Solid Biosciences Granted FDA Orphan Drug Designation for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
– Patient dosing expected to begin mid-to-late Q1 2024 –
“Obtaining ODD status for SGT-003, along with Fast Track Designation granted last month, furthers our efforts to meet the ongoing challenge of treating this devastating disease as expeditiously as possible,” said
The company is currently in the process of securing approvals from the institutional review boards (IRB) at the clinical trial sites for the planned Phase 1/2 clinical trial of SGT-003 and expects to commence patient screening shortly thereafter. Patient dosing in the trial is expected to commence in mid-to-late first quarter of 2024.
“SGT-003 therapy stands out among other Duchenne gene therapy candidates by leveraging a novel capsid and a muscle tropic vector delivering a microdystrophin that incorporates a neuronal Nitric Oxide Synthase (nNOS) binding domain. These attributes among others, have the potential to yield both more potent transduction than historical approaches, and a microdystrophin that may be able to more fully address muscle resiliency,” said
SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding a shortened form of the dystrophin protein (microdystrophin), containing the R16-R17 nNOS binding domain. Preclinical data suggests this may be important for both muscular function and durability of benefit in patients.
About Orphan Drug Designation
Duchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. Duchenne is a progressive, irreversible, and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 5,000 to 15,000 cases in
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the ability to successfully achieve and execute on the company’s priorities and achieve key clinical milestones; the company’s SGT-003 program, including securing IRB approval at clinical sites, initiating patient screening and initiating dosing, and the potential benefits of SGT-003; the potential benefits of ODD status; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the ability to recognize the anticipated benefits of Solid’s acquisition of AavantiBio; the company’s ability to advance SGT-003, SGT-501, AVB-401, AVB-202-TT and other preclinical programs and capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-501, AVB-401, AVB-202-TT and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any 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 discussions of potential risks, uncertainties and other important factors, in the company’s most recent filings with the
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Source: Solid Biosciences Inc.