Design Therapeutics
Design Therapeutics is a clinical-stage biopharmaceutical company developing proprietary GeneTAC small molecule therapeutics that selectively modulate gene expression at nucleotide repeat expansions to treat serious genetic diseases, with a clinical pipeline spanning Friedreich ataxia, Fuchs dystrophy, myotonic dystrophy, and Huntington's disease.
- Company typePublic
- Founded2019
- HeadquartersSolana Beach, United States
- Headcount51–100
- GTM typeB2B
- OfferingHardware or Manufacturing
What Design Therapeutics does
Design Therapeutics is a clinical-stage biopharmaceutical company headquartered in Carlsbad, California (NASDAQ: DSGN), founded to develop small molecule therapeutics for monogenic degenerative disorders caused by nucleotide repeat expansions. The company's core asset is its proprietary GeneTAC (Gene Targeted Chimera) platform, which engineers small molecules composed of a DNA-binding moiety linked to a ligand moiety to selectively dial gene expression up or down at disease-causing repeat sequences—without requiring gene editing or altering patient DNA. This architecture allows systemic, including CNS, distribution of a conventional small molecule while preserving wild-type gene function.
Design's pipeline targets four repeat-expansion disorders with no approved disease-modifying therapies: DT-216P2 for Friedreich ataxia (intravenous, Phase 1/2 RESTORE-FA), DT-168 for Fuchs Endothelial Corneal Dystrophy (eye drop, Phase 2 biomarker trial), DT-818 for Myotonic Dystrophy Type-1 (Phase 1 dosing initiated in Australia in H1 2026), and a preclinical Huntington's disease program targeting CAG repeats in HTT. May 2026 Phase 1/2 readout showed a 65% increase in frataxin mRNA and a 6.4-point mFARS improvement at the highest dose, with registrational path discussions planned with FDA in Q4 2026.
The business model is pre-commercial R&D with future pharmaceutical commercialization optionality; the company is currently funded by equity capital ($219.8M cash as of Q4 2025, runway into 2029) with no licensing revenue, partnerships, or commercialized products. Upon approval, distribution is expected to follow standard specialty pharmacy and hospital channels for rare disease therapeutics. The leadership team is anchored by repeat biotech operators including CEO Pratik Shah (former CEO of Auspex, acquired by Teva for $3.5B) and co-founder Aseem Ansari, the academic pioneer of synthetic transcription factors that underpin the GeneTAC platform.
Design Therapeutics firmographics
Firmographics- Name
- Design Therapeutics
- Legal name
- Design Therapeutics, Inc.
- Website
- https://designtx.com
- Company type
- Public
- Founded year
- 2019
- Operating status
- Operating
- Headcount range
- 51–100 employees
- Short description
- Design Therapeutics is a clinical-stage biopharmaceutical company developing proprietary GeneTAC small molecule therapeutics that selectively modulate gene expression at nucleotide repeat expansions to treat serious genetic diseases, with a clinical pipeline spanning Friedreich ataxia, Fuchs dystrophy, myotonic dystrophy, and Huntington's disease.
- Ownership category
- akta.pro rank
Design Therapeutics industry classification
Industry- Product category
- Genomic Medicine Therapeutics
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Research and Development in the Physical, Engineering, and Life Sciences (54171)
- SIC
- Pharmaceutical Preparations (2834)
- akta.pro primary industry
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)
- akta.pro secondary industries
- Neurology/CNS Gene Therapies (HLAAACAH), Ophthalmology Gene Therapies (HLAAACAG)
Keywords
Where Design Therapeutics is headquartered
LocationHeadquarters
- HQ city
- Solana Beach
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Design Therapeutics business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Personnel, Technology or R&D, Operations, Marketing or Sales, Others
Revenue model
- Pharmaceutical Development and Commercialization: As a pre-commercial clinical-stage biotechnology company, Design Therapeutics does not currently generate revenue. The company is developing small molecule therapeutics for genetic diseases with plans to pursue regulatory approval and commercialize treatments upon successful clinical trial outcomes.
Go-to-market motion1 record
Marketing channels3 records
Design Therapeutics product offering
Product offeringCore offering
Design Therapeutics is a clinical-stage biopharmaceutical company developing proprietary GeneTAC small molecule therapeutic candidates designed to selectively modulate gene expression at nucleotide repeat expansion mutations. Its pipeline targets serious monogenic genetic diseases with no approved disease-modifying therapies, including Friedreich ataxia (DT-216P2), Fuchs endothelial corneal dystrophy (DT-168), myotonic dystrophy type-1 (DT-818), and Huntington's disease. The GeneTAC approach uses DNA-binding and ligand moieties to dial gene expression up or down without requiring gene editing.
Product overview
Design Therapeutics is a biopharmaceutical company advancing novel small molecule therapeutic candidates through its proprietary GeneTAC® (Gene Targeted Chimera) platform. The company's pipeline consists of four clinical and preclinical programs targeting nucleotide repeat expansion disorders: DT-216P2 for Friedreich Ataxia (Phase 1/2), DT-168 for Fuchs Endothelial Corneal Dystrophy (Phase 2), DT-818 for Myotonic Dystrophy Type-1 (Phase 1 planned), and a Huntington's Disease research program. The GeneTAC® platform enables selective modulation of gene expression without gene editing by using small molecules with DNA-binding and ligand moieties.
Differentiator
Problem solved
Functional benefit
Products and services
- DT-216P2 A GeneTAC small molecule therapeutic candidate for Friedreich ataxia, administered intravenously, designed to increase frataxin RNA transcription in patients with GAA repeat expansions. Currently in Phase 1/2 RESTORE-FA trial for patients with Friedreich ataxia.
- DT-168 A GeneTAC small molecule designed to target CTG repeats in the TCF4 gene for Fuchs Endothelial Corneal Dystrophy, formulated as an eye drop to selectively block transcription of the expansion-containing allele. Phase 1 completed and Phase 2 biomarker trial initiated.
- DT-818 A GeneTAC small molecule designed to target CTG repeats in the DMPK gene for Myotonic Dystrophy Type-1, designed to selectively reduce transcription of the mutant expanded allele. Phase 1 MAD trial planned for H1 2026 in Australia.
- Huntington's Disease Program A GeneTAC small molecule program targeting CAG triplet repeats in the HTT gene for Huntington's disease, designed to selectively reduce toxic mutant HTT (mtHTT) gene product while preserving wild-type HTT (wtHTT) expression. Preclinical research program.
Quantifiable outcome
- 65% increase in frataxin mRNA levels in whole blood
- +3 more outcomes
Companies that use Design Therapeutics
Customer profileSegments4 records
Ideal customer profiles2 records
Design Therapeutics technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
Design Therapeutics partnerships and signals
Strategic signalScale indicators8 records
Recent moves6 records
Expansion highlights6 records
Design Therapeutics competitors and assessment
Company assessmentDirect peers
- Wave Life Sciences: Clinical-stage biotech developing stereopure oligonucleotide therapeutics for repeat expansion disorders including Huntington's disease and DM1. Most directly comparable peer as both companies target the same repeat expansion indications with novel genomic medicine approaches.
- Ionis Pharmaceuticals: Pioneer in antisense oligonucleotide (ASO) therapeutics for genetic diseases. Develops tofersen for SOD1 ALS and has programs in Huntington's disease (tominersen collaboration with Roche), making it a directly comparable peer targeting similar repeat expansion and rare neurological disorders.
- Sage Therapeutics: Biotech developing CNS-targeted small molecule therapeutics for neurological disorders including Huntington's disease. Comparable as a small molecule approach to CNS genetic/neurological diseases with similar development and clinical risk profiles.
- Crinetics Pharmaceuticals: Clinical-stage rare disease biotech developing oral small molecule therapeutics for endocrine disorders and rare diseases. Comparable as a peer-stage small molecule biotech with platform approach, multiple pipeline programs, and similar operational scale and capital structure.
Emerging players
- Beam Therapeutics: Clinical-stage gene editing company developing base editing therapies for genetic diseases including repeat expansion disorders. Competes with GeneTAC for the same Friedreich ataxia and DM1 patient populations using a different genomic medicine modality.
- Intellia Therapeutics: CRISPR-based gene editing company developing in vivo and ex vivo therapies for genetic diseases. Competes for repeat expansion disorder opportunities with a different modality approach, providing investor comparison for genomic medicine platform valuations.
- Solid Biosciences: Clinical-stage gene therapy company developing treatments for Duchenne muscular dystrophy and other genetic diseases. Comparable as a clinical-stage genetic disease biotech with similar development timelines, capital requirements, and pipeline focus on monogenic disorders.
- uniQure: Clinical-stage gene therapy company developing treatments for genetic diseases including Huntington's disease (AMT-130 in Phase 1/2). Comparable as a competing modality targeting the same Huntington's disease patient population with similar clinical timeline.
Broad incumbents
- Alnylam Pharmaceuticals: Established RNAi therapeutics leader with multiple approved products for rare genetic diseases (Onpattro, Givlaari, Oxlumo). Comparable as a nucleic acid-based approach to genetic disease modulation, though with broader portfolio and commercial-stage operations.
- BioMarin Pharmaceutical: Commercial-stage rare disease biotech with multiple approved enzyme replacement and small molecule therapies. Comparable as a strategic acquirer profile and as a benchmark for orphan disease biotech valuations reaching commercialization stage.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat3 records
Key risks6 records
Key highlights6 records
Customer concentration
Design Therapeutics social profiles
Digital presenceDesign Therapeutics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Design Therapeutics leadership team
Management profileNumber of profiles
Profiles10 records
Design Therapeutics funding detail
Funding detailFunding overview
Funding rounds3 records
Investors14 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Design Therapeutics M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Design Therapeutics
What does Design Therapeutics do?
Design Therapeutics is a clinical-stage biopharmaceutical company developing proprietary GeneTAC small molecule therapeutic candidates designed to selectively modulate gene expression at nucleotide repeat expansion mutations. Its pipeline targets serious monogenic genetic diseases with no approved disease-modifying therapies, including Friedreich ataxia (DT-216P2), Fuchs endothelial corneal dystrophy (DT-168), myotonic dystrophy type-1 (DT-818), and Huntington's disease. The GeneTAC approach uses DNA-binding and ligand moieties to dial gene expression up or down without requiring gene editing.
Is Design Therapeutics a public or private company?
Design Therapeutics is a public company. It is classified as public and is currently operating.
When was Design Therapeutics founded?
Design Therapeutics was founded in 2019. It employs 51 to 100 people.
Where is Design Therapeutics based?
Design Therapeutics is headquartered in Solana Beach, United States, in the North America region.
How does Design Therapeutics make money?
One revenue line is on record: pharmaceutical Development and Commercialization.
Who are Design Therapeutics's main competitors?
Direct peers on record are Wave Life Sciences, Ionis Pharmaceuticals, Sage Therapeutics and Crinetics Pharmaceuticals. Emerging players are Beam Therapeutics, Intellia Therapeutics, Solid Biosciences and uniQure. Broad incumbents are Alnylam Pharmaceuticals and BioMarin Pharmaceutical.
Does Design Therapeutics have an API?
No public API is recorded for Design Therapeutics.
What industry is Design Therapeutics in?
Design Therapeutics's product category is Genomic Medicine Therapeutics. Its primary akta.pro industry code is HLAAACAE, Gene Therapy for Rare/Monogenic Diseases, with a secondary code of HLAAACAH, Neurology/CNS Gene Therapies. Its NAICS code is 541714 and its SIC code is 2834.