PTC Therapeutics, Inc., reported the discontinuation of preclinical and early research programs in gene therapy as part of a strategic portfolio prioritization. PTC will continue its development and global commercialization of Upstaza, the first-ever approved gene therapy directly administered to the brain. As a result of the prioritization, estimated reductions of approximately fifteen percent in residual 2023 operating expenses (OPEX) are expected. Additional 2023 OPEX guidance will be provided as part of second quarter earnings.

The decision to deprioritize early-stage gene therapy programs reflects the company’s desire to focus resources on areas of the pipeline likely to deliver significant return on investment and transformative therapies for patients with high unmet medical need. The discontinuation of the gene therapy programs will also result in a reduction in workforce which will be conducted in accordance with appropriate labor regulations.

The discontinued gene therapy programs include preclinical stage programs in Friedreich ataxia and Angelman syndrome as well as several other programs targeting rare CNS and ophthalmological disorders of high unmet medical need at various stages of preclinical development.

In addition, the prioritization decision will allow for additional focus on PTC’s proprietary splicing platform as well as additional CNS and metabolic disorders that leverage its differentiated and innovative scientific expertise.

“We are incredibly proud of PTC’s accomplishments in the field of gene therapy, including the pioneering work associated with the approval of Upstaza,” said CEO Matthew Klein. “We believe that the decision to discontinue our pipeline gene therapy programs enables PTC to focus R&D efforts on our other innovative and differentiated scientific platforms and strongly positions us for long-term growth and success. Where possible, we will work to ensure that the discontinued gene therapy programs can be developed by other parties so that the therapies have the potential to benefit patients.”