Regel Therapeutics is pioneering a targeted gene therapy approach aimed at correcting abnormal gene expression specifically in disease-affected cells. Their technology leverages a deactivated Cas system (dCas) to interact with the epigenome without altering the DNA itself. This method restores normal gene expression by utilizing the body's natural gene regulation mechanisms, which is particularly crucial for treating severe genetic diseases that currently lack effective cures. The company’s innovative approach is built on years of research and aims to provide a first-in-class therapeutic intervention for patients suffering from intractable genetic disorders.

Central to Regel's strategy is their T3 platform, which integrates adeno-associated virus (AAV) delivery with proprietary technologies designed to modulate gene expression. This platform features a programmable dCas coupled with a modulator that fine-tunes gene expression back to healthy levels, alongside a regulatory element that ensures the intervention is restricted to the appropriate cells. This modular design allows for targeted delivery through a single-vector system, enabling efficient and selective therapeutic effects with the potential for long-term benefits. By addressing the challenge of off-target effects commonly associated with gene therapies, Regel Therapeutics aims to enhance the safety and efficacy of their treatments, thereby advancing the field of precision medicine.

United States

Regel’s technology utilizes a deactivated Cas system (dCas) which targets the epigenome without editing or damaging the DNA. This approach harnesses the natural mechanisms of gene regulation, allowing for efficient and permanent restoration of normal gene expression.

Cardiovascular Diseases

Genetic Diseases

Musculoskeletal Diseases

Nervous System Diseases

Regel Therapeutics

Dravet syndrome

RT101

Gene Therapy

SCN2A Haploinsufficiency

RT102

SYNGAP1 Haploinsufficiency

RT103

Muscular disease

RT201

Biologics

Cardiovascular disease

RT301

RT302

Massachusetts

Adeno-associated viral vector (AAV)

Antiviral Therapies

Devices

Therapeutic

Regel Therapeutics

Company Details