RARE Daily

Despite Pandemic Disruption, Orchard Doses First Patient with Gene Therapy for MPS IIIA

April 28, 2020

Despite the global COVID-19 pandemic’s causing delayed starts of planned clinical trials, slowed patient enrollment in ongoing trials, and ongoing trials put on hold, Orchard Therapeutics reported dosing its first patient with an experimental gene therapy to treat mucopolysaccharidosis type IIIA.

Mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome type A, is a rare, inherited, and life-threatening neurometabolic disorder caused by genetic mutations in the SGSH gene that lead to the buildup of sugar molecules called mucopolysaccharides in the body, resulting in progressive intellectual disability and loss of motor function. Children born with MPS-IIIA rarely live past adolescence or early adulthood, and no approved therapies currently exist to treat the disease.

Orchard Therapeutics’ experimental gene therapy OTL-201 is an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of MPS-IIIA. The study is an open-label, proof-of-concept trial designed to evaluate safety, tolerability and clinical efficacy, and is intended to enroll up to five patients between three and 24 months of age who will be followed for three years. The study also contains a number of key secondary outcome measures such as overall survival, cognition, and behavior to help inform future clinical development of HSC gene therapy for this indication.

“I am very encouraged that we, together with our research and clinical collaborators in Manchester, could achieve this important milestone in our efforts to develop a gene therapy for MPS-IIIA despite the current, challenging global health circumstances,” said Bobby Gaspar, CEO of Orchard.

Orchard’s news is encouraging in light of the coronavirus pandemic’s ongoing effects on clinical trials around the world. The company had reported expected delays in enrollment of several of its gene therapy candidates, including OTL-201, and regulatory and clinical disruptions.

A compendium put together by BioPharmaDive, which is collecting news of biopharmaceutical companies’ responses to the pandemic, shows that half of affected trials are in the early stage, and 17.4 percent (36) are studies of experimental treatments for rare diseases. Of the 77 companies in its list as of April 22, 42 are small biotechs reporting difficulties including delaying trials, halting enrollment in ongoing trials, or reporting that data readouts will be significantly delayed due to the pandemic.

Photo: Bobby Gaspar, CEO of Orchard

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