Regel Therapeutics is targeting gene regulation to fix various disease-affected cells. Within each cell gene expression is tightly regulated through epigenetics - alteration can lead to severe genetic diseases. Regel’s strategy is to target and restore the epigenome, without editing or damaging the DNA sequence. To achieve this, Regel is using a deactivated Cas system (dCas9) which harnesses the natural mechanisms of gene regulation allowing for efficient and permanent restoration of normal gene expression, in a safe manner. Clinical application will use AAV delivery and with the modular nature of dCas9 targeting, Regel can use their precision medicine approach across various genetic diseases.

Regel Therapeutics

Founded: 2020
Location: Cambridge, MA
Employee range: 11 - 50
Clinical stage: Clin0
Therapy area: Dravet syndrome
Drug types: NEU, GEN, PED, MUS, CVV
Lead product: RT-101

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