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NCT06775561RECRUITINGanonymous

Precision Diagnosis and Therapy for Rare Diseases by Interpreting Non-coding Genomes (PARADIGM)

Sponsor

Source record

IRCCS Azienda Ospedaliero-Universitaria di Bologna

Phase

Source record

Observational

Modality

AI-normalized

gene therapy

Target

AI-normalized

Non-coding genomic variants associated with Eye Diseases (ED) and Neuro-Muscular Diseases (NMD). The study aims to identify regulatory and splicing variants that contribute to the genetic heterogeneity of these disorders.

Indication / condition

AI-normalized

Neuromuscular Diseases

Intervention

Source record

PARADIGM study aims to streamline the process from genomic characterization of RGD patients with ED/NMD to identification of the suitable personalized therapy.

Source & freshness

Source record

NCT ID

NCT06775561

Original source

ClinicalTrials.gov

Source last updated

Jan 15, 2025

Ingested at

Jun 13, 2026

Internal sync

Jun 13, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

Open original registry record
View original source fields

NCT ID

NCT06775561

Title

Precision Diagnosis and Therapy for Rare Diseases by Interpreting Non-coding Genomes (PARADIGM)

Sponsor

IRCCS Azienda Ospedaliero-Universitaria di Bologna

Status

RECRUITING

Phase

Observational

Condition raw

Neuromuscular Diseases, Eye Diseases, Genetic Disease

Condition normalized

Neuromuscular Diseases, Eye Diseases, Genetic Disease

Modality raw

gene therapy

Modality normalized

gene therapy

Target raw

Non-coding genomic variants associated with Eye Diseases (ED) and Neuro-Muscular Diseases (NMD). The study aims to identify regulatory and splicing variants that contribute to the genetic heterogeneity of these disorders.

Target normalized

Non-coding genomic variants associated with Eye Diseases (ED) and Neuro-Muscular Diseases (NMD). The study aims to identify regulatory and splicing variants that contribute to the genetic heterogeneity of these disorders.

Interventions

PARADIGM study aims to streamline the process from genomic characterization of RGD patients with ED/NMD to identification of the suitable personalized therapy.

Public preview

Source record

The PARADIGM study, sponsored by IRCCS Azienda Ospedaliero-Universitaria di Bologna, is positioned to enhance the understanding and treatment of rare genetic disorders, specifically ED and NMD. By focusing on non-coding genomic variants, the study addresses a significant gap in current genetic diagnostics, potentially leading to improved patient outcomes and personalized therapies. The market for genetic testing and targeted therapies for rare diseases is expanding, driven by advancements in genomics and increasing demand for precision medicine. The outcomes of this study could provide a competitive edge in the rare disease therapeutics space, particularly for companies involved in gene therapy and genomic medicine. Diligence considerations should include the regulatory landscape for genetic therapies and the potential for partnerships with biotech firms specializing in RNA therapeutics and genome editing technologies.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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TrialSignal

Clinical trial intelligence report

Precision Diagnosis and Therapy for Rare Diseases by Interpreting Non-coding Genomes (PARADIGM)

Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.

Generated

Jun 16, 2026

NCT ID

NCT06775561

Status

RECRUITING

Phase

Observational

Sponsor

IRCCS Azienda Ospedaliero-Universitaria di Bologna

Executive brief

Investment-Ready Snapshot

The PARADIGM study, sponsored by IRCCS Azienda Ospedaliero-Universitaria di Bologna, is positioned to enhance the understanding and treatment of rare genetic disorders, specifically ED and NMD. By focusing on non-coding genomic variants, the study addresses a significant gap in current genetic diagnostics, potentially leading to improved patient outcomes and personalized therapies. The market for genetic testing and targeted therapies for rare diseases is expanding, driven by advancements in genomics and increasing demand for precision medicine. The outcomes of this study could provide a competitive edge in the rare disease therapeutics space, particularly for companies involved in gene therapy and genomic medicine. Diligence considerations should include the regulatory landscape for genetic therapies and the potential for partnerships with biotech firms specializing in RNA therapeutics and genome editing technologies.

Source & freshness

Provenance

SourceClinicalTrials.gov
Source updatedJan 15, 2025
Internal syncJun 13, 2026
Model versiontrialsignal-ai-v1

https://clinicaltrials.gov/study/NCT06775561

Indication

Neuromuscular Diseases

Modality

gene therapy

Target

Non-coding genomic variants associated with Eye Diseases (ED) and Neuro-Muscular Diseases (NMD). The study aims to identify regulatory and splicing variants that contribute to the genetic heterogeneity of these disorders.

Intervention

PARADIGM study aims to streamline the process from genomic characterization of RGD patients with ED/NMD to identification of the suitable personalized therapy.

Source record

Protocol Description

Detailed source ingestion pending.

Source record

Outcome Measures

Detailed source ingestion pending.

Source record

Eligibility

Detailed source ingestion pending.

AI analysis

Known Results And Readout Context

Detailed source ingestion pending.

IP intelligence

Patent And IP Landscape

Detailed source ingestion pending.

Source record

Contacts

Detailed source ingestion pending.