Published - May 24nd, 2023 @ 09:45 AM (GMT+2 )

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In a significant strategic move, PTC Therapeutics Inc. (NASDAQ:PTCT) has announced the discontinuation of its preclinical and early research programs in gene therapy. This move is part of a larger strategic portfolio prioritization, with the company concentrating its resources on areas of the pipeline that promise to deliver substantial returns on investment and transformative therapies for patients with high unmet medical needs.

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PTC Therapeutics discontinues preclinical gene therapy programs

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The decision to discontinue the gene therapy programs will result in an expected reduction of approximately 15% in PTC's residual operating expenses (OPEX) for 2023. The company has also indicated that there will be a reduction in the workforce as a result of this decision, which will be implemented in accordance with appropriate labor regulations.

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PTC continues development of gene therapy product, Upstaza

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Despite the discontinuation of these programs, PTC remains committed to the global development and commercialization of Upstaza, the first-ever approved gene therapy directly administered to the brain. This product is a testament to PTC's pioneering accomplishments in gene therapy.

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The discontinued gene therapy programs include preclinical stage programs in Friedreich ataxia and Angelman syndrome and several other programs targeting rare CNS and ophthalmological disorders of high unmet medical need at various stages of preclinical development. While these programs have been discontinued, PTC expressed its intention to work with other parties to ensure that these therapies can still benefit patients in the future.

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As part of this refocus, PTC will concentrate on its proprietary splicing platform and other CNS and metabolic disorders that leverage its differentiated and innovative scientific expertise. Dr. Matthew Klein, CEO of PTC, has expressed confidence in the company's ability to position itself for long-term growth and success by prioritizing its other innovative and differentiated scientific platforms.

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Emily Hill, CFO of PTC, steps down from her position

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In related news, PTC Therapeutics also announced that Emily Hill, the company's Chief Financial Officer, has been relieved of her duties and will leave the organization.

PTC Therapeutics is a science-driven, global biopharmaceutical company that focuses on discovering, developing, and commercializing clinically differentiated medicines that benefit patients with rare disorders. The company's mission is to provide access to best-in-class treatments for patients with little to no treatment options. PTC leverages its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients, thereby maximizing value for all its stakeholders.

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