Orchard Therapeutics is developing ex vivo autologous hematopoietic stem cell gene therapy for a variety of rare diseases. In this video, Mark Rothera, CEO of the company explains how the regulatory timeline the company has in place for two of its lead therapies – one to treat adenosine deaminase severe combined immunodeficiency (ADA-SCID) and one to treat metachromatic leukodystrophy (MLD).

“For ADA-SCID, we are filing what is called a rolling BLA (biologic licence application) with the FDA in the first half of next year, So we’re initiating the filing and then we’ll gradually complete that and then look forward to getting an approval for ADA-SCID,” noted Rothera, adding ”for MLD we’re filing that with the European Medicines Agency in the first half of next year.”

ADA-SCID (aka bubble boy disease) is a rare, life-threatening, inherited disease of the immune system due to mutations in the ADA gene. Symptoms usually begin during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive. Without treatment, the rare condition can be fatal within the first two years of life.

MLD is a life-threatening inherited disease caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body (liver, gallbladder, kidneys, and/or spleen). Children with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Currently, there are no effective treatments for MLD. Preliminary data from the gene therapy at Orchard Therapeutics is showing great promise for this population. Rothera said, you go from a child with this disease that is unable to move and unable to even do an IQ test to a child that is able to play basketball and be in the normal education system. So it is a remarkable transformation that we’re looking that – subject to getting an approval.”

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