JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present preclinical data from its proprietary J-Brain Cargo^®-applied adeno-associated virus (AAV) gene therapy preclinical research programs in a poster session at the European Society of Gene and Cell Therapy (ESGCT) 31^st Annual Congress, being held October 22-25, 2024, in Rome, Italy.

The poster presentation details are listed below, and the full program can be found on the ESGCT congress website at https://www.esgctcongress.com/.

Title: Incorporation of transferrin receptor binder into AAV enables efficient brain delivery for treatment of genetic CNS diseases

Abstract Number: P0133

Date: October 22, 2024, 7:30-9:00pm CEST

Presenter: Yuhei Ashida, Associate Senior Scientist, at JCR Pharmaceuticals

About the European Society of Gene and Cell Therapy (ESGCT)

Established in 1992, the European Society of Gene and Cell Therapy (ESGCT) seeks to support scientists and clinicians working in the fields of gene and cell therapy and to promote awareness and understanding of gene and cell therapy and the vast amount of related research in Europe. For more information, please visit https://www.esgct.eu/.

About the J-Brain Cargo^® Platform Technology

JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo^®, to bring biotherapeutics into the central nervous system (CNS). The first drug developed based on this technology is IZCARGO^® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder.

About JCR Pharmaceuticals Co., Ltd.

JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 49-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. For more information, please visit https://www.jcrpharm.co.jp/en/site/en/.

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