The FDA has granted fast track designation to Solid Biosciences’s gene therapy SGT-003 for the treatment of DMD.

The US-based company received clearance from the FDA to start Phase I/II trial for the gene therapy on 14 November. SGT-003 uses a novel capsid AAV-SLB101 to deliver the DNA sequence encoding the shortened form of the dystrophin protein (microdystrophin). It also has R16 and R17 nNOS binding protein domains.

DMD is a rare genetic condition that causes progressive muscle weakness. It affects approximately six in 100,000 individuals across Europe and North America, as per the US Muscular Dystrophy Association.

The first gene therapy for treating DMD was approved earlier this year. In June, Sarepta Therapeutics’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) received accelerated approval by the FDA for treating ambulatory patients aged 4-5 years with a confirmed mutation in the DMD gene.

There has been an increased interest in developing gene therapies in recent years. Multiple gene therapies have been approved for various indications such as sickle cell anaemia. In October, Japan-based Kyowa Kirin signed a definitive agreement to acquire Orchard Therapeutics.

Orchard’s catalogue includes Libmeldy (atidarsagene autotemcel), which has been approved to treat the rare genetic metabolic disorder metachromatic leukodystrophy (MLD) in the UK and Europe. The therapy was granted the rare paediatric disease designation by the FDA, with the Prescription Drug User Fee Act (PDUFA) action date set for 18 March 2024.

AstraZeneca has also made significant investments in developing gene therapies for various indications. Last month, the company partnered with Cellectis to develop up to ten cell and gene therapy candidates. The company also acquired Pfizer’s early-stage gene therapy portfolio for rare diseases in September.

The open-label Phase I/II trial for Solid’s SGT-003 will evaluate the therapy in paediatric patients. SGT-003 will be administered as a one-time intravenous infusion at a dose of 1E14vg/kg to two cohorts of patients, as per a 7 December press release. The two cohorts would consist of three patients each, with a potential for cohort expansion. The first cohort will enrol DMD patients aged 4-6 years. The long-term safety and efficacy of the therapy will be evaluated over five years.

Other therapies in Solid’s pipeline include SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and AVB-202-TT for the treatment of Friedreich’s ataxia.

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