an initial focus on genetic medicine and partners with pharmaceutical companies to apply its platform across other therapeutic areas.
Also known as MiNA Therapeutics · MiNA Therapeutics Limited · MiNA Therapeutics Ltd.
Investors · 1
Company profile
researched Aug 2026MiNA Therapeutics Limited is a London-based biotechnology company developing small activating RNA (RNAa, also referred to as saRNA) therapeutics, a class of drugs intended to harness innate mechanisms of gene activation to restore or boost the normal function of genes and the protein-modulated pathways they control. The company describes itself as a leader in the RNAa field and advances a proprietary pipeline with an initial focus on genetic medicine, while also collaborating with pharmaceutical companies to apply its platform in other therapeutic areas.
Its most advanced disclosed program is MTL-CEBPA, an saRNA targeting the myeloid transcription factor CEBPA, which has reached Phase 2 and has been studied in advanced hepatocellular carcinoma (including in combination with sorafenib), in combination with the PD-1 inhibitor pembrolizumab in advanced solid tumours, and in paediatric patients with mucopolysaccharidosis type 1H (Hurler syndrome). Preclinical programs include an saRNA aimed at fetal haemoglobin/γ-globin for sickle cell disease and an HNF4A-targeting saRNA associated with insulin resistance; other disclosed targets include C1-INH and SIRT1. The company states its first RNAa medicine has been evaluated in clinical trials in more than 130 patients, demonstrating what it terms clinical proof of platform, and that its team has developed a systematic approach to move new RNAa medicines from concept to a clinical trial in about two years.
Business model
MiNA develops its own pipeline of RNAa medicines while entering research collaborations and partnerships with pharmaceutical companies that apply its technology platform to additional therapeutic areas; the website maintains a dedicated business development contact for prospective partners.
Traction
MTL-CEBPA has advanced to Phase 2, with completed Phase 1 (with pembrolizumab, NCT04105335) and Phase 2 (OUTREACH2, NCT04710641) studies and a recruiting Phase 1/2 paediatric MPS1H study (ISRCTN10369994). The company reports its first RNAa medicine has been evaluated in over 130 patients, and it presented preclinical sickle cell disease data at the American Society of Hematology annual meeting in December 2024 and the European Hematology Association congress in June 2025. Phase 1 results for MTL-CEBPA plus pembrolizumab were published in Cell Reports Medicine in April 2025.
Latest developments
In April 2024 MiNA entered a research collaboration with Nippon Shinyaku to develop RNAa therapeutics for rare neurodegenerative diseases. In March 2025 it appointed Norbert Bischofberger to its board as non-executive director and Bríd Ryan as chief scientific officer. In June 2025 it presented new preclinical data from its sickle cell disease program at the European Hematology Association congress.
▸Full profile — market position, technology, go-to-market, geography, history
Market position
The company positions itself as a leader in small activating RNA therapeutics, a modality it characterises as a new class of medicines distinct from established gene-silencing RNA approaches.
Technology
The platform is based on small activating RNA (RNAa/saRNA) oligonucleotides that engage endogenous gene-activation mechanisms to upregulate expression of a target gene, in contrast to approaches that silence genes. Disclosed targets include CEBPA, γ-globin/fetal haemoglobin, HNF4A, C1-INH and SIRT1. Published Phase 1 work on MTL-CEBPA combined with pembrolizumab reported upregulation of CEBPA to relieve myeloid-driven immunosuppression, with paired biopsy analyses indicating increased T cell and antigen-presenting cell infiltration in the tumour microenvironment.
Go-to-market
Patients with genetic and other diseases addressed by its pipeline, including advanced hepatocellular carcinoma and other solid tumours, mucopolysaccharidosis type 1H, sickle cell disease and anaemia, and metabolic conditions; pharmaceutical companies serve as partners for platform application.
Geography
Headquartered in the United Kingdom at the Translation & Innovation Hub, 84 Wood Lane, London W12 0BZ, on Imperial College London's White City campus in west London, where it occupies offices and laboratories for RNAa drug discovery and development. Clinical and research activity extends internationally, including a collaboration with Japan-based Nippon Shinyaku and pipeline work involving institutions such as National Taiwan University College of Medicine and UT Southwestern Medical Center.
History
Corporate records list MiNA Therapeutics Ltd. as founded in 2008 in the United Kingdom. Its clinical programs date from at least 2019, when the Phase 1a/b study of MTL-CEBPA with pembrolizumab began, followed by the OUTREACH2 Phase 2 hepatocellular carcinoma study in 2022 and a paediatric MPS1H study in 2023.
Compiled by commissioned research from 8 cited public sources — announcements, filings, and press listed under research sources below.
Key figures
latest reportedCompany-reported or press-reported figures, each dated to when it was claimed — not independently audited.
Timeline · 5
launches, deals, and filingsThe company presented new preclinical data from its sickle cell disease program at the EHA 2025 Congress.
Publication reporting safety, tolerability, pharmacokinetics and efficacy of MTL-CEBPA combined with pembrolizumab in advanced solid tumours, with biopsy analyses indicating immunomodulatory effects.
MiNA appointed Norbert Bischofberger, Ph.D., to its board as a non-executive director and Bríd Ryan, Ph.D., MPH, as Chief Scientific Officer.
The company presented new preclinical data from its lead sickle cell disease program at the ASH annual meeting.
MiNA announced a research collaboration with Nippon Shinyaku to develop RNAa therapeutics targeting rare neurodegenerative diseases.
Dated company events from announcements, filings, and press; legal rows summarize public dockets and regulator releases.
▸Research sources · 8
primary sources listed
- Minatxminatx.com · web
8 public sources were cited for this profile; the first-party ones are listed here.
Frequently asked questions
- What does Minatx do?
- UK biotech developing small activating RNA (RNAa) therapeutics that restore or boost normal gene function.
- Who are Minatx's investors?
- Minatx's investors include Bootstrap Europe.