A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study. Cohort 1 will include participants 4 to <7 years of age. Cohort 2 will include participants 7 to <12 years of age. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

Start Date06 May 2024

Sponsor / Collaborator

Solid Biosciences, Inc.

NCT05657938 / CompletedNot Applicable

A Phase 0 Study to Evaluate the Clinical Readiness of a Wearable Sensor Device and Duchenne Video Assessment in the Home Environment on Participants With Duchenne Muscular Dystrophy (DMD)

This study is designed to evaluate the feasibility, wearability and participant satisfaction of novel outcome assessment tools in DMD patients which are performed in the home environment.

Start Date13 Oct 2022

Sponsor / Collaborator

Solid Biosciences, Inc.

NCT03680365 / CompletedNot Applicable

Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of DMD on the Lives of Families

The purpose of this study is to improve the understanding of the treatment goals that a person with Duchenne Muscular Dystrophy (DMD) or the caregiver may be most interested in, based on the severity of the person's disease. Data will be collected by online survey when the participant accepts the study invitation ("RSVP questionnaire") and telephone interview on the functional burden and self-identified treatment goals from the perspective of people with DMD and their caregivers. Interviews will be analyzed to help identify things important to Duchenne families to measure in clinical trials and to inform the selection of key concepts of interest and development of future clinical outcome measures, including observer reported outcomes/patient reported outcomes. The study will be conducted in the United States and will enroll between 45 and 120 participants 11 years or older living with DMD as well as their caregivers. The time commitment for the online survey and the telephone interview is about one hour. It is anticipated that the entire study will be completed within one year.

Start Date20 Sep 2018

Sponsor / Collaborator

Pfizer Inc.

[+11]

100 Clinical Results associated with Solid Biosciences, Inc.

0 Patents (Medical) associated with Solid Biosciences, Inc.

Literatures (Medical) associated with Solid Biosciences, Inc.

03 Sep 2024·Journal of Neuromuscular Diseases

Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy

Article

Author: McNiff, Megan ; Beaverson, Katherine L. ; Bateman-House, Alison ; Denger, Brian ; Camino, Eric ; Heslop, Emma ; Johnson, Alexandra ; Woollacott, Ione O.C. ; Donisa-Dreghici, Roxana ; Mansfield, Carol ; Fischer, Ryan ; Ganot, Annie ; Phillips, Dawn ; Thakrar, Sejal ; Armstrong, Niki ; Peay, Holly ; Cope, Heidi ; Fernandez, Vivian

Background: Duchenne muscular dystrophy (DMD) is a progressive, life-limiting, neuromuscular disorder. Clinicians play an important role in informing families about therapy options, including approved gene therapies and clinical trials of unapproved therapies. Objective: This study aimed to understand the perspectives of clinicians about gene therapy for DMD, which has not previously been studied. Methods: We conducted interviews with specialist clinicians treating patients with DMD in the United States (n = 8) and United Kingdom (n = 8). Interviews were completed in 2022, before any approved gene therapies, to gain insight into barriers and facilitators to implementing gene therapy and educational needs of clinicians. Results: Most respondents expressed cautious optimism about gene therapy. Responses varied regarding potential benefits with most expecting delayed progression and duration of benefit (1 year to lifelong). Concern about anticipated risks also varied; types of anticipated risks included immunological reactions, liver toxicity, and cardiac or renal dysfunction. Clinicians generally, but not uniformly, understood that gene therapy for DMD would not be curative. Most reported needing demonstrable clinical benefit to justify treatment-related risks. Conclusions: Our data demonstrate variability in knowledge and attitudes about gene therapy among clinicians who follow patients with DMD. As our knowledge base about DMD gene therapy grows, clinician education is vital to ensuring that accurate information is communicated to patients and families.

01 Aug 2023·Rheumatology

A novel estrogen receptor 1: sphingomyelin phosphodiesterase acid-like 3B pathway mediates rituximab response in myositis patients

Article

Author: Boehler, Jessica F ; Nagaraju, Kanneboyina ; Parkes, Joanna E ; Rider, Lisa G ; Miller, Frederick W ; Hoffman, Eric P ; Li, Ning ; Kendra, Ryan M ; O’Hanlon, Terrance P ; Peterson, Jennifer M

AbstractObjectivesThe B-cell depleting biologic, rituximab, is used to treat refractory autoimmune myositis. However, the beneficial effects of rituximab appear to outweigh the known contribution of B cells in myositis. We aimed to elucidate how myositis patients respond differently to rituximab and possible alternative mechanisms of action.MethodsHere we have: (i) comprehensively investigated concurrent mRNA and microRNA expression in muscle biopsies taken at baseline and 16 weeks post treatment in 10 patients who were part of the rituximab in myositis (RIM) trial; and (ii) investigated the beneficial effect of rituximab on myositis muscle cells.ResultsOur analyses identified an increased number of changes in gene expression in biopsies from patients who had a clinical response to rituximab (n = 5) compared with non-responders (n = 5). The two groups had completely different changes in microRNA and mRNA expression following rituximab therapy, with the exception of one mRNA, BHMT2. Networks of mRNA and microRNA with opposite direction of expression changes highlighted ESR1 as upregulated in responders. We confirmed ESR1 upregulation upon rituximab treatment of immortalized myotubes and primary human dermatomyositis muscle cells in vitro, demonstrating a direct effect of rituximab on muscle cells. Notably, despite showing a response to rituximab, human dermatomyositis primary muscle cells did not express the rituximab target, CD20. However, these cells expressed a possible alternative target of rituximab, sphingomyelinase-like phosphodiesterase 3 b (SMPDL3B).ConclusionIn addition to B-cell depletion, rituximab may be beneficial in myositis due to increased ESR1 signalling mediated by rituximab binding to SMPDL3B on skeletal muscle cells.

01 May 2023·Human Gene Therapy

Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials

Article

Author: Braun, Serge ; Duan, Dongsheng ; Ganot, Annie ; Morris, Carl ; Wagner, Kathryn R. ; Hunter, Nina ; Gonzalez-Alegre, Pedro ; Ziolkowski, Olivia ; Chamberlain, Jeffrey S. ; Brown, Kristy J. ; Danos, Olivier ; McDonald, Craig ; Tobolowsky, Mark ; Robb, Melissa

Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It is caused by mutations in the DMD gene and is characterized by progressive muscle degeneration that results in loss of function and early death due to respiratory and/or cardiac failure. Although limited treatment options are available, some for only small subsets of the patient population, DMD remains a disease with large unmet medical needs. The adeno-associated virus (AAV) vector is the leading gene delivery system for addressing genetic neuromuscular diseases. Since the gene encoding the full-length dystrophin protein exceeds the packaging capacity of a single AAV vector, gene replacement therapy based on AAV-delivery of shortened, yet, functional microdystrophin genes has emerged as a promising treatment. This article seeks to explain the rationale for use of the accelerated approval pathway to advance AAV microdystrophin gene therapy for DMD. Specifically, we provide support for the use of microdystrophin expression as a surrogate endpoint that could be used in clinical trials to support accelerated approval.

News (Medical) associated with Solid Biosciences, Inc.

23 Sep 2024

·www.globenewswire.com

Solid Biosciences to Participate at Chardan’s 8th Annual Genetic Medicines Conference

CHARLESTOWN, Mass., Sept. 23, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will participate in a fireside chat at Chardan’s 8th Annual Genetic Medicines Conference on Monday, September 30, 2024, at 11:00 am ET. A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for 30 days on the Events page. Institutional investors interested in meeting with management during the conferences may reach out to their Chardan representatives. About Solid BiosciencesSolid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com. Solid Biosciences Investor Contact:Nicole AndersonDirector, Investor Relations and Corporate CommunicationsSolid Biosciences Inc.investors@solidbio.com Media Contact:Glenn SilverFINN Partnersglenn.silver@finnpartners.com

Gene Therapy

12 Sep 2024

·www.globenewswire.com

Solid Biosciences to Participate at the 2024 Cantor Global Healthcare Conference

CHARLESTOWN, Mass., Sept. 12, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will participate in a fireside chat at the 2024 Cantor Global Healthcare Conference on Wednesday, September 18, 2024, at 10:20 am ET. A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for 30 days on the Events page. Institutional investors interested in meeting with management during the conferences may reach out to their Cantor Fitzgerald representatives. About Solid BiosciencesSolid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com. Solid Biosciences Investor Contact:Nicole AndersonDirector, Investor Relations and Corporate CommunicationsSolid Biosciences Inc.investors@solidbio.com Media Contact:Glenn SilverFINN Partnersglenn.silver@finnpartners.com

Gene Therapy

04 Sep 2024

·www.globenewswire.com

Solid Biosciences to Participate at Upcoming Investor Conferences

CHARLESTOWN, Mass., Sept. 04, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced participation in the following investor conferences: H.C. Wainwright 26th Annual Global investment Conference – New York, NYBo Cumbo, President and CEO, will participate in a fireside chat on Tuesday, September 10, 2024, at 9:30 am ET. Baird 2024 Global Healthcare Conference – New York, NYMr. Cumbo will present on Wednesday, September 11, 2024, at 12:50 pm ET. A live webcast of the H.C. Wainwright fireside chat will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for 30 days on the Events page. Institutional investors interested in meeting with management during the conferences may reach out to their H.C. Wainwright or Baird representatives. About Solid BiosciencesSolid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com. Solid Biosciences Investor Contact:Nicole AndersonDirector, Investor Relations and Corporate CommunicationsSolid Biosciences Inc.investors@solidbio.com Media Contact:Glenn SilverFINN Partnersglenn.silver@finnpartners.com

Gene Therapy

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100 Translational Medicine associated with Solid Biosciences, Inc.

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Pipeline Snapshot as of 07 Nov 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

SGT-003 ( micro-dystrophin )	Muscular Dystrophy, Duchenne More	Phase 2 Clinical
SGT-001 ( micro-dystrophin )	Muscular Dystrophy, Duchenne More	Phase 2 Clinical
UX-810 ( dystrophin )	Muscular Dystrophy, Duchenne More	Preclinical
AVB-501	Cardiomyopathy, Dilated More	Preclinical
AVB-401 ( BAG3 )	BAG3 mediated Dilated Cardiomyopathy More	Preclinical

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