Orchard discreetly axes license for 'bubble boy' gene therapy, despite recent positive long-term data update
Less than a month after publishing positive long-term data for an experimental gene therapy, Orchard Therapeutics has quietly terminated its licensing agreement over the program.
In an 8-K filing to the SEC last Friday, Orchard reported that it is ending the license related to its OTL-101 candidate, in development to treat adenosine deaminase severe combined immunodeficiency or ADA-SCID, known colloquially as “bubble boy syndrome.” It’s a rare genetic disease where patients have almost no immune system whatsoever.
The move comes after Orchard published long-term results in the New England Journal of Medicine showing all 50 patients who received the treatment were still alive after at least two years. Event-free survival was also high, with 97% of US patients and 95% of UK participants not needing to return to prior therapies.
Orchard had been developing the therapy after signing the agreement with UCLA and University College London back in February 2016. The biotech has another therapy for ADA-SCID known as Strimvelis, approved in Europe in 2016, but halted dosing after a patient treated under compassionate use began seeing treatment for leukemia.
The idea behind OTL-101 is to use a lentivirus with hematopoietic stem cells, administered ex vivo, to insert a functional copy of the ADA gene into patients’ cells. Orchard had been looking for an approval for a while, having previously won breakthrough therapy designation for OTL-101 from the FDA.
An FDA greenlight had eluded Orchard with the Covid-19 pandemic forcing them to push back plans to submit a rolling approval application. But last week’s SEC report may have been in the works for at least a year.
Back in May 2020, Orchard announced a new operational strategy involving laying off 25% of its staff and restructuring its pipeline to focus less on rare diseases. Part of that restructuring, Orchard said at the time, was to reduce investment in OTL-101 and another program — steering resources instead toward four other candidates classified as “top near-term priorities.”
The programs involved are being set up to treat: metachromatic leukodystrophy (OTL-200), Wiskott-Aldrich syndrome (OTL-103), Mucopolysaccharidosis type I (OTL-203) and Mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A (OTL-201).
Orchard went public in 2018, riding a $200 million IPO after two other nine-figure rounds with a $110 million Series B and $150 million Series B. The biotech worked with GlaxoSmithKline, too, acquiring the Big Pharma’s rare disease unit — including Strimvelis — in exchange for roughly 20% equity, also in 2018. The lack of an FDA-approved product at this stage, however, has left analysts with questions.
Social image: Bobby Gaspar, Orchard Therapeutics CEO