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Regel Therapeutics

Regel Therapeutics

Cambridge, United States· Est.
regeltherapeutics.com
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Private Company

Total funding raised: $15M

Overview

Regel Therapeutics, founded in 2021 and based in Cambridge, USA, is developing epigenetic therapies for severe genetic diseases. Its core technology, the Targeted EpiEditing platform, combines a deactivated CRISPR-Cas9 system with epigenetic modulators and cell-type-specific AAV delivery vectors to correct gene expression exclusively in affected cells. The company is led by an experienced team, including CEO Stephen Farr, who previously led Zogenix to acquisition, and is advancing two lead preclinical programs for Dravet Syndrome and SCN2A Haploinsufficiency.

NeurologyGenetic Disorders

Technology Platform

Targeted EpiEditing platform: a modular system combining a deactivated CRISPR-Cas9 (dCas9) fused to an epigenetic modulator for precise gene expression control, delivered via engineered AAV vectors with proprietary cell-type-specific regulatory elements to confine therapy to affected cells.

Funding History

1
Total raised:$15M
Seed$15M

Opportunities

The modular Targeted EpiEditing platform offers a pipeline-in-a-box opportunity, allowing the company to address numerous severe genetic diseases caused by haploinsufficiency or dysregulated gene expression beyond its initial neurology focus.
The high unmet need and premium pricing potential in rare genetic disorders like Dravet Syndrome create a significant commercial opportunity for a first-in-class disease-modifying therapy.

Risk Factors

The novel epigenetic editing approach carries unproven long-term durability and safety risks in humans, including potential off-target epigenetic effects.
The company is also dependent on complex AAV delivery, which faces challenges related to immunogenicity, manufacturing, and achieving truly cell-type-specific expression in patients.
As a preclinical, private company, it faces significant financing and execution risk to reach clinical proof-of-concept.

Competitive Landscape

Regel competes in the crowded genetic medicine space for neurological disorders, facing competition from gene replacement (e.g., Neurocrine Biosciences), antisense oligonucleotides (e.g., Ionis, Biogen), and traditional gene editing companies. Its primary differentiation is its focus on epigenetic correction without DNA alteration and its engineered vectors for cell-type specificity, a niche currently with few clinical-stage players.

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