Orchard Therapeutics Restructures, Cuts Rare Disease Programs to Extend Runway into 2024

Rare Daily Staff

Gene therapy biotech Orchard Therapeutics said it will discontinue its rare primary immune deficiency programs, including about 30 percent of its workforce, to focus its hematopoietic stem cell gene therapy platform exclusively on severe neurometabolic diseases and early research programs.

The discontinued programs include OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and Strimvelis, a gammaretroviral vector-based gene therapy approved in Europe for adenosine deaminase severe combined immunodeficiency (ADA-SCID).

“We recognize the significant need that persists for many patients suffering from these rare diseases of the immune system, and we sympathize with the individuals, families and healthcare providers affected by these announcements, as well as our clinical partners and colleagues who worked so hard to advance these programs,” said Bobby Gaspar, CEO of Orchard Therapeutics. “These therapies have shown the potential for significant benefit for many patients treated in the clinical studies and we will continue to look for alternative ways to advance them, which could include commercial partnerships.”

The restructuring is intended to extend the company’s cash runway into 2024 and focus operations on the highest value programs in its portfolio.

Orchard will continue its investment in Libmeldy (atidarsagene autotemcel) / OTL-200 for metachromatic leukodystrophy (MLD) to help sustain recent commercial momentum in Europe, as well as to support regulatory and future commercial activities for a potential U.S. approval and launch.

The company also will continue to advance clinical development of OTL-203 for mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA). The focus on these neurometabolic programs is expected to allow Orchard to leverage the clinical validation of HSC gene therapy demonstrated with Libmeldy and capture significant commercial synergies, given the unmet needs in these diseases.

Promising early-stage research programs that apply the HSC gene therapy approach in NOD2 Crohn’s disease, hereditary angioedema (HAE) and progranulin mutated frontotemporal dementia (GRN-FTD) also will remain an important part of the portfolio going forward given their promise in larger indications and as a possible source of future partnerships.

“In light of our experiences and knowledge gained in this current and rapidly evolving market environment for gene therapy, our plan is to concentrate resources on programs that have the potential to make a remarkable difference to patients while also providing sustainable value to the business to enable the achievement our long-term vision,” said Bobby Gaspar, CEO of Orchard. “As launch momentum for Libmeldy continues to build in Europe and we prepare for a regulatory filing in the U.S., a focused strategy that utilizes a common infrastructure for future neurometabolic disease launches is critical to our success as a commercial gene therapy company.”

Photo: Bobby Gaspar, CEO of Orchard Therapeutics