Cell therapy weekly: Kite enters exclusive license agreement with Refuge Biotechnologies for investigational gene expression platform
This week: AVROBIO completes patient dosing in first gene therapy clinical trial for cystinosis, Astellas invests US$50 million to support Taysha Gene Therapies’ AAV-based gene therapy programs and Kite enters exclusive license agreement with Refuge Biotechnologies for investigational gene expression platform.
The news highlights:
AVROBIO completes patient dosing in first gene therapy clinical trial for cystinosis
AVROBIO (MA, USA) has announced the completion of patient dosing in the collaborator-sponsored, Phase I/II clinical trial of an investigational hematopoietic stem cell (HSC) gene therapy to treat cystinosis. Cystinosis is marked by the accumulation of cystine in lysosomes and causes progressive multi-organ damage including early acute kidney disease progressing to end-stage kidney disease. Preliminary data suggest that this gene therapy is well tolerated with no adverse events related to the therapeutic reported to date.
Stephanie Cherqui, lead study investigator and associate professor of pediatrics at the University of California San Diego (USA) stated: “Completing the dosing of the first and only gene therapy trial for cystinosis represents a major milestone for a patient community living with a devastating genetic disease. Unmet medical needs impact the lives of patients and their family members every day. To date, the results from the trial show the potential of this investigational gene therapy to stabilize or reduce the impact of cystinosis on different tissues throughout the body with a one-time dose.”
Astellas invests US$50 million to support Taysha Gene Therapies’ AAV-based gene therapy programs
Astellas (Tokyo, Japan) and Taysha Gene Therapies (TX, USA) have announced a strategic investment to advance Taysha’s adeno-associated virus gene therapy programs for the treatment of Rett syndrome and giant axonal neuropathy. Under the terms of the agreement, Astellas will acquire 15% of the outstanding common stock of Taysha and receive an exclusive option to license two of Taysha’s clinical-stage programs for RET syndrome and giant axonal neuropathy for an investment of US$50 million.
Naoki Okamura, Chief Strategy Officer at Astellas stated: “Taysha is an industry leader in [central nervous system] gene therapies and this partnership fits strategically with our long-term vision of expanding Astellas’ gene therapy capabilities, allowing the company to impact the lives of a broader range of patients with urgent unmet medical needs.”
Kite enters exclusive license agreement with Refuge Biotechnologies for investigational gene expression platform
Kite Pharma (CA, USA), a Gilead company, and Refuge Biotechnologies (CA, USA) have entered an exclusive worldwide license agreement. Kite will receive exclusive rights to utilize Refuge’s proprietary gene expression platform to develop potential treatments for blood cancers. The platform utilizes an expression modulation strategy to repress or activate the transcription of target genes. Early pre-clinical data suggests the platform may have the potential to regulate target antigen-dependent gene expression to improve the efficacy and safety of first-generation CAR-T cell therapies.
Francesco Marincola, Global Head of Cell Therapy Research at Kite stated: “First-generation autologous CAR T-cell therapies have dramatically changed outcomes for people with certain blood cancers yet more work needs to be done to reach additional patients. As the global CAR T-cell therapy leader, Kite is working on the next generation of cell therapies with the goal to improve upon the great results we have today so more patients can benefit. Through this exclusive license agreement with Refuge’s platform, coupled with our unique in-house research capabilities, we aim to create a new generation of CAR T-cell products to induce long-term remissions for more patients.”
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