RARE Daily

Orchard Therapeutics Cuts Staff by 25 Percent, Narrows Rare Disease Focus, and Shuts California Facility

May 8, 2020

Rare Daily Staff

Gene therapy developer Orchard Therapeutics said it would cut its staff by 25 percent, narrow its rare disease focus, shut its California facility, and discontinue construction of its gene therapy manufacturing facility in Fremont, California in an effort to save $125 million and extend its runway into 2022.

The company said its strategy is designed to realize the potential of its hematopoietic stem cell (HSC) gene therapy technology by advancing high-value programs, strengthening its financial position, and building a commercial infrastructure.

“Moving forward, we are focusing on advancing therapies for high need and high value diseases, and our work in neurometabolic disorders is a clear example of this,” said Bobby Gaspar, CEO of Orchard. “We’re also excited to announce new research programs which we believe will demonstrate the breadth of the HSC platform approach.”

As part of the strategic plan, the company said it is prioritizing the development of OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-203 for MPS-I, and OTL 201 for MPS-IIIA. It is reducing investment in OTL-101 for ADA-SCID and OTL-300 for transfusion-dependent beta-thalassemia. 

The company also said it would accelerate research into genetic subsets of indications outside of rare disease including programs in frontotemporal dementia and Crohn’s disease.

The company also said the U.S. Food and Drug Administration recently provided written feedback on the sufficiency of the company’s data package, including the clinical endpoints, natural history analysis, and chemistry and manufacturing and controls (CMC) data for its U.S. biologics license application submission of OTL-200 for the treatment of metachromatic leukodystrophy. Orchard intends to use FDA’s guidance to further analyze the latest available CMC and clinical data, file an investigational new drug application, and seek Regenerative Medicine Advanced Therapy designation in 2020.

The company believes this will facilitate a more comprehensive dialogue with the FDA to resolve open matters before the intended BLA submission.

“Our new strategic plan positions Orchard to execute its mission and objectives at the highest level by matching our attention and resources to a set of core imperatives for the business,” said Frank Thomas, president and chief operating officer. “I believe that these are necessary steps, especially in light of the current environment in which we are operating, with focused investments in areas such as commercial and manufacturing operations supporting the needs we have now without a near-term dependence on the capital markets.”

Photo: Bobby Gaspar, CEO of Orchard Therapeutics

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