What Regel Therapeutics does

Regel Therapeutics is a privately held, clinical-stage gene therapy company founded in 2020 and headquartered at LabCentral in Cambridge, Massachusetts, with a research footprint at QB3-Bakar Labs in Berkeley, California. The company is pioneering a precision genetic medicine modality it calls Targeted EpiEditing, which uses a deactivated CRISPR-Cas9 (dCAS9) fused with an epigenetic modulator and delivered via AAV vectors carrying proprietary cell-specific regulatory elements. The intent of the platform is to permanently correct dysregulated gene expression only in disease-affected cells without altering the underlying DNA sequence, addressing the limitations of conventional editing-based gene therapies.

The company's lead programs are RT101 for Dravet Syndrome (addressing approximately 17,000 patients across the US and Europe) and RT102 for SCN2A Haploinsufficiency (approximately 10,000 patients across the US and Europe), both currently in preclinical/IND-enabling stages. The disclosed pipeline comprises six indications spanning three CNS targets and three muscle gene targets; the muscle programs are being developed in collaboration with an unnamed strategic industry partner. The platform was licensed from UCSF (CRISPRa) and is grounded in scientific work by co-founders Dr. Navneet Matharu (UCSF) and Dr. Jordane Dimidschstein (Broad Institute), with peer-reviewed publications in Nature, Science, Nature Neuroscience, and Nature Review Drug Discovery supporting the underlying science. A September 2025 Nature publication from the labs of Dr. Kevin Bender and Dr. Nadav Ahituv at UCSF provided preclinical proof-of-concept for the SCN2A program.

Regel operates as a pre-revenue, R&D-intensive biotech. The only disclosed funding event is a $1 million non-dilutive grant (September 2023) from the HS Chau Foundation through the Women in Enterprising Science Program at the Innovative Genomics Institute. Commercial economics will be derived from future one-time sales (or licensing) of gene therapy products for severe, rare genetic diseases, with pricing not publicly disclosed but expected to follow rare-disease pricing benchmarks post-approval. Distribution is expected to flow through specialty pharmacy networks and specialized treatment centers. Leadership comprises an experienced rare-disease commercial operator as CEO (Stephen J. Farr, ex-Zogenix/UCB), scientific co-founders in the President/COO and CSO roles, and a board and Scientific Advisory Board staffed with executives and academics from UCSF, Broad Institute, NYU, Harvard, Columbia, Intellia, and AvenCell.

Name

Regel Therapeutics

Website

https://regeltherapeutics.com

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

11–50 employees

Short description

Regel Therapeutics is a private, preclinical-stage gene therapy company developing a non-editing CRISPR-based "Targeted EpiEditing" platform to permanently correct gene expression in disease-affected cells, with lead programs in Dravet Syndrome and SCN2A Haploinsufficiency.

Ownership category

akta.pro rank

Product category

Gene Therapy

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Neurology/CNS Gene Therapies (HLAAACAH)

akta.pro secondary industries

Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

Keywords

• Epigenetic gene therapy
• Rare disease therapeutics
• CRISPR-based medicines
• Gene expression modulation
• AAV gene therapy

Headquarters

HQ city

Boston

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Others

Revenue model

1. Gene Therapy Product Sales: As a clinical-stage biopharmaceutical company, Regel Therapeutics plans to commercialize its proprietary gene therapy products (RT101 for Dravet Syndrome, RT102 for SCN2A Haploinsufficiency) upon regulatory approval. The company is developing a pipeline of clinical products across multiple indications.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

Regel Therapeutics is developing gene therapy products built on its Targeted EpiEditing platform, a non-editing CRISPR-based technology that uses deactivated Cas9 fused with an epigenetic modulator to permanently correct gene expression exclusively in affected cells. Its lead assets, RT101 for Dravet Syndrome and RT102 for SCN2A Haploinsufficiency, are preclinical gene therapy candidates targeting severe pediatric CNS disorders. The company also develops three additional CNS and three muscle-targeted programs through its broader pipeline.

Differentiator

Problem solved

Functional benefit

Products and services

• RT101 RT101 is a preclinical/IND-enabling gene therapy candidate for the treatment of Dravet Syndrome, a severe genetic epilepsy. The product uses Regel's Targeted EpiEditing platform to correct gene expression exclusively in affected cells without altering the genome; approximately 17,000 patients across the US and Europe are affected.
• RT102 RT102 is a preclinical/IND-enabling gene therapy candidate for the treatment of SCN2A Haploinsufficiency, a leading monogenic cause of neurodevelopmental disorders and early-onset epilepsy. The product uses the same Targeted EpiEditing platform approach as RT101 and addresses approximately 10,000 patients across the US and Europe.

Quantifiable outcome

• Preclinical proof-of-concept data published in Nature (September 2025) demonstrating CRISPR activation restores SCN2A expression and ameliorates disease-related phenotypes in mouse and human models of SCN2A-haploinsufficiency
• +1 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability4 records

Feature4 records

Partnerships

Three partnerships are on record, tiered core and minor.

• UCSF (University of California, San Francisco)coreTechnology or IntegrationRegel Therapeutics has licensed UCSF's CRISPRa technology to treat patients with haploinsufficiency disorders, including SCN2A loss of function. Laboratories of Dr. Kevin Bender and Dr. Nadav Ahituv at UCSF conducted landmark preclinical research published in Nature demonstrating functional rescue of SCN2A. Dr. Ahituv is a co-founder of Regel Therapeutics.
• Sarepta TherapeuticscoreStrategic or Co-development PartnerStrategic collaboration with Sarepta Therapeutics for development of proprietary programs. Mentioned in CEO Stephen Farr's appointment announcement as part of Regel's pipeline strategy.
• Strategic Industry Partner (Muscle Gene Programs)minorStrategic or Co-development PartnerCollaboration with a strategic industry partner for development of three muscle gene targets as part of Regel's broader pipeline expansion beyond CNS indications.

Scale indicators4 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Encoded Therapeutics: Encoded develops AAV-delivered gene regulation therapies for CNS disorders, including ETX101 for Dravet Syndrome (RT101's indication) and SCN1A+ programs. Like Regel, it targets CNS haploinsufficiency with cell-type specific regulatory elements and AAV delivery.
• Tevard Biosciences: Tevard is developing tRNA-based and other RNA-suppression therapies for haploinsufficiency disorders including Dravet Syndrome. CEO Stephen Farr sits on Tevard's board, indicating a close operational link to Regel's leadership. Both pursue upregulation strategies for the same target indications.
• Passage Bio: Passage Bio develops AAV-delivered gene therapies for rare monogenic CNS disorders, with clinical programs in GM1 gangliosidosis, frontotemporal dementia, and Krabbe disease. It directly overlaps Regel's modality (AAV + CNS) and patient population focus (rare monogenic diseases).
• Taysha Gene Therapies: Taysha is a clinical-stage CNS-focused AAV gene therapy company with a broad pipeline across rare neurological diseases, including multiple programs targeting haploinsufficiency. It is a directly comparable modality-and-indication peer.
• Voyager Therapeutics: Voyager develops AAV-based gene therapies for severe CNS diseases (e.g., Parkinson's, ALS, Huntington's). It shares Regel's CNS AAV delivery modality and rare neurological disease focus, and competes for similar KOL and clinical-trial-site resources.
• Stoke Therapeutics: Stoke is developing antisense oligonucleotides (ASOs) that increase expression of haploinsufficient genes, with a lead program (zorevunersen, Phase 3) in Dravet Syndrome — Regel's RT101 indication. Both companies pursue upregulation of disease genes via nucleic-acid-based platforms rather than conventional gene replacement.

Broad incumbents

• Beam Therapeutics: Beam is a clinical-stage base-editing company with CNS and rare disease programs. Like Regel, it pursues precise gene expression modulation, though via base editing rather than epigenetic modulation — positioning it as a broader incumbent in next-generation genetic medicine.
• uniQure: uniQure is a commercial-stage AAV gene therapy company (Hemgenix approval) with CNS programs including Huntington's and ALS. It is a broader incumbent in the AAV gene therapy space that competes for manufacturing capacity, regulatory expertise, and partner attention.
• Intellia Therapeutics: Intellia is a clinical-stage CRISPR-based gene editing company with in vivo and ex vivo programs across multiple tissues including CNS. While its modality (CRISPR/Cas9 editing) differs from Regel's non-editing EpiEditing approach, it competes in the same broad CRISPR-therapeutics category and shares key talent pipelines (Regel's Chairman Andrew Schiermeier was Intellia's COO).

Emerging players

• Ascidian Therapeutics: Ascidian is developing RNA editing therapeutics for severe neurological diseases, including Stargardt disease and other CNS indications. It shares Regel's interest in non-cutting approaches to gene expression modulation but uses RNA editing rather than CRISPR-based epigenetic modulation.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds1 record

Investors1 record

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Regel Therapeutics