The first gene therapy for treating DMD was approved earlier this year. Image Credit: Natali _ Mis / Shutterstock.

The FDA has granted fast track designation to 鈥檚 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.

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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, 鈥檚 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 signed a definitive agreement to acquire Orchard Therapeutics.

Orchard鈥檚 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 by the FDA, with the Prescription Drug User Fee Act (PDUFA) action date set for 18 March 2024.

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

The open-label Phase I/II trial for Solid鈥檚 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鈥檚 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鈥檚 ataxia.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 

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