Cell therapy weekly: Novel nanoparticles for delivery of genetic medicines
This week: GeneEdit (CA, USA) and Genetech (CA, USA), a member of the Roche Group (Basel, Switzerland), announced they will collaborate to develop novel nanoparticles to deliver genetic medicines for autoimmune diseases, Kyowa Kirin (Tokyo, Japan) acquired Orchard Therapeutics (London, UK) and ReLive Biotechnologies’ (Nanjing, China) cartilage repair product received approval from Singapore’s Health Sciences Authority.
The news highlights:
- Novel nanoparticles for delivery of genetic medicines
- Kyowa Kirin expands gene therapy portfolio with Orchard Therapeutics acquisition
- Approval from Singapore Health Authority for cartilage repair cell therapy
Novel nanoparticles for delivery of genetic medicines
GeneEdit and Genetech, have entered into a collaboration and license agreement, which will utilize GeneEdit’s polymer nanoparticle library, NanaGalaxy, to develop nanoparticles that can deliver Genentech’s nucleic acid-based medicines for autoimmune disease treatment.
GenEdit will receive a US$15 million upfront payment and stands to earn up to US$629 million in milestone payments along with tiered royalties on global net sales for resulting products. Genentech will handle preclinical, clinical, regulatory development and commercialization of products employing GenEdit’s nanoparticles.
“We are excited to work with Genentech to develop novel therapeutics for autoimmune disease,” said Kunwoo Lee, CEO of GenEdit. “Genentech is a leader in advancing breakthrough science to advance the development of novel therapeutic modalities. This collaboration underscores the promise of GenEdit’s NanoGalaxy platform to deliver nucleic acid-based medicines via our hydrophilic nanoparticle technology.”
Kyowa Kirin expands gene therapy portfolio with Orchard Therapeutics acquisition
Kyowa Kirin has acquired Orchard Therapeutics, a company developing and commercializing hematopoietic stem cell gene therapies, making it a wholly owned subsidiary. Orchard’s portfolio includes Libmeldy®, a gene therapy treatment for metachromatic leukodystrophy, which has been approved by the European Commission and UK Medicines and Healthcare products Regulatory Agency. Orchard is also advancing clinical programs for mucopolysaccharidosis type I Hurler’s syndrome and mucopolysaccharidosis type IIIA using the same hematopoietic stem cell gene therapy platform.
“We are truly excited about the acquisition of Orchard Therapeutics, a leading provider of HSC gene therapy. This platform offers significant potential to deliver more innovative treatments and breakthrough therapies and aligns with our purpose to deliver life-changing value for people living with rare and complex diseases,” said Masashi Miyamoto, Representative Director, President and CEO of Kyowa Kirin. “Going forward, our companies will build on the extensive experience of Orchard’s gene therapy platform and apply it to under-served indications and diseases where we believe it to be scientifically and clinically differentiated.”
Approval from Singapore Health Authority for cartilage repair cell therapy
Singapore’s Health Sciences Authority has approved a New Drug Application for ReLive Biotechnologies’ product, SpheChon. SpheChon is a chondrocyte spheroid-based cell therapy for cartilage repair. This New Drug Application approval covers the product’s use for the repair of symptomatic articular cartilage defects of the femoral condyle and the patella of the knee.
Xing Zhao, CEO of ReLive stated: “The approval in Singapore is more than just a regulatory milestone. It signifies ReLive’s growing influence and commitment to addressing unmet needs in the areas of orthopedic sports medicine and plastic and reconstructive surgery in Asia. This achievement aligns with our strategic vision of expanding our footprint in key markets and underscores our commitment to transforming patient care on a global scale.”
