Cell therapy weekly: First patient treated in frontotemporal dementia gene therapy trial
This week: The first patient has been dosed in a frontotemporal dementia gene therapy study, Kincell Bio (FL, USA) has acquired Imugene’s (New South Wales, Australia) North Carolina cGMP facility as part of a strategic partnership and Ferring Pharmaceuticals (NJ, USA) has partnered with SK pharmteco (PA, USA) to expand its gene therapy manufacturing capacity.
The news highlights:
- First patient treated in frontotemporal dementia gene therapy trial
- Imugene and Kincell Bio forge strategic partnership
- Ferring Pharmaceuticals partners with SK pharmteco to expand gene therapy manufacturing capacity
First patient treated in frontotemporal dementia gene therapy trial
The first patient has been treated in AviadoBio’s (London, UK) Phase I/II ASPIRE-FTD trial. This dose-escalation study is designed to evaluate the safety and preliminary efficacy of the company’s investigational gene therapy, AVB-101, for the treatment of frontotemporal dementia (FTD) with progranulin (GRN) gene mutations, which results in decreased levels of GRN in the brain.
AVB-101 is delivered to the thalamus using a minimally invasive surgical procedure and provides a functional copy of the GRN gene to restore GRN levels in the frontal and temporal cortex.
“AviadoBio is committed to bringing forward an innovative gene therapy treatment for FTD-GRN and this moment marks an important milestone for the FTD community and our company,” stated AviadoBio’s CEO Lisa Deschamps. “We are thankful for the dedication of the ASPIRE-FTD clinical investigators studying AVB-101 and immensely grateful for the families who participate in clinical trials for new treatment options that may change the future for generations of families living with FTD-GRN.”
Imugene and Kincell Bio forge strategic partnership
Imugene, a clinical-stage immuno-oncology company, and Kincell Bio, a contract development and manufacturing organization, have announced a strategic manufacturing and process development partnership. The partnership includes the sale of Imugene’s cGMP manufacturing facility based in North Carolina for a total consideration of up to US$6 million.
Both companies have also entered into a manufacturing supply agreement wherein Kincell will produce Imugene’s Azer-cel, an investigational anti-CD19 CAR-T cell therapy, to aid in continued clinical trials.
Leslie Chong, Managing Director and Chief Executive Officer of Imugene, commented, “We are delighted to have found a strong partner for the development and manufacturing of our CAR T Azer-cel program. We are confident that this strategic partnership with Kincell will enable Imugene to reach key upcoming data inflection points and extend the company’s cash runway to 2026.”
Ferring Pharmaceuticals partners with SK pharmteco to expand gene therapy manufacturing capacity
SK pharmteco and Ferring Pharmaceuticals have entered a commercial manufacturing partnership to scale up the manufacturing capacity for Ferring’s gene therapy ADSTILADRIN®. After technology transfer, SK pharmteco will be qualified to manufacture, test and release of ADSTILADRIN, subject to FDA approval.
ADSTILADRIN is approved by the FDA for the treatment of adult patients with high-risk Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, with or without papillary tumors. It utilizes an adenovirus vector to deliver the gene encoding interferon alpha-2b to bladder wall cells resulting in the production of high quantities of the interferon alpha-2b protein, a naturally occurring protein that aids in combating cancer.
Bipin Dalmia, Global Head, Uro-Oncology Franchise, Ferring Pharmaceuticals, stated: “Following our January announcement of full availability of ADSTILADRIN across the US and expansion of our clinical trials program, this agreement with SK pharmteco comes alongside major investments in our own manufacturing capabilities to assure the breadth of our long-term supply base. Stable and sustainable supply is a vital part of our mission to fill this unmet clinical need for the patients we serve.”
