Agilis Partners with Japanese Research Institute to Develop Gene Therapy for Angelman, Other Rare Conditions

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Agilis Biotherapeutics is collaborating with the Gene Therapy Research Institution (GTRI) in Japan to develop and manufacture a new adeno-associated virus (AAV) gene therapy for Angelman syndrome (AS) and other rare genetic disorders.

Angelman patients lack the maternal copy of the UBE3A gene. In certain brain areas, only the genetic copy inherited from a person’s mother is active (known as genomic imprinting), so patients are unable to make the UBE3A protein.

Agilis is working on effective viral particles as carriers of the missing UBE3A gene to the brains of patients. Using innovative DNA techniques, their goal is that neuronal cells begin making the UBE3A protein on their own.

Tokyo-based GTRI, with a pipeline of 20 diseases, is also developing gene therapies based on AAV. One of GTRI’s founders, Prof. Shin-ichi Muramatsu, has extensive experience in basic science and clinical research in gene therapy.

In their new joint venture, the two biotechs will focus on producing effective and safe gene therapies. They will develop and manufacture the AAV viral particles according to international standards, including pharmaceutical quality/manufacturing standards (CGMP). This is expected to accelerate the arrival of novel gene therapies to rare disease like Angelman syndrome.

The joint venture will be headquartered in Japan and operate a process development and production facility in Tokyo.

“We are pleased to collaborate with Agilis to leverage each organization’s capabilities and know-how, advance the manufacturing state-of-the art for gene therapy, and develop novel gene therapies,” Katsuhito Asai, CEO of GTRI and a director of the joint venture, said in a press release.

“Our partnership will seek to capitalize on the strong recent progress in the field of gene therapy and expedite the development of innovative gene therapies for patients in need, with a major emphasis on the quality production of safe, effective therapeutics,” Asai said.

Agilis CEO Mark Pykett, also a director of the joint venture, said Agilis Biotherapeutics is “thrilled” to partner with GTRI.

“We believe that our partnership will enhance the efforts of both organizations, build important shared production capabilities, and accelerate development and commercialization of important gene therapies. We look forward to working with GTRI on a range of initiatives,” Pykett said.