Solid Biosciences

Solid Biosciences helps people with rare heart and muscle diseases. It makes gene therapies that put healthy genes into the body. These medicines aim to stop or slow down serious genetic conditions.

What is Solid Developing?

• Duchenne Muscular Dystrophy: SGT-003 delivers a functional version of the dystrophin gene to muscles to preserve strength; currently in mid-stage clinical testing.
• Friedreich's Ataxia: SGT-212 uses a dual-delivery method to target the heart and brain; designed to restore essential frataxin proteins.
• Heart Rhythm Disorders: SGT-501 aims to stop life-threatening irregular heartbeats by helping the body regulate calcium flow in the heart.
• Cardiomyopathy Programs: Early-stage candidates like SGT-601 focus on repairing heart muscle function to prevent heart failure in patients with specific genetic mutations.

Strategic Deals and Partnerships

• Purchased assets from FA212 LLC in September 2024 to add a gene therapy for Friedreich's ataxia to the clinical pipeline.
• Partnered with the Mayo Foundation for Medical Education and Research in December 2024 to advance research for six new heart-related programs.
• Licensed heart-focused research from ICS Maugeri S.p.A. SB in June 2023 to expand development of treatments for rare cardiac conditions.
• Maintained a long-term collaboration with Ultragenyx Pharmaceutical Inc. to develop and sell new gene therapies for patients with Duchenne.

Proprietary Tech and Precision Delivery

The company uses its own delivery shells, called capsids, which are engineered to find and enter muscle and heart tissue more effectively. It also develops dual gene expression technology that allows it to pack multiple healthy genes into a single treatment to address complex diseases.

The Cost of Clinical Progress

• Operating loss reached $49.4 million in Q4 2025, deepening by 23.8% compared to the previous year.
• Net loss grew to $49.8 million in Q4 2025, a 16.9% deepening from Q4 2024.
• Free cash flow hit negative $54.1 million in Q4 2025, which deepened by 81.3% from the prior year.
• Market capitalization rose to $534.3 million in Q4 2025, up 149.3% from $214.3 million in Q4 2024.

Solid's Recent Evolution

Solid has transitioned from a single-disease focus to a developer of multiple genetic medicines for muscle and heart conditions. By combining its proprietary delivery technology with strategic medical partnerships, it has moved several experimental therapies into human testing.