Solid Biosciences
Solid Biosciences is a clinical-stage gene therapy company developing AAV-based treatments for rare neuromuscular and cardiac diseases including Duchenne muscular dystrophy (SGT-003, Phase 3), Friedreich's ataxia (SGT-212, Phase 1b), and CPVT (SGT-501, Phase 1b), leveraging its proprietary POLARIS-101™ next-generation capsid platform.
- Company typePublic
- Founded2013
- HeadquartersCharlestown, United States
- Headcount101–250
- GTM typeB2B
- OfferingHardware or Manufacturing
What Solid Biosciences does
Solid Biosciences is a clinical-stage precision genetic medicine company developing a portfolio of AAV (adeno-associated virus) gene therapies targeting rare neuromuscular and cardiac diseases. Founded in 2013 by Ilan and Annie Ganot after their son's Duchenne muscular dystrophy diagnosis, the company is headquartered in Charlestown, Massachusetts, trades on NASDAQ as SLDB, and has 51-100 employees. Its lead clinical program, SGT-003, is an investigational next-generation microdystrophin gene therapy using the proprietary POLARIS-101™ (AAV-SLB101) capsid platform, currently in Phase 1/2 (INSPIRE DUCHENNE, 40 participants dosed) and Phase 3 (IMPACT DUCHENNE, initiated May 2026) trials for Duchenne muscular dystrophy. Two additional clinical programs are advancing: SGT-212 (Friedreich's ataxia, Phase 1b FALCON trial initiated Jan 2026), which uses a first-in-class dual-route administration approach combining stereotactic MRI-guided cerebellar infusion with intravenous delivery, and SGT-501 (CPVT, Phase 1b Artemis trial), which delivers a functional CASQ2 gene to heart muscle. Three preclinical cardiac programs (SGT-601, SGT-401, SGT-701) round out the pipeline.
The company's core technology platform is the rationally designed POLARIS-101™ AAV capsid, which demonstrates enhanced muscle transduction and reduced liver biodistribution compared to first-generation capsids, supported by a broader capsid library with 50+ licensing and evaluation agreements. Manufacturing relies on a proprietary transient transfection-based gene therapy production process with in-house process development capabilities. The company pursues a pre-revenue clinical-stage business model with future revenue expected from gene therapy product sales (comparable DMD therapies priced at ~$3.2M per treatment) and non-exclusive licensing of capsid technology to other gene therapy developers. Distribution is structured through clinical trial sites globally (US, Canada, Italy, UK, Australia), with planned specialty treatment center commercialization upon regulatory approval.
The company is led by President and CEO Bo Cumbo (former Sarepta CCO), CFO Kevin Tan, CMO Gabriel Brooks (former Pfizer Rare Cardiovascular TA Head), and Chairman Ian Smith (former Vertex CFO/COO). Capital structure is anchored by repeat institutional investors including Perceptive Advisors, Bain Capital Life Sciences, and RA Capital Management, with cumulative private placement funding exceeding $880M since 2022. The most recent $240M placement in March 2026 extended cash runway into H1 2028, supporting advancement of three clinical-stage programs and expansion of the capsid platform through licensing partnerships.
Solid Biosciences firmographics
Firmographics- Name
- Solid Biosciences
- Legal name
- Solid Biosciences Inc.
- Website
- https://solidbio.com
- Company type
- Public
- Founded year
- 2013
- Operating status
- Operating
- Headcount range
- 101–250 employees
- Short description
- Solid Biosciences is a clinical-stage gene therapy company developing AAV-based treatments for rare neuromuscular and cardiac diseases including Duchenne muscular dystrophy (SGT-003, Phase 3), Friedreich's ataxia (SGT-212, Phase 1b), and CPVT (SGT-501, Phase 1b), leveraging its proprietary POLARIS-101™ next-generation capsid platform.
- Ownership category
- akta.pro rank
Solid Biosciences industry classification
Industry- Product category
- Gene Therapy Biologics
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)
- akta.pro primary industry
- AAV Vector Gene Therapy Developers (HLAAACAA)
- akta.pro secondary industries
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Neurology/CNS Gene Therapies (HLAAACAH), Rare Cardiovascular & Vascular Disorder Therapies (HLAIAIAH), Gene Therapy Delivery Devices & Administration Systems (catheters, intrathecal, ocular delivery) (HLAAACAL)
Keywords
Where Solid Biosciences is headquartered
LocationHeadquarters
- HQ city
- Charlestown
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Solid Biosciences business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Infrastructure, Marketing or Sales
Revenue model
- Gene Therapy Product Sales: Pre-revenue clinical stage company. Future revenue expected from commercialization of gene therapy candidates following regulatory approval. Gene therapies for rare diseases typically command premium pricing (e.g., $3.2M for comparable DMD therapies).
- Licensing and Technology Access: Non-exclusive licensing of proprietary capsid technology (AAV-SLB101/POLARIS-101™) to other gene therapy developers. Generates revenue through licensing fees and potential milestone payments.
Pricing tiers
| Model | Billing | Price |
|---|---|---|
| Other | One time/ perpetual license | No commercial products available for pricing |
Go-to-market motion2 records
Distribution channels3 records
Marketing channels5 records
Solid Biosciences product offering
Product offeringCore offering
Solid Biosciences is a clinical-stage precision genetic medicine company developing AAV gene therapy candidates for rare neuromuscular and cardiac diseases, including Duchenne muscular dystrophy (SGT-003), Friedreich's ataxia (SGT-212), and CPVT (SGT-501). The company leverages its proprietary POLARIS-101 (AAV-SLB101) next-generation capsid platform to deliver therapeutic genes and out-licenses the capsid technology to other gene therapy developers.
Product overview
Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases. The company's pipeline includes multiple clinical-stage programs: SGT-003 (Duchenne muscular dystrophy, Phase 1/2 and Phase 3), SGT-212 (Friedreich's ataxia, Phase 1b), and SGT-501 (CPVT, Phase 1b), along with preclinical cardiac programs (SGT-601, SGT-401, SGT-701). The company has developed POLARIS-101™ (AAV-SLB101), a next-generation muscle-tropic capsid platform that is being out-licensed to partners and is the delivery vehicle for its lead clinical programs. The technology platform includes a broader capsid library with over 50 evaluation agreements.
Differentiator
Problem solved
Functional benefit
Brands
- POLARIS-101™: Next-generation AAV capsid technology (AAV-SLB101) for muscle-targeted gene therapy with enhanced transduction and reduced liver biodistribution.
Products and services
- SGT-003 Investigational next-generation microdystrophin gene therapy for Duchenne muscular dystrophy using the POLARIS-101 (AAV-SLB101) capsid for improved muscle targeting and reduced liver biodistribution. Currently in Phase 1/2 INSPIRE DUCHENNE and Phase 3 IMPACT DUCHENNE trials.
- SGT-212 First-in-class dual-route gene therapy for Friedreich's ataxia delivering a functional frataxin (FXN) gene via intradentate nucleus infusion followed by intravenous administration to address neurological, cardiac, and systemic manifestations. Currently in Phase 1b FALCON trial.
- SGT-501 Gene therapy for catecholaminergic polymorphic ventricular tachycardia (CPVT) caused by RYR2 mutations, delivering a functional CASQ2 gene to heart muscle cells to address abnormal calcium signaling. In Phase 1b Artemis trial.
- SGT-601 Preclinical gene therapy candidate for TNNT2-mediated dilated cardiomyopathy that delivers the TNNT2 gene to address cardiac dysfunction caused by TNNT2 mutations.
- SGT-401 Preclinical gene therapy candidate for BAG3-mediated dilated cardiomyopathy.
- SGT-701 Preclinical gene therapy candidate for RBM20-mediated dilated cardiomyopathy.
- POLARIS-101 (AAV-SLB101) Capsid Platform Next-generation rationally designed AAV capsid technology with enhanced muscle tropism, reduced liver biodistribution, and robust transduction. Available for non-exclusive out-licensing to gene therapy developers; serves as the delivery vehicle for Solid's lead clinical programs.
Quantifiable outcome
- 60% mean microdystrophin expression in muscle fibers in Phase 1/2 trial participants
- +4 more outcomes
Companies that use Solid Biosciences
Customer profileNamed customers1 record
Segments4 records
Ideal customer profiles2 records
Solid Biosciences technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature5 records
Solid Biosciences partnerships and signals
Strategic signalPartnerships
Ten partnerships are on record, tiered strategic and core.
- NanoMosaicstrategicCo-marketing agreement combining Solid's POLARIS-101™ next-generation capsid technology with NanoMosaic's FDA AMT-designated Tessie™ platform for multi-omic capsid and transgene analytics. Partnership aims to provide gene therapy developers with integrated access to advanced delivery and measurement technologies.
- Andelyn BiosciencescoreNon-exclusive worldwide licensing agreement for use of proprietary next-generation capsid AAV-SLB101 (POLARIS-101™) in gene therapy manufacturing. The agreement supports further development of AAV-SLB101, which has demonstrated positive safety and efficacy signals in clinical and preclinical studies.
- ICS MaugeristrategicResearch collaboration with Dr. Silvia Priori's lab at ICS Maugeri in Pavia, Italy. Solid in-licensed the therapeutic approach for SGT-501 (CASQ2 gene therapy for CPVT) based on pioneering research from Dr. Priori's team.
- Friedreich's Ataxia Research Alliance (FARA)strategicOngoing partnership with FARA for Friedreich's ataxia clinical program development. FARA provides guidance and support shaping the clinical program. Collaboration helps advance clinically meaningful therapies for the FA community.
- SADS FoundationstrategicPartnership with SADS Foundation for CPVT community engagement. Plans to host community webinars to educate patients and families about investigational gene therapy SGT-501 and upcoming clinical trials.
- Parent Project Muscular Dystrophy (PPMD)strategicCharitable partnership with PPMD as part of Solid's community engagement. PPMD is a leading Duchenne advocacy organization.
- Cure DuchennestrategicCharitable partnership with Cure Duchenne as part of Solid's community engagement initiatives.
- Muscular Dystrophy Association (MDA)strategicCharitable partnership and conference participation with MDA. Solid presents clinical data at MDA Clinical & Scientific Conference.
- Cooperative International Neuromuscular Research Group (CINRG)strategicPPMD partnership with CINRG to support expanded Duchenne Natural History Study (eDNHS), involving participation from multiple pharmaceutical companies, academic institutions, and clinical sites worldwide.
- Mayo ClinicstrategicCollaboration with Mayo Clinic for cardiac gene therapy programs. Listed as partner on pipeline page with six undisclosed targets.
Scale indicators10 records
Recent moves7 records
Expansion highlights6 records
Solid Biosciences competitors and assessment
Company assessmentDirect peers
- Sarepta Therapeutics: Sarepta Therapeutics is the leading developer of gene therapies for Duchenne muscular dystrophy, with Elevidys (delandistrogene moxeparvovec) already approved and generating commercial revenue. It is Solid's most direct competitor in DMD gene therapy and the benchmark for both efficacy and pricing in the indication.
- Regenxbio: Regenxbio is an AAV gene therapy platform company that licenses its NAV capsid technology and develops internal rare disease programs including ABBV-RGX-314 (wet AMD) and RGX-121 (Hunter syndrome). It directly competes with Solid's POLARIS-101 capsid licensing model and has a similar DMD partnership history.
- uniQure: uniQure is an AAV gene therapy company focused on rare diseases with a clinical-stage DMD program and an approved hemophilia B therapy (Hemgenix). It directly competes with Solid in neuromuscular and AAV-mediated rare disease gene therapy development and shares a similar target customer base of rare disease specialists.
- Krystal Biotech: Krystal Biotech is a commercial-stage gene therapy company (Vyjuvek for DEB) with a pipeline including an AAV-based DMD program. It is comparable to Solid in its rare disease gene therapy focus and shares the same regulatory pathway and rare disease commercial model.
- Lexeo Therapeutics: Lexeo Therapeutics is a clinical-stage AAV gene therapy company with a strong focus on cardiac genetic diseases, including programs in Friedreich's ataxia cardiomyopathy and other inherited cardiomyopathies. It is one of Solid's closest direct peers in the cardiac AAV gene therapy space.
- Tenaya Therapeutics: Tenaya Therapeutics is a clinical-stage genetic medicines company focused on cardiac diseases, developing both gene therapies and gene editing approaches for inherited cardiomyopathies. It is comparable to Solid's SGT-501 and preclinical cardiac programs (SGT-601/401/701) in target indication and modality.
- 4D Molecular Therapeutics: 4D Molecular Therapeutics is a clinical-stage AAV gene therapy company that uses proprietary capsid engineering to develop targeted therapies for rare diseases including DMD and cardiac indications. Its Therapeutic Vector Evolution platform is comparable to Solid's POLARIS-101 capsid library.
- Rocket Pharmaceuticals: Rocket Pharmaceuticals is a clinical-stage AAV gene therapy company developing treatments for rare diseases including Danon disease and LAD-I. It shares Solid's model of targeting ultra-rare monogenic diseases with AAV-delivered gene therapy and operates in a similar commercialization context via specialty treatment centers.
Broad incumbents
- Pfizer: Pfizer is a large pharmaceutical company with a significant rare disease gene therapy franchise, including Beqvez (approved hemophilia B therapy) and a discontinued DMD gene therapy program. Its rare cardiovascular TA leadership also overlaps with Solid's cardiac focus, and Solid's CMO Gabriel Brooks joined from Pfizer.
- BioMarin Pharmaceutical: BioMarin Pharmaceutical is an established rare disease biopharmaceutical company with a commercial gene therapy (Roctavian for hemophilia A) and an extensive rare disease portfolio. It is comparable to Solid in its focus on ultra-rare genetic disease therapeutics with premium pricing and specialty distribution.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks6 records
Key highlights7 records
Customer concentration
Solid Biosciences social profiles
Digital presenceSolid Biosciences financial estimates
Financial estimateRevenue estimate
Valuation estimate
Solid Biosciences leadership team
Management profileNumber of profiles
Profiles15 records
Solid Biosciences funding detail
Funding detailFunding overview
Funding rounds11 records
Investors28 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Solid Biosciences M&A and investment
M&A and investmentM&A1 record
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Solid Biosciences
What does Solid Biosciences do?
Solid Biosciences is a clinical-stage precision genetic medicine company developing AAV gene therapy candidates for rare neuromuscular and cardiac diseases, including Duchenne muscular dystrophy (SGT-003), Friedreich's ataxia (SGT-212), and CPVT (SGT-501). The company leverages its proprietary POLARIS-101 (AAV-SLB101) next-generation capsid platform to deliver therapeutic genes and out-licenses the capsid technology to other gene therapy developers.
Is Solid Biosciences a public or private company?
Solid Biosciences is a public company. It is classified as public and is currently operating.
When was Solid Biosciences founded?
Solid Biosciences was founded in 2013. It employs 101 to 250 people.
Where is Solid Biosciences based?
Solid Biosciences is headquartered in Charlestown, United States, in the North America region.
How does Solid Biosciences make money?
Two revenue lines are on record. Gene Therapy Product Sales are the primary driver. The others are licensing and Technology Access.
Who are Solid Biosciences's main competitors?
Direct peers on record are Sarepta Therapeutics, Regenxbio, uniQure, Krystal Biotech, Lexeo Therapeutics, Tenaya Therapeutics, 4D Molecular Therapeutics and Rocket Pharmaceuticals. Broad incumbents are Pfizer and BioMarin Pharmaceutical.
Does Solid Biosciences have an API?
No public API is recorded for Solid Biosciences.
What industry is Solid Biosciences in?
Solid Biosciences's product category is Gene Therapy Biologics. Its primary akta.pro industry code is HLAAACAA, AAV Vector Gene Therapy Developers, with a secondary code of HLAAACAE, Gene Therapy for Rare/Monogenic Diseases. Its NAICS code is 541714 and its SIC code is 2836.