Solid Biosciences Inc. announced an update to its strategic priorities to focus on developing SGT-001 and SGT-003. SGT-001 Update: On March 14, 2022, Solid released data demonstrating that patients showed sustained benefit compared with natural history trajectories two years after treatment with SGT-001 across functional, pulmonary and patient reported outcome measures. Manufacturing Update: As the company focuses on advancing SGT-001, it will be streamlining operations and making the strategic shift to a commercially scaled transient transfection-based manufacturing process.

Following a robust manufacturing analysis, Solid believes that a new, outsourced process may provide improvements to manufacturability as well as additional organizational efficiencies. Clinical Update: The company also announced that it has concluded enrollment in IGNITE DMD, its Phase 1/2 clinical trial for SGT-001, and will continue monitoring dosed patients for five years post-treatment. The company anticipates that future patients will be treated with SGT-001 manufactured using the new transient transfection-based process.

Solid currently expects to continue dosing with SGT-001 in 2023. SGT-003 Update: On April 27, 2022, Solid released new preclinical data suggesting that a novel, next generation capsid candidate may have meaningful advantages for the delivery of muscle-related gene therapies. New data from a non-human primate study using a reporter transgene in Solid's novel capsid demonstrated increased muscle tropism, decreased liver biodistribution and improved efficiency compared with AAV9.

These results are consistent with earlier in vitro and in vivo studies in both dystrophic (MDX) and wild type mouse models, which suggested improved muscle tropism with Solid's novel capsid as well as improved expression of Solid's microdystrophin compared with AAV9. Solid's novel, muscle tropic capsid has been combined with the company's differentiated microdystrophin for the SGT-003 program for Duchenne. The company remains on track for an early 2023 IND submission for SGT-003.

The company also announced a reorganization of its corporate operations to prioritize the advancement of its key programs, SGT-001 and SGT-003. Solid anticipates that the use of transfection-based manufacturing processes for both SGT-001 and SGT-003 will allow the company to focus its operating structure and better leverage external manufacturing expertise. In addition, the company plans to narrow Research & Development activities to those related to SGT-001, SGT-003 and next generation capsids.

In connection with these activities, the company will reduce its headcount by approximately 35%. The reorganization will result in a reduction in planned corporate expenditures which is anticipated to extend funding of operations through important clinical milestones and into the second quarter of 2024.