Pharmaceutical companies are focusing on novel promising treatment options such as gene therapy to fulfill unmet needs in several rare and complex diseases in recent years. In Japan, among the various companies developing gene therapy programs, Takeda is the leading company with maximum activity in terms of deals with other companies, says GlobalData, a leading data and analytics company.

In Japan, cell therapy and gene therapy are categorized as regenerative medicine products based on the Pharmaceuticals and Medical Devices Law implemented in 2014.

According to GlobalData’s ‘Pharmaceutical Intelligence Center’, there are three gene-modified cell therapies and two gene therapies approved in Japan, whereas 17 gene-modified cell therapies and 13 gene therapies are in clinical pipeline by Japanese companies.

The approved gene-modified cell therapies in Japan include Novartis Kymriah (Mar 2019), Daiichi Sankyo’s Yescarta (Jan 2021), and Celgene’s Breyanzi (Mar 2021). The approved gene therapies include Mitsubishi Tanabe Pharma’s Collategene (Mar 2019) and Novartis / Suzuken Co Ltd’s Zolgensma (Mar 2020).

Venkat Kartheek Vale, Pharma Analyst at GlobalData, comments: “Personalized medicine is gaining more importance nowadays and cell and gene therapies have the potential to deal with unmet needs in several diseases and deliver better treatment outcomes. Takeda has realized the opportunities in this area and is investing in new capabilities and next-generation platforms to build a strong pipeline using its internal network as well as through partnerships and acquisitions.”

Currently, there are 2 Phase III, 7 Phase II, and 4 Phase I clinical trials for gene therapies while there are 11 Phase II and 6 Phase I clinical trials for gene-modified cell therapies in development by Japanese companies. However, majority of the clinical trials are for oncology indications.

Takeda has three gene-modified cell therapies in the pipeline: a Phase I/II product, TAK-007 (CD19 CAR-NK) and two Phase I products, TAK-940 (CD19 1XX CAR-T) for relapsed/refractory B-cell malignancies, and TAK-102 (GPC3 CAR-T) for solid tumors. The pipeline is relatively small considering the competition from other Japanese companies, but Takeda is very active in executing partnerships and acquisitions.

Mr. Vale concludes: “Takeda is gearing up to strengthen its portfolio with gene therapies in its core therapy areas including oncology, rare diseases, neuroscience, and gastroenterology. With a few early-stage products in oncology along with active collaborations and acquisitions in other areas, Takeda is moving fast to transform itself into a future-ready innovative company and derive maximum benefit from its products.”