Company profile: Regulus Therapeutics
1.1 - Company Overview
Company description
- Provider of microRNA-based therapeutics designed to inhibit dysregulated microRNA targets for genetically based orphan diseases, particularly orphan kidney diseases. Portfolio includes RGLS8429, a next-generation anti-miR-17 in Phase 1b for autosomal dominant polycystic kidney disease (ADPKD), supported by ongoing clinical trials to evaluate efficacy and safety.
Products and services
- MicroRNA-based Therapeutics: Regulus architects therapeutics that inhibit dysregulated microRNA targets for genetically based orphan diseases—particularly orphan kidney diseases—leveraging small microRNAs (20–25 nucleotides)
- MicroRNA-targeted
- RGLS8429: Regulus engineers a next-generation anti-miR-17 compound undergoing Phase 1b clinical evaluation for Autosomal Dominant Polycystic Kidney Disease (ADPKD), targeting miR-17 to inhibit dysregulated microRNA targets
- Next-generation
- Clinical Trials: Regulus conducts ongoing studies evaluating efficacy and safety of its therapeutic candidates, including RGLS8429 for ADPKD, advancing microRNA medicines via clinical evaluation
- Clinical-stage
Key contacts
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Financial details
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1.2 - Competitors and similar companies to Regulus Therapeutics
Rocket Pharmaceuticals
HQ: United States
Website
- Description: Provider of gene therapies for rare childhood disorders, developing lentiviral and AAV vector-based programs including RP-L102 for Fanconi Anemia, RP-L301 for Pyruvate Kinase Deficiency, an AAV program for Danon Disease, and gene therapies for LAD-I, PKP2 arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
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- Total funding raised: $●●●m
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Kriya Therapeutics
HQ: United States
Website
- Description: Provider of gene therapies for ophthalmology, neurology, and metabolic diseases, including KRIYA-825 for geographic atrophy (inhibiting C3/C5; one-time suprachoroidal), KRIYA-586 for thyroid eye disease (blocking IGF1 receptor), KRIYA-839 for diabetes (insulin and glucokinase), KRIYA-497 for NASH (FGF21), and KRIYA-748 and KRIYA-382 for trigeminal neuralgia and focal epilepsy via chemogenetically gated ion channels.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
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- Total funding raised: $●●●m
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Akamis Bio
HQ: United Kingdom
Website
- Description: Provider of clinical-stage oncology therapeutics using its Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform to develop viral vector-based gene therapies that target solid tumors and express therapeutic proteins to stimulate an immune response. Offerings include NG-350A (secreted CD40 agonist monoclonal antibody expressed within tumors), clinical trials of T-SIGn® agents, and collaborations with academia, research institutes, and biopharma.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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Sirna Therapeutics
HQ: United States
Website
- Description: Provider of biotechnology research and development focused on RNA interference (RNAi) for human disease therapy, creating RNAi-based therapies to ultimately treat patients.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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- Acquisitions: ●●
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Editas Medicine
HQ: United States
Website
- Description: Provider of clinical-stage genome editing medicines and a CRISPR gene editing platform, including Reni-cel for severe sickle cell disease and transfusion-dependent beta thalassemia (editing CD34+ cells at HBG1/HBG2 promoters), EDIT-101 for Leber congenital amaurosis 10 (repairing the IVS26 CEP290 allele), and ex vivo and in vivo gene editing medicines for ocular, blood diseases, and cancer.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
- Acquisitions: ●●
View full Editas Medicine company profile →
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2.M&A buyers
2.1 Potential strategic acquirers in the sector
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2.2 - Strategic buyer groups for Regulus Therapeutics
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3. Investors and private equity firms
3.1 - Buyout funds investing in similar companies to Regulus Therapeutics
2.2 - Growth funds investing in similar companies to Regulus Therapeutics
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4 - Top valuation comps for Regulus Therapeutics
4.2 - Public trading comparable groups for Regulus Therapeutics
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