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Solid Biosciences develops precision genetic medicines for rare neuromuscular and cardiac diseases. The company employs gene therapy candidates to address underlying causes of Duchenne Muscular Dystrophy, Friedreich's Ataxia, and CPVT. Their technical approach centers on advancing a pipeline of programs engineered for durable therapeutic benefits.
Founded in March 2013 by Ilan Ganot, Andrey J. Zarur, Matthew Bennett Arnold, Annie Ganot, and Gilad David Hayeem, Solid Biosciences' inception arose from a profound personal connection. This insight, rooted in direct experiences with Duchenne Muscular Dystrophy, drives a mission to improve daily lives for patients with rare diseases.
Solid Biosciences' therapies target patients with rare neuromuscular and cardiac disorders, primarily Duchenne Muscular Dystrophy. The company's vision is to deliver transformative genetic medicines, addressing critical unmet needs within these populations. They strive to enhance the long-term well-being and daily lives of affected individuals.
Solid Biosciences has raised $110.0M across 2 funding rounds.
Solid Biosciences has raised $110.0M in total across 2 funding rounds.
Solid Biosciences has raised $110.0M in total across 2 funding rounds.
Solid Biosciences's investors include Adam Koppel, Rajeev Shah, Biogen, Cormorant Asset Management, Foresite Capital, Janus Henderson Investors, Leerink Partners, Perceptive Advisors, RTW Investments.
Solid Biosciences has raised $110.0M across 2 funding rounds. Most recently, it raised $60.0M Other Equity in July 2019.
| Date | Round | Lead Investors | Other Investors | Status |
|---|---|---|---|---|
| Jul 26, 2019 | $60M Venture Round | — | — | Announced |
| Mar 30, 2017 | $50M Series C | Adam Koppel, Rajeev Shah | Biogen, Cormorant Asset Management, Foresite Capital, Janus Henderson Investors, Leerink Partners, Perceptive Advisors, RTW Investments | Announced |
Solid Biosciences is a precision genetic medicine company developing gene therapies for rare neuromuscular and cardiac diseases, with a primary focus on Duchenne muscular dystrophy (Duchenne).[1][2] Its mission is to improve the daily lives of patients living with devastating rare diseases, particularly by advancing therapies that address the underlying genetic causes, such as delivering functional genes via AAV-based vectors.[1][2][6] The company serves patients, families, and caregivers affected by conditions like Duchenne, Friedreich’s ataxia (FA), and catecholaminergic polymorphic ventricular tachycardia (CPVT), solving unmet needs in treating fatal genetic disorders through a diversified pipeline including SGT-003, SGT-212, and SGT-501, alongside next-generation capsids and genetic regulators.[1][2][7] Growth momentum includes ongoing clinical trials (e.g., Phase 3 for SGT-003), recent U.S. Department of Health and Human Services recognition of Duchenne for newborn screening, and active partnerships to accelerate innovation.[2][5][7]
Solid Biosciences was co-founded by individuals directly impacted by Duchenne muscular dystrophy, embedding patient perspectives into its DNA from inception.[2][3][6] This personal connection drove the company's formation as a patient-focused entity dedicated to advancing genetic medicines for neuromuscular and cardiac diseases, evolving from a Duchenne-centric mission to a broader pipeline targeting rare conditions like FA and CPVT.[1][2] Early traction stemmed from developing SGT-001, an AAV-based gene therapy for Duchenne, which progressed to clinical trials, alongside pivotal community engagement that positioned Solid as a center of excellence in rare disease science.[1][3] The company's growth reflects a commitment to rigorous science, collaborations, and putting patient needs at the forefront of research and development.[3][5]
Solid Biosciences rides the wave of precision genetic medicine, capitalizing on advances in AAV gene therapies to target monogenic rare diseases where traditional treatments fall short.[1][2] Timing is critical amid rising momentum in neuromuscular and cardiac gene therapy, bolstered by regulatory nods like Duchenne's addition to the U.S. newborn screening panel, which could expand early intervention and market access.[2] Market forces favoring Solid include growing investment in rare disease innovation, unmet needs in underserved patient populations, and cross-industry demand for its delivery technologies.[1][5] By prioritizing patient engagement and collaborations, Solid influences the ecosystem as a hub for expertise, potentially setting standards for community-driven biotech and enabling broader gene therapy breakthroughs.[3][5]
Solid Biosciences is poised to advance its pipeline with key milestones like Phase 3 data for SGT-003 and Phase 1/2 initiations for cardiac programs, potentially yielding approvals that transform Duchenne and rare disease care.[1][2][7] Trends like refined viral vectors, newborn screening expansions, and AI-enabled genetic regulators will shape its trajectory, amplifying delivery efficiency and trial success.[2][7] Its influence may evolve from niche innovator to ecosystem leader through partnerships, driving a future where genetic medicines meaningfully extend lives for rare disease patients—fulfilling its patient-rooted mission.[3][5]
