The company is currently working towards obtaining approvals from institutional review boards (IRB) at the designated clinical trial sites for the anticipated Phase 1/2 clinical trial of SGT-003
Solid Biosciences gets FDA ODD for Duchenne gene therapy candidate SGT-003. (Credit: Arek Socha from Pixabay)
Solid Biosciences has secured orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for SGT-003, the company’s advanced gene therapy candidate for Duchenne muscular dystrophy (Duchenne).
Solid Biosciences is a life sciences firm specialising in the creation of precision genetic treatments for neuromuscular and cardiac disorders, including SGT-003, for the treatment of Duchenne.
Duchenne is a genetic muscle-wasting disorder primarily impacting boys, typically manifesting symptoms between the ages of three and five. This progressive, irreversible, and ultimately fatal condition occurs in approximately one in every 3,500 to 5,000 live male births, with an estimated prevalence of 5,000 to 15,000 cases in the US alone.
Solid Biosciences president and CEO Bo Cumbo said: “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.
“These designations are important milestones for Solid, supporting the continued development of next-generation therapies for Duchenne.”
Currently, the company is actively working towards obtaining approvals from institutional review boards (IRB) at the designated clinical trial sites for the anticipated Phase 1/2 clinical trial of SGT-003. Following this, patient screening is anticipated to commence shortly.
The initiation of patient dosing in the trial is projected to take place in the mid-to-late first quarter of 2024.
Solid Biosciences chief medical officer Gabriel Brooks said: “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.
“Obtaining ODD status is an exciting development that we believe will aid our efforts to bring advanced treatment options to those patients affected by Duchenne.”
SGT-003 uses a proprietary, carefully designed capsid (AAV-SLB101) to administer a DNA sequence encoding a truncated version of the dystrophin protein (microdystrophin), featuring the R16-R17 nNOS binding domain.
Preclinical data indicates the potential significance of this domain for both muscular function and sustained benefits in patients.