Solid Biosciences outlines its strategic priorities for 2022 and announces corporate updates

– Continue to advance SGT-001 by administering additional patients in IGNITE DMD –

– Advance the new generation Duchenne gene therapy program (SGT-003) until submission to the IND; SGT-003 demonstrated an increase in muscle tropism and expression of microdystrophin –

– Company enters 2022 with around $ 210 million in cash and investments –

– Ian F. Smith, Chairman of the Board of Directors, will assume the role of Executive Chairman –

CAMBRIDGE, Mass., Jan. 10, 2022 (GLOBE NEWSWIRE) – Solid Biosciences Inc. (Nasdaq: SLDB), a life science company focused on promoting meaningful therapies for Duchenne (Duchenne) muscular dystrophy, made today ‘Today an update on its 2022 strategic priorities and other business initiatives ahead of its presentation at 40e JP Morgan’s annual healthcare conference scheduled for Thursday, Jan.13 at 9 a.m.ET.

“2022 will be an important year for Solid as we seek to advance genetic medicines for Duchenne. We intend to assay additional patients in the IGNITE DMD Phase I / II clinical trial of SGT-001 using our updated risk mitigation strategy and second generation manufacturing process. . During the first half of 2022, we plan to share additional data from IGNITE DMD, including data from patient 9, which was administered in November 2021. We are also moving our next-generation Duchenne program, SGT-003, to an IND submission scheduled for early 2023. Based on preclinical work to date demonstrating improved muscle tropism and microdystrophin expression, we believe that SGT-003 has the potential to provide further benefits. to patients with Duchenne, ”said Ilan Ganot, CEO, President and Co-Founder of Solid Biosciences. “We are entering 2022 with approximately $ 210 million in cash and investment, which we believe will support the continued progress of SGT-001 clinical development activities to enable the completion of IGNITE DMD and discussions with regulatory agencies, as well as the advancement of SGT-003 at the clinic. “

Highlights of the presentation to be given at the JP Morgan Healthcare conference include:

  • SGT-001: Following the implementation of the updated risk mitigation strategy, the Company has dosed a 9e patient in the IGNITE DMD Phase I / II clinical trial for SGT-001 in November 2021 using the company’s second-generation manufacturing process. Strong plans to continue assaying patients in IGNITE DMD in 2022 as well as sharing additional data on expression, functional, pulmonary, and patient-reported results from the trial during the first half of this year.

  • SGT-003: Solid will present additional data on SGT-003 demonstrating increased protein expression and more targeted biodistribution compared to AAV9. The company also intends to launch studies enabling IND in 2022 to support an IND submission in early 2023.

  • Platform technologies: The company will present and present data on the development programs of two platform technologies, Novel Capsids and Dual Gene Expression (DGE). These programs are part of the company’s ongoing research efforts to develop innovative technologies that Solid believes could potentially result in meaningful treatments and spur future expansion of the company’s pipeline.

The company also announces that the chairman of its board of directors, Ian F. Smith, has been appointed executive chairman. Mr. Smith is a highly accomplished life sciences executive with over 20 years of financial and operational management experience in public and private biopharmaceutical companies. The appointment as executive chairman reflects the relationship Mr. Smith has developed with Solid since joining the company’s board of directors in April 2020. As chairman of the board, Mr. Smith has worked closely collaboration with M. Ganot and other members of the management of Solid Executive. Team to advance Solid’s short-term priorities. Formalizing the relationship as Executive Chairman will allow Mr. Smith to provide additional leadership support as the company continues to develop new technologies to support meaningful treatments for patients.

About SGT-001
Solid’s SGT-001 is a novel adeno-associated viral-mediated gene transfer (AAV) therapy designed to treat the underlying genetic cause of Duchenne. Duchenne is caused by mutations in the dystrophin gene which results in the absence or near absence of the dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, into the body. This microdystrophin encodes a functional protein substitute that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s clinical program suggests that SGT-001 has the potential to slow or stop Duchenne’s progression, regardless of genetic mutation or disease stage.

SGT-001 is based on pioneering research in dystrophin biology conducted by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has received Rare Pediatric Disease Designation, or RPDD, and Expedited Designation in the United States and Orphan Drug Designation in the United States and the European Union.

About SGT-003
SGT-003, Solid’s next-generation gene therapy candidate for the treatment of Duchenne, uses an AAV-based vector rationally designed to provide the unique and differentiated microdystrophin construct that is also incorporated into SGT-001. SGT-003 demonstrated improved biodistribution over AAV9 in various in vitro and in vivo models, with increased delivery and expression in skeletal and cardiac muscle and reduced tropism for liver cells. Solid is aiming for an IND filing in early 2023.

About solid biosciences
Solid Biosciences is a life science company focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Disease-driven and founded by a family directly affected by Duchenne, our mandate is simple yet comprehensive – to work to fight the disease at its heart by correcting the underlying mutation that causes Duchenne with our leading gene therapy candidate, SGT- 001, as well as our recently announced next-generation gene therapy candidate, SGT-003. For more information, please visit

Financial information and forward-looking statements
The preliminary financial information presented in this press release is unaudited and based on information currently available, may be adjusted following the completion of the usual quarterly and annual review and audit procedures, not all present information necessary for a full understanding of the financial position as at December 31, 2021 or the results of operations of the company for the year ended December 31, 2021, and the actual financial results of the company may differ materially from the preliminary estimated financial information stated above.

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s ability to continue to assay patients in the IGNITE DMD trial, plans of the Company to present the IGNITE DMD data, the implication of the provisional clinical data, the safety or potential therapeutic benefits of SGT-001 in patients with DMD, the Company’s regulatory plans, the SGT- program 003 of the Company, including the Company’s expectations regarding the filing of an IND, timelines, sufficiency of cash, cash equivalents and available-for-sale securities to fund its operations, and other statements containing the words “anticipate”, “believe”, “continue”, “could”, “estimate”, “expect”, “intend,” “may”, “plan”, “potential”, ” plan ”,“ plan ”,“ should ”, “Target”, “should”, “work” and similar expressions. All forward-looking statements are based on management’s current expectations regarding future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those stated or implied. by these forward-looking statements. These risks and uncertainties include, without limitation, the risks associated with the Company’s ability to pursue IGNITE DMD on time or not at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; obtain and maintain the necessary approvals from the IGNITE DMD Clinical Trial Site Investigation Review Committees and the IGNITE DMD Independent Data Security Oversight Committee; enroll additional patients in IGNITE DMD and on schedule; the Company’s dosage strategy; replicate in clinical trials the positive results found in preclinical studies and early stages of clinical development; whether the provisional data referenced in this release will predict final trial results or demonstrate safe or effective therapeutic benefit from SGT-001; whether the methodologies, assumptions and applications that the Company uses to assess particular safety or efficacy parameters will produce statistically significant results; advance the development of its product candidates within the timeframes it anticipates in current and future clinical trials; successfully optimize and scale its manufacturing process; obtain, maintain or protect the intellectual property rights related to its product candidates; compete successfully with other companies seeking to develop treatments and Duchenne gene therapies; manage expenses; and raise the necessary substantial additional capital, on a timely basis, to further develop SGT-001, SGT-003 and other product candidates, achieve its other business objectives and continue to operate. For a discussion of other risks and uncertainties, and other important factors, each of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as the discussions on potential risks, uncertainties and other important factors, in the most recent documents of the Company with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the views of the Company as of the date hereof and should not be construed as representing the views of the Company as of a date subsequent to the date hereof. The Company expects subsequent events and developments to cause a change in the opinion of the Company. However, although the Company may choose to update these forward-looking statements at some time in the future, the Company specifically disclaims any obligation to do so.

Investor contact:
David Carey
FINN partners
[email protected]

Caitlin lowie
Solid biosciences
[email protected]

Media contact:
Erich Sandoval
FINN partners
[email protected]